Unlocking the Secrets to Treating Rare Childhood Disorders: A revolutionary new model for funding research, The Medulloblastoma Initiative (MBI), aims to shorten the timeline from lab discovery to patient treatment from seven to 15 years to just two years.
Curing Rare Childhood Diseases: A New Model for Funding Research
When Fernando Goldsztein’s son received a devastating diagnosis, he knew he had to act fast. The 12-year-old boy was diagnosed with relapsing medulloblastoma, a life-threatening pediatric brain tumor. Despite the bleak prognosis, Goldsztein refused to give up hope.
The Birth of a New Initiative
Goldsztein’s experience as an MBA student at MIT Sloan School of Management taught him the importance of networking and building relationships. He began reaching out to top medulloblastoma doctors and scientists around the world, asking for their help in finding a cure for his son’s disease.
In 2021, Goldsztein launched The Medulloblastoma Initiative (MBI), a nonprofit organization dedicated to finding a cure using a revolutionary new model for funding rare disease research. With a focus on collaboration and efficiency, MBI aims to shorten the timeline from lab discovery to patient treatment from seven to 15 years to just two years.
A Consortium Model
The key to MBI’s success lies in its open-source consortium model. By bringing together leading institutions from around the world, Goldsztein has created a network of researchers working towards a common goal. Each lab is assigned a specific question to answer and tasked with developing a therapy within a two-year timeline.
This approach is not only innovative but also unprecedented in the highly competitive field of medical research. The consortium model allows for data sharing and collaboration among labs, accelerating progress and reducing duplication of effort.
A New Way of Funding Research
MBI’s funding model is designed to be efficient and effective. Unlike traditional government grants, which allocate just 4 cents on the dollar towards pediatric cancer research, MBI focuses 100% of its funding on a singular goal: finding a cure for medulloblastoma. With no overhead or administrative costs, MBI is able to direct its resources directly towards research.
A Matter of When, Not If
Goldsztein’s determination and leadership have inspired a community of families affected by rare childhood diseases. With the support of his network of over 70 families, Goldsztein is confident that MBI will find a cure for medulloblastoma.
When asked about the impact of MBI’s success, Goldsztein shakes his head. “We are going to find the cure,” he says. “It’s not if, it’s a matter of when.”
Scaling Up and Replicating Success
Goldsztein’s next goal is to scale up MBI and make its playbook available for other childhood diseases. By sharing his model with others, Goldsztein hopes to create a ripple effect in the medical research community, leading to faster and more effective treatments for rare diseases.
As Goldsztein looks to the future, he remains committed to finding a cure for medulloblastoma. With MBI’s innovative approach and dedication to collaboration, it’s clear that this is not just a matter of hope – but a reality within reach.
