A common vegetable has given Australian scientists an unexpected lead in the search for better treatments for Friedreich ataxia.
A compound found naturally in broccoli may help correct one of the basic problems behind this rare and serious nervous system disease.
The compound is called sulforaphane.
Researchers at Swinburne University of Technology report that it can increase an important protein that is unusually low in people with Friedreich ataxia and may also help protect nerve cells from damage.
The findings were published in Antioxidants & Redox Signaling. Although the work is still at an early stage, researchers hope it could eventually lead to a treatment that is relatively affordable and widely available.
Friedreich ataxia is an inherited disease caused by a faulty gene. A child develops the condition after inheriting altered copies of the gene from both parents, who may carry the change without having the disease themselves.
The genetic change reduces production of a protein called frataxin. This protein is important for the tiny energy-producing structures inside cells, and having too little of it can interfere with normal cell function.
Over time, some nerve cells become damaged and die. The spinal cord and nerves that control movement are especially affected, causing problems with balance, coordination and muscle control.
Many patients first notice difficulty walking or frequent stumbling. As the disease progresses, some eventually need a wheelchair and can develop problems with speech, swallowing and other everyday functions.
Friedreich ataxia is not limited to the nervous system. It can also affect the heart and other organs, and these complications can have a major effect on health and life expectancy.
Only about 200 Australians are believed to have the condition. Because it is so uncommon, awareness is limited and research teams can face difficulty attracting funding for the expensive studies needed to develop treatments.
Associate Professor Faith Kwa and her colleagues at Swinburne have been investigating whether sulforaphane could offer a new approach. The compound occurs naturally in broccoli and related vegetables and has attracted scientific interest because of the way it affects the body’s defenses against cell damage.
In the new research, sulforaphane increased frataxin levels. That is important because a shortage of frataxin sits close to the root of Friedreich ataxia rather than being only a later consequence of the disease.
The compound also appeared to protect vulnerable nerve cells. Researchers found effects on processes involving inflammation and cellular stress, suggesting that sulforaphane may act on several pathways involved in the disease at the same time.
Kwa described Friedreich ataxia as a devastating condition that can gradually take away a child’s ability to walk, speak and play. Children make up a large proportion of affected patients, yet treatment choices remain limited.
The researchers believe sulforaphane has practical features that make it especially interesting. It has already been studied for safety in children and adults, and purified, biologically active forms of the compound are commercially available.
That does not mean sulforaphane is already an approved treatment for Friedreich ataxia. The next major step is to test it properly in people with the disease and determine whether the biological changes seen in research actually lead to better health.
Clinical trials would need to answer several questions. Researchers must learn what dose is needed, how long treatment should continue, whether it can slow loss of movement and other abilities, and whether there are important side effects during long-term use.
There is also an important difference between a compound found in food and a medical treatment made from that compound. The study does not show that simply eating more broccoli can prevent, reverse or cure Friedreich ataxia.
The strength of the findings is that sulforaphane appears to address more than one problem associated with the disease. Raising frataxin while reducing harmful stress and inflammation could make it a particularly interesting candidate for further study.
The biggest weakness is that encouraging laboratory evidence often fails to become an effective human treatment. The human body can absorb, process and distribute compounds differently from experimental systems, and a biological improvement does not always translate into slower disease progression.
Even so, the existing safety information and availability of purified sulforaphane could potentially shorten parts of the development process if future trials are successful. The researchers are now seeking support to move the work toward clinical testing.
For a disease affecting a relatively small number of families, greater awareness may be almost as important as the scientific discovery itself. More attention can bring research funding, patient participation and the clinical studies needed to determine whether this broccoli-derived compound can genuinely change lives.
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Source: Swinburne University of Technology.
