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Moon Shots Program update

Conquest - Summer 2013

Co-clinical trials speed targeted therapeutics to patients

By Hilary Graham

Carlo Toniatti and Tim Heffernan

Carlo Toniatti, M.D., Ph.D. (left), and 
Tim Heffernan, Ph.D., discuss 
development of the Co-Clinical Trials 
Platform, which will integrate unique 
expertise and cutting-edge resources to 
accelerate the pre-clinical evaluation of 
drugs; this information will later be used 
to inform the design and implementation 
of biomarker-driven clinical trials.
Photo: F. Carter Smith

Providing patients with effective therapies is the goal of researchers and clinicians at 
MD Anderson.

Unfortunately, the approval rate for new oncology drugs is low, limiting patients’ options to existing drugs, many of which have been the standard of care for decades. Only a meager 8% of all oncology drugs that enter clinical trials eventually receive approval from the U.S. Food and Drug Administration, with 65% of novel therapies failing in Phase III trials.

The high failure rate can partially be attributed to the fact that commonly used preclinical oncology models do not faithfully mirror the complexity of human tumors. Therefore, they cannot be relied on to predict a drug’s effectiveness in the clinic. Additionally, the preclinical criteria that govern the progression of a drug from the bench to the clinic have not been sufficiently strict.

The Center for Co-Clinical Trials is a unique preclinical research arm that will evaluate the efficacy and tolerability of single therapeutics or drug combinations in appropriate preclinical models that more closely resemble disease evolution in humans. This research arm is one of several platforms that will contribute to 
MD Anderson’s new Moon Shots Program.

These models will shed insight, at the molecular level, on a drug’s mechanism of action, as well as how cancer cells resist therapy. Molecular markers predicting sensitivity or resistance to therapy will be identified with the ultimate goal of discovering biomarkers that predict which patients will benefit from a specific treatment.

Why MD Anderson? 

MD Anderson provides an ideal setting for this integration between preclinical and clinical studies. It offers access to the largest cancer patient population and some of the best physician scientists in the nation, as well as a wealth of primary tumor samples. In addition, researchers perform cutting-edge functional genomic screens, as well as extensive genomic profiling, which identify genes that provide a survival advantage to cancer cells.

The Center for Co-Clinical Trials is led by Carlo Toniatti, M.D., Ph.D., head of research at the Institute for Applied Cancer Science, and Timothy Heffernan, Ph.D., senior associate director of target discovery at the institute. It will also coordinate some of its activities and expertise with the Moon Shots Program to accelerate the translation of preclinical discoveries and advances in experimental therapeutics into effective cures for cancer patients.

See MD Anderson’s online Newsroom and Cancer Frontline for more information on these and other basic, translational and clinical research findings at MD Anderson.

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