A drug for CML: Studies are Initiated
Fusion Pharma has received an approval for phase I CT of chronic myeloid leukemia (CML) drug.
Huge preliminary work has been made, including preclinical studies, protocol design, specialist training. Much depends on results of the study. PF-114 is intended for treatment of resistant forms of CML, including a T315I mutation form with no response to treatment with most authorized drugs.
Philadelphia chromosome is known to emerge in leukemia cells of CML as a result of chromosome translocation. It leads to emergence of chimeric oncogene BCR-ABL playing a key role in uncontrolled cell division and disease development. The product of pathological gene is the target for modern target therapy.
PF-114 is developed as a target drug inhibiting the activity of that chimeric protein.
Within the framework of the planned phase I study it is suggested to study safety and efficacy of the product in resistant patients, to search for its optimal dose. The study should begin at the end of May – early June 2016 at the Hematology scientific center of the Ministry of Health of the Russian Federation in Moscow. The project is also expected to involve V.A. Almazov North-Western Federal Medical Research Center in Saint Petersburg.