A recent newsletter announcement reads:  "MJFF Funds Two Promising Stem Cell Technologies".

I learn that "The Michael J. Fox Foundation (MJFF) announced funding for two projects leveraging the promise of engineered stem cells to speed new therapies and deeper understanding of Parkinson’s disease".  Excellent news to those of us who believe that the future of PD treatment lies in the field of regenerative medicine.  Thank you MJFF.
Reading on though, I am not so excited about the focus of these studies - more work on pre-clinical models, development of diagnostic tools and cell lines for the lab-bench.  The usual. Sigh. 
Why such conservatism?  After all, iPSCs have been transplanted into rats since 2010, and the Europeans have been conducting  hfVM transplant trials in real people since 2009.
 
My resignation turns to animated frustration however, when I click on the included link - MJFF Signs Letter Opposing the REGROW Act - and this is the subject of this rant.
 
It seems the Foundation that is devoted to doing whatever it takes to find a cure to PD have "joined a coalition of 10 patient advocacy and research groups opposing the REGROW Act, a piece of legislation moving through the U.S. Senate that seeks to establish a new way to approve stem cell therapies earlier in the testing process."    Hang on a sec...?  Read that again...
 
A little research tells me the REGROW Act in question - according to the bill's authors - "seeks to reduce barriers to medical innovation and accelerate the development of new regenerative medicine treatments, which have the potential to restore or establish normal function in damaged human cells, tissues and organs" by "requiring the FDA to collaborate with stakeholders to develop standards that will lead to manufacturing processes and controls for safe regenerative medicine products".  Hmmm.
 
Still confused, I return to the newsletter: the MJFF "believes that stem cell therapies should be proven effective before making it to market. The Foundation signed a coalition letter opposing legislation that would give conditional approval to stem cell therapies that have proven safety but not yet efficacy".   Ohh! Now I get it...  I can totally see why the Foundation would throw it's considerable weight (and effectively the combined weight of all PD patients everywhere) against this DANGEROUS legislation.  We Must continue to enable the FDA to protect me from myself!
Please excuse me while I kick the wall a few times and yell - you see I have this degenerative neuronal disorder in my brain which has no known cure, one of the non-motor symptoms of which is to lessen my patience with bureaucrats who would seek to make choices for me....  OK, breathe... breathe... (here, I'll just pop a little extra Sinemet...) Phew, I feel better now. 
 
So the concern is over insufficient data to determine efficacy.  Well, there seems to be a double standard operating here:  based on my doctor's advice, I take a really expensive FDA-approved pill of rasgaline every day - not because it is known to help people with PD, but because it might do.  The results in favor of it are still so thin that some clinicians do not prescribe it.  But it's good enough for the FDA (and for me for now).   Is regenerative medicine being held to a higher standard than pills and potions?
 
Maybe I'll find an answer if I continue reading that Letter of opposition to REGROW:
"There exist several pathways to get critical treatments to patients quickly while maintaining strong FDA safety and efficacy standards. "   
 Thump! thump! Yell!! Sorry...  I thought I had that wall-kicking tick under control.  Does the Foundation or it's brain-trust actually believe the status-quo is good enough?  Am I to sit on my hands while committees argue over how to "demonstrate efficacy",  and researchers have entire conferences devoted to how to plan and design standardized trials? (not to discuss actual clinical trials you understand - they talk about how to talk about clinical trails).  Should I  quietly endure while people argue over the impact of heterogeneous patient etiologies in small sample-size statistics?
 
The only way we are going to get past the small sample sizes and the unknowns is to try the treatments.  I wager some of us PWP's would be willing to try a few "probably-safe" treatments of uncertain efficacy.  Most of us do some form of this every day with the multitude of supplements, exercise routines and diet regimes we embrace in the hope of some small slowdown in progression.  What would we do for an alternative to a lifetime of taking toxic medications?  What might we do for a cure?  Maybe try some snake oil?   Buy in to some sham derivative treatment?  Sure.  Some of us.  But with DATA and reporting that the FDA can mandate, that number would be small as the rest are informed of outcomes.
 
I ask Dr. Isacson and Mr. Sherer of the Foundation - could we not add to FDA labeling a declaratory section on efficacy ?
Right alongside the warnings and contraindications (Warning: in some patients this treatment may cause nausea, headaches, psychosis or death), could we not have statements along the lines of "this treatment currently carries an FDA's efficacy rating of 3 out of a possible maximum of 5 - for more information on this standard and for specific individual results and outcomes, please click on the following URL..." ?
Patients could then be informed that a given treatment has been in use for many years and has demonstrated positive results in say, 85% of cases.  Or perhaps they could review the details of a "bleeding edge" treatment that has been proven "mostly-safe", but has an efficacy rating of zero, because it's been tried out in only a handful of individuals (perhaps small sample of whom saw significant benefit, but others saw none).  Would this be a bad thing?
 
Surely the aim is to bring safe, effective treatments to the PD community as quickly as possible.  Is the conservative FDA status quo really accomplishing this?  Are we so satisfied with the existing system that it's OK to turn our backs on any opportunity to make it better?  Instead of turning away, why not join the conversation and be a part of working out how to make this bill work?
 
/End Rant

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The Crooked Path is a blog written by Corey King, a CCPSG member and diagnosed with PD at the age of 47.  

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