Discussion in 'Parents of Children with Type 1' started by Brenda, Oct 29, 2014.
I am SO excited about this! If this encapsulation device works, I'd be happy enough to call it a cure. Even if my daughter had to have a new device implanted every two years (or more often), this beats the AP and BP hands down IMO. I wouldn't expect it to be commercially available for another 10 - 15+ years or so, however.
I agree. Much more excited about this stem-cell stuff than the bionic pancreas. Why do you think 10-15 years though? I thought being in human trials was a big deal and put this lightyears ahead of other biologic treatments/cures.
Does anyone know if there is immuno-suppressant therapy involved in this?
They just began Phase 1 trials, if I'm not mistaken. Have no idea how long these will last. Am pretty sure they will need to complete Phase 2 and perhaps even Phase 3 trials before the FDA approval process can begin. And, for better or worse, we all know how long that could take. I truly hope I am overestimating when I say 10 - 15 years...
That's the whole idea behind the semi-permeable membrane: so that no immunosuppressant therapy will be needed.
It is something to hope for. Apparently the implant has cells that evolve into islets/beta/alpha cells. In that immature state they do not require as much oxygen to start, giving the host a chance to vascularize the device as the cells mature.
I've copied the following from tudiabetes (I hope this is O.K.) and posted it here because it's one of the more concise and understandable pieces I've read about ViaCyte's VC-01TM device. Hopefully it goes it a little further than my answer above in explaining why it may be quite a few years before this device is available to the general T1D population. I also wanted to acknowledge jenm999's comment "I thought being in human trials was a big deal and put this lightyears ahead of other biologic treatments/cures" and say that I believe you are right, that the fact that a human trial has started does put the VC-01TM quite a ways ahead in the quest for better treatments/cures.
JDRF-Funded Islet Encapsulation Program Reaches Historic Milestone
Posted by Diabetes News and Interest on October 29, 2014 at 5:28pm in General Diabetes Topics and Pre-DiabetesSend Message View Discussions
First Ever Person with Type 1 Diabetes Receives Experimental Encapsulated Human Stem Cell-Derived Beta Cell Replacement Therapy
New York, NY, October 29, 2014 — JDRF, the leading research and advocacy organization funding type 1 diabetes (T1D) research, announced today that JDRF-funded partner, ViaCyte, Inc., a leading regenerative medicine company, has for the first time ever implanted a person with type 1 diabetes (T1D) with an experimental encapsulated cell therapy product candidate, called VC-01TM, which is being developed for the treatment of T1D. This individual, and others to follow, is participating in a trial to evaluate the safety and efficacy of the VC-01 product candidate, a potential replacement source of insulin-producing cells.
“JDRF is excited about the historic research advance of this encapsulated cell replacement therapy for type 1 diabetes,” said Derek Rapp, JDRF president and CEO. “Encapsulated cell therapies have the potential to fundamentally transform the lives of people with type 1 diabetes by restoring a person’s independence from insulin injections and reducing concerns of the complications that arise from living with the disease. We are enthusiastic about the progress of research in this field, and we look forward to the full enrollment in the trial and to results from this study which could move us one step closer to a new and effective treatment for type 1 diabetes. This day would not have been possible without the basic stem cell research and the insights learned to protect insulin-producing cells from the autoimmune attack that JDRF has been funding for years.”
The study of ViaCyte’s experimental therapy, VC-01, is initially being conducted at the University of California at San Diego (UCSD). The experimental product encapsulates immature human islets derived from a stem cell source in an immune-protective device called the Encaptra® drug delivery system. At first, a small group of people in the study will be followed for several months to ensure the safety of the therapy. After the initial evaluation of this first group of participants at the UCSD site, it is expected that additional sites will be activated to enroll more people in early 2015.
The primary goal of this first study is to evaluate the safety of the VC-01 product candidate in people who have had T1D for at least 3 years; not to make them insulin independent. However, the study is also designed to evaluate the effectiveness of the VC-01 product candidate in replacing the lost insulin-production function that is central to T1D, by measuring C-peptide levels as a marker of insulin production. This study will provide researchers with critical information about the functioning of the encapsulation device at the implantation site and about the maturation and survival of the implanted cells.
Dr. Paul Laikind, President and CEO of ViaCyte, said, “Treating the first patient with our stem cell-derived islet replacement product candidate is an exciting next step in our quest to transform the way patients with type 1 diabetes are impacted by the disease. Moving from a promising idea to a new medicine is a long and challenging journey and we are grateful to JDRF, and all its supporters, for the tremendous and continued support they have provided. At ViaCyte we have been working over a decade to reach this milestone, and JDRF has been there every step of the way, providing critical funding, excellent advice, and advocating on our behalf.”
People in the study will go about their daily lives with the implanted product for up to two years. They will be closely monitored by clinicians during this time. The encapsulated cell therapy here involves putting cells with the potential to mature and produce insulin in a protective barrier and implanting them in the body using a minimally invasive procedure. Once matured these encapsulated immature human islets might sense a person’s glucose levels and produce insulin while the barrier shields them from the body’s T1D autoimmune attack as well as foreign graft rejection. If they perform as they have in animal studies, the cells will continuously assess the amount of glucose in the blood and release the appropriate amount of insulin. Importantly, encapsulated cell replacement therapies have the potential to overcome the major obstacles that have limited the use of human islet transplantation in people with T1D: limited donated islets and the need for lifelong administration of immunosuppressive drugs to prevent destruction of the newly introduced islets. Stem cell-derived islet sources may represent an unlimited supply of replacement insulin-producing cells and the protective devices eliminate the need for immunosuppressive drugs.
