Decade-Old Compound Could Slow Huntington's Disease

Could there be a new way forward for Huntington's disease?
Could there be a new way forward for Huntington's disease?

An existing compound could offer hope for reducing symptoms of Huntington's disease (HD). The compound KD3010 has already gone through a Phase 1b clinical trial for diabetes without producing any adverse effects, so it has already been approved for human use.

“This is important since right now there is zero that can be done to alter the progression of this devastating disease,” said lead author the study Albert La Spada, MD, PhD. The study conducted by UC San Diego School of Medicine researchers, was published online in Nature Medicine.

This discovery of the drug as a potential treatment for HD comes after years of analyzing the inherited mutation in the huntingtin (htt) gene. This mutation misfolds htt proteins that gather in central nervous system cells and leads to a continued deterioration of movement control, cognitive decline and psychological problems.

In 2006, Dr. La Spada demonstrated how the htt mutated gene interferes with the regulatory protein PGC-1 which is central to the operation of mitochondria. Later in 2012, he found that PGC-1 disruption also inhibited brain cells' ability to degrade damaged molecules. With this discovery, Dr. La Spada tested elevated levels of PGC-1 in HD mice and found that it virtually eliminated the misfolded proteins and stopped neurological disease and neurodegeneration.

Testing the compound on mice last year, Dr. La Spada and his team witnessed significant improvements in neurodegeneration, “It also extended their lives by 16%,” he said. He is currently consulting with other researchers about a KD3010 clinical trial, which he hopes can begin within 18 months. If it proves to be successful, future tests on the compound's effectiveness for other related neurological diseases will be conducted.

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