Eugene Braunwald, Whose Research Reshaped Cardiology, Dies at 96
Jonathan Kandell / nytimes - His work changed how doctors understood heart attacks, heart failure and coronary artery disease, and helped lead to therapies that saved millions of lives.
AI Summary: Eugene Braunwald, a towering figure whose research reshaped modern cardiology, has died at 96. Colleagues remember his seminal contributions to cardiac physiology, therapeutics, and clinical practice that set the stage for decades of advances. His passing marks the end of an era for a field he helped steer.
WHO approves first malaria treatment for infants
medicalxpress - The World Health Organization announced Friday that it had given prequalification approval to a malaria treatment for newborns and infants for the first time.
AI Summary: The World Health Organization has cleared the first malaria treatment specifically for infants, granting prequalification that paves the way for broader procurement and use in endemic countries. Regulators' sign-off targets a vulnerable age group long underserved by effective pediatric therapies, potentially speeding distribution through global health channels and donor programs.
Distribution of Phase I lung cancer trials may be consolidating at top-performing US sites
medicalxpress - Between 2020 and 2024, the number of unique sites in the United States where phase I clinical trials for non-small cell lung cancer (NSCLC) were conducted decreased by 44% and became increasingly concentrated at the top 20 highest-volume clinical trial si…
AI Summary: A new analysis shows Phase I lung cancer trials are becoming concentrated at a relatively small number of leading U.S. centers, raising concerns about geographic inequity in access to early‑phase studies. The trend could speed drug development at elite sites while leaving patients in other regions with fewer experimental options and longer travel burdens.
- Conference highlights, care models and awareness (4)
- Consolidation at top U.S. Phase I centers (3)
- Early-phase trials and next-gen therapies (4)
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Conference highlights, care models and awareness
Consolidation at top U.S. Phase I centers
Early-phase trials and next-gen therapies
All Other Stories
FDA backs 3 psychedelic drug studies for mental illness
Ella Ruder / beckershospitalreview - The FDA is issuing national priority vouchers to three companies studying psychedelic drugs to treat serious mental illness. The vouchers were issued to companies studying psilocybin for treatment-resistant depression and major depressive disorder as well…
AI Summary: The FDA announced a targeted push to accelerate clinical research into psychedelics for mental illness, issuing three commissioner vouchers to support rigorous trials of psychedelic therapies. The move aims to fast-track evidence generation under regulatory oversight, signalling growing agency willingness to explore unconventional treatments while keeping safety and trial standards squarely in view.
- FDA voucher push and federal policy shift (5)
- Psilocybin and brain science (4)
- Real-world use, safety and clinical readiness (3)
- All Other Stories
FDA voucher push and federal policy shift
Psilocybin and brain science
Real-world use, safety and clinical readiness
All Other Stories
Gene therapy improves hearing in 90% of patients with inherited deafness in largest trial of its kind
livescience - A new gene therapy tested in China has improved the hearing of 38 people who were born deaf due to mutations in a gene called OTOF.
AI Summary: A gene therapy for inherited deafness delivered dramatic results, restoring hearing in roughly 90% of treated patients in the largest trial of its kind. Investigators report durable improvements over follow-up, signaling a potential one-time intervention for certain genetic deafness types and challenging the notion that auditory loss is always irreversible. Hope, meet hard data.
- FDA approves first-ever gene therapy for inherited hearing loss (6)
- Primate study finds human-like genetic cause of blindness (1)
- Trial shows durable hearing restored in most patients (3)
FDA approves first-ever gene therapy for inherited hearing loss
Primate study finds human-like genetic cause of blindness
Trial shows durable hearing restored in most patients
FDA approves 1st 2-drug HIV treatment
Ella Jeffries / beckershospitalreview - The FDA has approved Merck’s once-daily, two-drug regimen for adults with virologically suppressed HIV-1. The treatment combines 100 mg doravirine and 0.25 mg islatravir and is indicated for patients with no history of virologic treatment failure and no k…
AI Summary: The FDA has approved Merck’s once‑daily two‑drug antiretroviral regimen, marking a notable market entrant poised to compete with established single‑pill therapies. Regulators cleared the novel combination on efficacy and safety data, setting up potential shifts in prescribing, pricing and competition — and giving Gilead something new to grumble about.
Moderna, after losing US funding, rebounds to start mRNA bird flu vaccine trial
Kristin Jensen / healthcaredive - A program that got caught up in HHS’ decision to abandon mRNA research was revived by a public-private coalition and is now beginning a large, late-stage test that could support a future approval.
AI Summary: Moderna has initiated a clinical trial of an mRNA vaccine targeting a potentially pandemic bird‑flu strain, joining global efforts to have a rapid‑response countermeasure ready. Regulators and public‑health experts hail the move as sensible pre‑emptive science—because nothing says “we planned for this” like testing the vaccine before the outbreak arrives.
CMS delays Part D GLP-1 model amid skepticism from insurers
fiercehealthcare - The Trump administration is delaying a voluntary model that aimed to expand access to GLP-1s in Part D after pushback from insurers.
