News Release 

Ohio State-developed drug receives FDA approval for treatment of CLL and SML

Ohio State University Wexner Medical Center

COLUMBUS, Ohio - On Nov. 21, 2019, the U.S. Food and Drug Administration (FDA) approved the use of the drug acalabrutinib for first-line therapy in chronic lymphocytic leukemia (CLL) and small cell lymphoma (SCL).

This is the first full approval of the targeted drug therapy, which was developed and tested at The Ohio State University Comprehensive Cancer Center - Arthur G. James Cancer Hospital and Richard J. Solove Research Institute (OSUCCC - James) in collaboration with the pharmaceutical partner, Acerta Pharma.

Acalabrutinib (pronounced uh-KA-luh-BROO-tih-nib and marketed as Calquence®) is a second-generation Bruton tyrosine kinase (BTK) inhibitor, a newer class of drugs shown to improve the survival of patients with mantle cell lymphoma in addition to CLL and SML.

The drug works by permanently binding BTK, which is part of a chain of proteins that relays growth signals from the surface of the cancer cell to genes in the cell nucleus, enabling cancer cells to survive and grow. By blocking BTK, the drug halts the flow of these growth signals, and the cancer cells die.

Unlike the first generation BTK inhibitor (ibrutinib, marketed as IMBRUVICA®), preclinical and clinical data shows that acalabrutinib more selectively blocks the BTK pathway without disrupting other key molecular pathways important for preserving platelet and immune function, thereby preventing/minimizing certain side effects associated with cancer treatment.

The foundational basic-science research, initial phase I clinical trial and numerous sequential phase II and phase III clinical trials that led to this new FDA approval of acalabrutinib were performed by a team of researchers at the OSUCCC - James led by John C. Byrd, MD, Distinguished University Professor and the D. Warren Brown Designated Chair in Leukemia Research at the OSUCCC - James.

This research included collaborative clinical trials with the The Ohio State University College of Veterinary Medicine and the Comparative and Translational Oncology Program, a research collaboration that integrates nearly 40 scientific investigators from Ohio State's colleges of medicine, pharmacy, nursing and veterinary medicine, along with researchers from Nationwide Children's Hospital, to investigate cancers that occur in both humans and animals. William Kisseberth, DVM, directed the studies of acalabrutinib in dogs with lymphoma.

"Acalabrutinib is a highly potent and selective oral BTK inhibitor that has proven to be very effective for our patients affected by CLL and other blood cancers. It is remarkably well tolerated and results in longer progression-free survival. We are honored and thrilled that this research is helping patients thrive," says Byrd.

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Collaborator Jennifer Woyach, MD, will present data at the upcoming American Society of Hematology annual meeting on the mechanisms of resistance that cause some patients to stop responding to acalabrutinib.

The research with acalabrutinib was supported by the National Cancer Institute, Leukemia and Lymphoma Society, Four Winds Foundation, D Warren Brown Foundation, Connie Brown CLL Foundation, Sullivan CLL Foundation and Pelotonia.

To learn more about leukemia research and patient care at the OSUCCC - James, visit cancer.osu.edu.

About the OSUCCC - James

The OSUCCC - James strives to create a cancer-free world by integrating scientific research with excellence in education and patient-centered care, a strategy that leads to better methods of prevention, detection and treatment. Ohio State is one of only 51 National Cancer Institute (NCI)-designated Comprehensive Cancer Centers and one of only a few centers funded by the NCI to conduct both phase I and phase II clinical trials on novel anticancer drugs sponsored by the NCI. As the cancer program's 356-bed adult patient-care component, The James is one of the top cancer hospitals in the nation as ranked by U.S. News & World Report and has achieved Magnet® designation, the highest honor an organization can receive for quality patient care and professional nursing practice. At 21 floors and with more than 1.1 million square feet, The James is a transformational facility that fosters collaboration and integration of cancer research and clinical cancer care. For more information, visit cancer.osu.edu.

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