Very small safety study of mesenchymal stem cells at Mayo Rochester

There was a South Korean mesenchymal stem cell trial in MSA-C that published its results last year.  Mesenchymal stem cells are stem cells from bone marrow.  33 patients with MSA-C received either the placebo (17) or MSCs (16).  MSCs or placebo were infused first into arteries and then intravenously, over a one-year period.  This study was double-blind (neither the researcher nor the patient knew if the patient was receiving the placebo or MSCs).  The results showed that patients improved on the unified MSA rating score but not in the quality of life scale.  Also, improvement was transient.

One researcher who co-authored an editorial published in conjunction with the results is Phillip Low, MD at Mayo Rochester.  He is perhaps the leading MSA researcher in the US.  In the editorial, an argument was made that a multicenter, double-blind study needed to be done.  I guess Dr. Low hasn’t been able to organize a multicenter study but he has organized a very small one, taking place at Mayo Rochester.  It’s a phase I safety study; it is NOT an efficacy study.

Mayo Rochester is not recruiting for the study.  Enrollment is by
invitation only.  The study focuses on those in the early phase of
MSA.  There is a rigorous selection process.  Also, the study requires an extended period in Rochester, MN.

Here’s some general info posted about the study on the ShyDrager Yahoo!Group in late January:

“A couple of days ago, my dad was contacted by Mayo Clinic to see if he would be willing to participate in a new study/trial involving stem cells. There would be two groups, one would receive one dose of stem cells, the other would receive two. The gist of it is that they would remove stem cells from the fat in his thigh, then they would be reinserted directly into his spinal fluid to avoid the blood/brain barrier. Apparently this is a new way of inserting the stem cells (vis a vis the trials that have been going on in Korea). I believe the patients are in the hospital for about 3 days, then have to go for weekly checkups for a month (in Rochester), then check ups every 6 months. If patient are chosen for the double dose, this would be repeated for another month.  Apparently volunteers would start treatment immediately vs. waiting for a certain amount of people.  The study title is:  Intrathecal Autologous Mesenchymal Stem Cell Therapy in MSA – Effect of Dose and Natural History.”

I know some caregivers were discussing this at last Sunday’s support group meeting.  Thought you’d all like to know about this. We do have one local support group member who plans to participate.