A compound hidden in broccoli could help fight a devastating genetic disease

The disease, known as Friedreich ataxia, causes nerve cells in the brain and spinal cord to gradually deteriorate. As the damage progresses, patients lose their ability to coordinate movements, walk, and speak. The condition can also shorten life expectancy, and there is currently no cure. Approximately 200 Australians are living with the disease.

In a new study, scientists found that sulforaphane, a naturally occurring compound in broccoli, can increase levels of frataxin, a protein that is present in insufficient amounts in people with Friedreich ataxia. The researchers also found that the compound can help protect vulnerable nerve cells from damage.

How a Broccoli Compound Could Protect Nerve Cells

Frataxin plays an important role in keeping cells functioning properly, particularly those with high energy demands, such as nerve cells. In Friedreich ataxia, a genetic defect leads to reduced production of this essential protein, contributing to the progressive damage that characterizes the disease.

Sulforaphane appears promising because its potential benefits extend beyond increasing frataxin levels. Research indicates that it can also influence several biological processes involved in disease progression, including cellular stress and inflammation. Addressing multiple sources of damage at once could make the compound a valuable candidate for further investigation.

The findings are particularly encouraging for children with Friedreich ataxia. According to lead researcher Associate Professor Faith Kwa of Swinburne University, children account for a large proportion of those affected, yet no treatments specifically approved for pediatric patients are currently available.

"Friedreich ataxia is a devastating rare genetic disease that progressively steals a child's ability to walk, speak, play, and ultimately survive," she says.

"This novel therapeutic agent can target the underlying cause of the disease, while delivering an affordable and accessible therapy worldwide, improving treatment opportunities for families affected by this life-limiting disease.

"This discovery brings us closer to a future where children with Friedreich ataxia experience a better quality of life, greater independence, and an improved chance of long-term survival."

A Potential Treatment That Could Be More Accessible

One of the most encouraging aspects of sulforaphane is that it is not an entirely unfamiliar substance. The compound already has an established safety record in both children and adults, and purified, biologically active forms are commercially available.

These advantages could make it easier to develop a widely accessible treatment if future clinical trials confirm that sulforaphane is effective against Friedreich ataxia. However, further research is essential before scientists can determine whether the promising findings will translate into meaningful benefits for patients.

Kwa explains that the rarity of Friedreich ataxia creates additional challenges for researchers. Because relatively few people have the disease, public awareness is limited, making it more difficult to attract the funding needed to investigate potential treatments.

Researchers Seek Funding for Clinical Trials

The Swinburne team hopes to secure financial support for clinical trials that would examine whether sulforaphane can deliver the same promising effects in people living with the disease.

"We are hoping to gain funding to conduct clinical trials and further prove the positive impact the broccoli compound could have on those suffering from Friedreich ataxia," she says.

"Sulforaphane already has an established safety record in children and adults, and its pure and bioactive form is commercially available. This means if clinical trials prove successful, its rollout could accelerate the path to patient impact."

For patients and families facing a progressive illness with no cure, even improvements in everyday functioning and independence could make a meaningful difference. The researchers hope their work will not only advance treatment possibilities but also bring greater attention to a condition that remains largely unknown to the public.

"Even if we can raise awareness of this debilitating condition or improve one person's quality of life, our efforts will have been worth it."