NATIONAL INSTITUTES OF HEALTH

Updated LITESPARK-004 trial findings in The Lancet Oncology show belzutifan's sustained efficacy over 3 years in treating VHL disease–associated CNS hemangioblastomas, with 44% objective response and 46% stable disease. Grade ≥ 3 adverse events occurred in 46% of patients.

Scientists have made breakthroughs in regrowing damaged neural connections, leading to promising clinical trials for spinal cord injury patients. However, recent tax law and patent regulation changes threaten to derail these treatments, potentially stifling medical innovation. A bipartisan bill aims to repeal harmful Section 174 changes, emphasizing the need for policies that support innovation.

Black patients' decisions on cancer trial participation may be influenced by spiritual beliefs and historical distrust in clinical research, according to surveys at Baltimore medical centers. Despite trust in their medical teams, lingering distrust in research persists, stemming from historical unethical practices. Researchers suggest addressing these factors to improve recruitment and ensure trials reflect diverse populations.

Capricor Therapeutics signed a deal with Nippon Shinyaku for European commercialization of deramiocel, a DMD treatment, receiving $20 million upfront and potential $715 million in milestones. The partnership aims to bring deramiocel to market, addressing a disease with no cure, and extends Capricor's cash runway into 2026.

Case Western Reserve University researchers, with a $3.3M NIH grant, study if brain proteins like Eph receptors contribute to Alzheimer's, focusing on the blood-brain barrier's role. Collaborating with Texas Tech, UT, and Memorial Sloan Kettering, they aim to understand these proteins' functions in disease development.

A study by NIH's RECOVER Initiative and NYU Langone Health found no reliable biomarkers in routine clinical lab tests for diagnosing long COVID, highlighting the need for further research beyond standard methods.

Dr. Swee Lay Thein and Professor Stuart Orkin, both Shaw Laureates in Life Science and Medicine 2024, focus on BCL11A and fetal hemoglobin reactivation as therapeutic strategies, respectively. Their work at the National Institutes of Health and Harvard Medical School aims to advance sickle cell disease treatment.

The overturning of Roe v. Wade and subsequent abortion bans have led to IVF suspensions and mistrust in medical data sharing, potentially hindering clinical research on female reproductive health and exacerbating gender disparities in clinical trial participation.

The Institute of Herbal Medicine at UP Manila developed an ampalaya tablet to combat type 2 diabetes, proven effective in reducing blood sugar levels through clinical trials. This herbal medicine, distinct from supplements, offers a natural, safe alternative for diabetes management.

The Early Tracking of Childhood Health Determinants (ETCHED) study, conducted by NIDDK's PECRB, began enrolling pregnant women (aged 18+, AI/AN or Hispanic ethnicity) in April 2022 at VHMC, Phoenix. The study aims to follow 750 mothers and their offspring for 23 years to investigate maternal risk factors' impact on offspring's birth weight, weight, and adiposity. Secondary objectives include studying maternal factors' role in child growth, neurodevelopment, and gut microbiome changes. Data collection includes demographics, medical history, and biospecimens. The study is approved by NIH IRB and involves extensive community outreach.