Sometimes things in the stem cell world get complicated and this week is no exception with news of Orchard Therapeutics dropping a seemingly life-saving clinical trial. There are hundreds of kids who could possibly benefit.
Rotten apple decision from Orchard Therapeutics
From David Jensen at California Stem Cell Report we have something of a scoop: ‘Wicked’ Problems, Orchard Therapeutics and Bubble Babies: A California Stem Cell Story.
Orchard Therapeutics cited “business reasons” for terminating its seemingly successful stem cell clinical trial for SCID.
It feels to me like a tragedy to see a biotech stop a trial that seems to have already saved lives and could save many more.
What about the tens of millions of dollars that CIRM invested here? Should Orchard have to pay that back? Jensen has a follow-up piece at Capitol Weekly with more information about CIRM’s perspectives on the decision by Orchard Therapeutics.
The more encouraging news is that CIRM seems very devoted to continuing this work clinically. The hope is that another biotech will pick up the trial and Orchard will facilitate that.
Here’s a newer update from David Jensen.
Heart self-organization from stem cells
Some more recommended papers: T cells and more
- Generation of hypoimmunogenic T cells from genetically engineered allogeneic human induced pluripotent stem cells, Nature Biomedical Engineering.
- Human delta like 1-expressing human mesenchymal stromal cells promote human T cell development and antigen-specific response in humanized NOD/SCID/IL-2R $$\upgamma $$ γ null (NSG) mice, Scientific Reports
- MeCP2 controls neural stem cell fate specification through miR-199a-mediated inhibition of BMP-Smad signaling, Cell Reports
- SFRP2 induces a mesenchymal subtype transition by suppression of SOX2 in glioblastoma, Oncogene
- Study of inherited thrombocytopenia resulting from mutations in ETV6 or RUNX1 using a human pluripotent stem cell model, Stem Cell Reports
- Acute myeloid leukemia-induced functional inhibition of healthy CD34+ hematopoietic stem and progenitor cells, Stem Cells.
Dear James,
Perhaps you should research on how trials work, how they are developed, what the endpoints are, and how approval is obtained. It seems as if you’ve got some suppositions about the process, but very little actual understanding. While everyone is entitled to their opinion, you really should base it on facts. Actual facts, not some things one thinks they know, but have no understanding of in actuality.
Re the clinical trial in question, the company has never cited any significant technical difficulties. The company, in fact, last fall specified financial reasons for suspending OTL-101. Further, the NEJM this month carried an article that effectively touted the success of the trial. Orchard put it this way: “Results showed 100% overall survival and ≥95% event-free survival …”
Here is a link to the NEJM article. https://www.nejm.org/doi/full/10.1056/NEJMoa2027675?query=featured_home
Here is the latest from California Stem Cell Report concerning Orchard’s denial of compassionate use of OTL-101 for 20 children. https://david293.substack.com/p/bubble-baby-families-denied-compassionate
Dear Admin:
No matter what they may explain publicly about “business reasons”, pharma and biopharma companies don’t abandon successful clinical trials for an important medical need with a previously well-defined attractive market. If the trials are succeeding, they can always find waiting venture investment. Instead, they abandon clinical trials because they have encountered a technical problem in the trial that they can’t fix. Often it’s a fundamental flaw in the tech that they ignored from the start when early investments were flowing in. This is CIRM’s risk; and Orchard should not be expected to give them a penny back unless dishonest or criminal behavior is discovered. Most clinical trials fail. That’s the business this is. The science is not established. No matter what happened in mice and monkeys, it’s still experimental in patients.
Don’t forget they are clinical TRIALS, not clinical cure development, which is the way you and many others often mistakenly relate to them.
James @ Asymmetrex
I don’t see information on Orchard’s trial having tech problems, but I suppose it’s possible. It is important to be cautious in interpreting results from still ongoing trials, but with SCID it’s a unique situation where the usual outcomes are often fatal so this trial really does seem encouraging as a life saver.
@James,
You’ve never run any clinical trials, right? It’s funny to see you writing a holier-than-thou comment about trials not being cure development, but you tend to defend stem cell clinics you market cures all the time with zero real data.