Congratulations to Robert Steiner, MD, clinical professor, Genetics and Metabolism, who will serve as a Principal Investigator on a collaborative Small Business Innovation Research grant for the study, “Development of N-tert-(Butyl)hydroxylamine (NtBuHA) as a therapeutic agent for treating infantile neuronal ceroid lipofuscinosis (INCL).” Steiner has been diagnosing and caring for patients with INCL, a form of Batten Disease, for more than a decade and involved in research to develop treatments for the same period. The project aims to develop the first therapeutic option for reducing the negative effects of lost enzymatic activity, the underlying cause of INCL. There is a significant unmet medical need for a therapy to treat patients with INCL, a devastating neurologic disorder that is often diagnosed in infancy and is fatal by late childhood. A therapeutic would change the clinical practice paradigm from palliative and supportive care toward a curative strategy.
The two-year $1.44 million project is funded by a Phase II R44 grant from the National Institutes of Health/National Institute of Neurological Disorders and Stroke. Steiner’s Co-PIs include researchers at Circumvent Pharmaceuticals in Oregon, Charles River Laboratories in Massachusetts, and J-STAR Research in New Jersey.