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The LLS Blog

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FDA Approves First CAR T-Immunotherapy for Relapsed/Refractory Acute Lymphoblastic Leukemia

By Lee Greenberger, Ph.D. |
Leukemia Breaking News

The U.S. Food and Drug Administration (FDA) recently announced approval of brexucabtagene autoleucel (Tecartus®) as the first and only CAR T-cell treatment for adults with relapsed or refractory B-cell acute lymphoblastic leukemia (ALL). Roughly half of all ALL cases occur in adults, and unlike pediatric ALL, adults have historically had a poor prognosis. This approval, which follows an FDA Breakthrough Therapy Designation and priority review, is a meaningful advance for these patients.

This approval also marks the continuing progress in bringing the promise of CAR T-immunotherapy to more patients with a range of blood cancer types. There are now six CAR T-therapies approved in the U.S. that treat B-cell lymphoma, follicular lymphoma, mantle cell lymphoma, multiple myeloma and ALL.

The Leukemia & Lymphoma Society takes great pride in the expanding use these breakthrough therapies. LLS was among the earliest supporters of the CAR-T approach and the concept of harnessing a patient’s own immune system to fight their cancer. We continue to invest in ways to make CAR T accessible to even more patients.

Brexucabtagene autoleucel clinical trial outcomes show substantial benefit

FDA based its approval of brexucabtagene autoleucel on results from the ZUMA-3 study. After receiving a single CAR-T infusion, 52% of patients in the study had a complete response within three months. The duration of the response was estimated to exceed 12 months for more than half the patients. Without this new treatment, median survival time is about eight months.

Longer term data have also been published from earlier phases of the ZUMA-3 trial. Published phase 1 trial data reported that about one-third of patients who responded to the treatment were still in remission at the median follow up of 22 months.

Like other CAR T therapies, brexucabtagene autoleucel can cause cytokine release syndrome (CRS)—a dangerous immune overreaction with neurologic side effects. In ZUMA-3, CRS occurred in 92% of patients and neurologic toxicities occurred in 87%, but were generally well managed.

More information about ALL, including potential risk factors and helpful resources can be found here.

Looking to the future: cheaper, more effective, and more accessible CAR T

LLS was among the earliest supporters of the CAR T approach in the 1990’s and we continue to invest in ways to expand this breakthrough therapy to benefit more blood cancer patients.

Among our research priorities are “off the shelf” and gene therapy technology that would shorten the time it takes to deliver CAR T-treatment, targeting additional proteins implicated in other types of blood cancers—like BCMA for multiple myeloma; and finding ways to harness other parts of the immune system beyond T-cells, that could lead to even deeper and more long-lasting remissions.

We invested in much of this research through the LLS Therapy Acceleration Program® (TAP), a strategic venture philanthropy funding initiative. In 2015, TAP partnered with Kite, A Gilead Company to enhance the development of axicabtagene ciloleucel for the treatment of patients with refractory aggressive non-Hodgkin lymphoma (NHL). This funding helped lay the foundation to realize CAR T’s potential in other forms of blood cancers and LLS continues to drive innovation and further improvements in the advancement of CAR T.

LLS TAP is currently invested in Abintus Bio, which is developing cutting-edge in vivo CAR therapies that allow for powerful CAR T cells to be generated directly in a patient’s body, eliminating the need for time-consuming and costly collection, engineering and re-infusion of patient T cells. LLS is also supporting Caribou Biosciences, who are developing “off-the-shelf” CAR T-cell therapies, using healthy donor T cells for hard-to-treat blood cancers, instead of reengineering a patient’s own cells.

The future of CAR T continues to be bright and LLS is committed to continuing to support the development of potentially curative therapies for patients diagnosed with blood cancers.