A therapy developed at Ohio State is improving quality of life for some leukemia patients by reducing side effects associated with previous drugs. Learn more about acalabrutinib, which was recently approved for the treatment of chronic lymphocytic leukemia (CLL) and other blood cancers. When David Neiger, MD, now 63, was diagnosed with CLL in April 2014, his testing suggested he would likely not respond to traditional treatments for the disease. Those treatments would also leave his immune system susceptible to infection, making it difficult for him to continue his practice as a primary care physician. His hematologist, John C. Byrd, MD, co-leader of the Leukemia Research Program at The Ohio State University Comprehensive Cancer Center – Arthur G. James Cancer Hospital and Richard J. Solove Research Institute (OSUCCC – James), recommended an experimental oral therapy, which has since been approved (November 2019) by the U.S. Food and Drug Administration (FDA) for the treatment of CLL and small cell lymphoma (SCL). David would need to take just two pills a day — no invasive treatments — of the then-experimental drug, which is now known as acalabrutinib (pronounced uh-KA-luh-BROO-tih-nib) and marketed as Calquence®. “Having the opportunity to start this drug felt like a miracle at the time,” David recalls. “I knew as a physician that ibrutinib, the first BTK inhibitor (an earlier drug from which acalabrutinib was derived) changed CLL from an incurable to a chronic disease, but it was not without serious side effects for some patients. I was hopeful this second-generation drug would have fewer side effects, and I was absolutely willing to take the risk.” Now five years since starting the therapy, David remains disease-free. Aside from occasional joint pain and headaches, he jokes that his chief complaint is that the pill is no longer scarlet and gray and is now more reflective of the University of Michigan colors. As an Ohio State fan, he considers this to be a small but bitter pill worth swallowing for cancer control. He maintains a full clinical practice at Central Ohio Primary Care, where he currently serves as president of the group. Acalabrutinib Now Approved by FDA for Certain Cancers Acalabrutinib is a second-generation Bruton’s tyrosine kinase (BTK) inhibitor, a newer class of cancer drugs shown to improve the survival of patients with mantle cell lymphoma in addition to CLL and SCL. The drug works by permanently binding BTK, which is part of a chain of proteins that enables cancer cells to survive and grow. By blocking BTK, the drug halts the growth of the cancer cells, causing the cancer cells to die. Preclinical and clinical data shows that acalabrutinib more selectively blocks the BTK pathway without disrupting other key molecular pathways, 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 the 2019 FDA approval of acalabrutinib were performed by a team of researchers at the OSUCCC – James led by Byrd, who also holds the D. Warren Brown Designated Chair in Leukemia Research at the The Ohio State University College of Medicine. This research included collaborative clinical trials with 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, PhD, 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. To learn about leukemia research and patient care at The James, visit cancer.osu.edu/hematology or call 1-800-293-5066.