Breaking World Pharma News

Child cancer groups call for concerted action

Fri, 18 Sep 2026 10:00:00 +0200

It’s one of the cruelest ironies in health and medicine. Pediatric sarcomas are some of the rarest yet most aggressive forms of childhood cancer, and some of the hardest to cure. Whereas many adult cancers are now treatable with new therapeutic strategies, such as immunotherapies, pediatric sarcomas have not seen nearly as many advances. Survival rates have remained largely unchanged for decades.

Experimental study points to systemic sclerosis treatment

Thu, 17 Sep 2026 10:00:00 +0200

Investigators at Cedars-Sinai Health Sciences University used an experimental drug to reverse the effects of systemic sclerosis, a serious autoimmune disease. The study, conducted in a laboratory, was published in JCI Insight.

Systemic sclerosis, also called scleroderma, is a chronic, life-threatening condition that scars the skin, lungs, heart and other organs.

Protein build-up may shape cancer drug response

Wed, 16 Sep 2026 10:00:00 +0200

The way cancer-causing proteins accumulate in tumours may influence how effectively a promising new generation of medicines can remove them, a study suggests.

Researchers found that PROTAC (Proteolysis Targeting Chimeras) drugs were more effective at removing proteins that build up because they are not broken down properly than proteins that cancer cells continually produce in large amounts.

Sanofi and Cheplapharm to create new strategic partnership in mature medicines

Tue, 15 Sep 2026 10:00:00 +0200

Sanofi and Cheplapharm, a European leader in well-established medicines, announced their intention to create a strategic partnership under which Cheplapharm would take over from Sanofi a selection of 20 mature medicines and three manufacturing sites worldwide. In return, Sanofi will receive a 26.4% equity stake in Cheplapharm, building on a collaboration that started in 2014.

Hidden immune cells in the lung could lead to better flu vaccines

Mon, 14 Sep 2026 10:00:00 +0200

Influenza continues to cause major illness each year in the United States, leading to more than 35,000 deaths annually. Children, older adults, pregnant individuals, people with weakened immune systems, and those with chronic conditions such as heart disease, metabolic disorders, and cancer are especially vulnerable.

Vaccination remains the most effective way to prevent flu infection, but current vaccines do not always stop the virus from taking hold in the respiratory tract.

Novel drug shows promise in an early phase trial in heart failure

Fri, 11 Sep 2026 10:00:00 +0200

The oral relaxin agonist, AZD5462, improved cardiac function in patients with chronic heart failure, according to results presented in a Hot Line session at ESC Congress 2026 and published simultaneously in Circulation.

Despite several guideline-directed medical therapies for heart failure, many patients continue to experience considerable disease burden.

Cholesterol-lowering medication reduces heart attack and stroke risk in older people by 30%

Thu, 10 Sep 2026 10:00:00 +0200

A world-first clinical trial conducted by Monash University researchers has shown that cholesterol-lowering medication can reduce heart attack and stroke in people aged over 70 years.

Statins are commonly prescribed to manage high cholesterol in people up to 75 years, and are known to reduce the risk of heart attack and stroke.

Scientists decode gene circuits of human immunity, fueling new era of disease research

Wed, 09 Sep 2026 10:00:00 +0200

Scientists from Gladstone Institutes, UC San Francisco, and Stanford University, in collaboration with Biohub, have unveiled a massive, high-resolution functional map of human immune cells that promises to transform our understanding of how genetics control health and disease.

Published in the journal Cell, the study represents a landmark achievement in immunology and genomics.

FDA approves first in class targeted therapy for metastatic pancreatic cancer

Tue, 08 Sep 2026 17:24:16 +0200

The U.S. Food and Drug Administration approved Rasonque (daraxonrasib), a RAS inhibitor for the most common form of pancreatic cancer - delivering a new treatment option to patients with advanced pancreatic cancer months ahead of schedule.

This action demonstrates the agency’s commitment to moving with urgency, reducing unnecessary delays and advancing innovative treatments that can make a meaningful difference in the lives of American patients and their families.

Rethinking oral cancer treatment: A review of next-generation drug delivery systems

Mon, 07 Sep 2026 10:00:00 +0200

Delivering drugs directly to oral tumor sites remains challenging. A recent review involving researchers from Hasanuddin University examines barriers to local drug delivery and analyses strategies for targeting oral cancer lesions. Saliva, chewing, tongue movement, and individual tumor characteristics can affect how long a medicine remains at the treatment site and how consistently it is released.

AstraZeneca expands company’s $100m+ commitment to advance youth health equity in more than 60 countries

Fri, 04 Sep 2026 10:00:00 +0200

AstraZeneca announced a new five-year phase of its flagship Young Health Programme (YHP) in partnership with UNICEF and 60 non-governmental organisations (NGOs), extending its investment in youth health equity to over $100m since the YHP’s inception in 2010.

