Bellicum Pharmaceuticals Inc. said its BPX-501 drug showed lower cancer recurrence rates in an ongoing study among children with acute myeloid leukemia and primary immunodeficiencies.
Acute myeloid leukemia is a type of cancer affecting the blood and bone marrow, while primary immunodeficiencies, or PIDs, are a group of rare, chronic disorders where the immune system is missing or not functioning properly.
The study was conducted in 38 children with acute myeloid leukemia and 59 children with PIDs who received BPX-501 after a haploidentical stem cell transplantation, a transplant procedure that serves as an option for patients who lack matched donors for a certain tissue type.
At one year, 97.3% of the leukemia patients treated with the drug were still alive, while 91.5% survived without any cancer symptoms.
For the PIDs patient group, 88.1% of the children were alive without any disease symptoms, with 88.6% alive at one year.
Houston-based Bellicum will cooperate with investigators and the U.S. Food and Drug Administration for the development of a protocol to study the drug in children using data from the ongoing trial.
This study is expected to start at the end of 2018, subject to regulatory approvals.
