New research from a team of scientists in the McKelvey School of Engineering at Washington University in St. Louis suggests reconsidering whether high-frequency waveforms are as effective as the longer-duration waveforms that are already commonly available. The team worked in collaboration with researchers at the Medical University of Vienna, Austria, and the Friedrich-Alexander University in Erlangen-Nürnberg, Germany.

Ismael Seáñez, an assistant professor of biomedical engineering and of electrical and systems engineering at McKelvey Engineering and of neurosurgery at WashU Medicine, and Rodolfo Keesey, a doctoral student in his lab, examined whether high-frequency waveforms actually target the neural structures that lead to recovery as well as existing waveforms for conventional spinal cord stimulation. Seáñez’s team looked at the mechanisms behind responses prompted by high-frequency stimulation both in a human model and in computational models to determine any differences. Research results were published May 12 in Nature Biomedical Engineering. 

“Computational models and human experiments revealed that conventional spinal cord stimulation promotes motor recovery by recruiting sensory nerves, which subsequently activate motor nerves,” Seáñez said. “This is counterintuitive, because our end goal is to recruit motor nerves and help the muscles move. However, sensory activation allows patients to voluntarily control the movement evoked by stimulation,” Seanez said.

“Also, when you recruit directly in the motor nerves, initially you get a very nice response, but then the muscle starts fatiguing because you recruit all the neurons in that muscle,” he said. “Sensory activation avoids this. We wanted to see if high-frequency waveforms also recruit motor neurons through the sensory pathway. If they don’t, they could be less effective at improving recovery.”

For this study, Keesey conducted three experiments. In the first, he stimulated nerves in the legs of 28 unimpaired participants to artificially activate the sensory pathway to raise the resting potential of the motor neurons.

“Neurons that fire together, wire together,” Keesey said. “Because we have all these rich sources of connections with sensory activation, we can help with rehabilitation by activating a pathway that interacts with the brain and interacts with circuits in the spine. And that allows us to have this rehabilitative mechanism. However, kilohertz-frequency waveforms were found to be less effective than conventional waveforms at activating the sensory fiber pathway. Therefore, we may miss out on all these connections. This is a bad sign for recovery.”

In the second set of experiments, Keesey and his collaborators recreated the setup in a computational model. In the final set of experiments, they tested spinal cord stimulation of the cervical and lumbar portions of the spinal cord that control the arms and legs.

“What we see is that the kilohertz frequency currents require much higher stimulation intensities to elicit muscle responses,” Seáñez said. “(It appears) that kilohertz frequency waveforms have poor specificity for the target neural structure of spinal cord stimulation.”

Keesey said sensory fiber recruitment is important because otherwise, the stimulation simply activates the muscle with less opportunity for rehabilitation.

“You lose these rich sources of input from the brain, from the spinal cord and all these mechanisms for rehabilitation and the fatigue-resistant prosthetic effect,” Keesey said.

“The first wave of tSCS devices is now clearing FDA approval for motor recovery following spinal cord injury,” Keesey continued. “These devices all use high-frequency modulated waveforms, but they can be dramatically more expensive. Not only do we already have commercially available devices that are much more affordable and can deliver conventional waveforms, our research indicates that their waveforms may be more effective for rehabilitation.”

Keesey R, Hofstoetter U, Zhaoshun H, Lombardi L, Hawthorn R, Bryson N, Alashqar A, Rowald A, Minassian K, Seáñez I. Fundamental limitations of kilohertz-frequency carriers in afferent fiber recruitment with transcutaneous spinal cord stimulation. Nature Biomedical Engineering, May 12, 2026. DOI: 10.1038/s41551-026-01684-w.

Funding for this research was provided by the National Institutes of Health’s The Eunice Kennedy Shriver National Institute of Child Health and Human Development (K12HD073945), National Institute of Neurological Disorders and Stroke (K01NS127936); the McDonnell Center for Systems Neuroscience at Washington University in St. Louis; the General Federal Ministry of Education and Research; and Austrian Science Fund.

Originally published on the WashU McKelvey Engineering website

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“This inaugural professorship stands as a tribute to Dr. Llorin-Roa’s remarkable legacy — a career defined by compassion, a strong sense of professional and social responsibility, and unwavering dedication to her patients,” said Chancellor Andrew D. Martin. “Dr. Drewry’s work reflects those same values. Her commitment to patients and her care for the teams she leads are inspiring.”

Drewry, who serves as vice chair of the WashU Medicine Department of Anesthesiology and director of the Division of Critical Care Medicine, was installed by David H. Perlmutter, MD, executive vice chancellor for medical affairs, the Spencer T. and Ann W. Olin Distinguished Professor and the George and Carol Bauer Dean of the School of Medicine.

“This professorship recognizes Dr. Drewry’s academic and clinical excellence and the impact she has made in critical care medicine,” Perlmutter said. “A visionary in the field, she understands that its reach extends beyond any single ICU or hospital. By combining multidisciplinary expertise with telemedicine, she has helped build a critical care model that connects across our region — strengthening the infrastructure for the sickest patients and saving many lives.”

Drewry joined the WashU Medicine faculty in 2011 and has held multiple leadership roles at WashU Medicine and BJC HealthCare, including executive director of the WashU Medicine/BJC Telemedicine Critical Care Center. In this role, she leads a large, multidisciplinary critical care enterprise spanning academic intensive care units (ICUs), community hospitals and a system-wide telemedicine program. Under her leadership, the Division of Critical Care Medicine has expanded its clinical footprint, strengthened cross-departmental partnerships and prioritized consistent, high-quality care, while fostering professional development for faculty, advanced practice providers and trainees.

“Dr. Drewry is simply one of the most impactful leaders at WashU Medicine today,” said Michael S. Avidan, MBBCh, the Dr. Seymour and Rose T. Brown Professor of Anesthesiology and head of the Department of Anesthesiology. “In every role that she has held, she has made the people around her better and the systems she leads stronger. Importantly, she has championed the team-based model of critical care and played a seminal role in making WashU Medicine a national leader in training advanced practice clinicians in critical care.”

