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Experimental Drug Panobinostat Shows Little Activity In MDS Patients (ASH 2010)

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Published: Dec 29, 2010 9:17 am
Experimental Drug Panobinostat Shows Little Activity In MDS Patients (ASH 2010)

Preliminary results of a Phase 2 trial show that the experimental drug panobinostat has little activity in patients with relapsed and resistant myelodysplastic syndromes.

However, because the drug was reasonably well tolerated, the study authors suggested the drug be investigated further in combination with other myelodysplastic syndromes (MDS) treatments.

These results were presented at the 2010 meeting of the American Society of Hematology (ASH) earlier this month in Orlando by Dr. Ian W. Flinn of the Sarah Cannon Research Institute in Nashville who led the clinical trial.

Panobinostat (LBH589), which is being developed by the pharmaceutical company Novartis, belongs to a class of drugs called HDAC inhibitors that interfere with the maturation of cancer cells and kill them. The drug is currently being investigated as a treatment for a variety of cancer types including MDS.

In their study, Dr. Flinn and his colleagues investigated panobinostat’s activity in patients who had relapsed or refractory MDS.

The results presented at ASH were based on 10 MDS patients who had stopped responding to Vidaza (azacitidine) or Dacogen (decitabine) treatment. Patients with abnormalities in chromosome 5q had to have failed prior Revlimid (lenalidomide) treatment. None of the patients had received HDAC inhibitors before.

The patients received 30 mg of panobinostat orally three times a week for 21 days followed by one week of rest. If the patients did not experience any severe side effects after two cycles of treatment, the dosage was increased to 40 mg three times a week.

The best response that the researchers observed after two treatment cycles was stable disease in seven patients (70 percent). Two patients (20 percent) had progressed to acute myeloid leukemia. One patient did not complete the study and could therefore not be evaluated.

Non-blood-related side effects were generally mild and most commonly included loss of appetite, fatigue, and nausea.

Additionally, five patients (50 percent) experienced low red blood cell counts, eight patients (80 percent) had low platelet counts, six patients (60 percent) experienced low white blood cell counts, and seven patients (70 percent) had low neutrophil (a type of white blood cell that fights disease) counts.

For additional information, please see abstract 4015 at the ASH meeting website.

Photo by Hoggheff aka Hank Ashby aka Mr. Freshtags on Flickr – some rights reserved.
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