Iranian scientist develops drug for treatment-resistant leukemia

An Iranian researcher Anahita Rafiei along with hematologists from Goethe University Frankfurt, working with a Russian pharmaceutical company, have developed a new active substance that effectively combats the most aggressive forms of Philadelphia chromosome-positive leukemia.

The chances of patients with Philadelphia chromosome-positive leukemia (Ph+) being cured has greatly increased in recent years.

Nevertheless, a high percentage of patients have developed resistance to available medication. But now, hematologists from Goethe University Frankfurt, working with a Russian pharmaceutical company, have developed a new active substance that effectively combats the most aggressive forms of Philadelphia chromosome-positive leukemia, both in vitro and in vivo.

The research was reported in the most recent edition of the specialist journal ‘Leukemia’.

Patients with the Philadelphia chromosome develop chronic myelogenous leukemia (CML) or acute lymphatic leukemia (Ph+ ALL).

These are the first types of leukemia that are treatable thanks to the development of targeted molecular therapy.

Selective kinase inhibitor active substances act directly on the cancer-inducing gene BCR/ABL. However, after a while, the treatment becomes ineffective for many patients — either due to BCR/ABL mutations or as a result of other mechanisms that are as yet unknown.

At present, there is only one substance, Ponatinib, which is able to overcome nearly all clinical resistance. Unfortunately, Ponatinib can only be used with extreme caution due to some of its life-threatening side-effects.

“These results provide the basis for the administration of PF-114 in treatment-resistant patients with Ph+ leukemia. The favorable efficacy and a good side effect profile now need to be further tested on patients in clinical phase I studies,” the researchers said.

“PF-114 would not have reached this level of development without our colleagues in Frankfurt. On the basis of this data, in the first half of 2015, we will be able to start international phase I studies.”