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Malaria drug gets new use attacking brain cancer

A malaria drug is doing something it’s never done before: treat brain cancer.

When 26-year-old Lisa Rosendahl was diagnosed with an aggressive glioblastoma, a condition for which the survival rates are dismal, she tried every treatment plan available. Over time her brain became resistant to chemotherapy and targeted treatments. It wasn’t until her doctors included a malaria drug, chloroquine, that her quality of life improved, and four weeks later she was able to stand and regain control of her limbs.

The science behind this off-label use arose from the lab of Andrew Thorburn, deputy director of the University of Colorado Cancer Center, Aurora, starting in 2009. Thorburn’s lab studies the cellular process of autophagy, a process of cellular recycling in which the cell organelles, called autophagosomes, encapsulate extraneous or dangerous material and dispose of it. This process breaks down unneeded cellular components into building blocks of energy or proteins for use in times of low energy.

The treatment works because chloroquine stops this essential cellular process, re-sensitizing her cancer to the targeted treatments that had previously been effective. This treatment was effective for Rosendahl because her cancer had a mutation of the BRAF gene, making it especially dependent on autophagy. However, since the success of her treatment, the drug combination has been successfully used on two other brain cancer patients, who saw similar dramatic improvements.

“Based on our results, we hypothesize that by targeting an entirely different cellular process, i.e., autophagy, upon which these same tumor cells rely, it may be feasible to overcome such resistance and thus re-establish effective tumor control,” the paper published in the journal eLife states.

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