Phase I JAK2 inhibitor for myelofibrosis

The following information is provided by the University of California, San Francisco (UCSF), an ANCO Institutional Member.

We have recently been activated for an amended phase I protocol that is
investigating lower doses than initially tested when the original study
opened in 2007 (see below). The reason is that while activity was observed,
neuropathy was noted in some patients. This study is thus looking at lower
doses and alternate treatment schedules, and there will be careful
neurologic follow-up. UCSF is the first of the four sites to be open, and
we can accrue up to 12 patients, so we would like to accommodate as many
interested patients as we can.

A Phase I Dose-Escalation Study of the Safety, Pharmacokinetics and
Pharmacodynamics of XL019 Administered to Subjects with Myeloproliferative
Disorders

Dr. Neil Shah at UCSF is running a study involving XL019, a potent,
ATP-competitive and reversible inhibitor of JAK2. This is a first in human
study aimed at patients with myeloproliferative disorders. Many of these
patients have a dominant gain-of-function mutation in the JAK2 gene that the
drug targets, although JAK2-positive status is not required for eligibility.
Eligible patients will have one of the following diagnoses: primary
myelofibrosis (MF), post-polycythemia vera MF, or post-essential
thrombocythemia MF, who require therapy. Patients can be newly diagnosed
intermediate of high risk, or have relapsed or refractory disease. There
will be two dosing cohorts: once per day, or three times per week. Twelve
enrollment slots are currently available. The drug is given as an oral
formulation. If you have a potentially eligible patient, please contact Dr.
Shah’s research coordinator, Patty Olszynski, at 415-502-1564.