Don W. Cleveland Awarded 2018 Breakthrough Prize


On December 3, 2017, Don W. Cleveland, PhD, Distinguished Professor of Cellular and Molecular Medicine, Neurosciences and Medicine and chair of the Department of Cellular and Molecular Medicine, was honored with the Breakthrough Prize in Life Sciences for his work, "elucidating the molecular pathogenesis of a type of inherited ALS, including the role of glia in neurodegeneration, and for establishing antisense oligonucleotide therapy in animal models of ALS and Huntington’s disease.”

Dr. Cleveland has made seminal contributions to the understanding of how brain cells grow during normal mammalian development and how defects in those mechanisms lead to inherited neurodegenerative diseases, such as ALS and Huntington’s. Breaking with the mainstream belief in the field, Cleveland discovered that the genetic mutations that cause these diseases affect not just neurons, but the entire neighborhood of brain cells. He was the first to purify tau, the protein that mis-assembles and accumulates in Alzheimer’s disease and chronic brain injury, where it correlates with cognitive decline.

Based on these fundamental findings, Cleveland and his colleagues developed a ground-breaking approach to treating neurodegenerative diseases: designer DNA drugs that reduce synthesis of the toxic proteins produced by gene mutations or increase production from poorly expressed genes. This technology has already proven successful — it’s the underlying principle behind Ionis Pharmaceuticals’ FDA-approved therapeutic for spinal muscular atrophy, an inherited muscle wasting condition that was previously always fatal. Now, kids who were completely immobilized are walking. Similar designer DNA drugs are currently in clinical trials for the treatment of ALS, Huntington’s disease and Alzheimer’s disease.

Marilyn G. Farquhar Receives 2017 Revelle Medal


On November 17, 2017, Marilyn G. Farquhar, PhD, distinguished professor emerita of cellular and molecular medicine, was awarded the 2017 UC San Diego Revelle Medal, the highest honor bestowed by the Chancellor to current and former faculty members to recognize sustained, distinguished, and extraordinary service to the UCSD community, as well as a record of accomplishment that advances the UC San Diego mission of exceptional teaching, research, service and patient care.

Dr. Farquhar entered the field of science when female graduate students were rare. Today, her discoveries are part of the canon of cell biology. She joined UC San Diego in 1990 and served as the school’s first chair of the Department of Cellular and Molecular Medicine. She helped build the UC San Diego School of Medicine to become a top-ranked, national program. Farquhar is a founding member and former president of The American Society for Cell Biology. Her dedication to teaching and mentorship has made a powerful impact on the physicians and medical scientists of tomorrow.

After stints at the University of California, San Francisco, The Rockefeller University—where she became the first woman to be named a professor of cell biology—and Yale University, Farquhar joined UC San Diego in 1990, with her husband, famed physician-scientist George Palade. Farquhar served as the school’s first chair of the Department of Cellular and Molecular Medicine. She helped build the UC San Diego School of Medicine to become the top-ranked program it is today—the nation’s 18th best research-intensive medical school, according to U.S. News World Report.

Farquhar’s research accomplishments have been recognized by many prestigious awards including the E.B. Wilson Medal of the American Society of Cell Biology, the Distinguished Scientist Medal of the Electron Microscopy Society of America, the Homer Smith Award of the American Society of Nephrology and the Rous-Whipple Award of the American Society for Investigative Pathology. She is also well known for her dedication to teaching and mentorship, making a great impact in training the next generation of outstanding physicians and medical scientists.


Potential breakthrough drug for Huntington's to enter advanced clinical testing
A potential breakthrough drug for treating Huntington’s disease from Carlsbad’s Ionis Pharmaceuticals will begin an advanced clinical trial early next year. If successful, the trial could lead to marketing approval.

Ionis partner Roche, which is handling the clinical trials, made the announcement Sunday. The trial will take place globally.

Ionis is also developing drugs for Alzheimer’s and ALS, two other incurable and fatal neurodegenerative diseases.

The drug, RG6042, (originally IONIS-HTTRx) inhibits production of the mutant huntingtin. It uses an Ionis technology called antisense, which blocks or changes genetic activity. All Ionis drugs use antisense.

Ionis developed RG6042 in collaboration with researchers from the lab of Don W. Cleveland, Ph.D. at UC San Diego. It is injected into the spinal fluid, enabling it to reach the brain. Roche licensed the drug last December, assuming the lead in development.
UCSD and Nikon open new medical research center
UC San Diego Thursday announced the opening of its new Nikon Imaging Center, a research facility with cutting-edge microscopy and imaging technologies.

Nikon’s newest research center is the third of its kind in North America — along with Harvard Medical School and the Northwestern University Feinberg School of Medicine.

“UC San Diego’s partnership with Nikon is very exciting because it will enrich both the scientific research capabilities and educational activities on campus,” said UCSD professor Samara Reck-Peterson, Ph.D., a member of the School of Medicine’s department of cellular and molecular medicine.
Lab-grown brain bits open windows to the mind — and a maze of ethical dilemmas
The term "minibrain" vividly evokes the aspirational goal of this fast-moving area of research: to mimic the complexity of the human brain and illuminate the biology of the human mind, one of science’s darkest black boxes. As the technology, which scientists refer to in journal articles as “cerebral organoids,” improves, the more the “minibrain” title fits.

At the University of California at San Diego, stem-cell scientist Alysson Muotri, Ph.D. is attempting to “Neanderthalize” a brain organoid as part of his larger search for the biological basis of modern humans’ sophisticated social abilities. He used gene-editing technology to introduce a mutation found in Neanderthal genomes to a modern human stem cell. The early, unpublished results, he says, are an organoid that, instead of being smooth and spherical, is lumpy like popcorn — suggesting that the gene mutation significantly influences early brain development.
New National Training Program Aims to Mainstream Glycosciences
Over the next five years, the National Heart, Lung, and Blood Institute, part of the National Institutes of Health, will award approximately $20 million to four academic centers to launch a new national Career Development Consortium for Excellence in Glycosciences.

As part of the consortium, University of California San Diego will receive approximately $5 million over five years for its own Program for Career Development in Glycosciences. UC San Diego was also chosen to lead the consortium’s national administrative coordinating unit.

Ajit Varki, MD, Distinguished Professor of Cellular and Molecular Medicine, will lead the cross-disciplinary UC San Diego Program for Career Development in Glycosciences with Jeff Esko, PhD, Distinguished Professor of Cellular and Molecular Medicine, and Kamil Godula, PhD, assistant professor in the Department of Chemistry and Biochemistry.

“Our new training program is an attempt to correct [an] anomaly in the history of biomedical science by making glycoscience more accessible, transforming the field from a super-specialized research domain to an integrated part of mainstream biology,” said Esko, co-director of the Glycobiology Research and Training Center.
Why Humans Get Diarrhea and Other Mammals Don't
As an acute diarrheal disease, cholera is unique to humans. Its human specificity is a major world health problem — cholera affects between 1.4 and 4 million people annually, according to the World Health Organization. A recent report by researchers at the University of California San Diego School of Medicine may finally help explain this specificity — and the role of sialic acids.

The findings are published in the June 18 issue of PLOS Pathogens.

In their new paper, Ajit Varki, MD, Distinguished Professor of Medicine and Cellular and Molecular Medicine, Kim Barrett, PhD, Distinguished Professor of Medicine, and Victor Nizet, PhD, professor of pediatrics and pharmacy, have shown that this difference in sialic acid expression could explain why other animals do not suffer diarrhea when infected by the causative bacterium Vibrio cholerae.
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