Wide Variety of Tumors
Tumors with a mutated tumor suppressor gene called PTEN are especially sensitive to mTOR inhibition. Mutations and deletions of PTEN are common in a variety of cancers, including pancreatic and ovarian cancers, melanomas, leukemias, and gliomas, Dr. Clackson said. So AP23573 could possibly be used to treat a wide variety of tumor types.
The drug is now undergoing studies required for regulatory filings to initiate clinical trials. "We hope to file the IND [investigational new drug] application by the end of this calendar year," Dr. Clackson said.
In some studies, the ability of AP23573 to shrink tumors exceeded that of rapamycin, its chemical cousin, Dr. Clackson said. AP23573, an analog of rapamycin, was synthesized using structure-based drug design. Unlike rapamycin, it can be given intravenously or orally. "The availability of an orally active mTOR inhibitor represents an important advance in the development of this promising new class of antitumor agents that function by inducing tumor starvation," he concluded.