Lambert-Eaton Myasthenic Syndrome (LES)

lambert eaton myasthenic syndrome "LEMS" 101 words and what they ,,,,

2021-03-17T14:32:00.003-07:00

101 words you will start to hear,

This has been a year of firsts for Lambert-Eaton myasthenic syndrome (LEMS) patients. Two medications approved by the U.S. Food and Drug Administration became commercially available for patients. It is nearly unheard of to have approvals for a single rare disease within months of each other. Patients now have treatment options that are proven to be effective for LEMS.

In June, I was asked to be a columnist for Lambert-Eaton News and “LEMme Tell Ya” was born. I never imagined writing a weekly column about my disease. In addition to my column, Lambert-Eaton News publishes updated information multiple times a week for LEMS patients, their families, and providers. There was little to no information about LEMS when I was diagnosed in 2016, and now there is a weekly newsletter!

As we wrap up 2019, Lambert-Eaton News is taking patient information one step further by launching the Lambert-Eaton News Forums. The soft launch is this week!

Here are the subject areas on the Lambert-Eaton News Forums:

Diagnosed. What now?: This forum is the first place to go after you hear the words, “You have LEMS.” We understand it has been a long journey and being told that you have something that is ultra-rare is scary. Join members to discuss your diagnosis, what it means, and the next steps.
Autoimmune LEMS: For those of you who do not have cancer as the underlying cause of your LEMS, this is the forum for you. Many of us have multiple autoimmune diseases as well as LEMS. How do we juggle it all? Does diet help? What do we do next?
The Big C: In about 50 percent of LEMS cases, cancer is the underlying trigger. This forum is for those of you who have had cancer as the cause of your LEMS. If you are going through cancer treatment or have successfully completed it, this little corner of the web is yours.
Firdapse: Firdapse (amifampridine) was the first LEMS-specific treatmentapproved by the FDA in the United States. Join this forum to discuss the ins and outs of treatment and patient support programs through Catalyst Pathways. This forum is for discussions specific to Firdapse.
Ruzurgi: Formerly known as 3,4-DAP, and given on a compassionate basis to approximately 200 patients in the United States for 30 years, Ruzurgi(amifampridine) is now approved by the FDA for pediatric LEMS patients. This forum is specifically for Ruzurgi discussion.
IVIG and plasmapheresis: Immunomodulation is an option that many LEMS patients choose over immunosuppression. This forum is the space for patients to share experiences with intravenous immunoglobulin and plasmapheresis therapies and how they have managed the treatment schedules.
Immunosuppression: There are various forms of immunosuppression that many LEMS patients are on to stop the antibody attack on the calcium channels. This is your forum to share your experience with immunosuppression and how it has benefited your LEMS journey.
Family forum: LEMS affects the patient’s family as well as the patient. Consider this your space to network, share experiences, and support being a source of strength for your loved one with LEMS.
In the news: Our little-known rare disease has been in the news a lot in the past year. This forum is the space to share articles and stimulating discussion for those patients who want to delve into such topics.

I will moderate the forums with another LEMS patient who will start in January. We look forward to sharing conversation and support for all LEMS patients. Our soft launch this week is just prior to BioNews Services closing for the holidays. When BioNews reopens Jan. 2, we will be ready to engage in a type of patient conversation that hasn’t been available elsewhere.

We hope you will join us in the Lambert-Eaton News Forums.

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How-much-will-a-pack-of-joints-cost-the-future-of-mass-market-marijuana?

2021-03-04T14:17:00.001-08:00

How-much-will-a-pack-of-joints-cost-the-future-of-mass-market-marijuana?

Marijuana is for sale in Colorado and Washington, so it's seemingly only a matter of time before packs of joints are available as well — but how much will a pack run you? After doing some extensive research, Fast Company's Thor Benson came up with around $50.

He reached that number by comparing the estimated cost of marijuana production (about $2 to $3 per gram) with common industry margins (rumored to be between 40 percent and 50 percent) and factoring in the economies of scale wielded by large companies like Marlboro. At the very most, an average pack of joints might run you about $120 — the amount Denver-based store Medicine Man asks for a 20-pack of pre-rolled, one-gram joints.

Benson even sought input from Jeff Caldwell, a representative from Marlboro's parent company, but Caldwell wouldn't speculate on the potential costs of joint packs. Caldwell said that his company had "no plans to sell marijuana-based products," but Benson isn't so sure. "Marlboro is in the business of making money," he writes, and considering estimates that marijuana sales could drive $20 billion in taxes alone, Marlboro could find the opportunity too good to pass up. Another option for Marlboro would be selling less pricey "spliffs" — 50 percent marijuana, 50 percent tobacco joints — which Benson estimates could cost just $20 per pack.

Marijuana joints should cost about ten times as much as cigarettes, Benson claims, but does that mean they deliver ten times the pleasure?

On COVID-19: And LEMS Cancer patients and caregivers share their current life realities.

2021-03-04T14:03:00.003-08:00

On COVID-19: And LEMS Cancer patients and caregivers share their current life realities.

“I feel overwhelmed with uncertainty, fear, shock, worry, anxiety, sadness, and grief – similar to how I felt when I found out about my daughter’s cancer diagnosis.”

This honest response to the influx of news and conversations surrounding the coronavirus outbreak comes from Joan, whose daughter Sarah is currently in maintenance care for optical glioma bilateral. What’s more, Joan is temporarily out of work because of the ongoing crisis, yet another similarity to when she left her job to be by Sarah’s side during treatment.

From emotional distress to temporary losses in income, the parallels between living with cancer and navigating this global pandemic are undeniable. COVID-19 is also taking an especially harsh toll on those who are already vulnerable, and we recognize that the financial barriers and health concerns of cancer are only compounded by the spread of the virus.

Cancer won’t press pause because of COVID-19, and we have no intention of stopping either. We’re sticking to what’s always been our number one priority: helping patients get to the other side of cancer. In the spirit of meeting families where they’re at, we created the Family Reach COVID-19 Emergency Fund to stabilize cancer patients and their families during this time of crisis.

To better understand what this means for cancer patients and their families, we reached out to the experts themselves. Now more than ever, their voices will be our guiding light for offering meaningful support and solutions.

Going about business as usual

When you have a weakened immune system during cancer treatment, diligent hand washing and heightened concern for spreading germs becomes second nature.

“To be honest, it’s business as usual right now in our house,” said Nichole, mom of cancer-hero Ruby. “Ever since Ruby’s diagnosis, we have a strict policy of no shoes in the house and washing hands as soon as you come home. We are sticking to that, as well as staying home often, not taking the kids to crowded places, and making sure backpacks, books, and most toys are wiped clean with an antibacterial wipe.”

It’s clear that families who are currently facing cancer, and those who have previously experienced a diagnosis, are well-accustomed to the panic that many are feeling in response to the coronavirus pandemic. However, COVID-19 is also quickly presenting additional challenges for cancer patients and their families.

Facing more unimaginable decisions

After catching a fever, coughing, and showing low blood cell count last week, five-year-old Harper was admitted to the hospital. While a normal course of action for a pediatric leukemia patient, this time around is slightly different because of COVID-19. Worried Harper could now be under the same roof as coronavirus only adds to her parents’ concerns, and they must also follow the precautions initiated by the hospital.

“We are limited to only one visitor, which my husband and I decided would be me,” said Harper’s mom, Kim. “We don’t want Jeff to also put himself at risk by going to the hospital to visit her, as we need one parent to stay safe and care for our son Harry at home.”

Amy, on the other hand, has been facing cancer for over four years and is a single mother of two young children. As the virus continues to consume the world, she has to find the strength and resources to adjust to the current state of affairs.

“It is taxing because I have a strict budget due to my cancer,” she said. “I have to purchase certain food, extra cleaning supplies, and my limited income does not allow for any extras, like utilities or more groceries because my kids are now home from school.”

On top of these daily challenges, cancer patients like Amy don’t have the same ability to practice the social distancing and other cautionary measures advised by local and federal governments.

“What am I supposed to do when I need chemotherapy every other week?” Amy countered.

Like Amy, other families shared that traveling for treatment only exacerbates their growing fears about the virus.

“It’s scary for everyone, but even more so for immunocompromised cancer kids,” said Colleen, whose son Johnny is currently in treatment for neuroblastoma. “We are due to travel to and from New York for treatment later this month and it’s very worrisome.”

Continuing to support families facing cancer

Never failing to impress us with their resolve and compassion, the messages we received from our families expressed heartfelt concern – not only for themselves, but for everyone around them.

Studies have shown that certain patient characteristics have been linked to higher rates of fear of cancer recurrence. However, there are steps that individuals can take to mitigate these feelings of anxiety in cancer survivorship, according to recent research.

“One of the most common concerns of cancer survivors (after) cancer treatment is the fear of cancer coming back – what we refer to as fear of cancer recurrence,” says J , of the Queen’s University School of Nursing in Canada.

Galacia, of the Queen’s University School of Nursing, worked as on oncology nurse before pursuing her doctorate. After seeing firsthand the stress that survivors face regarding their cancer coming back, she decided to conduct research to determine if certain characteristics were associated with fear of recurrence.

“We used a theory, Leventhal’s Common Sense Model, which is commonly used to understand cancer recurrence. We used this to identify what could be important characteristics to look at amongst cancer survivors,” she said.

