Anti-Aging Stem Cell Treatment Proves Successful In Early Human Trials (newatlas.com)

An anonymous reader quotes a report from New Atlas: The results of two human clinical trials into a stem cell therapy that can reverse symptoms of age-associated frailty have been published, and the indications are that this landmark treatment is both safe and strikingly effective in tackling key factors in aging. Mesenchymal stem cells (MSCs) are a particular type of adult stem cell generating a great deal of interest in the world of science. This new MSC treatment is targeted at reducing the effects of frailty on senior citizens. This is the first anti-aging stem cell treatment directed specifically at the problem of age-associated frailty to move close to a final FDA approval stage. The treatment derives human mesenchymal stem cells from adult donor bone marrow and in these clinical trials involves a single infusion in patients with an average age of 76. Both Phase 1 and Phase 2 human trials have demonstrated the treatment to have no adverse health effects.

Although the two human trials were ostensibly designed to just demonstrate safety they do offer remarkable results in efficacy as well, paving the way for larger, Phase 3 clinical trials. 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." The next stage for the research is to move into an expanded Phase 2b clinical trial involving 120 subjects across 10 locations. After that a final, large randomized Phase 3 clinical trial will be the only thing holding the treatment back from final public approval. The results of the Phase 1 clinical trial were recently published in The Journals of Gerontology. The results of the Phase 2 clinical trial were recently published in The Journals of Gerontology. Further reading available via University of Miami, Miller School of Medicine.

Comments

Doesn't hold water

[philmarcracken]

Reposted from a more reputable interpreter of the results on reddit:

"I look at these results

(Fig. 2 from the journal article, supposedly demonstrating an improvement in frailty markers) and just see noise. No dose-response. No consistent benefit across measures for different treatment groups.

The figure shows four different tests for resilience to age-related frailty - each fig. 2a, 2b, 2c and 2d represent how each of the treatment groups performed on these tests at different time points.

There were three treatment groups: patients receiving 100 million cells (100-M), patients receiving 200 million cells (200-M) and a placebo group.
The 100 million cell group showed a stat dig improvement in the six minute walk test.

The problem, though, is that the 200 million group did not. So there is no dose response relationship. Generally, if a drug is real, the more drug you apply the stronger treatment effect you observe (lots of caveats to this generality, but none seem too relevant here).
Further, the 100 million group on showed a positive outcome in the six minute walk test. It failed to demonstrate efficacy consistently in the other three tests the researchers used to measure resilience against age-related frailty. In some cases it was even worse than placebo.

I would happily bet an amount of money that mattered to me that this result would fail to be replicated in a randomized, placebo-controlled study."

Re:Doesn't hold water

[Bill Hayden]

Very interesting results -- thanks for pointing them out.

It's notable that the 200M cell group performed at best the same, but usually worse than placebo on almost every test at every time frame. I'd have to disagree with you about dose response, though. Every medicine is going to have a bell curve of efficacy, and it looks like they just guessed too high on higher dose.

Also, you imply that the 100M cell group only improved on the 6 minute walk test. In fact, that group had statistically significant improvement on 3 out of 4 tests.

Re:Doesn't hold water

[drunken_boxer777]

I agree that it likely won't be replicated in a larger trial, but not for the reasons that you state.

Dose response can be straightforward in small molecule drugs, like aspirin. It becomes more complicated in biologic drugs, like granulocyte colony stimulating factor (filgrastim). We don't really know how it will work with stem cells, and the authors do list biologically plausible reasons why they don't see a dose response.

The real issue (besides the really low # of subjects) is the statistical analysis. They compare change from baseline (outcomes at 6 months vs. baseline) within a treatment group and look for statistical significance. What they should be doing is comparing change from baseline within a group vs. the change from baseline of the placebo group. See how the error bars of the 100 M group at 90 and 180 days overlap with the error bars of the placebo group in figure 2A? Not statistically different from placebo.

Granted, it would be crazy effective if it were statistically significant vs. placebo in such a small trial. However, their poor use of statistics (including no accounting for multiple analyses) is a major red flag. But this is about raising VC, as others might have pointed out. Multiple authors with MBAs rather than professional degrees (PhD, MD) is a giveaway.

Re:Doesn't hold water

[gurps_npc]

Dose response is for medication. This is not medicine, but instead a transplant of live cells.

Live cell transplants often do not get a dose response.

Compare with bread making - the difference between putting in 1 tablespoon of yeast and 2 tablespoons of yeast is minute.

I am not saying the study did great, but your major criticism is not appropriate for this type of treatment.