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New Exploratory Grant Will Help Scripps Florida Scientists Advance Treatment Development

JUPITER, FL – August 24, 2017 – Scientists from the Florida campus of The Scripps Research Institute have been awarded nearly $1 million from the National Institute of Neurological Disorders and Stroke, part of the National Insti­tutes of Health, to develop a genet­ically engineered animal model of a debilitating inherited disease that could accelerate the development of potential treatments.

Joseph Kissil, a TSRI associate professor in the Department of Molecular Medicine, is the principal investigator for the three-year grant. This is a phased award; if certain benchmarks are met, the grant is extended for a third year.

Kissil has been studying Neurofibromatosis Type 2 (NF2) for a number of years. The disease is caused by mutations in the anti-tumor gene NF2, which leads to tumors of the auditory nerve that connects the inner ear to the brain and causes severe hearing loss and impaired balance and walking. A majority of patients develop additional tumors throughout the nervous system, which can lead to fluid buildup in the brain and seizures.

While knowledge of the molecular mechanisms underlying the disease has improved significantly over the past two decades, there are still no effective treatments. Current options are non-curative and include surgery, radiation therapy, as well as temporary interventions for symptom control.

“Clearly, there is an urgent need to develop better options for these patients,” Kissil said. “One of the main obstacles is the lack of animal models that can be used in pre-clinical testing of drug candidates. This grant will help us develop a genetically engineered animal model of the disease that accurately portrays the biology of tumor development in patients – and that can be used to evaluate drug candidate efficacy.”

In 2013, Kissil and his colleagues identified a new drug candidate to treat NF2. The compound —known as FRAX97—slowed the proliferation and progression of tumor cells in some preliminary animal models of the disease. Originally developed for neurodegenerative disease, the compound targets a protein family known as p21-activated kinases or PAKs. These kinases (enzymes that add a phosphate group to other proteins and change their function) play a critical role in the development of NF2.

“While the models that we used then offered a good approximation, they were just that – an approximation,” Kissil said. “There’s still a great need for models that more closely reflect what happens in patients and that the new model we’re working on does just that.”

The number of the grant is1R21NS099417-01A1

About The Scripps Research Institute

The Scripps Research Institute (TSRI) is one of the world's largest independent, not-for-profit organizations focusing on research in the biomedical sciences. TSRI is internationally recognized for its contributions to science and health, including its role in laying the foundation for new treatments for cancer, rheumatoid arthritis, hemophilia, and other diseases. An institution that evolved from the Scripps Metabolic Clinic founded by philanthropist Ellen Browning Scripps in 1924, the institute now employs more than 2,500 people on its campuses in La Jolla, CA, and Jupiter, FL, where its renowned scientists—including two Nobel laureates and 20 members of the National Academies of Science, Engineering or Medicine—work toward their next discoveries. The institute's graduate program, which awards PhD degrees in biology and chemistry, ranks among the top ten of its kind in the nation. In October 2016, TSRI announced a strategic affiliation with the California Institute for Biomedical Research (Calibr), representing a renewed commitment to the discovery and development of new medicines to address unmet medical needs. For more information, see

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Joseph Kissil, a TSRI associate professor in the Department of Molecular Medicine. (High-res image)