Characteristics Associated With Fear of Recurrence

The findings ultimately showed that younger people had higher rates of fear of recurrence compared to their older counterparts, and females experienced it more than males.

Other findings were interesting – if not completely counter-intuitive. People who were more pessimistic tended to have lower rates of fear of recurrence. “This might be their type of coping… they’re maybe more pessimistic or more likely to deny or disengage and give into their circumstances,” Galacia says.

Additionally, as more time elapsed since the survivor’s diagnosis and treatment, fear of recurrence rates tended to go up, which could be a result of constant reminders of cancer as survivors continue to visit the clinic for follow-ups and screenings.

Finally, individuals who had symptoms that they perceived to be cancer-related and individuals with low self-esteem both had higher rates of fear of recurrence, Galacia found.

How Survivors Can Cope

Luckily, there are steps that survivors can take to decrease their feelings of fear of recurrence, which Galacia says can actually promote better behavior.

“Low levels of fear of recurrence can actually be motivating,” she says. “It can motivate us to make healthy behavioral changes, like (quitting) smoking, for instance.”

However, fear of recurrence can also have the opposite effect, where survivors do not go for their scheduled follow-up appointments or cancer screening, as that can trigger all the emotions from cancer.

If survivors feel that they have a debilitating level of fear – or if it frequently interferes with their daily living – they should seek out professional help, such as that from a cognitive behavioral therapist, Galacia says.

Survivors can also employ other methods to help curb their negative feelings, such as gratitude journaling, and talking to supportive people – especially other survivors who are feeling the same way. Doing so can help people realize that fear of recurrence is a common and normal emotion.

“Sometimes those thoughts will creep in – we’re human – but (it’s important to) reframe and focus on what’s true,” Galacia and Mr J Kersnowski says.

LEMS Patients with Cancer, and Survivors, Can Manage Stress panic attacks Through COVID-19 Uncertainty...

2021-03-04T14:00:00.000-08:00

How Patients with Cancer, and Survivors, Can Manage Stress Through COVID-19 Uncertainty.

The recent and rapidly evolving developments surrounding the novel coronavirus — also known as COVID-19 – has led to significant uncertainties around the world. Those unknowns have led to panic and have subsequently increased the anxiety and stress some may be feeling, particularly those who have survived cancer or are currently in treatment.

“Fear is a natural human emotion. We all have fear during this pandemic, yet the higher risk for health complications from COVID-19 may heighten the stress faced by individuals with cancer. We all need to focus on actions we can take to reduce our risk of contracting the virus, such as social distancing. We can also take practical steps to cope adaptively with chronic stress because the fear is probably not going away anytime soon,” Dr. Shelley Johns, a researcher with the Indiana University Melvin and Bren Simon Comprehensive Cancer Center, said in an interview with CURE®.

“Fear doesn’t have to blind us from taking actions that protect ourselves and our communities and connect us with our deeply held values.”

We spoke with Johns, a board-certified clinical health psychologist, about how people can recognize when they are experiencing anxiety, and what they can do to limit their exposure to stress related to the COVID-19 pandemic.

CURE: As concerns are heightened during times like these, how can people sense they are truly suffering from stress and anxiety?

Johns: Stress usually shows up in four aspects of our lives: our emotions, our bodies, our behaviors and our thoughts.

Some of the most common emotions right now with everything that is going on are fear, worry and sadness as well as feelings of confusion, frustration or anger. Some people may be experiencing loneliness due to social distancing. We can be observant of our emotions, accept them with grace and choose how we will use our emotions to our advantage. For example, fear of COVID-19 can motivate us to stay home to reduce our risk of contracting the virus. Loneliness can motivate us to call a friend or explore video chatting with family who live at a distance.

In terms of the effects of stress on our bodies, we may notice muscle tension, headaches, or more fatigue than usual. We may notice our heart racing or chest pain when we’re watching the news, or maybe getting a little short of breath and feeling unsettled or restless.

Some of the behavioral signs would be things like sleep disturbance, being irritable with others, appetite changes or exhibiting more nervous behaviors, such as nail biting, fidgeting, or pacing.

Stressful thoughts might include ‘the world is never going to be the same again,’ yet a more adaptive thought might be ‘this is an extremely challenging time with a great deal of uncertainty and adjustment, yet I can be resilient and take wise action in the face of COVID-19.’

How does stress and anxiety affect the health of a cancer survivor or someone recently diagnosed with cancer?

The most resilient people on Earth are people with cancer, because they are masters at adjusting to unwanted realities and uncertainty from the moment of diagnosis. People with cancer have looked death in the eye and are still finding their way.

One of my greatest concerns right now is for people with cancer whose immune systems may already be compromised. Chronic stress — which we’re all experiencing now – can have a negative effect on the immune system, which may already be depleted in individuals with cancer. I think it is essential for people who are immunocompromised, such as those with cancer, to be proactive to maximize their physical and emotional health during this COVID-19 threat. Social distancing, frequent and proper handwashing, keeping surfaces clean and disinfected, and calling your oncologist or primary care physician if you have a fever or cough are key strategies for physical health. For emotional health, I recommend a variety of research-supported stress management skills.

What kind of skills would you recommend?

One thing we can all do is decide what kind of person we want to be through this experience. In other words, what deeply held values do you want to embody during the COVD-19 crisis?

For instance, do you want to be compassionate and generous toward others? Do you want to deepen your faith or your connection with friends you’ve not spoken with recently? Do you want to be known as someone who maintains a sense of humor during stress? Letting our values guide our actions during uncertain times can be like a compass guiding our way through uncertain times.

Another suggestion is to find a practice that can help ground you. This could be deep breathing, prayer, or mindfulness meditation. Mindfulness is about being fully “awake” to this present moment rather than getting caught up in thoughts about the future or the past. Physical activity is also a healthy stress buster, and this can be as simple as taking a brisk walk while maintaining social distance or trying a few yoga stretches. Doing something to help others is also important. Everyone knows someone who lives alone, so keeping in close touch with that person by phone could give meaning to this experience for both of you. I also suggest keeping a gratitude journal. Making a list of three specific things you are grateful for each day can be a pleasant reminder that life is about more than cancer and COVID-19.

Would you recommend patients stick to a routine to help them with any stress and anxiety?

Absolutely! Maintaining a healthy routine and schedule for our day is one way we can exert some control over our lives. For example, going to bed and getting up at the same time each day and limiting nap time can help maintain energy and normalcy. Eating healthy, regular meals and limiting alcohol consumption is also key. Scheduling time for physical activity each day — to the extent possible for each person – is another great way to build endurance during COVID-19 stress. Other self-care strategies like meditation, prayer, and listening to mood-enhancing music could also be on the daily schedule.

What are some things that you would recommend people do to distract themselves from what’s going on?

I don’t think of it as a distraction, as much as choosing purposeful activities grounded in our values. Distraction is about turning away from what’s here, as where values-based action is more about turning toward what matters to us, which is much more meaningful and vitality-enhancing. For instance, you can text your family members, organize family photos, or learn something new online. Instead of feeling stuck at home, one of my patients has decided to take this opportunity to learn to speak Spanish using an online training program. Having purposeful activities scheduled into each day can help us maintain our sense of normalcy during this highly unusual time.

Is there anything that you would recommend against to help individuals avoid unnecessary stress and anxiety?

One thing I don’t find nurturing right now is too much news and social media. We all need to stay informed, yet too much media consumption can heighten our distress rather than reduce it. I have been limiting my news consumption to one hour per day, because research shows that during past public health crises, the more social media and news people consumed, the more anxious they felt. Consider replacing some of your news consumption with a funny movie or an hour with your favorite comedian instead.

2021 Stuck at home sick? marijuana seeds online.

2021-01-11T17:55:00.009-08:00

CBD Cannabis CBD SEED 101

Marijuana seeds are considered a cannabis product just like flower, edibles, and concentrates. Their legality depends on which state you live in. People living in states with adult-use legalization can buy, produce, and sell seeds within their own state, but seeds can’t cross state lines. People living in states with medical marijuana legalization can only buy seeds if they have a medical card.

Seed banks exist outside of the US and can sell them for “souvenir purposes,” but it is illegal to bring seeds into the US and Customs will seize any cannabis seeds they find in packages or on a person

Where can I buy cannabis seeds?

Many world-renowned seed banks are overseas in the Netherlands, the UK, Spain, and other countries where cannabis laws are less restricted. Seed banks provide seeds from a variety of different breeders.

In states with adult-use legalization or a medical marijuana program, you can buy seeds within your own state, either at a dispensary or through a specific seed company’s website.

Researching cannabis seeds online

Before you purchase seeds online, you’ll need to figure out what strain you want to grow and what breeder you want to buy from.

Because US federal law still prohibits cannabis, it can be hard to find information on seed banks and breeders. Breeders who have a long history and positive reputation are usually a good place to start. To get an idea of what well-established breeders look like, check out

You can also do some research and find an online grow journal that details the whole growing process of a specific strain from a particular breeder. Through these, you’ll be able to look over another grower’s specific notes and see pictures of the final results.

