New Cancer Drug Targets Hedgehog Signaling PathwayLast Updated: September 02, 2009. A new drug, GDC-0449, that targets the hedgehog pathway has shown promise in the treatment of basal-cell cancer and medulloblastoma, according to two reports and an editorial published online Sept. 2 in the New England Journal of Medicine.
WEDNESDAY, Sept. 2 (HealthDay News) -- A new drug, GDC-0449, that targets the hedgehog pathway has shown promise in the treatment of basal-cell cancer and medulloblastoma, according to two reports and an editorial published online Sept. 2 in the New England Journal of Medicine.
In one report, Daniel D. Von Hoff, M.D., of the Translational Genomics Research Institute in Scottsdale, Ariz., and colleagues randomized 33 patients with basal-cell carcinoma to receive 150 mg, 270 mg, or 540 mg daily doses of GDC-0449, a small-molecule inhibitor of smoothened homologue. In follow-up, 18 patients had an objective response with GDC-0449, while 15 patients had stable or progressive disease.
In a second report, Charles M. Rudin, M.D., of Johns Hopkins University in Baltimore, and colleagues analyzed tumor specimens of a 26-year-old man with refractory metastatic medulloblastoma and found evidence that the cancer involved activation of the hedgehog pathway and mutation of the patched homologue 1 (PTCH1) gene. Treatment with daily 540-mg doses of GDC-0449 resulted in rapid tumor regression and symptom reduction. However, while some lesions continued to respond to treatment, others resumed growth and the patient died approximately five months after treatment began.
"The two reports under discussion constitute compelling evidence that therapy directed against the hedgehog signaling pathway is a promising new approach for the treatment of advanced and metastatic basal-cell carcinoma, and they set the stage for studies using hedgehog pathway inhibitors in other cancers," write the authors of an accompanying editorial.
Both studies were supported by Genentech. Several authors from both studies and one of the two authors of the editorial reported receiving research grants from pharmaceutical companies.
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