Header Image: Rosa Rademakers (VIB-UAntwerp)
In April 2026, Rosa Rademakers (VIB-UAntwerp) appeared on the red carpet in Los Angeles to accept her Breakthrough Prize. The award is nicknamed the ‘Oscar of Science’, because the flashy ceremony is attended by both film stars and tech billionaires, in addition to the world-class scientists whose work is being celebrated on the stage.
Rademakers received the prize for a discovery made back in 2011, when she led research at the Mayo Clinic in Florida. Her team solved a decades-old mystery in neurodegenerative diseases by discovering the most common genetic cause of both frontotemporal dementia (FTD) — the most common form of dementia among people under the age of 65 — and amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease.
“I am the one who gets to accept the award, but I do so on behalf of the many people who dedicate themselves to fighting those diseases every day.” – Rosa Rademakers
“This is, of course, fantastic recognition,” Rosa Rademakers shared with VRT News in an initial statement. “Not only for my own work, but also for all colleagues in our laboratory who work hard every day. I am the one who gets to accept the award, but I do so on behalf of the many people who dedicate themselves to fighting those diseases every day.”
A Breakthrough Based on a Single Family
Back in 2011, researchers already knew there had to be a link between FTD and ALS, because the two conditions co-occurred in some families. With these relatives, some individuals developed ALS symptoms (such as cramps, weakened muscles, and eventual paralysis) while others would develop the signs of FTD (including drastic changes in personality, behavior, and emotions). Some people would have a combination of both, while other family members remained completely unaffected. The underlying genetic cause was a mystery.
Coincidentally, the conundrum was simultaneously solved by both Rademakers and another scientist called Bryan Traynor, with whom she shares the Breakthrough Prize. Working independently, both of their labs discovered the genetic culprit: a repetition in an otherwise unknown gene called C9orf72.
Working with the DNA from just a single family, Rademakers figured out that the individuals who remained healthy only had a few repeats of this specific same six-letter sequence of DNA, whereas family members that were affected by FTD or ALS had hundreds or even thousands of copies each.
A Landmark Moment for the Field
When Rademakers and her team verified their realization with more patients, the scope of their discovery became clear. “I remember the moment the results came in from about 100 patients we had examined,” said Rademakers to VRT News. “One in three had that same specific mutation. Then we knew it was going to be a major breakthrough.”
The publication represented a landmark moment in the study of these diseases. It shed light on the disease mechanisms, made it clear that FTD and ALS are connected, and opened new pathways for the development of treatments for these currently incurable diseases — including at least two therapies currently undergoing clinical trials.
“No medicines could be developed without the hard work of the many scientists in the laboratory.” – Rosa Rademakers
“This award proves how important basic scientific research is,” said Rademakers. “No medicines could be developed without the hard work of the many scientists in the laboratory. It also shows the younger generations that it is actually pretty cool to be a scientist, and that you do truly important work that benefits everyone.”
From Europe to the US and Back Again
Rademakers has been at the forefront of global dementia research for years. Born in the Netherlands in 1978, she moved to Antwerp at the age of 13 and eventually obtained her PhD at the University of Antwerp. She left Belgium for a postdoc at the Mayo Clinic the US in 2005, which is where this groundbreaking work took place.
In 2019, Rademakers returned to Antwerp to become Scientific Director of the Center for Molecular Neurology at VIB and UAntwerp. In 2022, she received the Generet Prize for Rare Diseases — the most prestigious prize for rare diseases in Belgium and Europe, worth 1 million euros.
“Our discovery was an important step forward, but of course the work is far from finished.” – Rosa Rademakers
Christine Durinx, Managing Director of VIB, warmly congratulated Rademakers on the Breakthrough Prize in a LinkedIn post, saying it’s “a proud moment for the scientific community — and an inspiring reminder of how fundamental research can change lives.”
Commenting on the prize herself, Rademakers said it is a “tremendous honor”. While she’s deeply grateful for the recognition, she also feels it is important in terms of the visibility it brings to dementia, and to the people and families living with its impact every day. “Our discovery of the C9orf72 hexanucleotide expansion was an important step forward, but of course the work is far from finished,” she says.
The Work Continues
Fifteen years on from Rademakers’ breakthrough, there still isn’t an effective treatment available for FTD and ALS patients, but her discovery has set a lot of promising work in motion.
The field has made real progress towards understanding the underlying disease mechanisms for the diseases, in particular the effects of toxic RNA and proteins in brain cells. Researchers have identified potential therapeutic targets, several of which are already being tested in clinical trials. “Understanding the biology of disease is often a long and gradual process, but it is what makes every later step toward timely diagnosis and therapy possible,” said Rademakers.
Read this article to learn about Belgian researchers Catching Cognitive Decline Early with Citizen Science!
This work has been particularly important in terms of diagnosis, which is exceptionally challenging for neurological conditions where the disease onset often occurs years before symptoms appear. Thanks to Rademakers’ research, scientists are now developing better biomarkers to predict whether someone will develop FTD or ALS and diagnose the diseases early. The hope is that, eventually, rapid interventions will help make treatments more effective.
Reflecting on her award, Rademakers said she’s deeply thankful to the many people who contributed, including colleagues in the US and in Belgium, and collaborators from all over the world. But above all, Rademakers thanked the patients and families who made the journey possible. “They remain the reason for this work, and why we will keep pushing forward.”
