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.


Surprise! Medical Research a Big Winner in New Federal Budget
Despite the president’s threats to cut funding, Congress approved budget increases for cancer, Alzheimer’s, and other medical research.

Scientists who rely on NIH for funding can exhale and get to work, at least for now.

“Overall, the increase is good news, it is very welcome news, because as always, the cost of disease far outweighs the amount we invest to treat disease,” Lawrence Goldstein, PhD, director of the University of California San Diego Stem Cell Program and scientific director of the Sanford Consortium for Regenerative Medicine, told Healthline.
Jekyll and Hyde and Seek
Writing in the February 27 online issue of Science Signaling , researchers at University of California San Diego School of Medicine and Moores Cancer Center describe how a signaling protein that normally suppresses tumors can be manipulated (or re-programmed) by growth factors, turning it into a driver of malignant growth and metastasis.

In the lives of cells, complex communications carrying proteins and other molecules along signaling pathways dictate cellular function and well-being. But in cancer cells, communications are often massively dysregulated. “Although the multiple signaling pathways in cells are typically conceptualized as independent entities, it is their complex crosstalk that shapes many aspects of cancer,” said senior author Pradipta Ghosh, MD, professor of medicine and cellular and molecular medicine at UC San Diego School of Medicine.
Repurposed Drug Found to be Effective against Zika Virus
In both cell cultures and mouse models, a drug used to treat Hepatitis C effectively protected and rescued neural cells infected by the Zika virus — and blocked transmission of the virus to mouse fetuses.

Writing in the current online issue of the journal Scientific Reports , researchers at University of California San Diego School of Medicine, with colleagues in Brazil and elsewhere, say their findings support further investigation of using the repurposed drug as a potential treatment for Zika-infected adults, including pregnant women.

“There has been a lot of work done in the past year or so to address the Zika health threat. Much of it has focused on developing a vaccine, with promising early results,” said senior author Alysson Muotri, PhD, professor in the UC San Diego School of Medicine departments of Pediatrics and Cellular and Molecular Medicine, director of the UC San Diego Stem Cell Program and a member of the Sanford Consortium for Regenerative Medicine.
Knowing How Your Neurons Respond to Stress Could Help Diagnose and Treat ALS
Gene Yeo, PhD, professor of cellular and molecular medicine at UC San Diego School of Medicine, and his team, published a paper in Cell today that describes stress granules and the role they play in neurodegenerative diseases, such as ALS. Their findings may provide a new way to diagnose the disease long before the onset of symptoms, and offer new drug targets for treating the disease. Yeo and first author of the study, postdoctoral fellow Sebastian Markmiller, PhD, explain their findings in the linked Q&A.
Lab-grown neurons showcase effects of autism mutations
Neurons derived from people with mutations linked to autism display diverse abnormalities that may help explain the origins of these individuals’ features, according to three new studies.

In all three studies, researchers reprogrammed skin cells from individuals with one of these mutations into stem cells that can mature into any cell type. They then turned these induced pluripotent stem (iPS) cells into neurons.

The studies also illustrate the promise of the iPS cell approach for understanding how genetic mutations lead to functional impairments, says Alysson Muotri, PhD, professor of pediatrics and of cellular and molecular medicine at the University of California, San Diego, who was not involved in the study. “This is a nice representation of the type of work that’s possible,” he says.
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