Brain Tumor & Neuro-Restoration Program

The Sanford Stem Cell Institute (SSCI) Brain Tumor & Neuro-Restoration Program houses a new, revolutionary effort to exploit the therapeutic weaknesses of brain cancers — the creation of stem cell-derived cancer “avatars,” CRISPR-edited to include common cancer mutations.

The program — co-directed by Frank Furnari, PhD, and Alex Khalessi, MD, MBA — involves partnership with a high-volume destination neurosurgical program that allows for spatial mapping and surgical treatment of the most complex brain tumors.

So far, the novel technique has produced highly accurate replicas of two types of glioblastoma found in adults, as well as pediatric atypical teratoid rhabdoid tumor — all fast-growing brain cancers that typically reduce life expectancy to 18 months or less. The technique has also produced a highly accurate model for the telomerase reverse transcriptase (TERT) promoter mutation found in 90% of glioblastoma tumors, as well as in other cancers such as bladder and endometrial cancers.

The SSCI Brain Tumor program — set to officially launch Fall of 2024 — will explore the heterogeneity of brain tumors, target identified therapeutic vulnerabilities, and seek to engineer the next generation of human tumor avatars for further innovation.

Frank Furnari, PhD, Co-Director

Frank Furnari, PhD, is a professor in the Division of Regenerative Medicine within the Department of Medicine at the University of California San Diego. He and his lab have made significant contributions to the fundamental understanding of mechanisms underlying therapeutic resistance in glioma, functionality of tumor heterogeneity, and the evolution of adult and pediatric brain tumors through genetic engineering of human pluripotent stem cell-derived avatar models. Furnari serves as associate editor for Neuro-Oncology and holds advisory board positions for the National Brain Tumor Society, the Society for Neuro-Oncology, and the Sontag Foundation. He is also co-founder of Trotana Therapeutics. 

Alex Khalessi, PhD, MBA, Co-Director

Alexander Khalessi, MD, MBA, is a board-certified neurosurgeon at UC San Diego Health who specializes in complex cranial surgery, endovascular neurosurgery, stroke care, and neurological oncology. He is chair of the Department of Neurological Surgery; the inaugural Don and Karen Cohn Chancellor's Endowed Chair in Neurosurgery; and professor of neurological surgery, radiology, and neurosciences at the UC San Diego School of Medicine. Khalessi is also the current president of the Congress of Neurological Surgeons (CNS), the leading academic society for neurosurgical professionals worldwide. Additionally, he sits on the American Association of Neurological Surgeons/CNS Washington Committee and the Board of Governors for the American College of Surgeons. He is also a founding member of the Medical Device Innovation Consortium and advises the FDA on early clinical feasibility for emerging neurosurgical devices.