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JAK inhibitors producing significant response in myelofibrosis patients
Date:12/6/2010

nimesh Pardanani, Ph.D. (http://mayoresearch.mayo.edu/staff/pardanani_a.cfm), the lead investigator on both studies. One of the two studies being presented at ASH is longer-term follow-up on the use of TG101348 in 59 patients with myelofibrosis who are being treated at Mayo Clinic, Stanford University Medical Center, MD Anderson Cancer Center, Dana-Farber Cancer Institute, University of Michigan and University of California, San Diego.

The study concludes that after 12 cycles of treatment, 95 percent of patients had some reduction in the size of their spleen. Within that group, 26 percent had a 100 percent reduction. The study also found that of the 48 patients who had a JAK2 mutation, 23 (48 percent) had a reduction in mutation burden. Patients who completed 12 cycles of therapy had the most reduction. Thirteen patients (72 percent) had a median 50 percent decrease. That response has lasted more than a year, says Dr. Pardanani.

The researchers also found that 56 percent of patients treated with 12 cycles of TG101348 achieved normal white blood cell counts.

"TG101348 represents the first proof of principle that a selective JAK2 inhibitor can shut down a signaling pathway and reduce the burden of neoplastic blood cells," Dr. Pardanani says. "It also offers significant clinical benefit in that it decreases spleen size, controls excess blood cell counts in more than half of patients, and improves symptoms such as fatigue and night sweats."

In the second study being presented at ASH, 32 of 36 enrolled patients had completed at least one cycle of CYT387 therapy in a phase I/II clinical trial. Within this group of patients, 41 percent achieved an improvement in anemia, as defined by the International Working Group criteria. And 97 percent had some degree of spleen-size reduction (at least a 50 percent reduction in 37 percent of those patients).

"CYT387 not only works to reduce spleen size and to help with other symptoms, but i
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Contact: Nicole Engler
newsbureau@mayo.edu
507-284-5005
Mayo Clinic
Source:Eurekalert

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