Winning ideas include restoring light sensitivity to the blind, precision correction of defective genes, and growing healthy tissue from stem cells for ocular tissue transplants
Ten winning submissions from a pool of nearly 500 entries have been selected by the National Eye Institute (NEI), part of the National Institutes of Health, for its Audacious Goals challenge, a nationwide competition for compelling, one-page ideas to advance vision science. Each winner will receive a $3,000 prize plus travel expenses to attend the NEI Audacious Goals Development Meeting, Feb. 24-26, 2013, at the Bolger Conference Center in Potomac, Md., outside Washington, D.C.
“The Audacious Goals initiative was born out of the NEI strategic planning process, however it is much more than a standard strategic planning exercise,” said Paul A. Sieving, M.D., Ph.D., NEI director. “We are envisioning the future. When we look back 10 to 12 years from now, what do we want to have accomplished? The Audacious Goals initiative will help propel us into that future.”
The NEI Challenge to Identify Audacious Goals in Vision Research and Blindness Rehabilitation is part of a government-wide effort to bring the best ideas and top talent to bear on our nation’s most pressing challenges through the awarding of prize money, among other types of awards. The challenge sought ideas that support the NEI mission to conduct and support research and other programs aimed at reducing the burden of vision disorders and disease worldwide. Prize competition entries were solicited not only from experts in vision research but from anyone in the private, government, and nonprofit sectors, including scientists, engineers, health care providers, inventors, and entrepreneurs, as well as the general public. Entries were de-identified and reviewed by experts on the basis of relevance to the NEI mission and whether the idea is bold, daring, unconventional, or exceptionally innovative; broad in scope; and potentially attainable in about 10 years.
Within a three-month period, 476 entries were submitted from people across the United States, including Puerto Rico. Topics ranged from regenerative medicine and stem cells to neuroscience, genetics, drug development, and artificial vision and prosthetics.
“We didn’t know what to expect when we issued this challenge,” said Richard S. Fisher, Ph.D., director of NEI’s Office of Program Planning and Analysis, which is spearheading the initiative. “Surprisingly, nearly half of the submissions we received came from people who had never been funded by NIH, which demonstrated that we captured the attention of a wide audience throughout the U.S. We invited anyone with an interest in vision research to submit an idea that began with the phrase, ‘It would be fantastic if…’ and in fact, we received many truly audacious ideas.”
During the judging process, more than 80 experts in the vision community helped narrow the field to 81 final candidates. A federal panel consisting of 13 clinicians and scientists then selected the winning ideas.
The winners and their audacious ideas are:
Dennis Clegg, Ph.D., University of California, Santa Barbara, Calif.
Regenerative Therapy for Retinal Disease
To treat degenerative retinal disease with an off-the-shelf tissue graft that could be implanted in the back of the eye to replace cells lost to disease.
Robert Duvoisin, Ph.D., Oregon Health and Science University,
Restoration of Vision by Opto-electronic Stimulation
To restore vision by making nerve cells in the eye sensitive to light so that images captured by a camera can be converted to nerve signals that are sent to the brain.
Yingbin Fu, Ph.D., University of Utah, Salt Lake City, Utah
Precise Gene Editing In Vivo
To permanently correct any disease-associated mutations in a patient through the use of molecules that are specially designed to target mutated DNA sequences and that can be delivered safely and efficiently into the eye.
Steven Pittler, Ph.D., University of Alabama, Birmingham, Ala.
Using Molecular Scissors Genome Editing to Cure Ocular Genetic Disease
To permanently correct gene defects in patients at the site of the mutation using molecules that act like scissors to precisely replace genome errors with the correct DNA sequence.
Rajesh Rao, M.D., Washington University School of Medicine in
St. Louis and The Retina Institute, St. Louis, Mo.
An Audacious Goal: Reprogramming the Retina
To directly reprogram easy-to-isolate skin or blood cells to retinal cells using gene therapy and other techniques to enable repair strategies for degenerative retinal diseases.
Tonia Rex, Ph.D., Vanderbilt University, Nashville, Tenn.
Functional and Structural Neuroregeneration
To restore functional vision in patients who experience loss of axons-the threadlike extensions of a nerve cell that conduct electrical impulses-from the optic nerve as a result of traumatic optic neuropathy or glaucoma by complete axon regeneration.
Julia Richards, Ph.D., University of Michigan, Ann Arbor, Mich.
Fountains of Youth for the Eye
To turn back the aging process in the eye so that ocular diseases like age-related macular degeneration or glaucoma start 10, 20, or 30 years later than they now do.
Jeffrey Stern, M.D., Ph.D., Capital Region Retina, PLLC, Albany, N.Y.
Endogenous Retinal Repair: Releasing our Inner Salamander
To repair the retina by activating stem cells residing within the eye, awakening reparative processes that occur naturally in amphibians and other animals but which lay dormant in human patients.
Russell Van Gelder, M.D., Ph.D., University of Washington, Seattle, Wash.
Reversing Retinal Blindness Using Small Molecules
To restore vision to patients with retinal diseases through the use of a photoswitch, a small molecule that is chemically modified to become active or inactive after exposure to certain wavelengths of light.
Janey Wiggs, M.D., Ph.D., Massachusetts Eye and Ear Infirmary, Harvard Medical School, Boston, Mass.
Vision BioBank - A Network of Ocular Phenotyping Centers Using Genomic and Epidemiologic Data to Promote Personalized Ophthalmology
To create a network of biobanks that collect corresponding phenotype (physical characteristics) and genotype (genetic) data of people with certain eye diseases; the biobanks could be used for a wide range of studies, including the development of sensitive and specific gene tests that could accurately determine a person’s risk for glaucoma, age-related macular degeneration, diabetic retinopathy, and other common complex blinding diseases as well as their likely response to certain therapies.
The winners have been invited to present their ideas at the NEI Audacious Goals Development Meeting, which will include about 200 vision researchers, patient advocates, ophthalmologists, and optometrists from the U.S. and abroad. The selected ideas will be discussed intensively for further expansion, development, and refinement. Following the meeting, NEI staff and members of the National Advisory Eye Council will finalize and publish a set of the most compelling audacious goals for the institute and the broader vision research community to pursue over the next decade.
“The selection of the winning entries marks the true starting point for NEI’s Audacious Goals initiative,” said Dr. Fisher. “We are now at the point where some of the world’s most prominent vision experts can discuss these ideas in-depth, establish a set of audacious goals, and weigh in on how we can realize those goals.”
For more information, visit the Audacious Goals website at http://www.nei.nih.gov/challenge.
NEI leads the federal government’s research on the visual system and eye diseases. NEI supports basic and clinical science programs to develop sight-saving treatments and address special needs of people with vision loss. For more information, visit https://www.nei.nih.gov.
About the National Institutes of Health (NIH): NIH, the nation’s medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov/.
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