Experimental Treatment Effective Against Aggressive Leukemia

Drug found to be as effective as current treatment without serious side effects.

Researchers are zeroing in on a means to overcome treatment resistance in the most aggressive forms of leukemia.

In a study published in a recent edition of the specialist journal Leukemia, German haematologists working with a Russian pharmaceutical company report on an experimental active substance that was found to effectively fight the most aggressive forms of Philadelphia chromosome-positive leukemia, both in vitro and in vivo.

Patients carrying the Philadelphia chromosome go on to develop chronic myelogenous leukemia (CML) or acute lymphatic leukemia (Ph+ ALL), which are the first types of the disease that are able to be treated due to targeted molecular therapy development.

Selective kinase inhibitor active substances, which directly treat the cancer-inducing BCR/ABL gene, become ineffective for many patients over time. The effect can be attributed to BCR/ABL mutations or other unknown mechanisms, the study noted.

Ponatinib is the only current substance that is able to overcome nearly all clinical resistance, but it also has some life-threatening side-effects.

An innovative experimental kinase inhibitor called PF-114 seeks to have the same effect on Ph+ leukemia as Ponatinib without the risky side-effects. Researchers found in the recent study that PF-114 treats resistant Philadelphia chromosome-positive leukemia as effectively as Ponatinib.

"These results provide the basis for the administration of PF-114 in treatment-resistant patients with Ph+ leukemia. The favorable efficacy and good side effect profile now need to be further tested on patients in clinical phase I studies," study co-lead Martin Ruthardt, MD, said in a press release.