I already asked why this wasn't a treatment to the MJFox foundation research group. They gave some justifications that according to me are not very convincing. One of them is that they can't give this in pill form. The funny thing is that they are testing sialidases now instead:https://www.michaeljfox.org/foundation/grant-detail.php?grant_id=1010
The funny thing is that this is infused directly into the brain ... so no pill either. If it has to be in pill form, then why throw away foundation money into something else that also is not a pill shape ? Or, why is the pill form a reason to not provide GM1 treatment, but for sialidases it is OK ? Another reason was that you can't mass produce this; but I found a research paper in which researcher claim it can be mass produced by means of a marine bacteria.
The only reason that made sense was that GM1 ganglioside is obtained from sheep and pigs (I also found papers showing it can be obtained from fishes) and therefore diseases can be transferred. I don't know whether it is impossible to avoid this during synthesis process. But I have the feeling it should be possible doing some pretreatments to remove bacterias and viruses.
In the end, I am very dissapointed about this fact. It looks to me that there already exists something to halt Parkinson's disease progression but for some reason this GM1 ganglioside is not provided to the patients. And I wonder why I even bother to spent money in PD research. You invest in it, they find a cure but then they don't provide it.
Looking at the clinical trial for GM1 Ganglioside (http://clinicaltrials.gov/ct2/show/study/NCT00037830?term=gm1+ganglioside&rank=1§=X0125
) it had to be injected subcutaneously
twice a day. That would be hopelessly impractical for a PD drug where it would have to be administered for years. The initial study was obviously a "proof of concept" study. The next step would be to chemically modify the molecule to make it orally available (ie taken as a pill). There are probably 3 or 4 chemists somewhere trying to do just that............
Bartobob, I understand it is not easy. But impossible ? Is it really impossible to go to your doctor for a daily injection ? I can understand that you don't go get an injection every day for throat pain. But for a disease as Parkinson ... ? What is the alternative ? Wait for a more practical form and in the meanwhile leave millions of people rotting away in a horrible disease like Parkinson ? Isn't it horrible to just rot away day by day, knowing that there is something out there that could have stopped your disease progression ?
I have a grandmother of 89 years old. She gets visited every day by a nurse. Why can't there be a Parkinson nurse doing the exact same thing for people with parkinson and provide them their daily injection ? Is this really so impossible ? We managed to send a spacecraft to the moon, we managed to see galaxies far away with special telescopes, we manages to analyse people's DNA in a few days/weeks, ... and then getting a daily injection is impossible ? Really ? Even if people are willing to pay for the costs ?
I share the concern expressed about the lack of information on this drug. Re the injections--- no need for the nurse. Millions of diabetics inject themselves umpteen times a day with insulin. And the Apo-pen.
I would like clarification of the progress and problems associated with this drug from the Research team, please.
Hi Mister X
Thanks for an interesting question.
As I’m sure you’re aware, new treatments have to go through a long process of development before they become available to patients. The clinical trial process is in place to make sure that any new treatments are not only safe and effective, but are better than existing ones.
The paper you linked to describes a very small trial that included just 26 people with Parkinson’s. As the authors state, additional study is needed to find out the degree to which GM1 ganglioside improves symptoms, and if it really can slow the progression of Parkinson’s.
Bartobob is right - it’s likely that research is happening right now to find better ways of harnessing the effects of GM1 ganglioside. The research into sialidases is a good example of this.
We’ll certainly be keeping an eye out for any publications and will report any interesting developments on our website and in our magazines.
GM1 ganglioside is just one of a number of potential treatments for Parkinson’s that are currently in the pipeline. We talked about some of the others in the latest issue of our research magazine Progress:http://www.parkinsons.org.uk/progress
I hope this is helpful.
Research Communications Officer
Hi Katie from the research group,
I appreciate your answer. But still I am not convinced. You know, I digged deeper into GM1 ganglioside. I was astonished of how long ago the beneficial effects of this drug on parkinson's disease were known. I found an article of GM1 tested on non-human primates in 1984 !!! What are we now ? 2012. That's almost 30 years later. If you take into account that it takes approximately 10 years for a product to get sold to people ... that means that this product should have been on the shelves 20 years ago. Where are we now with it ??? We finished a weird clinical phase 1 with it. Why weird ? Because a clinical phase 1 trial usually doesn't take 5 years. At this pace, it would take us 100 years instead of 10 years to get a medicin on the shelves.
So what is happening with this GM1 ? Why is this product being investigated this slowly, given the fact that it seems like THE best known product to modify disease progression of parkinson ? Why on earth aren't we doing a clinical phase 2 trial with it, but instead starting preclinical trials on sialidases ? Is this a joke ? Nobody knows whether these sialidases will even work. And by starting research on it, we will need to wait another 10 years before this reaches the shelves; if they will ever reach on the shelves because we don't know this will work. If a clinical trial phase 2 starts on GM1, it could be on the shelves in 5 years. Do you understand my concerns ? Do you understand my frustration ?
