Novel Cancer Drug Brings Hope for Leukemia Patients

A new cancer drug was found safe for cancer patients, according to the results of a phase I clinical trial about a new drug targeting mitochondrial function in human cancer cells. Researchers at Wake Forest Baptist Medical Center have discovered that this could be the first drug to inhibit mitochondria in this way and prove effective in further clinical trials to help fight this devastating form of blood cancer.

"This drug is selectively taken up by cancer cells and then shuts down the production of energy in the mitochondria," said Timothy Pardee, M.D., Ph.D., director of leukemia translational research at Wake Forest Baptist and principal investigator of the trial, in a news release.

The powerhouse of the cell as scientist refer to mitochondria, is used differently by healthy cells versus cancerous ones. For instance, healthy cells use mitochondria as a source of energy to carry out essential functions, whereas leukemia cells use it to reproduce faster and repair damage from chemotherapy.

For the findings, researchers examined 26 patients in a Phase I trial with relapsed hematological malignancies who were enrolled and given the drug CPI-613 via a two-hour infusion on days one and four for three weeks every 28 days. Of the 21 patients who completed the trial, 29 percent saw a benefit from the drug, including those who achieved a complete remission that lasted more than three years and another who had a partial response that lasted more than two years.

Researchers believe that the data suggests that agents targeting mitochondrial metabolism can be safely administered with activity in blood-related cancers, such as leukemia.

At this time, a Phase II clinical trial is currently underway.

More information regarding the findings can be seen via the journal Clinical Cancer Research.