Multiple myeloma treatment with KU origins enters clinical trial

LAWRENCE — A potential new treatment for multiple myeloma with ties to the University of Kansas has entered a clinical trial.

Biopharmaceutical company Cleave Biosciences this week announced it has begun a Phase 1 clinical trial to evaluate CB-5083, its top drug candidate in patients with relapsed and refractory multiple myeloma. CB-5083 is derived from a compound initially synthesized by KU’s Specialized Chemistry Center.

CB-5083 is an oral inhibitor of p97, a critical enzyme that controls various aspects of protein homeostasis. The clinical trial will evaluate CB-5083 in multiple myeloma patients who have relapsed/refractory or refractory disease after receiving two or more lines of therapy, including an immunomodulatory agent and a proteasome inhibitor.

Cleave expects to enroll up to 60 patients at multiple U.S. cancer centers. More information about the trial is available.

“Patients with multiple myeloma whose disease is resistant to existing therapies have run out of options,” said Frank Schoenen, associate director of the SCC. “As a researcher, it’s exciting and gratifying to know that our lab helped develop the molecule from which this promising new treatment is derived. We look forward to seeing how CB-5083 progresses, and we’ll continue working to create new therapies and cures for patients.”

Multiple myeloma, also known as myeloma, is a hematologic cancer, or cancer of the blood, that develops in the plasma cells in bone marrow. It is the second-most-common blood cancer after non-Hodgkin’s lymphoma. The National Cancer Institute estimates that in the United States approximately 70,000 people are living with multiple myeloma and approximately 24,050 new cases will be diagnosed this year. Worldwide, nearly 230,000 people are living with the disease, and approximately 114,000 new cases are diagnosed annually.

“Small molecules have proven to be extremely important to researchers to explore biological function at the molecular, cellular and in vivo level,” Schoenen said. “These molecules have also been proven to be valuable for treating diseases, and most medicines marketed today are from this class. A key challenge is to identify small molecules effective at selectively modulating a given biological process or disease state. That’s a big part of what we’re doing at the SCC and at KU.”

In addition to KU, Cleave Bioscience’s discovery partners include researchers from the California Institute of Technology and The Scripps Research Institute.

The SCC plays a prominent role in KU’s drug discovery efforts. The center is a member of the NIH’s Molecular Libraries Program, an elite group of five centers nationally whose aim is generate novel small-molecule probes by performing high-throughput screening, secondary screens and medicinal chemistry. The center is directed by Jeff Aubé, University Distinguished Professor in the Department of Medicinal Chemistry.

Additionally, the SCC is a key component of KU’s proposed Drug and Vaccine Development Institute, for which KU will seek financial support during the 2015 legislative session. The proposed institute would focus on the prevention and treatment of diseases and have two sub-institutes: the Institute for Translational Chemical Biology, which would focus on the creation of small-molecule drugs to treat emerging and rare diseases and the commercialization of those discoveries, and the Kansas Vaccine Institute, which would work on early-stage vaccine discoveries for preventing human and animal diseases.