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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

« What Sanofi Thinks About You | Main | A Response From Sanofi »

March 2, 2012

Stem Cells in Texas: Quite the Business

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Posted by Derek

I (and many of the readers here) have long thought that stem cells are perhaps the most overhyped medical technology out there - at least for now. I definitely agree that the possibilities for their use are staggering, and I very much hope that some of these pan out, but the gap between those possibilities and the current reality is just as huge. And it's a gap that really shows how hard medical progress is compared to how hard it is in the public imagination.

Nature has an article that bears on this, and on some other important topics. They've found that stem cell treatments are being sold to patients in Texas.

(The investigation) suggests that (Celltex Therapeutics) has supplied adult stem cells to Texas doctors who offer unproven treatments to patients, and that the company is involved in these treatments. One doctor claims that the treatments are part of a clinical study run by Celltex and that the company pays him US$500 a time to inject the cells into patients, who are charged up to $25,000 for a course. The US Food and Drug Administration (FDA) considers it to be a crime to inject unapproved adult stem cells into patients. David Eller, chief executive of Celltex, denies that the company is involved in treatment procedures, but would not comment on Nature's findings about how its cells are used or answer questions about them.

This makes me wonder about what is going on down there in Texas (and I can tell you, as an Arkansan, I'm willing to believe just about anything in that department). This latest business reminds me of the Burzynski cancer treatment stuff, in the way that definitions of "clinical trial" are stretched like rubber bands. Personally, I think that clinical trials are supposed to follow something very much like Yog's Law in publishing ("Money flows towards the writer"). If you're being asked to put up all kinds of money to get your book edited and published, you're very likely being scammed. And if you're being asked to pay thousands of dollars to be in a "clinical trial", well. . .you're being sold something. Real clinical trials reimburse their patients for time and effort, with money and/or medical care. They do not bill them for 25 long ones at the end of the dosing schedule

I should mention here that Slate also had an article up on Celltex, but there have been some problems. They've taken the piece down, citing editorial problems, but (as you'd figure), the cherchez le lawsuit rule applies here. Nature, though, doesn't seem to be getting sued for what they've written.

Now, back to the stem cell treatments. Among other things, Nature mentions a blog by a woman in Texas, who's written about her experiences being treated with adult stem cells from Celltex. It appears that she's receiving these treatments for multiple sclerosis, and was told that "This method has been successful with auto immune diseases such as Parkinson’s, arthritis, Multiple Sclerosis as well as others." She had apparently had a similar procedure done earlier in Mexico, but then:

". . .a friend told Larry about a doctor in Houston who went to South Korea two years ago for a stem cell transplant to treat the debilitating effects of psoriatic arthritis. He is now able to continue his medical practice, perform surgeries, and live without pain. Because our friends had noticed progress from my first stem cell transplant, they wanted us to know that Dr. Jones was now licensed to perform the procedure in Houston. To say the least, we were both excited about the possibilities and timing."

As that extract illustrates, at no point (that I have found) does this patient mention the phrase "clinical trial". One gets the strong impression, actually, that she believes that she is paying to undergo a new medical procedure, the latest thing, rather than participating in any kind of investigational study for a therapy that has not yet been reviewed by the FDA. The Nature writer, David Cyranoski, was able to speak with the physician involved, who says he's treated a number of people with cells from Celltex:

Lotfi says that most of his patients claim to get better after the treatment, but he admits that there is no scientific evidence that the cells are effective. “The scientific mind is not convinced by anecdotal evidence,” he acknowledges. “You need a controlled, double-blind study. But for many treatments, that's not possible. It would take years, and some patients don't have years.”

“The worst-case scenario is that it won't work,” he adds. “But it could be a panacea, from cosmetics to cancer.” He says that Celltex is conducting a trial in which patients “will be their own control”. “If you can compare before and after and show improvement, there's no need for a placebo,” he explains. “How can you charge people, and then give them a placebo?”

Indeed! Maybe you could try not charging them, and not making them spend their own money to find out whether your treatment is any good. Maybe you could get a large, statistically significant number of people together, who've been given thorough diagnostic workups, and give half of them the best standard of care for multiple sclerosis and half of them the stem cell treatment - at your expense - and see if they get better. How about that? (Oh, and just a little note - the worst case is not that nothing happens at all. It might be good for the people involved to think about that a bit).

This gets back to the discussions we've had around here about rethinking clinical trials. One of the things I'll say for the FDA is that they do force people to be rigorous, and to put new medical ideas to well-controlled tests. My worry about the "sell, then test" ideas was summed up in the first link in this paragraph: "I fear that there are any number of entrepreneurial types who would gladly stretch things out, as long as someone else is paying, in the hopes of finally seeing something useful. No one will - or should - pay for extending fishing expeditions." Read that Celltex article and see if that sounds familiar.

Comments (16) + TrackBacks (0) | Category: Clinical Trials | Regulatory Affairs | The Central Nervous System | The Dark Side


COMMENTS

1. Anon on March 2, 2012 9:38 AM writes...

Rick Perry got the mother load $1M injection. Fried off his brain in the process......

