May 26, 2020

The Niche

Knoepfler lab stem cell blog

New Stem Cell Clinic Trial for Alzheimer’s: Perspectives & Questions

A team at the University of Miami has launched a claimed first of its kind stem cell clinic trial for Alzheimer’s Disease (AD). The trial seems somewhat unique in using mesenchymal stem cells (MSC) for AD and is being run with biotech Longeveron.Test for Alzheimer's Disease

You can read more on the actual clinic trial including all the pertinent details here. I was unable to find a website for the company, but it is described here. Another stem cell trial for AD was announced last year that drew some notice and questions from me so I’m not sure that the Miami one is truly the first using MSCs. It doesn’t have to be first to be important.

The primary outcome measure in this new trial is safety and the secondary is efficacy for reduced AD symptoms and changes in quality of life. It is described this way officially:

“This is a randomized, placebo-controlled clinical trial designed to evaluate the safety and efficacy of LMSCs (Longeveron Mesenchymal Stem Cells) or placebo in subjects with Alzheimer’s Disease. Following a successful Safety Run-In Phase, a total of twenty-five (25) subjects will be randomized to (2:2:1) to receive low-dose LMSCs, high-dose LMSCs or placebo. After randomization, baseline imaging, and study product infusion, subjects will be followed up at 2,6,13,39 and 52 week post study product infusion. Intention-to-treat study population will be used for the purpose of the endpoint analysis and safety evaluations.”

Ultimately there will be three arms: 20 million Longeveron MSCs, 100 million Longeveron MSCs, and Placebo.

In a press release (PR) on the trial, the PI on the trial was quoted this way, “We believe infusions of these types of stem cells have the potential to be beneficial to individuals with Alzheimer’s disease,” said Bernard S. Baumel, M.D., assistant professor of neurology at the Miller School of Medicine, and principal investigator for the phase 1 clinical trial.”

The PR goes on to say, “Baumel is collaborating with Joshua M. Hare, M.D., Director of the Miller School’s Interdisciplinary Stem Cell Institute (ISCI) and Louis Lemberg Professor of Medicine, using mesenchymal stem cells developed by Longeveron, a UM life sciences spin-off company.”

In this kind of situation with systemic infusions of stem cells for neurological disorders, whether it is AD, autism, MS, or others, I always ask myself this kind of question, “Does a stem cell infusion clinic trial for Alzheimer’s make sense scientifically?”

I’m not 100% sure.

OK, yes, AD has an inflammatory component and MSCs can have anti-inflammatory immunomodulating properties, but is that a strong enough rationale for a trial? Maybe.

Also, in AD the blood brain barrier can be compromised so perhaps some MSCs put into the blood stream can get into the brain or could have a systemic anti-inflammatory effect. Again, maybe. Direct infusion of cells into the brains of AD patients might make more sense even if that seems like a more intense intervention.

One other issue that comes into play is consent in these kinds of trials. If the trial participants do indeed only have mild AD, then perhaps they can do a full informed consent, but that needs to be carefully evaluated. If there is uncertainty about whether their dementia has gone past a certain point, there is a key question of whether such consent is possible any longer. Does consent by family then come into play?

On the other hand, AD is a ubiquitous, fatal, and broadly devastating disease that goes beyond the patients themselves to family with a truly massive negative impact on society. There is no real treatment for AD. For these reasons, it seems reasonable to do trials such as this even if the rationale is a “maybe”. I hope the trial produces clear, encouraging data over the years.

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