The results of the research, conducted by the National Institutes of Health, appear in the New England Journal of Medicine.

Current Overall Response Rate Up

Earlier reviews of phase 1 oncology trials reported a rate of response to treatment of 4 to 6 percent and a toxicity-related death rate of 0.5 percent, according to the authors.

Christine Grady, R.N., Ph.D. and colleagues reviewed all nonpediatric phase 1 oncology trials sponsored by the Cancer Therapy Evaluation Program at the National Cancer Institute between 1991 and 2002.

The team reports the rates of response to treatment, of stable disease, of grade 4 toxic events, and of treatment-related deaths.

The analysis covered 460 trials involving 11,935 participants, all of whom were assessed for toxicity and 10,402 of whom were assessed for a response to therapy. The overall response rate -- that is, for both complete and partial responses -- was 10.6 percent, with considerable variation among trials.

Lower Rate in 'Classic' Phase 1 Trials

"Classic" phase 1 trials of single investigational chemotherapeutic agents represented only 20 percent of the trials and had a response rate of 4.4 percent.

Studies that included at least one anticancer agent approved by the Food and Drug Administration constituted 46.3 percent of the trials and had a response rate of 17.8.

An additional 34.1 percent of participants had stable disease or a less-than-partial response.

The overall rate of death due to toxic events was 0.49 percent. Of 3,465 participants for whom data on patient-specific grade 4 toxic events were available, 14.3 percent had had at least one episode of grade 4 toxic events.

Overall Death Rate Stable

Overall response rates among phase 1 oncology trials are higher than previously reported, the authors conclude, although they have not changed for classic phase 1 trials, and toxicity-related death rates have remained stable.

Rates of response and toxicity vary, however, among the various types of phase 1 oncology trials.