Texas is shaping up to be a showdown over physician innovation with stem cells. The Texas Medical Board (TMB) voted 11-4 on April 13, 2012 to pass a new rule (Chapter 198) that allows physicians to use stem cells that are not FDA approved under the practice of medicine, as long as certain conditions are met. The TMB will require IRB approval for stem cell use and that the patient understands that he or she is receiving an investigational therapy not approved by the FDA. Since we have always used IRBs to monitor our first use of stem cells and have always consented patients that they are receiving an investigational therapy, we applaud the TMB's initiative.
The board vote was not without controversy, with attacks coming far and wide from the commercial interests who want your cells to be drugs. In particular, a rather strange confusion of bioethics was presented to the media and board. Knowing a Texas university bioethicist I called him to confirm what I knew, that the "bioethical" cry wolf happening around the new TMB rules made little sense. The bioethical conundrum being shouted to the media was this: "Patients may be charged for medical research!" The problem with the statement goes back to the Belmont report, the seminal document which lead to our current NIH and HHS framework for research ethics.
Belmont clearly defines two types of research. The first is the type that's often done in universities, from which patients need the most protection. This is because the goal of the study is not in the individual research subject's best interest. For example, a randomized and placebo controlled trial starts with the assumption that only a half or fewer patients will be treated while the other half will get a fake therapy. While it may be in society's interest (and those involved in the study) to have the knowledge that compound X helps disease Y, the goal of the study is to get that knowledge and not to cure, mitigate, or treat any individual research subject. In fact the needs of any given subject are simply unimportant to obtaining the knowledge-hence a maximum number of patient protections needs to be in place.
This was why the bioethics experts were shouting from the hilltops – they were spinning that this type of research would happen with patients being charged. However, completely left out of the equation (and well known to these same bioethicists) is the fact that Belmont describes a different, but common format for medical research. In this type of research, the primary goal is to treat the patient, with the secondary goal being to obtain knowledge. In this second type of research, the patient or the insurer may be charged for the care delivered, as long as the goal is to treat the patient and the care has a reasonable hope of helping the patient. This is much more likely to be what happens as a result of the new TMB rule. So why would otherwise well trained and sound bioethics experts miss this distinction? Surely the TMB was approving the latter principle of the Belmont report and not the former. To see just how crazy the whole thing has become, let's take an example.
Bariatric surgery for severely obese patients has been popular since the 1980s. At that time, the surgeries were highly speculative in nature, as while we had prima facie evidence that they worked (you could see the patient dropping pounds after the surgery), we didn't know their long-term effects. For example, would the patient put all of the weight back on? Were there subtle types of malnutrition that would set in and hurt patients? Did a sudden loss of all of this weight do the patient any good or did it hurt the patient? These procedures were very risky, with as many as a few patients out of a hundred not surviving the surgery. Also, until recently insurance companies didn't pay for these procedures, so they cost patients tens of thousands of dollars. They were essentially an "investigational therapy" being advertised to patients that was risky and expensive. Despite this, the patients and their doctors saw the benefits. The physicians who performed these procedures were able to publish case reports, then case series, than more controlled trials-showing that the procedure worked and better quantifying it's risks. When there was a bad doctor producing bad outcomes, that issue was dealt with by the local medical board. Professional societies cropped up to establish guidelines and insurance coverage followed. From there, several large and well designed medical studies were launched, two of which were just completed. What did they show? That the surgery actually allowed about 3 in 4 severely obese type 2 diabetics to get off of diabetes medications whereas nobody in the medication management group got off of their medications. The conclusion? The surgery that began as medical innovation by doctors following the tenant of the Belmont report that physicians can innovate to help patients, decimated medication management for diabetes.
Now use the phrase "stem cells" and insert it for the phrase "bariatric surgery" above and you can see that the same physician innovation cycle is happening again. What's fascinating is that while about half of all the medical advances we all take for granted began this way (with physicians trying new things in one individually consented patient), the academic set won't recognize this approach as existing let alone valid. Why the difference between bariatric surgery and stem cells? There were no established commercial interests on the other side of the bariatric surgery equation-meaning the surgery couldn't be commercialized as a drug like is being attempted with stem cells. Therefore physician use of the surgery wasn't threatening pharma interests during it's development, until now...
The conclusion? I applaud the TMB's courage for standing up to commercial interests and putting into place a structure to help protect patients. I would also challenge the media reporting this story to think critically about why a time honored traditional of medical innovation that's responsible for many of the medical advances we take for granted is being attacked. Who stands to gain?
For more information, visit the Centeno Schultz Clinic web site.