Of mice, men and disease modifiyng treatments

StephanieStartz · 07-18-2012, 03:39 PM

Hi Laura,

No, this is different. This one is aimed at manipulating endogenous GM1 levels rather than introducing a GM1 or a GM1-like compound.

Hope that helps.

-Stephanie

07-25-2012, 05:00 PM

Quote:

Originally Posted by StephanieStartz

Hi Laura and Diego,

My name is Stephanie and I'm the social media specialist with The Michael J. Fox Foundation. I've been following the thread for the past two days and took your questions to our staff researchers in the hopes of providing context.

In regards to research on GM1, Jay Schneider has presented positive results using GM1 as a therapeutic in PD patients and evidence that it may be disease modifying. There are several challenges to treating with GM1 including its ability to cross the blood brain barrier, not being orally available to take via a pill, and low potency, but the main issue is the current source of GM1. Currently to get GM1 for drug trials, you have to extract it from pig or sheep and there are issues when extracting natural products from these animals (scale up of enough product, risk of transmitting disease, etc) so what Dr. Schneider is doing is testing a compound that aims to increase levels of GM1 in the brain by increasing the normal synthesis of GM1. This project is testing whether he can see similar results using this new compound to using the GM1 itself. (This is what the MJFF Rapid Response grant is funding, and why it is referred to as "innovative.")

The Foundation is working to move as many ideas forward as we can, understanding that the process is complex and never as straightforward as we would like it to be.

As to why this was under reported in scientific trade outlets, we don't really know why. We can speculate that perhaps enthusiasm was dampened by the uncertain future of this approach (the source of GM1). Additionally, the timeline is fairly long at this point given that any new product would need be shown to be safe.

For our full research strategy, including priority areas, please visit our website.

Please let me know if you have any questions, I am happy to take them back to our researchers.

Diego, I'm not sure who you've emailed at the Foundation, but please feel free to contact me at socialmedia@michaeljfox.org.

Thanks,
Stephanie

Stephanie,

Thanks for the reply. I don't know who I contacted. On the MJFF website you can contact them based on the category of your question. I sended a question related to the research priorities. I got following reply:

Jay Schneider has presented positive results using GM1 as a therapeutic in PD patients and evidence that it may be disease modifying. There are several challenges to treating with GM1 including its ability to cross the blood brain barrier, not being orally available to take via a pill, and low potency, but the main issue is the current source of GM1. Currently to get GM1 for drug trials, you have to extract it from pig or sheep and there are issues when extracting natural products from these animals (scale up of enough product, risk of transmitting disease, etc.) so what Dr. Schneider is currently doing is testing a compound that aims to increase levels of GM1 in the brain by increasing the normal synthesis of GM1. This project is testing whether he can see similar results using this new compound to using the GM1 itself. MJFF is supporting this project.

I get a huge lists of reasons that in fact don't seem very convincing reasons. Not orally available ? So what ? How did these people in the test phase got this med working for them ? I think people with PD wouldn't mind to get an injection on a daily basis. I knew I wouldn't mind if I had PD. There are people getting chemotherapy for cancer ... I think that's 1000 times worse ... but still people do this because this is their only hope.

I can go through all these reasons and counter them. You don't need to convince me that in its current shape it is not ideal. But ... come on ... does that matter ? There are people suffering and there is something that could help all of them ... it even helps people with HD. So are we gonna let them suffer and die because you can't make a pill of it and you might transfer a disease (are you sure that even this disease transfer could almost be avoided for 99 %)? I am convinced that if society really wants, this could be a safe med that reaches all PD patients. Seems to me that big pharmas are not willing to sell this product because it would be more profitable if it would have a pill form ???

Conductor71 · 07-25-2012, 09:00 PM

Diego,

My, you are a breath of fresh air! I believe this is how many of us feel but as patients who think that we are a huge untapped resource and seen by doctors and scientists, and all related organizations as cases or statistics, we rarely speak our mind. Not only are we rarely ever asked for our input; to be fair MJFF is an exception, but we know how we are perceived. We are afraid to step out of bounds because we are not taken seriously in the first place.

I get that as PWP we have a fairly self-centered sense of time, but wouldn't anyone whose near every movement depends on medication not be demanding a better process? We should not disregard safety, but the efficacy requirement of the FDA is ludricrous in our case given the unique situation with placebo. I really do not understand how some people sleep at night. It should not take the average 7-14 years of testing to see a drug come to market.

