In the last post, we saw that the FDA’s illegal fast-track approval of Genzyme’s cell-based Carticel product as a drug set a precedent that cells had to be treated as drugs and any cell-based therapy had to go through the extensive, expensive and invasive approvals for other types of pharmaceuticals.
This move put the kibosh on the development of cell-based therapies, since such therapies would have been massively expensive.
Centeno thinks that the FDA’s move stifled a potential renaissance in American medicine that would have been on par with the discovery of antibiotics. This might be a severe overstatement, but it seems clear to me, at least, that the FDA’s move did slow down the development of cell-based therapies.
Professional societies were no at all pleased with this regulatory move, since it would slow down the availability of therapies for patients. For example, te Society of Clinical Oncology, which represents 30,000 cancer specialists, sent a letter to the FDA on August 10, 1998 that read: “A striking aspect of the FDA’s proposal to regulate stem cell procedures is the virtual absence of any justification for the initiative. The principal “concerns” previously cited by the FDA in support of the new regulatory apparatus are preventing the transmission of communicable diseased and assuring that stem cell procedures are safe and effective.”
Certainly, these are valid concerns, but the letter continued: But, the FDA has adduced no evidence whatever to suggest that communicable diseases are presently being spread through stem cell procedures, or that stem cell transplants are unsafe or ineffective. Instead, the FDA has proposed to subject physicians and facilities performing stem cell procedures to an extraordinarily burdensome and restrictive regulatory regime based solely on theoretical issues.”
Wow! In other words, “your concerns are not real.” Furthermore, “you have no evidence to document your concerns and they only exist as possibilities.” In other words, according to these cancer specialists, the FDA created a policy to fix a problem that did not exist.
Certainly, the FDA could make the argument that the regulation guarantees that such problems will not pop up anytime soon, this misses the point of regulation – to address actual problems and not imaginary ones.
At this point Centeno makes a horrible historical blunder when he mentions the “Bush Administration’s ban on embryonic stem cell research.” For the umpteenth time, “THERE WAS NO BAN ON EMBRYONIC STEM CELL RESEARCH DURING THE BUSH ADMINISTRATION.” There I got that out. There was a moratorium on the use of federal funds for research that derived new embryonic stem cell lines, since such work required the destruction of human embryos. Private funding could fund such work, and embryonic stem cell research that used already established, approved ESC lines was eligible for federal funds. In the meantime, work on adult stem cells advanced during this time and some treatments became available.
Centeno thinks that drug companies supported by universities wanted to offer embryonic stem cells (ESCs) in vials, which would be administered and approved by the FDA in the way any drug would. Adult stem cells, on the other hand, came from the patient’s own body, and therefore, had a rough time fitting into this paradigm. The FDA, therefore, decided the alter the wording of a statute to include adult stem cells.
21 CFR 1271 regulations allowed any cells that came from a patient’s own body (autologous) to be regulated as a transplant or not at all. Therefore, any competitor of Genzyme could simply use their adult stem cell procedures without going through the lengthy and extensive approval process. It also meant that physicians, who are not directly regulated by the FDA, could start their own stem cell procedures without any FDA oversight.
The FDA however, solved this problem by altering one word in the statute from “into another human” to “into a human.” This change was apparently made without the public comment period required by the law. Even though this change seems minor, it means that cells from your own body cannot be placed into you without the FDA’s approval. In this manner, the FDA inserted itself into the lives of doctors.
Mary Ann Chirba, J.D., D.Sc., M.P.H. at the Boston College Law School gave the FDA a screen-full when she used her keyboard to critique the FDA’s language change. She said of this regulatory change that “in revising §1271.3(d) without the benefit of public input, the FDA has fashioned a regulation that imposes real and extensive burdens on patients and providers while achieving little if any benefit — at least with regard to low-risk, autologous adult stem cell therapies.” Not everyone is taking this change lying down.
One point Chirba makes is quite telling. She notes that the FDA has no such regulatory strategy when it comes to in vitro fertilization (IVF) therapies for infertile couples. IVF involves the harvesting of oocytes (eggs) from the presumptive mother and sperm from the presumptive father, the use of these cells to achieve fertilization in the laboratory, culture of the embryos outside the body, and then reintroduction of the cultured embryo into the mother’s body. Note the similarities of this treatment with autologous stem cell treatments – retrieval of cells from the patient, culturing the cells to make something different, but related to the originally harvested cells, and then the transplantation of those cells into the patient. The similarities are uncanny except that the FDA regulates autologous stem cell treatments but not iVF. Confused? Maybe you should be, because I am too.
If the FDA regulated IVF, they would achieve little in the way of safety, but the cost of the procedure would explode (however, the embryos might be better protected, but the FDA seems little concerned with human embryos these days, but that’s for another post).
The contradictory nature of this regulatory change has generated some terrific contradictions. For example, the FDA does not regulate bone marrow transplants, and in some cases, umbilical cord blood is used for such therapies instead of bone marrow. By 2009, there had been 15,000 cases of umbilical cord blood transplantation, and cord blood was saving lives and was relatively unregulated by the FDA. Then in 2009, the FDA changed tack and decided that cord blood was a drug and required approval. Cord blood “drugs” received approval in 2011, but nothing changed with regard to its safety profile and use. First it’s not a drug then it is; what’s up?
Another example is fat tissue used by plastic surgeons. Plastic surgeons have been using fat for transplantation for years without FDA approval, but now, according to the FDA, is the fat is broken down in any way before it is reintroduced, then it is a drug. Your own fat is now a drug! Ridiculous? You bet it is! Then there is Cytori Therapeutics who have designed and manufacture a machine that isolates fat-based stem cells from fat. According to the FDA, this machine manufactures and drug and must be vetted like a drug. The machine is approved in Europe, but not in the US because the FDA is too busy sending warning letters to Cytori telling them to cease and desist unless the almighty FDA tells them they can proceed.
There are also some genuine tragedies that come with this stunt pulled by the FDA. For example, Allosource and Parcell Laboratories process cadaver bone for bone transplants. However, because the bone contains stem cells, according to the FDA, they are making a drug and require the huge piles of money it takes to receive FDA approval. Then there is Provenge, a revolutionary treatment for prostate cancer that takes white blood cells from your own body and then reprograms them in culture to attack the prostate cancer. Thanks to the excessive regulation of the FDA, one dose of Provenge costs $93,000. Then there is the very sad case of Reprogenesis, who made a stem cell-based treatment for children with Vesicoureteral Reflux or VUR. Even though the treatment uses the patient’s own stem cells to build a new urinary valve in the ureter, the FDA said that they were making a drug and needed approval for it. Reprogenesis went bankrupt thanks to the FDA and children with VUR do not have that therapy.
If this sounds nuts, that’s because it is. Don’t get me wrong, we need the FDA and we need an agency to safeguard the safety and efficacy of the medicines we take, but the FDA is working on a model that is 3-4 decades out of date. American medicine is missing the renaissance in science because of overregulation and unwise regulation. It needs to change.