Several recent announcements have discussed new industry-academic collaborations as well as new funding opportunities to help translate scientific discoveries to improve patient care and health.
Harvard and AstraZeneca
AstraZeneca and Harvard University's Wyss Institute for Biologically Inspired Engineering recently announced a partnership "to use its organs-on-chips technology--miniature human organs made of a clear, flexible polymer that contain tiny tubes lined with living human cells--to help improve the way it tests drugs for humans," according to a press release.
"The chips are translucent, which could provide an insight into the inner workings of both animal and human organs," reported FierceBiotechResearch.
"This collaboration with AstraZeneca will help us to validate this approach as a potential alternative to animal testing by carrying out direct comparisons between organ chips containing cells from animals versus humans in organ-mimicking environments," Wyss Founding Director Dr. Don Ingber said in a statement. "If successful, this effort should lead to ways to streamline the drug development process and more effectively predict safety of drugs and chemicals in humans."
Wyss researchers have created chips that mimic the lung, heart and intestine, the article noted. "They are working to build 10 different human organs-on-chips in total and link them together to replicate the physiology of a whole human body."
"AstraZeneca and Wyss scientists will use the technology to develop new animal versions of the chips, which will be used alongside human models to test the safety of investigational drugs," Fierce reported.
"There is a huge need for alternative methods to replace traditional animal models since animal tests do not always accurately predict how humans will react to a new medication. Safety data from animal tests is needed before drug developers can get the green light for testing in humans, so more reliable preclinical models could lead to better drugs for humans. Researchers hope the chips could eventually replace animal testing altogether."
FDA Awards Over 12 Grants for Rare Disease Research
The U.S. Food and Drug Administration (FDA) recently announced the offering of more than $14 million in grants for the development of products for patients with rare diseases, as reported by Drug Store News.
The FDA announced that it had granted 15 grants for orphan drug research. A panel of outside experts with experience in the disease-related fields reviewed applications for the grants, which will be administered through the FDA's Orphan Products Grants Program.
As defined by the agency for drugs, orphan diseases affect fewer than 200,000 people in the United States. For devices, a rare disease occurs so infrequently in the country that there is no reasonable explanation that a medical device for such a disease will be developed without assistance.
According to the National Institutes of Health (NIH), there are about 6,800 rare diseases and conditions, affecting nearly 30 million Americans. Among the grants,
- Alkeus Pharmaceuticals received $167,000 for a drug for Stargardt disease currently in phase-1 development;
- Children's Hospital Medical Center in Cincinnati received $600,000 for a phase-1 study of a drug to treat Fanconi anemia;
- Children's Hospital received $600,000 for a phase-1 study of a drug for Wiskott-Aldrich syndrome; and
- Vanderbilt University received $1.59 million for a phase-2 study of montelukast, which Merck markets as the asthma and allergy drug Singulair, for the treatment of sickle-cell anemia.
"The FDA is committed to fostering and encoring the development of products for rare diseases, most of which have no available or adequate treatments," FDA Office of Orphan Product Development director Gayatri Rao said. "The grants awarded this year support studies in very vulnerable, difficult-to-treat populations who have no available options."
NIH Announces 15 Clinical and Translational Science Awards
The National Institutes of Health (NIH) recently announced more than $79 million in fiscal year 2013 funding to support 15 Institutional Clinical and Translational Science Awards (CTSA). A list of the recipients can be found in the press release.
Led by the National Center for Advancing Translational Sciences (NCATS), the CTSA program catalyzes improvements across the entire spectrum of translational research through efforts to broadly develop, demonstrate and disseminate health interventions. It serves as a connector to engage key partners including other NIH institutes and centers, patient groups, communities, health care providers, industry, and regulatory organizations.
"Science and technology are progressing at an unprecedented pace, and the CTSA program — which represents NIH's largest single investment in clinical research — is helping researchers harness these innovations and deliver improved diagnostics, treatments and cures for disease," said NIH Director Francis S. Collins, M.D., Ph.D.
These latest awards represent NIH's commitment to the CTSA program, which supports a consortium of more than 60 research institutions across the country that is focused on strengthening translational research. Under NCATS' leadership, the program is focused on solving roadblocks common to clinical translational efforts for all diseases.
"The CTSA Consortium is leading national efforts to enhance the efficiency, quality, and safety of translational research, no matter the disease or condition," said NCATS Director Christopher P. Austin, M.D. "This aligns with the NCATS mission to create new technologies and methods that can be applied widely to streamline development and implementation of interventions that improve human health.
NIH Launches New Centers
NIH also recently announced at the end of September the launch of a major initiative to improve how basic science advances and discoveries are translated into commercially viable products that improve patient care and advance public health.
