Myelofibrosis is the gradual replacement of bone marrow (place where most new blood cells are
produced) by fibrous tissue which reduces the body's ability to produce new blood cells and
results in the development of chronic anemia (low red blood cell count). One of the main
distinctions of myelofibrosis is "extramedullary hematopoesis", the migration or traveling of
the blood-forming cells out of the bones to other parts of the body, such as the liver or
spleen, resulting in an enlarged spleen and liver.
Treatment for myelofibrosis is unsatisfactory and there is no medication that is specifically
used in the treatment of myelofibrosis. There is a protein that is found to be present in the
majority of myelofibrosis patients (JAK2) and the drug Lestaurtinib is being studied to see
if it will stop this protein from functioning and thereby help control the disease.
This study is divided into two Phases (1 & 2). In phase 1 we will be looking for the dose of
study medication (Lestaurtinib) that will be the highest dose a patient can take without
experiencing serious side effects, maximum tolerated dose (MTD).
In phase 2, after the MTD dose has been established in phase 1, we will be investigating how
well CEP-701 (Lestaurtinib) works at suppressing the protein (JAK2).
The investigators also wish to find out important biologic characteristics or features of
myelofibrosis through an additional correlative biomarker study (MPD-RC #107). The
correlative biomarker study is a study that is related to the main study, but is looking to
answer different questions than the main study. The purpose of the biomarker study is to
understand the causes of MPD and to develop improved methods for the diagnosis and treatment
of these diseases, while the main study is trying to find out how well CEP-701 (Lestaurtinib)
will work in treating the myeloproliferative disease.
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
Ronald Hoffman
Collaborators:
Myeloproliferative Disorders-Research Consortium National Cancer Institute (NCI)