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Personalized Stem Cells to Treat Parkinson's

by Dr. Leena M on Mar 11 2025 2:32 PM
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Patients' own stem cells to replace damaged dopamine cells in Parkinson’s disease, offering new hope.

Personalized Stem Cells to Treat Parkinson`s
What if your brain could be repaired by your own cells? Researchers at Mass General Brigham are developing the first-ever personalized stem cell therapy for Parkinson's disease. Rather than merely treating symptoms, this innovative treatment attempts to repair damage by converting a patient's own blood cells into dopamine-producing neurons.
This approach is safer and more effective than traditional treatments because it does not require immunosuppressants. The therapy has already been administered to the first patients after decades of research. If effective, this strategy could transform the way Parkinson's disease is treated and lead to novel therapies for other brain conditions.

Is this the revolutionary development we've been anticipating?


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New Hope for Parkinson’s

A novel Parkinson's disease treatment that uses a patient's own stem cells to restore damaged dopamine cells is being tested in a new Phase 1 trial at Mass General Brigham. The Neuroregeneration Research Institute (NRI) at McLean Hospital conducted the research that led to this first-of-its-kind autologous stem cell transplant.

Brigham and Women's Hospital has treated three people thus far. Six people are involved in the trial, and their progress is monitored for a full year to guarantee its efficacy and safety. Researchers intend to expand the trial in Phase 2A if it proves successful.

This method makes use of patient blood stem cells that have been converted into induced pluripotent stem cells (iPSCs) (1 Trusted Source
Parkinson's disease - current treatment

Go to source
), which develop into neurons that produce dopamine. It is a safer substitute for immunosuppressive medications because no donor cells are required.


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Why This Study Matters

In contrast to existing treatments, cell replacement therapy for Parkinson's disease recovers destroyed dopamine neurons. This clinical experiment is the result of 30 years of groundbreaking research by Harvard Medical School neurology professor Dr. Ole Isaacson.

Isaacson is overjoyed to see his lab's efforts translate into effective patient care. He thinks that treatments for additional brain disorders may result from this discovery.

This strategy could revolutionize Parkinson's disease treatment (2 Trusted Source
Recent Advances in Drug Therapy for Parkinson's Disease

Go to source
) and beyond by replacing damaged brain cells, providing hope for longer-lasting and more efficient remedies.


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A Step Towards a Cure?

Beginning research in 2002, McLean's NRI created a stem cell therapy for Parkinson's disease under the direction of Dr. Ole Isaacson. They demonstrated efficacy using human iPSC-derived dopamine neurons by 2010, and in 2015, they demonstrated the long-term safety of the treatment in animal models.

The trial was approved by the U.S. FDA on August 23, 2023, and Brigham and Women's Hospital began treating patients on September 9, 2024. The investigation is being carried out by a team from Harvard Medical School and Mass General Brigham.

The accomplishment was hailed as a true "bench-to-bedside" innovation by McLean's Dr. Kerry Ressler. This is the first experiment to use blood-derived iPSC dopamine neurons for Parkinson's disease, and it is funded by the NIH's NINDS.

References:
  1. Parkinson's disease - current treatment - (https://pubmed.ncbi.nlm.nih.gov/37366218/)
  2. Recent Advances in Drug Therapy for Parkinson's Disease - (https://pmc.ncbi.nlm.nih.gov/articles/PMC9876715/)

Source-Mass General Brigham


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