Longevity briefs provides a short summary of novel research in biology, medicine, or biotechnology that caught the attention of our researchers in Oxford, due to its potential to improve our health, wellbeing, and longevity.
Why is this research important: Parkinson’s disease is the result of the death of dopamine-producing neurons in the brain. Conventional treatment for Parkinson’s disease involves drugs that increase dopamine levels, but introducing new dopamine-producing neurons is also possible. Research suggests that implanting dopaminergic neurons derived from stem cells can improve symptoms and reduce the reliance on drugs, but only a small number of patients have ever received this treatment.
What did the researchers do: In this phase I clinical trial, 12 Parkinson’s patients aged 50-78 received an experimental treatment called MSK-DA01. Human embryonic stem cells were converted into dopamine-producing cells and then surgically transplanted into the brains of participants. Participants had to take immune system-suppressing drugs for 1 year following the treatment (to prevent the cells from being rejected), and they were followed up for an additional year.
Key takeaway(s) from this research: The trial found no major safety problems associated with the treatment, which is the main goal of a phase I clinical trial. They also found that the transplanted cells were able to survive and engraft within the brain for at least one year following the treatment.
These transplanted cells should be able to form neural connections with existing cells and hopefully restore function in Parkinson’s patients, but we’ll have to wait for more advanced clinical trials to get an idea of how effective they might be. Using embryonic stem cells from humans raises both ethical and practical problems, as it would not be possible to scale it up to treat a large number of patients. Thanks to cellular reprogramming, however, we can produce dopaminergic neurons from adult cells extracted from the patient. Clinical trials for such cells are underway.
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Phase 1 Safety and Tolerability Study of MSK-DA01 Cell Therapy for Advanced Parkinson's Disease NCT04802733