Collaborative projects aim to accelerate new treatments for heart failure, macular degeneration, skin inflammation, lung cancer and Pompe disease
Five investigators at Duke received $100,000 each in May from the Duke Translational Research Institute (DTRI) to accelerate projects from preclinical to Phase I clinical trials.
DTRI awards these collaborative pilot funds to projects that link basic lab and clinical science investigators. The five projects funded in May 2014 aim to:
- Develop a home test that monitors a biomarker in the blood and allows patients with chronic heart failure to upload results to their health record via smart phone so physicians can adjust medication levels. (Ashutosh Chilkoti)
- Improve vision and slow down the macular degeneration disease process through a new class of drugs that block growth of unwanted blood vessels in the eye. (Scott Cousins)
- Improve the lives of those living with Pompe disease by increasing the effectiveness and decreasing cost of current enzyme replacement therapy. (Dwight Koeberl)
- Explore a new class of compounds that can be applied to the skin to treat itchiness and inflammation. (Wolfgang Liedtke)
- Optimize the best option for the first human-derived antibodies used to fight lung tumors. (Edward Patz)
Dwight Koeberl, MD, PhD
Dwight Koeberl, MD, PhD, associate professor in the departments of Pediatrics and Molecular Genetics and Microbiology, believes collaboration between clinicians and researchers is vital
to moving possible treatments forward. As he investigates new therapies for Pompe disease, collaboration with Dr. Nenad Bursac in Biomedical Engineering allows his team to culture muscle cells from patients and evaluate treatments in the laboratory. The goal is to improve efficacy and reduce the cost of the life-long enzyme replacement therapy required by patients with Pompe disease, a muscle-wasting disease.
“The neat thing about this project is we can now provide muscle biopsies to Dr. Bursac, and his team can culture them, form muscle bundles, and test new drugs with actual patient muscles,” Koeberl said.
Ashutosh Chilkoti, PhD
Another form of collaboration will take place between the departments of Biomedical Engineering and Medicine-Cardiology. Ashutosh Chilkoti, PhD, is collaborating with Kristin Newby, MD, MHS to improve the lives of chronic heart failure patients. They are making it easier to track the levels of a biomarker hormone that the FDA has approved as a diagnostic tool for this condition. The team is building an assay tool on a small chip that patients can use to test a finger-prick of blood. Patients will be able to upload an image of the assay result to their electronic health record via a smart phone app.
Chilkoti appreciates that the pilot funding includes research study support from DTRI, including regulatory support for working with the FDA and also “providing technical support as we try to integrate this data into the electronic health record,” he said.
Edward Patz, Jr, MD
Edward Patz, Jr, MD, professor of Radiology and professor in the department of Pharmacology and Cancer Biology, will be working with faculty and staff from the Human Vaccine Institute, where his team has already developed the first human-derived antibody against lung cancer tumors. Patz will use the DTRI pilot funds to determine in animal studies which antibodies are most effective at inhibiting tumor growth. At the same time, his team will study human cells to define factors that would predispose patients to benefit from the antibody treatments.
“This is a new approach,” Patz said. “It is a fresh look to actively use the human immune system to treat cancer.”
Wolfgang Liedtke, MD, PhD
Wolfgang Liedtke, MD, PhD, an associate professor in the department of Neurology, is partnering with dermatologists Drs. Russ Hall and Jennifer Zhang to improve the lives of people who have painful and itchy skin.
“100 million people in the U.S. and Europe are affected by skin conditions related to inflammation, allergy, itch and pain, and available medical options are not always satisfactory,” Liedtke said. Liedtke
and his collaborators are working to fill the gap in the availability of safe and effective skin treatments by creating a topical medicine that can dial down the inflammation, pain and itch signals sent by skin cells.
Scott Cousins, MD
Scott Cousins, MD, a professor in the departments of Ophthalmology and Immunology, is bringing together colleagues from ophthalmology, pharmacology and chemistry with overlapping expertise to study possible molecules that may reduce unwanted growth of blood vessels in the eye. This would not only benefit patients with macular degeneration patients receiving currently approved therapies, but also patients with other back of the eye diseases.
“This same class of medication could be useful in other diseases, all of which share similar inflammatory components,” Cousins said.
The primary source of funding for the DTRI Collaborative Awards is from Duke’s Clinical and Translational Science Award (CTSA), funded by the National Institutes of Health’s National Center for Advancing Translational Sciences (NCATS). DTRI is a component of the Duke Translational Medicine Institute.
Victoria Christian, Chief Operating Officer of DTRI, said pilot awards are tangible evidence of the impact of Duke’s CTSA. The applicants receive feedback about their projects from people who may not have been aware of their research prior to the submission. In addition, awardees and collaborators receive financial, project management and regulatory support from DTRI. Reviewers at DTRI create
portfolios of research and development projects and nurture teams by tapping into their experience and networks, which helps Duke’s research efforts become better known within Duke and within NIH.
“We hope these pilot funding programs make the benefits of Duke’s CTSA grant experiential for the research community,” said Christian, “especially at a time when workloads are heavy and pay lines are tight.”
For more information about the DTRI Supplemental Awards, visit the DTMI website.