On August 30, 2017, the United States Food and Drug Administration (FDA) approved the cell-based gene therapy Kymriah for treatment of children and young adults with a certain form of acute lymphoblastic leukemia (ALL), the most common childhood cancer in America. The approval greenlights the first gene therapy to be made available in the United States.
Each dose of Kymriah is customized to the individual patient by way of an emerging form of immunotherapy called chimeric antigen receptor (CAR) T-cell therapy. T-cells are extracted from the patient’s blood and genetically modified in the laboratory to produce chimeric antigen receptors, surface-level proteins that enable the T-cells to recognize and fight leukemia cells that possess the antigen CD19. The newly engineered T-cells are then infused back into the patient’s body. The goal of Kymriah and other forms of immunotherapy is to target and attack the cancer cells that they are programmed to destroy.
This historic approval follows clinical trials demonstrating durable safety and efficacy in children and young adults up to age 25 with relapsed or refractory B-cell precursor ALL.
Weill Cornell Medicine and NewYork-Presbyterian Hosptial has ongoing clinical trials evaluating CAR T-cell therapy in adults with certain forms of leukemia. To learn more, visit: https://jcto.weill.cornell.edu/.
The United States Food and Drug Administration (FDA) has approved CPX-351, a combination of chemotherapy drugs daunorubicin and cytarabine also known as Vyxeos, for treatment of two types of high-risk acute myeloid leukemia (AML).
Clinical trial participants with newly diagnosed therapy-related AML (t-AML) and those with AML accompanied by myelodysplasia-related changes (AML-MRC) demonstrated increased life expectancy when treated with CPX-351, as compared to those treated with separate administrations of daunorubicin and cytarabine.
The Weill Cornell Medicine and NewYork-Presbyterian Leukemia Program, in collaboration with our Joint Clinical Trials Office, participated in the expanded access protocol for CPX-351, and we continue to use the drug across our various studies.
We were also among the sites for the clinical trial that led to another of this week’s FDA approvals: Idhifa, a targeted therapy for relapsed or refractory AML patients with the genetic mutation isocitrate dehydrogenase-2 (IDH2). After treatment with Idhifa, 34 percent of the 157 trial participants who required blood or platelet transfusions at the start of the study no longer required transfusions.
We are proud to be among the first medical centers offering novel treatment options like CPX-351 and Idhifa to our patients and look forward to continued prompt delivery of therapies that may improve life expectancy and quality of life for those affected by leukemia.