New drug shows potential in slowing progress of Huntington’s disease

A potential new breakthrough in the treatment of Huntington's disease has been achieved with the discovery of a drug that could slow the condition's progression.

Researchers from University College London have tested a drug that can be injected into the spinal fluid to silence the gene responsible for generating the protein that causes damage to brain cells among people affected by the neurodegenerative disease.

For this study, 46 men and women with early-stage Huntington's disease were treated with the drug, with the concentration of harmful protein in the spinal cord fluid dropping significantly and in proportion with the strength of the dose.

This marks the first time that any therapy for this condition has successfully addressed the root cause of Huntington's, meaning this represents a significant breakthrough.

Lead researcher Professor Sarah Tabrizi told BBC News: "For the first time, we have the potential, we have the hope, of a therapy that one day may slow or prevent Huntington's disease."

The new drug was developed by Ionis Pharmaceuticals, which has sold the rights to the compound to Roche. Further clinical studies will now take place to assess the therapy's benefits.

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