For background, please read my previous blogging on Osiris: http://cureresearch4type1diabetes.blogspot.com/search/label/Osiris
The PROCHYMAL treatment has been shown safe in several phase-I, II, and even III trials for several immune diseases, so they are trying it with type-1 diabetes. This is an adult (actually self) stem cell treatment. Since safety is established, they went straight to phase-II clinical trials. The company's description is this: "Prochymal is a preparation of mesenchymal stem cells specially formulated for intravenous infusion. The stem cells are obtained from the bone marrow of healthy adult donors."
The results were a total failure: no change in the primary end point (C-peptide after a meal), or in any of the secondary end points. No safety issues turned up. This was after 1 year, and they will continue to follow the patients for another year.
News coverage: http://www.thestreet.com/story/11362832/1/osiris-stem-cell-therapy-fails-diabetes-trial.html
BTW: most news coverage of type-1 research is superficial. They just repackage the press release, occasionally adding a quote or two from the company; sometimes not even that much. But this article in "The Street" is much better than that, and is fun to read.
Background on mesenchymal stem cells: http://en.wikipedia.org/wiki/Mesenchymal
Should We Give Up On Adult Stem Cells?
The short answer is "no". But the full answer is a lot more interesting, but will need to wait for another blog posting. The short version of my opinion is this: based on all the studies done so far, including this one, I don't think that just dumping a bunch of bone marrow stem cells into the body is likely to cure type-1 diabetes. But that does not apply to stem cells specifically tailored for insulin production, because they are quite different, and so far we have no experience with them in people. Remember that there are many different types of stem cells, and many different ways of differentiating stem cells, and many different uses of stem cells. So while there is considerable bad news for the simple minded idea of dumping in a bunch of bone marrow stem cells and expecting a cure, there are many other avenues still open. That is why I think I will need a whole blog entry to sort out possible stem cell cures.
Dr. Taback et al in Winnipeg Hope for Funding for Vitamin D Prevention Study
As part of my recent policy change to cover treatments designed to prevent type-1 diabetes, I'm starting to cover this potential clinical trial of Vitamin D. I previously blogged once on Vitamin D, here:
http://cureresearch4type1diabetes.blogspot.com/2010/08/cinnamon-and-vitamin-d.html
Dr. Taback has put in the paperwork to ask for funding for a large, prospective study to see if giving people Vitamin D will lower the rate of type-1 diabetes. The basic plan is to screen 60,000 babies to find about 5,000 at higher risk for type-1, and then give those babies about 2000 IU of Vitamin D per day (current suggested dose is 400 IU). And then follow them for years to see if fewer develop type-1.
Although no mechanism is known, a few studies [r3] have had promising results, so it makes a lot of sense to test this in a larger group. Previous promising studies are summarized in [r1,r2].
An older study [r4] showed that Vitamin D consumption during pregnancy was NOT associated with markers for type-1 diabetes. Although this [r5] study just published recently suggests that it is associated with type-1 diabetes. The newer study was of much higher quality than the older one for two reasons. First, it measured actual cases of type-1, not markers. Second, it measured actual Vitamin D levels in the person, while the older study had people fill out a questionnaire about diet (which is vastly less accurate).
News coverage: http://www.cbc.ca/news/health/story/2011/12/28/diabetes-type1-vitamin-d-chasing-cures.html
[r1] http://www.ncbi.nlm.nih.gov/pubmed/18339654
[r2] http://www.ncbi.nlm.nih.gov/pubmed/15671235
[r3] http://www.ncbi.nlm.nih.gov/pubmed/11705562
[r4] http://www.ncbi.nlm.nih.gov/pubmed/20369220
[r5] News: http://www.foodconsumer.org/newsite/Nutrition/Vitamins/low_prenatal_vitamin_d_type_1_diabetes_0117120243.html
Study: http://diabetes.diabetesjournals.org/content/61/1/175.short
OmniBio Will Start another AAT Clinical Trial
OmniBio is testing AAT, an anti-inflammatory drug, which the body makes naturally, and which is already FDA approved for people who have a rare condition where a person don't make enough of it on their own. They started with a 15 person, phase-I study, and about 11 months ago expanded to a 50 person study, which I would consider phase-II. They just announced that they will start another study for type-1 diabetes and one for Graft-vs-Host disease. That's good news, of course, but I'd be a little more excited if they announced the results from their initial 16 person study.
Previous blogging on AAT: http://cureresearch4type1diabetes.blogspot.com/search/label/AAT
Previous discussion of inflammation based cures: http://cureresearch4type1diabetes.blogspot.com/p/common-ideas-and-opinions.html
News article: http://www.marketwatch.com/story/omni-bio-to-conduct-new-human-clinical-trials-2012-01-13
Corporate web site: http://www.omnibiopharma.com/
Joshua Levy
All the views expressed here are those of Joshua Levy, and nothing here is official JDRF or JDCA news, views, policies or opinions. My blog contains a more complete non-conflict of interest statement.