And then, to make it worse, I contact people and they give silly replies on why GM1 is not able to get on the shelves. You know, people are getting sick and dying. Parkinson's disease is not like a throat pain or ear infection that bothers you a few days and afterwards you get cured. People are really suffering because of this disease. The scientific/medical community has a responsibility towards patients; especially given the fact that many people donate tons of money to the community to cure the disease.
To be honest, I still didn't receive any comment from any researcher that gives me a satisfying explanation of why GM1 is not being developed at the pace it deserves.
Mister X I understand your frustration but if you say this product has been developed using pigs fish etc then do you not think the researches need time to make sure this treatment is safe, Yes i know it has been many years but what if when it was tried on a primate there was problems some years down the line that they may have now rectified ?. We all know of the mad cow disease problem with hormonal treatment and contaminated meat some years ago, do you want this treatment then find out you have contracted some thing else from the treatment later in life. Let the researches do there job. We all want a cure some more than others look at cancer how many years have they been researching for a cure ! I have had breast cancer and i know it will come back and by then they may have another treatment to give me more time on this earth with my family, the treatments are better but not a cure, they usually give a person longer life but no cure. So i have a double reason for a cure for both diseases but i need to be patient and even then it may not come in my life time. I am 53yrs old and full of life I get on with life, my Parkinson's is only part of my life i sign up for research programmes in Parkinson's and cancer trials for drugs and therapies that may help us now and people in the future.
I do agree that the researches could explain the process clearer to non medical people and the reason's for delay's in products being released to the public , but as i said i would rather wait until medicines are 100% safe even if it takes years than change having to deal with other diseases that have been inflicted due to researches rushing products through.
Mommah, the thing is I got a list of reasons. These reasons didn't make any sense. They were reasons that a simple person will believe; but if better informed, you can see that all reasons were not good. The only reason still standing is the one concerning the "infection from animals". But I wonder whether this one is really a reason too, given the fact that all the other reasons were useless. Is it really true that you can't desinfect it and eliminate all viruses and bacterias from it ? In the clinical trial there were 26 patients that received GM1 for 5 years. Usually a clinical trial phase 1 takes half a year; 6 times less. So this is the equivalent of a clinical trial of 6 months on 156 patients. And nobody got infected. So is it really as unsafe as they claim ? I tend to believe not.
When I first read these excuses, I believed them. But then I read an article a few months ago about a big breakthroug in huntington disease. Researcher were extremely happy and they wanted to make a therapy for people with HD as fast as they could. So I wondered ... if researcher in HD consider GM1 as a therapy for HD, then why researcher in PD say it isn't a proper therapy ? Then I started digging in it, and I found out that all reasons were nonsense. The only reason I can't proof nonsense is the animal transfer. But I guess it's more because of a lack of knowledge from my part. If people in HD see a valid therapy in it, I got all reasons to believe this is nonsense too.
Anyway, scientist have tested so many meds in the past 30-40 years. To me it is obvious GM1 for PD hasn't been investigated as it should have been. I wonder why ? They come with all kinds of new levodopa therapies. These new therapies were developed much after 1984 (when GM1 was already tested on monkeys). My question is ... WHY ??????? You don't agree with me that GM1 development has been extremely slow ?
Have discovered LigA20, a derivative of GM-1 which can be fed orally and reaches high concentrations in the brain.http://www.pnas.org/content/91/14/6303.full.pdf+html
So why wasn't that used in the clinical study?
Maybe because Fidia s.p.a. (an Italian drug company)hold european and US patents for its use in PD (and dont seem to be doing anything with it--probably using the patents as poker chips) or because "Unfortunately, however, LIGA20 is not currently available in the United States, due to problems that the manufacturer FIDIA has faced in exporting it.LIGA20 is produced by isolation from bovine brain, and the USDA has placed severerestrictions on importation of all such substances from Europe due to concerns about bovine spongioform encephalopathy."(2003)http://www.nidcd.nih.gov/staticresources/funding/programs/npp/pdf/archived/N01-DC-0-2108QPR11.pdf
They aren't studying it because the scientists think it is more usefull to start testing a completely new product related to GM1 gangliosides on fruit flies. They hope to prove by 2078 that it is disease-modifying for Parkinson's disease. They are doing a pre-clinical phase 1 on 2 fruitflies. They hope to be able to do a phase 2 on 30 fruit flies in 2025. They even think of doing 6 extra preclinical trials on fruit flies to be 100 % sure that the product they sell works. If for this, the product will be on the shelves in 2145 instead of 20178, so be it.
Did you also notice in the article you posted that those scientists are still thinking of doing tests with GM1 ganglioside ? I wonder where they got their science degree. Don't they know that GM1 ganglioside is very dangerous to people ?
