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What We Fund Highlight: Replacing Dopamine with Stem Cells and Brain Cells

Study on Rate of Dopamine Loss Emphasizes Need for Biomarkers

It’s a question we hear often: What’s new with stem cells? The answer in 2022: A lot. Our Foundation has issued a major grant to a project testing a stem cell-derived therapy, and the field is watching a similar trial that just hit a clinical milestone. 

The promise of stem cells comes from their ability to transform into other kinds of cells. Parkinson’s researchers are creating dopamine cells to replace those that are lost in the disease. This cell replacement could help ease some of the symptoms of the disease linked to dopamine loss, mainly the movement issues. Learn more about stem cells. 

Implanting Dopamine Cells Made from Stem Cells 

Recently The Michael J. Fox Foundation (MJFF) awarded $3 million to scientists at Arizona State University for a trial to implant dopamine cells made from stem cells into brains of people with Parkinson’s and a PRKN mutation. That’s a lot in one sentence, so let’s break it down: 

What: Dopamine cells created from induced pluripotent stem cells. This type of stem cell is created in a laboratory from a skin or blood sample. Then scientists engineer the stem cell into a dopamine cell. 

Where: Implanted into part of the brain where dopamine cells are lost in Parkinson’s. 

Who: Eight people with Parkinson’s and a rare mutation in the PRKN gene. These variants are most associated with young-onset Parkinson’s disease. 

Why: This population does not have some of the other pathology seen in the disease (such as clumps of alpha-synuclein protein). That means it may be easier to see an effect of the dopamine cell transplant in these individuals. 

When: The grant is supporting preparation for the trial, which may begin in about a year. 

Jeffrey H. Kordower, PhD, principal investigator of the study, shared, "This study will make a great impact in treatment of patients with Parkinson’s. I think the future looks incredibly bright, and I couldn’t be more excited.” 

Another trial of this kind just completed enrollment of its first group of volunteers. BlueRock Therapeutics is working with scientists in New York City; Irvine, California; and Toronto to test transplant of stem cell-derived dopamine cells in people with advanced Parkinson’s disease. The study is now recruiting a second group of people diagnosed five to 15 years ago. Learn more and contact the study team. 

Transforming Brain Cells into Dopamine Cells 

Another approach to replacing dopamine cells looks at their neighbor. In 2020, Xiang-Dong Fu, PhD, and his research team at the University of California, San Diego (UCSD) discovered they could reprogram astrocytes, one type of brain cell, into neurons, another type. With a $1.1 million MJFF grant — and working with Kordower — they now aim to explore astrocyte conversion to neurons in more advanced preclinical models. If safe and effective, this treatment could move to human studies. 

Fu and collaborators have also received a grant from the Aligning Science Across Parkinson’s (ASAP) initiative to investigate the biology behind this reprogramming. More information on this process may help optimize this therapeutic approach. [MJFF is ASAP’s implementation partner and issued the funding.] 

A cautionary note: Stem cell therapies have not yet been proven safe or effective for Parkinson’s disease though some clinics offer unproven stem cell treatments outside of clinical trials. Read more on what to know about these offerings.  

Interested in genetic testing? Above we discussed a rare PRKN mutation. There are more common gene variants linked to Parkinson’s disease. Learn about genetic testing options. 

Help contribute to research on Parkinson’s biology and experience — whether you have Parkinson's or not — join our Parkinson's Progression Markers Initiative (PPMI). 


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