Phase 1 clinical studies are designed to determine the proper dosage of an agent and to assess the safety of a drug. Phase 1 studies are not designed to determine if the patients who take the drug or agent benefit from it. Therefore, it is highly gratifying to see a medical agent produce distinct improvements in a phase 1 study.
The Tisch MS Research Center of New York (Tisch MSRCNY) has announced in an April 23rd press release that patients enrolled in their FDA-approved Phase I trial using autologous neural stem cells in the treatment of multiple sclerosis (MS) show significant improvements. These results were presented during the Multiple Sclerosis Highlights in the Field session at the 67th American Academy of Neurology (AAN) Annual Meeting in Washington, D.C.
MS is a chronic autoimmune disease of the central nervous system caused by attacks against the myelin sheath by the patient’s own immune system. The destruction of the myelin sheath causes systemic neurodegeneration. MS affects more than 2.3 million people worldwide.
In its interim analysis of their data, Tisch MSRCNY researchers reported that six of the nine patients treated with stem cells show increased motor strength, improved bladder function and an enhanced quality of life. Significantly, these treatments are well tolerated and, to date, no serious adverse events were reported.
“This preliminary data is encouraging because in addition to helping establish safety and tolerability, the trial is yielding some positive therapeutic results even at this early stage,” said Dr. Saud A. Sadiq, Chief Research Scientist at Tisch MSRCNY and the study’s principal investigator. Sadiq cautioned that these results result from an interim analysis and definitive conclusions will only be made upon completion of the trial.
The Tisch MSRCNY study investigates a pioneering regenerative strategy that utilizes stem cells harvested from the patient’s own bone marrow. Specifically, a special stem cell population called “MSC-NPs” or mesenchymal stem cell-derived neural progenitors are isolated from bone marrow and used in this clinical trial. MSC-NPs represent a neural subpopulation of bone marrow-derived MSCs with reduced mesodermal pluripotency and minimized risk of ectopic differentiation. In preclinical studies in laboratory mice afflicted with “experimental autoimmune encephalomyelitis” (an excellent model system for MS), Tisch MSRCNY scientists established that three doses of MSC-NPs delivered intrathecally (IT) resulted in improved neurological function associated with suppression of local inflammatory response and trophic support for damaged cells at lesion sites.
Once the MSC-NPs were isolated from the patient’s bone marrow stem cell, they were injected intrathecally, that is, into the cerebrospinal fluid surrounding the spinal cord, in 20 participants who meet the inclusion criteria for the trial. This is an open label safety and tolerability study, which means that both the physicians and patients know what treatments that are giving and receiving in contrast to blinded studies. All clinical activities in this study will be are conducted at Tisch MS Research Center of New York or at affiliated International Multiple Sclerosis Management Practiced. The interim analysis reports on the first nine patients who received at least one treatment of stem cells.
Study patient Vicky Gill, a married mother of two whose husband, Michael, also has MS, has already experienced the positive benefits of the therapy. “For the past six years, I would fall frequently, had very limited movement in my legs and always walked with my cane. After just two stem cell treatments, I am now gaining sensation and function I thought was totally lost, have not had any recent falls and at times don’t need a cane at all.”
The patients in this trial will receive three rounds of injections at three-month intervals. Safety and efficacy parameters will be evaluated in all patients through regular follow-up visits. Dr. Sadiq plans to continue and complete the Phase I study and if these positive trends continue, move on to a multi-center Phase II efficacy trial.