Stem cell good news on biotechs & cool new organoid paper

There’s some recent stem cell good news out there including new papers and biotech developments.

The stem cell and regenerative medicine field is really diverse and runs the gamut from really worrying stuff to very exciting, legit developments. In this post, I’m going to focus on the encouraging recent developments. What do I see as the good news out there so far in 2018? I’m going to go through some of the highlights below and include quotes from announcements in some cases.

“The European Commission has approved Alofisel (darvadstrocel), a stem cell therapy to treat complex perianal fistulas — one of the most disabling complications of Crohn’s disease. The cell therapy represents an alternative to multiple surgeries for patients with Crohn’s that have shown an inadequate response to at least one conventional biologic therapy.

Previously known as Cx601, Alofisel was developed by the Belgian biotech TiGenix and is licensed to the Japanese pharma Takeda. The marketing authorization comes with a €15M milestone for TiGenix, which will now proceed to transfer the authorization to Takeda.”

vascularized brain organoid paper
Pham, et al., Figure 2, NeuroReport

This provides concrete hope for Crohn’s patients suffering from this condition. If approved in coming years in the U.S. this would also be a milestone as a new type of approved stem cell therapy beyond HSCT and specific cord blood-related uses that are OK now.

One of the limitations of organoid technology generally and for brain organoids is simple diffusion. As much as we grow organoids in ways that maximize nutrient and oxygen availability, organoids can only grow healthy up to a certain point. Also, without vasculature, organoids are missing a key normal physiological component.

Now a new paper from right here at UC Davis by a team at our Stem Cell Program reports a groundbreaking methodology for generating human brain organoids that are vascularized.

This kind of technology could address the earlier mentioned roadblocks and catalyze exciting new developments. Kudos to the team of my great colleagues (listed in authorship order on the paper): Missy T. Pham, Kari M. Pollock, Melanie D. Rose, Whitney A. Cary, Heather R. Stewart, Ping Zhou, Jan A. Nolta, and Ben Waldau.

You can see Figure 2 above from this paper.

For a stem cell biotech to get orphan drug status for a product is a major positive development. Cynata recently got this good news on their CYP-001 product for treatment of acute graft versus host disease. From the company:

“CYP-001 is based on Cynata’s Cymerus technology platform, which uses induced pluripotent stem cells (iPSCs) to generate an unlimited number of uniform, therapeutic mesenchymal stem cell (MSC) products based on a one-time blood donation from one adult donor.”

You can see my interview with Cynata CEO Ross MacDonald here.

  • RMATs grow.

The list of regenerative medicine advanced therapy (RMAT) designations grew to 15 recent. You can see my full list here. I see this as good news and haven’t seen any approvals that stand out as particularly concerning, but the RMAT program is a regulatory experiment and we don’t know how it will turn out. I’m hopeful it’ll be a net positive for the field and patients with some RMATs ultimately turning into approved therapies in the future, proven safe and effective.

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