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McBurney said he hopes that the BEAT-MS trial will carefully develop analytical aspects so that the researchers can ensure that the cells are in the best possible state prior to transfusion. The regulatory environment needs to be in good shape, McBurney said, so that if future trials for aHSCT go well, then the therapies can reach the MS community as quickly as possible.Įarly results from using autologous hematopoietic stem cells to treat MS have spurred the development of another upcoming trial, called the BEAT-MS trial. Preliminary results indicate that certain groups of patients have a higher likelihood of achieving NEDA with aHSCT than with other available disease-modifying therapies, although additional work is needed to verify these findings ( Muraro et al., 2017a, b Sormani et al., 2017). The multi-step process of aHSCT involves ablating the patient's immune system and attempting to reconstitute it using hematopoietic stem cells. NEDA is indicated by three measures of MS activity: no relapses, no progression to disability, and no new or enlarging lesions upon magnetic resonance imaging. One therapeutic approach uses autologous hematopoietic stem cell transplantation (aHSCT) to attempt to reach an endpoint known as “no evidence of disease activity,” or NEDA. In fact, he said, while there are excellent academic and industry-sponsored studies going on outside of the United States, there is also a great deal of medical tourism aimed at MS patients. industry-sponsored trials that are patient-funded to not be traditional clinical trials. However, McBurney said, he considers the four U.S. Of the 16 studies based in the United States, 12 academic studies are completed or are no longer recruiting, and 4 industry-sponsored trials, primarily using MSCs, are in the recruitment phase. Currently, he said, there are approximately 50 clinical studies under way worldwide in these 3 areas.
Speaking about the regenerative medicine opportunities in MS, McBurney highlighted three areas in particular: rebooting the “confused” immune system with hematopoietic stem cells or MSCs repairing the damaged myelin sheath with oligodendrocytes regenerated from MSCs or iPSCs and regenerating lost nerve cells and reforming appropriate connections using MSCs or iPSCs. iConquerMS currently has 36 individuals on its governing board, research committee, and engagement committee, 22 of whom are lawyers, scientists, neurologists, and biorepository experts who have MS. The iConquerMS network includes approximately 4,000 MS patients who are involved in the conception and conduct of research studies, the dissemination of information, and advocacy as a means of improving care for MS patients. One advantage to these virtual collaborations, he said, is that they often do not require participating organizations to complete legal agreements, a feature which has accelerated collaborations. This mandated data return, he said, has allowed for the creation of virtual collaborations among participating organizations. The biorepository, McBurney said, has supported more than 100 studies with a mandated return of data from any researcher who receives samples from the biorepository. The Accelerated Cure Project 1-founded in 2001 by an engineer who was diagnosed with MS-has two main programs: a biorepository containing DNA, RNA, plasma, serum, and white blood cells from 3,200 consenting individuals collected at 10 MS clinics around the United States, and a people-powered research network called iConquerMS™ which was started in 2014 with funding from the Patient-Centered Outcomes Research Institute. All of the approved therapies, he said, address aspects of the immune system's role in MS, but none address the fundamental cause of the disease. Over the past 25 years, there has been remarkable progress made in treating the symptoms of the disease, he said, with 15 FDA-approved therapies available today and 2 or 3 more that should be ready for regulatory review over the next couple of years. People living with MS experience such symptoms as loss of mobility, loss of vision, cognitive problems, fatigue, sleep disorders, and mental health problems, he said. This damage can lead to conduction failures and, ultimately, the death of nerve cells, McBurney said. Multiple sclerosis (MS) is an autoimmune disorder of the central nervous system that causes damage to the myelin sheath that insulates the nerve fibers. IMPROVING STEM CELL–BASED PRODUCTS: PERSPECTIVES FROM A PATIENT-CENTRIC RESEARCH ORGANIZATION
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