FDA Approves an Experimental Treatment of Various Blood Malignancies for Clinical Trials

According to a story from First World Pharma, the biopharmaceutical company Mustang Bio, Inc. recently announced that the US Food and Drug Administration (FDA) has approved their investigational new drug (IND) application. This application pertains to the company’s experimental drug candidate MB-102. With this approval, MB-102 is now eligible for testing in phase 1/2 trials for three different blood malignancies, including high risk myelodyplastic syndromes, acute myeloid leukemia, and blastic plasmacytoid dendritic cell neoplasm (BPDCN).

About Acute Myeloid Leukemia (AML)

Acute myeloid leukemia, also known as acute myelogenous leukemia, is a type of blood cancer which affects myeloblasts, stem cells that would normally develop into myeloid white blood cells. Symptoms include an increased risk of infection, easy bruising and bleeding, fatigue, shortness of breath, fever, weight and appetite loss, anemia, and bone/joint pain. The five year survival rate for acute myeloid leukemia is only 27 percent in the US. To learn more about acute myeloid leukemia, click here.

About Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)

Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare blood disorder that affects plasmacytoid dendritic cells. Symptoms of the disease include ulcers, bruise-like patches, or skin lesions, swollen lymph nodes, liver, and/or spleen, anemia, and the presence of malignant pDCs throughout the body. The five year survival rate for BPDCN is low. There is an urgent need for more effective therapies for this disease. To learn more about BPDCN, click here.

About Myelodysplastic Syndromes

Myelodysplastic syndromes are a type of blood cancer in which developing blood cells remain immature and fail to transform into usable blood cells. Myelodysplastic syndromes rarely present with symptoms initially, but it can eventually present with anemia, neutropenia, thrombocytopenia, cell abnormalities, chromosome abnormalities, enlarged spleen and/or liver, easy bleeding and bruising, and infections. To learn more about myelodysplastic syndromes, click here.

About MB-102

MB-102 has earned Orphan Drug designation from the FDA as a therapy for acute myeloid leukemia. This drug is a form of CAR-T cell therapy, in which immune system T cells are extracted from the patient, modified to target the malignant cells, propagated in the lab setting, and then reintroduced into the body of the patient. With the clearing of the IND, clinical testing of this drug is expected to begin in the near future.


Share this post

Share on facebook
Share on google
Share on twitter
Share on linkedin
Share on pinterest
Share on print
Share on email
Close Menu