The director of the Adult Sickle Cell Clinic and associate professor at University of Alabama Birmingham discussed her experiences serving as an investigator for lovo-cel clinical trials.
“I think that bluebird and lovo-cel have laid the way for all the gene therapies to come. We identified that bone marrow harvest wasn't the optimal way to gather stem cells for a number of reasons, and instead, are now using plerixafor-mobilized stem cells. And that has really allowed us to get a much better stem cell product that's truly a pluripotent stem cell. So, I think that that's been one of the most important discoveries that has been made. And I think using transfusions pretreatment, preharvest has been really helpful as well.”
Lovotibeglogene autotemcel (lovo-cel; bluebird bio) could be the first gene therapy approved for the potential treatment of sickle cell disease (SCD). The therapy is up for review by the FDA with a Prescription Drug User Fee Act (PDUFA) date of December 20, 2023.1 Whether the therapy gets approved in December or not, the years-long development of lovo-cel has undoubtedly advanced the state of research in the field of SCD.
Julie Kanter, MD, director, Adult Sickle Cell Clinic and associate professor, hematology and oncology, University of Alabama Birmingham, has served as investigator on multiple of lovo-cel's clinical trials, including the HGB-206 (NCT02140554) and HGB-210 (NCT04293185) trials. CGTLive spoke with Kanter to learn more about her experiences with helping to investigate lovo-cel and some lessons learned during its development and trial evaluations. One of the most important lessons she touched on was shifting from the use of bone marrow as a source of stem cells to plexifor-mobilized stem cells.