Leadership

Director

Jaishri Blakeley is a Professor of Neurology, Neurosurgery and Oncology at the Johns Hopkins University School of Medicine and the Director of the Johns Hopkins Comprehensive Neurofibromatosis Center. She is an expert in clinical trial design and the development of translational studies to advance new therapies in rare neuro-oncologic diseases.

Scientific Advisor

Bruce Korf is the Wayne H. and Sara Crews Finley Chair in Medical Genetics and Professor and Chair, Department of Genetics and Director, Heflin Center for Genomic Sciences at University of Alabama at Birmingham. He has extensive expertise in all aspects of neurofibromatosis type 1, in particular plexiform neurofibromas, and is a world thought leader in the field of neurofibromatosis.

Scientific Advisor

Barbara Slusher is Professor of Neurology and Psychiatry and the Director of the Brain Science Institute (BSI) NeuroTranslational Drug Discovery Program at Johns Hopkins School of Medicine. She leads an integrated drug discovery program consisting of 20 veteran drug discovery scientists and is the expert for developing drug therapies for neurological and psychiatric disorders.

Chair

Bruce Korf is the Wayne H. and Sara Crews Finley Chair in Medical Genetics and Professor and Chair, Department of Genetics and Director, Heflin Center for Genomic Sciences at University of Alabama at Birmingham. He has extensive expertise in all aspects of neurofibromatosis type 1, in particular plexiform neurofibromas, and is a world thought leader in the field of neurofibromatosis.

Annette Bakker is the President and Scientific Director of the Children’s Tumor Foundation. She has extensive basic research skills in drug discovery and most recently created and headed the oncology research and development unit at Siena Biotech in Italy, where she focused on drug discovery and development. Dr. Bakker’s research has been internationally recognized by more than 30 peer-reviewed papers, patents, and innovation awards.

Larry Blankstein is a clinical research scientist with over twenty-four years’ experience in pharmaceutical and biotechnology clinical drug development and operational execution.  Having worked within CRO and Sponsor organizations, he brings a collaborative approach to CRO/Sponsor partnerships.  He has hands on experience in Phase 1-III clinical studies in project management, clinical operations, study budget management and CRO selection and management.  He has developed drug strategies and managed projects from product conception, through process development, pre-clinical development and clinical studies to filing with the FDA and EMA. His cross-functional leadership spans both internal team management as well as CRO oversight in the planning and execution of clinical studies.  He has led multi-national clinical teams in oncology, endocrinology, ophthalmology and orphan disease drug development across multiple technology platforms including small molecules, biologics, cell therapies and gene therapy. In addition, he has participated in many due diligence projects and has managed staff and project transitions during a number of successful mergers and acquisitions. As a consultant to the biotech and pharma industry since he left Genzyme in March 2014, he has been involved in projects focused on site and patient recruitment for large global trials, pre-IND reviews, clinical operations management, clinical development team leader, project management with Japanese companies, etc.

As the mother of a 16 year old son with NF1 and plexiform neurofibroma, Penny strives to promote education, awareness and acceptance of those affected by NF. She has served on the Board of Directors for NF, Inc. Mid-Atlantic and currently sits on the Camp New Friends (NF) Advisory Board. Penny’s son was diagnosed at 3 months of age with an orbital plexiform neurofibroma and has participated in numerous clinical trials for plexiform neurofibromas.

Marco Giovannini is a Professor in Residence at UCLA Health and the Director of the Neural Tumor Research Laboratory. His research interests lie in investigating the genetic and biochemical basis of tumor growth to find target proteins and pathways for rational drug design that will improve tumor treatments. An author and co-author of numerous articles on various aspects of tumor research, Dr. Giovannini’s work has been published in such journals as Genes & Development, Nature Genetics, Cancer Research and Proceedings of the National Academy of Sciences.

Sally is the co-founder and Chair of Moving Traditions, which finds, creates, and delivers resources to inspire people to draw on Judaism at key lifecycle moments. She has over twenty years’ experience as a management consultant to not-for-profit organizations. Sally is also on the Board of her family-owned-business, Edison Properities, LLC. She and her partner are the happy parents of three children, one of whom has NF1.

Lanny Kirsch is an independent consultant to the biotech/pharma industry with extended assignments focused on the development of antibody therapeutics and molecular diagnostics. Dr. Kirsch has been affiliated with Boston Children’s Hospital, the National Cancer Institute, the National Institute of Child Health and Human Development and Amgen. His research interests have been focused on cancer specific genetic instability and cancer genetics.

Vic Riccardi is the Director of the Neurofibromatosis Institute and the Medical Director of NF California. He has been a tireless advocate of the NF community and has lectured extensively on this condition. In June 2008, he was the recipient of the prestigious von Recklinghausen Award and is now working closely with the House Research Institute to establish the initial clinic for the California NF1 Program.

Jeff Rothstein is a Professor of Neurology at Johns Hopkins and the Director of the Packard Center for ALS Research. He works primarily in neuromuscular diseases, with a particular focus on amyotrophic lateral sclerosis (ALS). He is interested in molecular mechanisms of selective neurodegeneration in motor neuron diseases and identification of novel drug or peptide therapeutics to delay or prevent motor neuron degeneration in ALS thru the use of cell culture and transgenic models.

Barbara Slusher is Professor of Neurology and Psychiatry and the Director of the Brain Science Institute (BSI) NeuroTranslational Drug Discovery Program at Johns Hopkins School of Medicine. She leads an integrated drug discovery program consisting of 20 veteran drug discovery scientists and is the expert for developing drug therapies for neurological and psychiatric disorders.

Rachel Tiven is the Executive Director of Immigrant Justice Corps, a non-for-profit fellowship program dedicated to meeting the need for high-quality legal assistance for immigrants seeking citizenship and fighting deportation. Previously, she was executive director of Immigration Equality and Immigration Equality Action Fund. Rachel serves on the board of the Children’s Tumor Foundation and lives in New York City with her partner and three children, one of whom has NF1.

Brigitte Widemann is a Senior Investigator and Head of the Pharmacology and Experimental Therapeutics Section of the Pediatric Oncology Branch of the National Cancer Institute. Her research program focuses on the clinical development of new agents for the treatment of refractory childhood cancers and genetic tumor predisposition syndromes, in particular neurofibromatosis type 1 (plexiform neurofibromas and malignant peripheral nerve sheath tumors).