FSU chemist receives more than $500,000 grant to explore existing medicines for new treatments for pediatric brain cancer
Tallahassee, Florida – A Florida State University researcher is taking a different approach to the search for new cancer treatments, looking at medicines that already exist rather than starting from scratch with entirely new drugs.
Qing-Xiang “Amy” Sang, the Diane and Michael Bruton Professor for Cancer Research in FSU’s Department of Chemistry and Biochemistry, has received more than $500,000 from the Florida Cancer Innovation Fund. The grant will support research aimed at finding existing medicines that could potentially be used to treat rare brain cancers in children.
Sang plans to combine artificial intelligence with laboratory testing. The first step will involve using AI to examine a huge amount of existing scientific information and identify drugs that may have potential against specific types of brain cancer. Promising candidates will then be tested in the laboratory to see how they interact with tumor cells at the molecular level.
The approach could help researchers look at cancer treatments in a different way. Instead of developing a completely new medicine, scientists can examine drugs that have already been studied and, in some cases, approved for other diseases.
“Brain cancers are among the deadliest and hardest-to-treat cancers in both children and adults,” Sang said. “There are many types of brain cancers, so a drug that works well against one may do nothing against another. However, different tumors often share hidden similarities in their underlying biology. This grant will help us examine drugs that are already known to work against certain cancers and test whether they can also treat different, less-studied types of brain cancer.”
The research will focus particularly on rare pediatric tumors, including atypical teratoid rhabdoid tumors and diffuse intrinsic pontine glioma. Both are uncommon brain cancers affecting children and are currently considered incurable.
There is a large amount of information that could potentially help researchers understand these diseases. Nearly 600 cancer-fighting drugs are currently on the market, while millions of scientific findings have been published over the years.
Between 2010 and 2019 alone, more than 1 million cancer-related scientific publications were produced around the world. Those studies contain information about cancer biology, how drugs interact with tumors and how patients respond to different treatments.
The challenge is finding useful connections within all that information.
For a single scientist or even a research team, reviewing such a massive body of work and identifying patterns across different cancer types would be extremely difficult. Sang’s project will use AI as a tool to help narrow the field.
The technology will be used to analyze available data and identify which medicines appear to have the strongest potential against particular brain cancers. Researchers can then focus their laboratory work on the drugs that appear most promising.
Searching beyond traditional cancer drugs
Sang’s research will not be limited to medicines that were originally developed to fight cancer.
The project will also examine drugs approved by the U.S. Food and Drug Administration for other medical conditions. Such drugs may have biological effects that could make them useful against cancer even though cancer was not their original purpose.
One well-known example is tamoxifen. The drug was initially approved as a contraceptive medication before researchers discovered that it could be used to treat breast cancer and reduce the risk of tumors developing in some high-risk patients.
Sang hopes to find similar opportunities by comparing the biological features of different tumors.
“By identifying molecular similarities among tumors, we can make educated, data-driven hypotheses about which existing drugs might work in a new biological setting,” said Sang, who is also affiliated with FSU’s Institute of Molecular Biophysics. “We will take our top candidates for novel brain cancer treatments into the lab and test them directly on brain tumor cells to demonstrate that our approach works in practice, not just theory.”
The laboratory portion of the project will use patient-derived organoids. These are three-dimensional clusters of cells that can be grown in the laboratory and provide a model that more closely resembles the structure and behavior of an actual tumor than a traditional flat layer of cells.
Sang will use the organoids to study how potential treatments interact with brain tumor cells.
The use of these types of human-cell models also reflects a broader change in biomedical research. The U.S. Food and Drug Administration has shown a preference for computer-based tools and realistic human-cell models when they can be used effectively, as researchers look for alternatives to some forms of animal testing.
A potential shortcut in drug development
Finding a new cancer drug from the beginning can take years of research and testing. Repurposing a medicine that has already been approved or has already reached early clinical trials can offer researchers a different path.
Sang said that could be particularly useful for rare cancers, where the number of potential patients may be relatively small.
“Because we research drugs that are already approved or have gone through early-stage clinical trials, the odds of successfully moving them toward new patients are much higher than trying to build a brand-new compound,” Sang said. “This is especially important for rare brain cancers, which often attract little interest from pharmaceutical companies simply because the number of patients is small and potential profit is limited.”
Although the project has a strong focus on pediatric brain cancers, Sang’s research also includes more common brain tumors, including glioblastoma.
Brain tumors make up less than 2% of all new cancer cases in the United States, according to the National Foundation for Cancer Research. That is considerably smaller than the share represented by some of the country’s more common cancers, such as prostate and breast cancer.
The smaller number of cases can create additional challenges for research and drug development, particularly for rare pediatric tumors.
Sang’s project is supported by the Florida Cancer Innovation Fund, which was created in 2024 under the Florida Department of Health. The fund is designed to encourage collaboration between scientists and oncologists while supporting new approaches to cancer research and treatment.
FSU officials say the grant also reflects the university’s broader research goals.
“Dr. Sang is a highly regarded biochemist with remarkable creativity, persistence, and a research program yielding unique insights,” said Wei Yang, Department of Chemistry and Biochemistry chair. “Her new grant is also a testimony to the advancement of A Strategic Plan to Inspire Research Excellence, or the FSU ASPIRE mission, developed to identify strategic areas of focus and investment, ensuring the sustained growth and impact of FSU’s research enterprise, particularly in the area of drug discovery.”
The project is also connected to FSU Health, the university’s academic health system, which brings together hospitals, physicians, clinics, research, education and innovation.
For Sang, the ultimate goal is practical. The research is designed to identify treatments that could eventually help patients who currently have few options.
“Our goal is to extend the lives of cancer patients, enhance quality of life and when possible, provide a cure,” Sang said. “I hope that in our efforts to repurpose existing drugs, we have a realistic shot at finding effective treatments for difficult brain cancers in a quicker timeframe.”
The grant gives Sang and her team the opportunity to combine two rapidly developing areas of research — artificial intelligence and human-cell-based laboratory models — with medicines that are already known to scientists.
Rather than searching through the enormous universe of possible new compounds, the team will begin with drugs that already have a history of medical use. AI will help identify potential connections, while laboratory testing will determine whether those predictions hold up when the drugs are tested against actual tumor models.
The research will not guarantee that any particular medicine will become a treatment for pediatric brain cancer. But it provides a structured way to search for possibilities that might otherwise remain hidden within the enormous amount of existing cancer research.
For children and families facing rare brain cancers, finding those overlooked possibilities could be an important step toward expanding the number of treatments available in the future.
