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Grants support immunotherapy-based treatment for childhood cancer

Dr. William Decker, left, and Dr. Cliona Rooney, right.

Two Baylor College of Medicine researchers have received grants from the nonprofit organization Alex’s Lemonade Stand Foundation to advance their immunotherapy-based treatment approaches for childhood cancer from the bench to bedside.

Dr. William Decker, assistant professor of immunology at Baylor, and Dr. Cliona Rooney, professor in the Center for Cell and Therapy at Baylor, Texas Children’s Hospital and Houston Methodist, are two of a dozen researchers nationwide to earn the foundation’s new Reach Awards, which are designed to overcome barriers that impede the translation of innovative pediatric oncology research ideas from the lab to the clinic. The Reach Awards provide $250,000 over two years.

Decker’s research focuses on the development of a novel immunotherapeutic treatment approach for a type of brain cancer called Atypical Teratoid/Rhabdoid Tumor. Patients with this aggressive tumor have a poor survival rate, and current treatments can lead to significant neurocognitive side effects.

“There is a clear medical need for the development of more efficacious and less toxic treatment regimens for AT/RT and other pediatric brain tumors. Treatments should be able to both effectively fight cancer while sparing neurocognitive function,” Decker said.

Decker and his research colleagues propose using an immunotherapy approach that they have shown to be safe and potentially effective in brain cancer lab models. The treatment uses the patient's own blood cells and own tumor cells to generate a powerful vaccine that can attack the tumor while sparing normal tissue. Funding of this project will enable his research group to generate the necessary data to allow a clinical trial to proceed, Decker noted.

Rooney’s research also involves an immunotherapy-based approach. The goal is to develop potent immune-based therapies for high-risk neuroblastoma, a form of cancer that begins in childhood. Special immune system T-cells that have been modified to home in on a protein found on neuroblastoma cells can potentially eliminate tumors without causing toxic side effects or long lasting disability. Although these modified T-cells have successfully eliminated small tumors, eradication of large tumors or tumors that have spread throughout the body remains challenging.

To overcome the limitations associated with current immunotherapy-based approaches, Rooney has developed a novel approach in which she modifies T-cells with a form of vaccinia virus that attacks tumors such as neuroblastoma. The vaccine strain of vaccinia virus can be used both to kill tumor cells as well as to boost the function and numbers of the gene-modified vaccinia-specific T-cells. This cutting edge strategy should facilitate the treatment of bulky tumors and prolong the duration of T-cell activity after administration. Using the Alex’s Lemonade Stand Reach Award will allow Rooney and her colleagues will conduct studies in murine models of neuroblastoma to prepare for a trial in children with relapsed, high-risk neuroblastoma.

Rooney is also a professor of pediatrics – hematology/oncology at Baylor and Texas Children’s Cancer Center.

“We know firsthand how important these research trials are to bettering the lives of childhood cancer patients, and we are dedicated to bringing promising research from the lab to the clinic,” said Jay Scott, co-executive director of Alex’s Lemonade Stand Foundation, which was founded after his daughter, Alex, herself a cancer patient, held a lemonade stand to raise money for a cure for cancer.