Breakthrough Drug May Reverse Huntington's Disease Symptoms

Treatment in animal subjects stopped the effects of Huntington’s and produced long-term improvement

A single drug treatment may be able to slow down and silence the mutated gene responsible for the incurable Huntington’s disease, according to researchers at UC San Diego.

The disease is caused by the mutation of a single gene, causing toxic proteins to accumulate throughout the brain. Huntington’s affects roughly 30,000 Americans and people diagnosed with it often have uncontrolled movements and psychiatric problems. It often results in death. There is currently no effective treatment and Huntington’s disease.

Doctors from the Ludwig Institute for Cancer Research found that drug therapy that was tested in primates could help people with moderate to severe forms for the disorder. Treatment in mouse and primate subjects proved to have rapid results in destroying the mutation that causes the toxic protein in the brain.

In the study, animals with a form of Huntington’s disease were injected once with the advantageous drug. The animals began moving better within one month and achieved normal motor function within two – and the benefit persisted nine months after the drug was cleared from their systems.

“These findings open up the provocative possibility that transient treatment can lead to a prolonged benefit to patients,” Dr. Don W. Cleveland, author of the study, said in a prepared statement.

He also noted that the approach was particularly promising because this type of therapy has already been proven safe in clinical trials.

UCSD’s findings are published in the June 21 print issue of the journal Neuron and funding for this research partially came from the CHDI Foundation.

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