Acute myeloid leukemia (AML) is a cancer of the blood and bone marrow. AML is the most common type of acute leukemia in adults. Each year in the United States, about 19,900 people (usually older than 45 years of age) are diagnosed, and about 10,400 people die from all forms of the disease, according to the American Cancer Society. Unlike other cancers that start in an organ and spread to the bone marrow, AML is known for rapid growth of abnormal white blood cells that gather in the bone marrow, getting in the way of normal blood cell production. The lack of normal blood cells can cause some AML symptoms, including anemia, neutropenia (shortage of white blood cells that may lead to increased infections), and thrombocytopenia (shortage of platelets in the blood that may lead to excessive bleeding). Current treatment options for AML include chemotherapy, molecularly-directed small molecule inhibitors, and hematopoietic stem cell transplantation.
Our work in the relapsed/refractory setting and study at the NCI in the older, but fit for chemo patient population has garnered the interest and enthusiasm of clinicians. Working in collaboration with clinical investigators, consortia, partners and regulatory agencies across the globe, we have one goal in mind—to establish uproleselan as a foundational therapy across the entire spectrum of AML. Where most other AML approved therapies and development these days are focusing on narrow patient populations as defined by certain genetic markers, uproleselan targets the bone marrow microenvironment by inhibiting those pro-survival pathways that render cancer cells protected from the effects of chemotherapy. This is a novel approach that is not competitive to, but rather potentially broadly applicable across the spectrum and range of therapies used for relapsed/refractory and newly diagnosed patients fit for chemotherapy. In clinical studies to date, uproleselan has shown a very good safety profile; and in our Phase 2 trial, severe mucositis was reduced relative to expected levels compared to historical controls.