CIRM-funded clinical trial takes a combination approach to treating deadly blood cancers

Stained blood smear shows enlarged chronic lymphocytic leukemia cells among normal red blood cells. (UCSD Health)

A diagnosis of cancer often means a tough road ahead, with surgery, chemotherapy and radiation used to try and kill the tumor. Even then, sometimes cancer cells manage to survive and return later, spreading throughout the body. Now researchers at UC San Diego and Oncternal Therapeutics are teaming up with a combination approach they hope will destroy hard-to-kill blood cancers like leukemia.

The combination uses a monoclonal antibody called cirmtuzumab (so called because CIRM funding helped develop it) and a more traditional anti-cancer therapy called ibrutinib. Here’s how it is hoped this approach will work.

Ibrutinib is already approved by the US Food and Drug Administration (FDA) to treat blood cancers such as leukemia and lymphoma. But while it can help, it doesn’t always completely eradicate all the cancer cells. Some cancer stem cells are able to lie dormant during treatment and then start proliferating and spreading the cancer later. That’s why the team are pairing ibrutinib with cirmtuzumab.

In a news release announcing the start of the trial, UCSD’s Dr. Thomas Kipps,  said they hope this one-two punch combination will be more effective.

Thomas Kipps, UCSD

“As a result {of the failure to kill all the cancer cells}, patients typically need to take ibrutinib indefinitely, or until they develop intolerance or resistance to this drug. Cirmtuzumab targets leukemia and cancer stem cells, which are like the seeds of cancer. They are hard to find and difficult to destroy. By blocking signaling pathways that promote neoplastic-cell growth and survival, cirmtuzumab may have complementary activity with ibrutinib in killing leukemia cells, allowing patients potentially to achieve complete remissions that permit patients to stop therapy altogether.”

Because this is an early stage clinical trial, the goal is to first make sure the approach is safe, and second to identify the best dose and treatment schedule for patients.

The researchers hope to recruit 117 patients around the US. Some will get the cirmtuzumab and ibrutinib combination, some will get ibrutinib alone to see if one approach is more effective than the other.

CIRM has a triple investment in this research. Not only did our funding help develop cirmtuzumab, but CIRM is also funding this clinical trial and one of the trial sites is at UCSD, one of the CIRM Alpha Stem Cell Clinics.

CIRM’s Dr. Ingrid Caras says this highlights our commitment to our mission of accelerating stem cell therapies to patients with unmet medical needs.

“Our partnership with UC San Diego and the Alpha Stem Cell Clinics has enabled this trial to more quickly engage potential patient-participants. Being among the first to try new therapies requires courage and CIRM is grateful to the patients who are volunteering to be part of this clinical trial.”


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