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.