AI Summary: Federal regulators have paused a Medicare Part D pilot to expand coverage for GLP‑1 weight‑loss drugs amid payer skepticism and implementation concerns. The delay reflects worries about cost, program design and insurer buy‑in, forcing policymakers to revisit the model while patients and providers wait for clarity on whether Medicare will shoulder these high‑price therapies.
- CMS pauses Medicare GLP‑1 BALANCE pilot amid insurer pushback (4)
- Researchers chase GLP‑1 benefits, from gene therapy to Alzheimer’s (6)
- Telehealth and clinics scale GLP‑1 access, delivery and monitoring (3)
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CMS pauses Medicare GLP‑1 BALANCE pilot amid insurer pushback
Researchers chase GLP‑1 benefits, from gene therapy to Alzheimer’s
Telehealth and clinics scale GLP‑1 access, delivery and monitoring
All Other Stories
340B drug discounts are drifting from patients to profit, and reform is now on the table
medicalxpress - The 340B Drug Pricing Program must be reformed to better patient health and disincentivize institutional profit-seeking behaviors, says the American College of Physicians (ACP). In a new policy, "Reforming 340B to Promote Program Integrity and Better Serv…
AI Summary: The 340B drug-discount program is under renewed scrutiny after analyses and advocacy groups argue discounts intended to help patients are instead boosting institutional margins. Hospitals, provider groups and the AHA are contesting HRSA proposals and court rulings, sparking policy debates and potential regulatory fixes to curb markups and steer savings back to vulnerable patients.
RFK Jr. says China is 'eating our lunch' in biotech advances
Zachary Brennan / endpoints - China is "eating our lunch" on new drug approvals and clinical trial starts, HHS Secretary Robert F. Kennedy Jr. told Congress Tuesday, while praising the FDA's actions so far. "We are losing scientists, we're losing ...
AI Summary: Robert F. Kennedy Jr. publicly criticized U.S. biotech competitiveness—singling out China as gaining ground—and declined to fully endorse the new CDC vaccine director, blending industry critique with public‑health ambivalence. His comments underscore tensions between political positioning and health policy messaging while rattling stakeholders who prefer facts over theatrical proclamations.
- Kennedy defensive in hearings, balancing White House and base (4)
- Kennedy hesitates on CDC director, vaccine reports spark debate (5)
- On Pharma frontlines: Kennedy warns China is eating our lunch (3)
- All Other Stories
Kennedy defensive in hearings, balancing White House and base
Kennedy hesitates on CDC director, vaccine reports spark debate
On Pharma frontlines: Kennedy warns China is eating our lunch
All Other Stories
Ciltacabtagene autoleucel in high-risk smoldering multiple myeloma: the CAR-PRISM phase 2 trial
Omar Nadeem / nature - Nature Medicine, Published online: 20 April 2026; doi:10.1038/s41591-026-04365-yAs presented at the 2026 AACR Annual Meeting, in a phase 2 trial, treatment of patients with high-risk smoldering multiple myeloma with BCMA-targeting CAR T cell therapy cilta…
AI Summary: The CAR-PRISM phase 2 study reports that ciltacabtagene autoleucel, a BCMA-directed CAR T therapy, produced meaningful responses in patients with high‑risk smoldering multiple myeloma, suggesting potential to delay progression to symptomatic disease and defer immediate treatment. Early results show promising efficacy with manageable toxicities, hinting at a shift toward treating disease earlier rather than later.
- CAR-PRISM results: compelling MRD negativity, potential treatment shift (4)
- Experts weigh in: praise, safety concerns, and clinical enthusiasm (4)
- Policy and IO context: regulation, immune engineering, industry implications (3)
- All Other Stories
CAR-PRISM results: compelling MRD negativity, potential treatment shift
Experts weigh in: praise, safety concerns, and clinical enthusiasm
Policy and IO context: regulation, immune engineering, industry implications
All Other Stories
Long-term Use of Immunotherapy May Be Safe for Patients With Alveolar Soft Part Sarcoma
Kathleen Medora / aacr - SAN DIEGO – Long-term adverse events were rare and manageable among patients with alveolar soft part sarcoma (ASPS), which primarily affects adolescents and young adults, who received immunotherapy beyond the standard two years, according to results from …
AI Summary: Observational data indicate extended checkpoint inhibitor therapy can be tolerated by patients with alveolar soft part sarcoma and may provide sustained disease control for many. The findings support considering prolonged treatment in select cases, while underscoring the need for vigilant monitoring for late toxicities and randomized trials to confirm benefit.
Zoldonrasib Shows Early Strength in Previously Treated KRAS G12D NSCLC
oncodaily - KRAS G12D has long been one of the most frustrating targets in thoracic oncology. It is biologically important, clearly druggable in theory, and yet patients with KRAS G12D-mutant non-small cell […]
AI Summary: Investigational KRAS(ON) inhibitor zoldonrasib produced effective, durable responses in patients with advanced KRAS G12D–mutated non‑small‑cell lung cancer after prior therapy. Early‑phase data show meaningful tumour shrinkage and a manageable safety profile, offering a targeted option for a mutation long considered untargetable and breathing new life into KRAS drug development.