The programme, which supports young people in underserved communities, is committed to preventing conditions such as cancer, diabetes, heart disease and lung disease by educating and activating young people to

A ray of hope in the fight against cancer

Thu, 03 Sep 2026 10:00:00 +0200

Cancer is the second leading cause of death in Germany. In the search for new therapeutic approaches that target the uncontrolled growth of tumor cells, a class of enzymes known as cyclin-dependent kinases (CDKs) has come into focus. This is because this family of enzymes plays a fundamental role in gene expression and cell division. An overview of the current state of research on the transcriptional regulation of CDKs and the prospects for cancer treatment was compiled by Prof. Matthias Geyer of the University Hospital Bonn and the University of Bonn in collaboration with Prof. Robert P.

FDA approves first drug of its kind for polycythemia vera, a rare blood disorder

Wed, 02 Sep 2026 10:00:00 +0200

The U.S. Food and Drug Administration approved Mimrylo (rusfertide), a new treatment for adults with polycythemia vera, a rare blood disorder that causes the body to make too many red blood cells. The excess cells can thicken the blood and increase the risk of cardiovascular issues, such as blood clots, stroke and heart attack.

Novel combination therapy improves treatment outcomes in preclinical lung cancer models

Tue, 01 Sep 2026 10:00:00 +0200

A new preclinical study has demonstrated that combining HER3-DXd with olaparib significantly improves treatment responses in lung cancer models. This combination therapy induces irreparable DNA damage in cancer cells while also stimulating the body’s immune system to combat the disease.

Lung cancer is the most commonly diagnosed cancer and the leading cause of cancer-related deaths worldwide.

First-in-class therapy targets "undruggable" protein in hard-to-treat blood cancers

Mon, 31 Aug 2026 10:00:00 +0200

A first-in-class therapy to target MYC, one of the most sought-after and difficult targets in cancer biology, showed promise in hard-to-treat blood cancers, according to a new preclinical study from The University of Texas MD Anderson Cancer Center published in Blood.

Researchers led by Michael Andreeff, M.D., Ph.D., professor, and Yuki Nishida, M.D., Ph.D., assistant professor, both of Leukemia, found that experimental drug GT19630 interrupts a newly discovered cycle between MYC and GSPT1,

AI designs a novel E. coli killer

Fri, 28 Aug 2026 10:00:00 +0200

In science, the suffix “phage” means to devour. Thus, the name bacteriophage might conjure images of tiny creatures that “eat” bacteria. While that conception isn’t 100 percent scientifically accurate, bacteriophages are lethal bacteria killers nonetheless, and scientists are excited about engineering phage DNA as a path to new antibiotics.

And so it is that chemical engineer Brian Hie and bioengineering graduate student Samuel King came to study bacteriophage ΦX174 (pronounced “FYE-ex-1-7-4”).

GLP-1 drug linked to heart benefits for high-risk patients

Thu, 27 Aug 2026 10:00:00 +0200

Adding the GLP-1 receptor agonist drug tirzepatide (marketed as Mounjaro) to standard care for patients with type 2 diabetes and heart disease is associated with a lower risk of a major cardiovascular event, such as a heart attack or stroke, finds a study published by The BMJ.

Randomised trials and observational studies have shown non-inferior effects of tirzepatide compared to another GLP-1 receptor agonist, dulaglutide, for major adverse cardiovascular events (MACE) - a combined measure of heart attack, stroke, and death from any cause.

FDA approves daraxonrasib for metastatic pancreatic cancer following landmark clinical trial led by Dana-Farber

Wed, 26 Aug 2026 10:00:00 +0200

The U.S. Food and Drug Administration (FDA) has approved daraxonrasib, an oral multi-selective RAS(ON) inhibitor, for adults with metastatic pancreatic adenocarcinoma who have received at least one prior systemic therapy or are not candidates for multiagent systemic therapy. The approval is based primarily on the results from the pivotal randomized phase 3 RASolute 302 trial comparing daraxonrasib to chemotherapy as second-line therapy for patients with metastatic pancreatic cancer, led by Brian Wolpin, MD, MPH, director of the Hale Family Center for Pancreatic Cancer Research at Dana-Farber Cancer Institute.

Targeting MDA-9 restrains head and neck tumor growth and eliminates therapy resistance

Tue, 25 Aug 2026 10:00:00 +0200

Chemotherapy remains a cornerstone of treatment for head and neck cancers, the seventh-most common cancer type worldwide. However, the disease is often diagnosed at a late stage once it has already spread to other organs, and the majority of patients on active treatment will develop resistance to chemotherapy within a matter of months, underscoring a prominent avenue for designing more effective care strategies.

Engineered human neurons rebuild damaged spinal cord circuits

Mon, 24 Aug 2026 10:00:00 +0200

Spinal cord injuries affect an estimated 15 to 20 million people worldwide, often causing lasting impairments in movement, sensation, and independence. Such injuries can be especially devastating when they occur at the level of the neck, where damaged spinal circuits disrupt signals that control the diaphragm, the main muscle used for breathing.

Despite advances in emergency care and rehabilitation, no approved therapies exist to rebuild the neurons and connections lost after a spinal cord injury.