PBI-1402 is an orally active low molecular weight synthetic compound which is currently in a phase Ib/II clinical trial in patients with anemia associated with cancer and/or chemotherapy. PBI-1402 stimulates the in vitro/ex vivo proliferation and maturation of hematopoietic progenitors (erythroid and myeloid populations) with an activity comparable to EPO. Furthermore, an additive effect is observed in combination with EPO. PBI-1402 enhances the differentiation of pluripotent stem cells: CFU-GEMM, CFU-GM with a predominant effect on BFU-E. Clinical phase I study showed that PBI-1402 is devoid of significant side effects. In addition, the clinical phase I results showed statistical increase (100%, p<0.0001) of relative and absolute reticulocyte count in healthy volunteers after 21 days of oral treatment compared to placebo. Preclinical results demonstrated that PBI-1402 yields an increase in erythrocytes in the systemic circulation. In myeloblated mice that received a syngeneic bone marrow transplant, oral treatment with PBI-1402 resulted in a significant increase in peripheral erythrocyte count, hemoglobin, platelet and bone marrow erythroid progenitor cells. PBI-1402 is targeted as an adjunct to cytotoxic drugs, radiotherapy and bone marrow transplantation for the treatment of anemia.

Topics:
anemia, partial breast irradiation, penile brachial index, bone marrow transplantation, adverse effects, cancer, chemotherapy regimen, cytotoxic drug therapy, hemoglobin, radiation therapy

Disclosures: All authors except those noted below, are salaried employees of the therapeutic business unit of ProMetic Life Sciences Inc. (PLI). PLI is a publicly traded company listed on the Toronto Stock Exchance. D.C. Roy, M.J. Morin and G. Krosl are under contract with PLI since 2003.

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2006, The American Society of Hematology
2006
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