“There isn’t any remedy out there on your son. We will’t do something to assist him.”
When Fernando Goldsztein MBA ’03 heard these phrases, one thing inside him snapped.
“I refused to just accept what the medical doctors had been saying. I reworked my worry into my best power 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 right away. He needed to be taught to change into a distinct type of chief altogether.
Whereas Goldsztein by no means got down to change into a founder, the MIT Sloan Faculty of Administration 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 expertise in a brand new manner — boldly reaching out to the highest medulloblastoma medical doctors and scientists at hospitals all over the world to ask for assist.
“I knew that I needed to do one thing to save lots of Frederico, but in addition the opposite estimated 15,000 kids recognized with the illness all over the world every year,” he says.
In 2021, Goldsztein launched The Medulloblastoma Initiative (MBI), a nonprofit group devoted to discovering a remedy utilizing a outstanding 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 non-public 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 at present awaiting U.S. Meals and Drug Administration approval.
All of this in an business that’s infamous for bureaucratic crimson 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 purpose, with none overhead or administrative prices.
“There isn’t any 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 manner well being care analysis is funded and carried out by radically shortening the timeline.
From despair to power
After his preliminary analysis at age 9, Frederico went by way of a nine-hour mind surgical procedure and got here to america to obtain commonplace remedy. Goldsztein regarded on helplessly as his son obtained radiation after which 9 grueling rounds of chemotherapy.
First pioneered within the Eighties, this commonplace remedy protocol cures 70 p.c of youngsters. Nonetheless, it leaves most of them with lifelong unwanted effects like cognitive issues, endocrine points that stunt progress, and secondary tumors. Frederico was on the incorrect facet of that statistic. Simply three years later, his tumor relapsed.
Goldsztein grimaces as he recollects 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.
Finally, 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 explanation the remedy has not modified is that, till not too long ago, drugs has not superior sufficient to detect variations between the completely different tumor varieties. Packer explains, “Now with molecular genetic testing and methylation, which is a option to primarily kind tumors, that has modified.”
The issue for Frederico was that only a few researchers had 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 shortly?”
An open-source consortium mannequin
Packer suggested that they work collectively to “strive one thing completely different,” as an alternative of simply throwing cash at analysis with none guideposts.
“We arrange a consortium of main establishments all over the world doing medulloblastoma analysis, requested them to alter their lab method to concentrate on the Group 4 tumor, and assigned every lab a query to reply. We charged them with arising 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. Right this moment, the Treatment 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 comply with 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 total progress. This type 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 pricey pal ever since. An early-stage donor to MBI and a Houston-based govt within the power sector, Gargiulo sat down with Goldsztein as he first conceptualized MBI’s working mannequin.
“Often, startup enterprise fashions plot out the following 10-15 years; Fernando’s timeline was solely two years, and his benchmarks had 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 superb 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 potential to fundraise extra ambitiously.
In accordance 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 growth. This method goals to supply a flexible and cellular remedy that may very well be distributed globally.”
A matter of when
When Goldsztein just isn’t along 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 kids 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 goal are commodities that Goldsztein has in spades. “I don’t perceive the concept 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, not too long ago graduated highschool. He’s considering 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 can 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 purpose is to scale MBI and have it function a useful resource for teams that need to replicate its playbook to resolve different childhood illnesses.
“I’m by no means going to cease,” he says.
