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Illustrating the potential impact of the CTSA Program: a closer look at the Common Metrics Initiative

September 19, 2018

Common Metrics Data

Patients vs. paywalls: Is the U.S. ready for open-access publishing?

September 14, 2018

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Latest News from Around the CTSA Program Consortium

Penn State CTSI welcomes new KL2 scholar

Penn State CTSI

Penn State Clinical and Translational Science Institute has welcomed a new scholar into its program that invests in early-career faculty. Dr. Steven Hicks, assistant professor of pediatrics, was named into the institute’s KL2 Training Program, which gives researchers establishing themselves in their fields dedicated time to help their findings benefit human health more quickly while becoming successful, independent translational scientists.

“As a KL2 scholar I’ll have the opportunity to expand my expertise in biostatistics and community engagement through protected career development time,” Hicks said. “My research project will engage stakeholders from the community, academia and industry to advance autism research here at Penn State.”

Hicks’ research looks to use saliva samples from hundreds of children with autism spectrum disorder to find RNA differences that could be used to diagnose the disorder.

Hicks joins seven other scholars who started in January after a competitive application process.

KL2 Scholar

September 19, 2018

Xu takes a closer look at stem cell cultures under the microscope.

Kathleen Ferraro/Chicago ITM

ITM Researcher Building First Treatment for Cocaine Addiction

Chicago Institute for Translational Medicine

What could be the first medical treatment for cocaine addiction is being created by Institute for Translational Medicine (ITM) researchers Ming Xu, PhD, and Xiaoyang Wu, PhD. Their research edits a patch of normal skin using an engineering technology that puts an anti-cocaine gene into skin stem cells. This system allows Xu and Wu to take a small section of skin, add the anti-cocaine gene to the skin’s cells, and then put it back onto the patient through a process called grafting, a routine procedure where a piece of skin is added onto the body.

The genetically-modified cells produce anti-cocaine proteins that enter the bloodstream and break down cocaine in a way that makes it unrewarding and nontoxic to the brain and body. And because the tool uses a routine skin grafting procedure, the process is safe, minimally invasive, and affordable.

The treatment could also scale to address other addictions, such as opioids, alcohol and cigarettes. With more than 15 million Americans suffering from alcoholism, and cigarette smoking accounting for one in every five deaths in the U.S., Xu and Wu’s treatment has the potential to help millions of patients.

September 18, 2018

Penn State

Sekhar begins Community-Engaged Research Fellowship

Penn State Clinical and Translational Science Institute

There’s a difference between simply including in a research study the people a scientist wants to help and partnering with those people in the research process. That’s a lesson Dr. Deepa Sekhar learned while studying ways to better detect high-frequency noise-induced hearing loss in teenagers. She will continue to learn how to more effectively accomplish the latter through the year-long, Community-Engaged Research Core Faculty Fellowship of Penn State Clinical and Translational Science Institute.

The Community-Engaged Research Core Faculty Fellowship Program matches a researcher with a mentor and protects work time for research – important for a busy clinician helping patients. Community-engaged research meets the needs of the community by involving those who have an interest in improving health. This can include a community and its members, patients, clinicians, researchers, purchasers, payers, industry, hospitals and health systems, training institutions and policy makers. Applications are now being accepted for next year’s program.

September 11, 2018

Photograph by Emma Vought

Leukogene Therapeutics receives funding to develop compound for resistant multiple myeloma

Medical University of South Carolina --South Carolina Clinical and Translational Research Institute

A $2 million phase 2 Small Business Technology Transfer (STTR) grant to optimize a promising new compound that has shown efficacy in preclinical studies against treatment-resistant multiple myeloma has been awarded to a Medical University of South Carolina (MUSC) startup company, Leukogene Therapeutics, Inc., in collaboration with MUSC researcher and company founder, Nathan G. Dolloff, Ph.D. Dolloff and his team will use the STTR award to further develop the new compound into a drug that could be used with proteasome inhibitors in treatment-resistant multiple myeloma.While proteasome inhibitors kill cancer cells by preventing the breakdown of proteins, Dolloff's compound targets instead their synthesis, preventing proper folding, which is essential to protein function. Normally, unfolded or misfolded proteins would then be targeted to the proteasome for degradation to avoid the build-up of these dysfunctional proteins. However, in the presence of proteasome inhibitors, the breakdown is blocked, leading to the build-up of toxic misfolded proteins.In principle, the compound developed by Dolloff could offer a one-two punch when administered together with proteasome inhibitors.

August 30, 2018

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