Clinical Trial for DIPG Offers Hope for Children with Brain Cancer

By Doured Daghistani, MD, and Jorge E. Perez, MD

Brain cancer is the second most common cancer among children after leukemia. But while new therapies have significantly increased the cure rate of pediatric leukemia, effective treatment for brain cancer still lags.

I am currently conducting a pioneer phase I clinical trial called ONC201 at the Miami Cancer Institute (MCI) testing a new targeted therapy for brain tumors. I am hopeful that this new drug will become the go-to treatment for cancers such as diffuse intrinsic pontine glioma (DIPG), a cancer that currently has no long-term survival rate. This is one of the hundreds of clinical trials for cancer in which we’ve enrolled patients for the past three decades.


I joined the Children’s Oncology Group (COG) in 1996 to find cures for pediatric cancer working with experts worldwide. The COG is supported by the National Cancer Institute and COG researchers have been instrumental in numerous advances.

Doured Daghistani, MD

Through COG’s multiple clinical trials, acute lymphocytic leukemia (ALL) in children saw an increase in survivorship from 60 to 90 percent. Only 50 years ago, a diagnosis of ALL was a death sentence. The next target is to get the same results – or better – for brain cancer, DIPG and other pediatric cancers.

DIPG – Uniquely Difficult to Treat

In children with DIPG, the average survival is six months to 18 months after diagnosis. DIPG cannot be treated with surgery because the tumor is located where breathing and heart function are controlled. Removing the tumor would cause catastrophic injury to these vital centers.

The Miami Cancer Institute, where I serve as Medical Director of Pediatric Oncology, is one of five centers in the U.S. doing a trial with ONC201, a new generation of chemotherapy called “targeted therapy”.

We know our children are tolerating this new drug very well but it is too early to have any conclusion on the impact of the drug on survival. At some point, we may be able to combine ONC201 with chemotherapy or immunotherapy for a more robust response.

Jorge E. Perez, MD

While the children are monitored regularly in my office at MCI, the medication is given once a week by pill or via compounded liquid for younger children. The first dose is given under our supervision at MCI, after which the children return once every three weeks for assessment. There are currently 12 children participating in the trial, ranging in age from 5 years old to adolescents.

All of the Institute’s pediatric patients are under my care, including the majority of those receiving proton therapy. We regularly attract children from throughout Florida and other countries looking for our expertise in diagnosing and treating cancers like leukemia, brain tumors, kidney cancer and bone tumors, among others.

Regional Collaboration Offers Children More Opportunities

One of our goals is to expand COG treatment opportunities to patients of the other KIDZ Medical Services hematology/oncology offices at Nicklaus Children’s Hospital, Palm Beach Children’s Hospital at St. Mary’s Medical Center, West Boca Medical Center in Boca Raton and Naples Community Hospital.

The dedicated KIDZ Medical Services hematology/oncology researchers, physicians, and other healthcare staff have, for decades, been involved in ground-breaking research to improve cancer survival by offering patients the most advanced treatments, such as with proton beam therapy, and opportunities to participate in clinical trials.

Keys to the medical advancements are multispecialty collaboration, medical expertise, participation in the Children’s Oncology Group research and a deeply-rooted commitment – some might say calling – to increase survivability for pediatric cancer patients.

Research is the engine of everything and as long as we have hope in our heart and work hard on doing research and supporting research, we will beat cancer one day.

Dr. Doured Daghistani is Medical Director of Pediatric Oncology at Miami Cancer Institute. Dr. Jorge E. Perez is a Neonatologist and Co-founder of KIDZ Medical Services.