Sunday, October 1, 2017
Important Update For Stem Cells Arabia
If you are interested in potential cures for type-1 diabetes, then I urge you to read this report from the JDCA about Stem Cells Arabia:
http://thejdca.org/pc-2017-stem-cells-arabia
(Note that as a Fellow of the JDCA, I did contribute to their report.)
Stem Cells Arabia is in the middle of a clinical trial were they combine two stem cell procedures as a possible cure for type-1 diabetes. However, the real excitement is fueled by the results of a very small pilot study they did previously. The results from the pilot study were presented at a conference, but not published in a journal. But those results are very strong: all four treated patients went months without needing to inject insulin and never needed anti-rejection drugs. If you find that exciting (and I certainly do), then you'll want to read the JDCA report which contains more details, and you'll be looking forward to the results of their larger trial, which is expected to complete in early 2019.
Joshua Levy
http://cureresearch4type1diabetes.blogspot.com
publicjoshualevy at gmail dot com
All the views expressed here are those of Joshua Levy, and nothing here is official JDRF or JDCA news, views, policies or opinions. My daughter has type-1 diabetes and participates in clinical trials, which might be discussed here. My blog contains a more complete non-conflict of interest statement. Thanks to everyone who helps with the blog.
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5 comments:
The lack of transparency does not allow us to be so optimistic.
We must all keep our fingers crossed and, where appropriate, perhaps pray.
You seem like a down to earth guy, beats me why you have this strong believe in this small pilot study which is not published. Can you shed some more light on why the positivism?
Lost contact??? Consider patent (why not submit and publish the day after?). "Three of the patients were able to stop using insulin altogether and remained insulin independent for two full years before Al-Zoubi lost contact with them."
Faustman already demonstrated that if the autoimmune attack on the beta cells is stopped, they don't recover spontaneously to any clinically significant extent. She hoped INGAP polypeptide would stimulate regrowth, but it didn't work, and the search is still on. So the best this study could do would be to accomplish stage one of a necessarily two-stage process, with no guarantee that it is possible even in principle to accomplish stage two.
This is mostly a reply to cj:
I'm positive about this research for several reasons. But before I list them, I do want to be clear: I don't know if this research will be successful. No one does, and most research ends up failing. That's just the nature of research: it's uncertain. The reason I'm positive about this is that I think it has a better chance than other research I know about. Why? Because:
* The results seen so far (effectiveness and safety together) are the best actual results ever seen (In my opinion). Having people not using injected insulin for months, sometimes years (from a treatment which does not require long term immune suppression) is big.
* The future is clear and quick. They are running a trial which should give us a very clear signal about the success of the research. Those results will be known in 2-3 years, which is not that far away.
Obviously, there are downsides, which should not be ignored, (speaking at a conference rather than publishing in a journal, for example). But, for me, taken as a whole, this is the best we've got, and worth being positive.
In particular, I want to say two things about looking track of patients after two years:
* First, most trials follow people for two years or less. Yes, there are a few that go longer, but frankly, going shorter than 2 years is more common than going longer. But in any case, 2 years is the longest common follow up time for an intervention study like this. So while we all can wish the did longer (and complain that they did not), two years is a long time compared to other studies.
* Second, this four person study was a pilot study. It was just to see if the real study could be done. I could not find a clinical trial record for it. It was obviously exploratory in nature. It does not look (to me) like the had any predefined endpoints of success, or a predefined time range to conduct the study. In a sense, that is why we don't have a lot of details on the study and (I suspect) it is why they have lot track of most of the participants. But at this point, there is nothing to be done but to wait for the results from the Phase-I/II study. Yes, all of these things are potential problems, but none are fatal to me.
I do hope they publish more soon. With most research, I hope for better results. With this research, I hope for better publications. I think it is much easier to improve your publications than to improve your results! Since their clinical trial will end in early 2019, if they haven’t published good things by 2020, then that would be a very bad sign. But that is only 2-3 years away, and I am an optimist.
This is a reply to two of Oscar's quotes:
Oscar wrote: "Faustman already demonstrated that if the autoimmune attack on the beta cells is stopped, they don't recover spontaneously to any clinically significant extent."
I disagree completely. Nothing in Dr. Faustman's results lead me to think she has stopped the autoimmune attack. The changes she reported in immune cells were tiny, and there is no evidence that they would have lead to a stop in the autoimmune attack.
Oscar wrote: "She [Dr. Faustman] hoped INGAP polypeptide would stimulate regrowth, but it didn't work, and the search is still on."
I don't think Dr. Faustman has ever used INGAP. If you think she has, then please post a reference to it. In mice, she tried a spleen cell transplant, and that was unsuccessful in improving things, but in humans she has never used INGAP (or spleen cells, for that matter). I agree that other researchers have tried to use INGAP to grow new beta cells, and that research as been unsuccessful so far. There is still on small trial ongoing, as far as I know, but several larger ones have been unsuccessful, so I don't hold out much hope. (Although I am an optimist in general, multiple large trials which have been unsuccessful will dampen my enthusiasm.)
Finally Oscar wrote: "So the best this study could do would be to accomplish stage one of a necessarily two-stage process, with no guarantee that it is possible even in principle to accomplish stage two."
No. The JDCA summary is quite clear: these researchers are using two different stem cell treatments. One of those treatments is designed to regrow beta cells, the other is designed to stop the autoimmune attack. If both treatments succeed (a big "if"), this could be a complete solution.
Joshua Levy
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