Queensland University of Technology, School of Life Sciences, Faculty of Science and Technology, GPO Box 2434, QLD4001, Brisbane, Australia.
Expert Opin Investig Drugs. 2010 Mar;19(3):451-4. doi: 10.1517/13543780903571649.
The hedgehog signalling pathway is vital in early development, but then becomes dormant, except in some cancer tumours. Hedgehog inhibitors are being developed for potential use in cancer.
OBJECTIVES/METHODS: The objective of this evaluation is to review the initial clinical studies of the hedgehog inhibitor, GDC-0449, in subjects with cancer.
Phase I trials have shown that GDC-0449 has benefits in subjects with metastatic or locally advanced basal-cell carcinoma and in one subject with medulloblastoma. GDC-0449 was well tolerated.
Long-term efficacy and safety studies of GDC-0449 in these conditions and other solid cancers are now underway. These clinical trials with GDC-0449, and trials with other hedgehog inhibitors, will reveal whether it is beneficial and safe to inhibit the hedgehog pathway in a wide range of solid tumours or not.
hedgehog 信号通路在早期发育中至关重要,但随后会休眠,除了在一些癌症肿瘤中。 hedgehog 抑制剂正在被开发用于癌症的潜在用途。
目的/方法:本评估的目的是回顾 hedgehog 抑制剂 GDC-0449 在癌症患者中的初步临床研究。
I 期试验表明,GDC-0449 对转移性或局部晚期基底细胞癌患者以及一名髓母细胞瘤患者有疗效。GDC-0449 耐受性良好。
目前正在进行 GDC-0449 在这些疾病和其他实体瘤中的长期疗效和安全性研究。这些 GDC-0449 的临床试验以及其他 hedgehog 抑制剂的临床试验将揭示抑制 hedgehog 通路在广泛的实体瘤中是否有益和安全。