Goal to speed drug discovery and development
By Scott Merville
MD Anderson is building an industrial-strength bridge across the treacherous landscape that lies between basic science discovery and approval of new drugs for cancer patients by creating its new Institute for Applied Cancer Science (IACS).
Only 5-10% of potential cancer drugs survive the journey through the present drug development ecosystem, with many failing late in the process after years of work and millions of dollars spent.
“That’s a staggeringly unacceptable high rate of failure,” says Ronald DePinho, M.D., president of MD Anderson.
“Recent transformative scientific and technological advances provide the tools for improving drug discovery and development, but applying them requires a new organizational model,” he says. “Our institute will rely on industry-seasoned scientists to systematically secure the knowledge needed to fully understand key drug targets and develop a clear clinical path for new therapies.”
The IACS, established last year after DePinho was appointed as MD Anderson’s fourth president, is much like a biotechnology company embedded in an academic medical center.
By the end of 2012, IACS director Giulio Draetta, M.D., Ph.D., expects to have 70 senior scientists on board, “people from pharmaceutical research and development who know how to make drugs.”
“We’re building a professional drug development infrastructure that can tap into MD Anderson’s strengths: outstanding clinical care, a strong clinical trial infrastructure, great basic science and a fundamental commitment to translational research,” says Draetta. “We can discover drugs and bring candidates to clinical trial, all within a single institution.”
In traditional drug development, pharmaceutical companies take basic discoveries from academia through the preclinical developmental steps required to advance a new drug to phase I clinical trial. Pharmaceutical companies are abandoning this model, Draetta notes, while academic institutions, MD Anderson a leader among them, are taking on more translational research.
Swift, thorough process to kill projects
Fully bridging that gap requires a goal-oriented culture and scientists with expertise in genomics, computational biology, deep cancer biology, model systems and applied sciences such as medicinal chemistry.
"The institute will define and rapidly conduct key validation and drug discovery studies that allow for rapid decision making, regardless of the complexity involved,” says Lynda Chin, M.D., IACS scientific director. “It’s a design to weed out weak ideas early in the process. That differentiates us from other research labs and, for that matter, from my own academic lab.”
Culling projects is critical because more than half of candidate drugs that make it to phase III clinical trials fail at that final step.
Chin and Draetta envision the institute generating a robust drug development pipeline by characterizing and evaluating candidate drug ideas from MD Anderson scientists, as well as other academic institutions and private companies.
The institute’s goal-oriented industrial approach, combined with MD Anderson’s academic expertise, unique infrastructure and sheer critical mass, will provide a unique advantage over competitors in industry and academia, they say. It’s this integration of discovery and applied science that will enable MD Anderson to move swiftly from research discoveries to drug development to clinical application and ultimately make a real difference in the lives of patients.
The IACS goal is to develop at least one clinical drug candidate by the end of Fiscal Year 2013 and advance it into a phase I clinical trial during FY2014, with more to follow. Its present capacity allows it to evaluate three projects at a time.
Draetta and Chin developed this model with DePinho at the Belfer Institute for Applied Cancer Science in Boston, which DePinho led before coming to MD Anderson. The first 25 scientists hired at the IACS came from the Belfer Institute.
A self-sustaining financial model
MD Anderson has committed a maximum of $15 million per year for five years to the institute, as well as space, technology and support services.
The IACS is expected to achieve significant financial performance metrics, such as raising $2.5 million from philanthropy during its first year, and over time attract additional funds from corporate alliances, research grants, philanthropy and revenue generated by drugs and diagnostics it develops.
“As we generate compounds, we’ll have the opportunity to monetize those assets with the ultimate goal of becoming self-sustaining,” says Eric Devroe, Ph.D., executive director, strategic alliances.
Chin and Draetta: together again in new venture
Giulio Draetta, M.D., Ph.D., held appointments at Pharmacia and Merck as vice president and worldwide head of oncology drug discovery before becoming deputy director and chief research development officer at the Belfer Institute. He also is co-founder of Karyopharm Therapeutics, a biotech cancer therapeutics company.
In academia, Draetta studied processes of cell division and DNA damage identification and repair during appointments at Cold Spring Harbor Laboratory in New York, the European Molecular Biology Laboratory in Heidelberg, Germany, and the European Institute of Oncology in Milan, Italy.
As scientific director at Belfer, Lynda Chin, M.D., drove 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 of Research Excellence.
Chin serves on the central committee of The Cancer Genome Atlas (TCGA) of the National Institutes of Health, directs a Genome Analysis Data Center and chairs the TCGA glioblastoma and melanoma working groups. She co-founded AVEO Pharmaceuticals, a public cancer biotechnology company, and Metamark Genetics Inc., a cancer diagnostic company.
At MD Anderson, Chin is professor and chair of the new Department of Genomic Medicine. Draetta is a professor in the department.
Experienced leadership team
Among experts moving to the IACS from industry and the Belfer Institute:
• Philip Jones, Ph.D., head of drug discovery, led several of Merck’s oncology drug discovery programs. He took two Merck ideas from program initiation to candidate drug in fewer than 16 months each.
• Carlo Toniatti, M.D., Ph.D., head of research, has more than 20 years of experience in basic research, drug discovery and clinical development for biotech and pharmaceutical companies. He’s led preclinical drug discovery programs from target identification and validation to phase I and II clinical trials.
• Eric Devroe, Ph.D., executive director, strategic alliances, previously was vice president of business and strategy development at Metamark Genetics, where he completed 20 license and partnership agreements, including a major alliance with Janssen Biotech Inc.