Harley Kornblum, MD, PhD

Harley Kornblum, M.D., Ph.D. 

Professor, Psychiatry, Pharmacology and Pediatrics; Director, Neural Stem Cell Research Center, UCLA Semel Institute for Neuroscience and Human Behavior


Harley Kornblum, M.D., Ph.D., is a pediatric neurologist who studies how the brain develops and how this process can go awry in neurodevelopmental disorders and brain cancers. His lab takes an interdisciplinary approach to this research, collaborating with experts in genetics, genomics, and bioinformatics to better understand the genetic and cellular mechanisms that underlie healthy and abnormal brain development. Kornblum hopes this research will lead to the new treatments for brain cancers, spinal cord injuries and neurodevelopmental disorders such as autism and cerebral palsy.

Kornblum studies how stem cells within the brain and spinal cord proliferate, or self-renew, and differentiate to produce brain cells, including the neurons that process and transmit information and the glia that support and protect them. This work has led to novel theories on how autism develops. Kornblum and his team are pursuing these theories in order to develop potential treatment strategies. Another area of focus for the lab is studying how the production of new neurons declines with age. Kornblum hopes this research will inform the development of drugs that promote the growth of new neurons. His group is also generating spinal motor neurons, the cells most affected in amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy, from human embryonic stem cells. The team is conducting pre-clinical testing to evaluate if these stem cell-derived spinal motor neurons can be used as cell replacement therapies to treat disease and injury.

The Kornblum lab was among the first to identify cells within human brain tumors that have properties similar to neural stem cells. These cells, commonly known as brain tumor stem cells, are believed to be the root cause of brain cancer and a key mechanism of its resistance to therapy and recurrence. Kornblum studies how these cells grow and resist treatments in order to develop methods to prevent their growth and the formation of tumors. Kornblum and his collaborators are now studying how the cells surrounding brain tumor cells influence their growth in order to develop treatments that disrupt this process.

Kornblum earned his medical and doctorate degrees from UC Irvine. He subsequently completed post-doctoral fellowships at the University of Hawaii and UC Irvine, followed by a residency, internship and clinical fellowship in pediatric neurology at UCLA David Geffen School of Medicine.


Honors & Affiliations


  • JoAnne Brasel Memorial Lecture, LA BioMed, 2016 
  • Leslie Chair in Pioneering Brain Research, UCLA, 2006


  • Associate Director, UCLA Intellectual and Developmental Disabilities Research Center
  • UCLA Jonsson Comprehensive Cancer Center
  • UCLA Molecular Biology Institute


Kornblum's work is funded by the Adelson Medical Research Foundation, UCLA Specialized Program of Research Excellence (SPORE) in Brain Cancer, the National Cancer Institute, and the Eunice Kennedy Shriver National Institute of Child Health and Human Development.