Therapeutic Indications

Blood and Bone Marrow Cancers

Blood cancers affect the production and function of blood cells. Most of these cancers start in the bone marrow where blood is produced. Stem cells in the bone marrow mature and develop into three types of blood cells: red blood cells, white blood cells, or platelets. In most blood cancers, the normal blood cell development process is interrupted by uncontrolled growth of an abnormal type of blood cells. These abnormal blood cells, or cancerous cells, prevent blood from performing many of its functions, like fighting off infections or preventing serious bleeding.


The three main types of blood cancers are leukemia, lymphoma and myeloma. Leukemia is a type of cancer of the bone marrow, caused by the proliferation of abnormal cells in the bone marrow. Lymphoma is a type of blood cancer that affects the lymphoid organs where the immune cells are located. Myeloma is a cancer of the bone marrow caused by the proliferation of abnormal plasma cells, which are the cells producing the antibodies.

Multiple Myeloma (MM)

Multiple Myeloma (MM) is a cancer that affects a type of white blood cells called plasma cells that are specialized mature B cells, which secrete antibodies to combat infections. Multiple myeloma is characterized by the uncontrolled proliferation of neoplastic plasma cells in the bone marrow, where they overcrowd healthy blood cells. Although MM is a chronic disease and an exact cause has not yet been identified, researchers have made significant progress over the years in managing the disease through better understanding MM's pathophysiology.

In the last decade, the median survival rate of multiple myeloma patients has markedly improved, but each patient is unique depending on various factors: the stage of the disease, cytogenetic abnormalities, and response to therapy. However, despite this progress, many patients need additional options to treat their disease in order to manage it effectively. The progress in finding a cure needs to be continued as The American Cancer Society estimates that 32,270 new cases of MM will be diagnosed, and 12,830 deaths are expected to occur in 2020 in the U.S. alone.

Acute Myeloid Leukemia (AML)

Acute Myeloid Leukemia (AML) is characterized by infiltration of the bone marrow, blood, and other tissues by proliferative, clonal, abnormally and/or poorly differentiated cells of the hematopoietic system called blast cells. These cells interfere with normal hematopoiesis, thus contributing to the bone marrow failure which is the most common underlying cause of death.

In the US alone, it is estimated that there will be 19,940 new AML cases in 2020, with 11,180 deaths. Although it can occur in children and adults, AML is primarily a disease of older adults (median age of diagnosis is about 68 years old) and is uncommon before the age of 45. While complete response rates can be as high as 80% in patients undergoing initial induction cytotoxic chemotherapy, the majority of AML patients will ultimately be diagnosed with relapsed or refractory disease and require additional treatment options.

Acute Lymphoblastic Leukemia (ALL) & B-cell Acute Lymphoblastic Leukemia (B-ALL)

Acute Lymphoblastic Leukemia (ALL) and B-cell Lymphoblastic Leukemia (B-ALL) are a heterogeneous hematologic disease characterized by the proliferation of immature lymphoid cells in the bone marrow, peripheral blood, and other organs. The increase and accumulation of blast cells in the bone marrow results in suppression of the normal production of blood cell and blood plasma components, and can therefore cause anemia, thrombocytopenia, neutropenia and risk of infection. ALL can start either with early B-cells or T-cells at different stages of maturity. The American Cancer Society’s estimates for ALL in the United States for 2019 (including both children and adults) are about 6,150 new cases of ALL and about 1,520 deaths. Approximately 85% of ALL cases involve precursor B-cells (B-ALL).

The risk for developing ALL is highest in children younger than 5 years of age, but declines over time until the mid-20s, and increases again slowly after age 50. Overall, about 4 of every 10 cases of ALL are in adults. The cure rates and survival outcomes for patients with ALL have improved dramatically over the past several decades, primarily among children. Improvements are largely owed to advances in the understanding of the molecular genetics and pathogenesis of the disease, the incorporation of risk-adapted therapy, and the advent of new, targeted agents. Despite great progress in the development of curative therapies, ALL remains a leading cause of pediatric cancer-related mortality for patients presenting with a relapsed or refractory disease. New therapies are needed to overcome chemotherapy resistance and reduce non-specific treatment associated side effects.

Non-Hodgkin Lymphoma (NHL)

Non-Hodgkin lymphoma (NHL) is a heterogeneous disease resulting from the malignant transformation of lymphocytes with distinctive morphologic, immunophenotypic, genetic, and clinical features. Non-Hodgkin lymphoma is more common than the other general type of lymphoma — Hodgkin lymphoma. The past several decades have seen a steady increase in incidence rates of NHL, with overall rates in the United States nearly doubling over the period 1975 to 2008. In 2019, there is an estimated 74,200 new cases that will be diagnosed with NHL, with approximately 19,970 deaths.

Many different subtypes of non-Hodgkin's lymphoma exist. The most common non-Hodgkin lymphoma subtypes include diffuse large B-cell lymphoma and follicular lymphoma.