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ViaCyte, Inc. Announces FDA Acceptance of IND to Commence Clinical Trial of VC-01™ Candidate Cell Re

Discussion in 'Research' started by Ellen, Aug 19, 2014.

  1. Ellen

    Ellen Senior Member

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    http://www.marketwatch.com/story/vi...cement-therapy-for-type-1-diabetes-2014-08-19

    [h=1]ViaCyte, Inc. Announces FDA Acceptance of IND to Commence Clinical Trial of VC-01™ Candidate Cell Replacement Therapy for Type 1 Diabetes[/h]

    Published: Aug 19, 2014 9:00 a.m. ET
















    HEADLINE2Company Plans to Immediately Initiate Phase 1/2 Clinical Trial
    SAN DIEGO, Aug. 19, 2014 /PRNewswire/ -- ViaCyte, Inc., a privately-held regenerative medicine company, announced today that the U.S. Food and Drug Administration ("FDA") has accepted the company's Investigational New Drug Application ("IND") for its VC-01™ candidate cell replacement therapy to treat type 1 diabetes. The company plans to promptly initiate a Phase 1/2 clinical trial which it believes to be the first ever clinical evaluation of a stem cell-derived islet replacement therapy for the treatment of patients with type 1 diabetes.
    "We are pleased to have received FDA acceptance for our clinical trial protocol and look forward to initiating this study shortly" said Paul Laikind, Ph.D., President and Chief Executive Officer of ViaCyte. "The commencement of this clinical trial marks a significant milestone for ViaCyte, as we begin studying the use of the VC-01 product candidate for the treatment of type 1 diabetes. Instrumental in our ability to achieve this milestone are JDRF, the leading global organization focused on type 1 diabetes (T1D) research, and the California Institute for Regenerative Medicine, a leading organization focused on advancing stem cell research and regenerative medicine. Both organizations have been strong supporters of the work we are doing at ViaCyte."
    ViaCyte's VC-01 product candidate consists of pancreatic progenitor cells, called PEC-01™ cells, which are derived from a proprietary human embryonic stem cell line, encapsulated in ViaCyte's proprietary Encaptra® device. When implanted under the skin, the PEC-01 cells are specifically designed to mature and further differentiate into insulin-producing beta and other endocrine cells that regulate blood glucose in a manner similar or identical to the islets that normally comprise the endocrine pancreas.
    The Phase 1/2 clinical study will evaluate the VC-01 product candidate directly in patients with type 1 diabetes who have minimal to no insulin-producing beta cell function. In addition to evaluating the safety of the product candidate in these patients, the study is designed to demonstrate the effectiveness of the VC-01 product candidate in replacing the lost endocrine function that is central to the disease. In an open-label, dose-escalating format, ViaCyte expects to enroll approximately 40 patients in the study at multiple clinical sites.
    A primary goal of this first clinical trial will be to assess the safety and tolerance of the VC-01 product candidate in patients with type 1 diabetes. In addition, insulin production in the study subjects will be evaluated by measuring C-peptide, a biomarker for insulin produced by beta cells that is expected to provide a sensitive measure of efficacy in these patients. As proposed, the trial would also evaluate clinically-relevant secondary endpoints related to the need for administration of pharmaceutical insulin to control type 1 diabetes and the incidence of hypoglycemia, a common side effect associated with pharmaceutical insulin usage.
    Type 1 diabetes mellitus (previously called juvenile diabetes) is a life-threatening chronic condition in which the pancreas produces little or no insulin, a hormone needed to allow glucose to enter cells to produce energy. It is typically diagnosed during childhood or adolescence, though it can also begin in adults. Though far less common than type 2 diabetes, which occurs when the body becomes resistant to insulin, type 1 diabetes affects several million Americans, according to the JDRF. Currently, there is no cure for type 1 diabetes. Standard treatment involves multiple daily injections of insulin and rigorous management of diet and lifestyle.
    About ViaCyte
    ViaCyte is a privately-held regenerative medicine company focused on developing a novel cell therapy for the treatment of diabetes. ViaCyte's lead product candidate, the VC-01 combination product, is based on the production of pancreatic progenitor cells derived from human pluripotent stem cells. These progenitor cells are implanted in a durable and retrievable encapsulation device. Once implanted and matured, these cells are designed to secrete insulin and other regulatory factors in response to blood glucose levels. The VC-01 combination product is being developed as a potential long-term diabetes treatment without immune suppression, and without risk of hypoglycemia or other diabetes-related complications.
    ViaCyte is headquartered in San Diego, California with additional operations in Athens, Georgia. The Company is funded in part by the California Institute for Regenerative Medicine and JDRF.
    Logo - http://photos.prnewswire.com/prnh/20121026/LA00871LOGO-a
    SOURCE ViaCyte
    Copyright (C) 2014 PR Newswire. All rights reserved


     
  2. sszyszkiewicz

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    40 candidates! That sounds just terrific.
     
