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LOWELL, MA -- A startup company using UMass Lowell facilities and expertise has shown the feasibility of drug delivery to the brain in a large animal model, achieving a significant milestone in the development of their new medical device. The company, Perfusion Technology, says it is the first such demonstration of ultrasound-enabled penetration of the blood-brain-barrier.
“Many therapeutic drugs, such as chemotherapeutics, are unable to penetrate the brain. We have worked for several years to develop a device that safely opens the blood- brain barrier to allow delivery of large molecule therapeutic drugs,” says Ulrich Herken, M.D., Ph.D., founder and chief science officer of Perfusion. “Until now, our research has used rodent models with highly promising results. This new study shows that our device is effective in larger animals, with brains closer in size to humans. Success in a large animal model is a much better predictor of treating patients suffering from brain tumors.” The study is supported by a National Institutes for Health SBIR grant.
The experiment was conducted in collaboration with RxGen, Inc, a Connecticut-based privately held biotechnology company that leverages highly predictive pre-clinical models of human disease to accelerate the drug development process. “The initial results of our joint research using the Perfusion device are very encouraging,” says Steven R. Gullans, Ph.D., RxGen president and CEO.
“This is the kind of breakthrough that is needed to develop treatments of a variety of disorders of the brain and central nervous system,” says Dr. Gullans, former director of the Harvard Medical School-affiliated Brigham and Women’s Hospital Biotechnology Center. “Continued success will allow us to extend the research in several directions, including delivery of large molecule therapeutics and diagnostics to the brain.” Gullans has used advanced technologies to develop biomarkers and potential therapeutics for ALS, Parkinson's disease, and Alzheimer's disease.
FDA approval would likely follow several years of testing in humans, which could begin next year.
The initial focus of the company has been directed to delivery of conventional therapeutic drugs, followed by gene therapy for treatment of diseases of the brain and central nervous system. Perfusion is privately held, and has conducted its research at Massachusetts General Hospital, the Ohio State Cancer Center and with RxGen.
UMass Lowell, a comprehensive university with a national reputation in science, engineering and technology, is committed to educating students for lifelong success in a diverse world and conducting research and outreach activities that sustain the economic, environmental and social health of the region. UML offers its 11,000 students more than 120 degree choices, internships, five-year combined bachelor’s to master’s programs and doctoral studies in the colleges of Arts and Sciences, Engineering and Management, the School of Health and Environment, and the Graduate School of Education.www.uml.edu.
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