Author: Julio Marchi

“There is no treatment available for your son. We can’t do anything to help him.”

When Fernando Goldsztein MBA ’03 heard those words, something inside him snapped.

“I refused to accept what the doctors were saying. I transformed my fear into my greatest strength and started fighting.”

Goldsztein’s 12-year-old son Frederico was diagnosed with relapsing medulloblastoma, a life-threatening pediatric brain tumor. Goldsztein’s life — and career plan — changed in an instant. He had to learn to become a different kind of leader altogether.

While Goldsztein never set out to become a founder, the MIT Sloan School of Management taught him the importance of networking, building friendships, and making career connections with peers and faculty from all walks of life. He began using those skills in a new way — boldly reaching out to the top medulloblastoma doctors and scientists at hospitals around the world to ask for help.

“I knew that I had to do something to save Frederico, but also the other estimated 15,000 children diagnosed with the disease around the world each year,” he says.

In 2021, Goldsztein launched The Medulloblastoma Initiative (MBI), a nonprofit organization dedicated to finding a cure using a remarkable new model for funding rare disease research.

In just 18 months, the organization — which is still in startup mode — has raised $11 million in private funding and brought together 14 of the world’s most prestigious labs and hospitals from across North America, Europe, and Brazil.

Two promising trials will launch in the coming months, and three additional trials are in the pipeline and currently awaiting U.S. Food and Drug Administration approval.

All of this in an industry that is notorious for bureaucratic red tape, and where the timeline from an initial lab discovery to a patient receiving a first treatment averages seven to 15 years.

While government research grants typically allocate just 4 cents on the dollar toward pediatric cancer research — pennies doled out across multiple labs pursuing uncoordinated efforts — MBI is laser-focused on pushing 100 percent of their funding toward a singular goal, without any overhead or administrative costs.

“There is no time to lose,” Goldsztein says. “We are making science move faster than it ever has before.”

The MBI blueprint for funding cures for rare diseases is replicable, and likely to disrupt the standard way health care research is funded and carried out by radically shortening the timeline.

From despair to strength

After his initial diagnosis at age 9, Frederico went through a nine-hour brain surgery and came to the United States to receive standard treatment. Goldsztein looked on helplessly as his son received radiation and then nine grueling rounds of chemotherapy.

First pioneered in the 1980s, this standard treatment protocol cures 70 percent of children. Still, it leaves most of them with lifelong side effects like cognitive problems, endocrine issues that stunt growth, and secondary tumors. Frederico was on the wrong side of that statistic. Just three years later, his tumor relapsed.

Goldsztein grimaces as he recalls the prognosis he and his wife heard from the doctors.

“It was unbelievable to me that there had been almost no discoveries in 40 years,” he says.

Ultimately, he found hope and partnership in Roger Packer, the director of the Brain Tumor Institute and the Gilbert Family Neurofibromatosis Institute of Children’s National Hospital. He is also the very doctor who created the standard treatment years before.

Packer explains that finding effective therapies for medulloblastoma was complex for 30 years because it is an umbrella term for 13 types of tumors. Frederico suffers from the most common one, Group 4.

Part of the reason the treatment has not changed is that, until recently, medicine has not advanced enough to detect differences between the different tumor types. Packer explains, “Now with molecular genetic testing and methylation, which is a way to essentially sort tumors, that has changed.”

The problem for Frederico was that very few researchers were working on Group 4, the sub-type of medulloblastoma that is the most common tumor, yet also the one that scientists know the least about.

Goldsztein challenged Packer: “If I can get you the funding, what can your lab do to advance medulloblastoma research quickly?”

An open-source consortium model

Packer advised that they work together to “try something different,” instead of just throwing money at research without any guideposts.

“We set up a consortium of leading institutions around the world doing medulloblastoma research, asked them to change their lab approach to focus on the Group 4 tumor, and assigned each lab a question to answer. We charged them with coming up with therapy — not in seven to 10 years, which is the normal transition from discovery to developing a drug and getting it to a patient, but within a two-year timeline,” he says.

Initially, seven labs signed on. Today, the Cure Group 4 Consortium is made up of 14 partners and reads like a who’s who of medulloblastoma heavy hitters: Children’s National Hospital, SickKids, Hopp Children’s Cancer Center, and Texas Children’s Hospital.

Labs can only join the consortium if they agree to follow some unusual rules. As Goldsztein explains, “To be accepted into this group and receive funding, there are no silos, and there is no duplicated work. Everyone has a piece of the puzzle, and we work together to move fast. That is the magic of our model.”

Inspired by MIT’s open-source methods, researchers must share data freely with one another to accelerate the group’s overall progress. This kind of partnership across labs and borders is unprecedented in a highly competitive sector.

Mariano Gargiulo MBA ’03 met Goldsztein on the first day of their MIT Sloan Fellows MBA program orientation and has been his dear friend ever since. An early-stage donor to MBI and a Houston-based executive in the energy sector, Gargiulo sat down with Goldsztein as he first conceptualized MBI’s operating model.

“Usually, startup business models plot out the next 10-15 years; Fernando’s timeline was only two years, and his benchmarks were in three-month increments.” It was audaciously optimistic, says Gargiulo, but so was the founder.

“When I saw it, I did not doubt that he would achieve his goals. I’m seeing Fernando hit those first targets now and it’s amazing to watch,” Gargiulo says.

Children’s National Hospital endorsed MBI in 2023 and invited Goldsztein to sit on its foundation’s board, adding credibility to the initiative and his ability to fundraise more ambitiously.

According to Packer, in the next few months, the first two MBI protocols will reach patients for the first time: an immunotherapy protocol, which “leverages the body’s immune response to target cancer cells more effectively and safely than traditional therapies,” and a medulloblastoma vaccine, which “adapts similar methodologies used in Covid-19 vaccine development. This approach aims to provide a versatile and mobile treatment that could be distributed globally.”

A matter of when

When Goldsztein is not with his own family in Brazil, fundraising, or managing MBI, he is on Zoom with a network of more than 70 other families with children with relapsed medulloblastoma. “I’m not a doctor and I don’t give out medical advice, but with these trials, we are giving each other hope,” he explains.

Hope and purpose are commodities that Goldsztein has in spades. “I don’t understand the idea of doing business and accumulating assets, but not helping 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 threat of recurrence, recently graduated high school. He is interested in international relations and passionate about photography. “This is about finding a cure for Frederico and for all kids,” Goldsztein says.

When asked how the world would be impacted if MBI found a cure for medulloblastoma, Goldsztein shakes his head.

“We are going to find the cure. It’s not if, it’s a matter of when.”

His next goal is to scale MBI and have it serve as a resource for groups that want to replicate its playbook to solve other childhood diseases.

“I’m never going to stop,” he says.