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2. Jim on March 2, 2012 10:16 AM writes...

"The US Food and Drug Administration (FDA) considers it to be a crime to inject unapproved adult stem cells into patients."

I'm amazed that the verb "considers" is in play here. Is it or isn't it a crime? One would hope they'd have a bit more clarity around this. The only thing that's more amazing is the quote from the physician Lofti who doesn't understand that you don't charge patients in clinical trials.

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3. DCRogers on March 2, 2012 11:44 AM writes...

He says that Celltex is conducting a trial in which patients “will be their own control”.

I can't even think of something snarky enough to be worthy of this up=down description that eviscerates the entire concept of a clinical trial.

Even more than accepting money, this is the true corruption. Doesn't the FDA have to sign off on the protocol for this to be called a "clinical trial"? How the h*ll do you have a trial with no controls?

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4. PTM on March 2, 2012 11:59 AM writes...

I see no problem with such treatments as long as subjects are given accurate and up to date information about what is known and unknown about their risks and benefits.

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5. LF Velez on March 2, 2012 12:50 PM writes...

When the FDA 'considers' something to be misbranded [for instance, when a company claims efficacy for a treatment that has not been formally approved by the FDA, which is the legal arbiter of "efficacy" in the US, they are permitted to issue warnings and levy fines on that company. It is illegal to claim a pharmaceutical or device is "safe" or "effective" without prior FDA approval. ["Supplements" are a different story...]

I tried searching ClinicalTrials.gov to find a study that would fit the description from the Celltex group. No luck. You can see my results below:

Found 2 studies with search of:
stem cells AND texas | multiple sclerosis | United States, Texas

Study name: High-Dose Immunosuppression and Autologous Transplantation for Multiple Sclerosis (HALT MS) Study
Status: Active, not recruiting
Condition: Multiple Sclerosis
Interventions: Drug: Granulocyte-colony stimulating factor (G-CSF) and prednisone; Drug: Carmustine, etoposide, cytarabine, and melphalan (BEAM); Procedure: Autologous hematopoietic stem cell transplant


Study Name: High Dose Chemo/Radiotherapy and Hematopoietic Stem Cell Transplant for Patients With Multiple Sclerosis
Status: Completed
Condition: Multiple Sclerosis
Interventions: Drug: Cyclophosphamide; Drug: ATG; Drug: MESNA; Procedure: Radiation therapy

Neither of these studies was sponsored by Celltex. The blogger may be happily able to take many things on faith, but CBER is likely to take a dimmer view...

Permalink to Comment

6. Fenichel on March 2, 2012 1:41 PM writes...

"The US Food and Drug Administration (FDA) considers it to be a crime to inject unapproved adult stem cells into patients."

This statement is wrong; it misstates the jurisdiction of FDA. FDA regulates (1) the performance of clinical trials, and (2) the marketing of devices and pharmaceuticals, so it is a violation of the Food, Drug, and Cosmetic Act to market (in interstate commerce) products for medical purposes without FDA approval. Physicians' per-patient behavior is not regulated by FDA; a physician who provides non-approved therapy may be in trouble with his hospital or his state's licensing authority, but not with FDA.

The physician quoted here seems to realize that he's not doing a clinical trial; he's just (!) charging patients lots of money for a treatment not shown to be safe or effective. He shouldn't be doing that, but the FDA will not -- on the strength of what's described above -- be effectively able to tell him so.

Permalink to Comment

7. DV Henkel-Wallace on March 2, 2012 3:54 PM writes...

Maybe you could try not charging them, and not making them spend their own money to find out whether your treatment is any good.
Sigh. Derek, statements like this make you sound like you have scientific ethics (much less a moral sense), both of which are waaay out of fashion these days, and must be subordinated to Mammon. Get with the program! Permalink to Comment

8. Anonymous BMS Researcher on March 2, 2012 9:59 PM writes...

My wife's comment: there should be a special circle in Hell for lawyers who defend quacks from investigative journalists.

Permalink to Comment

9. Andrea on March 2, 2012 11:36 PM writes...

I think patients should have the right to choose a treatment involving adult stem cells as long as they are told all their pros and cons. Regarding charges, it may be morally wrong but it's not legally wrong to charge for your work

Permalink to Comment

10. basudin on March 3, 2012 12:45 PM writes...

Google has a cached copy of the original article if anyone wants to have a look (replace the xx with pp):

hxxp://webcache.googleusercontent.com/search?q=cache:c6eE_0l_hN8J:www.slate.com/articles/health_and_science/medical_examiner/2012/02/glenn_mcgee_and_celltex_an_ethics_scandal_in_bioethics_.single.html+&cd=3&hl=en&ct=clnk

Permalink to Comment

11. Timbo on March 3, 2012 5:48 PM writes...

Why doesn't somebody in the media follow up on these people who have been treated and see if they have improved.
That would really be worth reading!!!people get ripped off daily from used car dealers selling lemons to doctors administering chemotherapy and charging for it when they don't know if it's going to work ......your story is not intriguing just a redundancy .

