The key to any translational medicine initiative is to make sure the advances of the laboratory are finding their way into the lives of patients.

UCSF already has world-class research, along with top-notch patient care and educational programs in the health sciences. Biotechnology and pharmaceutical companies are setting up offices near the UCSF Mission Bay campus to work with UCSF labs on practical applications of new discoveries. The University’s Clinical and Translational Science Institute (CTSI) is a well-funded initiative bringing it all together. 

Mark Laret, chief executive officer, UCSF Medical Center

This effort will accelerate in 2014 with the opening of the new UCSF Medical Center at Mission Bay.

“What UCSF is particularly good at doing – and I think is our opportunity, particularly at Mission Bay – is the partnership of the research scientists and our clinicians with industry,” says Mark Laret, chief executive officer of UCSF Medical Center and UCSF Benioff Children’s Hospital. “It’s really the three legs of that stool that make for a great relationship and that are going to be the key to bringing discovery into common use.”

Two Key Steps

Translational medicine involves two key steps, according to S. Claiborne Johnston, MD, PhD, associate vice chancellor of research and director of CTSI. The first phase centers on moving discoveries from the bench to the bedside, that is, from labs to humans, usually in government-approved clinical trials. The second phase is equally critical, he says: advancing drugs, devices and therapies out of clinical trials and implementing and disseminating them into the wider world.

Many innovative partnerships at UCSF already exist, thanks to the early successes of facilities such as CTSI and the California Institute for Quantitative Biosciences (QB3), and the recent arrival of pharmaceutical industry heavyweights such as Bayer, Celgene and Merck in the Mission Bay neighborhood.

From left, Laura J. van ‘t Veer, PhD, professor of laboratory medicine and a leader in the Breast Oncology Program, and Laura J. Esserman, MD, MBA, professor of surgery and radiology, who directs the Carol Franc Buck Breast Care Center

The new UCSF Medical Center at Mission Bay will be a 289-bed complex with specialty hospitals for children, women and cancer patients.

“This, in a way, completes the loop and makes us a whole campus at Mission Bay – not just research, but research tied to clinical care,” Laret says. “That is what we think is the greatest potential at Mission Bay and the most important reason to build these specialty hospitals.”

Learning from Patient Care

Another key component of the translational medicine movement, according to Johnston at CTSI, is to enable researchers “to easily learn from patient care. The ability to learn from our errors and our successes has to be accelerated.”

One example, Johnston says, is by modernizing patients’ electronic health records and, with permission and in an appropriate manner, making them accessible in straightforward ways to researchers, so they can see the effects of their work in actual patients.

“If you get that system well greased, all parties can learn much more quickly,” Johnston says. “The cycles of innovation are much shorter.” 

Translational Medicine Well Underway

Numerous translational medicine projects geared to moving basic research more quickly from the laboratory to clinical trials and patient treatment are already underway.  Examples include:  

Robert V. Farese Jr., MD, an investigator with the UCSF-affiliated Gladstone Institute of Cardiovascular Disease, with postdoctoral researcher observing Drosophila. 

Alzheimer’s and dementia: In a major UCSF Alzheimer’s Disease Research Center endeavor, supported by the National Institutes of Health (NIH), UCSF faculty across all UCSF sites and affiliates are working to accelerate the pace of discovery regarding dementia diseases. Clinical researchers who diagnose and treat patients with dementia are collaborating with basic science researchers studying Alzheimer’s and related diseases in cell and animal models.

Neurologist William Seeley, MD, evaluates and treats patients with dementia and directs a laboratory focused on what makes brain tissues susceptible or resistant to degeneration. His ultimate goal is to translate these findings into novel treatments.

In one study published in the April 16, 2009, issue of the journal Neuron, Seeley and his colleagues reported strong evidence that neurodegenerative diseases target distinct neural networks that normally support healthy brain function. The discovery could help lead to earlier diagnoses, novel treatment monitoring strategies and, possibly, recognition of a common disease process among all forms of neurodegeneration.

Seeley also directs a neurodegenerative disease brain bank, which supports research by storing and distributing brain tissues from patients who have died of Alzheimer’s disease, frontotemporal dementia (FTD) and related disorders.

Robert V. Farese Jr., MD, an investigator with the UCSF-affiliated Gladstone Institute of Cardiovascular Disease, heads a lab working with the Consortium for Frontotemporal Dementia Research, a UCSF-based group of about a dozen laboratories. The group includes both basic science and clinical researchers, and is dedicated to finding therapies for FTD within 10 years. FTD is a devastating neurological disease and the second most common cause of dementia in patients under age 65.

Cancer: The UCSF Helen Diller Family Comprehensive Cancer Center, an interdisciplinary program that combines basic science, clinical research, patient care and epidemiology throughout UCSF, epitomizes the capacity for translational medicine.

Cancer Center programs in prostate cancer, breast cancer and brain tumors are supported by Specialized Program of Research Excellence (SPORE) grants from NIH. These grants are highly competitive and designed to drive cross-pollination of ideas between researchers in basic science and clinical science to improve cancer treatment, early detection and prevention.

Cancer specialist Laura van ’t Veer, PhD, is collaborating with Joe Gray, PhD, and Laura Esserman, MD, MBA, leaders of the Cancer Center’s Breast Oncology Program, on new opportunities to apply knowledge of genetic abnormalities and cancer molecular markers to benefit patients.

One project is focusing on genetic patterns in tumors that can help predict the combinations of standard or experimental drugs that may best target each tumor. Another project involves a major, unique clinical trial that the researchers hope will lead to better understanding of how early genetic and imaging measurements can be used to direct patients to treatments that are predicted to work best for them individually.

In previous work, van ’t Veer led bench-to-bedside development of the MammaPrint, a “ lab test on a chip” that detects patterns of gene activity in tissue samples prepared from breast tumors, indicating the likelihood of the return of cancer despite surgery to remove the tumor.

Imaging:  On the Mission Bay campus, a long term collaboration between UCSF and GE Healthcare has focused on developing novel magnetic resonance imaging technologies. In November 2010, this relationship led to the first use in humans of a new technology that monitors changes in hyperpolarized pyruvate, a naturally occurring sugar that cells produce during metabolism, to rapidly assess the aggressiveness of a tumor by imaging the metabolism.  The technique has the potential for dramatically changing treatment for many types of tumors by providing immediate feedback to clinicians on whether a therapy is working.

Sarah Nelson, PhD, director of the Surbeck Laboratory for Advanced Imaging, headed the collaborative research team.  

Synergy from Clinician-Research Teams 

The opening of UCSF Medical Center at Mission Bay will provide the most dramatic view of the way research and health care can interact. “To have that clinical facility right across the street from the research facilities will be fantastic,” UCSF Medical Center CEO Laret says. “Obviously, a lot of the research there will have an impact on patient care. There is a synergy you get from having clinicians and scientists working together.”