CPI-613 safe and somewhat efficacious for patients with leukemia

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According to a new study published in the journal Clinical Cancer Research, a phase 1 study has demonstrated that a novel investigational drug, known as CPI-613, was safe and somewhat efficacious for the treatment of patients with relapsed or refractory leukemia. In the study, researchers enrolled 26 patients with relapsed or refractory hematological cancers and administered CPI-613 on days 1 and 4 weekly for 3 weeks of a 28-day cycle.


Of 21 evaluable patients, 29% experienced some benefit from the treatment. One patient achieved a complete response and that patient's remission has lasted over 3 years. Another patient achieved a partial response with a remission that lasted greater than 2 years.


CPI-613 is the first drug to inhibit the production of energy in the mitochondria of cancer cells, thereby inhibiting proliferation and damage repair from chemotherapy. The drug is currently being studied in phase 2 studies in both solid tumors and hematological malignancies, and it was granted orphan drug status by the U.S. Food and Drug Administration (FDA) for pancreatic cancer. CPI-613 is being developed by Cornerstone Pharmaceuticals.

CPI-613 safe and somewhat efficacious for patients with leukemia
CPI-613 safe and somewhat efficacious for relapsed or refractory leukemia.

Results of a Phase I clinical trial showed that a new drug targeting mitochondrial function in human cancer cells was safe and showed some efficacy. The findings, reported by doctors at Wake Forest Baptist Medical Center, are published in the current online edition of the journal Clinical Cancer Research."

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. "This is the first drug to inhibit mitochondria in this way and if it proves effective in further clinical trials, it will open up a whole new approach to fighting cancer."

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