Acute myeloid leukemia (AML) is a rare disease which mainly affects the elderly. It is a cancer of the blood and bone marrow marked by rapid disease progression if untreated.
Acute myeloid leukemia is the most common acute leukemia in adults and affects 5/100,000 inhabitants in Europe, i.e., more than 20,000 new cases each year.1 The two-year survival rate of 75 years old patients with AML is below 10%.2
In the United States, the disease represents 1.2% of new cancer diagnoses each year, around one-third of all diagnosed leukemia cases, and is the cause of 10,000 deaths every year.
This cancer is a proliferation of immature cells, which means the cells are not sufficiently developed and so cannot function normally. These cancerous cells are present in the blood, this is why the term “leukemia” is used, and they arise from myeloid blast hence the term “myeloid”. The bone marrow is the tissue present in the bones where the blood cells, known as “blasts”, are produced.
How does AML develop?
The disease occurs when there are changes in the DNA of a stem cell of the bone marrow during the development stage. The affected stem cell transforms into a leukemic cell and multiplies into millions of “leukemic blasts”, known as myeloblasts.
Myeloblasts block the production of normal blood cells, both red and white blood cells, as well as platelets, blood cells produced by the bone marrow that play an important role in coagulation.
A decrease in blood cells can be revealed thanks to biological tests. Neutropenia, i.e. a low level of white blood cells, is one of the main effects observed and results in a weakening of the immune system, which is programmed to protect the body from infections. Anemia or a reduction in the number of red blood cells in the blood can also be observed, which often results in asthenia (significant fatigue), as well as thrombocytopenia (an abnormal decrease in the number of platelets in the blood).
AML risk factors
For most patients with AML, there are no obvious causes of the disease and the main one is rarely identified. Instead, there may be risk factors such as repeated exposure to benzene (cigarette smoke, petroleum products, work environment using benzene), certain genetic diseases such as Fanconi anemia or Down syndrome, certain chemotherapy or radiotherapy, or the development of certain cancers and blood diseases. These different risk factors sometimes alter the DNA of blood cells located in the bone marrow.
Therapeutic management of AML
The main treatment for AML is based on chemotherapy in order to eliminate the cancerous cells.
In general, there are two treatment phases. An induction phase and a consolidation phase. The induction phase generally lasts one month and helps to kill most cancerous cells. Consolidation therapy, meanwhile, prevents relapse, i.e. the recurrence of cancerous cells. It is possible to combine chemotherapy with targeted therapies in order to prevent the growth of cancerous cells. In certain cases, a stem cell transplant may be considered. In both treatment phases of AML, supportive therapy is offered to deal with the side effects of treatments and those induced by the disease.
To address the ever-increasing incidence of cancer and the growing need for therapeutic solutions, we have made oncology a priority focus for development. We allocate over half of our R&D budget in oncology, with the ambition to become an innovative player focused on developing medicines targeting hard-to-treat cancers with high unmet medical needs. We wish to initiate therapeutic progress where generally speaking needs are not yet met, known as hard-to-treat cancers, such as digestive cancers (stomach cancer, pancreatic cancer, and cholangiocarcinoma, a rare and aggressive form of bile duct cancer), glioma, or brain tumors, hematologic cancers (acute myeloid leukemia, acute lymphoblastic leukemia) and pediatric cancers.
Servier leads its R&D programs around two approaches:
KEEP IN MIND
 ESMO Guidelines 2020 – Acute myeloid leukemia in adult patients: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up
 National Cancer Institute. Surveillance, Epidemiology, and End Results (SEER) Program. Cancer Stat Facts: Acute Myeloid Leukemia (AML). https://seer.cancer.gov/statfacts/html/amyl.html