Haematologists 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, haematologists 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. They have reported
this in the current 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 able to be treated due 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 due to 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.
Moscow-based company Fusion Pharma has developed an innovative
kinase inhibitor, PF-114 with the aim of having the same effect on Ph+ leukemia
as Ponatinib, but with reduced side-effects. In the current edition of
'Leukemia', the team led by Dr. Afsar Mian, Professor. Oliver Ottoman and
lecturer Dr. Martin Ruthardt from the Haematology Department of Medical Clinic
II, have reported that PF-114 is as effective as Ponatinib against resistant Ph+
leukemia.
"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," explained Dr. Ruthardt. "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," explains Dr. Ghermes Chilov, CEO of Fusion
Pharma, the company that financed the project.
Posted By:-
Bioinformatics Department
Posted By:-
Bioinformatics Department
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