During the COVID-19 pandemic, Drewry displayed extraordinary leadership, directing the critical care clinical activities of several BJC HealthCare community hospitals as well as the surgical and cardiothoracic ICUs at Barnes-Jewish Hospital. In response to the surge of critically ill patients, she helped expand the ICU footprint at BJC hospitals by 40% by arranging for additional physician coverage and helping to convert non-ICU spaces into functional ICU spaces. She also chaired BJC’s COVID-19 Taskforce, helping to coordinate the critical care response to COVID-19 and standardize care and resources across ICUs. For her leadership during the pandemic, she received a COVETED award in 2021 from the anesthesiology department and a Dean’s Impact Award in 2023.

Drewry also was instrumental in coordinating the tele-critical care response to the pandemic, working closely with the BJC Transfer Center to help ensure patients were placed in hospitals best suited to their medical needs. She expanded tele-ICU staffing early in the pandemic, enabling remote monitoring and consultation across sites. Throughout this period, she also prioritized the well-being of the nurse practitioners, physician assistants, residents and physicians as they navigated the demands of caring for numerous critically ill patients.

Drewry also is a leader deeply committed to knowledge generation, focusing her scholarly work on the interplay between body temperature, immune response and outcomes in sepsis. She has served as site principal investigator on several studies funded by the National Institutes of Health (NIH).

Drewry earned her undergraduate degree in molecular, cellular and developmental biology from Yale University and her medical degree from WashU Medicine. She completed residency training in anesthesiology and critical care at Massachusetts General Hospital. She returned to WashU Medicine for a fellowship in critical care medicine and later earned a Master of Science in Clinical Investigation.

About Necita Llorin-Roa

Necita Llorin-Roa, MD, was a renowned educator, clinician and beloved colleague who specialized in pediatric anesthesiology. She earned a bachelor’s degree from the University of the Philippines in 1964 and a medical degree from its School of Medicine in 1969. Llorin-Roa completed an internship and residency at Philippine General Hospital in Manila. She came to WashU Medicine and Barnes-Jewish Hospital in 1971, where she completed an anesthesia residency and fellowship.

Beginning in 2014, Llorin-Roa served as president of the Philippine Medical Association of Greater St. Louis Mission Foundation, leading medical and surgical missions to underprivileged communities in her home country of the Philippines. Llorin-Roa was a member of the American Medical Association, the American Society of Anesthesiologists, the International Anesthesia Research Society, the American Society of Regional Anesthesia and the Society for Education in Anesthesia.

Llorin-Roa, who died in 2018, generously endowed the Necita L. Roa, M.D. Award in Anesthesiology for a graduating WashU Medicine anesthesiology resident, as well as a fund to support global health programs for anesthesiology trainees. She made a commitment in 2011 to establish and endow the Llorin-Roa Professorship in Anesthesiology in the Department of Anesthesiology at WashU Medicine.

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Researchers at WashU Medicine and collaborating institutions have developed a novel computational tool that can accurately identify a genetic problem in a gene called RFC1 that is linked to certain forms of peripheral neuropathy. Peripheral neuropathy is one of the most common neurological disorders and can cause pain, sensory loss, imbalance and weakness. It affects 12–20% of all people in the U.S. and can affect up to 30% of adults over age 65. The new research is published in Annals of Neurology. 

The disease-causing change, known as an RFC1 repeat expansion, has been associated with neuropathy, but its role across the broader spectrum of patients with unexplained, or “idiopathic,” neuropathy has remained unclear. One reason is that these repeat expansions — in which the set of DNA “letters” AAGGG is repeated many more times than normal — are difficult to detect using standard genetic testing methods. 

The research team led by senior author Sheng Chih (Peter) Jin, an assistant professor of genetics and of pediatrics, and first author Zitian Tang, a graduate student in Jin’s lab, set out to bridge this technical gap by developing a new computational pipeline coupled with machine learning that can reliably identify and classify repeat expansions from genome sequencing data. Using this approach, they found that RFC1 repeat expansions may account for more than 2% of cases of idiopathic peripheral neuropathy. 

The new tool offers a more affordable and reliable way to look for this extremely complex genetic variation in both clinical and research settings. The finding also supports broader genetic testing for people with unexplained peripheral neuropathy, including those who have muscle weakness as well as sensory symptoms. The team has made the tool public on GitHub, which could help expand testing to help more patients receive an accurate diagnosis and give families clearer information about the genetic causes of their condition. 

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Moran was a faculty member of WashU Medicine Mallinckrodt Institute of Radiology (MIR) for 45 years, during which he became a pioneer in a life-saving technology to treat brain aneurysms. He retired from research and clinical practice in 2025.

Moran was born in Detroit in 1948. He earned his bachelor’s degree from the University of Notre Dame in 1970 and his MD from Saint Louis University School of Medicine in 1974. He completed both a residency in diagnostic radiology and a fellowship in neuroradiology in 1978 at MIR and joined the faculty as an assistant professor of radiology in the same year. He rose to full professor of radiology and of neurological surgery in 2003.

Moran was part of the Pipeline for Uncoilable or Failed Aneurysms study, published in 2013. This multicenter clinical trial proved the safety and efficacy of the pipeline embolization device to treat brain aneurysms — dangerous bulges in blood vessels that can rupture and cause a stroke. The device is inserted into the affected vessel to reduce internal pressure on the aneurysm and allow it to heal.

Moran became an expert on the clinical use of the pipeline embolization device and traveled around the world and across the U.S. as a procedure trainer and proctor, visiting more than 200 medical facilities.

