New Drug for Myelodysplastic Syndromes (MDS) (BLI-1301)
Target Indication
BLI-1301 is extracted from a fungus, Grifola Frondosa, which enhances immunity. It is developed for enhancing the immune system of Myelodysplastic Syndrome (MDS) patients.
Drug Type
New drug category: Botanical new drug
Formulation: Oral
Foresight and Global Development
BLI-1301 is a natural anti-cancer ingredient extracted from a special fungus with patented process developd by Yukiguni Maitake Co. Ltd., Japan. American and Japanese scientists suggested that oral administration of BLI-1301 in animals showed effects on hematopoietic stem cells repair, immune system enhancement, and cancer treatment.
The indication of BLI-1301 is a rare hematological cancer - Myelodysplastic Syndromes (MDS). MDS patients suffer from heterogeneous malignant bone marrow disorders characterized by ineffective hematopoiesis, resulting in peripheral blood cytopenias. It was estimated that >10,000 new cases of MDS occur annually, and that there are 60,000 patients in the United States.
Retrospective analysis of 1,394,343 individuals in 2003 Medicare Standard Analytic Files have shown that, compared to overall Medicare population, the prevalence of infection is significantly increased in patients with MDS within 3-year follow-up. The risk of infection further increases in higher-risk MDS or transfusion-dependent MDS patients. Although 30% of MDS patients progress to acute myelogenous leukemia (AML), a retrospective study analysis of 273 deceased patients with lower-risk MDS demonstrated that the primary disease-related cause of death was infection (38% of all death), rather than transformation to AML (15%). Since the application and therapeutic effect of current prophylactic treatments are still limited, there is urgent unmet need of new drug in protecting MDS patients from infection.
Result
1.Enhancement of bone marrow colony formation and recovery of myeloid function in acute anemia mice model
Summary: In a paclitaxel-induced hematologic toxicity acute anemia mice model, BLI-1301 not only recovered neutrophil count by increasing colony forming units for granulocytes and macrophages, but also increased neutrophil and monocyte activity in peripheral blood as measured by ROS respiratory burst test.
2.No dose-limiting toxicity encountered in Phase I/II trial on breast cancer patients
Summary: In a phase I/II dose escalation trial on 34 postmenopausal breast cancer patients, BLI-1301 was taken orally at 0.1, 0.5, 1.5, 3, or 5 mg/kg twice daily for 3 weeks. No dose-limiting toxicity was encountered up to 10 mg/kg per day, and the optimal dose associated with the most prominent functional changes was 5-7 mg/kg per day. No serious adverse event was observed during the study period in any study subject.
3.Enhancement of ROS respiratory burst activity in Phase II trial on MDS patients
Summary: Following the phase I/II dose escalation trial, a phase II trial on MDS patients was conducted, where 21 MDS patients received BLI-1301 orally at 3 mg/kg twice daily for 12 weeks. The result from 18 evaluable MDS patients showed that BLI-1301 significantly enhanced the ROS respiratory burst activity of unstimulated neutrophils and monocytes, as well as that of E. coli or fMLP-stimulated monocytes isolated from peripheral blood of the participants.
Table 1. Mean (SD) baseline neutrophil and monocyte count and function in evaluable MDS patients and healthy controls, and mean change between baseline and 12 weeks in evaluable MDS patients, n=18.
Product Advantage
Several human and animal studies have shown that BLI-1301 enhances immune system and repairs hematopoietic stem cells.BLI-1301 has been tested by professional teams from Japan and US Cancer Centers in Phase I and II Clinical Trials.
BLI-1301 is a natural substance from a kind of fungus with long history of use. Tracing from its history of use as well as clinical studies, BLI-1301 has higher safety and fewer side effects compared to NCEs.
Oral administration enhances patient compliance.
BLI-1301 is covered by patents in 7 countries as the US, United Kingdom, Japan, Germany, France, Italy, and China.
