Targeted mitochondrial attack destroys glioma cells in preclinical study

Print 26 March 2015
John Carroll / FierceBiotech

Zeroing in on an enzyme that is over-expressed in brain tumor cells, two investigators at Houston Methodist say they have developed a new treatment for gliomas that has cleared its preclinical testing and should be ready for human testing within the next one to two years.

Using an in vitro test, the scientists say that their therapy--MP-MUS--destroyed anywhere from 90% to 95% of malignant glioma cells while leaving healthy cells largely undisturbed. 

The therapy is designed to attack cells' mitochondria, its power source. And the investigators say that an upcoming publication of a mouse study on the same drug will show that it worked in an animal model. That progress should open the door to human testing in 2016 or 2017, they add, after completing planned tox studies for regulators.

"We are very optimistic that we'll get there," said David Baskin, the vice chair of the Department of Neurosurgery at Houston Methodist Hospital. "Our past work has shown that MP-MUS has very low toxicity until it gets into tumor cells. Once it arrives, it is changed to its active form, doing a lot of damage where we want it to, leaving healthy brain cells alone--a bit like a 'smart bomb.' To our knowledge, this is the first known example of selective mitochondrial chemotherapy, which we believe represents a powerful new approach to brain cancer."

The investigators have a deal to develop this therapy with Virtici, a Seattle-based group that works with academic investigators on translational medicine projects.

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