RAD001

In a poster presentation, Jaroslaw Jac reported on a Phase 2 study of RAD001 (everlolimus) at a single institution, the Methodist Hospital in Houston, TX (this is a Robert Amato study). RAD001 is an mTOR inhibitor, where mTOR is a protein that can promote tumor cell growth and proliferation and angiogenesis. 41 patients were enrolled in the trial with 37 evaluable.

The treatment plan was 10 mg daily reduced to 5 mg for grade 3/4 toxicities. Over 80% of the patients had failed prior systemic therapy. Only two patients were non-clear cell.

There were 12 dose reductions, 7 due to pneumonitis (inflammation of the lungs). There were no other single significant grade 3/4 toxicities. For the 37 evaluable patients the response was.

RAD001 Response


Patients (%)

Partial Response (PR)

12 (32%)

Stable Disease (SD)

14 (38%)

Progressive Disease

11 (30%)

Median Duration of PR

23.8 mos.

Median Duration of SD

12.2 mos.

Progression-Free Survival

11.2 mos.

Median Survival

24.2 mos.

The authors conclude that RAD001 is well tolerated, shows a high level of anti-tumor activity given the partial response of 32%, and that mTOR is an appropriate therapeutic target in RCC. There was no mention of independent radiologic confirmation of the results. The Methodist group plans to enroll an additional 40 patients who have failed prior Nexavar or Sutent therapy.

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