A study published in the journal Multiple Sclerosis and Related Disorders, led by researchers at Mount Sinai in New York and Celgene Cellular Therapeutics revealed that an infusion based on cells derived from the placenta proved to be safe for patients with multiple sclerosis (MS) and a promising new therapy for the disease. The study is entitled “Human placenta-derived cells (PDA-001) for the treatment of adults with multiple sclerosis: A randomized, placebo-controlled, multiple-dose study.”
MS is a progressive neurodegenerative autoimmune disorder that results from an attack on the central nervous system (CNS) by the body’s own immune system, causing inflammation and damage to the myelin layer that covers and protects nerve fibers in the CNS. Myelin loss leads to impairment in signal transmission along the nerve fibers, affecting motor function (such as coordination, balance, speech and vision), causing irreversible neurological disability, paralysis and blindness. MS usually starts as an episodic disorder known as relapsing-remitting MS (RRMS), and in many patients it evolves into a more severe chronic condition with worsening disability named secondary progressive MS (SPMS). It is estimated that more than 2.3 million people in the world suffer from the disease.
It has been previously shown that therapeutic cell-based infusions have an immunomodulatory and repair action in MS. PDA-001 in particular is a preparation of cultured mesenchymal-like cells derived from healthy human placental tissue and designed for the treatment of MS as these cells have immunomodulatory, anti-inflammatory, pro-regenerative and neuroprotective properties. As these placenta cells are expanded in cell culture, one healthy donor is capable of supplying enough cells for several patients.
In the study, researchers tested the safety and possible exacerbation of the disease with this new MS treatment approach based on PDA-001. A phase 1b, randomized, multicenter, double-blind, placebo-controlled study was conducted with 16 MS patients (10 with RRMS and 6 with SPMS), aged between 18 and 65 years. Six patients received a high dose of PDA-001 (600×106 cells), other six were given a lower dose (150×106 cells), and the remaining four patients received a placebo. Patients were monthly monitored for brain lesions through brain magnetic resonance imaging scans to assess possible disease progression.
Researchers found that none of the patients had worsening of MS-related brain lesions one year after treatment with both PDA-001 doses, and that in fact, the majority of the patients had stable or improved levels of disability.
“We’re hoping to learn more about how placental stromal cells contribute to myelin repair,” said the study’s lead author and Professor of Neurology at Mount Sinai, Dr. Fred Lublin in a news release. “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.”
The research team concluded that PDA-001 treatment was overall safe and well tolerated by patients, and that preliminary evidence suggests that PDA-001 could be able to repair damaged nerve tissues in MS patients.
“This is the first time placenta-derived cells have been tested as a possible therapy for multiple sclerosis,” noted Dr. Lublin. “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.”