Weekly reads: CRISPR’d marsupial, outpatient recovery post stem cell transplants, Parkinson’s, pubs

This edition of The Niche weekly reads has a bit more good news than usual including on stem cell transplants. It’s nice to see positive developments like some recipients of hematopoietic stem cell transplants being able to stay at home afterwards.

Also, a reminder. You have until July 31 to enter our stem cell video contest to try to win $100. Your entry could be as simple as you talking about stem cells as you tape yourself on your phone or as complicated as animation. Our last winner years back did claymation. Click on the link above to see that video.

CBER newsy page helps find key new actions

By the way, this FDA page seems new, at least to me. The CBER branch of the FDA, which oversees cell therapies like stem cells, has a newsfeed-like page. You can easily track things like untitled letters, other communications, approvals, etc. Very helpful.

CRISPR opossums
CRISPR’d baby opossums.

But let’s start with some fun CRISPR gene-editing news.  The story from ScienceCRISPR creates first genetically modified marsupials. Here’s the source research paper from Current Biology: Targeted gene disruption in a marsupial, Monodelphis domestica, by CRISPR/Cas9 genome editing. You can see baby albino opossums above produced from the gene-editing. Is there any animals in which CRISPR won’t work?

Stem cell transplants with at-home recovery in some cases

From right here at UC Davis Health, Stem cell transplants now available for some on outpatient basis. This is great news for patients and the field, especially during the COVID-19 pandemic. From UCD oncologist Joseph Toscano:

“Outpatient stem cell transplants offer the full benefits of the standard inpatient version of this lifesaving procedure,” said Tuscano. “But we think there will be a decrease in recovery time and an increase in the mental wellness of patients who can become stressed by long hospitalizations and separation from family.”

Stem cell transplants for Parkinson’s research

News on the stem cells for Parkinson’s Disease front. FDA gives speedy review to Bayer’s Parkinson’s stem cell therapy FDA gives speedy review to Bayer’s Parkinson’s stem cell therapy, Pharmaphorum. Bayer-owner BlueRock Therapeutics got fast-track review. Parkinson’s is one of the most promising areas of cell therapy research with a good chance of an effective new treatment in the next 10-20 years.

Need for standards in the cell and gene therapy field

Evidence generation and reproducibility in cell and gene therapy research: A call to action, Molecular Therapy: Methods & Clinical Development. This piece by dozens of leaders including my colleague Gerhard Bauer right here at UC Davis is full of great ideas to make things better. Check it out. Here are some key points and recommendations:

Goals for optimal product development should include: (1) avoiding marketed products being withdrawn by manufacturers or regulators due to lapses in evidence generation; (2) systematic monitoring for potentially new and/or delayed adverse events not identified during clinical research phases (especially in orphan diseases with small pre-authorization studies); and (3) limiting the instances in which post-approval real-world evidence fails to confirm therapeutic benefits.

Additionally, the high upfront costs of some CGTs and their reimbursement challenges could potentially jeopardize their continued use and undermine confidence in the broader therapeutic category. In a field with such strong scientific prospects but also high degree of vulnerability due to limited clinical experience and evidence, transparency throughout research and development stages is key.

More news & pubs

3 thoughts on “Weekly reads: CRISPR’d marsupial, outpatient recovery post stem cell transplants, Parkinson’s, pubs”

  1. …if we make new dopaminergic cells for Parkinson’s won’t the alpha synuclein aggregates just destroy the new cells like they did they old cells?

    Isn’t that why the original fetal grafts failed?

    Any insight would be appreciated


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