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.

Senior Research Program Officer

Sang Y. Lee is a Senior Research Program Officer for the Neurofibromatosis Therapeutic Acceleration Program (NTAP) at the Johns Hopkins University School of Medicine. He is an experienced biomedical scientist and officer with diverse research background and keen knowledge of preclinical cancer drug development and the role of iron mismanagement in human disease with over 20 years of experience in academic and government research institutes. At present, he is interested in generating new grant initiatives, overseeing new and funded projects, and data monitoring and data analysis for uploaded neurofibromas data.

Director of Clinical Research

Carlos Romo is an Assistant Professor of Neurology, Oncology and Medicine at the Johns Hopkins University School of Medicine, an attending physician in the Johns Hopkins Comprehensive Neurofibromatosis Center, and the Director of Clinical Research of the Neurofibromatosis Therapeutic Acceleration Program. His research interests include early phase clinical trials for the treatment of neurofibromatosis and gliomas.

Scientific Advisor

Verena Staedtke is Associate Professor of Neurology at the Johns Hopkins University School of Medicine and the Director of Pediatric NF at the Johns Hopkins Comprehensive Neurofibromatosis Center. She is an expert in the clinical care of children and young adults with neurofibromatosis and other related neuro-oncological conditions. Her research is focused on developing novel treatments for these conditions, with a particular focus on gene and cell-based therapies.

Financial Manager

Jacquelyn Eubanks-Rudd is a certified public accountant with a certified global management accountant designation and a master’s degree in information systems. She has been a financial manager for over 30 years and for 15 of those years served as director of finance and administration at two institutions of higher learning. Her area of expertise is with institutions of higher learning and medical foundations. She has been the finance manager at the Neurofibromatosis Acceleration Program (NTAP) at Johns Hopkins since January 2018

Program Manager 

Rhonda Jackson is Program Manager for the Neurofibromatosis Therapeutic Acceleration Program at the Johns Hopkins University School of Medicine, Department of Neurology. She has over 20+ years of experience in administration and editorial management between Johns Hopkins University School of Public Health and School of Medicine and University of Maryland School of Medicine. She received her Associates in Healthcare Administration and Bachelor’s in Business Management. Her interests include research administration and editorial management.

Clinical Research Program Administrator

Joshua M. Roberts is the Clinical Research Program Administrator for the Neurofibromatosis Therapeutic Acceleration Program (NTAP) at the Johns Hopkins University School of Medicine. He has a degree in cognitive neuroscience and has applied this knowledge to overseeing complex multi-disciplinary research operations across numerous fields of study. With expertise in scientific writing, regulatory administration, database management, various neuroimaging modalities, and study coordination, he oversees clinical research projects examining effective treatments for people with Neurofibromatosis Type 1.

Nurse Practitioner

Christina Allen, CRNP, DNP is a board-certified family nurse practitioner at the Johns Hopkins Comprehensive Neurofibromatosis Center. She divides her time between providing clinical care for pediatric patients in the Neurofibromatosis Center with Dr. Staedtke and serving as the clinical nurse for research studies through the Johns Hopkins Comprehensive Neurofibromatosis Center and the Neurofibromatosis Therapeutic Acceleration Program.

She brings over 12 years of nursing experience, and a bulk of clinical knowledge. She truly enjoys caring for patients across the lifespan and has a keen interest in clinical research and therapeutic options for patients with 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.

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.

Scientific Advisor

Dr. Hirbe is currently an Assistant Professor of Medicine and Pediatrics in the Division of Medical Oncology in the Department of Medicine, and Director of the Adult Clinical Neurofibromatosis Program at Washington University in St. Louis.  Clinically, she cares for patients with bone and soft tissue sarcomas, other rare tumors, and patients with cancer predisposition syndromes including Neurofibromatosis Type 1, Li Fraumeni, and Retinoblastoma. Her laboratory is focused on genomics and mouse models of soft tissue sarcomas, particularly MPNST, with the goal of identifying drivers that can be exploited as diagnostic biomarkers or therapeutic targets.

