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DBL%20Hendrix%20small.png College chemistry, 1983

Derek Lowe The 2002 Model

Dbl%20new%20portrait%20B%26W.png After 10 years of blogging. . .

Derek Lowe, an Arkansan by birth, got his BA from Hendrix College and his PhD in organic chemistry from Duke before spending time in Germany on a Humboldt Fellowship on his post-doc. He's worked for several major pharmaceutical companies since 1989 on drug discovery projects against schizophrenia, Alzheimer's, diabetes, osteoporosis and other diseases. To contact Derek email him directly: derekb.lowe@gmail.com Twitter: Dereklowe

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In the Pipeline

« 2012 In Startups | Main | The Changing Literature »

January 4, 2012

Osiris And Their Stem Cells

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Posted by Derek

The topic of whether stem-cell therapies are overhyped - OK, let me show my cards, the topic of just how overhyped they are - last came up around here in November, when Geron announced that they were getting out of the business. And yesterday had a good example of why people tend to hold their noses and fan away the fumes whenever a company press-releases something in this area.

I'm talking about Osiris Therapeutics, who have been working for some time on a possible stem cell therapy (called Prochymal) for Type I diabetes. That's certainly not a crazy idea, although it is an ambitious one - after all, you get Type I when your insulin-producing cells die off, so why not replace them? Mind you, we're not quite sure why your insulin-producing cells die off in the first place, so there's room to wonder if the newly grown replacements, if they could be induced to exist, might not suffer a similar fate. But that's medical research, and we're not going to figure these things out without trying them.

This latest work, though, does not look fit to advance anyone's understanding of diabetes or of stem cells, although it might help advance ones understanding of human nature and of the less attractive parts of the stock market. Osiris, you see, issued a press release yesterday (courtesy of FierceBiotech) on the one-year interim analysis of their trial. The short form: they have nothing so far. The release goes on for a bit about how well-tolerated the stem-cell therapy is, but unfortunately, one reason for that clean profile might be that nothing is happening at all. No disease markers for diabetes have improved, although they say that there is a trend towards fewer hypoglycemic events. (I think it's irresponsible to talk about "trends" of this sort in a press release, but such a policy would leave many companies without much to talk about at all).

It's only when you look at Osiris and their history that you really start to understand what's going on. You see, this isn't Prochymal's first spin around the track. As Adam Feuerstein has been chronicling, the company has tried this stem cell preparation against a number of other conditions, and it's basically shown the same thing every time: no adverse effects, and no real positive ones, either. Graft-versus-host disease, cardiac events, cartilage repair, Crohn's disease - nothing happens, except press releases. You'd never know anything about this history if you just came across the latest one, though. The company's web site isn't a lot of help, either: you'd think that Prochymal is advancing on all fronts, when (from what I can see) it's not going much of anywhere.

So if you're looking for a reason to hold on to your wallet when the phrase "stem cell therapy" comes up, look no further. The thing is, some stem cell ideas are eventually going to work - you'd think - and when they do, they're going to be very interesting indeed. You'd think. But are any of the real successes going to come out of fishing expeditions like this? You don't want your clinical research program to be so hard to distinguish from a dose-and-hope-and-sell-some-stock strategy - do you?

Comments (13) + TrackBacks (0) | Category: Biological News | Business and Markets


COMMENTS

1. anchor on January 4, 2012 10:09 AM writes...

Osiris Therapeutic policy in addition to everything posted on their site ..is also to believe in tooth fairy and also to keep hope alive that there is one! Seems to me that stem-cell based therapy and the companies that have been floated are similar to dot.com companies of nineteen nineties! Many will go belly up and a handful will survive.

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2. johnnyboy on January 4, 2012 10:37 AM writes...

To postulate that you just have to inject some stem cells intravenously and they'll magically get themselves into the pancreas, form islets and start producing and releasing insulin and glucagon into circulation in a physiologically functional manner, you'd have to be either an idiot, psychotic, or a crook. In the case of Osiris, I'd vote for the latter.

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3. Anon on January 4, 2012 10:45 AM writes...

Osiris was the ancient Egyptian god whose annual death and resurrection personified the self renewing vitality and fertility of nature. Everything good comes from Osiris. This includes order, harmony, and the celestial periods.
Relax...... Everything will be OK

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4. ech on January 4, 2012 1:46 PM writes...

I agree that stem cells are as over-hyped as nanotech is, joining artificial intelligence and other buzzwords in attacting capital and press.

However, I thought there was a small human trial done at a university hospital that showed excellent results in using stem cells in heart attack patients. IIRC, left ventricle ejection fraction went up 12% after 1 year. The stem cells were said to have been placed in situ via catheter and not injected. Yes, it's a long way from small trials to routine clinical use, but this study seemed pretty solid.

