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8 | SEPTEMBER 1, 2016 | GENengnews.com | Genetic Engineering & Biotechnology News Graham Parker, Ph.D., and Jeanne F. Loring, Ph.D. A recent article 1 focused attention on the pro- liferation of unregulated clinics in the U.S. that promise stem cell cures. The term "stem cell" used to have meaning only to scientists. It referred to cells that can both make new copies of themselves and turn into different types of mature cells. Stem cell is a fitting term for the cells in bone marrow that are transplanted to people in an attempt to save them from leukemia. Bone marrow transplants require killing a patient's own cancerous bone marrow and replacing it with cells that produce all the types of blood cells found in the body. A bone marrow transplant to replace the blood system is the only stem cell therapy that is FDA approved as being safe and effective. But investments like California's $3 bil- lion for stem cell research in 2004 and simi- lar efforts worldwide have added stem cell to the vocabulary of a growing number of people. Positive press from funding agencies has raised not only the public's expectations for miraculous stem cell cures, but also a geographic and institutional bidding war for scientists pursuing cures for the most intrac- table diseases. Enter the opportunists. The Wikipedia description of the grifters who peddled snake oil in the early 1900s has a troubling simi- larity to the marketing by the unregulated stem cell clinics of today. " ...a 'doctor' with dubious credentials, selling fake medicines with boisterous marketing hype, often sup- ported by pseudo-scientific evidence. To in- crease sales, an accomplice in the crowd (a shill) would often attest to the value of the product in an effort to provoke buying enthusiasm." Like snake oil salesmen, clinics claiming astonishing curative results from stem cell treatments often do not have licensed phy- sicians administrating the treatments, no sci- entific evidence sup- porting their work, and they rely on tes- timonials for advertis- ing and promoting the value of their product. The first stem cell clinics started in coun- tries where the regulatory climate was less rigorous than that of the U.S. These clinics emerged in China, Mexico, the Bahamas, and even in European countries where loop- holes in regulations allowed the clinics to op- erate. In 2011, a large clinic in Germany was closed after the death of an 18-month-old boy who received an injection of cells into his brain. 2 The clinic's director was not pros- ecuted and soon established another stem cell clinic in Beirut, Lebanon, where new loopholes in regulation could be exploited. Like a money-making franchise, stem cell clinics have begun to spread to the U.S. Anyone who visits or lives in Florida or California will have noticed billboards and full-page advertisements for U.S. clinics of- fering stem cell therapies that have not been confirmed to be safe and effective. The websites are typically strong on mis- sion statements but short on specifics. They claim that their treatments are noninvasive and preferable to surgery. They emphasize the miracle of the customer's own cells being used to treat their condition. What's wrong with using someone's own cells? What is wrong is that the cells used by these clinics are not useful for therapy. The cells are either an extract of fat tissue that is removed from the patient by liposuction or cells from bone marrow. These cells are sim- ply incapable of turning into any of the cell types that are damaged by Alzheimer's dis- ease, Parkinson's disease, diabetes, multiple sclerosis, COPD, ALS, cerebral palsy, autism, spinal cord injury, or traumatic brain injury. In fact, cells taken from one source like fat, and put somewhere else, like the bloodstream, do not even survive beyond a few hours. They have no demonstrated medical benefit. The clinics offer no functional rationale for their treatments. Instead, they seek le- gitimacy by association. One strategy is to link their websites to scientific publications that relate to the given condition and stem cells. To give an example, there are reports that show that stem cell transplants can re- store short-term memory to mice that have some characteristics of Alzheimer's disease. As a preclinical investigation the work has real value. But this does not mean that such a therapy is likely to work in humans, and a great deal more research is required before any legitimate medical practitioner would consider trying it on people. Patient Testimonials Since there is no scientific basis for their treatments, the clinics use patient testimo- nials and patient ratings to give the illusion that there is a medical benefit to their activi- ties. The testimonials say that the patients are satisfied, they trust the organization, and they would recommend the treatment to oth- ers. But this is not science, and these are not verifiable clinical milestones and outcomes. This is advertising. It is important to understand the role of the FDA in regulating stem cell therapies at these clinics. The FDA has made it clear that two characteristics of a stem cell treat- ment make it subject to FDA regulations. POINT OF VIEW Protect Stem Cell Science from Stem Cell Quackery Graham Parker, Ph.D., is a professor in the department of pediatrics at the Wayne State University School of Medicine in Detroit and editor-in-chief of Stem Cells and Development, a peer-review journal published by Mary Ann Liebert, publishers. Jeanne F. Loring, Ph.D., is professor of developmental neurobiology and director of the Center for Regenerative Medicine, department of chemical physiology, on the California campus of the Scripps Research Institute. Dr. Loring is also on the editorial board of BioResearch Open Access, published by Mary Ann Liebert, publishers. Keep the Opportunities, Lose the Opportunists Although stem cells show great promise in regenerative medicine, they are not the stuff of miracle cures or all-purpose elixirs, whatever latter-day snake oil salesmen or their enthusiastic shills might claim. > After Completing $39B Acquisition, Teva Does Another Generics Deal with Allergan A day after completing its nearly $39 billion acquisition of Actavis Generics from Allergan, Teva signaled it wants to grow even bigger in generics through another smaller deal with Allergan, saying it agreed to ac- quire its Anda generic distribution business for $500 million. Anda distributes generic, brand, spe- cialty, and over-the-counter pharmaceutical products from more than 300 manufactur- ers to retail independent and chain phar- macies, nursing homes, mail-order pharma- cies, hospitals, clinics, and physician offices across the U.S. Through the acquisition, Teva said, it will gain 650 employees based at Anda's centers in Olive Branch, MS; Weston, FL; and Groveport, OH. Anda, the fourth-largest distributor of generics in the U.S., is expected to generate approximately $375 million in net revenues in the second quarter, and approximately $1.5 billion in third-party net revenue for all of 2016, Allergan said. > Pfizer Acquires Bamboo Therapeutics in a $645 Deal Pfizer acquired Bamboo Therapeutics, which focuses on developing gene therapies for the potential treatment of patients with certain rare diseases related to neuromuscular conditions and those affecting the central nervous system. This acquisition significantly expands Pfizer's expertise in gene therapy by providing Pfizer with clinical and several pre- clinical assets that complement the company's rare disease portfolio, an advanced recom- binant adeno-associated virus (rAAV) vector design and production technology, and a fully functional Phase I/II gene therapy manufactur- ing facility that Bamboo acquired from the University of North Carolina earlier this year, according to Mikael Dolsten, president, Pfizer worldwide research & development. Pfizer previously acquired approximately 22% of Bamboo's equity during the first quarter of 2016 for $43 million. Under the terms of this transaction, Pfizer acquired all of Bamboo's remaining equity for an upfront payment of $150 million. > Tokai Eliminating 60% of Work- force, Shrinking to 10 Employees Tokai Pharmaceuticals will eliminate approximately 60% of its workforce— about 15 jobs—by the end of the third quarter, in a cost-cutting move an- nounced days after the company's lead candidate galeterone failed a Phase III trial. The layoffs, disclosed after the close of the markets on July 29, will leave Tokai with 10 full-time employees. n News Industry Watch See Stem Cell Science on page 10