Kapil Jain retweetledi
Japan just approved stem cells for Parkinson’s disease. How will it work? Stem cells are special cells that can develop into other types of cells. In this case they are turned into dopamine-producing brain cells designed to replace the cells lost in Parkinson’s disease. Sawamoto and colleagues described in a new paper in Nature one of the first phase I/II clinical trial using induced pluripotent stem cell derived dopaminergic progenitor cells transplanted into the brains of folks living w/ Parkinson’s disease. These cells are generated from iPS cells and implanted into the putamen where dopamine neurons normally function.
Key Points:
- Seven folks w/ Parkinson’s disease received bilateral transplantation of dopaminergic progenitor cells derived from iPS cells into the putamen and were followed for 24 months.
- The transplanted cells survived, produced dopamine, and imaging revealed a roughly 44 percent increase in dopamine related activity in the putamen.
- The procedure appeared safe w/ no tumors or graft overgrowth and several participants experienced improvements in motor scores on Parkinson’s rating scales.
My take: Japan has a unique regulatory pathway for regenerative medicine that allows conditional approval once safety and early signals of benefit are demonstrated. For the therapy to advance in Japan data will be collected over the next 7 years. This paper represents an important milestone because it shows that lab grown dopamine cells can survive in the human brain and begin producing dopamine. The big questions ahead will be durability, patient selection, and whether the next 7 years of experience will confirm meaningful and sustained clinical benefit.
Here are 5 points that resonated w/ me:
1- Parkinson’s disease is caused by the loss of dopamine producing neurons and this therapy attempts to replace those missing cells.
2- The transplanted cells are generated from induced pluripotent stem cells which are adult cells reprogrammed to behave like embryonic stem cells.
3- Brain imaging revealed new dopamine activity in the transplanted region suggesting the cells were functioning.
4- Safety remains the first hurdle and this trial showed no tumors or uncontrolled cell growth over two years of follow up.
5- The future will likely combine cell therapy w/ medications, rehabilitation, and possibly gene therapy to maximize benefit for folks living w/ Parkinson’s disease. Here is the Nature paper that set the stage for the recent approval.
nature.com/articles/s4158… #parkinson @ParkinsonDotOrg @FixelInstitute
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