CTSI Research Award Helps Move Promise to Product at UCSF

What if testing for the disabling schistosomiasis parasite that plagues many developing countries was as simple as testing for pregnancy with an over-the-counter dipstick?

Researchers at UCSF have been pursuing these “dreams” for years. Now, they’re getting help to take the crucial steps closer to “real world” applicability thanks to an award offered by the Clinical and Translational Science Institute (CTSI), UCSF’s National Institutes of Health-funded program that helps accelerate research to improve health.

The T1 Translational Catalyst Award, first offered in 2010 and modeled off a successful award presented by the UCSF-affiliated J. David Gladstone Institutes, is designed to help drive promising, early-stage research through the lengthy and complex process connecting academia to medicines, tools or procedures that benefit patients. In the translational science spectrum, “T1” represents early translational research that has not yet been tested on humans.

While the award provides funding of up to $100,000, awardees note that what’s potentially even more helpful is the support offered from legal, financial, and regulatory knowledge experts in medical and health product development.

The granting process is phased, and although not all projects make it to the final level, each selected investigator receives some degree of individualized consultation.

“From past participants, we hear the real value is in the customized support that many receive during the course of the program participation,” said June H. Lee, MD, former associate director of Early Clinical Development at Genentech and now director of Early Translational Research for the CTSI and associate professor of pulmonary and critical care medicine.

“We are connecting outstanding UCSF scientists with the rich resources and expertise both in academia and industry to move science and inventions closer to becoming drugs, therapeutics or products that can improve health.”

Diverse Projects Share Potential for Real-World Solutions

So far, the T1 Catalyst Award has been given to a multidisciplinary group of UCSF investigators working on diverse issues. What they share is a pragmatic vision that might have large scale potential. These scientists talk of the exhilaration of being on the threshold of marketplace viability, and of the caution of practicing good science. Most are still relatively early in the process of product viability: testing, financing, patenting and licensing.

Take, for example, Conor Caffrey, PhD, UCSF associate researcher in the Department of Pathology, and scientist at UCSF’s Sandler Center for Drug Discovery. He is an expert in schistosomiasis, a chronic and morbid parasitic infection that is transmitted through contaminated water.

Caffrey, who is collaborating with colleagues at Stanford and UCSF’s Sandler Center for Drug Discovery, is trying to develop a quick, minimally invasive test for the condition, which responds well to medication.

The current practice of microscopically screening stool or urine for parasite eggs is limited by the unpredictable reproductive cycle of the worms, he said. A person can be infected, without a clear presence of eggs. It can also take days to get results from this method, which is not always convenient in the remote villages of Africa or Southeast Asia where the infection is widespread.

“We’re dealing with diseases of the very poor,” Caffrey said. “This isn’t like picking up a kit at your local drugstore.”

Caffrey’s T1 Catalyst Award is helping him to bioengineer a dipstick-type urine or blood test that changes color when it detects a protein unique to schistosomiasis. The award is giving him knowledge in financing and marketing, primarily from his main consultant, Russell Bromley, a businessman with expertise in innovative medical technologies. Bromley is connecting him with experts whose wisdom includes the major challenges of developing products for consumers who cannot pay for them.

Sports medicine expert and UCSF associate professor in the Department of Orthopaedic Surgery Anthony Luke, MD, MPH, talks of a similar synergy with his primary T1 Catalyst consultant, Peter Berger, an attorney, entrepreneur and software developer with a background in biotech. “Peter understands the gaming world, and the new technologies that could really help, and he brings a lot of expertise to planning the next steps,” Luke said.

Luke is the founder of RunSafe, an injury prevention clinic operated by the UCSF Department of Orthopedic Surgery that uses 2-D video techniques to assist runners and provide prevention counseling. The images allow specialists to analyze the gait, posture and other body mechanics of runners, observing patterns that may cause injury.

With his T1 Catalyst Award, Luke is ramping up efforts to find the most accurate, cost-effective, and easy-to-use body imaging techniques to advance what is used by RunSafe. His goal is a system that is easily replicated at multiple sites.

Luke has compared 2-D with 3-D imaging, and is scouring other technologies with his eye on the R&D hotbeds of the gaming industry, especially the motion sensor systems found on the Microsoft Kinect and Wii.

A major frustration for heart and lung doctors, blood clots typically cannot be detected until they are large and deadly. Severe symptoms, usually shortness of breath combined with risk factors such as obesity or smoking, are common emergency room triggers prompting doctors to look for clots. The most common technique is invasive angiogram X-ray.

Michael Page, PhD, UCSF assistant researcher in the lab of Charles Craik, professor of pharmaceutical chemistry, is also on the trail of finding a better way to detect blood clots. Page has invented something he calls CardioPaint, a peptide-based probe that detects small, early blood clots when injected in mice. The probe is activated by thrombin, a protein emitted in clotting. The new method shows clotting in real time, allowing doctors to evaluate immediate risks.

Page, who said he is already on the radar of big pharmaceutical companies, is using his T1 Catalyst Award funding to narrow down which imaging method gets the best results. He is also expanding his investigation to clots associated with cancers and infectious disease.

Page’s aim is to eventually test the approach in humans. His T1 Catalyst Award consultants include a Food an Drug Administration specialist, a venture capitalist and a cardiologist. “They each had different skills sets that were important and useful. I’m definitely picking their brains,” Page said.

In a classic example of a mutually beneficial collaboration, consultants teamed with grant recipients report being thrilled by their role. "It has been gratifying to work with young investigators early in their careers who are so focused and passionate about making a very concrete contribution in a challenging area of global health," Russell Bromely said.

Anthony Luke Treadmill photo by Mike Kennedy, Anthony Luke headshot photo by the RCL photo group, all other photos by Susan Merrell.

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