Progress in medicine is heavily dependent on research, especially well designed and executed clinical studies, and also to discoveries and innovations made by doctors using FDA approved drugs and therapies (and in some instances supplements, e.g., herbs, vitamins, amino acids, etc.) in novel ways and combinations in treating patients (“In-office tinkering” or experimentation, one might say). So long as what a doctor is using does not constitute a “new drug” by FDA criteria and does not violate agency rules that govern new devices or such, what the doctor is doing is considered the practice of medicine and is governed solely by his or her state medical board.
If a doctor were to treat ulcer patients using a combination of an approved drug such as cimetidine (Tagamet) and an herbal ulcer product such deglycyrrhizinated (DGL) licorice, this would fall under the practice of medicine and represent a form of low risk medical experimentation that most state medical boards allow provided informed consent requirements have been met, i.e., the patient has been told about known and suspected risks, side effects, or complications (Higher risk forms of experimentation might require the treatment regimen or protocol be approval by an Investigational Review Board aka as an ethical review board). If this particular combination happened to heal up ulcers better and surer than the drug alone then this would represent an innovation of the sort that propels medicine forward. If on-the-other-hand a doctor wanted to try a drug for advanced cancer approved in the EU but not in the US, he would have to comply with FDA rules and procedures concerning new drugs. He might, for instance, get be able to get permission to use the drug by filing an emergency IND (Investigational New Drug) application and having this reviewed by FDA officials. If they felt the risk v. benefits favored use in this instance, they would grant the doctor a new drug (IND) permit for the one-time use of the anticancer drug in question.
But what if a doctor wanted to use stem cells from a patient’s own body to treat his arthritic knees and bad back? Given the fact there is no risk of rejection, would the FDA take issue with a doctor taking a patient’s own fat or bone marrow stem cells and treating his bad knees and back?
The answer depends on what is done with the stem cells and how they are administered.
If a doctor wants to take a patient’s bone marrow stem cells and then culture them and increase their numbers or otherwise process them in ways that would substantially alter their form or function, this would constitute a new drug according to the FDA and would have to meet their body of regulations that govern biologics and how they are produced, processed and tested. If a doctor plans on harvesting stem cell rich bone marrow from a patient, doing very little to it (minimal processing or “manipulation” in FDA parlance) and then promptly administer it to her this does not constitute a new drug and thus does not fall under the FDA’s jurisdiction or purview.
Currently there are a handful of clinics here in the US treating patients with their own “minimally manipulated” stem cell rich fat or bone marrow tissue. Some are focused on treating orthopedic issues such arthritic joints, others on cosmetic procedures such as face lifts and breast reconstruction, and a few on neurologic conditions such as stroke as well as other medical challenges.
Critics have raised questions about the safety and scientific rationale of at least some of these treatments. For instance, while bone marrow stem cells have been shown to naturally mobilize and migrate to diseased, injured or infected tissues, then engraft, many scientists and doctors question whether harvesting and infusing huge numbers of bone marrow stem cells in patients might pose an unforeseen safety risk. The doctors doing this see it as augmenting a natural process and find that potential benefits and gains outweigh risks. Case-in-point: Chronic stroke. One osteopathic doctor (See Resource Box) who has been treating stroke patients with infusions of their own minimally manipulated bone marrow stem cells for more than five (5) years now has seen no problems crop up and has documented improvements in many of those treated. Recently some preliminary small-scale studies have confirmed that treating stroke with stem cell rich bone marrow or “bone marrow aspirate” is not only safe but produces clinical benefits in some patients.
In a February 1, 2012 article in US News & World Report’s HealthDay section titled “Stem Cell Therapy Shows Promise for Stroke, Studies Say” (Patients may regain some function after treatment, preliminary research suggests), journalist Maureen Salamon discussed the results of two separate studies done in India in which stroke patients received stem cells from their own bone marrow. In one of the studies 120 moderately affected stroke patients (18 to 75 years old) were divided into two groups. Those in the stem cell or experimental group were given intravenous infusions of bone marrow stem cells harvested from their hip bones, while those in the other group did not and served as controls. Approximately 73 percent of those in the experimental group achieved “assisted independence” during the ensuing six months, while only 61 percent of the control group did. This was not statistically significant.
And in the second group “…40 patients whose stroke occurred between three and 12 months prior were also split into two groups, with half receiving stem cells, which were dissolved in saline and infused over several hours. When compared to controls, stroke patients receiving stem cell therapy showed statistically significant improvements in feeding, dressing and mobility, according to the study. On functional MRI scans, the stem cell recipients also demonstrated an increase in brain activity in regions that control movement planning and motor function.”
