Approx. quotes from MJF: "Biomarkers to treat it prophylactically to effectively have a cure." "It's not finding the cause and then a cure , it's cutting it off." "If we can get there in the next 10-5 years I will be very happy."
I understand they are working on biomarkers but is there anything that you all know of that has evidence of treating it "prophylactically?" It is my belief that they are doing the massive biomarkers study in hopes of treating it prophylactically but at present there are not specific trials. Is that correct?
And, if my memory is correct, when my Dad was diagnosed 10+ years ago the "in 5 or 10 years" phrase was used consistently.
It is my hope that responses will be about the research, the science, and Parkinson's.
They say that 70 to 80% of the dopamine neurons are dead before physical symptoms appear. Any drug that stops or slow progression is likely to be very valuable in those that still have the majority of their neurons. In other words, any drug that is considered to possibly stop or slow is going to potentially prophylactic amongst those that have not yet lost a lot of neurons, and the biomarker research is to figure out who those poeple are.
Understood. I associate Exenatide with CPT but MJFF is "supporting" I hope they have more than just Exenatide. CPT has been working on that for over 10 years.
I quote:”A tool that could facilitate diagnosis and gauge the impact of therapies in development would truly change the way we're able to conduct research and how quickly we can move therapies through the pipeline. That's why finding a biomarker and research such as this are so critical.”
a precise bio marker could measure the progression of the disease , it would be a fundamental tool for observing the effectiveness of a potential cure in a research, determining its effectiveness in a short time. Pasteur with a microscope saw the microbes and I carry medical knowledge very far. A precise biomarker for the stage of PD would be something comparable.
It is not like they have been running into barriers for ten years -- every study, mice, open label and double blind has come out across with the study population universally successful at stopping PD in its tracks -- motor symptoms anyway; but I guess neuro protective is neuro protective -- or even going backward a small bit.
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Bydureon (a.k.a., Exenatide), a repurposed Type 2 Diabetes drug is now in third-stage trials in UK for stopping Parkinson’s in its tracks — results expected 2023. Testing — mice, open label, double blind — has been going on since 2010 and it has been positive every time.
According to a very sensitive test, Homeostasis Model Assessment for Insulin Resistance (HOMA-IR), 2/3 of Parkinson’s patients are supposed to be insulin resistant. IR may be treated with Bydureon (Exanatide). Just an angle that might get us one step closer to getting Bydureon.
Every time I hear about Exenatide it makes me wonder if fasting to control blood sugar levels or taking cinnamon to control blood sugar spikes or doing doing both fasting for say sixteen hours and taking cinnamon may be disease modifying.
in reply to
Which is why I fast 16 hours a day every day, do OMAD twice a week and no added sugar, not even fruit other than berries.
The key point about a biomarker, like the lady who can smell Parkinsons, is that IF you can detect it early using a biomarker, you can use a prophylactic like Exanatide earlier, and greatly extend that prohylactic's usefulness.
My understanding is that MJFF are sponsoring research in many areas, but one of their focusses is developing biomarker.
Much of the current research is into disease slowing solutions, like Exanatide or photobiomodulation, and damage repair solutions (like FUS and IPSC and Bristols GDNF) and proportionately less on symptom relief. All areas are valuable.
5-10 years is a rolling target that has been around forever
The "5-10 years" recurring statement is very strategic. Note he said, "10-5 years would make me happy." but listeners hear and hold on to the 10-5 years part not the "would make me happy" bit. It is dangling the carrot to keep those with PD and their families hopeful enough to keep funding. I remember telling my Dad over 10 years ago when he was newly diagnosed, "Daddy, you just have to make it 10 years and then the cure will be realized." How naive I was. I believed what the MJFF was touting. When neurologists who are not so strategic like Dr. Michael Okun give the more honest answer of most likely not even in 15 years, I trust that.
Which is why I put my emphasis emphasis on repurposed drugs. CPT appears to understand that due to the length and expense of new drug trials, seeking to slow the disease with repurposed drugs is the only realistic hope in the form of a pharma for currrent PWP. This of course does not negate the importance of the biomarker studies. But the biomarker studies will help the next generation, most likely, not us.
Most PWP in the US have not even heard of CPT let alone know of the repurposed drug trials. I want to help change that. Americans need to help fund CPT.
Your children, whose generation (well my childrens generation) will be the ones to benefit from those biomarkers enabling them to start disease slowing remedies, including possibly repurposed drugs, earlier. Remember that when you seek to mobilise them to the cause.
And be aware every repurposed drug I have seen, including the golden boy Exanatide, might help a bit. they are not spectacular. So far
As I previously stated, I am in no way minimizing the importance of biomarkers and the lengthy studies required to determine them. "might help a bit" is a lot better than, "sorry, nothing in time for you."
" CPT appears to understand that due to the length and expense of new drug trials, seeking to slow the disease with repurposed drugs is the only realistic hope in the form of a pharma for currrent PWP. "
Everyone understands the possible time-to-market advantages of repurposing, including the folks at MJFF.
"“Repurposing”— testing drugs often already approved by the U.S. Food and Drug Administration (FDA) for one disease to see whether they help people with another disease — can be an important strategy for speeding drug development. "
Consequently, MJFF has funded, or helped fund, various types of research into the following (amongst others) 'repurposing' candidates:
- Amitriptyline
- Fasudil
- Isradipine (including the big phase 3 trial)
- Terazosin
- Nilotinib
- DPP-4 Inhibitors
- Exendin-4 (exenatide, all the way back in 2009)
- Ambroxol (2011)
It isnt long ago at all (like, a couple of years only) that isradipine and nilotinib were the great white hopes for a PD DMT. They are yesterday's exenatide. They ultimately failed, but if they hadn't, it would be thanks to everyone that helped make it happen, not just whoever cut the cheque for the final phase 3 trial.
If you look at the funded studies of MJFF, you'll see that they tend to fund a lot of early-stage research. Why? Because no one else wants to, since the likelihood of a commercial payoff is very low. Current early stage research is boring if you have PD now, but it will be critical in the development of what eventually is proven to work.
It's a collaboration, not a competition. Why wasn't the CPT involved in the isradipine trial? Becuase they didn't need to be. Why isn't MJFF writing a cheque for the phase 3 trial of exenatide? They don't need to, because it is already funded.
I became a nurse in 1972. I started in neuro/psych. The "gold standard" was sinemet. At that time the doctors told the patients that there would be a cure in 5-10 years. Does any of that sound familiar? Sorry if I'm cynical but when I was diagnosed 7 years ago I was told the same thing
OUR BEST HOPE FOR A TREATMENT IS REPURPOSED DRUGS LIKE THOSE BEING TRIALED BY CURE PARKINSONS TRUST. (Sorry caps are obnoxious, I feel strongly about it) We should be more vocal about a “treatment in time for us.” Bc more funds should go to repurposed drug trials. Stem cells could be applicable in 10 years, possibly, if trials go well. Goodness I hope so.
Gene therapy, ProSavin, maybe 15 years? It pains me to type that but I think that’s optimistic but realistic.
I sure wish we could get it to 10 years or less.
What can we, PWP do to help support speeding up the process? Awareness of PD is not sufficient. Awareness and support for research and trials is what is needed.
When my Dad was diagnosed in approx. 2010, we were told 5-10 years. I think a “cure” will benefit my children’s generation. (They are 11)
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