University of West Virginia clinical trial offers hope for a rare and deadly blood cancer

CD19-positive mixed phenotype acute leukemia (MPAL) is one of the rarest forms of blood cancer. It’s also one of the deadliest; the median survival rate is usually months. And there has been no FDA-approved treatment — until now.

Ashkan Emadi, M.D., Ph.D., and the cancer team at the University of West Virginia Cancer Institute have completed a clinical trial of a subcutaneous formulation delivery for blinatumomab. Blinatumomab is an immunotherapy usually used to treat acute lymphoblastic leukemia, but it is a long, involved process. One treatment lasts 28 days and involves the patient being hooked to a pump for 24 hours a day. Emadi and his team have developed a way to deliver the drug in a shot.

While only one patient has received the treatment so far, after the first dose on Jan. 16, “the patient achieved complete remission with full hematologic recovery, including transfusion independence and the absence of the Philadelphia chromosome, an abnormal chromosome associated with certain types of leukemia, which is an outcome that is both unprecedented and highly encouraging,” Emadi said. As of Feb. 17, the patient was at home and recovering “with outstanding performance status.”

Phase II of the trial will involve enrolling up to 75 patients across three clinical cohorts across approximately 15 sites in North America. The cancer is so rare that enrollment is projected to be one to three patients per site per year, with applicants being accepted from across the world. If successful, the treatment could revolutionize not only treatment for MPAL, but also for delivering other immunotherapies in an easier, less invasive way that saves patients time in the hospital.

“Patients with MPAL have faced an extraordinary therapeutic void,” Dr. Emadi said. “This first-in-world treatment offers real hope that we can improve both outcomes and quality of life for a patient population that has been historically overlooked.”