Rutgers Scientists Develop Therapy for Fatal Childhood Disease

The R&D Council of New Jersey has honored two Rutgers University professors with its Edison Patent Award for discovering the cause of a rare but devastating and fatal childhood disease and developing a potential drug therapy now being tested in a clinical evaluation.

Peter Lobel and David Sleat, who conduct research at the Center for Advanced Biotechnology and Medicine of Rutgers Biomedical and Health Sciences, received the council’s Edison Award November 12 at its annual awards dinner.

The two scientists developed the first-ever effective method to deliver therapy for a progressive childhood illness known as Batten Disease, or Late Infantile Neuronal Ceroid Lipofuscinoses (LINCL). Batten Disease is a rare but devastating disorder caused by mutations in the CLN2 gene and protein (Tripeptidyl peptidase I, TPP1). Patients develop symptoms between ages 2 and 4 and die by 8 to 12 years. There is no effective treatment for this disease; symptoms are managed through the use of anti-epileptic drugs and physical, speech, and occupational therapies.

“The unique and effective approach that Professors Lobel and Sleat have taken in treating Batten Disease is the kind of innovation that is needed to improve the lives of patients facing diseases without effective therapies,” said Christopher J. Molloy, Rutgers senior vice president for research and economic development. “The development of new therapies for treating disease offers further potential to open new doors for even more medical discoveries and to eventually benefit more patients worldwide.”

Lobel and Sleat have developed a method for treating LINCL by administering CLN2 protein in an amount effective to reduce symptoms. This technology was exclusively licensed to BioMarin, a biopharmaceutical company that develops and commercializes treatments for serious diseases. BioMarin is currently conducting a clinical evaluation of a drug based on the discovery by Lobel and Sleat, which is a novel approach to developing enzyme replacement therapy for LINCL by concentrating on the TPP1 enzyme.

The R&D Council’s 36th Patent Award Ceremony and Reception was held place at the Liberty Science Center in Jersey City. The Liberty Science Center is home to the nation’s largest IMAX Theater, where a short original film paid tribute to the honored individuals and the patents of the inventors.  The segment with Lobel and Sleat is posted here.

Lobel is a professor of biochemistry and molecular biology and Sleat is an associate professor biochemistry and molecular biology in Rutgers’ Robert Wood Johnson Medical School.

Prof. Peter Lobel and Haiyan Zheng, assistant director of the Biological Mass Spectrometry Facility at Rutgers. (left)

Prof. David Sleat (right)

Rutgers Center for Advanced Biotechnology and Medicine (CABM) is a multi-disciplinary center with a mission to develop the next generation of researchers and to make fundamental discoveries in biomedical research and translate these to improve human health.  Research is focused in molecular medicine, developing and applying new technologies for therapeutic target discovery, characterization and validation.