Experimental Treatment Effective Against Aggressive Leukemia

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

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.