To save a life: Lessons for launching a medical R&D initiative

On Nov. 7, 2015, my family received the devastating news that our 9-year-old son Frederico was diagnosed with medulloblastoma, a pediatric brain tumor. At that shattering moment, I made two commitments: one to Frederico, a promise to dedicate my life to saving his; and the other to God, with a pledge to donate and raise money for research to find a cure. 

It was at this lowest point of my life that the seed of The Medulloblastoma Initiative (MBI) was planted — though I didn’t know it then. 

From our home in Porto Alegre, Brazil, we launched an international search for the best physicians and treatment plan for Frederico. We traveled to Massachusetts General Hospital, where Frederico received excellent medical care. Frederico lived, for which I thank God and his doctors. But he was transformed, and so was I. 

Our sweet boy endured a nine-hour surgery, followed by 30 sessions of radiotherapy and nine cycles of chemotherapy for nearly a year. This is a treatment protocol developed roughly 40 years ago, and while it boasts a 65% survival rate, Frederico’s doctors and I agree that figure is not high enough. Most patients will face severe side effects for life, given the high toxicity levels associated with this old therapy. There should be better options for children and their developing brains. There should be better options for children like Frederico.

Three years after Frederico’s successful treatment, his tumor was back. Once again, our world turned upside down — and from that point on, science could not offer any proven protocols for treatment. Two clinical trials were available to us, the first at Memorial Sloan Kettering Cancer Center in New York and then the next at Children’s National Hospital in Washington, D.C. 

Then, in 2022, the cancer returned yet again. We learned the hard way that science had not advanced beyond what we had already explored. I vowed to do something to change that. Frederico deserved better, as do all children and their families enduring this terrible disease. 

At the time, I was just a Brazilian businessman with no professional expertise in the healthcare field. But I was also the father of a child with cancer, and I could not accept that modern medicine had nothing to offer our family. 

The Talmudic teaching, “Whoever saves one life, saves the world entire” took on a heightened meaning in our household. 

We established the MBI with the aim of promoting research toward new, better treatments for medulloblastoma. We were blessed to join forces with an internationally acclaimed expert in this field, Dr. Roger J. Packer, director of the Brain Tumor Institute and the Gilbert Family Neurofibromatosis Institute of Children’s National Hospital. 

With an initial donation from my family and the support of many Brazilian donors, particularly from our Jewish community, we were able to establish a research consortium that currently brings together 13 top laboratories from the U.S., Canada, and Germany to achieve something extraordinary. 

Only two and a half years after we started, we have raised more than $10 million dollars (though the need is still far greater) and we are on the doorstep of clinical trials that offer promise for new treatments and ultimately, a cure. Researchers are examining trailblazing approaches to immunotherapy treatments for medulloblastoma which have proven effective for other tumors. Three trials are set to be launched over the next 6-12 months, a remarkable pace; typically the drug development process takes a decade, usually longer. 

We have also learned valuable lessons on how to establish a viable scientific research and development initiative. 

First, it is critical to find the right partner with expertise in this scientific field. In teaming up with Dr. Packer, we allied ourselves with the world’s foremost clinical expert and applied science researcher of pediatric brain tumors and medulloblastoma. His guidance and direction inspired us to establish the MBI. It is also not lost on us that Dr. Packer is the son of Holocaust survivors and that his family are supporters of and volunteers at the United States Holocaust Memorial Museum. We have a shared understanding of the preciousness and fragility of life. 

Second, identify a focus for research and concentrate your investment, measured in both money and time, on this one objective. The National Cancer Institute allocates only four cents to pediatric cancer for every dollar invested in research. Resources are finite and pediatric cancer is not a top priority for research funding. Our work seeks to overturn that dynamic. 

Third, establish a structure to allow the team of scientists to work synergistically toward a common goal, not in competition. Throughout the process, continue to follow up periodically. Since MBI was established in 2021, we have slowly added labs and researchers to the consortium. Our success so far is due to this community of experts. The greatest value I provide is the passion and hope that a cure can be found to save my son and other children, coupled with a drive to raise critical funds to support the MBI. 

Fernando Goldsztein presenting the MBI project during an annual alumni meeting of MIT Sloan School of Management.

Finally, public awareness is vital. The MBI mission cannot be confined to the scientific research community, discussed solely in the laboratory setting or at medical conferences. Donors play a vital role in sharing stories like Frederico’s, using the power of example to show why pediatric cancer research deserves funding and investment. 

Today, Frederico is 17 years old. He has lived with cancer for eight long years. He attends high school, loves reading, enjoys traveling and lives a relatively normal life. And yet, his cancer is very likely to return yet again, a heavy weight our family lives with every day. 

The MBI is our expression of the belief that whoever saves one life, saves the world entire. 

Fernando Goldsztein with his son Frederico at age 15 in Washington, DC.

Fernando Goldsztein is a Brazilian businessman and founder of The Medulloblastoma Initiative.