UCSF has a long history of leadership in neuroscience research, including a Nobel Prize. In 2010, it signaled its intention to maintain that leadership when it announced construction of a new neurosciences building at the UCSF Mission Bay campus.

“The building will bring together under one roof all of the elements needed for truly translational research to prevent, treat and cure the pervasive neurological diseases and disorders that afflict individuals and families,” says Stephen Hauser, MD, chair of the Department of Neurology.

Adam Gazzaley, MD, PhD

Adam Gazzaley, MD, PhD

The new Neurosciences Laboratory and Clinical Research Building will foster collaboration among some of the world’s top researchers: from the UCSF Department of Neurology, UCSF Institute for Neurogenerative Diseases (IND), W.M. Keck Foundation Center for Integrative Neuroscience, and UCSF Memory and Aging Center. Moreover, when the building is complete in 2012, it will be only steps away from the Helen Diller Family Cancer Research Building, which includes neuro-oncology, neuroscience and neurosurgical researchers.

“The opportunity for major progress is tremendous,” says Stanley Prusiner, MD, director of the IND and winner of the 1997 Nobel Prize in Physiology or Medicine for discovering an entirely new class of disease-causing proteins, called prions, that lead to neurodegeneration. Much evidence argues that prion-like proteins feature in Alzheimer’s disease and Parkinson’s disease.

Understanding the Aging Brain

As the building takes shape, UCSF basic science and clinical researchers continue their wide-ranging assault on neurologic disorders. Some examples:

In 2010, Bruce Miller, MD, and Lennart Mucke, MD, received the Potamkin Prize for Research, the most prestigious prize for Alzheimer’s disease research in the world. Miller directs the UCSF Memory and Aging Center, while Mucke directs the Gladstone Institute of Neurological Disease, part of the J. David Gladstone Institutes, an independent, nonprofit biomedical research institution affiliated with UCSF.

“Drs. Miller and Mucke exemplify the University’s bench-to-bedside ethic in their studies and treatment of these complex neurodegenerative disorders,” says Hauser.

Adam Gazzaley, MD, PhD, director of the Neuroscience Imaging Center at UCSF, complements their work. His lab combines behavioral assessments with advanced imaging techniques to shed light on the ways in which memory and attention change with normal aging and dementia – and how to intervene therapeutically to alleviate deficits.

Gazzaley’s lab was the first to prove that practicing simple visual tasks can improve the accuracy of short-term visual memory. The research, which also measured mental performance and changes in neural activity, confirmed that the brains of older adults can change in response to focused training.

Treating Brain Tumors

If work at the UCSF Brain Tumor Research Center – part of the UCSF Brain Tumor Center – is successful, the brain may also be able to change its response to tumor growth. Using a $19.1 million grant from the California Institute for Regenerative Medicine, a group led by Berger is refining a strategy it successfully used on rodents to target glioblastoma multiforme, the most aggressive form of brain tumor, with genetically engineered stem cells.

“If the FDA approves this work for humans, it would be an important advance in treating brain tumors of all kinds,” says Berger.

UCSF neurosurgeon Andrew Parsa, MD, PhD, mines a similar vein. He has developed an experimental therapy for glioblastoma multiforme that could enable physicians to manage the disease with a personalized vaccine. After resection of a patient's tumor, Parsa uses heat shock proteins from the  tumor to provoke an immune response specific to the cancer cells. In early tests, the work appears to extend patient survival time.

The Division of Neuro-Oncology at UCSF is internationally recognized for its ability to design and execute cutting-edge clinical trials for newly diagnosed and recurrent brain tumors. About 200 patients are enrolled in neuro-oncology clinical trials at UCSF each year.

As a member of two multi-institutional organizations funded by the National Cancer Institute (NCI) – the Adult Brain Tumor Consortium and the Pediatric Brain Tumor Consortium – UCSF is able to give patients access to the most advanced treatments emerging from research. These consortia are funded by the NCI and support multidisciplinary clinical trials using surgery, radiation and systemic therapies.

The Brain Tumor Research Center is also one of only four centers nationwide to receive a Specialized Program of Research Excellence (SPORE) award from the NCI that specifically funds translational research for brain cancer. This program supports projects examining the epidemiology of the disease, neuroimaging advances, new targeted therapies and small molecule inhibitors, and immunotherapy. The SPORE award also supports junior investigators performing research ranging from stem cell studies to quality-of-life initiatives.

Multiple Sclerosis

Hauser and his Multiple Sclerosis Research Group have long been leaders in understanding the causes of, and advancing the therapeutic options for, the devastating symptoms of the disease.

Recently, Hauser’s group conceived a study that became the most advanced genomic analysis ever conducted on identical twins, in which one sibling has multiple sclerosis and the other does not. The work earned the cover story in the April 29, 2010, issue of Nature.

“The results put us a step closer to teasing out the relative contributions of genetic and environmental factors to multiple sclerosis,” says UCSF researcher Sergio Baranzini, PhD, the study’s lead author.

Understanding Headaches, Epilepsy and Beyond

Severe headaches afflict millions worldwide, but science has struggled to understand them. The work of Peter Goadsby, MD, PhD, who directs the UCSF Headache Center, is an exception. He led the first controlled trial to definitively show that self-administered, high-flow inhaled oxygen at the onset of a cluster headache provides substantial pain relief. And Goadsby’s pioneering basic science discoveries have set the stage for creation of a novel medication for migraine with aura, a condition characterized by a variety of mostly visual disturbances.

In epilepsy research, neurologist Dan Lowenstein, MD, is the driving force behind the national, NIH-sponsored Epilepsy Phenome/Genome Project. With UCSF as the coordinating center, the project collects detailed information on patients with specific types of epilepsy to identify causes of the disease and better understand which drugs will work best for particular patients.

Andrew Parsa, MD, PhD

Andrew Parsa, MD, PhD

The Department of Neurological Surgery is also completing a number of important clinical trials, including one that examines whether Gamma Knife radiosurgery for mesial temporal sclerosis – a loss of neurons and scarring of the brain’s temporal lobe – is a viable, noninvasive alternative to traditional surgery.

Similarly, UCSF research on stroke prevention and treatment continues to lead advances for the third-leading cause of death in the United States.  One team, for example, is overseeing clinical trials on the Merci Retriever which takes its name from mechanical embolus removal in cerebral ischemia.  Ischemia is the condition of decreased blood supply to the brain, and this treatment is designed to extend the treatment window for stroke.  UCSF also has helped develop ways for physicians to stratify risk when treating transient ischemic attack (TIA), a warning sign for stroke. 

Other prominent research evaluates the best treatments for traumatic brain injury and examines how to mitigate the long-term effects of concussion, the latter being done in conjunction with the National Football League.

Research reaches across all UCSF schools. The UCSF School of Nursing recently teamed with Red Hill Studios, a video game developer, to demonstrate the use of inexpensive gaming equipment to create therapeutic games. The games are being designed to improve the gait and balance of people with Parkinson’s and to improve motor control in children with cerebral palsy.

The list goes on, and the new neurosciences building at Mission Bay exemplifies UCSF’s commitment to maintaining leadership in neuroscience research and care. Scheduled to open in 2012, the facility will bring together under one roof basic science and clinical research programs seeking cures for intractable neurological disorders.  According to UCSF Chancellor Susan Desmond-Hellmann, MD, MPH, “This approach will accelerate research discoveries and drive them toward patient care.”