journal club

Perspectives on new stem cells for paralysis paper & media coverage

stem cells for paralysis

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 unproven RNA therapy, supportive oligonucleotide therapy or SOT, from Greek firm RGCC

SOT, supportive oligonucleotide therapy

A few dozen clinics in the US sell an unproven RNA therapy called supportive oligonucleotide therapy or SOT. 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 intended to act by …

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

stem cells for heart disease

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

heart stem cells

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 …

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

methuselah mouse, anti-aging

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

ExoFlo from Direct Biologics

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 …

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Paper sparks renewed interest in transdifferentiation & clinical potential

transdifferentiation, iOPCS

There was a time when I thought transdifferentiation based approaches might quickly move into clinical trials. Then things kind of cooled off. We didn’t see many papers reporting methods to transdifferentiate cells. I still think that this technology, sometimes called direct reprogramming, has major potential. A new paper on making human brain cells this way got …

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Nature paper claims young CSF fights brain aging in mice

old mice get young CSF

A new Nature paper argues that young CSF fights brain aging. Young CSF vs. young blood CSF is the cerebrospinal fluid that surrounds the brain, including those of young mice. The claim is that when young CSF is injected into the brain/CNS cavity of old mice, it makes the aged brains seem younger. And function …

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Monkey human embryo chimera Cell paper: exciting, ethically complex direction

human embryo chimera

A new Cell paper from an international team led by Juan Carlos Izpisua Belmonte pushes human embryo chimera embryo research further than ever before. It is both exciting work and raises many complex bioethics questions at the same time. What is a human embryo chimera? By way of background, chimeric human embryos contain a mixture …

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Journal club: Jaenisch lab paper on epigenetic CRISPR-Cas9 rescue of Fragile X in a dish

Liu-et-al.-Figure-6C-Fragile-X-CRISPR-Cell-e1519673510685

There’s much more to CRISPR-Cas9 than just gene editing and a new paper from the lab of Rudy Jaenisch in Cell highlights that in an exciting way. It reports epigenetic reversal of a Fragile X Syndrome phenotype in induced pluripotent stem cell (IPSC) neurons. Fragile X Syndrome is a neurological disorder in boys resulting from CGG repeat …

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