An ingredient naturally found in broccoli may one day help treat a rare inherited disease that slowly damages the nervous system.
Researchers at Swinburne University of Technology have found that the plant compound can act on several of the biological problems involved in Friedreich ataxia.
Friedreich ataxia is a rare genetic condition that mainly affects the brain, spinal cord and nerves. Over time, it can make walking, speaking and carrying out everyday activities increasingly difficult.
The disease often begins during childhood or the teenage years, although symptoms can sometimes appear later. Around 200 people in Australia are estimated to live with the condition, making it unfamiliar to much of the public.
Friedreich ataxia is caused by changes in a gene responsible for producing a protein called frataxin. People with the disease do not make enough of this important protein, which helps cells manage iron and produce energy properly.
When frataxin levels are too low, cells can become damaged by chemical stress and problems with energy production. Nerve cells are particularly vulnerable, helping explain why movement, balance and coordination gradually worsen.
The condition can also affect other parts of the body. Some patients develop heart problems, diabetes, hearing loss or changes in vision, and the severity of the disease can vary considerably between individuals.
There is currently no cure, and treatment largely focuses on managing symptoms and complications. The lack of treatment options is especially difficult for children and their families because the disease can continue to progress for many years.
Researchers led by Associate Professor Faith Kwa at Swinburne investigated a natural compound called sulforaphane. Sulforaphane is found in vegetables such as broccoli and is especially associated with broccoli sprouts and other vegetables in the same plant family.
The study found that sulforaphane could increase levels of frataxin, the protein that is deficient in Friedreich ataxia. It also helped protect vulnerable nerve cells from damage in the researchers’ experimental models.
The compound appeared to work on more than one part of the disease process. In addition to improving frataxin levels, it affected cellular stress and inflammation, two processes that may contribute to damage in Friedreich ataxia.
The research was published in the journal Antioxidants & Redox Signaling. The findings provide early evidence that sulforaphane deserves further investigation as a possible treatment rather than showing that eating broccoli can treat the disease.
Kwa said Friedreich ataxia can progressively take away a child’s ability to walk, speak and participate independently in everyday life. She believes a treatment that acts on the underlying biology of the disease could potentially improve quality of life and long-term outcomes.
One possible advantage of sulforaphane is that scientists already know considerably more about its safety than they would about a completely new drug. Purified forms of the compound are available, and it has previously been studied in both adults and children.
That could make future development easier if clinical trials show that the treatment works. However, a compound being considered safe in earlier research does not guarantee that it will be effective against Friedreich ataxia or establish the correct dose for patients.
The researchers are now hoping to secure funding for clinical trials. Such trials would need to test whether the encouraging laboratory findings translate into meaningful improvements in people living with the disease.
The rarity of Friedreich ataxia creates another challenge. Rare diseases often receive less public attention, and recruiting enough patients for large clinical trials can be difficult, which can slow the development of new treatments.
The study is promising because sulforaphane appears to influence the central protein problem in Friedreich ataxia while also protecting cells from other forms of damage. A treatment capable of acting on several disease processes could have advantages over one that addresses only a single effect.
Still, these results should be viewed as an early step rather than evidence of a new treatment ready for patients. Clinical studies must establish whether purified sulforaphane reaches the right tissues, improves symptoms or slows disease progression, and remains safe when used at therapeutic doses over time.
The research also should not be interpreted to mean that eating large amounts of broccoli will raise frataxin enough to treat Friedreich ataxia. The amount, preparation and delivery of a medical treatment can be very different from normal dietary exposure.
If future trials are successful, however, an already available natural compound could offer an unusually practical path toward treatment. For families affected by this life-limiting disease, that possibility gives researchers a strong reason to continue the work.
If you care about nutrition, please read studies about the harm of vitamin D deficiency, and Mediterranean diet may preserve brain volume in older adults.
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Source: Swinburne University of Technology.
