At the Forefront of Pediatric Brain Tumor Therapy

One of the advantages of a leading-edge Brain Tumor Program, like the one at Texas Children’s Hospital, is the ability to offer patients the expertise of outstanding pediatric subspecialists at every point on their journey. Texas Children’s Cancer and Hematology Center, home to one of the largest brain tumor programs in the country, has expertise in treating children with both common, as well as rare, pediatric brain tumors. Physician-scientists in the Brain Tumor Program are committed to increasing cure rates and decreasing the short- and long-term side effects of currently available treatments for children with brain tumors. They are nationally and internationally recognized for working to achieve these goals and are on the forefront of translating their most promising research discoveries into innovative therapies, including clinical trials of novel targeted drugs or cellular immunotherapies. 

At the forefront of diagnosis and treatment 

The Brain Tumor Program, led by co-Directors Murali Chintagumpala, MD, and Will Parsons, MD, PhD, is a multidisciplinary team comprised of pediatric-trained experts in cancer genetics and genomics, neuro-oncology, radiation oncology, neurology, neurosurgery, neuropathology, and neuropsychology, neuro-ophthalmology, physical therapy, endocrinology and more. This team, which is rounded out by social workers, child-life workers, pharmacists, nurses and others, delivers comprehensive cutting-edge care that is tailored to improving the outcome and quality of life for each child.  

Historically, little was known about the biology of some types of brain tumors, such as those occurring in the brainstem, because it was difficult to safely remove the tumor – let alone biopsy it.  

“With advances in surgical and genomic technology, we can now safely determine the tumor’s molecular make-up, The goal of this molecular analysis is to more precisely diagnose the tumor so that we can tailor treatment recommendations to maximize their therapeutic potential, while simultaneously reducing treatment-related toxicities and the potential for treatment-related late effects,” Dr. Chintagumpala said.“Each individual tumor is different. Based on the results of sophisticated genomic testing, we can target our treatment for the individual patient.”  

Another treatment modality that’s delivered in a targeted manner is the use of radiation therapy. Proton radiation therapy delivers the appropriate dose to the delineated target while sparing the surrounding normal brain tissue. This characteristic contributes to the amelioration of long-term neurocognitive and neuroendocrine deficits that patients may experience with conventional radiation therapy.  

“Results of research led by neuropsychologist, Dr Lisa Kahalley, a key member of our team, showed that the neurocognitive effects of proton therapy were lessened in patients with medulloblastoma compared with conventional radiation,” Dr. Chintagumpala said. “This is critical information since our focus is not only on providing curative treatments, but also treatments with the potential for fewer short-term or long-term side effects.” 

Novel clinical trials  

Unfortunately, there has been little to no improvement in the outcomes for some types of pediatric brain tumors over the past several decades. Children with high-grade gliomas or diffuse intrinsic pontine gliomas (DIPG), a subtype of diffuse midline gliomas, have a dismal prognosis despite treatment with radiation therapy. At Texas Children’s Cancer Center, investigators in our Cell and Gene Therapy Program are developing promising new immunotherapies for these patients. For example, Dr. Omar Bilal has developed a chimeric antigen receptor (CAR) T cell that recognizes GD2 antigen-expressing tumors such as DIPGs. The addition of C7R to the CAR T cells provides a constant supply of cytokines that allows the cells to survive and recognize tumors for a longer duration. This trial is actively accruing patients, and preliminary results are encouraging.  

Dr. Meena Hegde and her team have developed a trial for children with another type of childhood brain tumor known as ependymoma. These tumors commonly express the HER2 antigen. A clinical trial for HER2 CAR T cells was developed based on promising preclinical data from Dr. Hegde’s laboratory. This FDA-approved trial is also enrolling children who have other recurrent or high-risk brain tumors that express HER2. We hope that these novel cell therapy strategies, when combined with conventional cancer therapies, will lead to increased cure rates for some of the most challenging to treat pediatric brain tumors. 

“The availability of novel clinical trials such as these at Texas Children’s Cancer Center allow us to offer innovative frontline therapies to patients who would otherwise not be able to access them,” Dr. Chintagumpala said.  

Texas Children’s is a founding member of the NCI-funded Pediatric Brain Tumor Consortium, a founding member of the Southern Pediatric Neuro-Oncology Consortium, a member of the Children’s Oncology Group (COG), and a member of NCI’s Pediatric Early Phase Clinical Trial Network. Our membership and leadership activities in these consortia also provide our patients with access to additional innovative clinical trials. 

Children treated in our Brain Tumor Program are seamlessly followed by experts in the Long-Term Survivor Program once they are three years from their initial diagnosis. The Long-Term Survivor team, in collaboration with the patient’s primary neuro-oncologist, continues to monitor and provide survivors with individualized recommendations for monitoring and care throughout their entire life. 

For referring providers 

Whether patients self-refer for a second opinion or are referred by their oncology team, the Texas Children’s team works collaboratively with colleagues around the country for the benefit of their patients.  

“We encourage physicians to reach out with questions or for a consultation,” said Dr. Chintagumpala, “and we will rapidly respond.” 

Learn more about the Brain Tumor Program at Texas Children’s or call 1-800-226-2379 with questions or to discuss a patient case.