As chair of MIR’s Continuous Quality Improvement Committee, Moran led an interdepartmental effort to reduce the time between a patient’s arrival in a medical facility and the start of an intervention. That success was recognized with the Team Award for Quality from Barnes-Jewish Hospital’s Endovascular Acute Stroke Committee in 2017.

Moran received additional awards and honors over the course of his career and was elected a fellow of the American Heart Association, the Society of NeuroInterventional Surgery and the American College of Radiology.

He is survived by his wife, Eleanor Moran; children John Moran (Molly), William Moran (Courtney) and Mary Catherine Moran; nine grandchildren; siblings Jeffrey Moran (Cindy), Susan Moran and Thomas Moran; and numerous nieces, nephews and in-laws.

Funeral services will be held at the Saint Louis Abbey Church, 500 S. Mason Road in Creve Coeur, Mo., at 11 a.m. Saturday, May 16. In lieu of flowers, the family requests Masses in his honor, memorial donations to the St. Louis Abbey or St. Louis Priory School or to the Detroit Catholic Central High School.

Read more about his life in the family obituary.

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Now, a clinical trial, led by researchers at Washington University School of Medicine in St. Louis, shows that a stem cell transplant in which the donor cells have been genetically engineered to remove a particular protein helps prevent toxic side effects and potentially improves the effectiveness of therapies given after a transplant to help prevent cancer recurrence.

The study was conducted at Siteman Cancer Center, based at Barnes-Jewish Hospital and WashU Medicine, and 14 other sites in the U.S. and Canada. The findings are published May 12 in the journal Nature Medicine.

According to the study’s corresponding author, John F. DiPersio, MD, PhD, the Virginia E. & Sam J. Golman Professor of Medicine at WashU Medicine, this gene-editing technology could help address a longstanding frustration in the field: CAR-T cell therapy — an immunotherapy that effectively treats some aggressive blood cancers — has not worked against all blood cancers, including acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS).

According to DiPersio, who treats patients at Siteman and is a research member there, myeloid cancers like AML and MDS are tricky to treat with CAR-T cells because the same proteins on cancer cells that the immunotherapy homes in on for destruction are also present on healthy myeloid cells, including therapeutic donor stem cells. As such, the anti-cancer therapy carries a high risk of toxicity because it also destroys healthy blood stem cells, which can trigger a dangerous inflammatory cascade. This effect also could dilute the effect of the anti-cancer therapy because so many of the CAR-T cells are attacking the wrong targets, leaving many cancer cells untouched.

This basic concept was first described by Miriam Y. Kim, MD, now an assistant professor of medicine at WashU Medicine. She began this research as a postdoctoral researcher at the University of Pennsylvania and continued the work in the DiPersio lab before becoming an independent investigator in the WashU Medicine Division of Oncology. She treats patients at Siteman and is also a research member there.

For this clinical trial, patients with AML and MDS received donor stem cells that had a target protein, called CD33, removed, in hopes that immunotherapy targeted against CD33 would kill the cancer and ignore the healthy cells.

“We are encouraged by the results of this study showing that a CD33-deleted stem cell transplant looks very similar to the outcomes of standard stem cell transplantation,” said DiPersio, who also directs WashU Medicine’s Center for Gene and Cellular Immunotherapy. “In the future, we are hopeful we will be able to combine this with CD33-targeted immunotherapies, such as CAR-T cells, and improve treatment options for patients with these very aggressive blood cancers.”

To that end, DiPersio and his collaborators have also published a single case study of a patient with high-risk AML who received a CD33-deleted stem cell transplant and later, upon relapse after the transplant, received a CD33-targeted CAR-T cell therapy, which used T cells from the same donor who provided the stem cell transplant. The patient — who had one of the most aggressive types of AML — achieved complete remission and remains cancer free over one year after receiving the CAR-T cell therapy. The patient also had normal blood cell production return with all blood cells lacking CD33, providing evidence that the genetically engineered donor cells had established themselves in the bone marrow. DiPersio is the senior author of this study, published in October 2025 in JCO Precision Oncology.

Shielding healthy cells

CD33 is an appealing protein to delete from donor stem cells because it is only present on blood-forming cells and not in other tissues, and because there is evidence it is not required for the proper function of blood stem cells, given that individuals born without CD33 have no apparent health problems. After a patient has successfully received this type of stem cell transplant, any remaining cells in the body with CD33 on the surface should, in theory, only be the cancer. Then, CAR-T cells or another immunotherapy designed to target CD33 would kill only the cancer cells and leave healthy donor stem cells untouched.

In this phase 1/2 multicenter clinical trial, 30 adult patients with AML or MDS at high risk of relapse received a stem cell transplant in which CD33 had been removed from the donor cells using CRISPR gene editing technology before the transplant procedure. The CD33-deleted stem cell product is called tremtelectogene empogeditemcel (trem-cel) and was made by Vor Biopharma, which funded the study.

As proof of concept, the patients also received a maintenance therapy that targets CD33, after they underwent the stem cell transplant. While not a CD33-targeted CAR-T cell, the maintenance therapy, called gemtuzumab ozogamicin, is a type of engineered antibody that targets CD33 and carries an anti-cancer drug. Gemtuzumab ozogamicin is approved by the Food and Drug Administration to treat CD33-positive AML and is in clinical trials for CD33-positive MDS. While it helps prevent relapse, the drug’s use is limited because it can cause liver toxicity and damage to blood cells, including dangerously low counts of white blood cells, red blood cells and platelets.

All patients achieved engraftment of their transplanted stem cells by day 28, meaning the cells had gathered in the bone marrow and started working. Some patients met this goal sooner, and platelet production returned by day 16, on average. These timeframes are comparable to those of standard transplanted stem cells.

Average survival was just over 14 months. Nineteen patients received at least one cycle of the antibody maintenance therapy as part of a dose-escalation protocol, and the researchers were able to establish the recommended dose. The researchers found that patients maintained blood cell counts across all doses, suggesting that the gene-edited stem cell transplant protected patients from the dangerously low blood cell counts typically seen during this maintenance therapy following a standard stem cell transplant.

