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Kids Connection
March 2007
Children's Research Institute News Brief

Dr. Maria
Bernard L. Maria, MD, MBA
Executive Director
Darby Children's
Research Inst.
Inderjit Singh, PhD
Inderjit Singh, PhD
Scientific Director
Darby Children's
Research Inst.


Researchers win sought-after CTSA Awards

Three cross-disciplinary pilot projects involving DCRI investigators have won highly coveted CTSA awards.

The awards are funded by a CTSA internal institution grant, explains Dr. Kathleen Brady, director of the General Clinical Research Center and part of a committee that selected the winning studies.

Of nearly 100 applications, the committee chose to fund nine pilot projects, all of which were translational and collaborative in nature.

That three of the CTSA awardees have come out of the DCRI is pretty amazing, says Dr. Bernie Maria, executive director.

"For an institute that's been operating for just two years, we effectively competed for these awards," he notes. "That really says something about the performance of this institute."

The three winning DCRI projects were:
  1. the development of a new oral cancer screening method to improve early detection of oral cancer;
  2. the use of brain positron emission tomography (PET to predict atypical response to antipsychotic drugs;
  3. the use of hyaluronic acid oligomers (o-HA) in malignant gliomas to treat human brain tumors.
"We're supposed to be thinking creatively, out-of-the-box about ways that we can transform the system to facilitate research," says Dr. Brady. "To make it as easy as possible, to break down barriers to improve communications between basic and clinical scientists.

"We've awarded pilot project money to research projects that are designed to catalyze new collaborations, which is part of the criteria required to receive CTSA funds," she continues.

The novelty of the oral cancer screening study, spearheaded by the Clemson bioengineering program and principal investigator Dr. Bruce Gao, is its use of a laser to produce a holographic optical configuration, thereby avoiding tissue slice preparation and cell staining.

"Because oral lesions are being seen in younger and younger ages because of chewing tobacco, this research is significant for children's health," says Dr. Maria of the DCRI.

The project aims to translate an optical technique already developed in the team's lab into a practical application for oral cancer detection, explains Dr. Hai Yao of the DCRI Clemson bioengineering program, who is a collaborator on the project.

"The advanced optical technique already exists but the application is unique," says Dr. Yao. The team hopes to ultimately create a hand-held optical-biopsy tool for oral cancer screening.

"Our goal is to develop a novel instrument with clinical applications," he explains. "The project is multi-disciplinary, combining engineering and biomedical research. It involves researchers from the engineering, dental and cell biology departments from both Clemson University and MUSC."

It's a project that could have broad impact. "This technique could be used to detect many other kinds of cancer, too."

Also highly translational and innovative, the PET/antipsychotic drug study uses brain imaging in a new way, says principal investigator Dr. Jun-Sheng Wang.

"Right now, we have no means to study drug concentrations in the human brain," he explains. "The PET technique will show us exactly what happens after patients receive the commonly used antipsychotic drug, risperidone (which was recently approved for use in children). This will help predict clinical response and effective dosages of the drug." The team's long-term goal is preventing drug resistance in patients with schizophrenia.

"We now have evidence that the drug transporter protein, P-glycoprotein, acts as a gatekeeper, preventing antipsychotic drugs from entering the central nervous system," says Dr. Wang. "Within a few years, we hope to find a novel therapeutic solution to overcome this P-glycoprotein gate keeping activity."

"This is fundamental work, tied to the transport of drugs in and out of the brain," explains Dr. Maria. "Its implications for children center around the placenta, with preventing drugs from crossing the placenta into the fetal brain. It's pre-birth children's research."

The research team includes members from the departments of psychiatry, radiology, the Clemson-MUSC Bioengineering Program, pharmacy and surgery.

The third CTSA pilot project targets brain tumors, which are the leading cause of death from disease in children.

"We are developing a new treatment that antagonizes hyaluronic acid, which cells use to promote malignant properties," explains Dr. Maria, a pediatric neuro-oncologist and principal investigator of the study. "We are developing new oligomers, never used in humans before, to control the growth of brain and spinal cord tumors. It's a new biologic therapy that manipulates Mother Nature."

The departments of cell biology and anatomy, pharmacology and pharmaceutical sciences are working together on the project. "It's a multi-departmental and intercollegiate project between the Colleges of Medicine and Pharmacy," notes Dr. Maria.

The team expects to take the study to clinical trials within the next year or two. "It could be effective against a variety of cancers particularly since preliminary studies show that the oligomers potentiate the effects of radiotherapy and chemotherapy," says Dr. Maria.

All nine winning pilot projects reflect a direction and attitude crucial for transition to a CTSA.

"The CTSA planning grant we received from the NIH provides funding for us to plan the submission of a very large infrastructure grant designed to provide much broader support for clinical and translational research at MUSC than we have previously had," explains Dr. Brady. "These nine pilot projects are part of that transformation in our approach to research."

DCRI Second Anniversary


Dr. Pai, Dr. Maria, Dr. Johnston (Johns Hopkins University), and Dr. Singh




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