Future directions and Conclusion
The treatment landscape for cancer therapy has evolved significantly over the past few decades. Cellular therapies, such as tisagenlecleucel, offer tremendous promise in patients with treatment refractory B-ALL.
However, much work remains to be done to optimize the utility of cellular therapies in order to harness their full potential, including increasing accessibility to these agents, managing potentially life-threatening side effects, preventing disease relapse, and translating these successes in B ALL to solid tumors and other diseases. In addition, studies are underway to expand the success of tisagenlecleucel beyond r/r B-ALL to patients earlier in therapy. The Children’s Oncology Group phase II trial (AALLL1721) is evaluating the 5-year disease free survival rate in patients with high-risk B-ALL who are MRD positive at the end of consolidation to then receive tisagenlecleucel to determine if earlier administration of CAR T cells would have an impact on EFS and the ability to avoid HSCT. Continued understanding into basic immunology, genetic engineering, and ALL biology will surely result in more advances in cellular therapy that continue to improve outcomes in r/r childhood B-ALL and offer the opportunity for curative therapy in our sickest and most refractory patients.