Scientific Advisory Board

Allan E. Rubenstein, MD, Chairman SAB

Dr. Rubenstein has over 20 years of experience in management of biotechnology and medical equipment companies. Dr. Rubenstein is presently Clinical Professor of Neurology and Pediatrics at the NYU Langone Medical Center. He is the founder and the former CEO of NexGenix Pharmaceuticals Inc., a biotechnology company focused on drug development for neurofibromatosis and related sporadic tumors. He also serves as the Vice-Chairman and Lead Director of the Cooper Companies (COO, NYSE), a medical products company. Dr. Rubenstein was one of the founding members of the Children's Tumor Foundation and also founded the Mount Sinai Neurofibromatosis Research and Treatment Center. He has been recognized in Who's Who in the East and Who's Who in Science and Technology. He has provided counsel to numerous research and governmental agencies, including the NINDS, the Neurogenetics Study Section of the National Cancer Institute, and the Department of Defense NF Research Program. He is a reviewer for several scientific publications and was a Contributing Editor for the Mount Sinai Journal of Medicine for many years. Dr. Rubenstein graduated from Cornell University. His medical education began at the University of Rochester, School of Medicine, and was completed at the Tufts University School of Medicine in Boston. His Neurology specialty training was completed at the Columbia Presbyterian Medical Center- Neurological Institute in New York.


Arthur L. Horwich, MD

Dr. Horwich is Sterling Professor of Genetics and Pediatrics at Yale School of Medicine and Investigator of the Howard Hughes Medical Institute. He received undergraduate and MD degrees from Brown University, trained in Pediatric Medicine at Yale, then was a postdoctoral fellow in the Tumor Virology Lab at the Salk Institute and in Genetics at Yale, where he subsequently joined the Genetics Faculty and has remained. His independent work identified a mutant affecting the proper folding of imported mitochondrial proteins, which proved to be a double ring assembly, Hsp60, a chaperonin. He then focused on structure and mechanism of action of the related bacterial chaperonin, GroEL and its cooperating “lid” component, GroES. His most recent work focuses on misfolding-induced ALS produced by mutant superoxide dismutase 1. He has been recognized by membership in the National Academy of Sciences and Institute of Medicine, and has received a number of international awards including: Gairdner International Award, Wiley Prize in Biomedical Sciences, Rosenstiel Award in Basic Medical Science, Louisa Gross Horwitz Prize, Shaw Prize in Life Science and Medicine, and Lasker Award for Basic Medical Research.


Santosh Kesari, MD, PhD, FANA, FAAN

Dr. Kesari is currently Chair, Department of Translational Neuro-Oncology and Neurotherapeutics, John Wayne Cancer Institute. He is also Director of Neuro-Oncology, Providence Saint John’s Health Center and Member, Los Angeles Biomedical Research Institute. Dr. Kesari is also Director of the Translational Neuro-Oncology Laboratories at the Moores Cancer in addition to a Professor of Neurosciences at the UC San Diego School of Medicine. Dr. Kesari is ranked among the top 1% of neuro-oncologists and neurologists in the nation, according to Castle Connolly Medical Ltd. He is a winner of an Innovation Award by the San Diego Business Journal. He is on the advisory board of American Brain Tumor Association, San Diego Brain Tumor Foundation, Chris Elliott Fund, Nicolas Conor Institute, Voices Against Brain Cancer, and Philippine Brain Tumor Alliance. He has been the author of over 200 scientific publications and is the inventor on several patents and patent applications. He completed ahis residency at the Massachusetts General Hospital/Brigham and Women’s Hospital in Boston and ahis fellowship at the Dana-Farber Cancer Institute. He earned a Ph.D. degree in molecular biology and a medical degree from the University of Pennsylvania, School of Medicine. He is a member of the Society of Neuro-Oncology, American Society of Clinical Oncology, Indian Society of Neuro-Oncology, and American Academy of Neurology.


Nicholas DP Cosford, PhD

Dr. Cosford is Professor at Sanford Burnham Medical Research Institute, La Jolla, California. His research is involved in investigating the interactions of small molecule compounds with therapeutically important proteins and cellular signaling pathways. One aspect of his research emphasizes the use of medicinal chemistry and chemical biology approaches to probe intracellular pathways that regulate cell survival and cell growth. Another area of active research is the development of synthetic chemistry methodology using microfluidic technology for the rapid synthesis of biologically active small molecules. Therapeutically, his group is primarily focused on the discovery and optimization of compounds that have the potential to treat cancer, CNS diseases and infectious diseases. He has more than 20 years of experience leading small-molecule drug discovery projects in the pharmaceutical industry. At Sibia Neurosciences and at Merck Research Laboratories he directed multidisciplinary research teams focused on small- molecule hit-to-lead optimization and was responsible for moving several lead compounds through to the clinical phase. Examples include taking a nicotinic receptor agonist (Altinicline, SIB-1508Y) from initiation of research through to Phase II clinical for PD; taking mGluR5 negative allosteric modulators from HTS hits through in vivo proof-of-concept to Phase I clinical (ongoing); and design, synthesis and optimization of an mGluR5 PET tracer clinical candidate; design, synthesis and optimization of an Akt allosteric inhibitor preclinical candidate. To date, he has published more than 55 peer reviewed scientific publications, has more than 30 issued patents, and more than 30 additional patent applications pending. In 2006 he received the FRAXA Research Foundation Award for Outstanding Contributions to Fragile X Research.


Prof. Krishna K. Sharma, PhD

Dr. Sharma is Professor of Ophthalmology at University of Missouri, Columbia, USA. As PI on several previous grants during the last 20 years, he has investigated lens crystallin structure-function and the molecular mechanisms involved in lens protein aggregation. His research focuses on mechanisms that underlie age-related changes in lens alpha-crystallin, the major protein in the lens, and how such changes can be modified to prevent age-related loss of lens transparency and cataract formation. Understanding protein interactions in the lens and how these interactions alter the function and structure of alpha-crystallin is fundamental to identifying the mechanisms of cataract development, and to developing modulators that will prevent lens changes and cataract formation. His work lead to the identification of chaperone functional sequences in alpha A and B-crystallins, and to the synthesis and characterization of peptide chaperones derived from these two crystallins. At this time his laboratory is conducting studies to develop alpha-crystallin- derived peptides that will inhibit protein aggregation and misfolding that leads to age- related loss of lens transparency. Another facet of his research is to identify the initiating events for age-related lens changes by characterizing proteolytic mechanisms responsible for crystallin fragmentation and aggregation. His group has identified specific crystallin fragments in aging human lenses that induce protein aggregation. He is recipient of several awards and honors, including, Lew R. Wasserman Merit Award, Research to Prevent Blindness, Robert E. McCormick Scholar Award, Research to Prevent Blindness and Cataract Research Award, National Foundation for Eye Research. He also serves on National Advisory Eye Council of NIH.