If you grow some seeds and like the results, try growing another strain from that same breeder and see how it goes

SEEDS

Regular seeds

If you buy a packet of regular seeds, they’ll come with a mix of males and females. A lot of cultivators prefer to grow these because they haven’t been backcrossed—essentially inbred—as much as feminized or autoflower seeds. You’ll need to sex out the seeds once their reproductive organs show during the flowering phase and discard the males—because they don’t produce buds and will pollenate females, resulting in seeded flowers.

Feminized seeds

Seeds can come feminized, meaning you can just put them in soil and start growing for buds. These seeds are guaranteed to be bud-producing females and growing them cuts out the step of having to sex out plants and discard the males.

It also reduces the risk of having a stray male sneak into your crop—just one male can pollinate a huge crop, causing your females to focus their energies on producing seeds instead of buds.

Autoflower seeds

Autoflower plants change from the vegetative to flowering state with age, not the changing of their light cycle. They have a short grow-to-harvest time and can be ready to harvest in as little as 2 ½ to 3 months from when you put the seeds in the ground. The downside is that, typically, they are less potent, but autoflower seeds are great for people who want to grow cannabis but don’t want to spend a lot of time doing it.

How much do marijuana seeds cost?

Cannabis seeds usually come in a pack of 10 or 12 seeds and start at around $40 a pack and go up from there. Some high-end genetics can run between $200 to $500 a pack. 80% OFF

Feminized and autoflower seeds will cost more because more breeding work was put in to creating them and they take less time for the grower to get buds

When you grow any amount of seeds, a percentage of them won’t germinate, even if you get them from a reputable breeder. Always count on a few not germinating or dying off, or roughly 1/4 of the total you put in the ground.

When growing regular seeds, some won’t germinate and some will have to be discarded because they’ll turn out to be males. With feminized seeds, some won’t germinate, but a higher percentage of them will turn into flowering plants because there won’t be any males.

If you want six total cannabis plants to harvest for buds and are growing from regular seeds, start with about 4 times as many, or 24 seeds. Some won’t germinate and some will turn out to be males, and then you’ll want to discard down to the six best phenotypes. If growing feminized seeds, you can probably start with about twice as many seeds in this case (about 12); a couple won’t germinate, and then discard down to the six best phenotypes.

Make sure to always stay within your state’s legal limit of growing plants.

How do I buy strain-specific cannabis seeds?

Blue Dream, Gelato, and Original Glue have gained in popularity in recent years. To try your hand at growing them, check out these resources:

HERE IS THE PLACE!

Tips for Dealing With Pain When You Have LEMS CBD Cannabis

2021-01-11T17:49:00.001-08:00

Tips for Dealing With Pain When You Have LEMS

What causes pain in LEMS?

In LEMS, the immune system mistakenly attacks nerve cell endings where they meet muscle cells. This impedes the transmission of electrical impulses from the nerve cells to the muscle cells. Over time, they waste away due to inactivity, causing pain.

Where does it hurt?

Arm and leg movements are affected in LEMS. People with the disorder may find it difficult to walk, get up from a chair, climb steps, or exit a car.

Although the levels of severity vary, LEMS patients commonly experience pain in their lower limbs, including the thighs, and in the lower back. Your muscles may ache and become tender. In some cases, simple exertion can cause this.

Pain treatments

Doctors usually address pain and improper gait — a person’s manner of walking — in LEMS after disease treatment starts. Therapies such as intravenous immunoglobulin can help block the immune system from attacking the nerve cells, which may help relieve pain.

Cannabidiol or CBD,LEMS CBD Cannabis which is derived directly from the hemp plant and can be used in many forms, also is thought to help with LEMS symptoms, including pain and fatigue. In addition to reducing inflammation, CBD may promote better sleep quality. This may be helpful for some patients, as people with chronic pain often experience sleep disruption. Speak with your physician about whether the treatment is right for you. works!!

While more research is needed, physiotherapy may help strengthen muscles and improve patients’ range of motion. That, in turn, could help reduce pain. Discuss your exercise goals with a physiotherapist who can help you design a safe exercise routine.

In addition, over-the-counter medications such as acetaminophen or nonsteroidal anti-inflammatory drugs (NSAIDs) may help, sometimes in conjunction with prescribed treatments. NSAIDs include aspirin, ibuprofen, and naproxen sodium, which is known by the brand name Aleve. Discuss these options with your physician.

Meditation also has been shown to reduce the intensity of pain. One thought is that the practice decreases stress, which in turn may lessen pain.

Support

An effective support network can help you as you deal with pain. Not only can members encourage and motivate each other, but they also can share ideas about what worked, and what didn’t, for them.

Last updated: June 7 2020

***

Lambert-Eaton News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

LEMS CBD Cannabis

Cancer

2018-07-15T20:17:00.001-07:00

https://www.google.com/url?rct=j&sa=t&url=https://hillaryhq.com/2018/07/14/sandhill-capital-partners-has-decreased-eaton-plc-com-etn-position-by-6-84-million-dollar-general-dgs-sentiment-is-1-07/&ct=ga&cd=CAEYACoTNTQyMTIyNDcxNjY1MzA3MjMzMTIaNjUzNmMwZDcxNjk0OWJjYzpjb206ZW46VVM&usg=AFQjCNFAPV6SMEcORYOc_faj0MAaKUvO7A

2015-02-05T20:30:00.001-08:00

http://norml.org/news/2015/02/05/portland-norml-responds-to-olcc-s-first-meeting-on-implementation-of-measure-91-marijuana-legalization

Press release info feb

2015-02-03T07:28:00.001-08:00

S&P 500 2,037 +15.99 +0.79% PRESS RELEASE Catalyst Pharmaceuticals Announces Encouraging Pre-NDA Meeting With the FDA for Firdapse(TM) as a Treatment for Lambert-Eaton Myasthenic Syndrome (LEMS) By Published: Feb 2, 2015 8:03 a.m. ET SHARE CORAL GABLES, Fla., Feb 02, 2015 (GLOBE NEWSWIRE via COMTEX) -- Catalyst Pharmaceutical Partners, Inc. CPRX, +0.00% (Catalyst Pharmaceuticals), a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating diseases, today announced it has held a productive pre-New Drug Application (NDA) meeting with the U.S. Food and Drug Administration (FDA) regarding Firdapse™ for the treatment of LEMS. Based on this meeting, Catalyst believes that its Phase 3 clinical program will provide acceptable support for submission of an NDA for Firdapse™ for LEMS. The Company plans to complete a full NDA submission during the 3rd quarter of 2015. Catalyst will confirm the overall regulatory path forward upon receipt of formal meeting minutes from the FDA in the coming weeks and will provide a further update at that time

New test news oct 8.

2014-10-08T10:22:00.001-07:00

http://m.bizjournals.com/southflorida/news/2014/10/06/catalyst-pharma-to-hire-25-people-after-successful.html?utm_source=feedburner&utm_medium=feed&utm_campaign=Feed%3A+industry_6+%28Industry+Health+Care%29&r=full

News oct 8tt

2014-10-08T10:20:00.001-07:00

http://money.cnn.com/news/newsfeeds/articles/globenewswire/10101677.htm

2014-10-01T12:21:00.003-07:00

http://mobile.reuters.com/article/idUSL3N0RU5QS20140929?irpc=932

2014-10-01T12:21:00.001-07:00

http://www.bidnessetc.com/26483-catalyst-surges-on-positive-trial-results/

2014-10-01T12:20:00.001-07:00

http://globenewswire.com/news-release/2014/09/29/669185/10100382/en/Catalyst-Pharmaceuticals-Announces-Positive-Top-Line-Phase-3-Data-From-Pivotal-Firdapse-Clinical-Trial-in-Patients-With-Lambert-Eaton-Myasthenic-Syndrome-LEMS.html

Investigational Drug, Firdapse, Available at No Cost to U.S. Patients with LEMS

2014-05-23T07:11:00.000-07:00

Investigational Drug, Firdapse, Available at No Cost to U.S. Patients with LEMS

Coral Gables drug company to make new medicine available at no cost

April 23, 2014
Evan S. Benn

Coral Gables-based Catalyst Pharmaceutical Partners soon will make its investigational drug Firdapse available at no cost to patients with a certain autoimmune disorder.

The drug, which is supposed to relieve muscle weakness in people with Lambert-Eaton Myasthenic Syndrome, is entering the final phase of its clinical trials. Catalyst said it expects to submit an application for Firdapse’s approval with the U.S. Food and Drug Administration next year.

Catalyst has been leading the development of Firdapse in the United States since 2012; the drug already is government-approved and commercially available in Europe.

During the U.S. approval process, Catalyst said it will establish an expanded-access program to make Firdapse available for free to patients with LEMS.

“As part of our ongoing commitment to the LEMS community, we are making Firdapse available to those who would like access to this investigational drug but are unable to participate in clinical studies or travel to sites where it is available,” Catalyst CEO Patrick J. McEnany said in a statement.

Catalyst steps up to provide LEMS patients expanded Firdapse access
April 23, 2014
Donna Young

After recognizing some patients with a rare autoimmune neuromuscular disease, known as Lambert-Eaton myasthenic syndrome (LEMS), were unable to access treatment for their disease, Florida drug maker Catalyst Pharmaceutical Partners stepped up and initiated the process to establish an expanded access program (EAP) for its experimental potassium channel inhibitor Firdapse (amifampridine phosphate).

