Imagine receiving the devastating news that your child has been diagnosed with a rare and aggressive brain cancer. This is the heart-wrenching reality that Brazilian businessman Fernando Goldzstein faced when his nine-year-old son, Frederico, was diagnosed with medulloblastoma—a diagnosis that would not only change their lives forever but also spark a global movement. But here’s where it gets controversial: What if the treatments available for such cancers are decades old, toxic, and often ineffective? This is the stark truth Goldzstein uncovered, and it’s a reality that far too many families face in the rare disease community.
In the world of rare diseases, the term diagnostic odyssey describes the grueling journey families endure to identify and treat their loved one’s condition. For Goldzstein, this odyssey began on November 7, 2015, when Frederico’s diagnosis froze their world. “It’s very difficult to put into words what a parent feels in circumstances like this,” he shared. Determined to save his son, Goldzstein embarked on a relentless quest for answers, collaborating with leading institutions like Children’s National Hospital in Washington, D.C. His efforts not only led to innovative treatments for Frederico but also birthed the Medulloblastoma Initiative (MBI), a global organization dedicated to accelerating research and cures for rare pediatric cancers.
And this is the part most people miss: Despite advancements in biotech, treatments for childhood brain cancers like medulloblastoma remain shockingly outdated. “The treatment is from the ’80s,” Goldzstein revealed. “It’s toxic, ineffective, and leaves survivors with devastating side effects like stunted growth, cognitive issues, or even secondary tumors.” This glaring gap in medical progress is what drives the MBI’s mission to push for faster, safer, and more effective clinical trials.
On November 18, 2025, Goldzstein spoke with the National Press Foundation’s Rare Disease Reporting fellows, sharing his journey and emphasizing the urgent need for collaboration. “For rare disease parents, giving up is not an option,” he declared. His story highlights the resilience of families who refuse to accept hopeless diagnoses, even when faced with uncharted territory. “You’re in uncharted waters,” he explained. “There’s no proof the treatment will work, but you have no other choice.”
Goldzstein’s advocacy has already yielded remarkable results. Since its founding in 2021, the MBI has secured approval for two clinical trials in just two and a half years—a process that typically takes a decade. Philanthropy and partnerships with 12 U.S. research institutions, one in Canada, and one in Germany have been key to this success. “I never imagined MBI would become a global movement,” he admitted. With two more trials in the pipeline and ongoing treatment for children like Frederico, the initiative is making strides, though challenges remain.
Frederico, now a thriving teenager, defied the odds. “He’s a smart, curious kid who loves to travel and read,” Goldzstein proudly shared. “He’s been a warrior, and fighting pediatric brain tumors has become the goal of my life.” But not every family is as fortunate, which is why Goldzstein continues to push for systemic change.
Here’s the controversial question: Are we doing enough to prioritize research for rare pediatric cancers, or are we leaving these children behind? Goldzstein’s story challenges us to rethink our approach to rare disease treatment and invites us to join the fight. What do you think? Share your thoughts in the comments below.
Access the full transcript of Goldzstein’s session here. This fellowship is funded by Fondation Ipsen, with NPF solely responsible for the content.
