PDA001 is a preparation of mesenchymal stromal (connective tissue) stem cells derived from human placental tissue, which is believed to be a potent, novel approach to treating autoimmune diseases such as Multiple Sclerosis (MS). Such allogenic transplants (from a different donor) are preferred in these cases due to minimum immunogenicity and fewer chances of rejection by both animal and human models alike. A large amount of this formulation can be prepared from a single donated placenta, which makes it a better option for further therapeutic developments. Research on this therapeutic formulation is being sponsored by Celgene Cellular Therapeutics (a subsidiary of Celgene Corporation).
Previous studies have already shown that therapeutic cell-based infusions have had an immunomodulatory, anti-inflammatory and pro-regenerative properties. Having been derived from the human placenta, these cells are all the more potent (multiipotent) and have significantly more immunomodulatory effects than other available cell-based formulations.
In order to test for the efficacy of any therapeutic formulation, its safety profile needs to be assessed and one needs to make sure that it does not cause further worsening of the condition. For PDA001, human studies have been conducted separately as well as in the form of clinical trials in this regard. An individual case of Primary Progressive Multiple Sclerosis (PPMS) was subjected to intrathecal and intravenous infusion of umbilical cord derived mesenchymal stem cells along with existing drug-based therapies often prescribed for the disease. A few weeks into the therapy, there was significant improvement in sensory impairment and muscle strength. Extensive monitoring at the end of 56 days showed improvements in neurological signs as well as the number of brain lesions as seen in the MRI scans.
In 2014, a safety profile study which tested whether the formulation worsened exacerbations and flare-ups in patients was performed over six sites in the United States and two sites in Canada. The study, a phase 1b, multicenter, randomized, double-blind, placebo-controlled, 2-dose ranging study, included patients with relapsing-remitting multiple sclerosis or secondary progressive multiple sclerosis. Ten patients with relapsing-remitting multiple sclerosis and six with secondary progressive multiple sclerosis were selected and were given intravenous infusions randomly, six were given a higher dose and six were given a lower dose, with the remaining four receiving a placebo. After following up for 12 months with extensive monitoring, it was seen that the doses were well tolerated by patients and it did not worsen flare-ups and exacerbations.
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