Sorting Hype From Fact in Stem Cells

Every so often an article comes across an editor’s desk that makes so much sense that she is tempted to reprint all of it. Such an article is one by Harold DeMonaco, Director, Innovation Support Center, Massachusetts General Hospital, Boston and medical editor of Healthnewsreview.org. DeMonaco wrote about the hype presently surrounding stem cells. Below, with his permission, is some of what he had to say, edited for space and to highlight his principal points.

“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.

“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.

“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. If mesenchymal stem cells are indeed drugs, they should be approved only after extensive clinical study. At least that is the FDA view.

“The 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 to 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.

“A counter argument is that companies like Celltex and Regenerative Sciences are not altering a patient’s own cells. What they do is 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? 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 regulate the practice of medicine. The good news is that physicians can innovate to their hearts content. Physicians are free to use a patient’s own stem cells for any purpose. They do not need to have any agency approve the use.

“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.”