A new drug has shown a lot of potential to fight chronic myeloid leukemia (CML), which has become resistant to regular treatment.

According to the study conducted by the Tufts Medical Center, the drug named DCC-2036 decreased the mutant enzyme, BCR-ABL1 in human leukemia cells. However, in mice, the drug wiped out the cancer cells and increased survival rates. The study is the first published report to appear in the journal Cancer Cell.

DCC-2036 is a tyrosine kinase inhibitor (TKI) – a class of drugs that block the action BCR-ABL1. Other TKI drugs such as imatinib (Gleevec) noticeably improved the diagnosis for patients with CML, but about a third of patients are known to suffer serious setbacks mainly due to mutations that make BCR-ABL1 resistant to the TKI. Those patients may simply have to resort to bone marrow transplantation.

The drug has been developed by Deciphera Pharmaceuticals LLC and the crystal structure of the enzyme was calculated so the drug could be precisely made for the mutant enzyme.

Richard Van Etten, MD, PhD, Director of Tuft’s Medical Center’s Cancer Center has stated the findings demonstrate that DCC-2036 is an exceptional entrant for clinical development as a treatment for resistant CML. He also pointed out that not all drugs that work in a test tube will be successful in a living organism such as the mouse model.

DCC-2036 is presently being tested in a phase 1 clinical trial among patients who have been unsuccessful with two other TKIs. The trial is pro-actively taking on patients at Tufts Medical Center, MD Anderson Cancer Center, and University of Michigan Cancer Center.

The study exemplifies the power of designing drugs based upon structures of the target and preliminary testing of these drugs in mouse models before taking them to the clinic. It also shows how cancer treatments will be developed in the 21st century.

What is CML?

Chronic myeloid leukemia (CML) is an uncommon cancer that causes the body to make too many white blood cells. It usually develops very slowly, and that’s why it is also called ‘chronic’ leukemia.

It starts in the bone marrow in a special type of blood cell called a stem cell. CML can occur at any age; however it is more common in middle-aged and older people and is rare in children.

Chronic myeloid leukemia CML is diagnosed in approximately 5000 people in the U.S. on a yearly basis and a third of patients who relapse have to go through a bone marrow transplant.

In the UK, about 700 people are diagnosed with CML annually.

1) National Cancer Institute
2) Macmillan Cancer Support