Research and Development of the Effective Components of Panaxdiol Saponin as New Chinese Patent Medicine for Treating Hemocytopenia

Pancytopenia (hemocytopenia) such as primary immune thrombocytopenia (ITP), aplastic anemia and chronic neutropenia (agnogenic leukocytopenia) were often treated by glucocorticoids, androgen and immunosuppressive agents at present, but the response to these treatments has not been always satisfactory, and may cause serious adverse events. Our research has identified a biological active component in ginseng extract and the active component, panaxadiol saponins component (PDS-C), was isolated from total saponins of ginsenosides, and formulated into capsules named as Painengda. We successfully obtained approval from State Food and Drug Administration (SFDA) of China in 2010 to conduct clinical trials of PDS-C as class-five new Chinese patent medicine. Phase I and phase II clinical trials of PDS-C and Painengda Capsule were carried out in the treatment of ITP and agnogenic leukocytopenia. The composition and content of PDS-C have been analyzed and defined by high-performance liquid chromatography-mass spectrometry (HPLC-MS) and HPLC using specific monomers of ginsenosides as the reference standards. PDS-C is very efficacious for treating mice and rats with ITP and aplastic anemia, and myelosuppression caused by chemotherapy or radiation. Our animal model studies and cell biology and molecular biology experiments demonstrated that PDS-C possessed dual activities, namely that of promoting proliferation and differentiation of hematopoietic progenitor cells, and that of regulating the immune function. PDS-C and Painengda Capsule as a new Chinese patent medicine have been successfully transferred to industry. We believe that PDS-C is effective and safe in the treatment of refractory hemocytopenia. The advantages are that it is effective in small doses, it is convenient to use because of its oral administration, its lack of adverse events, it could be used alone or in combination with pharmacological agents, which improve the efficacy and decrease adverse events.