Precision Medicine - NFCR Cancer Research Focus

Precision Medicine

Precision Medicine

What is Precision Medicine?

Although cancer research is ultimately about finding a cure for cancer, it also encompasses efforts to make cancer a more manageable disease with less toxic side effects.

Decades of research led us to a new era in the treatment of cancer known as precision medicine or precision oncology. Gone are the days of the “one-size fits all” approach to cancer treatment. Instead, doctors are beginning to administer treatments based on the needs of each individual person, their genetic information, health history and specific conditions.

NFCR Research Highlights

Dr. Daniel A. Haber’s laboratory focuses on understanding the genetic abnormalities of cancer and the research aims to guide targeted drug therapies. His lab is responsible for identifying a specific mutation in the epidermal growth factor receptor (EGFR) in a subset of NSCLC. By linking EGFR mutations to lung cancer, Dr. Haber made it possible to identify patients who will respond well to certain cancer-fighting drugs that block EGFR mutations. In July 2016, the FDA approved the drug Iressa® as a front-line treatment for patients with these specific tumor mutations that Dr. Haber identified. Currently, his lab is defining the genes and proteins that contribute to the dormancy (resting state) of breast cancer cells and the awakening of the cells decades later after the cancer was removed. Ultimately, therapeutic strategies will be developed to target these key genes and proteins with the aim to prevent the distant metastatic recurrence of cancer after surgery.

Dr. Wei Zhang has devoted his entire career to the pursuit of precision oncology – specifically to the key molecular and genomic events that drive the development and progression of cancer. Over the last 20 years, Dr. Zhang and his team have identified multiple novel cancer markers and oncogenic signaling molecules. Currently, Dr. Zhang is studying how genetic expression, amplification and mutations relate to and regulate each other. Using data from next-generation sequencing, Dr. Zhang is identifying growth-promoting genes of a patient’s cancer.

Dr. Cesare Spadoni is developing specific treatments for childhood cancers. Currently there are not many specific drugs for childhood cancer. Medulloblastoma is a childhood brain cancer and Dr. Spadoni’s team is developing a therapy with the ‘2Hit approach’ – a compound or combination of agents that attack two or more therapeutic targets in medulloblastoma cancer cells. The 2Hit approach aims to simultaneously increase effectiveness and reduce potential drug resistance. His team is also developing a treatment for rhabdomyosarcoma, the most common pediatric soft tissue sarcoma that results from the fusion of two genes, PAX3 and FOXO1. The small molecule drug, volasertib, is a potent inhibitor of the enzyme PLK1- resulting in reduced activity and stability of the abnormal fused proteins. The NFCR AIM-HI Translational Research Initiative is supporting the development of volasertib and a Phase 1 clinical trial is planned to begin in 2020 to treat rhabdomyosarcoma with this promising drug combination.

With support from the NFCR AIM-HI Translational Research Initiative, Dr. Ronald DePinho and colleagues have developed a promising new drug that inhibits, STAT3, a major signaling protein in over 50% of cancers. STAT3 controls networks of genes for numerous cellular processes, including 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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