DEC 13, 2017 2:35 PM PST

Study Finds Promise for Huntington's in a Cancer Drug


Image credit:

The most intricate man-made origami structure may pale in comparison to the body’s natural origamis: 3-dimenstional proteins. The folding of amino acids in specific arrangements create highly sophisticated proteins in composition, structure, and also function.

When this orchestrated process goes right, human biology is a well-oiled machine. However, when the folding is off even for just a little bit, the results can be devastating. In the case of Huntington’s disease, the huntingtin protein is misfolded causing the proteins to clump in the brain. These turn into toxic clusters that interfere with the function of neurons and leading to the mental and physical decline in patients.

Huntington’s disease is a neurodegenerative disorder that is progressive and fatal. In patients with the disease, the huntingtin protein is misfolded and these proteins form clumps in the brain. In turn, these proteinaceous clumps turn into toxic clusters that interfere with the function of neurons and leading to the mental and physical decline in patients. In addition to finding a cure for this tragic disease, researchers are also working to slow its progression.

Spurred by previous results involving repurposed drugs, researchers wanted to test whether a cancer drug can help patients with Huntington’s disease.

The research team involved top scientists from Duke University, the University of California, San Diego, the Salk Institute in La Jolla, CA, and the Johns Hopkins University in Maryland.

In this study, the repurposed drug is bexarotene (trade name: Targretin), an anti-cancer drug approved for the treatment of cutaneous T cell lymphoma. While this retinoid drug is FDA-approved for skin lymphoma, it has been prescribed off-label for other cancer types, including non-small cell lung cancer and breast cancer.

Using a mouse model of Huntington’s disease, the research team tested the effects of bexarotene on the marked degeneration typically associated with the disease.

Mice treated with bexarotene showed remarkable changes, including “improved motor function, reduced neurodegeneration, and increased survival.” These protective qualities appeared to stem from the drug’s ability to correct the oxidative metabolism in Huntinton’s disease neurons. Bexarotene also seemed to improve cell quality control. Overall, the drug seemed to correct activities that would otherwise be dysfunctional in Huntington’s disease.

"It's not just the response from the drugs, but the mechanistic pathways these drugs are targeting," said Dr. Albert La Spada at Duke University. "These pathways are relevant to other neurodegenerative disorders and potentially the aging process, itself in addition to Huntington's disease."

Although the results are promising, the team cautions that further studies are required before the rug can be confidently prescribed to help Huntington’s patients. Furthermore, the team thinks bexarotene may be more potent if combined with other therapies. In particular, preliminary tests in cells show that bexarotene taken with KD3010, a diabetes compound, yielded better results.

"With this approach, we could minimize side effects with lower doses of each compound, even when together the treatments provide a higher effect than either one alone,” said Dr. Audrey Dickey, the study’s lead author.

Additional sources: MNT

About the Author
  • I am a human geneticist, passionate about telling stories to make science more engaging and approachable. Find more of my writing at the Hopkins BioMedical Odyssey blog and at
You May Also Like
NOV 23, 2019
NOV 23, 2019
Can lithium heal damage from radiation?
Research suggests that lithium could play a role in minimizing the negative effects of radiation on the brain. The research was published in Molecular Psyc...
DEC 08, 2019
DEC 08, 2019
Why do colder, wetter climates have a higher prevalence of cancer?
You might want to sit down, because this news may come as a surprise: researchers have found an association between cold, wet climates and increased cancer...
DEC 23, 2019
Health & Medicine
DEC 23, 2019
Are Popular Gyms Promoting Indoor Tanning?
You might assume that by now, most humans are aware of the dangers of indoor tanning beds. However, many popular gyms include tanning beds as a perk of mem...
DEC 25, 2019
Drug Discovery & Development
DEC 25, 2019
New Drug to Make Breast Cancer Treatment More Affordable
The US Food and Drug Administration has granted accelerated approval to new breast cancer drug, trastuzumab deruxtecan. The drug’s increasing recogni...
JAN 20, 2020
JAN 20, 2020
Open-Source Software Judges The Accuracy of Cancer Predicting Computer Programs
Cancers are generally composed of diverse cells that vary in genetics—these variations often make a particular cancer more susceptible or resistant t...
FEB 14, 2020
FEB 14, 2020
Rewired natural killer cells show promising results in leukemia patients
Natural killer (NK) cells are a subset of white blood cells that are key players in the innate immune system, orchestrating host-rejection responses agains...
Loading Comments...