Catalyst to include safety data from new 3,4DAP expanded access program in LEMS as part of NDA filing executivesApril 23, 2014
Jennifer Smith-Parker

Catalyst Pharmaceutical Partners (NASDAQ:CPRX) plans to collect safety data from a new expanded access program for its Lambert-Eaton Myasthenic Syndrome (LEMS) candidate and add it to its NDA filing, said COO and CSO Steve Miller and Doug Winship, vice president of regulatory operations. The program can meet the needs of at least 500 people, said CEO and cofounder Patrick McEnany.

Tags: Featured Coverage, Health Communications, Biotech, corporate communications, Life Science Communications, pharmaceutical public relations, Brand and Product Communications, health sciences

www.netag.nhs.uk/files/appraisal-reports/Firdapse - NETAG appraisal report - Nov 2011 -WEB VERSION.pdf

2014-05-23T07:08:00.001-07:00

www.netag.nhs.uk/files/appraisal-reports/Firdapse - NETAG appraisal report - Nov 2011 -WEB VERSION.pdf

Firdapse

2014-05-23T07:07:00.000-07:00

Firdapse® (currently approved in the EU) is the first and only approved drug for the symptomatic treatment of Lambert-Eaton Myasthenic Syndrome in adults, a rare autoimmune disease with the primary symptoms of muscle weakness.

Firdapse at a Glance

Approved in the European Union
Manufactured and commercialized by BioMarin in the European Union
Designated as an orphan drug in the United States, the European Union and and Switzerland

Regulatory Status

In December 2009, Firdapse received marketing approval from the European Commission making it the first approved therapy for the symptomatic treatment of LEMS. Firdapse has been designated an orphan drug in the EU, which grants market exclusivity for ten years.

Commercialization Plan

Firdapse is commercialized by BioMarin in the European Union.

In October 2012, BioMarin out-licensed North American rights of Firdapse to Catalyst Pharmaceutical Partners. For more information on Firdapse development plans in the U.S., please refer to the Catalyst website.

Catalyst Pharmaceutical Partners Receives Breakthrough Therapy Designation From FDA for Firdapse(TM) for the Treatment of LEMS -

2014-05-23T07:04:00.002-07:00

Catalyst Pharmaceutical Partners Receives Breakthrough Therapy Designation From FDA for Firdapse(TM) for the Treatment of LEMS -

CORAL GABLES, Fla., Aug. 27, 2013 (GLOBE NEWSWIRE) -- Catalyst Pharmaceutical Partners, Inc. (Nasdaq:CPRX), a specialty pharmaceutical company focused on the development and commercialization of novel prescription drugs targeting rare (orphan) neuromuscular and neurological diseases, today announced that its investigational product Firdapse™ (amifampridine phosphate) has received "Breakthrough Therapy Designation" by the U.S. Food and Drug Administration (FDA) for the symptomatic treatment of patients with Lambert-Eaton Myasthenic Syndrome (LEMS). Firdapse™ is Catalyst's investigational therapy that is being evaluated for the treatment of the debilitating symptoms associated with LEMS, including muscle weakness.

"We are very pleased to have received Breakthrough Therapy Designation for Firdapse™ and we are excited by the FDA's decision to place our product in a category that may enable expedited development and review for patients with LEMS," said Patrick McEnany, President and Chief Executive Officer of Catalyst. "With no approved or effective symptomatic treatment currently available for LEMS, Firdapse™ has the potential to be the first-line treatment option for patients with this rare condition."

Breakthrough Therapy Designation for Firdapse™ was based on clinical data from several previously published clinical trials of amifampridine (3,4-DAP) in patients with LEMS. Firdapse™ has the potential to provide significant relief of the often debilitating symptoms of the disease, including muscle weakness (e.g. difficulty walking), difficulty swallowing and talking, drooping of eyelids and facial weakness.

About Breakthrough Therapy Designation

Breakthrough Therapy Designation was enacted as part of the 2012 Food and Drug Administration Safety and Innovation Act (FDASIA). FDASIA defines breakthrough therapy as a drug that is "intended, alone or in combination with one or more other drugs, to treat a serious or life-threatening disease or condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development."

A breakthrough therapy designation conveys all of the fast track program features, as well as more intensive FDA guidance on an efficient drug development program. The FDA also has an organizational commitment to involve senior management in such guidance. The Breakthrough Therapy Designation is a distinct status from both accelerated approval and priority review, which can also be granted to the same drug if relevant criteria are met.

About Firdapse™

Firdapse™, also known as amifampridine phosphate, 3,4-diaminopyridine phosphate or 3,4-DAP phosphate, is a potassium channel blocker. It delays repolarization of the pre-synaptic neuron, causing voltage gated Ca2+ channels to remain open longer. The increase Ca2+ influx causes more acetylcholine to be released, making it more likely that a muscle action potential will be initiated, thereby reducing muscle weakness. The North American rights to Firdapse™ were licensed to the Company in 2012 by BioMarin Pharmaceutical. BioMarin currently markets Firdapse™ in the EU for the treatment of LEMS.

In the United States, where the product has previously received orphan drug designation, Firdapse™ is in a Phase III, multicenter, double-blind, placebo-controlled, randomized discontinuation study followed by an open-label extension period to evaluate the efficacy and safety of Firdapse™ in patients with LEMS. In addition to LEMS, other potential orphan neuromuscular indications for Firdapse™ include Myasthenia Gravis and Congenital Myasthenic Syndrome, among others.

About LEMS

Lambert-Eaton Myasthenic Syndrome, LEMS, is a rare autoimmune disease that can be severely disabling, with the primary symptom of muscle weakness. The weakness is generally more marked in the proximal muscles, particularly of the legs and trunk. Other problems include reduced reflexes, drooping of the eyelids, facial weakness and problems with swallowing. Patients often report dry mouth, impotence, constipation and feelings of light headedness on standing. These problems can be life threatening when the weakness involves respiratory muscles. The muscle weakness in LEMS is caused by autoantibodies to voltage gated calcium channels, which cause a reduction in the amount of acetylcholine released from nerve terminals. The prevalence of LEMS is estimated at approximately 3,000 patients in the United States and Canada. Approximately 50 percent of LEMS patients diagnosed have small cell lung cancer. Patients with LEMS typically present with fatigue, muscle pain and stiffness. A diagnosis of LEMS is generally made on the basis of clinical symptoms, electromyographic and compound muscle action potential (CMAP) testing and where available, the presence of autoantibodies against voltage gated calcium channel.

About Catalyst Pharmaceutical Partners

Catalyst Pharmaceutical Partners, Inc. is a specialty pharmaceutical company focused on the development and commercialization of novel prescription drugs targeting rare (orphan) neuromuscular and neurological diseases, including Lambert-Eaton Myasthenic Syndrome (LEMS), infantile spasms, and Tourette Syndrome. Catalyst's lead candidate, Firdapse™ for the treatment of LEMS, is currently undergoing testing in a global, multi-center, pivotal phase III trial. Catalyst is also developing a potentially safer and more potent vigabatrin analog (designated CPP-115) to treat infantile spasms, and epilepsy, as well as other neurological conditions associated with reduced GABAergic signaling, like post-traumatic stress disorder and Tourette Syndrome.

Forward-Looking Statements

This press release contains forward-looking statements. Forward-looking statements involve known and unknown risks and uncertainties, which may cause Catalyst's actual results in future periods to differ materially from forecasted results. A number of factors, including the timing of completion of Catalyst's currently ongoing Phase III trial of Firdapse™, whether the Phase III trial will be successful, whether the receipt of breakthrough therapy designation for Firdapse™ will expedite the development and review of Firdapse™ by the FDA or the likelihood that the product will be found to be safe and effective, whether an NDA for Firdapse™ will ever be accepted for filing by the FDA, the timing of any such NDA filing or acceptance, whether any of Catalyst's product candidates will ever be approved for commercialization or successfully commercialized, and those other factors described in Catalyst's Annual Report on Form 10-K for the fiscal year 2012 and other filings with the U.S. Securities and Exchange Commission (SEC), could adversely affect Catalyst. Copies of Catalyst's filings with the SEC are available from the SEC, may be found on Catalyst's website or may be obtained upon request from Catalyst. Catalyst does not undertake any obligation to update the information contained herein, which speaks only as of this date.

