As each distinguished guest spoke at the Capitol Hill briefing event on September 8, 2016, presented by the Alliance for Childhood Cancer and The American Cancer Society, I couldn’t help but picture a racing ambulance, with red lights flashing, bullhorn blaring, and somebody yelling, “Help is on the Way!” The briefing outlined how we are chasing after better treatments for pediatric cancers, including brain tumors.
The event began with the nightmare story told by a grieving father, Carlos, about the loss of his young daughter Althea to childhood leukemia. Instead of referring to her medical care as treatment, he called it torture, a familiar feeling amongst the pediatric brain tumor community. Before I could quell and compose my emotions, the story grew worse, as he revealed the cancer diagnosis of his second child. There before me was a photo of a healthy, happy boy, about eight years old, posted next to the podium, and I hoped that as the story unfolded, I would learn that this boy, Phineas, was still here on earth…
Before I tell you more about Phineas’ story, I need to convey how excited and intrigued I am about the many R&D opportunities highlighted during the expert panel. New technologies and data are available to researchers, and new discoveries are happening all the time.
Take Dr. Malcolm Smith of the National Cancer Institute (NCI), for instance. He kicked off the panel presentation with the discussion of the upcoming NCI Pediatric MATCH Trial. Pediatric patients are eligible for the trial regardless of their tumor location. The focus of the trial is on the tumor’s genetic make-up and its irregularities and mutations. Each prospective patient’s tumor is analyzed and sequenced. If the abnormality is one of the mutations that might respond to one of the eight compounds in the study, the patient will be enrolled into the MATCH Trial. This approach of testing many compounds in one trial – sometimes called a “basket trial” – is a new approach for the NCI, and has the potential to end with eight new agents approved for different pediatric cancer indications. One of these agents could potentially be applied to a pediatric brain cancer sub-type.
Dr. Smith also outlined the 3 pediatric initiatives in the Cancer Moonshot effort, an initiative being led by Vice President Biden. David Arons, CEO of National Brain Tumor Society, is a member of the Blue Ribbon Panel, the advisory committee to the Moonshot. The Moonshot will include a provision for studying the basic science of how tumors form in children, advancing the work in pediatric immunotherapy, and developing new agents specifically designed for children. All of these initiatives may help pediatric brain tumor research.
And there was Dr. Martha Donoghue of the US Food and Drug Administration (FDA), who shared how the FDA is adapting two key pieces of federal policy – BPCA (Best Pharmaceuticals for Children Act) and PREA (Pediatric Research Equity Act) – guidelines to create market incentives for industry to conduct pediatric cancer studies of their agents. She also highlighted a hearing held the month before which encouraged doctors to include biopsy in the standard of care for a brainstem tumor (specifically, DIPG) diagnosis. These samples can be sequenced, and could lead to a better understanding and treatment of pediatric brain tumors.
Dr. Tahira Khan of Genentech also shared how her lab was also looking at trials based on molecular targets. She referenced Genentech’s MATRIX Trial which is based on genetic biomarkers. And Dr. Susan Blaney of Texas Children’s Cancer Center – who also happens to be on the National Brain Tumor Society Strategic Scientific Advisory Council for the Defeat Pediatric Brain Tumors Research Collaborative – reassured us that researchers are broadcasting the message that the pediatric population is not responding to adult cancer treatments. She made it clear that these treatments are extremely painful and debilitating, and that we need to move quicker than ever to identify and establish a standard of care and treatment for these kids. The word is getting out quickly and proactively – the deaths of these kids with pediatric brain tumors and other cancers are unacceptable, and we need to change this scenario now. Such a wonderful, encouraging and powerful message to send throughout the entire cancer community.
Now I get back to Carlos’ story. Phineas declined quickly, similar to his sister, but for Phineas, there was a new approach – a new treatment. His tumor was analyzed and sequenced with the very latest precision-medicine techniques. The Children’s Oncology Group, a clinical trial network funded by the NCI, had uncovered a new sub-type of his cancer. After discovering Phineas’ tumor would not respond to the current standard of care, they developed a personalized, precision-based treatment, and an alternative protocol was developed for Phineas. His “ambulance” reached him in time.
Help may FINALLY be on the way. Innovation and technology could truly lead to promising treatments for pediatric brain tumors in the future.