Side effects during the treatment were similar to those of standard transplants, including anemia, low platelets, fever, infections and graft-versus-host disease, in which the donor cells attack the patient’s healthy tissues. Seven patients died during the study, with four due to the cancer progressing and three due to transplant-related causes, including kidney failure, liver toxicity and sepsis.

DiPersio said the results of the study lay the groundwork for developing paired CD33-deleted stem cell transplant and CD33-targeted immunotherapy interventions that avoid destruction of healthy donor cells in the course of cancer treatment.

DiPersio JF, Koehne G, Shah NN, Bernard L, Suh HC, Koura D, Tamari R, Mushtaq MU, Maakaron J, Rimando J, Kennedy VE, Patel SS, Hudson C, Loken M, Stanizzi DA, Lee-Sundlov MM, Thosar S, Mundelboim G, Guo G, Ge HG, Li BE, Xavier-Ferrucio J, Hyzy SL, Lin MI, Raffel GD, Cooper BW. CRISPR−Cas9 CD33-deleted allogeneic hematopoietic cell transplantation with gemtuzumab ozogamicin maintenance in AML: a phase 1/2 trial. Nature Medicine. May 12, 2026. DOI: 10.1038/s41591-026-04362-1.

This work was supported by Vor Biopharma. Several co-authors were employees of the company when the work was conducted.

About WashU Medicine

WashU Medicine is a global leader in academic medicine, including biomedical research, patient care and educational programs with more than 3,000 faculty. Its National Institutes of Health (NIH) research funding portfolio is the second largest among U.S. medical schools and has grown 83% since 2016. Together with institutional investment, WashU Medicine commits well over $1 billion annually to basic and clinical research innovation and training. Its faculty practice is consistently among the top five in the country, with more than 2,000 faculty physicians practicing at 130 locations. WashU Medicine physicians exclusively staff Barnes-Jewish and St. Louis Children’s hospitals — the academic hospitals of BJC HealthCare — and Siteman Cancer Center, a partnership between BJC HealthCare and WashU Medicine and the only National Cancer Institute-designated comprehensive cancer center in Missouri. WashU Medicine physicians also treat patients at BJC’s community hospitals in our region. With a storied history in MD/PhD training, WashU Medicine recently dedicated $100 million to scholarships and curriculum renewal for its medical students, and is home to top-notch training programs in every medical subspecialty as well as physical therapy, occupational therapy, and audiology and communications sciences.

Originally published on the WashU Medicine website

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In patients with an especially aggressive form of glioblastoma, the vaccine caused no serious side effects and prolonged patients’ overall survival compared to historical outcomes after standard-of-care surgery and chemo-radiotherapy. One long-term survivor remains recurrence-free nearly five years later.

The results of the phase 1 trial, conducted at Siteman Cancer Center, based at Barnes-Jewish Hospital and WashU Medicine, were published May 12 in Nature Cancer. The study was led jointly by Mass General Brigham and Geneos Therapeutics, a Philadelphia-based biotechnology company.

“We are extremely encouraged by these results,” said Tanner M. Johanns, MD, PhD, lead author of the study and an assistant professor in the Division of Oncology in the John T. Milliken Department of Medicine at WashU Medicine. “This kind of vaccine is a first for glioblastoma, and it is exciting to think how we can leverage this individualized therapeutic DNA cancer vaccine platform to make a positive impact on the lives of patients who are fighting this disease. Additionally, combination therapies leveraging this personalized platform are currently being investigated at WashU to test if outcomes may be improved further.”

The novel treatment uses engineered DNA molecules designed to stimulate the patient’s immune system against the cancer. Each patient’s tumor has unique proteins specific to that tumor, and this vaccine activates the patient’s immune system to recognize those proteins and eliminate the tumor cells.

Johanns said that although some immunotherapies targeting glioblastoma have shown promise in previous studies, they ultimately are ineffective in significantly delaying or preventing recurrence. That’s likely because glioblastoma can evolve and escape immune attack, but Johanns’ vaccine was designed to help the immune system recognize many different targets on cancer cells. So even if the tumor loses several of these targets, the vaccine is still able to generate responses to many others.

Additionally, glioblastoma is termed a “cold” tumor, meaning that the tumor environment is able to hide from the immune system. The cancer vaccine that was used in this trial, developed by Geneos Therapeutics, transforms cold tumors into “hot” tumors that are then susceptible to immune-mediated eradication. The vaccine is thus able to improve the patient’s immune response by targeting proteins on the cancer cell and by making the environment within the tumor more favorable to immune activation.

“We chose a DNA-based platform because it would allow us an opportunity to target more cancer proteins than any vaccine had targeted before,” said Johanns, who treats patients at Siteman and is a research member there. “Our thinking was that if we could generate a broader range of immune responses against those proteins then it may lead to a more potent vaccine compared to other vaccine platforms with more limited protein targets.”

This DNA-based vaccine platform was able to activate each patient’s immune system to seek out as many as 40 cancer proteins specific to each patient’s tumor — twice as many as had been targeted by any cancer vaccine therapy to date.

More targets, more chances for success

The vaccine in the study, called GNOS-PV01, targets so-called neoantigens — proteins unique to an individual patient’s cancer cells that their immune cells can recognize. The neoantigens were identified and selected using an algorithm developed at WashU Medicine by computational biologists and co-authors Obi Griffith, PhD, a professor of medicine, and Malachi Griffith, PhD, an associate professor of medicine, both in the Division of Oncology and research members at Siteman. Johanns and his colleagues selected neoantigens from different regions of a patient’s tumor, a method they incorporated to further increase the number of cancer cell proteins targeted by the vaccine.