For Further Information Contact:
Patrick J. McEnany
Catalyst Pharmaceutical Partners
Chief Executive Officer
(305) 529-2522


Melody Carey
Rx Communications Group
Co-President
(917) 322-2571

- See more at: http://globenewswire.com/news-release/2013/08/27/569448/10046173/en/Catalyst-Pharmaceutical-Partners-Receives-Breakthrough-Therapy-Designation-From-FDA-for-Firdapse-TM-for-the-Treatment-of-LEMS.html#sthash.n7kWvT6Q.dpuf

wikipedia.Lambert-Eaton_myasthenic_syndrome

2014-05-23T06:58:00.004-07:00

Lambert–Eaton myasthenic syndrome

From Wikipedia, the free encyclopedia

(Redirected from Lambert-Eaton myasthenic syndrome)

Lambert–Eaton myasthenic syndrome
Classification and external resources
Neuromuscular junction. Lambert–Eaton myasthenic syndrome is caused by autoantibodies to the presynaptic membrane. Myasthenia gravis is caused by autoantibodies to the postsynaptic acetylcholine receptors. 1. Presynaptic terminal 2. Sarcolemma 3. Synaptic vesicle 4. Nicotinic acetylcholine receptor 5. Mitochondrion
ICD-10	G 73.1
ICD-9	358.1
DiseasesDB	4030
MedlinePlus	000710
MeSH	D015624

Lambert–Eaton myasthenic syndrome (LEMS, also Lambert–Eaton syndrome, or Eaton–Lambert syndrome) is a rare autoimmune disorder that is characterized by muscle weakness of the limbs. It is the result of an autoimmune reaction in which antibodies are formed against presynaptic voltage-gated calcium channels, and likely other nerve terminal proteins, in the neuromuscular junction (the connection between nerves and the muscle that they supply).^[1] The prevalence is 3.4 cases per million.^[2] Around 60% of those with LEMS have an underlying malignancy, most commonly small cell lung cancer; it is therefore regarded as a paraneoplastic syndrome (a condition that arises as a result of cancer elsewhere in the body).^[3]

People who develop LEMS are usually over 40, although it may occur at any age. The diagnosis is usually confirmed with electromyography and blood tests; these also distinguish it from myasthenia gravis (MG), a related autoimmune neuromuscular disease.^[1]

If the disease is associated with cancer, direct treatment of the cancer often relieves the symptoms of LEMS. Other treatments often used are steroids, azathioprine andintravenous immunoglobulin, which suppress the immune system, and pyridostigmine and 3,4-diaminopyridine, which enhance the neuromuscular transmission. Occasionally, plasma exchange is required to remove the antibodies.^[1]

Signs and symptoms[edit]

The weakness of LEMS typically involves the legs and arms. Leg involvement is more striking than in myasthenia gravis. The proximal muscles (those closest to the trunk) are predominantly affected; this leads to difficulties climbing stairs and rising from a sitting position. Physical exercise and high temperatures can worsen the symptoms. Weakness of the bulbar muscles (supplying the mouth and throat) is occasionally encountered.^[1] Weakness of the eye muscles is uncommon. Some may have double vision, drooping of the eyelids and difficulty swallowing,^[1] but generally only together with leg weakness; this distinguishes LEMS from myasthenia gravis, in which eye signs are much more prominent.^[3] In the advanced stages of the disease, weakness of the respiratory muscles may occur.^[1] Some may also experience problems withcoordination (ataxia).^[4]

Three quarters of people with LEMS also have disruption of the autonomic nervous system. This may be experienced as a dry mouth, constipation, blurred vision, impaired sweating, and orthostatic hypotension (falls in blood pressure on standing, potentially leading to blackouts). Some report a metallic taste in the mouth.^[1]

On neurological examination, the weakness demonstrated with normal testing of power is often less severe than would be expected on the basis of the symptoms. Strength improves further with repeated testing, e.g. improvement of power on repeated hand grip (a phenomenon known as "Lambert's sign"). At rest, reflexes are typically reduced; with muscle use, reflex strength increases. This is a characteristic feature of LEMS. The pupillary light reflex may be sluggish.^[1]

In LEMS associated with lung cancer, most have no suggestive symptoms of cancer at the time, such as cough, coughing blood and unintentional weight loss.^[3] It has been suggested that LEMS associated with lung cancer is more severe.^[4]

Causes[edit]

LEMS is often associated with lung cancer (50–70%),^[1] making LEMS a paraneoplastic syndrome.^[4] Of the people with small cell lung cancer, 1–3% have LEMS.^[3] In most of these cases, LEMS is the first symptom of the lung cancer, and it is otherwise asymptomatic.^[3]

LEMS may also be associated with autoimmune diseases, such as hypothyroidism (an underactive thyroid gland) or diabetes mellitus type 1.^[1]^[5] Myasthenia gravis, too, may happen in the presence of tumors (thymoma, a tumor of the thymus in the chest); people with MG without a tumor and people with LEMS without a tumor have similar genetic variations that seem to predispose them to these diseases.^[3] HLA-DR3-B8(an HLA subtype), in particular, seems to predispose to LEMS.^[5]

Mechanism[edit]

In normal neuromuscular function, a nerve impulse is carried down the axon (the long projection of a nerve cell) from the spinal cord. At the nerve ending in the neuromuscular junction, where the impulse is transferred to the muscle cell, the nerve impulse leads to the opening of voltage-gated calcium channels (VGCC), the influx of calcium ions into the nerve terminal, and the calcium-dependent triggering of synaptic vesicle fusion with plasma membrane. These synaptic vesicles contain acetylcholine, which is released into the synaptic cleft and stimulates the acetylcholine receptors on the muscle. The muscle then contracts. ^[1]

In LEMS, antibodies against VGCC, particularly the P/Q-type VGCC, decrease the amount of calcium that can enter the nerve ending, hence less acetylcholine can be released from the neuromuscular junction. Apart from skeletal muscle, the autonomic nervous system also requires acetylcholine neurotransmission; this explains the occurrence of autonomic symptoms in LEMS.^[1]^[3] P/Q voltage-gated calcium channels are also found in the cerebellum, explaining why some experience problems with coordination.^[4]^[5] The antibodies bind particularly to the part of the receptor known as the "domain III S5–S6 linker peptide".^[5] Antibodies may also bind other VGCCs.^[5] Some patients have antibodies that bind synaptotagmin, the protein sensor for calcium-regulated vesicle fusion.^[5] Many people with LEMS, both with and without VGCC antibodies, have detectable antibodies against the M1 subtype of the acetylcholine receptor; it is thought that their presence participates in a lack of compensation for the weak calcium influx.^[5]

Apart from the decreased calcium influx, there is also a disruption of active zone vesicle release sites, which is also thought to be antibody-dependent since LEMS patients have antibodies to components of these active zones (including voltage-dependent calcium channels). Together, these abnormalities lead to the decrease in muscle contractility. Repeated stimuli over a period of about 10 seconds eventually lead to sufficient delivery of calcium, and an increase in muscle contraction to normal levels, which can be demonstrated on electromyography by increasing amplitude of repeated compound muscle action potentials.^[1]

It has been shown that the antibodies found in LEMS associated with lung cancer also bind to calcium channels in the cancer cells, and it is presumed that the antibodies originally develop as a reaction to these cells.^[1] It has been suggested that the immune reaction to the cancer cells suppresses their growth and improves the prognosis from the cancer.^[3]^[5]

Diagnosis[edit]

Chest X-ray showing a tumor in the left lung (right side of the image)

The diagnosis is usually made on electromyography (EMG), which is one of the standard tests in the investigation of otherwise unexplained muscle weakness. This involves the insertion of small needles into the nerves supplying several muscles, administering small electrical impulses through these needles, and measuring the electrical response of the muscle in question. Two EMG investigations can be characteristic in LEMS: compound motor action potentials (CMAP) and single-fiber examination.^[1]

CMAP shows small amplitudes but normal latency and conduction velocities. If repeated impulses are administered (two per second or 2 Hz), it is normal for CMAP amplitudes to become smaller as the acetylcholine in the motor end plate is depleted. In LEMS, this decrease is larger than observed normally. Eventually, stored acetylcholine is made available, and the amplitudes increase again. In LEMS this remains insufficient to reach a level sufficient for transmission of an impulse from nerve to muscle; all can be attributed to insufficient calcium in the nerve terminal. A similar pattern is witnessed in myasthenia gravis. In LEMS, in response to exercising the muscle, the CMAP amplitude increases greatly (over 200%, often much more). This also occurs on the administration of a rapid burst of electrical stimuli (20 impulses per second for ten seconds). This is attributed to the influx of calcium in response to these stimuli.^[1]^[3] On single-fiber examination, features may include increased jitter (seen in other diseases of neuromuscular transmission) and blocking.^[1]

Blood tests may be performed to exclude other causes of muscle disease (elevated creatine kinase may indicate a myositis, and abnormal thyroid function tests may indicatethyrotoxic myopathy). Antibodies against voltage-gated calcium channels can be identified in 85% of people with EMG confirmed LEMS.^[1] Once LEMS is diagnosed, investigations such as a CT scan of the ches

t are usually performed to identify any possible underlying lung tumors. 50–60% of these are discovered immediately after the diagnosis of LEMS. The remainder is diagnosed later, but usually within two years and typically within four years.^[3] As a result, scans are typically repeated every six months for the first two years after diagnosis.^[1]While CT of the lungs is usually adequate, a positron emission tomography (PET) scan of the body may also be performed to search for an occult tumour, particularly of the lung.^[6]

Treatment[edit]

Molecular structure of 3,4-diaminopyridine, a commonly used drug treatment for LEMS.