Blog: http://cureresearch4type1diabetes.blogspot.com
Mice cures: http://t1dcuredinmice.blogspot.com/
News coverage: http://www.thestreet.com/story/11362832/1/osiris-stem-cell-therapy-fails-diabetes-trial.html
BTW: most news coverage of type-1 research is superficial. They just repackage the press release, occasionally adding a quote or two from the company; sometimes not even that much. But this article in "The Street" is much better than that, and is fun to read.
Background on mesenchymal stem cells: http://en.wikipedia.org/wiki/Mesenchymal
Should We Give Up On Adult Stem Cells?
The short answer is "no". But the full answer is a lot more interesting, but will need to wait for another blog posting. The short version of my opinion is this: based on all the studies done so far, including this one, I don't think that just dumping a bunch of bone marrow stem cells into the body is likely to cure type-1 diabetes. But that does not apply to stem cells specifically tailored for insulin production, because they are quite different, and so far we have no experience with them in people. Remember that there are many different types of stem cells, and many different ways of differentiating stem cells, and many different uses of stem cells. So while there is considerable bad news for the simple minded idea of dumping in a bunch of bone marrow stem cells and expecting a cure, there are many other avenues still open. That is why I think I will need a whole blog entry to sort out possible stem cell cures.
Dr. Taback et al in Winnipeg Hope for Funding for Vitamin D Prevention Study
As part of my recent policy change to cover treatments designed to prevent type-1 diabetes, I'm starting to cover this potential clinical trial of Vitamin D. I previously blogged once on Vitamin D, here:
http://cureresearch4type1diabetes.blogspot.com/2010/08/cinnamon-and-vitamin-d.html
Dr. Taback has put in the paperwork to ask for funding for a large, prospective study to see if giving people Vitamin D will lower the rate of type-1 diabetes. The basic plan is to screen 60,000 babies to find about 5,000 at higher risk for type-1, and then give those babies about 2000 IU of Vitamin D per day (current suggested dose is 400 IU). And then follow them for years to see if fewer develop type-1.
Although no mechanism is known, a few studies [r3] have had promising results, so it makes a lot of sense to test this in a larger group. Previous promising studies are summarized in [r1,r2].
An older study [r4] showed that Vitamin D consumption during pregnancy was NOT associated with markers for type-1 diabetes. Although this [r5] study just published recently suggests that it is associated with type-1 diabetes. The newer study was of much higher quality than the older one for two reasons. First, it measured actual cases of type-1, not markers. Second, it measured actual Vitamin D levels in the person, while the older study had people fill out a questionnaire about diet (which is vastly less accurate).
News coverage: http://www.cbc.ca/news/health/story/2011/12/28/diabetes-type1-vitamin-d-chasing-cures.html
[r1] http://www.ncbi.nlm.nih.gov/pubmed/18339654
[r2] http://www.ncbi.nlm.nih.gov/pubmed/15671235
[r3] http://www.ncbi.nlm.nih.gov/pubmed/11705562
[r4] http://www.ncbi.nlm.nih.gov/pubmed/20369220
[r5] News: http://www.foodconsumer.org/newsite/Nutrition/Vitamins/low_prenatal_vitamin_d_type_1_diabetes_0117120243.html
Study: http://diabetes.diabetesjournals.org/content/61/1/175.short
OmniBio Will Start another AAT Clinical Trial
OmniBio is testing AAT, an anti-inflammatory drug, which the body makes naturally, and which is already FDA approved for people who have a rare condition where a person don't make enough of it on their own. They started with a 15 person, phase-I study, and about 11 months ago expanded to a 50 person study, which I would consider phase-II. They just announced that they will start another study for type-1 diabetes and one for Graft-vs-Host disease. That's good news, of course, but I'd be a little more excited if they announced the results from their initial 16 person study.
Previous blogging on AAT: http://cureresearch4type1diabetes.blogspot.com/search/label/AAT
Previous discussion of inflammation based cures: http://cureresearch4type1diabetes.blogspot.com/p/common-ideas-and-opinions.html
News article: http://www.marketwatch.com/story/omni-bio-to-conduct-new-human-clinical-trials-2012-01-13
Corporate web site: http://www.omnibiopharma.com/
Joshua Levy
All the views expressed here are those of Joshua Levy, and nothing here is official JDRF or JDCA news, views, policies or opinions. My blog contains a more complete non-conflict of interest statement.
Blog: http://cureresearch4type1diabetes.blogspot.com
Mice cures: http://t1dcuredinmice.blogspot.com/
3 comments:
Thanks for keeping us up to speed Josh.
Thanks Josh. The Prochymal news is particularly disappointing and the press release indeed appears to be misleading by still labeling the treatment as a "breakthrough", though we should not underestimate the importance of the safety/tolerability achievement, particularly noting these are exogenous stem cells. Let's see how it evolves, interesting to note that the Osiris share price has recovered substantially after plunging on the day of the press release (January 3rd) and it is now significantly above the press-release value.
And To Whome Would you Leave Some $ to In your Will?
JDRF or ?
Thank you
Dennis
T1
@DennisIDDM@aol.com
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