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Clinical & Translational Science Institute / Stories / September 2017 / Past CTSI Pilot Awardee Lands in PLOS ONE’s Top 25%

Past CTSI Pilot Awardee Lands in PLOS ONE’s Top 25%

RNA word cloudA bladder cancer study, funded in part by the UR CTSI, is one of the most cited papers published by PLOS ONE in 2016. The study, led by Carla Beckham, M.D., Ph.D., assistant professor of Urology at the University of Rochester Medical Center and Wilmot Cancer Institute, has been cited over 6,000 times landing it among the journal’s top 25 percent.

The study, which was published as a result of a UR CTSI Faculty Pilot Award project, suggested that a type of RNA molecule may serve as a biomarker - and possibly a treatment target - for bladder cancer, the fifth most common cancer and the most expensive to treat over a patient’s lifetime. This could help patients get treatment earlier, prevent cancer progression, contain the cost of treatment, and lead to new medical breakthroughs in the lab.

“We set out to find markers of tumor progression in the urine of bladder cancer patients that could help advance new chemotherapy research,” said Beckham, who treats patients for bladder cancer and other urological diseases, and conducts research in RNA biology. “We focused on small membrane bound vesicles called exosomes, which facilitate tumor progression. We wanted to know which RNA are important in tumor progression and may be present in exosomes.”

With funding from the UR CTSI, Beckham sequenced RNA from urinary exosomes, tumors, and healthy tissue of patients with bladder cancer, as well as from exosomes of healthy age-matched donors. She found that a long non-coding RNA (lncRNA) known as HOTAIR, is enriched in the urinary exosomes of patients with high-grade, muscle-invading bladder cancer. 

HOTAIR, which has previously been shown to play a role in tumor initiation and progression in many cancers, could also be a good target for bladder cancer treatment. When Beckham and her team knocked out expression of the HOTAIR gene in bladder cancer cells in the lab, those cells lost their invasive traits.

Beckham cautions that a single molecule is not likely to be an effective biomarker on its own or lead to a silver bullet treatment for bladder cancer. Her team identified four additional lncRNAs in urinary exosomes from bladder cancer patients that could be included with HOTAIR as part of a panel of biomarkers for the disease. However, further investigation in larger patient populations will be necessary before this panel can be used to identify and treat patients.

To read Beckham’s full study in PLOS One, click here.

The project described in this article was supported by a CTSI Pilot Award through the University of Rochester CTSA award (UL1TR000042) from the National Center for Advancing Translational Sciences of the National Institutes of Health. The CTSI Pilot Studies Program provides seed funding for highly innovative translational and clinical research that addresses translational research questions, and provides insights for future projects.

Read more about the Pilot Studies Program, and check out a list of our current and past awardees.

Michael Hazard | 9/7/2017

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