If LEMS is caused by an underlying cancer, treatment of the malignancy usually leads to resolution of the symptoms.^[1] Treatment usually consists of chemotherapy, withradiation therapy in those with limited disease.^[3]

The three treatment modalities aimed at improving LEMS symptoms directly are improving the neuromuscular conduction and suppression of the immune system. Two classes of medication are used to improve neuromuscular transmission. Pyridostigmine decreases the degradation of acetylcholine after release into the synaptic cleft, and thereby improves muscle contraction. In LEMS, the potassium channel blocker 3,4-diaminopyridine (amifampridine) is also used. It delays the repolarization of nerve terminals after a discharge, thereby allowing more calcium to accumulate in the nerve terminal.^[1]^[3] An older agent, guanidine, causes many side-effects and is not recommended. 4-Aminopyridine (dalfampridine), an agent related to 3,4-aminopyridine, causes more side-effects than 3,4-DAP and is also not recommended.^[3]

Immune suppression tends to be less effective than in other autoimmune diseases. Prednisolone (a glucocorticoid or steroid) suppresses the immune response, and the steroid-sparing agent azathioprine may replace it once therapeutic effect has been achieved. Intravenous immunoglobulin (IVIG) may be used with a degree of effectiveness. Plasma exchange (or plasmapheresis), the removal of plasma proteins such as antibodies and replacement with normal plasma, may provide improvement in acute severe weakness. Again, plasma exchange is less effective than in other related conditions such as myasthenia gravis, and additional immunosuppressive medication is often needed.^[1] According to a systematic review by the Cochrane Collaboration, the best evidence in the treatment of LEMS exists for 3,4-aminopyridine and IVIG.^[7]

History[edit]

Anderson and colleagues from St Thomas' Hospital, London, were the first to mention a case with possible clinical findings of LEMS in 1953,^[8] but Lambert, Eaton and Rooke at the Mayo Clinic were the first physicians to substantially describe the clinical and electrophysiological findings of the disease in 1956.^[9]^[10] In 1972, the clustering of LEMS with other autoimmune diseases led to the hypothesis that it was caused by autoimmunity.^[11] Studies in the 1980s confirmed the autoimmune nature,^[5] and research in the 1990s demonstrated the link with antibodies against P/Q-type voltage-gated calcium channels.^[1]^[12]

References[edit]

^ Jump up to:^a ^b ^c ^d ^e ^f ^g ^h ⁱ ^j ^k ^l ^m ⁿ ^o ^p ^q ^r ^s ^t ^u ^v ^w Mareska M, Gutmann L (June 2004). "Lambert–Eaton myasthenic syndrome". Semin. Neurol.24 (2): 149–53. doi:10.1055/s-2004-830900. PMID 15257511.
Jump up^ Titulaer MJ, Lang B, Verschuuren JJ (December 2011). "Lambert–Eaton myasthenic syndrome: from clinical characteristics to therapeutic strategies". Lancet Neurol 10 (12): 1098–107.doi:10.1016/S1474-4422(11)70245-9. PMID 22094130.
^ Jump up to:^a ^b ^c ^d ^e ^f ^g ^h ⁱ ^j ^k ^l ^m Verschuuren JJ, Wirtz PW, Titulaer MJ, Willems LN, van Gerven J (July 2006). "Available treatment options for the management of Lambert–Eaton myasthenic syndrome". Expert Opin. Pharmacother. 7 (10): 1323–36.doi:10.1517/14656566.7.10.1323. PMID 16805718.
^ Jump up to:^a ^b ^c ^d Rees JH (June 2004). "Paraneoplastic syndromes: when to suspect, how to confirm, and how to manage". J. Neurol. Neurosurg. Psychiatr. 75 Suppl 2 (Suppl 2): ii43–50.doi:10.1136/jnnp.2004.040378. PMC 1765657.PMID 15146039.
^ Jump up to:^a ^b ^c ^d ^e ^f ^g ^h ⁱ Takamori M (September 2008). "Lambert–Eaton myasthenic syndrome: search for alternative autoimmune targets and possible compensatory mechanisms based on presynaptic calcium homeostasis". J. Neuroimmunol. 201-202: 145–52.doi:10.1016/j.jneuroim.2008.04.040. PMID 18653248.
Jump up^ Ropper AH, Brown RH (2005). "53". In Ropper AH, Brown RH.Adams and Victor's Principles of Neurology (8th ed.). New York: McGraw-Hill Professional. p. 1261. doi:10.1036/0071469710.ISBN 0-07-141620-X.
Jump up^ Maddison P, Newsom-Davis J (2005). "Treatment for Lambert–Eaton myasthenic syndrome". In Maddison, Paul. Cochrane Database of Systematic Reviews (2): CD003279.doi:10.1002/14651858.CD003279.pub2. PMID 15846654.
Jump up^ Anderson HJ, Churchill-Davidson HC, Richardson AT (December 1953). "Bronchial neoplasm with myasthenia; prolonged apnoea after administration of succinylcholine". Lancet 265 (6799): 1291–3.doi:10.1016/S0140-6736(53)91358-0. PMID 13110148.
Jump up^ Lambert-Eaton-Rooke syndrome at Who Named It?
Jump up^ Lambert EH, Eaton LM, Rooke ED (1956). "Defect of neuromuscular conduction associated with malignant neoplasms".Am. J. Physiol. 187: 612–613.
Jump up^ Gutmann L, Crosby TW, Takamori M, Martin JD (September 1972). "The Eaton–Lambert syndrome and autoimmune disorders". Am. J. Med. 53 (3): 354–6. doi:10.1016/0002-9343(72)90179-9.PMID 4115499.
Jump up^ Motomura M, Hamasaki S, Nakane S, Fukuda T, Nakao YK (August 2000). "Apheresis treatment in Lambert–Eaton myasthenic syndrome". Ther. Apher. 4 (4): 287–90. doi:10.1046/j.1526-0968.2000.004004287.x. PMID 10975475.

Lambert Eaton Myasthenic Syndrome (LEMS): An Autoimmune Syndrome Causing Muscle Weakness

2014-05-23T06:55:00.003-07:00

Lambert Eaton Myasthenic Syndrome (LEMS): An Autoimmune Syndrome Causing Muscle Weakness

Lambert Eaton Myasthenic Syndrome belongs to the class of disorders referred to as neuromuscular transmission disorders. That is to say, that this group of diseases affects the ability of the nerves to communicate with the muscles and thus leads to muscle weakness.

Lambert Eaton Myasthenic Syndrome (LEMS) is also an autoimmune disorder because it is a product of the body that is acting to cause the disorder. In other words, the body is acting against itself to cause disease.

Finally, LEMS is often a paraneoplastic disorder. The term paraneoplastic refers to a disease that is indirectly caused by a neoplasm or cancer. While we will discuss LEMS in detail, it is important to know that a variety of paraneoplastic syndromes exist and can be the first signs of a malignancy.

PATHOPHYSIOLOGY

In order to understand LEMS, it is necessary to first understand how muscles are made to work. We will take a look at the transmission of information from the brain to the muscles and analyze the steps that allow this remarkable process to occur.

One of the first things that occurs when a person wants to make a voluntary movement (i.e. a movement that the individual decides to make and not a reflex) is that the frontal lobe will communicate the intention to the parietal lobe on the appropriate side of the brain. Remember that the left side of the brain controls motor movement on the right side of the body and vice versa. While there are a few minor exceptions to this rule, this holds for nearly all muscle actions and certainly those that we are interested in.

The parietal lobes around found on both sides of the brain and are located above the ears. Each lobe is divided into specific segments that are responsible for controlling different muscles in the body. When one of these segments fires an impulse (electrical), it travels down nerve fibers on its way to the appropriate muscle. Nerves from the brain carry information down the spinal cord to the appropriate level where they make a connection to another nerve. The location where two nerves meet once another is referred to as a neural synapse.

At a synapse, nerves do not directly touch one another. Instead, they only come close to touching. The space between the end of one nerve and the beginning of the next is referred to as the synaptic cleft. The cleft is just a tiny space between nerves. Communication between nerves occurs through the release of chemicals, usually acetylcholine, that travel from the end of the first nerve (called the axon) to the beginning of the next nerve (called the dendrite) by diffusing across the very tiny synaptic cleft.

Once the chemical signal reaches the next nerve, it is converted back into an electrical signal and continues its journey toward the muscle. When the electrical impulse reaches the end of this second nerve, it is again converted into a chemical signal at another cleft. This cleft is referred to as a neuromuscular junction and deserves some special attention.

When an electrical impulse reaches the end of a nerve at a neuromuscular junction, several special things happen. First, the depolarization caused by the presence of the electrical action potential causes specialized calcium gates on the surface of the nerve ending to open. These specialized calcium gates are referred to as voltage gated calcium channels and when they open, they allow calcium to enter the axon of the nerve. High levels of calcium inside the axon cause special packages of chemicals (called vesicles) to hook to the surface of the nerve and release their chemicals into the cleft between nerve and muscle.

The chemical that is release is acetylcholine and it diffuses across the cleft from the nerve axon to the muscle where it binds to special receptors on the muscle. When it binds to these receptors, sodium and potassium (salts) are allowed into the muscle cell. This causes depolarization of the muscle cell, which allows calcium to enter that cell and bind to specialized proteins that allow the muscle to contract. As long as acetylcholine (ACh) is present, the muscle cell will remain depolarized. In order to stop the process and allow the muscle to relax, special enzymes called cholinesterases break down the ACh. These enzymes will become important later when we discuss the diagnosis and treatment of LEMS (1).

This is a basic overview of how the brain tells a muscle to contract and move. The most important part of this process in regard to LEMS is the neuromuscular junction. In particular, acetylcholine and the calcium channels are important. In LEMS, the calcium channels are prevented from working properly by the presence of antibodies against these channels. Without proper functioning of the calcium channels in the neuron (nerve), calcium cannot enter the axon and trigger the release of ACh and thus depolarization of the muscle fiber cannot occur. The result is muscle weakness.