A vaccine platform using a different DNA-based technology developed for breast cancer by co-author William Gillanders, MD, the Mary Culver Distinguished Professor of Surgery at WashU Medicine who treats patients at Siteman, inspired the idea to bring Geneos’ GNOS-PV01 vaccine to WashU Medicine for use against glioblastoma, Johanns said.

The trial enrolled nine adult patients who had been recently diagnosed with glioblastoma. All patients were treated at Siteman Cancer Center. The team prepared a synthetic DNA molecule encoding the unique information for each patient’s tumor neoantigens. The vaccine was manufactured at the Biologic Therapy Core Facility at Siteman during the patient’s post-operative recovery and subsequent radiation treatment.

The vaccine injections started, on average, 10 weeks after the patient’s surgery and were administered every three weeks for a nine-week period, and then every nine weeks thereafter as long as patients were able to participate. All participants, except one who was taking an immune-suppressing steroid, showed an increase in immune-cell activity indicating a response to the vaccine intervention.

Two-thirds of the patients had no progression of their cancer six months out from their surgeries, and two-thirds survived one year. Typically, around 40% of glioblastoma patients reach either milestone.

One-third of the participants were still alive after two years, which is twice the historical survival rate for this patient population. One participant is still alive and recurrence-free today, almost five years after her initial diagnosis.

An investment in the future

Kim Garland is a retired school nurse who lives in Kirkwood, Mo., with Scott, her husband of 31 years. In June 2021, at age 62, Kim was volunteering at a youth camp in Ironton, Mo., when her daughter-in-law, also volunteering at the same camp, noticed that Kim was struggling with confusion and forgetfulness, as well as headaches that would come and go throughout the day.

“I was forgetting things, things that should have been very obvious,” said Kim.

A scan at a local hospital’s emergency room back in St. Louis revealed a 6.5-centimeter mass in Kim’s brain — about the size of a small avocado. Within the week, Albert Kim, MD, PhD, the August A. Busch, Jr. Professor of Neurological Surgery at WashU Medicine, director of the Brain Tumor Center at Siteman, and co-author of the study, performed the initial surgery to remove her tumor. The grim diagnosis of grade 4 glioblastoma came after the tumor was removed.

When offered the opportunity to participate in a clinical trial, Kim Garland agreed in hopes that her participation would improve future treatments. After receiving this prognosis, both Kim and Scott did not expect that she would be alive with no recurrence nearly five years after her initial diagnosis.

“We know we are fortunate to have the kind of care that Kim has been able to receive, just a 30-minute drive from our home,” Scott said. “We see many other patients who are traveling long distances for their treatments. Having this level of care and treatment so close to home has been a huge blessing.”

With the support of their team, the couple have gained the confidence to make longer-term plans, including a long-delayed vacation this summer and spending quality time with their children and 15 grandchildren — a big change from the week-by-week life they were living in the aftermath of Kim’s initial diagnosis.

“Cancer vaccines have a long history, and the development of personalized neoantigen-targeting therapeutic vaccines now represents a highly compelling approach in glioblastoma and in other cancers,” said co-senior author Gavin Dunn, MD, PhD, a neurosurgical oncologist at Mass General Brigham Cancer Institute. “These programs require a high degree of integrated teamwork, and we are fortunate to have collaborated with many dedicated team members in this effort.”

Kim Garland’s cancer, along with those of the other patients in the trial, was an unmethylated MGMT subtype of glioblastoma, which is particularly hard to treat because it is not responsive to available treatment options such as chemotherapy. Johanns said the next step is to assess the vaccine’s efficacy in a larger group of patients, and to expand the treatment to all types of glioblastomas. The goal of Johanns and his team is to improve the vaccine response to ensure that more patients can experience benefits like those experienced by Kim Garland.

The knowledge that their participation in the trial has potentially advanced care is a comfort to the Garlands, who still need to steel themselves before each follow-up appointment, out of concern that Kim’s tumor could yet return.

“What we’re hopeful for is that through research like this, someday, when another person hears the words ‘you have glioblastoma’ as their diagnosis, it will not cause as much anxiety,” said Scott. “Maybe, they will be told ‘this is the cancer you have, but it is very treatable.’ We are fortunate and blessed to be at the right place and at the right time, to be part of this clinical trial and have a small part in the battle against this terrible disease.”

Garfinkle EAR, Perales-Linares R, Gimple RC, Livingstone AJ, Kaleigh F. Roberts KF, Butt OH, Goedegebuure. SP, McLellan MD, Chang GS, Hundal J, Yan J, Navarro JB, Paxton SA, Chattopadhyay S, Cooch N, Perales-Puchalt A, Stavroulaki K, Rochestie S, Peters J, Junker B, Campian JL, Chheda MG, Chicoine MR, Kim AH, Willie JT, Zipfel GJ, Dowling JL, Miller CA, Griffith OL, Griffith M, Gillanders WE, Miller, KE, Mardis ER, Sardesai NY, Dunn GP, Johanns TM. Adjuvant personalized multivalent neoantigen DNA vaccination induces tumor-specific immune responses in newly diagnosed glioblastoma patients. Nature Cancer. May 12, 2026. DOI: 10.1038/s43018-026-01163-w

Funding for this study came from the Mark Foundation for Cancer Research Momentum Fellowship, National Institutes of Health (NIH) National Institute of Neurological Disorders and Stroke (NINDS) grants R01NS117149 and R01 NS107833, the Nationwide Foundation Pediatric Innovation Fund, NIH K12CA167540 and The Alvin J. Siteman Cancer Center Investment Program along with The Foundation for Barnes-Jewish Hospital, NIH NINDS R01NS112712 and The Schnuck Family Fund and The Knight and Christopher Davidson Family Fund. Additional study support for development, manufacture, and administration of the treatment and monitoring of the immune responses was provided by Geneos Therapeutics. The content is solely the responsibility of the authors and does not necessarily represent the official views of the NIH.

