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.

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

Acute Lymphoblastic Leukemia (ALL) and B-cell Lymphoblastic Leukemia (B-ALL) is a heterogeneous hematologic disease characterized by the proliferation of immature lymphoid cells in the bone marrow, peripheral blood, and other organs. The proliferation and accumulation of blast cells in the marrow results in suppression of hematopoiesis and, thereafter, anemia, thrombocytopenia, and neutropenia. 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 5,930 new cases of ALL and about 1,500 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.

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 U.S. alone, it is estimated that there will be 21,450 new AML cases in 2019, with 10,920 deaths. Although it can occur in children and adults, AML is primarily a disease of the elderly, with an incidence of 15 cases per 100,000 for those over 60 and a median age of patients with AML of 67 years. 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 with a poor prognosis. CD123 is highly expressed on acute myeloid leukemia (AML) leukemic stem cells and blast cells, as well as in other hematologic malignancies, and constitutes an attractive target for AML.

Multiple Myeloma (MM)

Multiple Myeloma (MM) is a clonal plasma cell malignant neoplasm characterized by the proliferation of a single clone of plasma cells producing a monoclonal immunoglobulin. This clone of plasma cells proliferates in the bone marrow and often results in extensive skeletal destruction with osteolytic lesions, osteopenia, and/or pathologic fractures. In the United States, the lifetime risk of getting MM is 1 in 132 and it is anticipated that in 2019 32,110 new cases of MM will be diagnosed, with 12,960 deaths.

Nine drugs have been approved over the past fifteen years for the treatment of MM, substantially expanding the number of treatment regimens available for patients in all stages of the disease. In the last decade, survival of multiple myeloma (MM) patients has markedly improved with a median survival of approximately 5 to 7 years but with major variation depending on a host 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.

Hodgkin Lymphoma (HL)

Hodgkin Lymphoma (HL) is a cancer of the lymphatic system that affects people of many ages, most commonly in early adulthood (around the age of 20) and those in their later adult years (after age 55). As lymph tissue lives in many parts of the human body, HL can start almost anywhere. HL can be broken down into Class Hodgkin Lymphoma, which has 4 subtypes based on whether the patient is lymphocyte-rich, lymphocyte-depleted, presenting with nodular sclerosis or mixed cellularity. There is also another type called Nodular lymphocyte-predominant Hodgkin lymphoma which accounts for about 5% of cases.

In 2019, it is expected that 8,110 new cases will be diagnosed and approximately 1,000 deaths will occur due to HL. While there are currently no screening tests for HL, it is most often found by recognizing its common symptom of enlargement or swelling of one of more lymph nodes. Survival rates have continued to improve over years, especially due to advancements in treatment and technology. The 5-year relative survival rate of people diagnosed with HL is around 86%, and can vary based on age of diagnosis.

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.