Huntington Disease May Hold Key to Novel Cancer Treatment
The mutated huntington (HTT) gene causes the body to generate a class of small molecules that are toxic to cancer cells but not most healthy cells, according to the results of a new study published in EMBO Reports (https://doi.org/10.15252/embr.201745336). The researchers went as far as to call it a “super assassin against all tumor cells.”
Patients with Huntington disease have an 80% lower risk of cancer than the general population, and the researchers developed a murine study to better determine how the HTT gene reduces risk. HTT is present in every cell of the body for healthy patients as well as those with Huntington disease. When mutated, the gene produces small interfering RNAs that attack the brain’s nerve cells in Huntington disease—but also cancer cells.
The researchers delivered the molecule in nanoparticles to mice with human ovarian cancer. The treatment significantly reduced tumor growth with no toxicity to the mice, and the tumors showed no sign of developing resistance. The researchers also found that it killed all cancer cells in subsequent tests for human and mouse ovarian, breast, prostate, liver, brain, lung, skin, and colon cancer cell lines.
Additional work is being done to refine the treatment’s efficacy in reaching a tumor and to stabilize the nanoparticles for storage. The researchers believe that their findings may result in a novel treatment that could be administered on a short-term basis to kill cancer but not long enough to cause the neurologic damage associated with Huntington disease.