The NIH Centers for Accelerated Innovations (NCAIs), funded by the NIH's National Heart, Lung, and Blood Institute (NHLBI), will target technologies to improve the diagnosis, treatment, management, and prevention of heart, lung, blood, and sleep disorders and diseases.
"The NCAIs will foster a transformational change in the way basic science discoveries move from the laboratory into commercial products," said Dr. Gary H. Gibbons, director of NHLBI. "These centers essentially will offer a one-stop shop to accelerate the translation of early-stage technologies for further development by the private sector and ultimate commercialization. As a result, the public will gain access sooner to new biomedical products that improve human health while also benefiting from the economic growth associated with the creation of new companies and the expansion of existing ones."
NHLBI issued grants totaling $31.5 million to establish three inaugural, multi-institution NCAIs.
- Boston Biomedical Innovation Center:Brigham and Women's Hospital, Boston Children's Hospital, Massachusetts General Hospital, and President and Fellows of Harvard College
- Cleveland Clinic Innovation Accelerator: The Cleveland Clinic Lerner College of Medicine; Case Western Reserve University, Cleveland; Cincinnati Children's Hospital; The Ohio State University, Columbus; and University of Cincinnati
- UC BRAID Center for Accelerated Innovation: University of California, Los Angeles; University of California, Davis; University of California, Irvine; University of California, San Diego; and University of California, San Francisco
NCAIs will provide an integrated, systematic, and comprehensive approach to navigating the translation of early stage biomedical innovations from the research laboratory to commercial development and successful deployment to patients. Each center will be a consortium of academic, government, non-profit, and private sector organizations that will provide funding for feasibility studies; regulatory, legal, and business development expertise; and entrepreneurial training and mentorship. To enhance the scope and impact of the NHLBI investment, each awardee has secured non-federal funding equal to or greater than the NHLBI award.
NCAI awardees will leverage existing federal government resources, including those offered by NIH and by other federal program partners including the U.S. Food and Drug Administration, the United States Patent Office, and the Centers for Medicare & Medicaid Services.
"NIH and NHLBI have long been committed to supporting resources that enable pre-clinical studies," said Dr. Jodi Black, deputy director of the Division of Extramural Research Activities, NHLBI. "This landmark program will help NHLBI derive maximum benefit from its existing research and development investments and ensure that the resulting breakthrough innovations move rapidly and effectively into available products that reduce the health burden of heart, lung, blood, and sleep disorders and diseases."
According to NHLBI's Fact Book for the 2012 fiscal year, in 2009, the estimated economic cost for cardiovascular, lung, and blood diseases was $424 billion—23 percent of the total economic costs of illness, injuries, and death in the United States. In addition, cardiovascular and lung diseases accounted for three of the four leading causes of death in the United States and four of the 10 leading causes of infant death.
NIH Announces 2013 High-Risk High-Reward Research Awards
Finally, NIH announced 78 awards to support innovation in biomedical research to scientists "proposing highly innovative approaches to major contemporary challenges in biomedical research, under the High Risk-High Reward program supported by the National Institutes of Health Common Fund." The press release gives a full list of the various award recipients.
"NIH is excited to continue support of visionary investigators, among all career stages, pursuing science with the potential to transform scientific fields and accelerate the translation of scientific research into improved health, through the Common Fund's High Risk-High Reward Research Program,'' said NIH Director Francis S. Collins, M.D., Ph.D. "This program allows researchers to propose highly creative research projects across a broad range of biomedical research areas, that involve inherent risk, but have the potential to lead to breakthroughs."
NIH Pioneer, New Innovator, Transformative Research, and Early Independence Awards encourage creative thinkers to pursue exciting and innovative ideas about biomedical and behavioral research. The Pioneer Award, in its 10th year, challenges investigators at all career levels to develop highly innovative approaches that could have a powerful impact on a broad area of biomedical or behavioral research.
The New Innovator Award initiative, established in 2007, supports investigators who are within 10 years of their terminal degree or clinical residency, but who have not yet received a Research Project Grant (R01) or equivalent NIH grant, to conduct exceptionally innovative research.
The Transformative Research Awards program, established in 2009, promotes cross-cutting, interdisciplinary approaches and is open to individuals and teams of investigators who propose research that could create or challenge existing models.
The Early Independence Award, with the first awards given in 2011, provides an opportunity for exceptional junior scientists, who have recently received their doctoral degree or finished medical residency, to skip traditional post-doctoral training and move immediately into independent research positions.
In 2013, NIH is awarding 12 Pioneer Awards, 41 New Innovator Awards, 10 Transformative Research Awards, and 15 Early Independence Awards. The total funding, which represents contributions from the NIH Common Fund and multiple NIH institutes and centers, is approximately $123 million.