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Neuro Innovations - A Rare Chance to Cure A Difficult Cancer

Collaborations in Discovery

A Rare Chance to Cure A Difficult Cancer

Date: November 1, 2011

Ziya Gokaslan, Johns Hopkins Neurosurgery
Ziya Gokaslan

Ziya Gokaslan tells the graduate students who come to join his research at the Johns Hopkins Neurosurgical Spine Center that they have a shot at something that most researchers can only dream about: developing a full cure for a deadly form of cancer. “That’s not something we can say very often,” explains Gokaslan.

The target of Gokaslan’s efforts is chordoma, a malignant tumor that grows along the spine anywhere between the skull and the tailbone, gradually damaging nearby nerves and bone, causing pain, weakness and a loss of function. Gokaslan’s team is attacking these life-threatening tumors—diagnosed in about 300 people each year in the United States—on several fronts. Because the tumors surround and invade the spine, surgically removing one of these tumors whole is difficult. And if the tumor ruptures, it will recur. So Gokaslan and colleagues have helped develop a surgical technique that involves coming at the tumor from several different directions. “Each different angle gives us a better shot at safely freeing up one part of the tumor, so we can get it out in one piece,” says Gokaslan. “Then we can reconstruct part of the spine if it’s damaged, so the patient can regain mobility.”

Gokaslan and colleague Jean-Paul Wolinsky have also helped develop new surgical techniques for patients whose tumors are too entwined with bone and tissue to be removed whole, often the case with tumors at the base of the skull. Guided by advanced computer imaging, he can remove the tumor in pieces through a tube inserted in the neck, avoiding damage to the face and jaw that’s otherwise typical of the surgery. He’s also pioneering the use of radiation consisting of focused beams of protons to demolish the tumors, cutting treatment times down from two months to two days, and reducing the chances of recurrence.

What most excites Gokaslan, though, is the work his team and colleague Alfredo Quiñones-Hinojosa have done with the single, overactive gene believed to be mostly responsible for chordoma. That gene provides a unique opportunity, because most cancers are caused by activity in 200 or more genes, presenting too complex a target. Gokaslan and colleagues have helped identify compounds that, when injected into mice that have chordomas, seem to shut the gene down and stop tumor growth. “We’re still exploring different strategies,” says Gokaslan. “But having a way to silence this gene, combined with better surgery and radiation, means we have a real chance to cure chordoma.”

  • Challenge: Remove chordomas cleanly and less invasively—and even cureing them
  • Approach: Guide surgery with computers, and shut down the gene that causes them
  • Progress: Chordomas are coming out intact, and drugs have stopped
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