The Regulation of Stem Cells
In the last year or two, the regulation of stem cells has dramatically changed. My take on it is that the FDA got tired of the wild west stem cell clinics which had proliferated in the mid to late 2000’s. I take some of the credit/blame for it as I was one of the first attorneys (if not the first to clear doctors to use fat cells for autologous use for arguably homologous use. Here is some background:
From a regulatory point of view, there are two types of stem cells: embryonic and adult. There is not much to say about the regulation of the clinical use of embryonic stem cells because embryonic stem cells are not in clinical use. So long as President Bush continues to bar the development of new embryonic stem cell lines, it is unlikely that such cells will be in clinical use anytime soon.
Adult stem cells come in two basic flavors: cells obtained from living, breathing humans, and stem cells obtained from umbilical cords or placental materials. Stem cells are either autologous, meaning that they come from the same individual, or allogenic, meaning that they come from a different individual.
Another important concept is whether the use of the cells is for a homologous use or purpose, which is a use of the stem cells for the same type or purpose as the origin of that particular stem cell. Thus, a homologous use for a stem cell obtained from the bone marrow would be for a blood or hematological condition. A nonhomologous use would be for any other use or purpose. Using umbilical cord blood stem cells to treat a disease such as multiple sclerosis or ALS would be a nonhomologous use.
Finally, the FDA considers it important whether or not the stem cells are “more than minimally manipulated” which means whether the cells have been changed or altered in any significant way. Cells which are more than minimally manipulated transfer the product into a new drug, which cannot be used presumably without going through the FDA approval process (IND and NDA).
Under current federal law and regulations, the use of adult stem cells is legal for any homologous use, where the cells have not been more than minimally manipulated and the removal and reinsertion happen during the same surgical procedure. For example, adult bone marrow stem cells can be transfused into the same or another patient who has a hematological condition, or who has a need for replacement blood cells, like in bone marrow transplants for high-dose radiation and chemotherapy cancer treatment.
The FDA takes the position that the autologous use of stem cells for a homologous use is not regulated by the FDA, so long as the removal of the cells and their reintroduction to the person occurs during the same procedure and the cells are not more than minimally manipulated.
However, the autologous use of stem cells where the reintroduction does not take place during the same procedure as the removal, while legal, falls within the jurisdiction of the FDA. Thus cord blood banking is legal. The pitch that cord blood banks make to expectant mothers is that if the cord blood is saved, it can be used by the child at a later time. This is true, but it’s not the whole story. The other part is that the cord blood can only be used for a homologous use, even if it is autologous, i.e. even if the blood came from the cord from the child’s own mother. That is because under current federal regulation, the nonhomologous use of even autologous cord blood stem cells is not permitted without an IND or special exception. (Unless of course it is done during the same procedure which in this case would be birth, but then there would be no blood banking).
As a result of these regulations, it is basically not FDA legal to receive any adult stem cells for a nonhomologous use to cure or treat a disease. That is because the FDA does not as yet recognize that adult stem cells have been proven to be safe and effective for any non homologous condition.
Based on the recent FDA final guidance documents, the FDA takes the position that the separation of cells from fat, or even concentrating platelets for reinjection is problematic, read illegal without an IND.