Dr. Lu Le, MD, PhD is a Dermatologist with an interest in Neurofibromatosis. He received his Ph.D. degree in Immunology and Molecular Genetics, and a medical degree (M.D.) from the Medical Scientist Training Program at the University of California, Los Angeles.  He completed an Internship in Internal Medicine at UCLA/St. Mary Medical Center, residency training in Dermatology and a postdoctoral fellowship in Cancer Biology from University of Texas Southwestern Medical Center.  He is board certified by the American Board of Dermatology.  Dr. Le sees patients in the Dermatology clinic and at the UTSW Comprehensive Neurofibromatosis clinic.  As a principal investigator in cancer biology, Dr. Le’s laboratory investigates how initiating genetic and tumor microenvironmental events interplay to regulate NF1-associated tumorigenesis.  A major contribution of Dr. Le’s laboratory is the generation and exploitation of novel Neurofibromatosis models to decipher mechanisms that initiate Neurofibroma development and drive their malignant transformation.  His laboratory has identified the cells of origin for different types of Neurofibroma, defined developmental “window-of-opportunity” within Schwann cell lineage for Neurofibroma development and delineated vital cancer pathways for its malignant transformation.  These studies have addressed fundamental, unanswered questions in the Neurofibromatosis field and could lead directly to potentially effective therapies for NF1 patients, where none exist today.

June Lee is a physician-scientist with over 20 years in the biotechnology and pharmaceuticalindustry. Most recently, she was Founder and CEO of Esker Therapeutics. She previouslyserved as Executive Vice President, Chief Development Officer and Chief Operating Officer ofMyoKardia where she built and led a world-class development organization that was acquiredby Bristol Myers Squibb for $13.1 billion in November 2020. Prior to MyoKardia, Dr. Lee wasProfessor of Medicine at UCSF School of Medicine, where she served as Director ofTranslational Research and built the Catalyst Program, an internal accelerator for early-stagetechnologies. As the therapeutic area head at Genentech, Dr. Lee led early clinical developmentprograms in cardiovascular and metabolic diseases, infectious diseases, and respiratorydiseases. Dr. Lee serves on numerous boards in the healthcare industry including the AdvisoryBoard for Johns Hopkins University Center for Therapeutic Translation, the Board of Directorsfor Tanaya Therapeutics, Eledon Pharmaceuticals Inc. and GenEdit, and is a member of theScientific Advisory Board for Foresite Labs. Dr. Lee received her undergraduate degree inchemistry at the Johns Hopkins University, earned her medical degree at the School ofMedicine at University of California, Davis, and completed her clinical training in internalmedicine and pulmonary & critical care at UCLA and UCSF.

Marcus Ratley is an NF Fighter. Diagnosed with Neurofibromatosis type 1 at a young age. NF has played a role in his life daily. Throughout the years he had the opportunity to meet and build friendships with many other NF Heroes (People Living with Neurofibromatosis). One of his passions is bringing awareness about Neurofibromatosis and being an advocate for others with Neurofibromatosis. Marcus believes in research and the importance of clinical trials that focus on the concerns of those living and surviving with Neurofibromatosis.

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.

Advisor

Marjorie B. Tiven has devoted her professional and philanthropic work to cultural diplomacy, education, and public service. She is the founder and president of Global Cities, Inc., a program of Bloomberg Philanthropies. Through her signature program, Global Scholars, Tiven works with a worldwide network of cities committed to developing global competency among the next generation of students through international digital exchange. Tiven’s program focuses students on issues of public health, environmental stewardship, and a sustainable future—and grounds them in the scientific mindset they will be able to draw on as adults in tackling the world’s problems. This global education program has grown over the last five years from 2,000 students to over 13,000 students in 64 cities in 29 countries. Tiven’s global scholars inspire her to collaborate in every way possible to solve the world’s problems. It is in that spirit that she is thrilled to support the work of her daughter Rachel Tiven and Sally Gottesman in their efforts to hasten a cure for neurofibromatosis, introducing them to Jaishri Blakeley, MD at Johns Hopkins Medicine.

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 (cutaneous neurofibromas and malignant peripheral nerve sheath tumors).