Areas of Focus | Targeted Cancer Therapies - NFCR

Targeted Cancer Therapies

Targeted Cancer Therapies

What are Targeted Cancer Therapies?

Targeted cancer therapies use drugs to more precisely identify and attack cancer cells, based on a person’s genes, as compared to traditional cancer treatments. As such, targeted cancer therapies are sometimes called “molecularly targeted drugs” or “molecularly targeted therapies” and allow for a more precision medicine approach.

In addition to being molecularly-focused, targeted therapies are often cytostatic (which means they block tumor cell proliferation), whereas standard chemotherapy agents are cytotoxic (which means they kill tumor cells). Therefore, many targeted drugs go after the mechanisms that make cancer cells different than normal cells and leave the healthy cells alone.

NFCR Research Highlights

Dr. Daniel Von Hoff is developing monoclonal antibodies (Mabs) that target a key protein in the fibrotic or ‘stromal’ cells that surrounds many types of tumors. When the protein is inactivated when bound by the Mab, it can no longer signal to the tumor cells to metastasize from the primary tumor. With funds from the NFCR AIM-HI Translational Research Initiative, the best monoclonal antibodies are in final pre-clinical stages and the most promising one may soon enter clinical trials to treat colorectal cancer patients who need a new effective treatment to save their lives.

The immunotherapy called ‘checkpoint inhibitors’ is revolutionizing treatment for some cancers but it is not effective in the aggressive brain cancer, GBM. Dr. Rakesh Jain is using treatments to target factors in abnormal blood vessels combined with the checkpoint inhibitors to successfully stop cancer growth in complex models of GBM. The combination approach has high potential to improve treatment outcomes and increase survival in GBM patients.

Dr. Cesare Spadoni’s team is developing treatments for childhood cancers. In the brain cancer, medulloblastoma, research is identifying the most promising drug or a drug combination that inhibits two targets in pre-clinical models of medulloblastoma. The scientists have also determined that the small molecule, volasertib, serves as an effective treatment for the pediatric soft tissue sarcoma, rhabdomyosarcoma. Volasertib targets and inhibits an enzyme resulting in reduced expression and activity of two abnormal fused genes responsible for causing rhabdomyosarcoma. With NFCR support, Dr. Spadoni’s efforts have led to this promising therapeutic now being commercialized through the AIM-HI Translational Research Initiative. To learn more, click here.

With support from the NFCR AIM-HI Translational Research Initiative, Dr. Ronald DePinho and colleagues have developed a promising new drug that targets one of the major signaling proteins in over 50% of cancers. The protein, STAT3, controls networks of genes for numerous cellular processes, such as proliferation, survival, angiogenesis, metastasis, invasion, and immune escape. The drug is now in Phase 1 clinical trials to treat various types of advanced cancers.

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