“There isn’t a remedy out there in your son. We are able to’t do something to assist him.”
When Fernando Goldsztein MBA ’03 heard these phrases, one thing inside him snapped.
“I refused to simply accept what the medical doctors have been saying. I reworked my concern into my best energy and began preventing.”
Goldsztein’s 12-year-old son Frederico was recognized with relapsing medulloblastoma, a life-threatening pediatric mind tumor. Goldsztein’s life — and profession plan — modified instantly. He needed to be taught to change into a unique form of chief altogether.
Whereas Goldsztein by no means got down to change into a founder, the MIT Sloan School of Management taught him the significance of networking, constructing friendships, and making profession connections with friends and college from all walks of life. He started utilizing these abilities in a brand new approach — boldly reaching out to the highest medulloblastoma medical doctors and scientists at hospitals world wide to ask for assist.
“I knew that I needed to do one thing to avoid wasting Frederico, but in addition the opposite estimated 15,000 youngsters recognized with the illness world wide annually,” he says.
In 2021, Goldsztein launched The Medulloblastoma Initiative (MBI), a nonprofit group devoted to discovering a remedy utilizing a exceptional new mannequin for funding uncommon illness analysis.
In simply 18 months, the group — which remains to be in startup mode — has raised $11 million in personal funding and introduced collectively 14 of the world’s most prestigious labs and hospitals from throughout North America, Europe, and Brazil.
Two promising trials will launch within the coming months, and three extra trials are within the pipeline and presently awaiting U.S. Meals and Drug Administration approval.
All of this in an trade that’s infamous for bureaucratic pink tape, and the place the timeline from an preliminary lab discovery to a affected person receiving a primary remedy averages seven to fifteen years.
Whereas authorities analysis grants sometimes allocate simply 4 cents on the greenback towards pediatric most cancers analysis — pennies doled out throughout a number of labs pursuing uncoordinated efforts — MBI is laser-focused on pushing one hundred pc of their funding towards a singular objective, with none overhead or administrative prices.
“There isn’t a time to lose,” Goldsztein says. “We’re making science transfer sooner than it ever has earlier than.”
The MBI blueprint for funding cures for uncommon illnesses is replicable, and more likely to disrupt the usual approach well being care analysis is funded and carried out by radically shortening the timeline.
From despair to energy
After his preliminary analysis at age 9, Frederico went by way of a nine-hour mind surgical procedure and got here to the USA to obtain customary remedy. Goldsztein regarded on helplessly as his son acquired radiation after which 9 grueling rounds of chemotherapy.
First pioneered within the Nineteen Eighties, this customary remedy protocol cures 70 % of youngsters. Nonetheless, it leaves most of them with lifelong unwanted effects like cognitive issues, endocrine points that stunt development, and secondary tumors. Frederico was on the improper facet of that statistic. Simply three years later, his tumor relapsed.
Goldsztein grimaces as he remembers the prognosis he and his spouse heard from the medical doctors.
“It was unbelievable to me that there had been nearly no discoveries in 40 years,” he says.
In the end, he discovered hope and partnership in Roger Packer, the director of the Mind Tumor Institute and the Gilbert Household Neurofibromatosis Institute of Kids’s Nationwide Hospital. He’s additionally the very physician who created the usual remedy years earlier than.
Packer explains that discovering efficient therapies for medulloblastoma was complicated for 30 years as a result of it’s an umbrella time period for 13 varieties of tumors. Frederico suffers from the most typical one, Group 4.
A part of the rationale the remedy has not modified is that, till lately, drugs has not superior sufficient to detect variations between the totally different tumor varieties. Packer explains, “Now with molecular genetic testing and methylation, which is a strategy to basically type tumors, that has modified.”
The issue for Frederico was that only a few researchers have been engaged on Group 4, the sub-type of medulloblastoma that’s the most typical tumor, but additionally the one which scientists know the least about.
Goldsztein challenged Packer: “If I can get you the funding, what can your lab do to advance medulloblastoma analysis rapidly?”
An open-source consortium mannequin
Packer suggested that they work collectively to “strive one thing totally different,” as a substitute of simply throwing cash at analysis with none guideposts.
“We arrange a consortium of main establishments world wide doing medulloblastoma analysis, requested them to alter their lab strategy to give attention to the Group 4 tumor, and assigned every lab a query to reply. We charged them with developing with remedy — not in seven to 10 years, which is the traditional transition from discovery to growing a drug and getting it to a affected person, however inside a two-year timeline,” he says.
Initially, seven labs signed on. In the present day, the Remedy Group 4 Consortium is made up of 14 companions and reads like a who’s who of medulloblastoma heavy hitters: Kids’s Nationwide Hospital, SickKids, Hopp Kids’s Most cancers Middle, and Texas Kids’s Hospital.
Labs can solely be part of the consortium if they comply with observe some uncommon guidelines. As Goldsztein explains, “To be accepted into this group and obtain funding, there aren’t any silos, and there’s no duplicated work. Everybody has a bit of the puzzle, and we work collectively to maneuver quick. That’s the magic of our mannequin.”
Impressed by MIT’s open-source strategies, researchers should share information freely with each other to speed up the group’s general progress. This sort of partnership throughout labs and borders is unprecedented in a extremely aggressive sector.
Mariano Gargiulo MBA ’03 met Goldsztein on the primary day of their MIT Sloan Fellows MBA program orientation and has been his expensive good friend ever since. An early-stage donor to MBI and a Houston-based government within the power sector, Gargiulo sat down with Goldsztein as he first conceptualized MBI’s working mannequin.
“Often, startup enterprise fashions plot out the subsequent 10-15 years; Fernando’s timeline was solely two years, and his benchmarks have been in three-month increments.” It was audaciously optimistic, says Gargiulo, however so was the founder.
“Once I noticed it, I didn’t doubt that he would obtain his targets. I’m seeing Fernando hit these first targets now and it’s wonderful to observe,” Gargiulo says.
Kids’s Nationwide Hospital endorsed MBI in 2023 and invited Goldsztein to take a seat on its basis’s board, including credibility to the initiative and his capacity to fundraise extra ambitiously.
In keeping with Packer, within the subsequent few months, the primary two MBI protocols will attain sufferers for the primary time: an immunotherapy protocol, which “leverages the physique’s immune response to focus on most cancers cells extra successfully and safely than conventional therapies,” and a medulloblastoma vaccine, which “adapts related methodologies utilized in Covid-19 vaccine improvement. This strategy goals to offer a flexible and cellular remedy that may very well be distributed globally.”
A matter of when
When Goldsztein will not be together with his circle of relatives in Brazil, fundraising, or managing MBI, he’s on Zoom with a community of greater than 70 different households with youngsters with relapsed medulloblastoma. “I’m not a health care provider and I don’t give out medical recommendation, however with these trials, we’re giving one another hope,” he explains.
Hope and function are commodities that Goldsztein has in spades. “I don’t perceive the thought of doing enterprise and accumulating property, however not serving to others,” he says. He shared that message with an auditorium of his fellow alumni at his 2023 MIT Sloan Reunion.
Frederico, who defied all odds and lived with the specter of recurrence, lately graduated highschool. He’s curious about worldwide relations and obsessed with pictures. “That is about discovering a remedy for Frederico and for all children,” Goldsztein says.
When requested how the world could be impacted if MBI discovered a remedy for medulloblastoma, Goldsztein shakes his head.
“We’re going to discover the remedy. It’s not if, it’s a matter of when.”
His subsequent objective is to scale MBI and have it function a useful resource for teams that wish to replicate its playbook to unravel different childhood illnesses.
“I’m by no means going to cease,” he says.