The development of autoantibodies in LEMS is poorly understood. In fact, the antibodies can arise through a variety of mechanisms and even though LEMS can be its own, free-standing autoimmune disease, it is often associated with cancer. Estimates vary, but it is generally accepted that 50 - 70% of LEMS cases are the result of an underlying malignancy (2). That is to say, in the majority of cases auntoantibodies arise as the result of a cancer. Most often, the cancer is small cell lung cancer (SMLC), but lymphoma, non-small cell lung cancer, prostate cancer and bladder cancer have also been implicated in LEMS (3).

SYMPTOMS

Often, the symptoms of LEMS develop before the presence of a malignancy is known. Thus, it is important to recognize and correctly diagnose the condition so that an evaluation of an occult cancer can be performed.

The major symptom of LEMS is muscle weakness. Usually the proximal muscles of the lower legs are most commonly affected (2). This means that the thigh muscles and hamstrings become weak. Patients will not difficulty climbing stairs, rising from a chair, or lifting their legs. The weakness is generally insidious in onset and progressive over time. This means that the weakness is usually mild to begin with, but becomes more severe over weeks or months (4).

Any other muscle can be affected, but the proximal legs are most common while muscles of the face and eyes are affected in up to 70% of patients (2). Symptoms due to affects of the facial muscles include ptosis (inability to open the eyelids), facial drooping, and difficulty swallowing (5). When the muscles of the eye are affected, double vision (diplopia) is the major symptom because the muscles are unable to keep both eyes pointing in the same direction.

The symptoms of LEMS are very similar to those of a related condition referred to as myasthenia gravis (MG), which also causes weakness of the muscles of the face and body. It is important to distinguish MG from LEMS as the treatments are different and the need to evaluate for occult malignancy is more urgent in LEMS. There are diagnostic tests that can be sued to separate the two conditions, but there are symptomatic differences as well.

One of the ways in which the symptoms of LEMS differ from those of MG is referred to as facilitation. In MG, use of the affected muscle in a repeated fashion leads to increased weakness. In LEMS, the opposite is true. Repeated use or stimulation of a muscle affected by LEMS will result in a decrease in weakness of the muscle. This phenomenon is referred to as facilitation.

Another group of symptoms present in LEMS that generally is not present in MG is autonomic dysfunction. That is, improper functioning of the nervous system responsible for all of the "automatic: functions in the body. This includes such things as sweating and heart beat. Patients with autonomic dysfunction will often experience dry mouth, dizziness when standing, impotence, and rapid fluctuations in blood pressure (5).

While specific symptoms of cancer may also be present, they usually are not. If present at all, symptoms of cancer in early LEMS generally fall into the category of "B" type symptoms and include such things as unintended weight loss, night sweats, fevers, and chills. Usually the symptoms of LEMS present before symptoms of the cancer do.

DIAGNOSIS

Diagnosis of LEMS depends on both physical exam findings and laboratory testing. We will focus on physical exam findings first.

Deep tendon reflexes are generally absent or diminished in a patient with LEMS. One helpful diagnostic maneuver involves having a patient contract a muscle for 10 seconds and testing for the reflex both before and after. The return of a previously absent reflex in this setting is characteristic of LEMS. An example would be the return of the knee reflex after prolonged contraction of the quadriceps muscles.

The tensilon test involves the administration of a cholinesterase inhibitor to help differentiate between LEMS and MG. A cholinesterase inhibitor prevent the enzyme that breaks down ACh from working, thus leading to an increased amount of ACh in the synaptic cleft and an increased strength in muscle contraction (please see above). Generally, this test works only in MG and not in LEMS. Thus, administration of a cholinesterase inhibitor followed by increased muscle strength is diagnostic of MG and not LEMS. Unfortunately, this test is not incredibly reliable and is generally not used any longer (4).

Direct measurement of antibodies against the calcium channels yields a definitive diagnosis of LEMS. However, antibodies are only present in 85% of cases, making this a specific, but not very sensitive test (2). The presence of antibodies makes the diagnosis, but their absence does not rule out LEMS. If LEMS is suspected, but the antibody testing is negative, one final test can be performed to make the clinical diagnosis of LEMS.

Electrodiagnostic studies involve the use of needles and mild electric shocks in order to stimulate individual muscles. The level of electrical shock need to stimulate a muscle and the movement obtained after a given shock can be used to diagnose LEMS and also to differentiate it from MG. In LEMS, the following things are found on electrodiagnostic testing:

- The amount of shock needed to stimulate a muscle is decreased after exercising. This is the opposite of what is found in MG.

- The gain following exercise rapidly diminishes

- Rapid repetition of the shock leads to enhanced muscle stimulation while slow repetition leads to decreased muscle stimulation.

This test can be painful, but the data obtained are often essential to diagnosis and to staging the severity of muscle involvement (4).

TREATMENT

The treatment of LEMs can be broken down into four categories as follows:

Emergency treatment - When the muscles responsible for breathing are affected in LEMS, supportive therapy is needed. This involves the use of supplemental oxygen and, in some cases, the use of a tube and respirator to do the breathing that a person cannot do on his or her own (4).

Medication - Cholinesterase inhibitors are used in LEMS, but generally do not result in significant improvement. Nevertheless, they are used. Generally, a small amount of benefit is obtained by combining a cholinesterase inhibitory with a potassium channel blocker. This second class of drugs works by preventing potassium channels from working properly at the end of the axon and thus allowing for a prolonged depolarization. The prolonged depolarization of the axon allows more calcium channels to open, more calcium to enter, and thus more ACh vesicles to release their ACh. The cholinesterase inhibitor then allows the ACh that is released to remain in the synapse longer, leading to a stronger depolarization of the muscle fiber and thus stronger muscle contraction. Potassium channel blockers, of which 3,4,-diaminopyridine is most effective, are very toxic and must be used carefully be physicians experienced in their administration (4).

Immune modulation - This refers to the use do of drugs and procedures designed to minimize the effectiveness of the autoantibodies or rid them from the body altogether. The administration of antibodies through IV (called IVIG) that are designed to bind to the autoantibodies and render them ineffective can be moderately effective in some patients. (4)

Treatment of the Cancer - If LEMS is the result of an underlying malignancy, the symptoms can be reduced or eradicated through treatment of the tumor. While complete removal of a tumor and cure of a cancer is always the ultimate goal, it is not necessary to remove the tumor in its entirety to treat LEMS. Often, simply shrinking the tumor through the use of chemotherapy or radiation is enough to decrease the level of circulating autoantibody and thus decrease the symptoms of LEMS.

PROGNOSIS

Despite these treatments, LEMS is generally progressive over time. Despite its progressive nature, LEMS is seldom accountable for death in patients who suffer from it. Usually, the underlying malignancy results in death before LEMS does.

Catalyst Pharmaceutical Partners Announces Recommendation of Data Monitoring Committee to Continue Pivotal Phase lll Clinical Trial for Firdapse(TM)

2014-05-23T06:52:00.001-07:00

Catalyst Pharmaceutical Partners Announces Recommendation of Data Monitoring Committee to Continue Pivotal Phase lll Clinical Trial for Firdapse(TM)

CORAL GABLES, Fla., Oct. 15, 2013 (GLOBE NEWSWIRE) -- Catalyst Pharmaceutical Partners, Inc. (Nasdaq:CPRX), a specialty pharmaceutical company focused on the development and commercialization of novel prescription drugs targeting rare (orphan) neuromuscular and neurological diseases and disorders, announced today that, at a recently held meeting, the independent Data Monitoring Committee (DMC) overseeing the Company's ongoing pivotal Phase III clinical trial in the United States and Europe evaluating Firdapse(TM) for the treatment of Lambert-Eaton Myasthenic Syndrome (LEMS) recommended that the Company continue the trial as planned based on the committee's review of safety and clinical data from the trial.

The DMC is a group of experts responsible for the independent review of accumulated, unblinded clinical safety and efficacy data obtained in the Company's clinical trial, in order to safeguard the interests and safety of participants and future patients. The DMC considers study-specific data, as well as relevant background knowledge about the disease, test agent or patient population under study.

The Firdapse(TM) Phase III clinical trial is designed as a randomized, double-blind, placebo-controlled, discontinuation trial enrolling 36 patients diagnosed with LEMS at sites in the U.S. and Europe. To accomplish this, the Company, in addition to the 7 active sites at the time of acquisition, has initiated 12 additional sites and expects to initiate shortly an additional 6 sites in the U.S., Europe, Canada and South America. The Company expects to complete enrollment in the trial about the end of the fourth quarter of 2013 and to announce top line data from the trial during the second quarter of 2014.

About LEMS

Lambert-Eaton Myasthenic Syndrome, or LEMS, is a rare autoimmune disorder characterized by muscle weakness of the limbs. The disease is caused by an autoimmune reaction where antibodies are formed against the connection between nerves and the muscles they supply. Often, LEMS is associated with an underlying malignancy, most commonly small-cell lung cancer, and in some individuals, LEMS is the first symptom of such malignancy. LEMS generally affects the extremities, especially the legs. As the disease most affects the parts of limbs closest to the trunk, difficulties with climbing stairs or rising from a sitting position are commonly noted. Physical exercise and high temperatures tend to worsen the symptoms. Other symptoms occasionally seen include weakness of the muscles of the mouth, throat, and eyes. Individuals affected with LEMS also may have a disruption of the autonomic nervous system, including dry mouth, constipation, blurred vision, impaired sweating, and/or hypotension.