  3. Ellen

    Ellen Senior Member

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    I just watched this video. Did they only trial this in STZ mice, and no other rodent model? I guess since they are using immunosuppression, they don't care as much about having a spontaneously autoimmune model?

    https://www.youtube.com/watch?v=M2Z3c4D6jRQ
     
  4. rgcainmd

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    I contacted ViaCyte to inquire about the possibility of enrolling my daughter in their clinical trial for the Encaptra device. They are only considering candidates between the ages of 18 and 55 at this time.
     
  5. mmgirls

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    I thought that this was a "without immune suppression", confused???
     
  6. sszyszkiewicz

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  7. mmgirls

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    that is pretty much what I say years ago at a JDRF conference when I learned of this company.

    I am so very hopeful for this to go thru trials, it is so very different and I might jump at it because it can be removed from the body.
     
  8. Don

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    I have grown skeptical of results from mice studies but the concept sure seems transferable to humans. The Encaptra device just needs to block the human killer Tcells. Interesting that normal mice bg is 160-200, no correction needed, lol.
     
  9. Ellen

    Ellen Senior Member

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    I'm probably confused. If there is no immunosuppression, this is even more confusing because if they only trialed it in a mouse that is not spontaneously autoimmune, it's an even more huge step to go from STZ mice to humans. JMO.
     
  10. Don

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    I suppose the trial was about proving that encapsulated immature human beta cells can thrive & mature and start producing insulin and maintain healthy bg levels. I guess Viacyte is already confident that their semi-permeable membrane will not allow killer Tcells to pass through. Maybe Tcells are bigger or the wrong shape to pass through ???
     
  11. Ellen

    Ellen Senior Member

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    But why test on STZ mice instead of NOD mice?
     
  12. Parody

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  13. Don

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    Tx for this well-composed writeup. So the device may work for up to 2 yrs. Fingers-crossed.
     
  14. joshualevy

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    I'm not saying it's a good reason, but STZ mice are much cheaper and easier to work with than NOD mice.
    STZ you just buy mice "in bulk" and inject them with a beta cell toxin when you want to work with them.
    NOD mice are specially bred mice (so more expensive) and they get autoimmune diabetes, but you don't know exactly when they will come down with it, and not all do. So you need to buy more than you need, and planning the timing of the study is more complex.

    From my point of view, I would say two things:
    First, if you are testing a device or a type-1 treatment, then STZ is a pretty good way to go. Because treatments only care if you have the disease or not (and not how you got it), and devices don't really interact with the immune system at all. Now encapsulated beta cells are like a device, but they might have immune system interactions. So NOD probably is better than STZ, but how much better? Second, and maybe more important: Once you start human trials, the results from your mice trial become much less important. So one way to look at this is that the STZ mice were good enough to get them to human trials, and that's all that matters. So even if NOD is better than STZ when they were doing mice work, now they're doing human work, so it's all in the past.

    Joshua Levy
     
  15. sszyszkiewicz

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    There are going to be 40 human beings walking around with this device soon. 40 is a decent number. Sounds like a big bet! I have been searching on clinicaltrials.gov for viacyte, but have not found anything regarding the details of the study. Does anyone know anything past the announcement?
     
  16. Parody

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  17. joshualevy

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    I still have not found a ClinialTrials.gov record for this trial. I've searched for "ViaCyte" and also for "VC-01". Does anyone have the NCT number?

    Joshua Levy
     
  18. Don

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    I guess Viacyte is still gearing up (and securing more funding). I have checked clinicaltrials.gov several times, too. Given the potential bg volatility if undergo this experimental device, it seems best to have CGM on board. Off topic but it is interesting how the San Diego area is a magnet area with Viacyte, Dexcom, and Tandem.
     
  19. Parody

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  20. Don

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