ROAD TO THE CURE UPDATE MAY 2017Category: Road to the Cure
WHY STEM CELL THERAPY MAY NOT BE THE ANSWER
And stem-cell therapies have been associated with other adverse effects as well... such adverse effects are probably more common than is appreciated, because there is no reporting requirement…
USFDA CLARIFIES STEM-CELL THERAPY’S BENEFITS AND RISK
[The New England Journal of Medicine, 376 (11), 1007 (2017)] The US Food and Drug Administration (USFDA) share the current excitement over the potential of stem-cell therapy to improve patient outcomes or even cure diseases. The USFDA states that increasingly hematopoietic stem cells (stem cells that give rise to all other blood cells) and stem cells derived from sources such as adipose (especially of body tissue) used for the storage of fat tissues are being used to treat multiple orthopedic, neurological, and other diseases. Often these stem cells are being used in practice on the basis of minimal clinical evidence of safety or efficacy, sometimes with the claim that they constitute revolutionary treatment for various conditions.
USFDA’s VIEW ON THE EFFECTIVENESS OF STEM-CELL THERAPY
“Despite the absence of compelling evidence from adequate, well-controlled clinical trials, some practitioners assert that stem cells have a unique capacity to restore health because they can sense their environment and differentiate in a manner that repairs any defect. A separate argument is that conducting controlled trials and meeting regulatory standards for such promising therapies is too complex for all, except large industrial sponsors and that therefore broad use in clinical practice should be allowed and encouraged while evidence regarding efficacy is gathered. Proponents of both arguments generally assert that stem-cell therapies are quite safe, particularly when the cells are derived from an autologous (of cells or tissues) obtained from the same individual source. Outside the setting of hematopoietic reconstitution and a few other well-established indications, the assertion that stem cells are intrinsically able to sense the environment into which they are introduced and address whatever functions require replacement or repair — whether injured knee cartilage or a neurologic deficit — is not based on scientific evidence. Published data derived primarily from small, uncontrolled trials plus a few well-controlled randomized trials have not reliably demonstrated the effectiveness of stem-cell treatments even in some of the most systematically studied conditions, such as heart failure and graft-versus-host heart failure and graft-versus-host [References: JAMA, Cardiol., 2016; 1: 831; Expert Rev Clin., Immunol., 2016; 29 : 1-14]”. This lack of evidence is “worrisome”. The literature is replete with examples of therapeutic interventions pursued on the basis of expert opinion and patient acceptance that ultimately proved ineffective or harmful when studied in well-controlled trials comparing them with the standard of care. One of the most unfortunate therapeutic misadventures in contemporary times was the widespread use of autologous stem-cell transplantation for treating metastatic breast cancer, a practice ultimately shown to be ineffective, costly and risky.
USFDA’s VIEW ON THE SAFETY OF STEM-CELL THERAPY
The safety of stem-cell therapies for indications other than hematopoietic reconstitution also cannot be taken for granted. In one recent case, a patient was treated with multiple injections of allogeneic stem cells from different sources that were intended to reduce neurologic deficits stemming from a middle cerebral artery stroke [(Reference: N Engl J Med 2016; 375 : 196)]. The injections were associated with the development of a glioproliferative lesion, which led to paraplegia and ultimately required radiotherapy. Although autologous stem cells may typically raise fewer safety concerns than allogeneic stem cells, their use may be associated with significant adverse events. Autologous hematopoietic stem cells injected into the kidneys of a patient with renal failure resulting from systemic lupus erythematosus were associated with the development of tumors (angiomyeloproliferative lesions) that eventually led to nephrectomy [ Reference: J. Am. Soc. Nephrol., 2010; 21 : 1218].
In another instance, autologous stem cells derived from adipose tissue and injected intravitreally into the eyes of people with macular degeneration were associated with worsening vision in three people, two of whom became legally blind [Reference: Presented at the American Academy of Ophthalmology Annual Meeting, Las Vegas, November 13–14, 2015]. Stem-cell therapies have been associated with other adverse effects as well. Such adverse effects are probably more common than is appreciated, because there is no reporting requirement when these therapies are administered outside clinical investigations. The occurrence of adverse events highlights the need to conduct controlled clinical studies to determine whether these and allogeneic cellular therapies are safe and effective for their intended uses. Without such studies, the USFDA will not be able to ascertain whether the clinical benefits of such therapies outweigh any potential harms.
According to the article published by the USFDA, even after more than three decades of research and billions spent in R&D expenses, the stem-cell research is still in its infancy. The USFDA is “worrisome” about the lack of evidence for the efficacy and safety of the stem cells. The reason for this is that mammalian cells comprise tens of thousands of different proteins, lipids, carbohydrates, and other molecules, all interacting in an extraordinarily complex manner. This complexity makes it challenging, if not impossible, to predict cellular behavior a priori when stem cells are introduced into a new environment, and empirical data are therefore necessary to document safety. There is no scientific reason to believe that demonstration of efficacy and safety for stem-cell products should be any different from that for other biologic products. Therefore, stem-cell therapy is far from being ready for use in humans.
Patients and their loved ones should not expect access to stem-cell therapies sometime soon in the future. We at PRO and the scientists at ICB International, Inc., (“ICBI”), highly recommend all patients afflicted with neurodegenerative and other diseases and their families to carefully review the research article published in the above referenced “The New England Journal of Medicine” by the US Food and Drug Administration (USFDA). The copies of this article are available from Jo Rosen, President of Parkinson’s Resource Organization (PRO). The patients must make their own decisions objectively and rationally with the help of their physicians without any bias regarding the current status of stem-cell therapy. Conflict of interest on the part of medical professionals and practitioners can do more harm than good. The information published in the foregoing article is for educational purpose only. If you meet certain qualifications, you too may be able to take part in “history-making” science. You can still get in at ground floor if that appeals to you. Or you can continue waiting until SOMEONE ELSE handles this pandemic. Please get in touch with Jo Rosen at Parkinson’s Resource Organization with whom we are in constant communication, updating her on our progress, while learning from her why it is so important to continue working to eradicate this horrific disease. IMAGINE a world without Parkinson’s or Alzheimer’s disease. Just Imagine.