Researchers at Swinburne University have identified a natural compound found in broccoli that could offer a new approach to treating Friedreich ataxia, a rare and progressive genetic disorder. The study suggests that sulforaphane may help increase levels of frataxin, a protein that is critically low in patients with the condition.
Friedreich ataxia causes nerve cells in the brain and spinal cord to deteriorate over time. As the disease progresses, patients lose the ability to coordinate movements, walk, and speak. The condition also reduces life expectancy, and there is currently no cure. Approximately 200 Australians live with the disease, and pediatric cases make up a significant portion of those affected.
Sulforaphane Targets Underlying Protein Deficit
The study focused on sulforaphane, a naturally occurring substance in broccoli. Scientists found that the compound can increase frataxin production in cells. Frataxin is essential for keeping cells functioning properly, particularly nerve cells that have high energy demands. In Friedreich ataxia, a genetic defect leads to insufficient production of this protein, which contributes to the progressive damage seen in patients.
Beyond boosting frataxin, sulforaphane appears to influence other biological processes involved in the disease. Research indicates it can help reduce cellular stress and inflammation. Addressing multiple sources of damage simultaneously makes the compound a strong candidate for further medical investigation.
Associate Professor Faith Kwa, the lead researcher at Swinburne University, emphasized the potential impact on children. She noted that no treatments specifically approved for pediatric patients currently exist for this condition.
“Friedreich ataxia is a devastating rare genetic disease that progressively steals a child’s ability to walk, speak, play, and ultimately survive,” Kwa said. “This novel therapeutic agent can target the underlying cause of the disease, while delivering an affordable and accessible therapy worldwide.”
Path to Clinical Trials
One advantage of sulforaphane is its established safety record in both children and adults. Purified, biologically active forms of the compound are already commercially available. This existing safety profile could simplify the development of a widely accessible treatment if future clinical trials confirm its effectiveness against Friedreich ataxia.
However, the rarity of the disease presents challenges for research funding. Public awareness remains limited, which makes it difficult to attract the financial support needed for large-scale studies. The Swinburne team is now seeking funding to conduct clinical trials that would examine whether sulforaphane delivers the same promising effects in people living with the disease.
Kwa stated that the team hopes to prove the positive impact the compound could have on sufferers. She added that if clinical trials prove successful, the rollout of the treatment could accelerate due to the compound’s existing availability.
For families facing a progressive illness with no cure, even small improvements in everyday functioning and independence could make a meaningful difference. The researchers hope their work will advance treatment possibilities and bring greater attention to a condition that remains largely unknown to the general 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,” Kwa said.
Source: ScienceDaily