That being said, I have to stick up for MJFF a bit here. I have mixed feelings. I feel confident they are doing what they can but it is within the confines of the FDA, academia, pharma, etc. Is still don't ghet why MJFF cannot just break protocol and go for it. We have lots of well known people with this disease; why couldn't they fund a new approach to research that challenges the status quo. This is not that we ignore safety regulations, but there must be a faster way.

As for Dr. Schneider, We do not know how involved he is with pharma,so no fair to call out.

I do agree with you in principle though. Diego. The gist is that there are other meds that are still derived from animals. I think maybe researchers get hung up over delivery because having a med device or any "part" takes that much longer to jump through the ridiculous hoops of the FDA. Their restrictions are our problem. It costs pharma huge money to see a drug through to market.

07-26-2012, 06:39 AM

Quote:

Originally Posted by Conductor71

As for Dr. Schneider, We do not know how involved he is with pharma,so no fair to call out.

I am not calling out on Dr. Schneider. Maybe it seems I did because I made a wrong construction in my sentence. English is not my native language so sometimes I write things a bit differently than I want. I am not attacking anyone. I don't attack MJFF nor Dr. Schneider. See it as a broad general complaint.

Quote:

Originally Posted by Conductor71

I do agree with you in principle though. Diego. The gist is that there are other meds that are still derived from animals. I think maybe researchers get hung up over delivery because having a med device or any "part" takes that much longer to jump through the ridiculous hoops of the FDA. Their restrictions are our problem. It costs pharma huge money to see a drug through to market.

You know ... I might seem a bit harsh ... but I am just stating what I think. If scientist would notice a meteorite that would hit earth and destroy all life on this planet, they would do everything they could to deviate the meteorite from his normal path. Seems to me that this is more difficult than extract some ganglioside from sheep and pig. Do you know how many sheeps and pigs people eat each year ? I think people eat more sheep and pigs a year than the amount of pigs and sheep needed to extract ganglioside for PD patients. And given the fact that PD patients really suffer a lot, I think it is unethical to not provide these people ganglioside.

People can get disease from animals from it ... how big are the odds for this to happen ? And if this happens ... are people gonna die or can they be cured with some antibiotics ? So I am not convinced at all about this argument.

I work in the Netherlands. People are very direct. And being direct may give the impression, I am an ***hole. But you know ... being direct is also being honest. And it gives room for discussions. As for now, according to me the only reason ganglioside is not yet provided to PD patients is because pharmas don't want to sell it because it is not a pill form. If this is the case ... maybe pharmas shouldn't sell this, but another structure is needed to provide ganglioside to PD patients. Maybe local hospitals with their own sheep and pigs sponsored by government and PD patients (PD patients could stop "wasting" their money in meds only relieving symptoms and spend them on a disease modifying ganglioside); to give a quick idea (don't know whether this is possible).

Conductor71 · 07-26-2012, 07:22 PM

Quote:

Originally Posted by Diego24

You know ... I might seem a bit harsh ... but I am just stating what I think. If scientist would notice a meteorite that would hit earth and destroy all life on this planet, they would do everything they could to deviate the meteorite from his normal path. Seems to me that this is more difficult than extract some ganglioside from sheep and pig. Do you know how many sheeps and pigs people eat each year ? I think people eat more sheep and pigs a year than the amount of pigs and sheep needed to extract ganglioside for PD patients. And given the fact that PD patients really suffer a lot, I think it is unethical to not provide these people ganglioside.

People can get disease from animals from it ... how big are the odds for this to happen ? And if this happens ... are people gonna die or can they be cured with some antibiotics ? So I am not convinced at all about this argument.

I work in the Netherlands. People are very direct. And being direct may give the impression, I am an ***hole. But you know ... being direct is also being honest. And it gives room for discussions. As for now, according to me the only reason ganglioside is not yet provided to PD patients is because pharmas don't want to sell it because it is not a pill form. If this is the case ... maybe pharmas shouldn't sell this, but another structure is needed to provide ganglioside to PD patients. Maybe local hospitals with their own sheep and pigs sponsored by government and PD patients (PD patients could stop "wasting" their money in meds only relieving symptoms and spend them on a disease modifying ganglioside); to give a quick idea (don't know whether this is possible).

Diego,

I am grateful, not critical. At least that was not my intent.

I was just part of a small international group of people working on a project, and we made a similar observation. One of our members was German and she too said we are direct, so she could not understand the style of communication coming from a contractor in California. I see what you are saying. I think we as children are very direct but then learn all the nuances, read between the lines, passive-aggressive tone used by adults and without thinking we just perpetuate it. Not sure why we tend to do it; maybe to avoid confrontation?

Thanks for speaking up

Laura

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