R.P.L., J.Y., N.C., A.P.P., S.R., J.P., and N.Y.S are either current or previous Geneos Therapeutics employees.

About WashU Medicine

WashU Medicine is a global leader in academic medicine, including biomedical research, patient care and educational programs with more than 3,000 faculty. Its National Institutes of Health (NIH) research funding portfolio is the second largest among U.S. medical schools and has grown 83% since 2016. Together with institutional investment, WashU Medicine commits well over $1 billion annually to basic and clinical research innovation and training. Its faculty practice is consistently among the top five in the country, with more than 2,000 faculty physicians practicing at 130 locations. WashU Medicine physicians exclusively staff Barnes-Jewish and St. Louis Children’s hospitals — the academic hospitals of BJC HealthCare — and Siteman Cancer Center, a partnership between BJC HealthCare and WashU Medicine and the only National Cancer Institute-designated comprehensive cancer center in Missouri. WashU Medicine physicians also treat patients at BJC’s community hospitals in our region. With a storied history in MD/PhD training, WashU Medicine recently dedicated $100 million to scholarships and curriculum renewal for its medical students, and is home to top-notch training programs in every medical subspecialty as well as physical therapy, occupational therapy, and audiology and communications sciences.

Originally published on the WashU Medicine website

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Compared with students who received a referral, those who were offered the app reported fewer symptoms of mental health problems in follow-up testing six weeks, six months and two years later. They were also more likely to be free of any mental health disorders.

“Universities like WashU already have excellent mental health services, but not all students will take the steps to make an appointment,” said Denise Wilfley, the Scott Rudolph University Professor, a professor of psychiatry, medicine and pediatrics at WashU Medicine, and a professor of psychological and brain sciences in Arts & Sciences, all at WashU. “We were able to offer students an effective resource that they could download on their phones right then and there.”

Wilfley was the senior author of the study published in Nature Human Behavior. Ellen Fitzsimmons-Craft, an associate professor of psychological and brain sciences in WashU Arts & Sciences and an associate professor of psychiatry at WashU Medicine, was a co-first author. Michelle Newman of Penn State and Daniel Eisenberg of the University of California, Los Angeles, were also co-authors. 

The app is designed to deliver a digital version of cognitive behavioral therapy (CBT), a well-established therapeutic approach that aims to identify and change the negative thought and behavior patterns that can drive anxiety, depression and eating disorders.

Responding to prompts, users completed interactive modules where they received psychoeducational content and engaged in exercises to help them learn and practice the content. The coaches could then review their progress and provide personalized responses and feedback. “The coaches help students implement the things they’re learning through the mobile app,” Fitzsimmons-Craft said. “They provide feedback on progress and get students thinking about what they’re doing to achieve positive change.”

The app’s accessibility turned out to be a major advantage. Nearly 75% of students randomly chosen to receive the app used it at least once. In contrast, only 30% of students who received a referral to campus mental health services reported receiving any mental health treatment in the following six months. The accessibility advantage of the app was evident for all student groups, including those from disadvantaged backgrounds and those who generally face greater barriers to care. “Having something right on their phone made a big difference for students,” Wilfley said.

Campus-based counseling services, including those offered at WashU, are still an invaluable resource for students, Wilfley said. “We’re not using digital tools to replace counseling services,” she said. “We’re developing a way to make evidence-based intervention available to as many students as possible. We’re removing barriers to care.”

Unlike some other digital mental health platforms, the app used in the study doesn’t run on artificial intelligence (AI). That’s an important distinction, because generative AI-based therapy remains largely untested and carries certain risks, including the possibility of misinformation and harmful advice. In November 2025, the American Psychological Association recommended against the use of generative AI chatbots and wellness apps as a replacement for standard mental health care.

Artificial intelligence could still be an important tool for addressing mental health concerns on campus. Leading a team that includes Wilfley, Fitzsimmons-Craft is the principal investigator on a five-year $3.7 million grant from the National Institutes of Health (NIH) to develop a self-guided, chatbot-based digital intervention designed to help students with eating disorders. The chatbot uses carefully controlled rules-based AI, not generative AI.

Student mental health should be a top concern for campuses around the country, Fitzsimmons-Craft said. In the current study, nearly half of the 39,194 students who completed initial screening were identified as either having or being at high risk for depression, anxiety or an eating disorder. In addition to the physical and emotional toll, such conditions can make it difficult or impossible for students to succeed academically, she said.

“Many students wait until they reach a crisis point to reach out to the counseling center,” Fitzsimmons-Craft added. “By pairing screening with immediate access to the app, students have an opportunity to take a more proactive approach to their mental health.”

Wilfley, Fitzsimmons-Craft and colleagues are now working to make the app available to all students who are struggling with mental health. “Sometimes evidence-based research can be locked away for many years before it reaches the public,” Wilfley said. “Digital interventions should be available to everybody who needs it. The fact that this study started with large-scale screening on college campuses shows the potential for reaching large populations. ”

Given the prevalence of mental health disorders on campuses across the country, it would make sense for colleges and universities to screen all incoming freshmen for anxiety, depression and eating disorders, Wilfley said.  

This work demonstrates that the combination of population-based mental health screening and digital interventions can not only reduce psychiatric symptoms and improve quality of life but also prevent psychiatric disorders. “This approach can simultaneously reduce the prevalence of mental disorders, expand equitable access to care and improve affected individuals’ symptoms,” Wilfley said.

“WashU already has a program that promotes awareness about alcohol use disorders, which, of course, is an extremely important issue,” Wilfley said. “But universities could also take a more proactive approach to mental health.”

