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SURAMIN AUTISM TRIAL UPDATE!

Discussion in 'Other Health News and Research' started by Murph, Mar 8, 2018.

  1. Murph

    Murph :)

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    https://tacanowblog.com/2018/03/08/suramin-autism-trial-update/

    SURAMIN AUTISM TRIAL UPDATE!
    Posted by Lisa Ackerman on March 8, 2018
    By Gita Gupta

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    Last year, we posted about Dr.Robert Naviaux’s success with the 100 year old drug suramin in a small double-blind, placebo controlled trial of children with autism. All the children who received a single dose of suramin showed improvements in the core symptoms of autism. You can read the parent stories of improvements in their children here, but these are some of the improvements that were seen –

    · new language

    · longer and more complex spontaneous sentences

    · improvements in voice quality

    · improved appetite

    · less picky eating

    · interest in trying new things

    · greater resilience to changes in routine

    · increased social interaction with other children including playing games.

    This study was small due to a lack of funding. Funding was entirely from private donations. Larger, carefully designed clinical trials are needed to replicate the results, learn how to use low-dose suramin safely in autism, identify drug interactions as well as detect rare side effects and collect all the data that the FDA requires to approve the use of suramin in autism.

    After the success of the initial small trial, we had hoped it would be quick and easy to raise money to fund the path to FDA approval. As some of you know, the road has been much longer and rockier than anyone expected. However, we finally have good news! Read on for an update.

    Suramin Autism Trial Update – Q&A with Dr. Naviaux
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    What is the status of the suramin autism trial(s) – has funding been found? If so, when will the trials start?
    Dr.Naviaux: I am very happy to share that a biotech company, backed by very reputable investors with a proven track record, has agreed to make suramin and fund the next autism trials.

    Unfortunately, Bayer decided not to provide suramin for the proposed clinical trials in autism. So, the biotech company that will be funding the trials has agreed to manufacture suramin. They will also collect all the data that the FDA needs in order to approve the drug for use in autism.

    It will take about a year for suramin to be available from this biotech company. Even factoring in the year that it will take for suramin to be ready, we expect that this will be a much faster and smoother path to FDA approval than attempting to fund each step through charitable donations.

    The company will make a public announcement when suramin is available for the next clinical trials. (Per the legal agreement that the Naviaux Lab has with this company, their name cannot be disclosed until then.) The suramin autism treatment 2 (SAT2) trials will start as soon as suramin is available.

    We are hopeful that the new supplier of suramin will, in the long run, offer a simpler path forward and fewer potential delays in testing. If future clinical trials show that suramin is safe and effective in treating autism, then the FDA will have all the data they need to make a decision on approval in a shorter period of time than before. Ultimately, this means that the drug can become available for patients with autism or ME/CFS faster than was possible before.

    We appreciate your support on the long road we traveled trying to raise funds for this trial. We are very grateful for all your donations, which have helped support the lab while this important agreement was being worked out.

    Can you describe the trials that are planned? How many patients, what age, what gender etc.? Will there be a multi-dose trial or just the bigger trial?
    Dr.Naviaux: Several clinical trials will be needed. The first two will test a few doses of suramin given monthly for a few months. These will be done in about 50 boys, ages 5-17 years old. The exact ages have not yet been decided. After that, a study will be done that will include girls with ASD. Other studies will look at drug-drug interactions so we know if suramin interacts with drugs that are commonly used in ASD. The last clinical trial that the FDA will need will be a Phase III, multicenter trial in 200 or more participants. The multicenter trial will test the safety and efficacy of several doses of suramin given monthly for several months.

    Will suramin be available on a compassionate use basis prior to FDA approval?
    Dr.Naviaux: This is not known yet. The safety and efficacy of several doses of suramin must be shown first.

    How long will it take to get FDA approval?
    Dr.Naviaux: This will depend on the results of the next few studies. In principle, it usually takes about 5 years to complete all the needed studies after the first feasibility study. This would include the time to conduct and analyze the results of the required multicenter, Phase III study. If the next studies showed that suramin was exceptionally safe and effective at the doses used, this time might be decreased by a year or two.

    Here’s to smooth sailing ahead
    After many roadblocks and funding challenges, we are happy that a path has finally emerged for suramin to be extensively tested in autism! Here’s wishing Dr. Naviaux and his biotech partners a smooth and quick path for testing suramin, and if proven safe and effective, winning the FDA approvals needed to make it widely available to children with autism.
    Want to learn more about suramin in autism? Check out the Naviaux Lab page on autism research. http://naviauxlab.ucsd.edu/science-item/autism-research/
     
  2. Murph

    Murph :)

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    This is terrific. But not fast! All the parents of autistic kids out there who are helping this trial along are really investing in helping the next generation of autistic kids.

    A year for suramin. An unknown amount of time for the first feasibilty study. Plus five years to finish the Phase III. I'm thinking 2026 at the earliest for results to be known.

    And as we know all too well, at the end of the Phase III you can often be left with disappointment. (I do fear with Naviaux that chance of disappointment might be even higher . He has a radical new conception of chronic disease, which to me seems like a high-risk, high-reward kind of science, rather than boring incremental science. If he's right, it is an end to a lot of suffering and Nobels akimbo. But reward comes with risk. I like his logic and it makes sense to me, but I know that a good hypothesis and a good result are oceans apart.)

