About Dr. John Ravits

Learn from yesterday, live for today, hope for tomorrow.
Albert Einstein
Dr. John Ravits, MD, FAAN
Dear friends,
I am incredibly grateful to be able to pursue something as important as breakthroughs in humanity’s understanding and treatment of ALS. My team and I work tirelessly to find new ways of tackling this devastating illness. Thanks to our friends, and supporters, we are able to focus on the most promising leads.
The past year has been an incredible time of progress. As you’ll see we have had several research and clinical trials take shape, critical efforts that can translate good ideas into potential cures. We have also maintained our designation as an ALS Association Certified Treatment Center of Excellence, meaning we’re one of the best centers of our kind in the country. The quality of our research and clinical care programs is thanks to an incredible group of individuals on my team who are passionate about understanding and treating ALS– individuals you’ll have a chance to meet in the coming pages. Behind all of this is an extraordinary community of donors and Neurological Sciences advocates who stand with us in seeking cures for ALS. Philanthropic partners and advocates like you offer us the freedom and flexibility to pursue our most promising ideas and meet our patients’ greatest needs.
I hope as you read these pages you can see just how special our people and our work are, and that you are encouraged by the progress being made. There is hope on the horizon for ALS, thank you for being on the journey with us.
Sincerely,

Biography
I am the principal investigator of the Ravits Lab and a physician-scientist in the Department of Neurosciences at UC San Diego. I also teach in UC San Diego’s neurosciences graduate program, neurology residency and neurology fellowship. My research activities fall into three areas: basic research on ALS mechanisms, translational research on ALS treatments, and clinical care for people with ALS.
In the basic research lab, my group is focused on: (1) gene expression (“genomics”) of motor neurons and molecular neuropathology; (2) strategies of gene therapies especially targeting C9ORF72, the gene which is the most common genetic form of ALS, and targeting ATXN2, which offers a way to treat people with sporadic disease; (3) microRNAs, small genes that are relevant as part of a new frontier in ALS research; and (4) TDP-43 phosphorylation by a protein called CK1ε, which we are excited about because it is a mechanism of disease that could provide an upstream molecular switch for treatments that use small molecule inhibitors or gene therapies.
In the translational program, my group performs clinical trials to test treatment for patients, the most exciting of which involve antisense oligonucleotides, which are gene therapies, and various small molecules targeting different pathways. We also collect blood and tissue for repositories for researchers around the world.
In the clinic, I lead a team of providers (including therapists and nurses) to meet the needs of current patients- we serve between 100-150 patients with ALS. I began my journey with pure clinical observations of patients and created models of how the disease spreads, while at the same time establishing biorepositories for patient samples and studying them using several methods, including lasers, whole genome technologies, and computers.
I believe ALS can be defeated and, while doing this, seek to parlay our unique perspective on neurodegenerative diseases into an opportunity to better understand other neurodegenerative diseases, such as frontotemporal dementia and Alzheimer’s disease.
As a physician-scientist with innovative ideas in an exciting neurosciences environment, I am excited to bring my research forward and at the same time, help develop the next generation of physicians and scientists.
