Is This the New Playbook for Curing Uncommon Childhood Illnesses?

By Danna Lorch | MIT Sloan College of Administration | MIT Information

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

When Fernando Goldsztein MBA ’03 heard these phrases, one thing 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 recognized with relapsing medulloblastoma, a life-threatening pediatric mind tumor. Goldsztein’s life — and profession plan — modified instantly. He needed to study to turn into a special form of chief altogether.

Whereas Goldsztein by no means got down to turn into a founder, the MIT Sloan College 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 approach — boldly reaching out to the highest medulloblastoma medical doctors and scientists at hospitals around the globe to ask for assist.

“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 group devoted to discovering a treatment 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 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 crimson tape, and the place the timeline from an preliminary lab discovery to a affected person receiving a primary therapy averages seven to fifteen years.

Whereas authorities analysis grants usually 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 100% of their funding towards a singular purpose, with none overhead or administrative prices.

“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 uncommon illnesses is replicable, and prone 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 america to obtain commonplace therapy. Goldsztein appeared on helplessly as his son obtained radiation after which 9 grueling rounds of chemotherapy.

First pioneered within the Nineteen Eighties, this commonplace therapy protocol cures 70 p.c of youngsters. Nonetheless, it leaves most of them with lifelong unwanted side effects like cognitive issues, endocrine points that stunt development, and secondary tumors. Frederico was on the incorrect aspect 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 almost 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 therapy years earlier than.

Packer explains that discovering efficient therapies for medulloblastoma was advanced for 30 years as a result of it’s an umbrella time period for 13 forms of tumors. Frederico suffers from the commonest one, Group 4.

A part of the explanation the therapy has not modified is that, till just 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 way to essentially sort tumors, that has changed.”

The issue for Frederico was that only a few researchers have been engaged on Group 4, the sub-type of medulloblastoma that’s the commonest 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 research quickly?”

An open-source consortium mannequin

Packer suggested that they work collectively to “try something different,” as an alternative of simply throwing cash at analysis with none 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. Immediately, 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 Heart, and Texas Kids’s Hospital.

Labs can solely be a part of the consortium if they comply with observe some uncommon guidelines. 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.”

Impressed by MIT’s open-source strategies, researchers should share knowledge freely with each other to speed up the group’s general 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 good friend ever since. An early-stage donor to MBI and a Houston-based govt within the vitality sector, Gargiulo sat down with Goldsztein as he first conceptualized MBI’s working mannequin.

“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, however 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.

Kids’s Nationwide Hospital endorsed MBI in 2023 and invited Goldsztein to sit down on its basis’s board, including credibility to the initiative and his means to fundraise extra ambitiously.

Based on Packer, within the subsequent few months, the primary two MBI protocols will attain sufferers for the primary 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 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 youngsters 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 goal 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 specter of recurrence, just lately graduated highschool. He’s interested by worldwide relations and enthusiastic about images. “This is about finding a cure for Frederico and for all kids,” Goldsztein says.

When requested how the world could be impacted if MBI discovered a treatment for medulloblastoma, Goldsztein shakes his head.

“We are going to find the cure. 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 wish to replicate its playbook to unravel different childhood illnesses.

“I’m never going to stop,” he says.

—

Reprinted with permission of MIT Information

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The submit Is This the New Playbook for Curing Uncommon Childhood Illnesses? appeared first on The Good Males Mission.

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