JDRF is the leading global organization focused on type 1 diabetes (T1D) research. JDRF’s goal is to progressively remove the impact of T1D from people’s lives until we achieve a world without T1D. JDRF collaborates with a wide spectrum of partners and is the only organization with the scientific resources, regulatory influence and a working plan to bring life-changing therapies from the lab to the community. As the largest charitable supporter of T1D research, JDRF is currently sponsoring $568 million in charitable research in 17 countries. For more information, please visit www.jdrf.org
I was wondering if Joshua would be willing to venture an educated guess as to when (if all goes well and this device performs as hoped) he believes this might be available to the general population. I realize that there are potentially more hurdles and roadblocks than we can even begin to imagine, BUT if someone held a gun to your head, Joshua, and made you place a bet on when this device might be approved and on the market, what would you answer? If you are not comfortable venturing a guess and answering this question, I understand and respect your decision.
FWIW-in future, rather than wholesale copying of another's blog post, it might be better to simply provide a link.
Guessing about when something will be available is easy; it's being right (even close to right) that is hard. :smile:
I think your 10-15 year guess is the same as I would make. Since no cure has ever gone though the process, we have no idea how long it will take, but 10-15 years is as good a guess as I can make. As to why, here is a quick summary: Phase-I, Phase-II, and Phase-III trials (about 3-4 years each), and marketing approval (about 1-2 more years). I would make the exact same guess about any treatment that is starting phase-I trials.
It's hard for the approval process to move any faster. The only way it could is:
* If the treatment were already approved for another use (so could be prescribed "off label").
* If the treatment has already done phase-I for another disease, so it could go straight to phase-II.
* If you were willing to travel to a less regulated foreign country.
* If you could market it as a "dietary supplement", in which case the FDA doesn't test/approve it.
Obviously, none of these apply to Viacyte's VC-01.
Remember that 3-4 years for each phase is start to start. People will sometimes say things like "they are only testing it for a year in phase-I trials, so it will be much faster. But it won't. For starters they need to recruit people, they don't have 10 people waiting to start their trial on day one. (Well, not usually.) So even if the trial only runs for a year, it will often take them a year to recruit all the people. Then they need to analyse the data, get it published, and (most time consuming) figure out the design of the next phase. That usually takes more than a year as well. Of course, if they need to raise money for the trial, that can take much longer. So the clinical trial that collects data for one year, actually takes 3 years to complete to the point of starting the next phase. For type-1 diabetes, phase-III trials often involve 300 people. Even if you are recruiting at 10 or even 20 cities, it can take over a year to fully enroll a trial like that. Also for phase-III, you must do two trials of that size. Now you can run them at the same time, but you still need to wait until the second one is complete, before getting marketing approval.
Now, lets take a look at this clinical trial they are starting for VC-01. Technically, they are calling it a phase-I/II trial. I have not seen the official protocol for this trial, but I think I heard through the grape vine that they are talking about 40 or 50 people total. Usually what this means is that they are going to treat about 10 people, look at that data, assuming there are no safety issues, treat the rest of the people. The first part is the phase-I part and the second part is the phase-II part. Their Clinical Trial Record says they hope to finish in August 2017.
Now, the optimists reading that will say (no, they will shout): but even if it takes them a year to organize the phase-III, they'll be starting that in late 2018, which is only 4 years away, not 6 like you said it would take them! On paper, that might be true. But in real life, no. They won't finish a phase-II trial by August 2017. They won't be ready to start a phase-III in late 2018. Medical clinical trials just don't move that quickly, or that smoothly. And that date assumes that the phase-I/II trial is so successful, both as a phase-I trial and as a phase-II that there is no need to run a repeated phase-I trial or a separate phase-II trial. It's just not going to happen that way; I'm speaking as a realist.
Consider LCT. LCT developed an encapsulated stem cell device, in many ways similar to VC-01. LCT started their first phase-I trial in June 2007. Now, 7 1/2 years later, they are conducting phase-II trials. (Results to date have not been "cure level". People who go their device often used much less insulin, and were much more stable, but few of them went "insulin free" for any length of time, and those that did, typically only did for a few weeks.) In any case, in terms of speed, these guys are even slower than 4 years per phase.
Finally, in terms of who is ahead of who, ViaCyte is in the middle of the pack of leaders:
LCT is in phase-II trials, but the results so far have not been cure level.
A research group at Hotel St. Luc has finished phase-I trials (I think), but results were not good (I think).
AZ-VUB: is in phase-I trials, but I know very little about it. I'm behind in my research. I always am.
Viacyte just started phase-I, and so there are no results to see.
Beta-O2 just started phase-I, and so there are no results to see.
Behind those guys are DRI and Serova who are testing infrastructure which could end up being part of an encapsulated beta cell device, and also a large group of companies and researchers who are testing similar devices in animals. Examples include Islet Sheet Medical, Nuvilex, Mellon's group at Harvard, the Chicago Project, and possibly many more. (Remember, I don't follow animal research in detail. There is way too much of it, and it is too far away from general availability.)
I know this posting is a little long, but I hope it was helpful.
Thank you, Joshua; your post was very helpful in putting this all in perspective.
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