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developmental therapeutics

Hematology and Oncology

Finding Novel Treatments for Cancer

The Developmental Therapeutics team at Children’s of Alabama is working to develop new treatments for children with cancer.

Having cancer is challenging enough for a child and their family, but when the standard treatments aren’t working, they find themselves searching for answers. As Director of Developmental Therapeutics at Children’s of Alabama and the University of Alabama at Birmingham (UAB), Jamie Aye, M.D., is responsible for finding those answers.

Aye was appointed to the position in August 2025 after serving as associate director since 2023. In her current role, she leads a team dedicated to developing more effective, less toxic therapies for children with cancer. In some cases, the cancers are rare; in other cases, the cancers are recurrent with no available curative treatment. Either way, developmental therapeutics provides hope for solutions.

“It provides the family with hope that we will discover newer options that their cancer hasn’t seen,” said Aye, who’s also an associate professor of Pediatrics at UAB.

The team achieves this through a two-pronged strategy. One prong involves working to develop new therapies for cases that are recurrent or difficult to treat. “It’s really hard to see a patient go through that,” Aye said. The other prong is developing less-toxic therapies that reduce immediate and long-term side effects—for example, “If we’re able to use a new immunotherapy in conjunction with radiation that allows us to give the radiation at a lower dose,” Aye said, “now that patient has a lower risk for a secondary cancer from radiation.”

The program has a history of success. A few years ago, Gregory Friedman, M.D., one of Aye’s predecessors, led a successful early phase trial that used a new type of immunotherapy to treat patients with recurrent or progressive brain tumors.  Friedman’s novel treatment was the result of an investigator-initiated trial, where the team takes the work its researchers are doing and turns it into a clinical trial. Developing these types of trials is the overarching goal of the program. And the research avenues are broad, Aye says, “from researchers within pharmacology, radiology, immunology or genetics,” to name a few. Aye works to bring these researchers together “so that hopefully we can see commonalities between the different projects that we’re working on, so that people aren’t siloed and can collaborate through either new technologies, new biomarkers to measure how much disease, new immunotherapies, or new ways of detecting disease by imaging,” she said.

Any type of research the team is doing has the potential to turn into an effective treatment. “It could even just be a pathway a researcher is investigating,” Aye said. “Hopefully through collaborations that we’re establishing, we can see how that pathway may relate to a particular cancer that we’re hoping to find a treatment for—and then from that, test a new therapy, and then if it works in the lab, seeing how we can get it to a patient.”

Collaborations are one of the keys to making these trials happen. As director of the program, Aye works to build relationships both near and far. Locally, she leads an annual research retreat to bring together researchers—both basic science and clinical researchers—to work on developing investigator-initiated trials using novel therapies. She developed this retreat during her time as associate director of the program. Nationally, she works with industry and pharmaceutical companies—to investigate new therapeutic options—and with consortia to add to the program’s portfolio of trials available to patients at Children’s and UAB.

Aye is already working toward more investigator initiated trials, “which, hopefully in the coming years, will come to fruition,” she said, to provide more options and more hope for children with cancer. “It’s why we do what we do,” she added. “Because we want to improve their outcomes and also find treatments that don’t leave them with lifelong side effects.”

Hematology and Oncology

Developmental Therapeutics Offers Next Generation of Cancer Medicines

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One of the most devastating things a doctor has to do is inform a family that their child with cancer has run out of options. At Children’s of Alabama, however, clinicians often have one more thing to offer: access to early-stage clinical trials for potentially lifesaving drugs through its Developmental Therapeutics Program.

“Development therapeutics is about the next generation of medicines that will hopefully improve outcomes and reduce side effects for children with cancer,” Program Director Gregory Friedman, M.D., said.

Children’s is able to provide access to investigational compounds because it is one of just 21 pediatric oncology programs in the country invited to participate in the Children’s Oncology Group Phase 1 and Pilot Consortium. Consortium studies, Friedman said, “are how we’ve improved survival rates in recent years.”

Studies offered through the Developmental Therapeutics Program are generally Phase 1 or Phase 2 trials designed to demonstrate proof of concept, safety, tolerability and efficacy of new compounds. Currently, the program is managing more than 40 such studies.

Friedman is also developing a translational program to quickly move investigator-initiated research from laboratories at the University of Alabama at Birmingham (UAB) and Children’s into the clinical setting by providing an interface between the investigators and regulatory bodies, including the Food and Drug Administration. In addition, the Developmental Therapeutics team works with industry to develop new agents and is exploring participation in disease-specific pediatric consortiums.

Through the Developmental Therapeutics Program, Children’s also participates in the NCI-COG Pediatric MATCH trial, the largest precision medicine trial of its kind, in which tumor tissue is sent to the NCI for molecular testing to identify genetic abnormalities driving cancer growth. The goal is to then identify investigational or existing drugs to target those abnormalities and conduct clinical trials of the agents.

Friedman stresses that it takes a team to run the program including other physicians, a nurse manager, a clinical trial specialist, a data information coordinator and regulatory experts.

The goal, he said, is to continue to grow the program. “Our ultimate goal is to have a trial available for every child and family in these very difficult situations where few treatment options exist,” he said.

Viral Therapy in Malignant Brain Tumors Offers Hope

Developmental Therapeutics Program Director Gregory Friedman, M.D., is the principal investigator on one of the most exciting studies underway in pediatric cancer: evaluating the use of a cold sore virus to treat children with progressive malignant brain tumors. The approach is based on the finding that pediatric brain tumors are highly sensitive to genetically engineered oncolytic HSV-1 G207, which lacks genes essential for replication in the normal brain. The Phase 1 trial is designed to evaluate the safety, tolerability and preliminary efficacy of G207 delivered directly into the brain via intratumoral catheters.

Friedman and his team presented preliminary results on the treatments effects during the International Symposium of Pediatric Neuro-Oncology Meeting in Denver in June 2018. There were no dose-limiting toxicities or serious adverse events related to the virus and five of the six patients who received treatment demonstrated positive responses. One patient is now more than 21 months out from the treatment with a significant improvement in their well-being.

Since then, at least three other children have been treated safely with evidence of favorable drug activity. The next phase of the study will test the treatment’s safety in combination with a single dose of radiation, which is designed to enhance an immune response against the tumor.

Cancer Fighters
Learn more about the Alabama Center for Childhood Cancer and Blood Disorders. www.childrensal.org/cancer.