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Molecular Therapy Holds Promise in the Treatment of Amyotrophic Lateral Sclerosis

manageable, chronic disease."

Within a year, Cleveland hopes the first clinical trial will be initiated in humans. In order to deliver the antisense drug directly to the nervous system, surgeons will insert a small pump into a patient using a fairly routine surgery that has already been approved for management of pain. A small catheter is then implanted into the area surrounding the spinal cord, in order to pump antisense oligonucleotide drugs directly into the nervous system.

The investigators noted that if the antisense approach works for ALS – by delivering therapeutic agents for neurodegenerative diseases across the highly impermeable blood-brain barrier – it would likely also work in other neurodegenerative conditions, including Alzheimer's, Parkinson's and Huntington's diseases.

"We know we're on target with the pathogenic mechanism," said Cleveland. The remaining question is whether the genetic-based therapy will be tolerated. "If tolerated, this sets the stage for broader treatment of neurodegenerative disease, especially Huntington's disease, where there is currently no treatment, but key genes involved in promoting disease are known."

Source :Eureka

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