I spoke with Dr. Riordan yesterday. I found him very open and willing to discuss his work.
To date he has performed the cell transplant procedure on three young adults with Duchenne. Ryan is 23. Another young man was 28. The procedure was considiered Compassionate use.
Dr. Riordan is using MSCs (human mesenchymal stem cells). The origin of these cells is from bone marrow donors, age 18-65 and/or umbilical cord cells. Dr. Riordan said there is greater potency of cells from young donors (18+) and /or cord cells. The cells are grown in culture and prepared for transplant. Adult derived cells are capable of 20/30 doublings. Umbilical cord derived -200 doublings. He suggested that there is significant data to suggest MSCs integrate well and become muscle cells. But what if the environment has more fat/fiber than muscle? What evidence do we have the the MSCs will become muscle? Is it possible, the MSCs could actually make things worse? These are my questions and will be discussed in the near future.
Dr. Riordan said none of the young men are on an immune suppression protocol, that they have had no problems with rejection.
Ryan's biopsy was peformed in his home state, Dystrophin testing was done by Athena. Dr. Riordan thought the biopsy was done on the Quadriceps muscle, but would need to check.
Strength testing pre and post was done by physical therapists at the Stem Cell facility. Dr. Riordan did not have methods of testing, nor the pre and post testing available for the call.
Dr. Riordan is not accepting patients at this time. The procedure is quite expensive, in the neighborhood of $100,000 and the three young men who received stem cells were done at no charge. Dr. Riordan is interested in developing collaborations and clinical trials with academic centers.
Dr. Riordan is kind and genuinely interested in understanding the potential of MSCs in DMD, but significant questions remain before we would be able to jump on this bandwagon. Dr. Riordan was leaving Costa Rica today and will be traveling for a few days. We agreed, I would connect with Lee Sweeney and Gillian Butler Browne to schedule a conference call.
More on this in the next days/weeks.