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Mesothelioma Treatment Breakthrough May Come From Another Rare Disease

Veterans Affairs Mesothelioma Treatment

Mesothelioma patients may someday benefit from research into Huntington’s Disease, another rare disease. Researchers discovered that patients with Huntington’s Disease have a significantly less chance than the general population of getting cancer. Now, the researchers report that better understanding what it is about the disease that can kill off cancer cells could lead them to a breakthrough treatment for all types of cancer.

A team of researchers from Northwestern University found that the gene that drives the progression of Huntington’s Disease, a rare neurodegenerative disorder, is also highly toxic to cancer cells. The huntingtin gene, that plays an important role in neurons in the brain, can become mutated by an over abundance of repeating RNA sequences, leading to the rare disorder. The nerve cells of the brain are vulnerable to these deadly genes, and, according to the researchers, cancer cells are even more susceptible to the genes. In fact, the scientists have dubbed it the “super assassin gene” due to the fact that it is so toxic that it kills off cancer cells.

“This molecule is a super assassin against all tumor cells,” said senior author Marcus Peter, the Tom D. Spies Professor of Cancer Metabolism at Northwestern University Feinberg School of Medicine. “We’ve never seen anything this powerful.”


In the United States, a rare disease status is assigned to a disease or disorder if it affects fewer than 200,000 Americans at any given time. There are approximately 30,000 cases of Huntington’s Disease in the U.S. An estimated 3,000 Americans are diagnosed with mesothelioma each year.

Huntington’s Disease deteriorates a person’s physical and mental abilities, most often affecting adults between the ages of 30 and 50. There is no cure for the  fatal genetic disorder. Mesothelioma, an asbestos-caused cancer, typically affects men in their 60’s or older who worked around asbestos decades earlier. Mesothelioma is also an incurable, fatal condition.

The researchers found a way to deliver the molecule as a treatment compound via a nanoparticle, a microscopic drug delivery system, to mice models with human ovarian cancer tumors. The team reported three key results:

  • The tumor growth was “significantly reduced;”
  • There was no toxicity to the mice; and
  • The tumors did not build up resistance to the drug.

Pleased with the results, the team will begin to explore using the molecule as a novel form of anticancer reagents. There is much more research to be done, however, the team is hopeful that this breakthrough will be useful on all types of cancer, possibly even mesothelioma.

The study was published in the Feb. 12 issue of the journal EMBO Reports.


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