Hematology and Oncology

Developmental Therapeutics Offers Next Generation of Cancer Medicines


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.

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