New research institute at The University of Texas MD Anderson Cancer Center

Organization focuses academic, private sector strengths on process in dire need of improvement

Academic and government leaders announced today the establishment of a major new research institute at The University of Texas MD Anderson Cancer Center that will blend the best attributes of academic and industrial research to identify and validate new cancer targets, convert such scientific knowledge into new cancer drugs, and advance these novel agents into innovative clinical trials.

"The Institute for Applied Cancer Science will exploit the enormous opportunities provided by recent truly transformative scientific and technological advances to improve the appallingly low rate of success in the nation's current cancer drug development system," Ronald DePinho, M.D., president of MD Anderson, said.

"Only 5-10 percent of potential cancer drugs make it from initial discovery all the way to patients as approved treatments. And more than half of those fail in phase III clinical trials, the final step of development. That's costly not just economically but costly to patients who are subjected to largely ineffective treatments," he said. "Improving this unacceptable performance requires that we hit the reset button and develop a new organizational model that systematically secures the knowledge needed to fully understand key targets and develop a clear clinical path for new therapies."

The institute is designed to convert basic discoveries into effective new drugs and complementary diagnostics for cancer patients via multidisciplinary collaboration using a full range of state-of-the-art technologies, said Raymond DuBois, M.D., Ph.D., MD Anderson's executive vice president and provost. It will enhance and align with many other programs at MD Anderson, spanning basic, translational and clinical research.

DePinho and institute leaders joined Gov. Rick Perry and University of Texas System Board of Regents Chairman Gene Powell to announce creation of the institute today.

Cultural divide is 'valley of death' for drug development

Researchers in academic medicine excel in identifying molecular changes that drive cancer development and growth, leading to the identification of potential drug targets, DuBois noted. "In recent years, academic researchers have moved into translational research, those final steps between the lab and first-in-human Phase I clinical trials that were once largely the domain of pharmaceutical companies."

As pharmaceutical companies focus their resources more on later stage clinical trials and commercialization, and entrepreneurial biotech companies struggle financially during slow economic times, that shift is important.

Yet, the cultural divide between academic research and the intense, goal-oriented focus of biotechnology companies constitutes what the institute's new leaders call "the valley of death" in drug development.

"Efficient conversion of discoveries into effective medicines will require seamless integration of not only discovery and applied science, but also the exploratory and goal-oriented cultures in academia and industry," DuBois said. "Our institute leaders are highly accomplished in both realms and have outstanding experience in bridging the gap between them."

Draetta, Chin lead new institute

Giulio Draetta, M.D., Ph.D., director, and Lynda Chin, M.D., scientific director, are former leaders of the Belfer Institute for Applied Cancer Science in Boston. Chin also chairs MD Anderson's new Department of Genomic Medicine, where Draetta is a professor.

Before his leadership role as deputy director and chief research development officer at Belfer, Draetta held appointments at Pharmacia and Merck as vice president and worldwide head of oncology drug discovery. As an entrepreneur, he is co-founder of Karyopharm Therapeutics, a biotechnology company focused on novel therapies for cancer.

In academia, Draetta was a leading researcher in the processes of cell division and DNA damage identification and repair during appointments at Cold Spring Harbor Laboratory, the European Molecular Biology Laboratory in Heidelberg, Germany, and the European Institute of Oncology. As deputy director at Belfer, he also was a Presidential Scholar at Dana-Farber Cancer Institute.

As scientific director at Belfer, Chin drove the scientific foundation for collaborations with Merck and Sanofi-Aventis. She was professor in the Harvard Medical School Department of Dermatology and the Dana-Farber Department of Medical Oncology, where she led the Melanoma program and the Harvard Skin Cancer Specialized Program in Research Excellence.

Chin also is a leader in The Cancer Genome Atlas (TCGA) of the National Institutes of Health, serving on the central committee, directing a Genome Analysis Data Center and chairing the TCGA glioblastoma and melanoma disease working groups. In 2002, she also co-founded AVEO Pharmaceuticals, a public cancer biotechnology company, and most recently founded Metamark Genetics, a cancer diagnostic company.

Goals: Biomarker-driven clinical trials, external funding

Draetta and Chin will be joined by an experienced leadership team with proven track records in industry and at the Belfer Institute to establish a robust science-driven drug development pipeline at MD Anderson. Together, the team has set ambitious goals of developing novel drug candidates for biomarker-driven clinical trials in five years, internally as well as jointly with corporate partners.

MD Anderson will commit a maximum of $15 million per year for five years to the institute, as well as space, cutting-edge technology and support services. In return, DuBois said, the institute will be expected to achieve significant financial performance metrics, such as raising $2.5 million in external funding during its first year, with a total goal of raising at least $42 million by the fifth year. Successful external fundraising will reduce MD Anderson's financial commitment.

The Institute for Applied Cancer Science will be housed in South Campus Research Buildings 3 and 4, located on East Road.

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