journal club Archives

Longeveron’s Laromestrocel for Frailty: Breaking Down Cell Stem Cell Data & Problematic Nature Coverage

March 4, 2026 / Professor Paul Knoepfler, Ph.D. / Leave a Comment

Longeveron is a biotech studying clinical applications of mesenchymal stromal/stem cells or MSCs . MSCs are interesting, usually heterogeneous cell preparations. They are probably most well known for modulating the behavior of other cells via their secretome. The clinical potential of MSCs is often overstated, typically based on small, uncontrolled trials. So, when randomized, double-blinded, […]

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Early encouraging mitochondrial donation study produced 8 babies but long road ahead

July 18, 2025 / Professor Paul Knoepfler, Ph.D. / 3 Comments

A new reproductive method called mitochondrial donation has reached a milestone. The goal of this approach is to prevent mitochondrial diseases in children whose mothers have disease-associated mitochondrial DNA. Two linked NEJM papers on report encouraging results in humans from the innovative approach. The first small paper reported on this first-of-its-kind mitochondrial donation study, focusing

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Some skepticism as Shift Bioscience reports secret purported rejuvenation gene SB000 in preprint

June 11, 2025 / Professor Paul Knoepfler, Ph.D. / 12 Comments

Brendan Swain, Shift Biosciences, SB000, rejuvenation gene

What if there was a rejuvenation gene that made adult cells younger without turning them into potentially destructive embryonic, fetal, or tumor cells? It could be transformative for how we think about cellular aging and have many impacts on health. A new preprint posted on June 6th reports on a possible rejuvenation gene. The gene

Some skepticism as Shift Bioscience reports secret purported rejuvenation gene SB000 in preprint Read More »

Mostly discouraging trial of MSC-NPs or purported neural cells from MSCs for MS

June 11, 2024 / Professor Paul Knoepfler, Ph.D. / 2 Comments

The idea of mesenchymal cells or MSCs for MS has been around for a long time. However, there haven’t been that many strong clinical trials testing it. There’s a new paper out that reports one of the stronger MSC-related trials for MS in terms of design. In this case, they didn’t use just MSCs but

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Perspectives on new stem cells for paralysis paper & media coverage

April 10, 2024 / Professor Paul Knoepfler, Ph.D. / 5 Comments

A new paper on stem cells for paralysis from a Mayo Clinic team has sparked a bit of legitimate hope but also some hype in the media. The publication was in Nature Communications. It is entitled “Intrathecal delivery of adipose-derived mesenchymal stem cells in traumatic spinal cord injury: Phase I trial.” The approach was direct injection

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US clinics widely selling supportive oligonucleotide therapy (SOT) or Q-REstrain therapy from Greek firm RGCC

March 12, 2024 / Professor Paul Knoepfler, Ph.D. / 13 Comments

A few dozen clinics in the US sell an unproven RNA therapy called supportive oligonucleotide therapy or SOT, now known as Q-REstrain. The product comes from a Greek firm RGCC or Research Genetic Cancer Center, which also pre-tests patients’ gene expression to design the RNA product. Clinics marketing SOT in the US say it is

US clinics widely selling supportive oligonucleotide therapy (SOT) or Q-REstrain therapy from Greek firm RGCC Read More »

Put on a happy face? Perspectives on Mayo Clinic stem cells for heart disease Ph3 pub

January 11, 2024 / Professor Paul Knoepfler, Ph.D. / 6 Comments

One of the first regenerative medicine talks I ever saw was on stem cells for heart disease. I was still a trainee at the time. The professor, whom I won’t name, presented data from an early phase trial reporting that direct injections of some kind of cells right into the heart improved outcomes. Stem cells

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Heart stem cells trial mostly disappoints but is oversold

March 2, 2023 / Professor Paul Knoepfler, Ph.D. / 6 Comments

Are there endogenous adult heart stem cells that are useful in cardiac disease or can stem cell infusions help ailing hearts? As to the first question, the adult heart does not appear to have meaningful numbers of true stem cells. Further, testing the idea of infusions of stem cells for heart disease has so far been giving

Heart stem cells trial mostly disappoints but is oversold Read More »

Perspectives on David Sinclair anti-aging Cell pub & in vivo reprogramming

February 7, 2023 / Professor Paul Knoepfler, Ph.D. / 2 Comments

About twenty years ago a science story made big news of a so-called anti-aging Methuselah gene. Methuselah gene and anti-aging The claim was that this DNA conferred long life on people. Hence the name Methuselah, which refers to a man from the Bible who reportedly lived 969 years. The so-called Methuselah gene was at first not a

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Review of ExoFlo paper used to push Direct Biologics IND inside FDA finds red flags

November 3, 2022 / Professor Paul Knoepfler, Ph.D. / 7 Comments

I wrote recently about how political pressure on FDA Commissioner Stephen Hahn in 2020 may have helped the Texas firm Direct Biologics move their product ExoFlo forward inside the agency. Today I analyze the ExoFlo Phase I clinical trial paper used in that push on Hahn to get the firm the IND for a Phase II

Review of ExoFlo paper used to push Direct Biologics IND inside FDA finds red flags Read More »

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