Weekly reads: NurOwn, key gene screen, MS, Croce mess

I get a lot of questions about stem cells for ALS and more recently people have been specifically interested in NurOwn from BrainStorm Cell Therapeutics.

Nurown BrainStorm
A diagram of Nurown from BrainStorm.

Recently, the FDA denied BrainStorm’s request for a BLA for NurOwn. The company felt its Phase III trial was encouraging. It sounds like they will move forward with a follow-up meeting with the FDA to discuss this situation.

NurOwn is an autologous mesenchymal cell product that secretes neurotrophic factors. Another name is MSC-NTF cells.

It is also being marketed for MS.

More recommended reads

The phenotypic landscape of essential human genes, Cell. This paper has a lot of cool data. As someone who has spent so much time studying MYC, it was intriguing to see both it and its dimeric partner MAX (their genes) pop up here. My only issue with the paper is that they did the screen in HeLa cells. HeLa’s are very abnormal cells so what’s essential in them may not be the case in normal cells.

Stem cell therapy strikes out in progressive MS, MD Edge. This is discouraging. In other forms of MS, stem cell therapy paired with chemo seems more promising.

The ISSCR Announces New Stem Cell Reports Early Career Editorial Board, Stem Cell Reports. It’s a great idea to have an editorial board with early career scientists. I hope we see more of this.

FDA finalizes umbrella trial guidance for cell and gene therapies, RAPS. Did you know that you can try out different versions of a drug (say slightly different formulations of cells) for a disease in a single clinical trial?

Papers in Croce case with “blatantly obvious” problems still aren’t retracted after misconduct investigation: sleuth, Retraction Watch. I get the feeling more generally that a surprising number of journals just try to avoid dealing with misconduct issues. Maybe they feel like it’s in their best interest to not retract papers they published. So oftentimes they stall and dodge highly problematic papers. They might just correct papers where retraction is needed.

In world-first trial, lab-grown blood was just injected into two people, The Verge. I imagine a likely, positive future where at some point there are large quantities of synthetic blood available for use in many scenarios. This area is that promising.

3 thoughts on “Weekly reads: NurOwn, key gene screen, MS, Croce mess”

  1. I truly hope for the eventual success of NurOwn….it is an ingenious platform that I hope can bring hope to the ALS community. If anyone from BrainStorm should read this post: PLEASE KEEP TRYING, and remember the Mission.

  2. Nurown: it’s important to understand that FDA did not reject Nurown on the basis of wonky clinical trial data, rather FDA issued a Refusal to File notice. Once upon a time not that long ago, sponsors would send tons of disorganized papers to FDA and the agency had to rummage thru the mess to see if there was data sufficient to support marketing approval. Nowadays thankfully if required sections are missing or or the application is a mess of several thousand pages, FDA can issue a Refusal to File letter.

    This should not happen with a competent sponsor and there are many entities and consultants who specialize in writing NDAs so there’s no excuse for getting a Refusal to File letter.

    What’s up with BrainStorm management?

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