Dr. Shah on the Use of Tisagenlecleucel in Pediatric ALL

Bijal D. Shah, MD, associate professor, Department of Malignant Hematology, Moffitt Cancer Center, discusses the use of CAR T-cell therapy in patients with pediatric acute lymphoblastic leukemia (ALL).

Tisagenlecleucel (Kymriah) is approved for pediatric and young adult patients ≤25 years of age with B-cell precursor ALL that is refractory or in second or later relapse. The therapy has been particularly beneficial to patients with extramedullary disease, says Shah, particularly in those with refractory central nervous system disease. This is an area where the monoclonal antibodies blinatumomab (Blincyto) and inotuzumab ozogamicin (Besponsa) have shown much efficacy.

Any patient with refractory disease, irrespective of disease origin, could derive benefit from the CD19-directed CAR T-cell therapy. However, there are some limitations, cautions Shah. For example, the product is only approved for use in patients with B-cell ALL, not those with T-cell ALL. To that end, Autolus Therapeutics is developing a CAR T-cell product that may prove to be beneficial in this subset. Additionally, researchers are evaluating agents targeting CD5, CD7, and CD1A, which could fill the current absence of effective targeted therapies for patients with T-cell ALL, concludes Shah.