These are email responses to my request for help initiating a clinical trial of orally ingested trehalose. Of interest I would note that one doctor had this to say:
"There are a few patients I would like to share this information with while we figure out next steps."
Dear Joe,
Very many thanks for your email and for taking the time to write to us.
Your thoughts are certainly interesting, and there does indeed seem to be a rationale for studies along these lines. In the first instance I would request a couple of weeks for us to digest this and consider a) existing data as we are coming to this afresh, and b) possible approaches to tackling this. I would propose that we then schedule a call to discuss in greater detail.
In the meantime, if you have carried out a literature review or similar, then I would be grateful if you could forward any such material.
I have also been interested in the potential of trehalose as a putative therapy for HD and other polyQ neurodegenerative diseases.
While I was very sceptical of the initial trehalose studies, there does seem to be a body of evidence suggesting that this might be reasonable to consider for eventual human clinical trials.
I do not currently run any studies in SCA1, and I am fully occupied with ongoing clinical trials in HD and as the co-chair of the HSG, my primary focus has to remain in HD.
Clinical trials are very expensive and difficult to organize. I have no experience with anyone funding a clinical trial using donations or fund-raising from families. The most likely path forward would be to generate a proposal to the NIH an/or FDA and seek funding from these sources. Unfortunately, As you may soon hear this is not currently the best time to be trying to push forward medical science in the United States. I believe that the recent Minocycline and creatine trials in HD were funded using this approach.
At this time any trial would need to be in HD for me to be involved.
Generating a viable proposal and clinical trial design is not something that I can take on at the current time, but I would be supportive of the effort and would be willing to help out as possible.
Cheers,
Blair
Blair R. Leavitt MDCM, FRCPC (Neurology)
Co-Chair, Huntington Study Group
Professor, Department of Medical Genetics, and Division of Neurology, Department of Medicine (Associate)
Interim Director & Senior Scientist, Centre for Molecular Medicine and Therapeutics (CMMT) - Researching Life to Change Lives
The University of British Columbia | Child & Family Research Institute
Room 2020, 950 West 28th Ave | Vancouver, BC Canada V5Z 4H4
Dear Joseph - thank you for sharing your experience and the papers. I have heard of trehelose for ALS and am interested in learning more. A few questions
how did you pick dose to take? Can trehelose be taken orally? Any advice on where to obtain. There are a few patients I would like to share this information with while we figure out next steps.
I would like to connect you with Dr Schmahmann who is our SCA expert - there is also a new ataxia network forming very interested in clinical trials- he is part of that.
I would be interested in pursuing this also possibly in als.
I will ask my HD colleagues also for input. More soon. Tx for your help and answer to above questions. Merit
Merit Cudkowicz, MD
Chief of Neurology, Massachusetts General Hospital
Julieanne Dorn Professor of Neurology Harvard Medical School
Perhaps AtaxiaUK might consider joining in and helping at this point? BioBlast only has a patent on IV administered trehalose not oral, nasal, or sublingual, or any combination of the 3.
Joe I think you are incredible. ...thank you so much for all you are doing. You are the only source of hope I have come across in the 29 months since my husband was diagnosed. I will be talking to his neurologist about trehalose when we next see her....although it's not something we look forward to........who needs to be told how much worse things are/will get if there's no help avaluable! Is there anything else you think any of us could do? May be stupid but what about contacting one of the news style talk shows like Jeremy Vine on Radio 2? They cover lots of stuff on medical conditions.....they might be interseted in it...if packaged as having potential for lots of conditions. ....Ataxia probably isn't interesting enough on by itself!
I have tried to contact science and health editors of major newspapers. It has generated some minor interest, but I think you should send an email to Jeremy Vine and see what he says.
The potential for trehalose is much bigger than ataxia as it also holds potential for treatment in Alzheimer's and Parkinson not too mention it has already been proven to be "heart healthy" by the trial I linked on vascular health. I think it's safe to say "the ball is rolling". It will still be quite some time before we have definitive answers, but I am very excited because although I am only speaking here about trehalose, my real end game is a drug "cocktail" treatment. The exciting news is I think it's here and just needs a seal of doctor approval. What I'm thinking is one drug to clean out the toxic protein, one drug to improve cellular health, and one drug to improve brain function: autophagy, mitochondrial enhancement, and glutamate enhancement. There are multiple candidates for all 3 avenues. Obviously I think trehalose can handle autophagy and in my original posting I mentioned Niagen because I believe that handles the second and there are several glutamate enhancers already on the market plus there is one in clinical trial for ataxia right now: BHV-4157. The combination of trehalose, niagen, and BHV-4157 or some similar combo should be a real possibility relatively soon. I believe it will be available long before gene therapy, but most importantly if effective it will not just treat symptoms but provide real benefit in stopping progression. Best of all I was on a conference call today with multiple doctors and they agree the time is now to try a "cocktail" in clinical trials.
Long story short, I foresee a real "treatment" becoming available in the next 5 years and one that is not expensive nor overly invasive, but like I said "the ball is rolling" but we have a "long row to hoe."
Great idea about trying to get the word out there about trehelose and to try and drum up some public awareness. This might be tricky for Ataxia UK, I expect they have a fairly clear comms strategy. Perhaps some of us could get together and form a type-of pressure group. A starting point might be for members of this blog to write to their MPs. We could produce some standard wording, perhaps drawing a bit on you expertise Joe (if you don't mind). If enough of us do this, it would be easier to peak the interest of Jeremy Vine, Victoria Derbyshire etc.
We have introduced this pack size because it can be shipped outside the UK throughout more cheaply. A 1.9 kg Trehalose pack costs £13.85 to post throughout most of europe.
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