I started to dig deeper into ganglioside. I came to the interesting conclusion that in all neurodegenerative diseases, there is a problem with the gangliosides in the brain. In Parkinson's disease and Huntington's disease there is a lack of GM1 ganglioside; and as far as I understood, in Alzheimer there is too much GM1 ganglioside. In fact, as far as I understood, it is the binding with the excessive GM1 that leads to the plaques. So I was wondering ... is it possible that by giving people a GM1 pill, that you halt Parkinson's disease but pave way for Alzheimer in that patient ? Looks like you have to be carefull and keep the levels within certain limits or many things can go wrong.
I'm glad they are pushing ahead with israpidine to slow down PD. It looks really promising. As it's already out there as a calcium channel blocker it should be approved by the FDA quite quickly.
I am also happy they are working on isradipine. But I have to say, this is only 1 agent and it is not proven it slows down PD. It is only proven that it is safe. I hope it works though. On the other hand, maybe the article is a bit poorly made. I know MJFF is also working on the creatine study; it is not mentioned in the article. Besides that, I also think MJFF is funding the Cogane studies, another promising lead which is almost at the end of clinical phase II. And I don't know what they are planning for GM1. I really hope a phase III trial will come for GM1. From the article you can derive that dipraglurant recenlty finished phase II and that it is possible to have it on the market in 2016-2017. GM1 is in similar phase. So if they don't throw GM1 in the garbage, PD patients might have something that dramatically slows down PD by 2016-2017.
I dont know if people have seen this fantastic news:http://www.sciencecodex.com/promising_drug_slows_down_advance_of_parkinsons_disease_and_improves_symptoms-102996
The thing I cannot understand is why this is not big news across all our newspapers and TV. I've only come across this in a couple of obscure websites.0
But as far as I can tell, this is the first double-blind, placebo controlled phase II study that has established statistically significant evidence of slowing disease progression.
If this is true, it is without question the biggest breakthrough since the advent of levedopa. You could actually argue (if it succeeds in phase III) it's more significant than levedopa because this slows disease progression whereas l-dopa only affects symptoms.
Perhaps the reason this isnt making headlines is that it's a university study funded by government, rather than by big pharma with a huge PR department to push out the results.
Either way, I think this is really great news. Just a question of when phase III will get underway. Apparently the main issue is producing it in quantities sufficient for 1000s of people in the trial and then obviously on to the market.
Indeed, I am also surprised this is no big news. As I was also very surprised that in the Alzheimer community nobody spoke about LMTX. I am not an expert in medicine or parkinson but it took me 2 months of research to find out that LMTX is the current best thing for AD and GM1 for PD. (LMTX might also be very promising for PD). But nobody ever speaks about it.
Anyway, I was really pissed of on the MJFF foundation because I fund them and they are pursuing the most crazy things sometimes like green tea trials and stuff like that, but they didn't invest a penny yet on GM1. This does not mean that they shouldn't do trials on teas and other stuff. Of course they should. But when something like GM1 is out there, and money has to be divised wisely because clearly there is a lack of money, then GM1 should have been on the top priority list anyway. You see, it took the poor professor more than 30 years to be able to carry out a clinical phase II trial. If it wasn't for the NINDS (I think) GM1 would have never even been tested on humans at all !!! If GM1 would have been dealt with properly, GM1 would probably be a standard treatment at this moment. Do people realize how many PD patients have suffered and are still suffering because of the mismanagement of the PD scientific community ? And the worst part of all is that I even doubt GM1 will ever be provided to people. It makes me angry and sad at the same time that people are suffering now while a "cure" should be only 3 to 4 years away (a clinical trial III is the only hurdle to provide this to the PD patients).
In the past I already complained about this on the neurotalk forum, but instead of getting support to put the PD community under pressure to pursue GM1, I got insulted and banned by the moderators of the forum. With exception of 1 lady on the forum, all the others just nodded "yes, the mod is right and PD community is doing fantastic not pursuing GM1". I find it shocking to see how people that are sick are not willing to fight for a cure.
I would like to provide some perspective on the GM1 ganglioside thread that may answer some peoples questions. GM1 ganlioside as a treatment for many different neurologic disorders was started many years ago by an Italian pharma called Fidia. Their GM1 was produced from slaughter house cow brains and when Mad cow disease became a concern, they basically went out of business. Dr. Schneider's work was all done with the bovine source material. Their is no synthetic GM1 and attempts at semisynthetic products have still not succeeded in suitable quantities. We have been working for almost 20 years to develop a sheep source for GM1 production from animals that have a genetic condition that results in massive accumulations (40X) of GM1 ganglioside in there tissues. We have developed a source verified raw material that could be scaled to meet therapeutic needs for a variety of disorders including Parkinson's. We know how to purify the GM1. We are currently trying to work with the Huntington's disease community as HD is an orphan disease and the approval process is somewhat streamlined. Unfortunately, we have hit numerous road blocks along the way. It appears that people are more interested in money than treatments and protecting turf than working together to find treatments. If any are interested in our project, you can go to our website, Glycoscienceresearch.com. We are hopefull that GM1 ganglioside can soon be available for use in PD and HD...I believe that these very special lambs were created by God solely for this purpose. If you have any questions, feel free to contact me. Larry Holler DVM,PhD