Newman, MG, Fitzsimmons-Craft EE, Baik SY. et al. Population-based RCT of a digital cognitive-behavioural guided self-help intervention for anxiety, depression and eating disorders in college students. Nat Hum Behav (2026) DOI: https://doi.org/10.1038/s41562-026-02454-z

This study was supported by the National Institute of Mental Health (NIMH) (grant R01 MH115128, M.G.N., E.E.F.-C., D.E., C.B.T. and D.E.W., and NIMH grant K08 MH12034, E.E.F.-C.). This manuscript is the result of funding in whole or in part by the National Institutes of Health (NIH).

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Led by first author Ning Shen, PhD, a staff scientist; Daniel Kerschensteiner, MD, the Bernard Becker Professor of Ophthalmology and Visual Sciences; and Philip Ruzycki, PhD, an assistant professor, the researchers performed a genome-wide CRISPR screen to look for genes whose loss worsened retinal degeneration in a mouse model of retinitis pigmentosa. They then developed and tested two gene therapies to boost the expression of the identified candidate genes — UFD1 and UXT — to promote the clearing of toxic proteins. Injection of either gene therapy to the eyes of mice with retinitis pigmentosa reduced photoreceptor death, maintained the light-sensing capabilities of the retina and preserved vision.  

Likewise, delivery of either gene therapy to a human retinitis pigmentosa model developed by the Bright Center for Human Vision at WashU Medicine — using human donor retinas cultured in a dish — preserved photoreceptors, demonstrating strong therapeutic promise. 

“This study bridges a critical bottleneck in translating laboratory-based findings to the clinic by leveraging both mouse and human models of retinitis pigmentosa to develop and test human-compatible gene therapies,” said senior author Kerschensteiner, who also leads the Bright Center for Human Vision. The genome-wide screen results, shared as a public resource, point to additional candidate genes for future therapeutic development to preserve vision. 

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The newly discovered process appears to protect liver cells from the toxic effects of the buildup of misfolded, aggregation-prone proteins inside those cells. The findings could help clarify the wide variation in disease severity sometimes seen among patients with alpha1-antitrypsin deficiency and inform new approaches to predicting which patients are at highest risk of eventually needing a liver transplant.

The study appears online in the journal Nature Communications.

Cells have tightly controlled processes to make all the proteins necessary for healthy functioning. There are processes for manufacturing proteins, for folding them into their proper shapes, for transporting them to the correct parts of the cell, and for degrading and disposing of them when they’re no longer needed. Because disruptions in any part of this process — called proteostasis — can lead to disease, cells also have safety measures to handle mistakes that crop up along the way.

“What is truly remarkable about proteostasis is that it’s set up to have multiple fail-safes for handling a bad protein,” said senior author David H. Perlmutter, MD, executive vice chancellor for medical affairs, the Spencer T. and Ann W. Olin Distinguished Professor and the George and Carol Bauer Dean of WashU Medicine. “That’s good for cellular economy because it means the cell doesn’t have to spend all its energy on making every protein perfectly. Our study identifies a completely new way that cells manage potentially harmful proteins.”

Perlmutter and his colleagues dubbed the newly identified fail-safe the polymerized protein response. The new study suggests that this response allows cells to tolerate misfolded proteins that also polymerize and aggregate, maintaining normal cell function despite their presence. It complements a separate, well-characterized quality-control process, the unfolded protein response, that governs how cells handle unfolded proteins.

Both unfolded proteins and aggregated proteins can accumulate in a cell’s endoplasmic reticulum — the protein manufacturing, packaging and shipping center of cells. And the new study now shows that cells have different processes for handling each.

Studying human cell lines and mouse models of alpha1-antitrypsin deficiency, Perlmutter and his colleagues showed that aggregated proteins in the endoplasmic reticulum of liver cells trigger the polymerized protein response through a molecule called Derlin-2, which then activates an important molecule called an NF-kappa-B p50 homodimer. These interactions set in motion a genetic program that protects the cell. Perlmutter and his team are continuing this research to work out the details of that protection.

Perlmutter said the identification of this new process could help researchers design ways to identify which patients with alpha1-antitrypsin deficiency are at highest risk of developing severe liver disease and will likely require a liver transplant. Early identification — before the liver damage becomes apparent — could help guide treatment decisions and reveal potential routes to new therapies and prevention strategies.

“We think the polymerized protein response is a cellular adaptation that protects most patients from liver damage due to these aggregated proteins building up in their liver cells,” Perlmutter said. “It allows the cells to be healthy despite the presence of the proteins. As long as this signal is present, the cells are able to handle the protein load.”

Importantly, the study found that the polymerized protein response could apply to aggregated proteins in diseases beyond alpha1-antitrypsin deficiency. Their experiments showed that the response can be triggered in a rare age-dependent dementia, inherited diabetes insipidus and amyotrophic lateral sclerosis (ALS) because each of these is caused by a genetic variant that aberrantly polymerizes in the endoplasmic reticulum of the cells in which they are made.

“We are continuing to investigate the molecular details of the polymerized protein response and how it plays a role in a host of diseases caused by aggregation-prone proteins, in addition to alpha-1 antitrypsin deficiency,” Perlmutter said.

Munanairi A, Rudnick DA, Huang J, Perlmutter DH. The polymerized protein response (PPR) is activated by genetic variants that polymerize in the ER and is mediated by NFkappaBp50. Nature Communications. April 27, 2026. DOI: 10.1038/s41467-026-72369-w.

This work was supported by the National Institutes of Health (NIH) through U.S. Public Health Service grants P01-DK096990 and R01-DK131215. Additional support was provided by the Genome Engineering and Stem Cell Center at Washington University in St. Louis; the Genome Technology Access Center at WashU Medicine; researchers at Washington University School of Medicine in St. Louis; and researchers at the University of Basel, Switzerland. This content is solely the responsibility of the authors and does not necessarily represent the official view of the NIH.

