Radically different
Biogen and Denali Therapeutics said Thursday that their experimental therapy for Parkinson’s disease failed to slow the degenerative brain disorder in a randomized trial, dealing a substantial blow to a scientific approach that stoked excitement among advocates and academics.
In the study, 648 adults with Parkinson’s were randomized to receive either a placebo or a pill targeting a protein called LRRK2. In 2004, researchers discovered that mutations in the LRRK2 gene can cause a rare, inherited form of Parkinson’s. And in 2018, another group of scientists showed that blocking the protein might actually benefit all patients with the disease.
Thursday’s results are a significant setback to the latter idea.
Thursday’s Senate Appropriations Committee hearing was intended to focus on President Trump’s proposed budget for the National Institutes of Health’s fiscal year 2027 budget.
Instead, many of the senator’s questions for NIH Director Jay Bhattacharya and the five institute directors who joined him focused on more immediate concerns: a leadership vacuum at the agency’s sprawling infectious disease institute amid two outbreaks, the slow pace of funding over the course of the current fiscal year, and the impact of various Trump administration policies on the research community.
Here are three questions that were raised during the hearing:
A type of targeted chemotherapy developed by China-based Kelun-Biotech and licensed to Merck cut the risk of tumor progression by 65% in patients with lung cancer, according to Phase 3 study results reported Thursday.
A preliminary survival benefit favoring the Kelun-Merck drug, called sacituzumab tirumotecan, or sac-TMT, was also seen in the study, but will require longer follow-up to confirm.
The study, conducted in China, is the first successful combination of an antibody-drug conjugate with a PD-1-targeted immunotherapy in patients with advanced but previously untreated non-small cell lung cancer.