2024 Year In Review | Page 25

Replacing and protecting cells
Efforts to deliver dopamine-producing cells and tissues or growth factors into the brain continue to progress . BlueRock Therapeutics is planning a Phase II trial following findings indicating its stem cell-derived dopamine replacement therapy improves motor functioning . Aspen Neuroscience and Kenai Therapeutics are working to replace dopamine cells in the brain with neurons engineered from induced pluripotent stem cells ( made from reprogrammed adult cells ). Asklepios BioPharmaceutical advanced its growth factor gene therapy to Phase II testing .

Improving Non-motor Symptoms

There is a growing focus on medicines specifically designed to treat the non-motor symptoms linked to Parkinson ’ s . Treatments targeting PD-related cognitive changes include two drugs in Phase II testing , and a new drug for apathy is in development . Two different drugs , ketamine , a dissociative anesthetic with known hallucinogenic effects that has been studied for treating depression , and the psychedelic psilocybin , are being studied for treating PD-related depression . For non-motor symptoms less responsive to pharmacological therapy , technology-based interventions show promise . Sibel Health is conducting Phase III testing of a wearable wireless device to reduce excess saliva and swallowing difficulty . A smartphone application to improve speech volume and clarity , called Understand Me for Life , is in Phase II testing .

De-risking Lowers Barriers to New Treatments

Since its start , MJFF has used a research funding strategy called “ de-risking ” to jump-start the development of new treatments for PD . At the heart of this strategy is the understanding of the risk profile of developing new treatments , especially in brain disease . The astronomical financial investment required
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