May 24, 2012

Michael J. Fox and Parkinson's: Stem Cells, Biomarkers, Clinical Trials

In his recent interview with ABC News anchor Diane Sawyer, Michael J. Fox said that stem cell research may not yield solutions for people with Parkinson’s disease as soon as other potential therapies.

Appearing with Debi Brooks, his foundation’s co-founder, Fox said that there’s a lot of good work being done on stem cell research, and that it should continue. But he now acknowledges that there have been “problems along the way,” and believes that new drug therapies, experimental surgeries, or earlier diagnoses are more likely than stem cells to provide effective treatment – even a cure -- for Parkinson’s.

In the interview, Brooks explained that scientists have had good results developing new dopamine-producing neurons – healthy brain cells – from undifferentiated stem cells. Treatment with stem cells has made great sense, in theory. The trouble, she added, was in the applied biology – how to get those news cells into a PD brain… and getting the deteriorating brain to accept those new, implanted neurons.

Brooks added that treating PD may very well involve more than simply putting fresh, dopamine-producing neurons into a patient’s brain. She said that looking at “other therapeutics” could yield broad benefits for people with Parkinson’s.

The shift away from the key position of stem cells in Parkinson's research represents an interesting development for the popular actor and advocate, whose dyskinesia – the tics and shakes associated with the disease -- has become the public image of Parkinson’s. (Dyskinesia is not a symptom of the disease, but a side effect from long-time use of the standard PD medication.)

Parkinson’s Progression Markers Initiative
If Fox seems a little less bullish on stem cells, he’s now an absolute believer in the importance of much earlier diagnoses, which, he thinks, will prove critical in developing treatment or cure. Said Fox, “By the time my pinkie twitched {his first symptom}, about 80% of my dopamine cells were gone.”

To develop earlier diagnoses, Fox and his foundation team have recently begun the Parkinson’s Progression Markers Initiative (PPMI), a clinical study to find biomarkers of PD. Fox hopes the initiative will move the science beyond providing some relief for symptoms – which is essentially where we are now.

Toward that end, Fox and his foundation are looking for “400 newly-diagnosed Parkinson's patients who have not yet begun medication, and 200 control participants who do not have Parkinson's disease or a close relative with Parkinson's.”

The Fox Foundation PPMI site shows that -- as of this morning – they have successfully recruited over half of the required 200 control subjects, and nearly half of the required 400 people with recent PD diagnoses. So far, PPMI studies are set for 18 U.S. cities and six international cities.

Clinical Trials and The Fox Trial Finder
PPMI is only one study. Brooks estimated that there are about 200 others PD studies listed online.

During the Sawyer interview, Fox and Brooks said that 85% of all clinical trials are slow to complete their work because of recruitment issues. They reported a shocking 45% of all trials never recruit a single patient.

Less than 10% of the estimated one million people with Parkinson’s in the U.S. participate in clinical studies. Wishing that percentage were higher, Fox said that the recruitment slogan should be: “YOU may be what you are looking for!”

Interested in participating in a clinical PD study near you? The Michael J. Fox Foundation is looking for tens of thousands of volunteers, and they want to make it easy to match people with appropriate studies. Check here:  MJF Trial Finder.

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