09/23/2025 | Gabriela Maraccini | CNN

https://www.cnnbrasil.com.br/saude/iniciativa-criada-por-brasileiro-acelera-pesquisas-contra-cancer-infantil/ 

The Medulloblastoma Initiative, created by Fernando Goldsztein from Rio Grande do Sul, funds studies that promise to transform the treatment of a rare type of brain tumor

At the age of nine, the son of Rio Grande do Sul businessman Fernando Goldsztein began to experience headaches and vomiting for no apparent reason. After a period of seeking medical help and undergoing tests, the young boy, named Frederico, was diagnosed with medulloblastoma, a type of malignant brain tumor.

But confirming the diagnosis in 2015 was not an easy process. “It's not a general rule, but sometimes doctors tend not to perform imaging tests immediately to rule out a brain tumor because it is considered a rare disease,” Goldsztein notes in an interview with CNN. “But unfortunately, it affected my son, and he was diagnosed after a certain period,” he says.

Treatment options for medulloblastoma include surgery to remove the tumor and the use of chemotherapy and radiation therapy. However, after attempts to cure the disease, Goldsztein heard from doctors that there were no effective treatments for his son. "They said there was nothing they could do because my son was among the 20 to 30% of patients who do not respond to treatment. The tumor had returned and there was no cure,“ he says. ”But giving up on my son was not an option."

Unwilling to accept the poor prognosis, Goldsztein decided on his own to seek a cure for his son and thousands of other children who are also affected by the disease. Based on this experience, the entrepreneur built a research model that combines cutting-edge science, direct funding, and international cooperation. The result of his work was the creation of the Medulloblastoma Initiative (MBI) in 2021.

In just 30 months, the MBI raised US$ 11 million (approximately R$ 59 million) and established partnerships with 17 prestigious institutions in the United States, Canada, and Germany, accelerating discoveries that previously took more than a decade to reach patients. The key to this acceleration was the Cure Group 4 Consortium, developed alongside pediatric neurosurgeon Roger Packer in the United States.

"The institutions work collaboratively and in total synergy. Each one forms a piece of the puzzle. There are no silos [of information], no duplication of work. This synergy allows our research to move at a faster pace than if each laboratory were working in isolation," explains Goldsztein.

The initiative has been recognized by the Massachusetts Institute of Technology (MIT) as a model for funding research into rare diseases that can be applied to other diseases, not just medulloblastoma.

"An important difference at MBI is the sense of urgency. Initiatives like ours, which start with families, are not uncommon in the United States, but many of them are created as tributes to patients who have already passed away. In my case, I created MBI to save my son and other children. This gives it a unique sense of urgency, where every day and every week counts in our efforts to conduct studies focused on finding a cure for this disease," she says.

In the coming months, MBI will begin the first clinical trial with advanced immunotherapy, approved by the U.S. Food and Drug Administration (FDA), which stimulates the body's own immune response against tumor cells.

Another ongoing study is developing a vaccine against medulloblastoma based on messenger RNA technology, similar to that used in the Covid-19 vaccine, with potential for global distribution.

“It's not a question of ‘if,’ it's a question of ‘when’ we will find the cure,” says Goldsztein.

Understanding medulloblastoma
Medulloblastoma is an aggressive tumor that develops in the cerebellum, the region of the brain responsible for balance and motor coordination. Although rare in the general population, it is the most common malignant brain cancer in childhood, accounting for about 20% of pediatric brain tumors.

“Most diagnoses occur between the ages of 5 and 9, with a slight predominance in boys. Although it can occur in adults, these cases are much less frequent,” explains pediatric hematologist and oncologist Cláudio Galvão de Castro Junior, advisor to MBI.

Symptoms may include persistent morning headaches, vomiting without apparent cause, changes in balance and vision, and, in infants, rapid increase in head circumference. “It is important for parents and healthcare professionals to be vigilant. These signs may seem nonspecific at first, but early detection makes all the difference in prognosis,” warns Galvão.

Standard treatment combines surgery, radiation therapy, and chemotherapy, but still imposes severe side effects, such as cognitive deficits, hormonal changes, and hearing loss. "Medicine is moving toward more targeted therapies. Current studies seek to reduce the radiation dose in low-risk children, in addition to exploring immunotherapies and new targeted drugs capable of increasing survival and, above all, quality of life," adds the specialist.