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Turmoil at US health agencies

Last Friday night, Peter Marks, the Director of the Center for Biologics Evaluation and Research at the FDA resigned from his position. Marks, who was the top vaccine regulator at the FDA, wrote in his resignation letter  that he had been willing to work with US Health and Human Services (HHS) Secretary, Robert F. Kennedy Jr.’s “concerns regarding vaccine safety and transparency.” However, he continued, “it has become clear that truth and transparency are not desired by the Secretary, but rather he wishes subservient confirmation of his misinformation and lies.” Following Marks’ departure, biotech stocks plummeted, and John Crowley, the President and CEO of the Biotechnology Innovation Organization which is an advocacy organization for the biotechnology industry, said in a statement, “We are deeply concerned that the loss of experienced leadership at the FDA will erode scientific standards and broadly impact the development of new, transformative therapies to fight diseases for the American people.”

On Tuesday, Kennedy then followed through on his announced plans for massive layoffs at the FDA, CDC, and NIH. Cuts affected leaders of multiple departments at the NIH, GEN  reported. At the FDA, scientists studying bird flu, contaminants in the food supply, and antimicrobial resistance were let go, STAT  reported. Researchers at the CDC working in reproductive health, birth defects, and substance abuse were fired as well as people aiming to prevent tuberculosis and HIV, STAT  found.

About the layoffs, FDA Commissioner during Donald Trump’s first term, Scott Gottlieb, wrote on X, “Today, the cumulative barrage on that drug-discovery enterprise, threatens to swiftly bring back those frustrating delays for American consumers, particularly affecting rare diseases and areas of significant unmet medical need.”

Former two-time FDA Commissioner, Robert Califf wrote on LinkedIn, “The FDA as we've known it is finished, with most of the leaders with institutional knowledge and a deep understanding of product development and safety no longer employed.” He continued, “It will be interesting to hear from the new leadership how they plan to put ‘Humpty Dumpty’ back together again.”

Biotech stocks continued to fall in reaction to the layoffs. Republican senator from Louisiana and chair of the Senate health committee, Bill Cassidy, has asked Kennedy to testify before the panel about his overhaul of HHS on April 10, 2025. – Stephanie DeMarco

A new blood test for Alzheimer’s disease

Researchers have developed a new blood test to help diagnose Alzheimer’s disease (AD) and monitor its progression in patients with mild cognitive impairment (MCI) (1). The test detects eMTBR-tau243, a fragment of the tau protein, which, along with amyloid beta, is considered a pathological marker of AD and closely correlates with its clinical symptoms. In a study involving 902 participants, the researchers found that individuals with MCI had elevated blood levels of eMTBR-tau243 compared to cognitively healthy controls, and those with AD-related dementia had even higher levels. However, the test did not detect eMTBR-tau243 in patients with cognitive impairments not associated with AD. The new biomarker could serve as a valuable tool for diagnosing AD, monitoring tau accumulation, and evaluating the effectiveness of tau-targeted treatments in clinical trials. – Dika Ojiakor

CDC withholds the release of measles forecast report

Leaders at the CDC blocked the release of an expert assessment that warned of a high risk of measles in areas with low vaccination rates, according to an article by ProPublica. The withheld report would have emphasized the importance of vaccination in preventing the spread of the highly contagious disease, which has now affected 19 US states. The move seems to signal a shift in how the agency is responding to pressure from Robert F. Kennedy Jr., a vaccine skeptic and the current US Secretary of HHS. The CDC continues to recommend that most people receive two doses of the measles, mumps, and rubella (MMR) vaccine, which is widely available at travel clinics and pharmacies across the US. – Dika Ojiakor

First siRNA drug for hemophilia

The FDA approved Qfitlia from Sanofi and Alnylam to treat patients 12 years and older with hemophilia A or B with or without neutralizing antibodies to factor VIII or IX. Qfitlia is the first small interfering RNA (siRNA) therapeutic for hemophilia and the first drug that works by reducing the antithrombin protein, which inhibits blood clotting. Rather than being used during a bleeding episode on-demand, Qfitlia is injected subcutaneously every two months to prevent bleeding episodes. The approval is based on two clinical trials with 177 patients, which showed that Qfitlia reduced the annualized bleeding rate by around 70 percent.  – Allison Whitten

A novel target to prevent pancreatic cancer

Pancreatic cancer remains one of the most lethal cancers, with only around 10 percent of patients surviving five years after their diagnosis. Precancerous pancreatic lesions often do not progress to cancer, but when they do, scientists have shown that Kirsten rat sarcoma virus (KRAS) mutations are frequently to blame. Now, researchers at Cold Spring Harbor Laboratory revealed that the fibroblast growth factor receptor (FGFR2) gene also contributes to pancreatic tumorigenesis by enhancing KRAS signaling in mice (2). The scientists showed that combining FGFR2 inhibitors with epidermal growth factor receptor (EGFR) inhibitors — both of which are already approved to treat other cancers — reduced the formation of precancerous lesions, suggesting that these inhibitors could be tested in a clinical trial in patients at risk of developing pancreatic cancer.  – Allison Whitten

A starfish for a heart disease diagnostic

From models of neural regeneration to an actual model for a wearable heart disease monitor, starfish, it seems, can do it all. In a new study, biomedical engineers at the University of Missouri designed a wearable sensor that measures both mechanical and electrical signals from a person’s heart while they’re moving — a difficult feat (3). The device has five flexible arms like a starfish that can shrink and extend as the wearer moves. The team trained a machine learning model to filter out movement-related noise and to diagnose atrial fibrillation, myocardial infarction, and heart failure with 91 percent accuracy. – Stephanie DeMarco

References

1. Horie, K. et al. Plasma MTBR-tau243 biomarker identifies tau tangle pathology in Alzheimer’s disease. Nat Med (2025).
2. Tonelli, C. et al. FGFR2 Abrogation Intercepts Pancreatic Ductal Adenocarcinoma Development. Cancer Res (2025).
3. Chen, S. et al. Starfish-inspired wearable bioelectronic systems for physiological signal monitoring during motion and real-time heart disease diagnosis. Sci Adv  11, eadv2406 (2025).