The clinical assistant professor at Stanford Medicine also shared his excitement on the recent approvals of lovo-cel and exa-cel.
“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.