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RFU Researchers Help Drive Precision Medicine

RFU's new Innovation and Research Park (IRP) and Helix 51 bioscience incubator, designed for collaboration between academic and industry scientists, will help speed the translation of discoveries aimed at lifting the burden of disease and improving health and wellness.

Our efforts to foster innovation will ultimately, we believe, help lower the cost of care, including the enormous custodial and treatment costs for patient populations afflicted with devastating neurological disease. A paper published in the Annals of Neurology in 2017 estimates the annual U.S. cost of the most common neurological diseases as $789 billion in 2014 dollars. The conditions include Alzheimer's disease, and other dementias, low back pain, stroke, traumatic brain injury, migraine, epilepsy, multiple sclerosis, spinal cord injury and Parkinson's disease.

Grace "Beth" Stutzmann, PhD, who has studied Alzheimer's disease (AD) for more than 16 years, is investigating upstream, searching for early changes in the brain that precede the onset of AD.

"The challenge is to find early mechanisms, because by the time memory lapse occurs, the damage to the brain is often too extensive to reverse," said Dr. Stutzmann, founder of the nationally recognized startup NeuroLucent and director of the Brain Science Institute's Center for Neurodegenerative Diseases and Therapeutics.

Molecular biologist Judith Potashkin, PhD, an investigator in the same center, is making progress in the search for biomarkers of Parkinson's disease (PD), a chronic, debilitating, often misdiagnosed illness that affects 1 to 5 percent of the population over the age of 60. Her research is helping to distinguish atypical Parkinsonian disorders and subcategories of PD patients, which may make the disease easier to diagnose.

Dr. Potashkin and other RFU scientists and partners, including Michelle Hastings, PhD, are helping to drive precision medicine, which can increase the value of care through precise diagnosis, individualized treatment and the identification of optimal populations for the testing of new drugs. Dr. Hastings, director of the Center for Genetic Diseases, is working to develop gene-directed therapies that more precisely target aspects of rare, inherited diseases, including Usher syndrome, Batten disease and cystic fibrosis (CF). Our work on CF, which is caused by a defect in the CFTR gene, consists of a multi-pronged investigation among multiple labs within the center with expertise in the mechanism of disease and approaches for treating disease conditions.

Scientists and innovators are working across RFU's six disease-focused therapeutic centers of excellence and Helix 51 incubator to translate discoveries that can help decrease the cost of R&D and reduce overall healthcare costs driven by hospitalizations, ineffective treatments and the growing need for long-term care.

Posted November 20, 2019
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