Parkinson’s disease research has ended in numerous dead ends despite substantial efforts over many years. Recently, Biogen and Sanofi scrapped their Parkinson’s candidates, cipanemab and venglustat respectively, owing to lack of efficacy, and a disease-modifying therapy has yet to materialise.
But the push to find drugs that help beyond reducing symptoms continues, and Evaluate Vantage has delved into the pipeline of projects in active late-stage clinical trials. This year is shaping up to be crucial for the field, with 10 studies expected to yield data or to complete in 2021.
One target that crops up multiple times is GLP-1; this approach, traditionally employed in type 2 diabetes, is also being tested in Alzheimer’s. Among other avenues of research, it is hoped that gene therapy could offer a one-time cure for Parkinson’s.
@cclemonade, sorry to cast some Coldwater on your enthusiasm and hope for a second time today 🤨
I have been on Exenatide since 2006 and I personally don't think it would have made a difference. I got Parkinson's anyways and it didn't do much to help with my symptoms which is tremor dominant.
It's a disease modifier not a symptom reliever. It might be that with exanatide it takes 20 years to progress to stage 3 instead of the 10. I'd take that as a good start.
@winniethepoo, you perhaps do have a point. My diagnosis symptoms have not progressed over nearly 10 years and come to think of it, it is certainly possible.
Your symptoms have not progressed for nearly 10 years and you are on Exenatide. I doubt that is a coincidence. I’m perplexed then by your stating that it didn’t do much to help.
Exanatide is being trialed as a disease modifier. Something which slows the rate at which your parkinson's progresses. Why would you expect it to do anything for your existing symptoms?
@winniethepoo. Your point well taken. Never viewed my plight / fortune with this perspective. However, your position speculates that disease modification and symptoms are synonymous with each other and one is indicative of the other. At least that is the way, I have seen most research being presented or perceived.
I think the way trials like the exanatide one (or my SPARK trial) measure disease progression is to look at symptoms over time, and compare the rate at which symptoms progress (off meds)
Initially it is hard to be sure whether the symptomatic result is due to the drug relieving the symptom or the drug "putting out the fire" that causes the symptoms to get worse.
There are 2 ways of trying to isolate the disease modifying / progression slowing effect from simple symptomatic relief
"Wash out" - after (say) 12 months drug use, stop drug use at month 12 and observe the symptom at month 12 and (say) month 15. If the benefit remains at month 15 it is likely it is due to the disease slowing properties rather than simple symptomatic relief. If you stopped Sinemet on month 12 and assessed symptoms at month 15 - they would be much worse
The 2nd method is to review at multiple points. If you measure the symptom at month 12 and month 24 and the result remains constant, then it is likely to be a progression slowing effect. Especially when you consider the measurement is off-meds (No sinemet, pramipexole etc for 24 hours before the assessment)
Bass of Spades, your upbeat attitude is wonderful and very healthy! Along the path to genomic therapy I foresee a disease slowing treatment of some sort and stem cells and FUS.
The thing with stem cells is that it doesn't stop the disease process, so those replaced cells might feel good for a while but they eventually die back from the same things as the old ones.
Gene therapy will target the specific defects, in protein synthesis for example. New methods in gene mapping will allow us to see exactly where things are going wrong and target the specific areas of the genome. Cancers will be cured like you've never imagined, too! They're already having success with things like sickle cell anemia and beta thalassemia, which are rare blood disorders. Just trust me , it's totally do-able and it's coming!
Conceptually, I'm not sure why fixing up my genes will help with the damage already done. But i have to confess to knowing absolutely nothing about it.
As for the theories about transplanting new cells into a hostile environment, I'm not convinced that its futile. The death of dopamine transporters is actually fairly slow. If we say you start with 1m DTs, and wr accept the 10 year / 70% estimates, then you lose 7% or 70,000 every year. If you can load up on another 500,000, then that might buy you another 500,000/70,000=7 years.
A study done in Lund Sweden in the 80’s, proved that the implanted neurons lived and were effective for over 20 years. I am new to understanding the trial process.
Is it reasonable to think that if a stem cell trial is said to take 7 years that in reality it could be 15+ years bc multiple rounds of trials might be necessary? Of course I hope they hit it out of the park in the first go but that seems rather unlikely.
I understand. But to my knowledge stem cells are farther along and therefore have a greater likelihood of being accessible to us someday. That was my only point. Hopefully I’m wrong. Are there in human trials for gene therapy for PD?
Assuming that stem cells are proven to be an effective treatment, the problem I see with them is one of scale. It is a highly invasive surgical procedure, and given its current state I don’t see how that realistically scales to the masses.
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