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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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Weekly reads: Vertex, stem cells for MS, Athersys, 900-day paper review at Nature

“While still early, these results support the continued progression of our VX-880 clinical studies, as well as future studies using our encapsulated islet cells, which hold the potential to be used without the need for immunosuppression,” said Bastiano Sanna, Ph.D., executive vice president and chief of cell and genetic therapies at Vertex.

Vertex Pharmaceuticals has a lot going for it right now on the cell therapy front. For instance, they have the strongest type 1 diabetes cell therapy pipeline after some recent acquisitions. Still it’s not a simple matter to succeed in the cell therapy space even with one therapy for one targeted disease. Trials are tough …

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Weekly reads: Xist & autoimmune disease in women, Crohn’s disease, dumb headline of the week

Xist ,auto immune disease

Readers of The Niche have asked me many questions about stem cells for autoimmune disease but the puzzle of why women get these conditions more often than men hasn’t come up before here. For instance, why is MS so much more common in women than men? It’s remained somewhat of a mystery over the years. …

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Textbook case of bad media on ‘stem cell treatment’ clinic: Kirk Cousins Caribbean trip

Kirk Cousins, stem cell treatment

It’s frustrating to see so much bad media coverage of celebrity trips to clinics to get supposed stem cell treatment of various kinds. Part of the concern here stems from ordinary people taking risks by following the example of the famous people.  Good journalism should ask tough questions, talk to experts, and not yield an …

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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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20 stem cell & regenerative medicine predictions for 2024

Stem cell, crystal ball, predictions

Each year I make predictions for the stem cell and regenerative medicine field for the following year. Today’s prediction post is focused on what to expect in 2024. You can also see my stem cell predictions for 2023 and my grades on how I did. I also have made a video version of these predictions so please …

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2023 The Screamers Science Hype Award goes to anti-aging media coverage

The Screamers Science Hype Awards.

Every year I give out The Screamers Science Hype Award. The point of The Screamers is to raise awareness about science hype and catalyze new ways to combat it. In that spirit, I award what I see as the most extreme examples of science hype. Sometimes in addition to the one main winner, I give “honorable” …

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Weekly reads: RMAT jump, tissues as liquid crystals, gov stem cell advisor scammed of $650K

RMAT, FDA data

The FDA has a unique mechanism called RMAT or regenerative medicine advanced therapy designation. It’s intended to move investigational regenerative therapies forward more effectively. RMAT data I recently updated The Niche RMAT list and it has jumped to 85. The FDA has approved many new RMATs this year. The data on how many applications the FDA …

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Another kind of stem cell tragedy: William P. Murphy passes at 100

William P. Murphy, US Stem Cell

It’s kind of a tragedy that William P. Murphy, former chairman of unproven stem cell clinic firm, US Stem Cell, might be most remembered after his recent passing for his doings there.  For example, I didn’t know until now of his prolific past as a biomedical inventor who did a lot of good. Instead, my impression of …

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Weekly reads: FDA OKs 2 gene therapies for sickle cell, new Paolo Macchiarini series, Fujifilm

Marie Tornyenu, Casgevy gene therapies for sickle cell

The big news broke Friday that the FDA approved two new gene therapies for sickle cell disease. While the approvals were expected and the actual use of these treatments will be complex on several levels, this is a historic development. 2 FDA-approved gene therapies for sickle cell Friday’s news follows on recent UK approval for …

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