“We have no treatments for your son. There’s nothing we can do.”
Hearing those devastating words shattered Fernando Goldsztein MBA ’03’s world.
“I refused to accept the doctors’ prognosis. I turned my fear into action and began to fight.”
Fernando’s 12-year-old son, Frederico, was diagnosed with relapsing medulloblastoma, a severe pediatric brain cancer. In that moment, Goldsztein’s life trajectory shifted dramatically, requiring him to become a different type of leader.
While he never envisioned becoming a founder, his experience at the MIT Sloan School of Management emphasized the value of building relationships and networking. He began leveraging these skills to reach out to top experts and researchers in medulloblastoma across the globe, seeking assistance.
“I knew I had to act, not just for Frederico, but for the approximately 15,000 children diagnosed with this disease each year worldwide,” he explains.
In 2021, Goldsztein established The Medulloblastoma Initiative (MBI), a nonprofit focused on accelerating research to find a cure through an innovative funding model for rare diseases.
In just 18 months, MBI — still in its startup phase — has raised an impressive $11 million in private funding, uniting 14 esteemed laboratories and hospitals from North America, Europe, and Brazil.
Looking ahead, two promising clinical trials are set to commence soon, with three additional trials awaiting approval from the U.S. Food and Drug Administration.
This progress comes in an industry often burdened by bureaucratic hurdles, where it usually takes seven to 15 years for a lab discovery to reach patients.
Conventional government research grants allocate a mere 4 cents of every dollar toward pediatric cancer research — funds typically spread thinly across multiple projects with no coordinated direction. In stark contrast, MBI channels 100 percent of its funding toward a unified mission without incurring overhead costs.
“Time is of the essence,” Goldsztein asserts. “We’re propelling scientific advancement faster than ever.”
The MBI approach to funding solutions for rare diseases holds the potential to disrupt the traditional funding landscape in healthcare research by significantly reducing development timelines.
Transforming Despair into Strength
Frederico’s journey began with a brain surgery lasting nine hours at age nine, followed by standard treatment protocols in the U.S. These included radiation and nine intense rounds of chemotherapy. This protocol, established back in the 1980s, cures 70% of children; however, it often leaves lasting effects such as cognitive impairments and secondary tumors. Regrettably, Frederico was among those who relapsed just three years later.
Goldsztein recalls with a grimace the bleak prognosis they received. “It was shocking to realize almost no advancements had been made in 40 years,” he reflects.
Hope arrived through a collaboration with Dr. Roger Packer, director of the Brain Tumor Institute at Children’s National Hospital. Packer, the architect of the treatment protocol, explained the complexity of developing effective therapies for medulloblastoma, which encompasses 13 tumor types. Frederico suffers from Group 4, the most prevalent subtype, yet the one least understood.
Recent advancements in molecular genetic testing have opened new doors for treatment development. “Before, we couldn’t distinguish these tumors well enough,” Packer says. “The landscape is changing now.”
The challenge lies in the fact that few researchers concentrated their efforts on Group 4. Goldsztein posed a pivotal question to Packer: “If I can secure funding, how quickly could your lab push forward medulloblastoma research?”
Creating an Open-Source Consortium
Packer suggested a novel approach: establishing a consortium among leading research institutions focused on medulloblastoma, where each lab would address a specific question about Group 4 tumors. Their projected timeline? Not the usual seven to 10 years from discovery to patient treatment — but a groundbreaking two years.
Seven labs agreed to join initially; now, the Cure Group 4 Consortium boasts 14 participants, including prominent institutions like Children’s National Hospital and SickKids.
To join this consortium, labs must adhere to unique guidelines: no redundant work and no protective silos exist. Goldsztein explains, “Everyone contributes a crucial piece, facilitating rapid progress. This collaborative spirit is our model’s core strength.”
Drawing inspiration from MIT’s open-source philosophy, researchers share their data openly, accelerating collective advancements. Such cross-institutional collaboration is rare in this highly competitive field.
Mariano Gargiulo MBA ’03, who met Goldsztein during orientation at their MIT Sloan Fellows MBA program, quickly became a close ally and early supporter of MBI. An executive in Houston’s energy sector, Gargiulo noted the uniqueness of Goldsztein’s two-year goal. “In a sea of standard timelines, his focus on rapid benchmarks set a bold precedent.”
Goldsztein’s ambitions gained traction when Children’s National Hospital recognized MBI’s potential in 2023, inviting him to join its foundation’s board, which enhanced his fundraising opportunities.
According to Packer, MBI’s first two clinical trials will soon reach patients: one focusing on immunotherapy to target cancer cells safely and effectively, and the second a medulloblastoma vaccine inspired by methodologies from COVID-19 vaccine development — a versatile treatment strategy with global applicability.
Ensuring a Brighter Future
When not advocating for MBI or spending time with his family in Brazil, Goldsztein connects with over 70 other families dealing with relapsed medulloblastoma through Zoom. “I’m not a doctor, and I don’t give medical advice, but these trials provide us with hope,” he shares.
Hope and purpose fuel Goldsztein. “I can’t fathom dedicating oneself to business success without giving back,” he expressed at his 2023 MIT Sloan Reunion, instilling motivation in his peers.
Remarkably, Frederico, who has faced his battles against recurrence, recently graduated high school. He’s found passion in photography and international relations. “This is about finding a cure for Frederico and all children affected by this disease,” Goldsztein emphasizes.
When asked about the implications of MBI discovering a medulloblastoma cure, Goldsztein confidently replies, “It’s not a question of if, but when.”
His next ambition is to expand MBI, providing a framework for organizations aiming to tackle other pediatric diseases using their methodology.
“I will never give up,” he vows.
Photo credit & article inspired by: Massachusetts Institute of Technology
