The Future of Brain Disease Research: a Patient-Centric Perspective
What does the future of brain disease research hold?
Degenerative diseases of the brain affect millions worldwide: 55 million people are living with Alzheimer’s disease or other dementias, and an estimated 2.8 million live with multiple sclerosis. Their prevalence continues to skyrocket as the global population ages. These conditions, in which neurons in the brain and nervous system become damaged and sometimes die, currently have no cures. How can stem cells help us to tackle this growing challenge and finally deliver effective therapies to patients?
Parkinson’s disease researcher and clinician Vikram Khurana, MD, PhD (Brigham and Women’s Hospital) and stem cell scientist and MS patient Valentina Fossati, PhD (NYSCF) discuss how they are leveraging stem cells to develop personalized, patient-focused treatments for brain diseases. The discussion was moderated by NYSCF’s Raeka Aiyar, PhD.
What you’ll learn:
- The current major obstacles to unlocking new, effective brain disease treatments
- Why stem cells are essential for solving the mysteries of brain diseases and finding effective treatments
- How patient-focused research is helping advance novel drugs to clinical trials
Read highlights from the discussion here.
Panelists
Vikram Khurana, MD, PhD
Chief of the Division of Movement Disorders, Brigham and Women’s Hospital, Harvard Medical School
- Dr. Khurana is a leading physician-scientist in Parkinson’s disease and similar neurological diseases like multiple system atrophy.
- Dr. Khurana’s research uses patient stem cells to study alpha-synuclein, a protein in the brain that becomes toxic in Parkinson’s patients. He received the NYSCF – Robertson Stem Cell Investigator award to fund this work.
Dr. Khurana is Chief of the Division of Movement Disorders at Brigham and Women’s Hospital and Harvard Medical School. He is Principal Faculty at the Harvard Stem Cell Institute, and an Associate Member of the Broad Institute of Harvard and MIT. He is a member of the Board of Directors of the MSA Coalition. His clinical and research interests relate to neurodegenerative disorders focusing on Parkinson’s disease and related dementias, rarer disorders including multiple system atrophy and ataxias. Patients with these disorders are seen by him at Brigham and Women’s Hospital in the P+A+MSA clinic.
His current research continues to bring stem-cell technologies toward personalized and precise diagnostics and therapeutics for neurodegenerative disorders. In 2018, he was named a NYSCF – Robertson Stem Cell Investigator, in 2019 a George C. Cotzias Fellow of the American Parkinson’s Disease Association and in 2020 an investigator of the Aligning Science Across Parkinson’s Initiative.
Valentina Fossati, PhD
Senior Research Investigator
The NYSCF Research Institute
- Dr. Fossati leads NYSCF’s multiple sclerosis program, where she uses methods she has pioneered to convert patient stem cells into all major brain cell types, with the ultimate aim of enabling drug discovery and regenerative therapies.
- As an MS patient and researcher, Dr. Fossati’s diagnosis inspired her to focus her stem cell expertise on this understudied disease with such wide-ranging patient outcomes
Dr. Fossati leads NYSCF’s multiple sclerosis (MS) program and is a NYSCF–Druckenmiller Postdoctoral Fellow Alumna. She obtained her PhD in Biotechnology from the University of Bologna, and during her postdoctoral work at Mount Sinai School of Medicine, she studied immune system development. After being diagnosed with MS in 2009, Dr. Fossati shifted her research to better understanding this disease. Bringing stem cell expertise to MS research, she has pioneered methods to convert patient stem cells into all major brain cell types, including astrocytes, oligodendrocytes and microglia, which provide vital support to neurons. By building living models of the brain in a dish, Dr. Fossati is uncovering the role of these cells in diseases like MS and Alzheimer’s, with the ultimate aim of enabling drug discovery and cell replacement therapies. Dr. Fossati also leads NYSCF’s efforts to develop miniature 3D versions of the brain called “organoids” from human stem cells, which recapitulate the brain’s environment and have become very useful for studying brain development and disease.
Raeka Aiyar, PhD
Vice President, Scientific & DEIB Outreach
The NYSCF Research Institute
- Dr. Aiyar is an experienced geneticist and science communicator
- She leads NYSCF’s DEI strategy, including our internal affinity groups to support marginalized groups, our DEIB-focused events and communications, our initiative to build a diverse stem cell biobank, and our inclusive grantmaking efforts
Dr. Aiyar is an experienced geneticist turned science communicator with a passion for advancing diversity in science. Trained in biology and bioinformatics at the University of Waterloo, she received her PhD at the European Molecular Biology Laboratory in Germany, where she used genomics technology to investigate new therapeutic strategies for mitochondrial diseases. Since then, Dr. Aiyar has dedicated her career to science communication, engaging a variety of audiences through writing, training, and outreach. As Director of Communications and Development at the Stanford Genome Technology Center, she led a range of efforts in patient outreach, scientific strategy, and collaboration building. In her current role, she oversees NYSCF’s scientific communication initiatives, developing content for diverse audiences through print and digital media as well as event programming. Since 2020, she has led NYSCF’s DEIB initiatives across internal affinity groups, events and communications, biobank diversification, and grantmaking.