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Orthopedists need to be realistic about stem cells and FDA regulations
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There have been ongoing debates and discussions among cellular biologists, scientists and physicians as to the potential risks of not regulating the applications of the expanding cell therapy use in clinical medicine. The discussions have included what measures are necessary to ensure patient safety, how to protect patients from unproven applications and if FDA regulations will slow stem cell innovation.
Unfortunately, whenever a new field emerges in which there is minimal oversight, as is the case with regenerative medicine, many clinicians promote unproven treatments beyond acceptable efficacy and safety levels. Another reason to consider oversight of the different protocols and data is that there has been no proven scientific basis for many applications of adult stem cells injected in freestanding clinics in the United States to date. This debate appears to have been settled by a recent decision from a federal trial court pending appeals. If upheld, the decision may give the FDA the authority to regulate stem cells similar to drugs.
Adult stem cells
As a result of stems cells becoming easier to extract and isolate from accessible bone marrow sites, fat, skin and muscle, more clinicians encourage patients to try adult stem cells as a new treatment. These adult stem cells are isolated from the patient, grown and multiplied in a lab, and then returned to their donors. Oftentimes, the treatments are devised and promoted by the physician and are usually on a cash-only basis. These cell therapies are advertised and promoted as individualized as they use the patient’s own tissue to accelerate or promote “natural” healing. Some patients are told the process will regenerate damaged tissue or result in new tissue replacement. Because these cells are obtained from the consenting patient are often done in outpatient settings and the cells not sold or transported across state lines, much of the usual scrutiny has been avoided up until this point.
While adult stem cells have demonstrated the potential to differentiate into many kinds of tissues in culture, it is a big step for the stem cells to renew damaged body parts when placed in an existing harsh, degenerative biologic environment. Proliferation techniques used in culture expose cells to possible contaminating proteins and culture media that can change their expression and behavior. Grown adult stem cells are not the same as the stem cells extracted from the patient’s body. In addition, these cells may be processed to alter their biological characteristics or prepared for a different biologic use than is native to the body. Manipulated cells clearly become a biological product. Manipulations, even if minimal, increase the potential for unexpected patient risks.
Douglas W. Jackson
The current promises of the outcomes made to patients are getting ahead of the trailing and often lacking scientific evidence. Many experts and skeptics have serious doubts that extracted adult stem cells can do anything helpful in the patient for degenerative conditions.
Ethical and political issues
The science of using stem cells as clinical treatments has been challenged and impacted by many political and ethical issues. The most heated discussions and restrictions have involved the use of embryonic stem cells. In the United States and around the world, embryonic stem cells in the lab have been transformed into many different cell types, which offers hope of using them as the means for the repair and regeneration of diseased tissue and possibly organs. Because embryonic cells are most often derived from human embryos, many individuals and organizations oppose their use on ethical and religious grounds. Under the Bush administration, federal funding for embryonic stem cell research was significantly restricted. The Obama administration relaxed many obstacles.
Political pressures from patients and certain organizations within the United States have increased access to many types of stem cell treatments. This has been heightened and highlighted by prominent figures, such as Gov. Rick Perry (R) of Texas, who talks about it in relation to his back treatments. At this point, most of the proposed human clinical treatments have not been tested to the satisfaction of many knowledgeable scientists or to the degree desired prior to widespread patient use. This delay is partially because the use of many unproven stem cell treatments in orthopedics, as well as other fields of medicine, has caused the impetus for more formalized oversight from the FDA.
Potentially dangerous adverse events still need to be clarified, and there is a desire by many people to protect patients from financial exploitation as insurance does not cover the stem cell treatments. Because of the current restrictions to access in the United States, many American patients seek treatments from labs outside the country, especially in developing countries. Some companies and entrepreneurs, including individual physicians, have moved their labs out of the United States and now recruit patients to travel for their treatments.
Neurologic conditions
It is understandable why desperate patients with progressive and deteriorating life threatening neurologic conditions are willing to travel and try some of the new treatments. Several legitimate programs exist and are developing in the United States. Many countries have had less federal restrictions, and countries such as Great Britain, China and Israel may be ahead of the United States in stem cell research and treatments. There are some exciting and well-designed studies underway that involve the first clinical trials of medical treatments derived from human embryonic stem cells. An example of one of these human studies involves a possible treatment for age-related macular degeneration. The eye is well-suited for this initial study on the use of stem cells and is based in part on encouraging results in rats with retinal disease. The eye is a somewhat isolated tissue and is easily amenable to ongoing direct visual monitoring.
Another exciting application in the field of orthopedics is for spinal cord injury. New studies involve cord paralysis with treatment started within 14 days of injury. The embryonic stem cells are applied to coat the damaged neurons and hopefully facilitate transmission of electrical impulse across the area of injury. This study also may further help establish the safety in human patients and, at best, may result in partial repair. At this time, it is unrealistic to expect full return of lost motor function in a completely paralyzed patient. In the beginning, the goals have to be realistic and may include improvement in some bowel and bladder function or an improved ability for some movement in the involved extremities. We have a responsibility to present realistic and proven treatments and outcomes to our patients.
Be realistic
We must all remember that it takes the human embryo 9 months to go through the cascade of complicated events and inter-related cellular changes to eventually become an infant living outside of the uterus. The infant then takes another year to start walking. We have to be realistic about what the injection or placement of stem cells at one point in time on mature human tissue can achieve.
The use of stem cells is an exciting area of medicine with great potential. The last thing our patients need is any exploitation and generation of false hopes not based on science. The potential treatments should be based on well-designed and studied applications. In addition, it appears future studies will be impacted by the ruling of U.S. v Regenerative Sciences LLC, which should help to clarify the FDA’s role in the regulation of certain types of cell-based products and patient safety.
A note from the editors:
For more information about the U.S. v Regenerative Sciences LLC ruling, read the Orthopedic Medical Legal Advisor column.