Permalink to Comment

12. Timbo on March 3, 2012 5:50 PM writes...

Why doesn't somebody in the media follow up on these people who have been treated and see if they have improved.
That would really be worth reading!!!people get ripped off daily from used car dealers selling lemons to doctors administering chemotherapy and charging for it when they don't know if it's going to work ......your story is not intriguing just a redundancy .

Permalink to Comment

13. Timbo on March 3, 2012 5:50 PM writes...

Why doesn't somebody in the media follow up on these people who have been treated and see if they have improved.
That would really be worth reading!!!people get ripped off daily from used car dealers selling lemons to doctors administering chemotherapy and charging for it when they don't know if it's going to work ......your story is not intriguing just a redundancy .

Permalink to Comment

14. David Cyranoski on March 5, 2012 6:14 AM writes...

@Fenichel

FDA jurisdiction is a tricky issue. In my research for writing that article, I knew that there was a question mark about jurisdiction, so I addressed the question to one source who formerly worked as an advisor with the FDA on cell therapy regulatory issues (who wants to stay anonymous) and a current spokesperson at the FDA. Both of their comments support the idea of FDA jurisdiction applying here.

(1) Former FDA advisor: "It is extremely difficult for a company to avoid interstate commerce, since FDA has previously interpreted this to include materials used to prepare the medical therapy. For example, if they purchase cell culture media or even flasks that were manufactured outside the state, then they have violated interstate commerce and therefore FDA can state they have jurisdiction."

(2) FDA spokesperson Rita Chappelle (the nub is in the last sentence):

"FDA takes a risk based approach to the regulation of human cells, tissues and cellular and tissue-based products (HCT/Ps). Under the authority of Section 361 of the PHS Act, FDA established regulations for all HCT/Ps to prevent the transmission of communicable diseases. These can be found in Title 21 of the Code of Federal Regulations (CFR) Part 1271. The regulations in Part 1271 identify the criteria for regulation solely under Section 361 and Part 1271. HCT/Ps that meet all of these criteria are regulated solely under Section 361 and implementing regulations in Part 1271, and no pre-market review (application to FDA) is required. To satisfy these criteria, an HCT/P must be:

ï‚· Not more than minimally manipulated

ï‚· Intended for homologous use only

ï‚· Not combined with another article (with some limited exceptions)

ï‚· Without systemic effect and not dependant on the metabolic activity of living cells for their primary function, or, failing this, intended for autologous use or use by a first-or second-degree blood relative

HCT/Ps that do not meet all of the above criteria are also regulated under Section 351 of the PHS Act and/or the FD&C Act as drugs, devices and/or biological products.

Please note, 21 CFR 1271.1 (b) provides that the regulations in Part 1271 apply “whether or not the HCT/P enters into interstate commerce.”

Permalink to Comment

15. Morten G on March 5, 2012 6:59 AM writes...

First person to develop a tumor wins! Weeee!

It's not impossible to do a clinical trial on one person. If you are able to repeat a dose-response with double blinded dosages that would in my mind be sufficient to justify treating the patient. Unfortunately this is treatment with a replicator so that's not relevant.

Permalink to Comment

16. Robert Fenichel on March 5, 2012 2:35 PM writes...

@David Cyranoski
I didn't express myself clearly. There's no doubt that FDA has jurisdiction over *marketing* of stem cells, and the interstate-commerce requirement is interpreted so broadly -- as the quotes in #14 show -- that it scarcely reduces FDA's power at all. The point I was trying to make is that FDA does not directly regulate the non-experimental *practice of medicine*.

In the original post, we heard that Celltex was manufacturing a product and selling it to doctors, surely for medical purposes, although Celltex "denies that the company is involved in treatment procedures, [and] would not comment on Nature's findings about how its cells are used." That would be the proper response, if Celltex were selling industrial chemicals and were shocked! shocked! to find that doctors were using them for medical purposes. Celltex may be in trouble with FDA, for exactly the reasons that David Cyranoski states.

On the other hand, the doctor involved will hear from FDA -- if at all -- only as a fact witness, if FDA is trying to build a case against Celltex. The doctor may, as I said in #6, expect to be asked some tough questions by his hospital and, perhaps, by the Texas licensing authority. FDA will then be involved only to verify that the substances in question have no approved medical use.

Anecdote: When I was a medical intern, we had a patient who suffered from intractable nausea, but his liver function was so poor that the then-available anti-emetics were all contraindicated. Someone had heard that undiluted Coca-Cola syrup was a powerful anti-emetic, so we got some from the cafeteria and tried it, with equivocal results. FDA would have had some interest if the Coca-Cola company had promoted this use, but of course it hadn't. [Decades later, when I was at FDA, we'd occasionally hear about incidents similar to this, and we'd refer our informants to state & hospital authorities.] The hospital's Department of Medicine discussed the issue, and some of those involved were chided for adventurism.

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