My daughter just got a position at a major cardiac research center that is also working on a stem cell therapy for heart attack damage, but they are still in animal trials.

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5. emjeff on January 4, 2012 2:04 PM writes...

The best biomarker for a worthless drug is a flurry of indications. When you start testing it in Alopecia, Bursitis, Cough, Dementia... it is time to quit.

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6. Jim H on January 4, 2012 10:51 PM writes...

Prochymal for blood related disorders is certainly a good approach and likely very effective therapy.
I agree that the "hype" is far greater than the potential, in many cases. You are missing the link with type 1 diabetes (or maybe I am). They are looking at MSC's immunosupressive properties. Since Type I Diabetes can be classified as an "autoimmune" disorder, MSC are know to be effective modulators of immune response, so they not looking at engraftment as an end point.

That said, I think they are stretching the boundaries of what we know about the immunomodulatory elements of BM derived MSCs (if we can even define them in finite terms). I think it will eventually be boiled down to the "small molecule" mechanisms you pharma folks are obsessed with, but until we understand the lymphatic system, MSCs may be the best weapon we have.

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7. T on January 5, 2012 2:22 AM writes...

There are certainly a lot of stupid stem cell "treatment strategies" being tried in areas where we don't yet have the knowledge to attempt anything more sensible, but that doesn't mean that the whole topic is hype with nothing useful to show for itself. There is at least one form of stem cell treatment that is proven effective and has been in clinical use for many years: bone marrow transplantation. And where this intercepts with gene therapy (ie isolating hematopoietic stem cells from the patient, correcting the genetic defect and then transplanting them back, rather than using cells from a healthy donor, for which good matches are hard to find, and which leads to a risk of graft versus host disease), there have recently been a number of succesful clinical trials, where it was possible to cure life threatening genetic disieases of the hematopoietic system like X-linked severe combined immunodefficiency (X-SCID).

http://www.ncbi.nlm.nih.gov/pubmed/21624615

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8. MStudent on January 5, 2012 10:37 AM writes...

I thought the same thing when I read the news bit - sure, lack of adverse effects is good news for a phase I, but it's not something that you can call "groundbreaking". Osiris has been really unlucky with this product. TBH I get the impression that after it failed to provide results in it's original indications, they began to run it against everything and anything to see if they can actually market it for some indication.

(To be fair, there are cell therapy companies out there which are getting results -so far-, though)

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9. Jason on January 5, 2012 11:51 AM writes...

I was in a Prochymal trial for Crohn's, and I can say for certain that it had major positive effects. Just because it hasn't worked for diabetes doesn't prove anything against it to me after it lowered my C-Reactive protein count from 65 to 16, without the side effects of crap like Prednisone and Imuran (two horribly overused drugs) and also made my severe psoriasis completely disappear. One clinic miscalculated some results, and that protocol was canceled, which was probably a black day for Osiris, but so what -- I saw a big difference. Certainly diabetes is probably the wrong fix to try, but I'd use it again right now for my out-of-control inflammation.

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10. Henrik Olsen on January 5, 2012 1:49 PM writes...

At least they're not like the Ukrainian quack company Emcell, who claim to use stem cell therapy to cure just about everything including Alzheimer's, ALS, cancer, diabetes, HIV/AIDS and Parkinson.

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11. anonymous on January 5, 2012 9:55 PM writes...

"nothing happens, except press releases"

Good one, Derek, I love it! OK, 2012 has only just begun, but that line will still probably qualify for one of my favorite quotes of the year by December.

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12. Jim on January 6, 2012 1:02 PM writes...

My understanding was that this was not a trial to replace insulin producing cells, but to stop further beta cell destruction by immune cells in the early stages of the diseases. This would make sense because the company has been pushing the anti-autoimmune effects in their recent trials. Please correct me if I am wrong in my assumption.

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13. agentsmith on January 13, 2012 4:45 PM writes...

Well. I understand Derek's bias towards a pharmacentric model, and that audience of this blog is mainly (not only) medchemists and other pharma-related scientists. So I rarely comment here.

But this one. This is like saying gene therapy is flawed as a concept and should 'never' be tried because it is too dangerous. Like saying that the internet is useless for business/commerce after the dot com bust. The mechanism of stem cell therapeutic action (in cases they are effective) is quite complex and very poorly understood. A lot needs to be done. Even so, we may be able to find stem-cell based cures before understanding everything. But it is safe to say it will be a long time before a cocktail of small molecules with sufficient amount targeting will prove equivalent.

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