Stem cell trials are here

Fred D. Lublin, James D. Bowen, John Huddlestone, Marcelo Kremenchutzky, Adam Carpenter, John R. Corboy, Mark S. Freedman, Lauren Krupp, Corri Paulo, Robert J. Hariri, Steven A. Fischkoff DOI:

Background:Infusion of PDA-001, a preparation of mesenchymal-like cells derived from full-term human placenta, is a new approach in the treatment of patients with multiple sclerosis.
Objective: This safety study aimed to rule out the possibility of paradoxical exacerbation of disease activity by PDA-001 in patients with multiple sclerosis.
Methods:This was a phase 1b, multicenter, randomized, double-blind, placebo-controlled, 2-dose ranging study including patients with relapsing-remitting multiple sclerosis or secondary progressive multiple sclerosis. The study was conducted at 6 sites in the United States and 2 sites in Canada. Patients were randomized 3:1 to receive 2 low-dose infusions of PDA-001 (150 million cells) or placebo, given 1 week apart. After completing this cohort, subsequent patients received high-dose PDA-001 (600×million cells) or placebo. Monthly brain magnetic resonance imaging scans were performed. The primary end point was ruling out the possibility of paradoxical worsening of MS disease activity. This was monitored using Cutter’s rule (≥5 new gadolinium lesions on 2 consecutive scans) by brain magnetic resonance imaging on a monthly basis for six months and also the frequency of multiple sclerosis relapse.
Results Ten patients with relapsing-remitting multiple sclerosis and 6 with secondary progressive multiple sclerosis were randomly assigned to treatment: 6 to low-dose PDA-001, 6 to high-dose PDA-001, and 4 to placebo. No patient met Cutter’s rule. One patient receiving high-dose PDA-001 had an increase in T2 and gadolinium lesions and in Expanded Disability Status Scale score during a multiple sclerosis flare 5 months after receiving PDA-001. No other patient had an increase in Expanded Disability Status Scale score >0.5, and most had stable or decreasing Expanded Disability Status Scale scores. With high-dose PDA-001, 1 patient experienced a grade 1 anaphylactoid reaction and 1 had grade 2 superficial thrombophlebitis. Other adverse events were mild to moderate and included headache, fatigue, infusion site reactions, and urinary tract infection.
Conclusion:PDA-001 infusions were safe and well tolerated in relapsing-remitting multiple sclerosis and secondary progressive multiple sclerosis patients. No paradoxical worsening of lesion counts was noted with either dose.

So in this study stem cells were given to MSers and there as no disease worsening. However was their any improvement,  Maybe but too early to tell but effects were clearly not miraculous, or it would be plastered over the media. However what happens to the cells that are transplanted?

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  • I thought the hallmark of effective stem cell treatment is that they kill off the immune system before administering the new stem cells. It appears they didn't do that in this study. Is that correct?

    • You're thinking of bone marrow transplants, which destroys the immune system of the recipient and repopulates with bone marrow stem cells of the donor.
      This study is an infusion of mesenchymal stem cells into MSers whose immune system is intact to see if it's safe to do with the object of regulating the immune system rather than repopulating it.

  • So, in this trial they reports about treatment to be safe.
    I know that MESEMS trial results should have been reported
    at Actrims/Ectrims.
    Any news on this Profs.?

  • More cell based therapies to modulate the inflammatory process and promote repair….. stem cells for everyone!

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