Bringing CNS Members Together to Make Children’s Lives Better


Peter T. Tsai, MD, PhD

Profile written by Mustafa Sahin, MD, PhD

Peter T. Tsai, MD, PhD

Peter Tsai, MD, PhD, the 2013 recipient of the Philip R. Dodge Young Investigator Award, has been immersed in science from early in life. With two accomplished developmental biologists as parents, Peter was involved in research projects as a child. he recalls learning to run cesium chloride gradients and polyacrylamide gels with his parents. In addition, during high school he worked on projects involving migratory patterns of arctic squirrels with Brian Barnes at the university of Alaska, and on satellite imagery with Jobea Way at the Jet Propulsion Laboratory at the California Institute of Technology. These led to his love of Alaska, a state that he has visited several times since.

Peter then joined the MD/PhD program at the uCLA School of Medicine. There, he worked with Dr. hong Wu on two different projects. His Ph.D. thesis focused on characterizing the role of FOxN1 in thymic epithelial differentiation and the roles of BMP4 and FGF7/10 in thymopoiesis. his results were published as a first author paper in the journal, Blood. In addition, he characterized novel roles for the erythropoietin receptor in neuronal development and neuroprotection from ischemia. This led to a first author publication in the Journal of Neuroscience.

After completing his MD/PhD training, Peter did a pediatrics residency at Boston Children’s hospital and joined the Department of Neurology as a pediatric neurology resident in 2007. As a result of caring for patients with autism, he developed an interest in the role of the cerebellum in cognition and social behavior and in the roles for cerebellar dysfunction in neurodevelopmental disorders. This latter focus led to the development of the Cerebellar Disorders Clinic that Peter started and currently directs. At the same time, he sought to examine the role of the cerebellum in higher cognitive function using mouse models, and joined the lab of Dr. Mustafa Sahin using TSC a model.

In the Sahin lab, Peter generated a conditional knockout of the Tsc1 gene specifically in Purkinje cells, and found that these mice display autistic- like features, which can be prevented by mTOR inhibition. This study provided the first direct proof that the cerebellum is involved in the neural circuitry underlying autism and was published this summer in the prestigious journal, Nature with Peter as first author. The fact that Peter dove into basic science after a long hiatus and quickly generated important data published in a high-impact journal illustrates his dedication to investigation and organizational skills, originating from very early in his life.

Peter has also established himself as an excellent team member and collaborator both in the clinic and in the lab. he has had many productive collaborations including with Dr. David Kwiatkowski’s lab in characterizing the first allelic series of a Tsc2 mouse model. he also collaborated with Dr. Simon Warfield’s laboratory investigating the volume of cerebellum in TSC patients using MR imaging. Both of these studies have lead to publications in Human Molecular Genetics and Pediatric Neurology, respectively. On a more personal note, Peter is always optimistic, unselfish, cheerful with a great sense of humor. Outside of medicine and science, Peter’s life revolves around his family and his two children, who are 6 and 4 years old. Although it may seem incredible, based on his slender physique, he also has a deep enjoyment of food and loves to cook and eat.

Based on his creative ideas and hard work, Peter received a Clinical Research Training Fellowship from the American Academy of Neurology. This year, his application for a K08 award was funded by the NINDS on the first try. The combination of his basic science and clinical training, his character, and passion for his work make Peter an outstanding role model for child neurology trainees everywhere. There is no doubt that this year’s recipient of the Philip R. Dodge Young Investigator Award has the experience, determination, and creativity to make significant contributions to child neurology and autism research.