Before It's Too Late: Multistakeholder Perspectives on Compassionate Access to Investigational Drugs for Pediatric Patients With Cancer.

Patients and their families, physicians, drug companies, and regulatory agencies have common goals: to find effective therapies for life-threatening conditions. In oncology, the lines between clinical research and treatment are often blurred; parents and physicians of patients who have exhausted standard-of-care treatments and cannot participate in a clinical trial are likely to consider seeking compassionate use access to investigational drugs; however, knowledge and perspectives about compassionate use may differ among these groups. There are unique considerations associated with providing compassionate use to children diagnosed with cancer, including evaluation for potential developmental toxicities, the need for pediatric-specific dosing and formulations, informed consent, and, when appropriate, patient assent.

Phase I trial of combretastatin a-4 phosphate with carboplatin.

Purpose: Preclinical evidence of synergy led to a phase I trial employing combretastatin A-4 phosphate (CA4P), a novel tubulin-binding antivascular drug, in combination with carboplatin.

Experimental Design: Based on preclinical scheduling studies, patients were treated on day 1 of a 21-day cycle. Carboplatin was given as a 30-minute i.

Future directions with angiogenesis inhibitors in colorectal cancer.

The recent US Food and Drug Administration's approval of bevacizumab has reinvigorated antiangiogenic research and has moved colorectal cancer to the forefront of study for the most promising drug candidates in this class. Predicting future directions in this field requires a return to the challenges of the past. Antiangiogenic drugs have necessitated new study design paradigms and imaging techniques in the assessment of drug activity and in dose selection.