In this interview, Dr. Packer and Fernando show how connected science expands access, improves decision-making, and creates impact far beyond the laboratory.

Read the full story by clicking here.

O post Brazilian businessman’s initiative creates a chance of cure for a rare type of cancer apareceu primeiro em The Medulloblastoma Initiative.

In the rare disease realm, the term “diagnostic odyssey” refers to the long, difficult process required to confirm a patient’s illness. Families must then endure the even more excruciating path to find an effective treatment.

For Brazilian businessman Fernando Goldzstein, that odyssey began on Nov. 7, 2015.

“We received this news that our son was diagnosed with a brain tumor, and at that time, our lives froze. It’s very difficult to put in words what a father or a mother feel in circumstances like this.”

Nine-year-old Frederico Goldzstein was diagnosed with medulloblastoma, the most common malignant brain cancer in children, and his father was determined to save his life. His journey toward an effective treatment led to a collaboration with Children’s National Hospital in Washington, D.C., and other research institutions, intended to speed up the research and diagnostic process. It also yielded the launch of the Medulloblastoma Initiative, an international organization that seeks cures for these rare cancers by supporting scientific research.

On November 18, Goldzstein spoke with NPF Rare Disease Reporting fellows about the need for more collaboration in the race to save the lives of children like Frederico. Here are some highlights from his session:

For rare disease parents, giving up is not an option

Goldzstein said that while his son’s treatment began in their hometown of Sao Paolo, it soon became clear that he’d have to travel to find answers. That’s because at one point, the tumor relapsed, and doctors in Boston told him there was nothing else to do but wait for Frederico to die.

Goldsztein rejected that advice. He began the arduous process of educating himself about his son’s condition and reaching out to the best research institutions in the U.S. He was directed to Dr. Roger Packer, Director of the Brain Tumor Institute at Children’s National Hospital. He recommended that Frederico participate in clinical trials in New York and Washington.

But for parents of children with brain cancer, hope can bring deeper anxiety.

“There is no scientific proof that things are going to work or not, so you are in unchartered waters. It might be toxic, you don’t know, but you don’t have options. So you have to do something or the tumor is going to move ahead until the patient dies.”

Frederico participated in the trials, even though they were not designed for his type of cancer.

“There was no trial specific for Frederico’s disease. I was hoping for the best, but I had to be prepared for the worst.”

Children with brain cancer need better treatments

Goldzstein said that in this age of biotech innovation, treatments for children with brain cancer have not kept pace.

“Believe it or not, the treatment is very old. It’s from the ’80s. The treatment is very toxic and ineffective because 30 percent, more or less, of the kids relapse. And if they relapse, there’s no cure. And you might think that, ‘well, we have 70 percent of the kids cured.’ Yes, we do. However, the treatments are very, very toxic and the kids suffer from collateral effects that affect their quality of life, like they don’t grow, or they have cognitive problems of even secondary tumors.”

The Medulloblastoma Initiative pushes clinical trials

The Medulloblastoma Initiative, founded in 2021, has been able to get two clinical trials approved in two and a half years – significant speed in a field where that process can take a decade. Goldzstein said that philanthropy and collaboration between 12 U.S. research institutions, one in Canada and one in Germany made it possible.

“When we started this, we didn’t know exactly where we are going to be in two years, four years. I would never imagine that MBI would be a global movement.”

The initiative also has a third and fourth trial in the pipeline, and some children whose tumors relapsed are also being treated.

Not everyone is as fortunate, but Frederico defied the dire odds he’d been given.

“He is a very smart guy. He likes to travel very much. He likes to read, he loves school as well. Fred has been a warrior, and I decided that fighting the pediatric brain tumor will be the goal of my life from now.”

O post The Medulloblastoma Initiative Fast Tracks Research To Help Kids with Cancer apareceu primeiro em The Medulloblastoma Initiative.

Originally published in: People Magazine.

O post Dad Is in Race Against Time to Save Teen Son and Thousands of Other Kids with Same Rare Brain Tumor apareceu primeiro em The Medulloblastoma Initiative.

O post MIT Sloan Recognizes MBI Among Top Alumni Stories of 2025 apareceu primeiro em The Medulloblastoma Initiative.

