Honors Gallery

FDA Approval: Personalized Cancer Treatment to Treat Lethal Blood Cancers

Award: Excellence in Technology Transfer

Year: 2018

Award Type: National

National Cancer Institute (NCI)

The National Cancer Institute (NCI), in collaboration with Kite Pharma Inc., a Gilead Company (Kite), developed a new treatment, approved by the U.S. Food and Drug Administration (FDA) which has been shown to be effective for several types of fatal blood cancers.

This unique therapy utilizes live cells and harnesses the power of a patient’s immune system to combat their cancer. A subset of a patient’s own immune cells, called T cells, are genetically modified to recognize the patient’s own tumors. The patient’s cells are collected, modified, and grown in the laboratory until they number in the billions. These programmed cancer-fighting cells are then reintroduced into the patient, now armed with the ability to potentially recognize and attack the cancer cells. This treatment is termed a “live therapy” and it requires only a single infusion.

NCI’s Technology Transfer Center (TCC) played a proactive role in this discovery-to-commercialization story. Beginning with facilitating a partnership with an industry partner, Kite, strongly committed to advancing this novel approach for the treatment of B-cell Non-Hodgkin’s lymphoma (NHL). This led to negotiating a complex, clinical trial Cooperative Research and Development Agreement (CRADA) involving both preclinical studies and later clinical trials using treatments made under stringent conditions (i.e., “Good Manufacturing Practice,” or GMP). Several other types of transactional agreements were also necessary to support advancement.

Successful development of the technology required effective coordination of diverse, multidisciplinary programs within and external to NCI—clinical compliance, regulatory affairs, GMP grade manufacturing, research scientists and T2 professionals (including licensing, patenting, and information technology staff). Kite’s expertise included preclinical and clinical research and development, regulatory, manufacturing, quality systems, and GMP production.

Its expertise enabled further development of the treatment and larger clinical studies in cancer patients. Armed with positive patient data from the early clinical trials, Kite submitted a Biologics License Application (BLA) to the FDA in March 2017 and was granted Priority Review.

The synergy of NCI’s scientific/clinical expertise with Kite’s clinical, regulatory, manufacturing, operation, and business capabilities was essential to the successful development and commercialization of this therapy and other products to address these cancers. Persistence was essential to endure the course of this lengthy translational process. However, that endurance has resulted in the development of this immunotherapy that can now benefit patients who are not responding to any other treatments, and would otherwise have little chance for survival. This is a significant medical breakthrough for this type of cancer, as evidenced by the FDA’s swift approval of the therapy in October of 2017.

Contact: Dr. Steven Rosenberg, (301) 496-4164, [email protected]