In August, clinical research teams activated a phase 4 study, led locally by Nirav Shah, MD, Professor of Hematology and Oncology and Medical Director of the Cancer Center Cell Therapy Shared Resource (CTSR). This study follows patients who previously participated in the original trial of pirtobrutinib, a targeted therapy for chronic lymphocytic leukemia, small lymphocytic lymphoma, and other non-Hodgkin lymphomas. The goal is to track long-term safety and continued benefit for a drug that patients often take daily for years.
“It’s important to understand the long-term side effects of drugs that are taken indefinitely until progression. This study gives us a critical opportunity to monitor how patients do over time and ensure that pirtobrutinib remains both safe and effective,” said Dr. Shah.
Pirtobrutinib is a BTK inhibitor, a type of drug that blocks a protein cancer cells use to grow and survive. Unlike older BTK inhibitors, which bind permanently to the protein, pirtobrutinib is the first non-covalent BTK inhibitor approved by the FDA. This difference allows it to keep working even in patients whose disease has become resistant to other BTK inhibitors. Because it targets cancer cells more precisely, it also reduces the risk of side effects that can occur when healthy cells are affected.
“This trial is an extension study allowing patients who were receiving pirtobrutinib on prior protocols to continue the drug for as long as it is providing benefit to them,” Dr. Shah explained. “That continuity is incredibly important for patients who may have few remaining treatment options.”
The study also reflects MCW’s leadership in advancing new treatments for blood cancers. The Cancer Center was a leading site in the original pirtobrutinib studies that led to its FDA approval, underscoring its ability to collaborate with pharmaceutical partners and help bring new therapies into practice.
Researchers are also exploring pirtobrutinib in combination with CAR T-cell therapy, a treatment that engineers a patient’s own immune cells to recognize and attack cancer. “We have an investigator-initiated trial that combines pirtobrutinib with our in-house dual-targeted CAR20.19 T cells to see if a combined treatment can result in better outcomes,” said Dr. Shah. “The idea is that pirtobrutinib may prime the immune system to create a stronger CAR T-cell product and then help prevent relapse when used as maintenance.”
Dr. Shah noted that these efforts will gain even more momentum with the opening of the new CTSR in the Center for Cancer Discovery. “It’s exciting to see the new building up and running, giving our physician-scientists the space they need to advance the field and foster collaborations,” he said.
As he celebrates a decade at MCW, Dr. Shah is energized by the pace of progress. “I’m excited to be part of this growing and innovative program. Trials like this not only extend access to promising therapies but also position us to keep driving forward in blood cancer research,” he added.