MASSACHUSETTS GENERAL HOSPITAL

A $75.4 million NIH grant enables a UCSF-led clinical trial testing three drugs for progressive supranuclear palsy (PSP), aiming to slow its progression. The trial, involving up to 50 sites, uses a platform model to quickly identify effective therapies, prioritizing enrollment of diverse populations. CurePSP collaborates in recruitment, offering hope for patients and families.

New research shows low doses of carbon monoxide, similar to smoker exposure, protect against Parkinson's by preventing neurodegeneration and alpha-synuclein accumulation, suggesting further investigation into its disease-slowing potential.

ProJenX, Inc. forms Clinical Advisory Board (ClAB) with experts in ALS and neurodegenerative diseases to guide clinical development of prosetin, a novel brain-penetrant MAP4K inhibitor. Prosetin is being evaluated in Study PRO-101, a Phase 1 trial assessing safety and tolerability in healthy volunteers and ALS participants, with positive early results.

Vaderis announces positive results from its HHT clinical trial with VAD044, showing favorable safety, tolerability, and efficacy in key disease manifestations. Ongoing OLE data at 6 months confirm consistent safety and improvements in bleeding parameters.

NeuroSense Therapeutics reports positive 12-month biomarker data from its Phase 2b ALS trial, showing PrimeC's regulation of iron levels linked to improved survival and disease mitigation. PrimeC demonstrated a 36% slowing of disease progression and a 43% improvement in survival, supporting its advancement to Phase 3 testing.

Rezatapopt, a first-in-class p53 Y220C reactivator, shows promise in targeting the previously 'undruggable' p53 Y220C mutation in advanced solid tumors. Currently in phase 1/2 PYNNACLE study, it has demonstrated efficacy and tolerability across various tumor types, with a recommended phase 2 dose of 2000 mg daily. Early results indicate notable activity in refractory cancers, with ongoing phase 2 studies aiming to further clarify its effectiveness and potential for FDA approval.

Denali Therapeutics reported Q1 2024 financials, highlighting progress in neurodegenerative and lysosomal storage disease treatments. Key updates include positive clinical data for tividenofusp alfa in MPS II, DNL343's Phase 2/3 ALS trial enrollment completion, and advancements in Parkinson’s and Alzheimer’s therapies. The company raised $500M in financing, focusing on BBB-crossing therapeutics.

A phase 1 clinical trial for a novel CAR-T therapy targeting glioblastoma showed dramatic tumor reduction in patients, with one achieving near-complete regression. Despite eventual tumor progression, the results are promising, prompting further research to enhance treatment durability. The approach combines CAR-T and bispecific antibodies, aiming for broader efficacy against solid tumors.

The World's Best Hospitals 2024 ranking highlights 12 top U.S. hospitals, including Mayo Clinic, Cleveland Clinic, and Johns Hopkins Hospital, known for their excellence in patient care, research, and medical innovations. These institutions lead in various specialties, from cardiovascular care to organ transplantation and cancer research, showcasing the U.S.'s prominence in global healthcare.

Denali Therapeutics and Sanofi's ALS drug failed Phase 2 trial, missing the primary endpoint on the ALSFRS-R scale. Despite the setback, Sanofi continues testing the drug for multiple sclerosis. Denali is also developing another ALS drug, DNL343, targeting nerve cell death mechanisms.