The Korean-based stem cell biotech, RNL Bio, which also does business in the U.S. as RNL Bio and Human Biostar Inc, reportedly (seems to be a press release, PR) has filed an investigational new drug (IND) application with the Korean FDA (KFDA). The purpose of the application is to test the efficacy of autologous adult stem cells for the treatment of cerebral palsy.
In the PR, it says the trial would treat up to 45 children using a product called RNL-AstrostemTM given intravenously (IV).
By way of background, cerebral palsy is a relatively common disease of brain injury in children, affecting about 3 kids out of a 1000. Stem cells have been proposed to treat this condition, but it is still early days in the investigation of this approach.
I found 12 clinical trials on stem cells and cerebral palsy, but only about half are actually related to this area of clinical investigation. Only one has results posted and it was an allogeneic trial. It suggested some subtle, transient (lasting a few months) improvement in function in transplant recipients. There was no clear evidence of an increase in major adverse events, which is good, but there was an increase in other adverse events. A limitation is that patients were only followed at most 6 months.
Getting back to RNL Bio, as evidence that the stem cell product is safe, the PR cites a RNL Bio paper that I felt, as a stem cell scientist, was relatively unconvincing as I discussed in a recent post (see here, the 4th paper discussed).
In today’s PR on the IND filing, Dr. JC Ra, leader of RNL Bio, is quoted:
“It is our mission to find cures for incurable diseases, such as the terrible pediatric curse of cerebral palsy, through autologous stem cell technology.”
RNL Bio has been in the news in the last year or so for a patient lawsuit filed in California, alleged smuggling of stem cells across international borders, and its legal conflict (both sued the other) with US partner Celltex.