New hope for the treatment of Kennedy’s disease
30 November 2006
Kennedy’s disease, also known as Spinal Bulbar Muscular Atrophy is a rare, slowly progressing form of MND that only affects men. In order to develop potential therapies for this condition, it is necessary to understand the underlying cause of the disease.
In a session on the opening morning of the 17th International Symposium on ALS/MND two methods for increasing our understanding of this condition were discussed.
The approach Prof Gen Sobue from Nagoya University School of Medicine, Japan, was to develop a mouse model of Kennedy’s disease, that showed both the physical symptoms of the human condition, and reflected the changes seen in the spinal cord of those affected by the disease.
One of the explanations of why Kennedy’s disease only affects men is that it affects the function of the male sex hormone testosterone. In animals, Prof Sobue’s data showed the extreme solution was to castrate them. However, he acknowledged that ‘castration is not a very nice therapeutic approach’ in humans and that a drug had been developed to reduce the levels of testosterone.
Recruitment is underway for a Japanese clinical trial to test the beneficial effects of this drug, measured by improvement in the swallowing ability. An earlier trial had shown that the drug was safe in people with Kennedy’s disease.
Genetic disease are sometimes classified according to whether the gene defect causes an additional toxic property, or whether the disease occurs as a result of the loss of normal function of the gene. To date, researchers investigating the cause of Kennedy’s disease have concentrated on the former.
In his presentation later in the same session on ‘Lessons from other motor neuron disorders’ Dr Thomas based at the University of Washington, Seattle used an elegant animal model to study the previously neglected area of the contribution of the loss of function of the gene that causes Kennedy’s disease. He and his colleagues found that this can lead to physical symptoms of weight loss and muscle weakness, they also observed changes to cells in the spinal cord.
In a session on the opening morning of the 17th International Symposium on ALS/MND two methods for increasing our understanding of this condition were discussed.
The approach Prof Gen Sobue from Nagoya University School of Medicine, Japan, was to develop a mouse model of Kennedy’s disease, that showed both the physical symptoms of the human condition, and reflected the changes seen in the spinal cord of those affected by the disease.
One of the explanations of why Kennedy’s disease only affects men is that it affects the function of the male sex hormone testosterone. In animals, Prof Sobue’s data showed the extreme solution was to castrate them. However, he acknowledged that ‘castration is not a very nice therapeutic approach’ in humans and that a drug had been developed to reduce the levels of testosterone.
Recruitment is underway for a Japanese clinical trial to test the beneficial effects of this drug, measured by improvement in the swallowing ability. An earlier trial had shown that the drug was safe in people with Kennedy’s disease.
Genetic disease are sometimes classified according to whether the gene defect causes an additional toxic property, or whether the disease occurs as a result of the loss of normal function of the gene. To date, researchers investigating the cause of Kennedy’s disease have concentrated on the former.
In his presentation later in the same session on ‘Lessons from other motor neuron disorders’ Dr Thomas based at the University of Washington, Seattle used an elegant animal model to study the previously neglected area of the contribution of the loss of function of the gene that causes Kennedy’s disease. He and his colleagues found that this can lead to physical symptoms of weight loss and muscle weakness, they also observed changes to cells in the spinal cord.
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Notes to editors
The 17th International Symposium on ALS/MND is taking place in Yokohama, Japan, between Thursday, 30 November and Saturday, 2 December. The event is organised by the UK-based Motor Neurone Disease (MND) Association and hosted this year by the Japanese ALS Association (JALSA).
