A new Phase I clinical trial has demonstrated that Multiple Sclerosis (MS) patients were able to safely tolerate treatment with cells cultured from human placental tissue. The results of this study were recently published in the journal Multiple Sclerosis and Related Disorders. This pioneering study was conducted by researchers at Mount Sinai, Celgene Cellular Therapeutics, which is a subsidiary of Celgene Corporation, and collaborators at several other institutions, including the Swedish Neuroscience Institute in Seattle, WA, MultiCare Health System-Neuroscience Center of Washington, London Health Sciences Centre at University Hospital in London, the Clinical Neuroscience Research Unit at the University of Minnesota, the University of Colorado Denver, The Ottawa Hospital Multiple Sclerosis Clinic, and the MS Comprehensive Care Center at SUNY.
Even though this clinical trial was designed solely to determine the safety of this treatment, the data collected from the participating patients suggested that a preparation of cultured cells called PDA-001 may repair damaged nerve tissues in patients with MS. PDA-001 cells resemble “mesenchymal,” stromal stem cells, which are found in many tissues of the body. However, in this study, the cells were grown in cell culture systems, which means that one donor was able to supply enough cells for several patients.
“This is the first time placenta-derived cells have been tested as a possible therapy for multiple sclerosis,” said Fred Lublin, MD, Director of the Corinne Goldsmith Dickinson Center for Multiple Sclerosis, Professor of Neurology at Icahn School of Medicine at Mount Sinai and the lead investigator of the study. “The next step will be to study larger numbers of MS patients to assess efficacy of the cells, but we could be looking at a new frontier in treatment for the disease.”
MS is a chronic autoimmune disease. The body’s immune system attacks the insulating myelin sheath that surrounds and protectively coats the nerve fibers in the central nervous system. The myelin sheath greatly improves the speed at which nerve impulses pass through these nerves and without the myelin sheath, nerve impulse conduction becomes sluggish, and the nerves also eventually die off. Long-term, MS causes extensive nerve malfunction and can lead to paralysis and blindness. MS usually begins as an episodic condition called “relapsing-remitting MS” or RRMS. Patients will have occasional outbreaks of nerve malfunction, pain, or numbness. However, many MS patients will see their condition evolves into a chronic condition with worsening disability called “secondary progressive MS” or SPMS.
This Phase I trial examined 16 MS patients, 10 of whom had RRMS and six of whom were diagnosed with SPMS and were between the ages of 18 and 65. Six patients were given a high dose of the placental-based cell line PDA-001, and another six were given a lower dose. The remaining four patients were given placebos. Dr. Lubin noted that alteration of the immune system by any means can cause MS to worsen in some patients. Therefore, all participating subjects were given monthly brain scans over a six-month period to ensure they did not acquire any new or enlarging brain lesions, which are indicative of worsening MS activity. However, none of the subjects in this study showed any paradoxical worsening on MRI and after one year. The majority had stable or improved levels of disability.
“We’re hoping to learn more about how placental stromal cells contribute to myelin repair,” said Dr. Lublin. “We suspect they either convert to a myelin making cell, or they enhance the environment of the area where the damage is to allow for natural repair. Our long-term goal is to develop strategies to facilitate repair of the damaged nervous system.”