David Shyr, MD, on ASH 2023 Highlights, Further Research for Nula-Cel SCD Therapy

“This is the first step right now, the patient who needs a cure, who transplant is not indicated ,but has another option. Could this option replace transplant? I think so. I think that's the future, that you don't need to do allogeneic transplant for this disease anymore. We can do gene therapy. I think competition is good; competitions allows improvement.”

The CRISPR/Cas9-edited cell therapy KMAU-001 (nulabeglogene autogedtemcel; Kamau Therapeutics) yielded a marked improvement in quality of life with zero vaso-occlusive events (VOEs) or other manifestations of SCD in the first patient with sickle cell disease (SCD) treated in a phase 1/2 clinical trial (NCT04819841). However, the patient first experienced low cell counts and decreasing amounts of the corrected hemoglobin A allele and the mechanism of the therapeutic benefit is unknown. Investigators found that she was mostly producing fetal hemoglobin, which KMAU-001 is not designed to have an effect on, although she recovered after receiving eltrombopag and became transfusion-independent.

These data were presented by David Shyr, MD, clinical assistant professor, pediatric stem cell transplantation, Stanford Medicine, at the 2023 American Society of Hematology (ASH) Annual Meeting & Exposition, held December 9-12, in San Diego, California. CGTLive spoke with Shyr to learn more about further research that needs to be done with nula-cel. He also discussed things he was excited to see at the ASH meeting.

Click here to view more coverage of the ASH 2023 meeting.

REFERENCE

Shyr DC, Lowsky R, Miller W, et al. One Year Follow-up on the First Patient Treated with Nula-Cel: An Autologous CRISPR/Cas9 Gene Corrected CD34+ Cell Product to Treat Sickle Cell Disease. Presented at: ASH 2023 Annual Meeting; December 9-12; San Diego, California. Abstract 5000