    Still, this is the exact and only way progress is made. So fingers crossed!
     
    sb4, Sing, Learner1 and 4 others like this.
  3. hixxy

    hixxy Senior Member

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    I wonder why it takes so long? It's not like they need to prepare to mass produce suramin.
     
  4. Runner5

    Runner5 Senior Member

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    PNW
    My Vegas bet is they'll raise a whole lot of money and a few people walk away mysteriously owning private islands.
     
  5. anni66

    anni66 mum to ME daughter

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    Why only boys?
     
  6. Neunistiva

    Neunistiva Senior Member

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    They need to figure out how to make generic suramin and set up production, I assume. There's a saying, that it costs a billion to produce the first pill, and 10 cents to produce the second. I assume it's the same with time.

    ASD is diagnosed more often in male patients than female. It could be it's easier finding boys that fit the criteris, or maybe they want to keep variables down to a minimum. If boys and girls have different reaction to suramin it would muddy the results of research.
     
    sb4 and Murph like this.
  7. anni66

    anni66 mum to ME daughter

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    Yes, but again the differences in responses etc between gender not studied. Difficult to be able to extrapolate results .
     
  8. Neunistiva

    Neunistiva Senior Member

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    "After that, a study will be done that will include girls with ASD." It's in @Murph 's post
     
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  9. anni66

    anni66 mum to ME daughter

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    Thanks - not much sleep this week.
     
    Neunistiva likes this.
  10. hixxy

    hixxy Senior Member

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    That's for drug development not manufacturing a generic.
     
  11. Wally

    Wally Senior Member

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    Does anyone know when Bayer’s suramin drug went off patent?

    I know it is a drug that has been around for a long time, so I was wondering why no other company has made a generic form of the drug? Not enough profit/need for the drug?

    I have not been able to find any independent information from Bayer (press releases, annual statements) where Bayer has publically stated that it will discontinue manufacturing this drug. Has anyone seen any public statement from Bayer on this subject?

    Has Bayer already discontinued manufacturing this drug? If yes. when did manufacturing of the product stop? Or are prior statements about Bayer stopping manufacturing of this drug incorrect?

    I did find information about Bayer supplying this drug to the W.H.O. for African Sleeping Sickness at http://www.who.int/trypanosomiasis_african/partners/bayer/en/

    I was interested in trying to get more information on why manufacturing of this drug was something that Bayer decided to stop manufacturing. If Dr. Naviaux has hit upon a possible new use for this drug in treating Autism, why would Bayer not want their drug to be available for (potentially) a very large worldwide market? Also, what company (if any) will be supplying a drug (with similar efficacy to Bayer’s suramin) for African Sleeping Sickness?

    It just feels like there is more to this story and I was wondering if anyone else shares my curiosity about the manufacture and availability of Suramin?
     
    Last edited: Mar 9, 2018
  12. Neunistiva

    Neunistiva Senior Member

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    Yes, I know it's not exactly the same, research is not calculated into the price and timefrime (which is why it will take a year, not decades), I was just trying to illustrate a point.

    Try imagining what it takes to set up a factory to produce a certain chemical compound based on a formula. That's the part that takes a long time. Once the factory is making it, whether they will produce 10 or a million makes a difference of days. It's the part before the production that takes time.
     
  13. Neunistiva

    Neunistiva Senior Member

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    AFAIK it's a drug that's used for a very narrow purpose, as you said yourself, African sleeping sickness. Bayer is giving it away for free there. No incentive.

    Why do you think they stopped? This is all it says "Unfortunately, Bayer decided not to provide suramin for the proposed clinical trials in autism. "
     
  14. Wally

    Wally Senior Member

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    @Neunistiva - I had thought I had seen the subject of a manufacturing change by Bayer for this drug mentioned in another article or in another thread. However, I could not readily locate such a reference, so I added a question asking if Bayer had actually stopped manufacturing the drug.

    When I read the recent Q & A with Dr. Naviaux, he only says that “Bayer decided not to provide suramin for the proposed clinical trials in autism”. No mention is made of Bayer no longer manufacturing this drug, so I was a bit confused not knowing if there was an issue with Bayer’s manufacturing of the drug or not.

    So, if the drug is still being manufactured by Bayer, here are my questions for any PR members who might have answers to these questions? If no one responds with answers to these questions, then perhaps I will contact Dr. Naviaux to see if he might be willing to answer my questions.

    1) Why is Bayer not interested in providing their drug for these proposed autism clinical trials?
    2) Is Naviaux asking for the drug to be provided for free?
    3) What is the cost of the drug? I was under the impression that it is a relatively inexpensive drug.
    4) Does a drug manufacturer have to sign off on the use of one of its drugs, if the drug will be used in an outside clinical trial?
    5) Are there additional costs (time and resources) that would be incurred by a drug manufacturer (besides the cost of supplying the actual drug) for a clinical trial? In other words, is there more involved for the manufacturer than just supplying the drug?
    6) Do these types of clinical trials always have to be a joint project between the drug manufacturer and the outside researcher? If yes, why?
     
  15. Neunistiva

    Neunistiva Senior Member

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