In the article, experts emphasize the scientific and social impact of bringing experimental therapies for pediatric brain tumors to Brazil, a milestone made possible by MBI’s collaborative model

The story also presents the journey of Fernando Goldsztein and his son Frederico, showing how a personal experience evolved into an international consortium that now brings together 16 leading institutions, has raised US$ 11 million, and allowed the record-time approval of two clinical trials by the FDA.

Among the approaches highlighted are personalized RNA vaccines and CAR-T cell therapies – technologies designed to activate the patient’s own immune system against the tumor and considered especially promising in cases of recurrence, when treatment options are more limited.

The article also reports on the signing of the consortium’s first agreement with a Brazilian institution: the Einstein Hospital Israelita, which now joins the MBI network through scientific collaboration, data sharing, and priority participation in future clinical trials.

This recognition reinforces the strength of a mission that began in Brazil and now mobilizes scientists, families, and donors around the world, all united by the same purpose: accelerating the development of new therapies for children who cannot wait.

Read the full story – clicking here

O post National recognition for a global mission apareceu primeiro em The Medulloblastoma Initiative.

By sharing the origins of the MBI, VivaBem underscores both the human impact and the scientific reach of this cause: a network that crosses borders, connects leading research centers, and accelerates the pathways that allow children around the world to access more advanced and promising treatments.

This national recognition reinforces the strength of a mission that began in Brazil and has since become a bridge between cutting-edge research, international collaboration, and real hope for thousands of families.

Read the full story – clicking here

We remain steadfast in our commitment to accelerating the cure through science, collaboration, and purpose.

O post The VivaBem/UOL portal featured the story of the Medulloblastoma Initiative (MBI) apareceu primeiro em The Medulloblastoma Initiative.

Founded in 1955 by São Paulo’s Jewish community, Einstein Hospital Israelita is a leading reference in healthcare, research, and social responsibility. Guided by the principles of Tikun (Transformation), Chinuch (Education), Refuá (Healthcare), Tsedaká (Social Justice), and Mitzvá (Good Deeds), Einstein combines excellence, humanized care, and knowledge generation to advance medicine and create a positive impact on society.

According to Newsweek’s ranking, the Hospital stands out as the leading hospital in the Southern Hemisphere and Latin America.

This first collaboration between MBI and a Brazilian healthcare institution strengthens international cooperation, promotes knowledge exchange, and supports future clinical trials.

After the signing, Dr. Roger J. Packer delivered the lecture “Transforming the Landscape of Medulloblastoma Research and the MBI Model for Global Impact,” highlighting the role of strategic collaborations in the search for new therapies.

Together, we move forward to ensure that every child has the future they deserve.

O post A powerful agreement for global impact apareceu primeiro em The Medulloblastoma Initiative.

O post MBI on The Lead in Peds apareceu primeiro em The Medulloblastoma Initiative.

Recognized for its excellence in care, education, research, and social responsibility, Einstein combines tradition and innovation to transform medicine through knowledge, ethics, and social commitment.

The MBI, in turn, is an international philanthropic initiative that connects families, scientists, and world-class institutions to drive discoveries that can change the future for children with medulloblastoma. Since its creation, it has promoted unique collaborations among universities, hospitals, and research centers of global relevance.

This partnership with Einstein represents another strategic step toward strengthening a global network dedicated to impactful science, one that values scientific rigor, transparency, and a commitment to real results.

Purpose-driven science always finds worthy allies.

O post MBI innovates once again with a new partnership: Einstein Hospital Israelita apareceu primeiro em The Medulloblastoma Initiative.

One of the most respected magazines in the country mentioned MBI in a report about scientific advances in the treatment of medulloblastoma.

With more than 50 years of history, Exame is a national reference in economic, scientific, and innovation journalism. Since 2020, it has been part of the BTG Pactual Group, consolidating itself as one of the most prestigious and far-reaching publications in Brazil.

The article highlights the beginning of the first clinical trials conducted by MBI, involving two patients treated through personalized immunotherapy: an innovative approach designed to strengthen the immune system to attack the tumor’s DNA.

O post MBI mentioned by Exame apareceu primeiro em The Medulloblastoma Initiative.