Clinical trial delivers good results in leukemia patients

November 30, 2012

Huntsman Cancer Institute (HCI) researchers Michael Deininger, M.D., Ph.D., and Thomas O'Hare, Ph.D., were part of a team that found a potent oral drug, ponatinib, effective in patients who have developed resistance to standard treatments for chronic myeloid leukemia (CML) and Philadelphia chromosome positive acute lymphoblastic lymphoma (Ph+ ALL). The New England Journal of Medicine released results of the trial today.

In the phase I clinical trial conducted at five cancer centers nationwide, ponatinib was highly active in patients with CML and Ph+ ALL who had developed resistance to currently approved (TKIs), the standard treatment for CML and Ph+ ALL. Ponatinib is a rationally developed drug, designed in the labs of study sponsor ARIAD Pharmaceuticals, Inc. to address limitations of currently available treatments. Deininger, professor and chief of Hematology and at Huntsman Cancer Institute at the University of Utah, and O'Hare, a research associate professor, helped to develop ponatinib, and predicted its efficacy against all known mutant forms of BCR-ABL1, the abnormal protein tyrosine kinase that causes CML.

"Ponatinib is arguably the most potent and broadest BCR-ABL1 available thus far, covering even the T315I mutant, which is completely resistant against all approved TKIs", says Deininger, a senior author on the study who leads an ongoing ponatinib trial at Huntsman Cancer Institute. "The results of this study as well as preliminary data from a larger phase 2 trial show that ponatinib has remarkable activity in patients with resistant CML and Ph+ ALL, suggesting that this new TKI will expand our therapeutic armamentarium very significantly. It is a poster child for personalized cancer therapy."

Explore further: New drug shows potential for treatment-resistant leukemia

Related Stories

New drug shows potential for treatment-resistant leukemia

April 11, 2011

A study from Tufts Medical Center researchers published today finds that a novel drug shows promise for treating leukemia patients who have few other options because their disease has developed resistance to standard treatment.

Recommended for you

Current cancer drug discovery method flawed, study finds

May 2, 2016

The primary method used to test compounds for anti-cancer activity in cells is flawed, Vanderbilt University researchers report May 2 in Nature Methods. The findings cast doubt on methods used by the entire scientific enterprise ...

Shape of tumor may affect whether cells can metastasize

April 28, 2016

Only a few cells in a cancerous tumor are able to break away and spread to other parts of the body, but the curve along the edge of the tumor may play a large role in activating these tumor-seeding cells, according to a new ...

0 comments

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.