Sanford-Burnham selects organizations to participate in Florida Translational Research Program

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The collaborations are enabled by the Florida Translational Research Program, which provides Florida-based scientists with access to Sanford-Burnham's drug discovery technology and expertise.

Sanford-Burnham Medical Research Institute at Lake Nona (Sanford-Burnham) announced the selection of the first five research organizations that will participate in the Florida Translational Research Program (FTRP) to advance drug discovery in the state. The projects focus on cancer, diabetes, and obesity, and are led by scientists from the University of Central Florida, the University of Florida, the University of Miami, Scripps Florida, and Sanford-Burnham. The Program provides Florida-based scientists with access to drug discovery expertise and state-of-the-art infrastructure at Sanford-Burnham.

"We fielded approximately 50 specific inquiries for the program and received a total of 15 valid applications for five project slots," said Layton Smith, Ph.D., director of Drug Discovery Florida at Sanford-Burnham, who oversees the program. "We selected these projects based on scientific merit, technical feasibility, and potential for commercialization. Work on the first collaboration began in February and we expect to complete all projects by the end of the year."

Toward new cancer therapeutics

Three of the projects focus on discovering new treatments for cancer and other tumors. Claes Wahlestedt, M.D., Ph.D., and his team at the University of Miami are trying to find chemical compounds that stop MLL3, a protein that plays a central role in the development of several leukemias, as well as breast and colon cancers. Researchers led by Daiqing Liao, Ph.D., at the University of Florida have shown that a novel drug target for cancer, acetyltransferase p300, is a master regulator of cancer-cell survival. Novel inhibitors of p300 are thus expected to prevent the development of tumors in a variety of cancers. Cristina Fernandez-Valle, Ph.D., at the University of Central Florida and her team seek compounds that block the protein merlin, which has a similar function as p300.

While the research focus of each project varies greatly, all participating scientists agree that having access to state-of-the-art screening technology enhances their ability to do research. "The immediate and potential long-term impact of this funding on my research cannot be overstated," said UCF professor and research scientist Fernandez-Valle. "There is currently no drug treatment for neurofibromatosis, a devastating tumor syndrome diagnosed in children and young adults. The results of this project and the partnership with Sanford-Burnham are critical for us to successfully compete for the continuously diminishing federal funds that we need to eventually develop an effective drug therapy for neurofibromatosis."

On the road to treating diabetes and obesity

The other two projects represent the first steps in the discovery of new medicines to treat diabetes and obesity. Patricia McDonald, Ph.D., at Scripps Florida will collaborate with Sanford-Burnham to identify molecules that block the function of a protein called GPR21, which is known to reduce the effects of insulin on the body. Fraydoon Rastinejad, Ph.D., and his lab at Sanford-Burnham seek to block the action of two other proteins that control metabolism and the expansion of fat cells, Rev-Erbα and Rev-Erbβ.

The Florida Department of Health and Sanford-Burnham established the FTRP as a competitive grant program that provides funding for collaborative drug discovery projects. The overall goal of the program is to translate research discoveries made in Florida laboratories into the medicines of tomorrow.

"As a native Floridian, I am extremely proud to see the first FTRP projects come to life. There is no doubt we have the talent here in Florida to develop the medicines of tomorrow. Through the FTRP collaborations, we have the opportunity to match this talent with our infrastructure and technology platforms at Sanford-Burnham," says Smith.

This first year of the FTRP is a pilot phase with five project slots that were available; the number of projects is expected to increase next year. Applications were invited from investigators who had already developed innovative assays for use both in basic research and in therapeutic development, and who were interested in having their assay screened using Sanford-Burnham's small-molecule library. In the future, the program will accept proposals for more comprehensive projects that may have a chemical component, not yet be ready for high-throughput screening, or require assay development services from Sanford-Burnham.

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