The new study was a phase 1 study, which means it was designed only to assess the safety of the medication, not its effectiveness.
The initial safety study had 70 patients with one of the three ALK-driven cancers. Mosse said the drug was extremely well-tolerated, with mostly mild side effects, such as diarrhea or nausea. She said some children had mild vision changes, but there was no significant effect on sight, and some children had changes in the results of liver function tests.
Although the study wasn't designed to measure the drug's effectiveness, its benefits quickly became apparent, especially for anaplastic large cell lymphoma cancers. Eighty-eight percent (seven out of eight children with an ALK abnormality) had a complete response to the drug, and now have no detectable disease. At this point, the study volunteers have been on the drug for as long as 18 months, the researchers noted.
Seven children with inflammatory myofibroblastic tumors were enrolled in the trial, and the majority have had either tumor shrinkage or complete tumor regression. Said Mosse: "Right now, the only effective way to cure this type of cancer is to remove it surgically. If you can't remove it all, it comes back. Our first patient had a tumor in the shoulder and amputating the entire arm was the only cure possibility they were given. We gave this patient a very low dose and saw a dramatic benefit of the drug."
Neuroblastomas are proving to be more of a challenge. The researchers found two of 27 study volunteers showed complete tumor regression. Eight have had no disease progression. Giving higher doses of crizotinib seemed to be more effective, and it was still well-tolerated.
Mosse said the drug will go to phase 2 testing for neuroblastomas so researchers can try to distinguish who might respond best to the drug. The researchers also wan
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