Glioblastoma and Melanoma metastasized to the brain are incredibly deadly brain cancers. However, pharmaceutical company Moleculin Biotech, Inc. hopes the diseases have met their match in a small molecule compound known as WP1066.

The company has initiated a Phase 1 clinical trial of the new first-in-class cancer drug candidate discovered by Professor Waldemar Priebe at The University of Texas MD Anderson Cancer Center. In animal models, WP1066 has shown to both inhibit an important cell signaling protein STAT3 that is involved in cell growth and proliferation, and also stimulate an immune response. 

The first glioblastoma patient has received the initial doses of the compound at MD Anderson Cancer Center, and is said to have tolerated the dose well.

“Treating the first brain tumor patient with WP1066 is the start of a very exciting and encouraging program for doctors treating the worst types of brain cancers. There has been very little progress in recent years toward improved therapies for glioblastoma and other aggressive primary or metastatic brain tumors.  WP1066 has shown extremely promising results based on animal studies where we have seen inhibition of tumor growth and improvements in survival,” said Dr. Sandra Silberman, an oncologist and Moleculin’s CMO.

WP1066, which is built from the chemical backbone of the active ingredient in propolis, a natural product of honey bees, not only directly kills tumor cells, but also has the ability to overcome the tumor’s ability to evade the natural immune response, which would otherwise be working to eliminate the cancerous activity.

It is also the first anticancer agent that appears to consistently inhibit the activated form of STAT3 within cells, a target that, as Dr. Silberman explains, has been long-sought due to its many pathways of promoting tumor growth. “Although STAT3 has long been identified as an important target for treating tumors, for years most efforts have focused on attempts to indirectly inhibit STAT3 from upstream signaling, not from within the cancer cell itself. WP1066 appears to be unique in its ability in vitro and in animal models to consistently and directly inhibit the activated form of STAT3 and produce significant anticancer effects, including tumor growth inhibition and increased life span of treated animals.”

Walter Klemp, chairman and CEO at Moleculin added, “Although the industry has been struggling to find a way to target STAT3, we at Moleculin believe that most of these efforts have been mechanistically misguided and ended in failure because their approach would ultimately be ineffective at adequately blocking the activation of STAT3 and lack the necessary drug-like properties to succeed. The opportunity to test a unique STAT3 therapy in these patients is significant in supporting Moleculin’s mission to provide benefit for those who need new and better treatments.”

(Source: Moleculin Biotech, Inc.)