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By Alex Morrisson

NEW ORLEANS -- December 1, 2013 -- For the first time, a novel investigative agent appears to induce complete responses among patients diagnosed with myelofibrosis, researchers said here at the 55th American Society of Hematology (ASH) Annual Meeting and Exposition.

·In this pilot study we were pleasantly surprised to find that 5 of our 22 evaluable patients achieved a complete response to treatment with imetelstat -- we have never seen this type of response before in myelofibrosis,· said Ayalew Tefferi, MD, Mayo Clinic, Rochester, Minnesota.

Of the patients that achieved a complete response, 4 exhibited documented reversal of fibrosis in bone marrow and in 2 patients there has been no molecular sign of the disease.

·We also believe that we now have molecular clues as to which patients are going to respond to the treatment with imetelstat,· said Dr. Tefferi in his oral presentation on December 9.

Overall, 13 of the 22 patients responded to therapy with imetelstat. The other 9 patients either didn·t respond or the response was too small to be considered even a partial improvement.

The trial has recruited 33 patients, but just 22 have been treated for at least 6 months.

·These are early results but they are promising,· said Dr. Tefferi said. ·Typically, myelofibrosis is characterised by marrow scarring and although patients may derive symptomatic relief from other treatments, they usually do not revert back to normal bone marrow.·

Funding for this study was provided by Geron Corporation.

[Presentation title: Imetelstat, a Telomerase Inhibitor, Induces Morphologic and Molecular Remissions In Myelofibrosis and Reversal Of Bone Marrow Fibrosis. Abstract 662]
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