Is this the fresh approach for curing uncommon childhood illnesses?
When life dealt a devastating blow to Fernando Goldsztein, he channeled his despair into relentless determination. His 12-year-old son had been diagnosed with a relapsing form of medulloblastoma, a rare and life-threatening brain tumor in children.
"The doctors' prognosis was bleak," Goldsztein recalls. "But I refused to accept it. I turned my fear into my greatest strength and fought back."
With a career in business and an education from MIT Sloan, Goldsztein had the skills to network and build connections. He applied these talents to an unexpected cause – saving his son and thousands of other children around the world.
In 2021, he launched The Medulloblastoma Initiative (MBI), a non-profit organization with an audacious mission: to find a cure for medulloblastoma using a groundbreaking new approach to funding rare disease research.
After raising $11 million in just 18 months, MBI brought together 14 of the world's top labs and hospitals, spanning North America, Europe, and Brazil. Two promising trials will start soon, and more are in the pipeline, awaiting approval from the U.S. Food and Drug Administration.
The industry of healthcare research is notorious for being bogged down by bureaucracy, with a typical timeline of seven to fifteen years from lab discovery to treatment. MBI's remarkable speed has the potential to disrupt the entire field, condensing the timeline significantly.
Crucially, MBI is focused on eliminating overhead and administrative costs, ensuring that 100% of the funding goes directly towards finding a cure. Unlike government research grants, which allocate only 4 cents on the dollar towards pediatric cancer research, MBI is laser-focused on making a meaningful impact.
"There is no time to waste," Goldsztein emphasizes. "We're not just doing science – we're making it move faster than it ever has."
MBI's innovative model of funding research could have far-reaching implications, speeding up the pace of research for rare diseases and transforming the way healthcare research is carried out.
Goldsztein's personal journey started when his son was initially diagnosed at nine years old. After a nine-hour brain surgery and standard treatment in the U.S., Goldsztein watched on helplessly as his son underwent radiation and nine rounds of chemotherapy. devastating side effects like cognitive problems, growth issues, and secondary tumors.
The standard treatment protocol has a success rate of 70%, but it leaves most children with lifelong effects. Frederico, unfortunately, was one of those who fell into the 30% category. After three years, his tumor relapsed.
Renowned doctor Roger Packer, who created the standard treatment years earlier, offered Goldsztein hope and partnership. Packer explained that the complexity of medulloblastoma has stymied research for 30 years, as medulloblastoma is an umbrella term for 13 different types of tumors. Each subtype requires unique treatments, making research difficult unless researchers focus on a specific tumor type.
Goldsztein seized the opportunity to partner with Packer, launching the Cure Group 4 Consortium, a collaborative effort involving 14 leading institutions from around the world. Their goal? To develop effective therapies for Group 4, the most common and least-understood subtype of medulloblastoma, within a two-year timeline.
Unlike traditional research collaborations, MBI's open-source consortium model demands transparency and cooperation. Researchers must share data freely to accelerate collective progress, a revolutionary strategy welcomed by MBI's partners.
Supporting MBI in this unprecedented endeavor is friend Mariano Gargiulo, who met Goldsztein during their time at MIT Sloan. Inspired by Goldsztein's unyielding optimism and ambitious timeline, Gargiulo has been a crucial contributor to the organization's success.
Frederico, who defied expectations and graduated high school, has ambitions of his own. Passionate about photography, he remains a source of hope and inspiration for Goldsztein.
When asked about the potential impact of MBI's success, Goldsztein is unequivocal: "We're going to find the cure. It's not a matter of if – it's when."
With MBI's relentless pursuit of progress, it seems that "when" could be closer than we imagine.
- Goldsztein's son's diagnosis with medulloblastoma, a rare and life-threatening brain tumor, prompted a switch in focus from business to health.
- Recognizing the gravity of the situation, Goldsztein mobilized his network and skills from his MIT Sloan education to save his son and other children worldwide.
- In 2021, he launched The Medulloblastoma Initiative (MBI), aiming to find a cure for medulloblastoma using a groundbreaking new approach to funding research.
- MBI's research resolves to conquer neurological disorders, health-and-wellness challenges, and medical-conditions like cancer, with a focus on children and rare diseases.
- The organization brings together 14 top labs and hospitals globally, collaborating on two promising trials to treat medulloblastoma, with more in the pipeline.
- MBI's innovative funding model aims to minimizing overhead and administrative costs, ensuring that all funding goes directly towards finding a cure.
- Unlike traditional government grants, MBI is laser-focused on making a meaningful impact in pediatric cancer research, with the goal of drastically reducing the timeline from lab discovery to treatment.
- With MBI's groundbreaking approach to research, the future of healthcare may see faster progress in understanding and treating various health conditions, starting with medulloblastoma.