About Catalyst Pharmaceutical Partners

Catalyst Pharmaceutical Partners, Inc. is a specialty pharmaceutical company focused on the development and commercialization of novel prescription drugs targeting rare (orphan) neuromuscular and neurological diseases, including Lambert-Eaton Myasthenic Syndrome (LEMS), infantile spasms, and Tourette Syndrome. Catalyst's lead candidate, Firdapse(TM) for the treatment of LEMS, is currently undergoing testing in a global, multi-center, pivotal phase III trial and recently received "Breakthrough Therapy Designation" from the U.S. Food and Drug Administration (FDA). Catalyst is also developing a potentially safer and more potent vigabatrin analog (designated CPP-115) to treat infantile spasms, and epilepsy, as well as other neurological conditions associated with reduced GABAergic signaling, like post-traumatic stress disorder and Tourette Syndrome.

Forward-Looking Statements

This press release contains forward-looking statements. Forward-looking statements involve known and unknown risks and uncertainties, which may cause Catalyst's actual results in future periods to differ materially from forecasted results. A number of factors, including the timing of completion of Catalyst's currently ongoing Phase III trial of Firdapse(TM), whether the Phase III trial will be successful, whether the receipt of breakthrough therapy designation for Firdapse(TM) will expedite the development and review of Firdapse(TM) by the FDA or the likelihood that the product will be found to be safe and effective, whether an NDA for Firdapse(TM) will ever be accepted for filing by the FDA, the timing of any such NDA filing or acceptance, whether any of Catalyst's product candidates will ever be approved for commercialization or successfully commercialized, and those other factors described in Catalyst's Annual Report on Form 10-K for the fiscal year 2012 and other filings with the U.S. Securities and Exchange Commission (SEC), could adversely affect Catalyst. Copies of Catalyst's filings with the SEC are available from the SEC, may be found on Catalyst's website or may be obtained upon request from Catalyst. Catalyst does not undertake any obligation to update the information contained herein, which speaks only as of this date.

Catalyst Pharmaceutical Announces Social Media Presence - WSJ.com

2014-05-23T06:50:00.001-07:00

Catalyst Pharmaceutical Announces Social Media Presence - WSJ.com

Catalyst Pharmaceutical Announces Social Media Presence!!!

Catalyst Pharmaceutical Partners, Inc. (Nasdaq:CPRX), a specialty pharmaceutical company focused on developing safe and effective approved medicines targeting orphan neuromuscular and neurological diseases, today announced that it has joined the social media conversation with a company blog and Twitter handle, which can be found at CatalystPharmaBlog.com and @CatalystPharma respectively.

"We are embracing social media to engage with and better understand the patient communities we intend to serve," said Patrick J. McEnany, Chairman and CEO of Catalyst. "We are developing drugs for patients with rare, orphan diseases, and that means we need to go beyond traditional methods of communications to reach and learn from these patients, their families and their care providers."

Catalyst will use these social media channels to foster conversations between Catalyst and the Lambert-Eaton Myasthenic Syndrome (LEMS) patient community and company stakeholders. Information distributed via the corporate blog and Twitter will provide meaningful updates, valuable information and relevant resources for the LEMS patient community and stakeholders. Catalyst also will use social media to engage with communities as it relates to the pipeline for infantile spasms, complex partial seizures and Tourette syndrome.

A link to our full social media guidelines can be found here.

About Catalyst Pharmaceutical Partners

Catalyst Pharmaceutical Partners, Inc. is a specialty pharmaceutical company focused on the development and commercialization of novel prescription drugs targeting rare (orphan) neuromuscular and neurological diseases, including Lambert-Eaton Myasthenic Syndrome (LEMS), infantile spasms, and Tourette syndrome. Catalyst's lead candidate, Firdapse(TM) for the treatment of LEMS, is currently undergoing testing in a global, multi-center, pivotal Phase 3 trial and recently received "Breakthrough Therapy Designation" from the U.S. Food and Drug Administration (FDA). In 2012, Catalyst licensed Firdapse(TM) from BioMarin and Catalyst assumed management of the Phase 3 pivotal trial, previously initiated by BioMarin. Firdapse(TM) is the first and only European approved drug for symptomatic treatment in adults with LEMS.

Catalyst is also developing a potentially safer and more potent vigabatrin analog (designated CPP-115) to treat infantile spasms, and epilepsy, as well as other neurological conditions associated with reduced GABAergic signaling, like post-traumatic stress disorder and Tourette syndrome. CPP-115 has been granted U.S. orphan drug designation for the treatment of infantile spasms by the FDA and has been granted EU orphan medicinal product designation for the treatment of West Syndrome by the European Commission.

Forward-Looking Statements

This press release contains forward-looking statements. Forward-looking statements involve known and unknown risks and uncertainties, which may cause Catalyst's actual results in future periods to differ materially from forecasted results. A number of factors, including the timing of completion of Catalyst's currently ongoing Phase 3 trial of Firdapse(TM), whether the Phase 3 trial will be successful, whether the receipt of breakthrough therapy designation for Firdapse(TM) will expedite the development and review of Firdapse(TM) by the FDA or the likelihood that the product will be found to be safe and effective, whether an NDA for Firdapse(TM) will ever be accepted for filing by the FDA, the timing of any such NDA filing or acceptance, whether Catalyst will be the first company to receive an approval for 3,4-DAP, giving it 7-year marketing exclusivity for its product, whether any of Catalyst's product candidates will ever be approved for commercialization or successfully commercialized, and those other factors described in Catalyst's Annual Report on Form 10-K for the fiscal year 2012 and other filings with the U.S. Securities and Exchange Commission (SEC), could adversely affect Catalyst. Copies of Catalyst's filings with the SEC are available from the SEC, may be found on Catalyst's website or may be obtained upon request from Catalyst. Catalyst does not undertake any obligation to update the information contained herein, which speaks only as of this date.

CONTACT: Media/Investor Contacts: 
         Donna LaVoie or David Connolly 
         LaVoie Group 
         (617) 374-8800 
         dlavoie@lavoiegroup.com 
         dconnolly@lavoiegroup.com 
 
         Company Contact: 
         Patrick J. McEnany 
         Catalyst Pharmaceutical Partners 
         Chief Executive Officer 
         (305) 529-2522 
         pmcenany@catalystpharma.com

Catalyst Pharma updates on Firdapse development activities

2014-05-23T06:39:00.001-07:00

Catalyst Pharma updates on Firdapse development activities

The Myasthenia Gravis Association will be supporting Rare Disease Day 2014 on Friday 28 February as a way of raising awareness of Myasthenia Gravis.

2014-05-23T06:35:00.001-07:00

The Myasthenia Gravis Association will be supporting Rare Disease Day 2014 on Friday 28 February as a way of raising awareness of Myasthenia Gravis.

The association’s next support group meeting will take place on Thursday 6 March at Demesne Media Arts Centre, Bowness Road, Middleton, M24 4NU at 7.30pm.

The Myasthenia Gravis Association exists to promote the welfare of suffers from Myasthenia Gravis, Lambert- Eaton Myasthenic Syndrome (LEMS) and CMS Congenital Myasthenic Syndrome throughout the United Kingdom and the Republic of Ireland.

Myasthenia Gravis is a neuro-muscular condition that affects approximately 1 in 10,000 people in the UK. Myasthenia Gravis is a condition characterised by fluctuating muscle weakness, which in rare cases may be fatal, particularly if left untreated.

Regional Organiser Sarah Sanders said: “There are over 600 people with Myasthenia Gravis in the North West Region and so many more that we are not currently aware of.”

Myasthenia Gravis, like most rare diseases is often chronic. Undiagnosed and left untreated it can be life-threatening and can affect all ages and races. The number of reported cases in children is also on the increase.

The condition is so rare that most people have never even heard of it. As it stands, many doctors only have a vague knowledge of the disease so many cases tend to go undiagnosed.

Currently, there is no existing cure for the disease with control by medication being the best thing available to sufferers.

Sarah added: “The association aims to provide the best possible support services for people living with Myasthenia and their families.”

In order to continue the support they offer to sufferers of the disease, the association is also appealing for help from volunteers. Sarah added: “We are appealing to local people who may have an hour or two to spare to volunteer for us.

“Our volunteering opportunities range from helping us to deliver our support services to assisting with fundraising initiatives. We aim to tailor those volunteering roles to the individual to ensure that they are involved in projects which they will find appealing and in line with their own interests.”

Student Volunteering Week 2014 takes place from 24 February to 2 March 2014 so the association is hoping to appeal to students who would like to get involved.

For more information on the association, contact Sarah Sanders:

sarah.sanders@mga-charity.org

Catalyst Pharmaceutical Partners Observes Rare Disease Day Today - WSJ.com

2014-05-23T06:27:00.001-07:00

Catalyst Pharmaceutical Partners Observes Rare Disease Day Today - WSJ.com