Posted by Gary Schwitzer in Business of health
I got a news release recently offering the chance to interview the doctor who “Performed Stem Cell Therapy on New York Yankees pitcher Bartolo Colon.” The news release says the doctor was “eager to discuss”:
How can stem cell therapy help you function better?
How can stem cell therapy offset the signs and symptoms of arthritis?
How can stem cell therapy help rebuild the cartilage in joints?
How does joint health impact our ability to stay active?
(and other stuff)
I asked one of our expert editors, Harold DeMonaco, to write about such promotions.This is his column.
Few other treatment modalities exemplify what is both good and bad with American medicine as stem cell therapies. For years, we have been inundated with promises that cures for some awful diseases were just around the corner. And while there is progress, that progress has been painfully slow. That is until entrepreneurs stepped into the picture.
Stem cells can be derived from many different tissues. The most promising cells, mesenchymal stem cells, are harvested from bone marrow and fat. These cells, like any stem cell, can be programmed to do many functions. Enter the entrepreneurs. Companies like Celltex and Regenerative Sciences and others have quickly moved into the stem cell business supplying cells to physicians and researchers alike. Texas, in large measure due to the efforts of its governor, has become the epicenter of mesenchymal stem cell companies. Texas has recently made stem cell therapies, not yet approved by the FDA, legal to administer in the state under a set of minimal requirements.
Normally, this would be viewed by everyone as a good thing. Private investment in a technology has been shown to push innovation. Making new technologies available has been an economic engine for many parts of the country. But there is an important legal issue associated with stem cell processing. When do stems cells become drugs? If deemed to be drugs, their use and processing are subject to FDA oversight well beyond that of simple blood products. The distinction between drug and blood product is murky at best at the moment.
All prescription drugs are approved by the FDA after exhaustive clinical trials. The gold standard is the randomized, double blind, controlled trial where the drug in this case stem cells, is compared to an inactive placebo. While there is debate about this approach, most would agree that it is a valid determination of the true value of a drug in the treatment or mitigation of a disease. Regardless of the study design employed, if mesenchymal stem cells are indeed drugs, they should be approved only after extensive clinical study. At least that is the FDA view.
A counter argument is that companies like Celltex and Regenerative Sciences are not altering a patient’s own cells. What they do is not different that what blood banks do with whole blood, separating out cells for specific use. Since platelets, whole blood, packed red cells and the like are not drugs, they are not subject to FDA oversight other than those activities specific to cell processing. Shouldn’t mesenchymal stem cells fall into the same paradigm? Of course, there is a third point of view from folks who don’t like anyone telling them what is good or bad for them. They are the “It’s my body and I’ll try if I want to” crowd.
There are a host of issues related to the use of stem cells for medical therapy. The above regulatory issues are important because they frame the clinical issues. If the cells are sufficiently manipulated to fall under the FDA’s drug regulations, they are subject ot a long and arduous road to approval. Their sale would be limited to only those indications for which there was sufficient data. Their manufacture would be subject to more intense FDA scrutiny. Perhaps this is not the right approach. After all, the folks at the stem cell facilities know what they are doing and minimal oversight is all that should be necessary.
The folks at Celltex were recently visited by the FDA who found their processing of cells to be deficient.* According to the FDA report, Celltex has serious issues related to the sterility, uniformity and integrity of the stems cells it obtains from patients, stores and grows for eventual use. Sound like good practices to you? Maybe then, the FDA should have a larger oversight role. The FDA and Regenerative Sciences have been locked in a court battle since 2008 over whether or not stem cells, when used for specific therapies, are in fact drugs.
In either case, drug or not, the use of the stem cells processed by commercial sources by a physician, for any purpose, is the practice of medicine. The FDA does not and cannot regulate the practice of medicine. That is up to the individual state’s boards of medicine. The good news is that physicians can innovate to their hearts content. Any physician can use just about any commercially available product for any medical purpose. Again, the state’s board of registration of medicine is the only regulatory agency with oversight. Physicians then are free to use a patient’s own stem cells for any purpose. They do not need to have any agency or oversight approve the use. This regardless of the lack of the confirmation that the therapy would work or it has a positive benefit to risk.
Mesenchymal stem cells may prove to be of great benefit to people suffering from joint diseases. Unfortunately, there are no compelling data to support their use at the moment. There are ongoing clinical trials and answers may be forthcoming. For the moment, if a member of my family asked about seeking this treatment at a local clinic, I would advise them to either enter into a clinical trial or explore other options. I would want to make certain that the cells were handled properly, that I received my own stem cells and not someone else’s and that the effects of the treatment were being reviewed, so that a body of knowledge could be developed as to whether or not the treatment did more good than harm. Call me cautious. I think that the phrase, “Caveat Emptor,” is appropriate here.
Of course these are my views and not the views of my employer.
* Publisher’s note: The Houston Chronicle recently reported, “FDA report faults Houston stem-cell company.“ That’s Houston. As in Texas. As in: “The Sugar Land company involved in Gov. Rick Perry’s unlicensed adult stem-cell procedure is rife with basic manufacturing problems, according to the U.S. Food and Drug Administration.”
And this week, Adam Feuerstein of TheStreet.com reported, How To Tell When a Drug Company Fibs About Clinical Trial Results,” about a company’s news release concerning its stem cell therapy in heart attack patients.
Just this morning, Larry Husten on the Cardiobrief blog added an update to the issues raised by Feuerstein:
“I have heard from several investigators in the trial that the Osiris press release was issued without any input or consultation from the site investigators. In fact, the site investigators, including several who are extremely experienced clinical trialists, have expressed frustration and disappointment because their input has not been sought at any point during the trial. In most multi-center trials it is common practice to consult with the sites, and in particular the top-enrolling sites. In this case, the highest enrolling sites have had no significant involvement in the trial design or conduct. One investigator said he “had never worked with a company like this.”Another member of the steering committee told me that the committee had not met in a long time and has not seen the trial data. In fact, steering committee members were not even aware that Mark Vesely, an assistant professor at the University of Maryland, was the principal investigator of the study. One steering committee member said he’d never heard of him before reading the press release.”
Addenda on July 9:
Nature News Blog posted, “Patients Seek Stem Cell Compensation.“
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