Cells Weekly is a digest of the most interesting news and events in stem cell research, cell therapy and regenerative medicine. Cells Weekly is posted every Sunday night!
1. “Money back guarantee” for cell-gene therapy drug
High prices for marketed cell/ gene therapy products is a hot and controversial topic. For example, approved gene therapy drug Glybera was priced in 2015 as high as 1 million euros in Europe. The second approved in Europe gene therapy drug Strimvelis was recently priced for 594,000 euros. Still very expensive, but the twist is that manufacturer GSK offers “money back guarantee” policy:
“The drug has to deliver what you say or we don’t pay,” says Luca Pani, director general of the Italian Medicines Agency, known as AIFA, which set the price and terms during negotiations with the British drug giant. “If it does not work, they will return the money.”
This is historic moment for the field of cell/ gene therapy. GSK was considering different options in pricing/ reimbursement and decided to blaze a trail in such new model as “money back guarantee”. We will continue to watch post-marketing commercialization sales and adoption of Strimvelis.
2. CRISPR whistleblower
This week, email from whistleblower revealed some information, related to ongoing CRISPR patents fight:
In the e-mail, sent on February 28, 2015, Lin called the Broad’s claims “a joke” and “unfair to me and [to] science history.”
Lin’s account is striking not only because he worked in Zhang’s lab at the time but because he is listed as an inventor on Broad’s earliest patent filing, from December 2012.
The e-mail was sent as part of a job request to Doudna. In it, Lin, who is from China, seemed ready to barter inside information and assistance with the patent case in exchange for a job. “I am willing to give more details and records if you are interested or whoever is interested to clear the truth,” he said.
The Broad Institute posted very long and detailed response:
Although the rotation student’s email makes several claims, the Opposition Document does not include any evidence to support them.
Abundant evidence already shows that the student’s claims are false…
In a meantime Editas Medicine spent $10.9M this year only to pay Broad “legal bills in patent dispute over CRISPR”.
3. Results of RENEW cardiac cell therapy trial
Phase 3 clinical trial to assess autologous CD34+ cells in refractory angina was initiated by Baxter Corp. in 2012 (later managed by Bazalta – Baxter’s spin off). Couple of years ago, company suspended enrollment and later decided to terminate the trial. The results of terminated trial were recently published. The study analyses enrolled 112 patients (planned enrollment 444). Primary end points were not met. Some secondary end points were barely met. So, strategic business decision to stop the trial was probably based on interim analysis of the data.
4. Antibody-based conditioning in hematopoietic stem cell transplantation
Researchers from Stanford University described a new method for antibody-based conditioning of bone marrow in experimental hematopoietic stem cell transplantation. They added anti-CD47 antibody to previously used anti-c-Kit:
We show that host HSC clearance is dependent on Fc-mediated antibody effector functions, and enhancing effector activity through blockade of CD47, a myeloid-specific immune checkpoint, extends anti–c-Kit conditioning to fully immunocompetent mice. The combined treatment leads to elimination of >99% of host HSCs and robust multilineage blood reconstitution after HSC transplantation.
Antibody-based conditioning is not new approach and such antibody as anti-CD45 underwent clinical trials before. Antibody combinations could be more efficient and less toxic.
5. Advances in hematopoietic cells expansion
Sara Nolte of the Signals blog wrote a nice post on recent advances in hematopoietic stem cell expansion. She covered recent study about role of miR-125a and approach of startup company ExCellThera.
6. Testing self-renewal of human CD271+ MSC
Researchers from University of Lund (Sweden) described isolation and testing of self-renewal potential of CD271+ human mesenchymal stromal cells:
Furthermore, CD271pos/CD140alow/neg BMSCs gave rise to non-adherent sphere colonies (mesenspheres) with typical surface marker profile and tri-lineage in vitro differentiation potential. Importantly, serial transplantations of CD271pos/CD140alow/neg BMSC-derived mesenspheres (single cell and bulk) into immunodeficient NOD scid gamma (NSG) mice showed increased mesensphere numbers and full differentiation potential after both primary and secondary transplantations. On the other hand, BMSC self-renewal potential decreased under standard adherent culture conditions.
6. Plans of Fujifilm in regenerative medicine
Japanese company Fujifilm made a big buzz with aquisition of US-based iPS cell developer Cellular Dynamics International in 2015. CIRM’s blogger Karen Ring recently interviewed Fujifilm’s manager of Regeneratvie Medicine devision Toshikazu Ban:
FCDI plans to start iPS cell therapy clinical studies in the U.S. for age related macular degeneration in the year 2017, and clinical studies for retinitis pigmentosa, Parkinson’s and heart failure around 2019.
7. Gene therapy patient stories
Ricki Lewis of DNA Science Blog posted an overview of few patient (and companies!) stories, who underwent pioneering gene therapy interventions. Highly recommended to read!