Accelerating the Development of New Drugs and Diagnostics Maximizing the Impact of the Cures Acceleration Network: Workshop Summary (2012) / Chapter Skim
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2 Approaches to Accelerating Translational Science
2 Approaches to Accelerating Translational Science
Pages 15-28
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From page 15... ...
· Effectiveness of and communication among the project management team is a key element of success. · A consensus-based traditional funding review process could undermine sup port for needed breakthrough projects.
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From page 16... ...
When lung function drops below 40 percent, death is likely from one of a variety of causes. About 15 years ago, Vertex Pharmaceuticals and the Cystic Fibrosis Foundation began working together to lower lung decline to 1 percent per year, said Joshua Boger, who founded Vertex Pharmaceuticals in 1989 and still serves on its board.
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From page 17... ...
"This is a success story in large part due to unique cooperation between Vertex and the Cystic Fibrosis Foundation, which has become a model in the field," said Boger. Partnerships and Approaches Until the late 1990s, the Cystic Fibrosis Foundation supported "wonderful science," according to Boger, but the foundation's leadership realized it was not directly helping patients.
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From page 18... ...
The successful treatment of some cases of cystic fibrosis, as described above, or the soon-to-be-accomplished victory over hepatitis C virus, demonstrates what can be done. But failures in such areas as Alzheimer's disease, heart disease, or the development of an HIV vaccine illustrate both the scientific and business challenges of drug discovery.
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From page 19... ...
First, CAN could support and catalyze research to develop and validate a new generation of animal models created to exhibit clinically relevant phenotypes. This likely will require multiple genetic manipulations that are carefully selected to bring the models into more faithful representation of human disease.
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From page 20... ...
A next generation of cellular reprogramming techniques is now emerging by which mouse or human fibroblast or white blood cells can be converted directly into a variety of cell types to model human diseases in culture. In particular, reprogrammed human neurons can form multi cellular networks and recapitulate important features of neurodegenerative diseases.
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From page 21... ...
Bradner described a very rare and very lethal cancer called NUT midline carcinoma, which affects approximately 100 people per year in the United States. A molecular pathologist at Brigham and Women's Hospital named Christopher French cultivated cells from patients who have the disease, and Bradner and his colleagues used these cells to test 4 This section, including subsection, is based on the presentation by James Bradner, Instructor in Medicine, Dana-Farber Cancer Institute, and Assistant Professor, Harvard Medical School.
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From page 22... ...
"They're under no special obligation to call us back, but they almost always do, to share the exciting findings of their research." In multiple myeloma, JQ1 downregulates what Bradner called "the central horseman of the cancer apocalypse," a gene called MYC that triggers growth. Mice with multiple myeloma driven by MYC had a complete response.
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From page 23... ...
The company has put together a multistage, multi-institute, public private translational research program centered on the compound Aes-103. The members of the collaboration include AesRx, the National Heart, Lung, and Blood Institute, the Therapeutics for Rare and Neglected Diseases program -- which used to be part of the National Human Genome Research Institute and is now part of NCATS -- the NIH Clinical Center, and the NIH Clinical Pharmacy.
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From page 24... ...
"The ecosystem has changed so dramatically that it's become doubtful whether companies like us can continue to provide the role that we have traditionally had in the drug development supply chain." Venture capital funds are dramatically reducing their commitment to early-stage biotechnology companies for several reasons, Seiler said. First, the suppliers of venture capital increasingly have required more mature programs, which has put early-stage biotechnology projects beyond venture capital's investment horizons.
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From page 25... ...
Staff members also mine the society's research grant portfolio for projects that have moved out of 6 This section, including subsection, is based on the presentation by Louis DeGennaro, Exec utive Vice President and Chief Mission Officer, the Leukemia & Lymphoma Society (LLS)
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From page 26... ...
DeGennaro concluded by briefly mentioning a novel partnership among the society, NCATS, and the University of Kansas to repurpose existing drugs to treat hematological malignancies. A memorandum of understanding set objectives and responsibilities, with commercialization a prominent objective.
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From page 27... ...
For example, most university researchers have little understanding of what biotechnology companies actually do and of the levels of expertise, creativity, and imagination that are required. "There's a tendency to think of the latter stages of the development pathways as turning the crank on routine, uninteresting work, and that's a misperception." He also said that academic researchers have a tendency to overvalue what they have, "and therefore they don't come to the table with realistic views." DeGennaro emphasized the importance of better training for clinical investigators about the regulatory process.
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From page 28... ...
Williams sought to distinguish the demands of the science from the demands of regulation. He said that although animal models are not required for drug development, each situation has different requirements.
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