According to one US expert Ms. Salamon consulted, Dr. Matthew Fink, chief of the division of stroke and critical care neurology at New York-Presbyterian Hospital/Weill Cornell Medical Center, “the therapy’s safety is the only thing the two studies seemed to demonstrate.” Dr. Fink also stated that “the cells taken from study participants’ hip bones can only be characterized as “bone marrow aspirates” since the authors didn’t prove that actual stem cells were extracted.”
As you might expect, in instances in which doctors have used patient stem cells that were cultured or taken beyond “minimal manipulation” the FDA has gone on the offensive. In one well publicized and on-going case, in 2005 Colorado-based Centeno-Schultz Clinic began doing orthopedic treatments (Regenexx-C) in which patient’s own bone marrow mesenchymal stem cells were expanded in a special solution and the re-injected to treat moderate to severe joint, tendon, ligament, and bone pain. This was done as part of “IRB approved and supervised clinical studies using both same day and cultured stem cells for orthopedic injuries”, according to Chris Centeno, MD in a blog entry to the Regenexx website blog on February 6, 2012.
Following two years of treating patients who were part of the IRB approved and supervised study, Dr. Centeno and his associates felt they had sufficient evidence of efficacy to begin offering the procedure on a limited basis to its regular patients.
In 2008 the FDA sent a letter challenging the Regenexx™ procedure as constituting creation of a new drug. Dr. Centeno and his colleagues provided the FDA with numerous legal opinions declaring that what had been doing did not qualify as creation of a new drug, and invited agency representatives to meet with them to discuss the issues involved. The FDA declined Centeno’s invitation which resulted in his firm (Regenerative Sciences LLC) filing suit against the FDA first in 2008 and again in 2010; lawsuits aimed at getting the agency to present their reasoning for categorizing a patient’s own cells as “drugs” and cite their authority for this. Two years later (2010) the FDA filed suit against Regenerative Sciences LLC. According to Dr. Centeno “As part of an agreement between the parties, we state that we will only use same day procedures in the U.S. and not culture cells until a judge decides who is right in this dispute.”
In July 2011 Texas Governor Rick Perry revealed he had a longstanding back problem treated with spinal fusion surgery and injections of his own lab grown and cultured fat stem cells by his orthopedic surgeon Stanley Jones in Houston. According to an August 3, 2011 article that appeared in The Texas Tribune, a few weeks after his procedure Gov. Perry had declared in an AP (Associated Press) interview “that he felt 80 percent recovered and was swimming and using the treadmill”.
Since then Gov. Perry, Dr. Jones and other prominent Texans have actively pressed to have the Texas Medical Board pass rules that would allow and regulate as opposed to hindering use a patient’s own stem cells by Texas doctors. Various drafts have been proposed since then and are being debated at this time.
Not surprisingly the issues surrounding the Centeno lawsuits and the initiative in Texas to allow but properly regulate doctor use of a patient’s own stem cells has triggered a fierce, often contentious debate over matters such as how to maintain the latitude needed by physicians to do their own in-office informal research vs. concerns over patient safety. Some are wondering whether a court decision favoring the FDA’s decision to treat more than minimally manipulated autologous (a patient’s own) stem cells as a new drug might not result in states defying this much as seventeen of them have with respect to medical use of marijuana (Allowing and regulating at the state level what has been declared illegal at the federal).
For patients who wish to try some form of stem cell therapy the options boil down to either pursuing either what is legal or permitted, or what isn’t.
On the legally permitted side are FDA approved clinical studies they can try to get into (provided they qualify, i.e., meet inclusion criteria) plus treatments offered by private doctors who use a patient’s own stem cells that have been harvested and not more than “minimally manipulated” as part of the same procedure. They can also travel abroad to have adult (nonembryonic) stem cell therapy in a country that permits this such as Mexico, Panama, Thailand or the Cayman Islands.
On the technically illegal side are various clandestine clinics and treatment programs (plus the few well known ones in Texas) that treat with autologous stem cells that have been cultured or otherwise more than “minimally manipulated”. How many of these “underground clinics” exist and where they are located is difficult to determine for obvious reasons.
In the final analysis, many people writing and blogging about this “stem cell turf war” want medical progress that is not predicated on throwing patient safety to the wind nor so hamstrung by restrictive laws and regulations as to be driven to its knees. Such progress is contingent on physician’s being able to do in-office informal patient research. When it comes to autologous stem cells, some doctors feel the FDA has no business declaring more than minimally manipulated stem cells a drug subject to their regulation and the new drug approval process. However, until the issue is settled in the courts or through other venues, doctors who use autologous bone marrow or fat stem cells as part of their quest for and development of more effective medical treatment approaches must walk the FDA’s “minimal manipulation”/prompt use line.
Disclosure: The author serves osteopathic physician David Steenblock as a professional writer but otherwise has no connection, commercial or otherwise, to any clinic, program or individual named in this article or in The Resource Box.