Spring / Summer 2025 | Research 9
Investigative Therapeutics Exchange( LITE), the initiative focuses on bridging basic science advances to industry-led drug development.
New Tools Improve Clinical Trials
The outlook for testing the effectiveness of these PD drugs is improving as new tools become available. Last summer, the FDA issued a letter of support encouraging the industry to adopt the biomarker test for PD’ s hallmark misfolded protein, alpha-synuclein, to ensure that trials enroll people with the right biology to test a given medication.
The Foundation is at the helm of a robust treatment pipeline, monitoring progress on more than 150 drugs and therapies in clinical trials.
said Catherine Kopil, PhD, head of clinical research at MJFF.
LRRK2-Targeted Therapies Another area of active development focuses on the LRRK2 gene. Mutations in the gene hyperactivate LRRK2, triggering cellular dysfunction that leads to PD. Strategies to reduce this hyperactivity hold potential to help the four percent of people with inherited LRRK2 mutations and potentially people without mutations as well.
Several PD drugs targeting LRRK2 are currently in clinical trials, including Phase II trials at Neuron23 and Denali Therapeutics. Several other strategies to target LRRK2 have been identified and await further evaluation.
In 2024, MJFF launched a major new program to expand LRRK2 insights and expedite therapeutic development. Called the LRRK2
MJFF has provided funding to over a dozen groups focused on improving the available biomarker test and finding others. Researchers are also developing a test that uses skin or blood, rather than spinal fluid, to detect the presence of alpha-synuclein, making testing less invasive and less expensive. These tests are also designed to be quantitative, so that they not only detect the disease, but also reveal changes in its severity. Such quantitative tests are crucial to gauging whether experimental therapies slow or stop the disease from progressing.
Remarkable progress is also occurring in imaging for PD. At least six alpha-synuclein tracers have advanced to testing in human trials. Positive results from any of these trials could deliver the breakthrough alphasynuclein imaging tool that the Parkinson’ s field has long been seeking.
“ The field is energized for what’ s to come: a day when we can clearly measure, quantify and visualize brain pathology in PD,” said Jamie Eberling, PhD, head of research resources and imaging lead at MJFF.“ When that day comes, it will fully transform what’ s possible in drug development.”