About WashU Medicine

WashU Medicine is a global leader in academic medicine, including biomedical research, patient care and educational programs with more than 3,000 faculty. Its National Institutes of Health (NIH) research funding portfolio is the second largest among U.S. medical schools and has grown 83% since 2016. Together with institutional investment, WashU Medicine commits well over $1 billion annually to basic and clinical research innovation and training. Its faculty practice is consistently among the top five in the country, with more than 2,000 faculty physicians practicing at 130 locations. WashU Medicine physicians exclusively staff Barnes-Jewish and St. Louis Children’s hospitals — the academic hospitals of BJC HealthCare — and Siteman Cancer Center, a partnership between BJC HealthCare and WashU Medicine and the only National Cancer Institute-designated comprehensive cancer center in Missouri. WashU Medicine physicians also treat patients at BJC’s community hospitals in our region. With a storied history in MD/PhD training, WashU Medicine recently dedicated $100 million to scholarships and curriculum renewal for its medical students, and is home to top-notch training programs in every medical subspecialty as well as physical therapy, occupational therapy, and audiology and communications sciences.

Originally published on the WashU Medicine website

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He was installed by Chancellor Andrew D. Martin and David H. Perlmutter, MD, executive vice chancellor for medical affairs, the Spencer T. and Ann W. Olin Distinguished Professor and the George and Carol Bauer Dean of the School of Medicine. The professorship is designated for the head of ophthalmology department and was established by Arthur W. Stickle, MD, a pediatric ophthalmologist and former WashU Medicine clinical faculty member, and his wife, Emily.

“The Stickles’ generosity creates a lasting legacy that will advance vision research and patient care,” Martin said. “Dr. Lee’s work at the intersection of artificial intelligence and ophthalmology exemplifies the kind of innovation that defines WashU Medicine and that will benefit patients here in St. Louis and far beyond.”

Lee served on the faculty at the University of Washington in Seattle for a decade before being recruited to WashU Medicine in 2025 to lead the ophthalmology department.

“We are fortunate to recognize Dr. Lee for his research and leadership in artificial intelligence,” Perlmutter said. “His recruitment signals our clear commitment to lead nationally and globally in applying artificial intelligence across all areas of medicine. We are taking our expertise in genomics and informatics and integrating it with AI technologies to further advance personalized medicine and have an enormous impact on the way we diagnose disease and care for patients.”

Early in his research career, Lee leveraged his medical training and computer programming expertise to assemble a pioneering team of machine learning scientists. His team was among the first to successfully apply deep learning techniques to vision science, helping to establish a new field of research in ophthalmic imaging.

His work has led to many innovations, including improving the ability of AI to assess the severity of age-related macular degeneration — a common retinal disease — based on retinal images. His work also has helped develop a novel severity score for macular telangiectasia type 2, a rare retinal disease that causes a gradual loss of vision. In addition, he has created foundational AI models that can be trained for various types of retinal imaging without a lot of additional data.

In recent work, he has used advanced data-driven methods to identify genetic factors influencing the formation of the macula, the central part of the retina. Together, these advances have greatly improved imaging techniques used to detect retinal diseases and are helping physicians identify disease earlier and tailor treatment more precisely.

Lee chairs the Digital Imaging and Communications in Medicine (DICOM) working group, which establishes standards for ocular imaging across the medical device industry. He also co-chairs the Ryan Initiative for Macular Research Age-Related Macular Degeneration Consortium, a nonprofit collaboration uniting life sciences companies, device manufacturers and academic groups to study age-related macular degeneration.

Lee has worked to create an ecosystem for the entire lifecycle of medical AI devices. This includes collaborating with the U.S. Food and Drug Administration to develop policies for regulating machine learning tools, developing report guidelines for AI studies and running the first comprehensive multicenter study of autonomous AI devices for screening of diabetic eye disease.

With his wife and scientific partner, Cecelia Lee, MD, the Jane Hardesty Poole Distinguished Professor at WashU Medicine, he leads the National Institutes of Health (NIH)’s Bridge2AI Common Fund Data Generation Project, a four-year, $32 million effort to develop a flagship dataset from 4,000 participants for AI research in type 2 diabetes.

At the national level, he also chairs the Intelligent Research in Sight Registry Academic Consortium and the Steering Committee on Information Technology of the American Academy of Ophthalmology, the latter of which has integrated big data and AI concepts into the core curriculum of ophthalmology residency programs nationwide.

After completing his undergraduate studies at Harvard University, Lee earned his medical degree from WashU Medicine in 2009 and was among the first WashU medical students to complete the school’s newly established Master of Science in Clinical Investigation, which prepares investigators for academic careers in clinical research. He completed his residency training at WashU Medicine and Barnes-Jewish Hospital and a medical retina fellowship at Moorfields Eye Hospital in London, followed by a surgical retina fellowship the University of British Columbia in Vancouver. He joined the University of Washington faculty in 2015, becoming a full professor in 2024, before being recruited to lead the WashU Medicine ophthalmology department faculty.

About Emily and Arthur W. Stickle

Arthur Stickle, MD, came to WashU Medicine in 1943 to study ocular motility under the renowned late Richard Scobee, MD, and to complete his residency after graduating from the University of Oklahoma medical school. He later joined the clinical faculty as an assistant clinical professor of pediatrics and founded the St. Louis Eye Clinic, which eventually expanded to seven satellite offices across the metropolitan area.

Arthur Stickle became known nationally as a leading expert in the treatment of strabismus — the misalignment of the eyes — and is credited with developing new surgical treatments for the condition. He enjoyed teaching residents and played a pivotal role in the development of strabismus specialists in St. Louis. He served on the board of the American Association of Certified Orthoptists for many years. He retired in 1992 and died in 2010 at the age of 91.

Emily Stickle was a volunteer in several St. Louis organizations, including Volunteer Service Council, Girls Inc., Saint Louis Symphony Orchestra and Dance St. Louis. She died in 2023.

In 1995, the Stickles endowed the Arthur W. Stickle, MD, Lecture in Pediatric Ophthalmology, the first annual lecture created by a former member of the department.

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