FDA OKs first-in-human trial of cell therapy for hard-to-treat AML
Blood cancer treatment developed on University of Colorado campus
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The U.S. Food and Drug Administration (FDA) has given the green light for a first-in-human clinical trial to test a new cell therapy for acute myeloid leukemia (AML), an aggressive form of blood cancer.
The trial is expected to start recruiting participants in June at a site in Colorado. It will test a chimeric antigen receptor (CAR) T-cell therapy developed by scientists at the University of Colorado Anschutz Gates Institute.
This marks the first time that the FDA has cleared in-human testing of a CAR T-cell therapy developed entirely on the CU Anschutz campus, according to a news story from the university.
“Advancing this therapy from an idea to a first-in-human clinical trial reflects the collaborative nature and diverse expertise on campus,” said the therapy’s inventor, M. Eric Kohler, MD, PhD, a professor at CU Anschutz. According to the university, the therapy’s human-made molecular receptor, or CAR, “underwent years of testing, engineering, and optimization in the laboratory before making it to this point” and winning FDA clearance for testing.
“From the basic science departments to the translational teams of Gates Institute, to the clinical expertise in cellular therapy, bringing new therapies forward is what the Gates Institute is built to do,” Kohler said.
T-cells are immune cells that can kill cancer cells. In CAR T-cell therapy, these cancer-killing immune cells are engineered to express a CAR that directs them to attack a specific target. In this case, the CAR targets a protein called CD64, which is often expressed on AML cells.
“This work was built on the seminal findings of Craig Jordan’s laboratory, identifying CD64 as a marker of leukemia cells that escape modern therapies,” Kohler said.
Trial to enroll adults with treatment-resistant, relapsing AML
The upcoming trial will enroll adults with AML that is refractory or relapsed, meaning the cancer has failed to respond or has come back after initially responding to available treatments. The researchers are also hoping to launch a similar trial in children and adolescents later this year. That trial is expected to be held at Children’s Hospital Colorado, per the university.
The adult trial will be led by Mathew Angelos, MD, PhD, an assistant professor at the CU Anschutz School of Medicine. Angelos said that FDA clearance of the trial “reflects tremendous dedication and collaboration across our scientific, clinical and regulatory teams.”
“I’m excited to lead this study and believe it has the potential to meaningfully impact patients with difficult-to-treat myeloid blood cancers, where new treatment strategies are urgently needed,” Angelos said.
[This cell therapy takes] a different approach [to treating] this very aggressive disease, one we hope will ultimately change how myeloid leukemias are treated.
Terry Fry, MD, executive director of the Gates Institute, said this milestone “represents years of work by a team committed to moving cellular therapies from discovery into meaningful clinical impact.”
“This achievement highlights the strength of our growing cell and gene therapy ecosystem and the collaborative expertise required to advance a novel therapy from discovery to clinical testing,” Fry said. “It’s a powerful example of how teams across campus are working together to translate science into meaningful impact for patients.”
According to Fry, the cell therapy takes “a different approach [to treating] this very aggressive disease, one we hope will ultimately change how myeloid leukemias are treated.”