- Experts on site: RAS inhibitors reshape NSCLC treatment landscape (5)
- Industry moves: BridgeBio reshuffles as KRAS race heats up (1)
- On the AACR floor: zoldonrasib delivers durable G12D responses (4)
- Perioperative and biomarker research reshapes precision care in NSCLC (4)
- All Other Stories
Experts on site: RAS inhibitors reshape NSCLC treatment landscape
Industry moves: BridgeBio reshuffles as KRAS race heats up
On the AACR floor: zoldonrasib delivers durable G12D responses
Perioperative and biomarker research reshapes precision care in NSCLC
All Other Stories
Three gene therapy pioneers just won the Breakthrough Prize. This is their story
Ryan Cross / endpoints - A trio of scientists behind the first gene therapy approved in the United States have been awarded a Breakthrough Prize in Life Sciences. It’s arguably one of the most prominent recognitions yet for the field ...
AI Summary: Three gene‑therapy researchers received the Breakthrough Prize in recognition of foundational work that enabled the first CRISPR-based sickle cell therapy. The award highlights decades of basic and translational research that turned genome editing from lab curiosity into a tangible clinical remedy, with winners sharing a multimillion‑dollar prize and public acclaim.
First-Line Zongertinib in Advanced HER2-Mutant NSCLC: A New Targeted Standard Begins to Take Shape
oncodaily - For years, HER2-mutant non-small-cell lung cancer sat in an uncomfortable place in thoracic oncology. It was clearly an oncogene-driven disease, but unlike EGFR-mutant or ALK-rearranged NSCLC, it did not have […]
AI Summary: Clinical trial data show zongertinib delivers strong antitumor activity as a first‑line treatment for patients with advanced HER2‑mutant non‑small cell lung cancer, positioning the drug as a potential new targeted standard. Robust response rates in previously untreated patients are prompting clinicians to rethink initial treatment choices while attention turns to long‑term safety and access.
FDA updates mifepristone safety study status
Ella Jeffries / beckershospitalreview - The FDA updated its mifepristone guidance, detailing ongoing work on a safety study and the current status of the drug’s Risk Evaluation and Mitigation Strategy program. According to an April 8 news release, the agency said it is still collecting and eval…
AI Summary: The FDA updated the safety review status for mifepristone, yet the regulatory change hasn’t translated into widespread retail availability. Many community pharmacies are still hesitant to dispense the drug due to logistical, legal and reputational concerns, leaving access limited despite federal moves intended to broaden distribution — so yes, policy changed, but practice lagged.
First-in-human trial primes immune system to accept donor livers
medicalxpress - UPMC and University of Pittsburgh clinician-scientists have weaned and kept multiple liver transplantation patients off of all immunosuppressant drugs for more than three years through a first-in-human clinical trial of a unique "immune priming" therapy.
AI Summary: A first‑in‑human study reports a novel immune‑priming approach that enabled three liver transplant recipients to stop standard lifelong immunosuppression. The intervention appears to induce tolerance to donor tissue, reducing dependence on anti‑rejection drugs and their toxic side effects, offering a potential shift in transplant management if larger trials confirm these early results.
Eli Lilly Buys Startup CrossBridge Bio to Bring a More Powerful Strike to Tumors
Frank Vinluan / medcitynews - Eli Lilly is paying up to $300 million for CrossBridge Bio, a startup developing antibody drug conjugates that deliver two drug payloads to cancers. Beyond potentially better efficacy, CrossBio’s dual approach could also fight drug resistance. The post El…
AI Summary: Eli Lilly agreed to acquire CrossBridge Bio for up to $300 million, adding small‑molecule oncology assets to its pipeline. The deal accelerates Lilly’s tumor‑targeting strategy, reflects Big Pharma’s preference for buying nimble biotech innovation, and highlights ongoing consolidation trends that determine where promising oncology programs ultimately end up.
ESMO 2026 Guideline: Redefining the Management of Metastatic Colorectal Cancer
oncodaily - Metastatic colorectal cancer (mCRC) represents a complex and biologically diverse disease in which treatment decisions increasingly depend on molecular characteristics, disease burden, and patient-specific factors. While therapeutic options have expanded …
AI Summary: The European Society for Medical Oncology released updated clinical practice guidelines for metastatic colorectal cancer, redefining diagnostic pathways, systemic therapy sequencing, and follow‑up standards. The guidance incorporates recent targeted and immunotherapy evidence to recommend practical treatment algorithms, prompting oncology centers to update protocols and payers to reassess coverage as clinicians translate recommendations into routine care.
FDA approves Travere's Filspari as first drug for the kidney disease called FSGS
Nicole DeFeudis / endpoints - The FDA expanded the label for Filspari on Monday to add another kidney condition. The drug is now the first therapy approved in the US for focal segmental glomerulosclerosis (FSGS). The pill may be taken ...
AI Summary: The FDA approved Filspari for focal segmental glomerulosclerosis (FSGS), delivering the first specifically authorized treatment for this rare kidney disease. The approval provides a targeted therapeutic option for patients and marks a commercial milestone for Travere, raising hopes for better outcomes while spotlighting questions about access, pricing, and long‑term real‑world effectiveness.