Early AML trial finds experimental therapy was generally well tolerated
CR-001 was tested in 11 patients; six of 10 evaluable had stable disease
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The experimental anticancer therapy CR-001 was generally well tolerated in a first-in-human clinical trial that tested several doses in 11 people with acute myeloid leukemia (AML), a type of blood cancer.
Following treatment, six of the 10 patients evaluable for effectiveness had stable disease, meaning their cancer did not worsen. The researchers said CR-001 may have potential as an AML maintenance therapy, although the preliminary findings need to be evaluated in further studies.
“Based on its superior safety profile, CR-001 may have a potential as AML maintenance therapy,” scientists wrote in the study, “Phase I, open-label, multi-center dose-finding and expansion study to investigate the safety, tolerability, and preliminary efficacy of CR-001 (5-FU-miR-15a) in patients with acute myeloid leukemia,” which was published in Leukemia. The work was funded in part by Curamir Therapeutics, the company developing CR-001.
How CR-001 is designed to work
In AML, cancer cells produce various proteins that help drive abnormal cell growth. CR-001 contains a modified version of miR-15a, a naturally occurring microRNA, or short piece of genetic material, that helps regulate several proteins involved in AML. The modification is designed to make miR-15a more stable and help it enter cells, where it may reduce levels of proteins that support cancer growth and treatment resistance.
Researchers conducted a first-in-human clinical trial to evaluate the therapy’s safety and tolerability in people with AML. The study enrolled 11 participants, most of whom had AML that had returned or failed to respond after multiple prior lines of therapy. Participants received CR-001 by infusion once weekly for four weeks at various doses. Those whose disease remained stable after the first four infusions could receive up to four additional weekly infusions.
Results showed CR-001 was generally well tolerated, and no dose-limiting toxicities were observed. Infusion-related reactions occurred in six patients and included shivering and a rapid heart rate. The reactions lasted about 30 minutes and resolved completely. The researchers noted that CR-001 was generally administered in an outpatient setting, without requiring an overnight hospital stay.
“In general, CR-001 was well-tolerated and administered on an outpatient basis,” the scientists wrote.
In addition to stable disease in most evaluable patients, preliminary analyses suggested that CR-001 may have lowered levels of some proteins that help AML cells survive, grow or resist treatment, providing early evidence that the therapy may be acting on its intended targets.
“CR-001 was well-tolerated and showed signals of biological activity as evidenced by single-cell protein profiling and disease stabilization,” the researchers concluded.
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