[orting]

The phase 2 study primarily looked at safety. There was no correction for multiple hypothesis testing on the efficacy endpoints. So it seems that the only conclusion that is warranted is that the study shows no adverse health effects and that "larger clinical trials are warranted to establish the efficacy of hMSCs in this multisystem disorder." as they state in the conclusion.

It is interesting if it works, but lets wait for the next phase before assuming it does.

[nopinsight]

“In the first trial 15 frail patients received a single MSC infusion collected from bone marrow donors aged between 20 and 45 years old. Six months later all patients demonstrated improved fitness outcomes, tumor necrosis factor levels and overall quality of life.

The second trial was a randomized, double blind study with placebo group. Again no adverse affects were reported and physical improvements were noted by the researchers as "remarkable".

"There are always caveats associated with interpreting efficacy in small numbers of subjects, yet it is remarkable that a single treatment seems to have generated improvement in key features of frailty that are sustained for many months," writes David G. Le Couter and colleagues in a guest editorial in The Journals of Gerontology praising the research.”

[orting]

I am not criticizing the study. I am highlighting that the conclusion that the paper arrives at i the correct one: That it warrants large scale studies.

This work is a prime candidate for being misrepresented as showing that this stem cell treatment is effective for age related health issues.

There are 30 participants in the phase 2 trial. There are two treatment groups (100M and 200M) with different dose and one placebo group. Each group has 10 participants.

None of the treatment groups showed adverse effects.

There is a difference between asking "Are there any adverse effects?" and "are there positive effects for parameter 1 to n"? If you ask the second kind of question and do not correct for multiple hypothesis testing, you will make many errors.

The small dose treatment group (100M) showed improvement in many parameters vs placebo, whereas the other (200M) showed improvement in fewer parameters vs placebo. Since no corrections where made for testing, this only tell us that there where no statistically significant adverse effects.

As I noted initially, I think it is interesting. Once we have seen the results of a couple of large studies, we can talk about the effects of this treatment.

[nopinsight]

Thank you for your reply, in particular for the specific issue with the results. I quoted the article to show that the researchers seem to believe that the trial results are more promising than a formal analysis would suggest. I agree that further studies are required.