Cynthia J. Tifft, MD, PhD, to receive the 2027 Roscoe O. Brady Award
WORLDSymposium is excited to announce Cynthia J. Tifft, MD, PhD, as the recipient of the 2027 Roscoe O. Brady Award. Dr. Tifft is a Senior Clinician at the National Human Genome Research Institute where she serves as Deputy Clinical Director and directs the Pediatric Undiagnosed Diseases Program.
Dr. Tifft received her B.A. in biology from Revelle College at the University of California at San Diego and her M.S. in genetic counseling from Rutgers University. She received her Ph.D. in genetics from the University of Texas Graduate School of Biomedical Science at UT M.D. Anderson Cancer Center and her M.D. from the McGovern Medical School at UT Health Houston. She completed her pediatric residency at Johns Hopkins Hospital and fellowship in medical genetics at the National Institutes of Health. Dr. Tifft joined the faculty of the George Washington University School of Medicine at Children’s National Hospital in 1991, becoming chair of the Division of Genetics and Metabolism in 1996. In 2009, she returned to the NIH to focus her attention on children with rare and undiagnosed diseases.
For many years, Dr. Tifft’s clinical and research interests have been lysosomal disorders affecting the central nervous system. She is the principal investigator of a longitudinal natural history study of children and adults with glycosphingolipid and glycoprotein disorders, including Tay-Sachs and Sandhoff diseases, GM1 gangliosidosis and type 1 sialidosis.
In May 2019 Dr. Tifft and her “all-woman team from five continents” launched the initial first-in-human gene therapy trial for patients with Type I and Type II GM1 gangliosidosis. Connecting with desperate families and enrolling patients in the middle of a worldwide pandemic gave participants the chance at a better life. For their work the team received the NHGRI GREAT Award in 2020 and the NIH Director’s Award in 2021.
As director of the Pediatric Undiagnosed Diseases Program, Dr. Tifft has coordinated the selection and clinical evaluation of nearly 600 children whose diagnoses have long eluded the medical community. Using comprehensive phenotyping and next generation sequencing, she and her team have provided diagnoses for a third of selected patients, including newly discovered disease genes, and have strong candidate genes under study for many more.
The 2027 Roscoe O. Brady Award will be presented at the beginning of the WORLDSymposium 2027 Basic Science Session on Monday, February 1 at 08:00 PST.
About the WORLDSymposium™ Roscoe O. Brady Award
The Roscoe O. Brady Award is given annually by WORLDSymposium to recognize one individual for outstanding innovation and accomplishment in the field of lysosomal disease research and therapy. Dr. Brady was the third recipient of the “Award for Innovation and Accomplishment.” After decades of research on the biology and biochemistry of lysosomal diseases, Dr. Brady undertook experimental administration of exogenous proteins to replace the patients’ genetic enzyme deficiencies. His ongoing work led to many discoveries in the field, mentoring and inspiring numerous other investigators.
In the process of treating three patients with Gaucher disease, biomarkers were discovered and validated. His work ultimately resulted in FDA approval (1991) and commercialization of the first “enzyme replacement therapy” (ERT). This was an intravenous biologic medication alglucerase for Gaucher disease, the human enzyme purified from massive quantities of normal human placenta. This landmark accomplishment was followed by approval of imiglucerase made using recombinant DNA technology. Later in his career, Dr. Brady investigated substrate depletion treatment, molecular chaperone therapy, and gene therapy for patients with lysosomal disorders.
After a lifetime (October 11, 1923 – June 13, 2016) with decades of work in the field, and with the approval from his family, the WORLDSymposium award for innovation and accomplishment was renamed in his honor at the WORLDSymposium 2018 meeting.
Past Award Recipients:
2026 Peter Marks, MD, PhD
2025 James M. Wilson, MD, PhD
2024 Elsa Shapiro, PhD
2023 William A. Gahl, MD, PhD
2022 Stuart A. Kornfeld, MD
2021 Ellen Sidransky, MD
2020 John F. Crowley
2019 Danilo A. Tagle, PhD, MS
2018 Mark E. Haskins, VMD, PhD
2017 Konrad Sandhoff, PhD
2016 Emil D. Kakkis, MD, PhD
2015 Stephen C. Groft, PharmD
2014 Gregory A. Grabowski, MD
2013 Robert J. Desnick, PhD, MD
2012 John J. Hopwood, PhD
2011 Steven U. Walkley, PhD
2010 William S. Sly, MD
2009 Elizabeth F. Neufeld, PhD
2008 Roscoe O. Brady, MD
2006 Henri A. Termeer
2004 William Krivit, MD, PhD