Our therapeutic areas
Acute Myeloid Leukemia (AML)
- AML is a rare and aggressive cancer of the blood and bone marrow. It prevents white blood cells from maturing, causing an accumulation of blasts which do not allow room for the normal blood cells. In AML, these immature forms of myeloid cells become abnormal blood cells1, or leukemic cells which rapidly divide and enter the blood stream, sometimes spreading to other organs2.
- In the European Union, 5 to 8 cases will be diagnosed among 100,000 people every year. AML is more common in older people, with an almost 10-fold increase in the number of cases among elderly patients9.
Myelofibrosis (MF)
- MF is 1 of the 3 main types of myeloproliferative neoplasms (MPNs)3. Bone marrow is gradually replaced by scar tissue causing the body to produce fewer normal blood cells4-6. The cause of MF is not known but most patients share certain mutations that may explain why MF develops7.
- Approximately 1 in 100.000 people has MF8.
Polycythemia (PV)
- PV is the second most common type of myeloproliferative neoplasm (MPN)10. In PV the body system that controls blood cell production does not function properly. This causes the body to produce too many blood cells in the soft tissue, inside the bones or in the bone marrow1.
- Globally, PV affects up to 3 in every 100.000 people each year11.
Immune Trombocytopenia (ITP)
- ITP is a rare autoimmune disease characterised by increased destruction and impaired productions of blood cells called platelets. Platelets allow the blood to clot properly and keep blood vessels intact12. When platelet levels are very low, this can cause dangerous internal bleeding and bruising13.
- ITP affects approximately 1 in 50.000 adults each year and is slightly more common among adult women12,14.
References
1. National Cancer Institute: Adult Acute Myeloid Leukemia Treatment (PDQ®)–Patient Version. Available at: https://www.cancer.gov/types/leukemia/patient/adult-aml-treatment-pdq. Accessed January 2019
2. American Cancer Society: What is acute myeloid leukemia? Available at: http://www.cancer.org/cancer/leukemia-acutemyeloidaml/d tailedguide/leukemia-acute-myeloid-myelogenous-what-is-aml. Accessed January 2019
3. Zimmerman M et al. Myeloproliferative Disorders and Myelofibrosis. Am J Manag Care. 2012 May; (3 Spec No.): SP131-3.
4. Leukemia & Lymphoma Society. “Myelofibrosis Facts.” Accessed August 2017.
5. Dictionary of Cancer Terms. National Cancer Institute. US National Institutes of Health. Available at: https://www.cancer.gov/publications/dictionaries/cancer-terms. Accessed August 2017.
6. National Center for Biotechnology Information, US National Library of Medicine, National Institutes of Health. Myelofibrosis. Available at: http://www.ncbi.nlm.nih.gov/pubmedhealth/PMH0001558/. Accessed February 2016
7. Cazzola M, Kralovics R. From Janus Kinase 2 to Calreticulin: The Clinically Relevant Genomic Landscape of Myeloproliferative Neoplasms. Blood.2014 Jun 12;123(24):3714-9.
8. Cervantes F. How I treat splenomegaly in myelobrosis. Blood Cancer J. Oct 2011; 1(10): e37
9. Leukemia & Lymphoma Society®: The AML ESMO ACF Patient Guide Series based on the ESMO Clinical Practice Guidelines. Acute Myeloblastic Leukaemia. Available at: https://www.esmo.org/content/download/6583/114891/file/EN AML Guidefor-Patients.pdf Accessed January 2019
10. Leukemia & Lymphoma Society. Polycythemia Vera Facts. Available at: http://www.lls.org/content/nationalcontent/resourcecenter/freeeducationm.... Accessed August 2017.
11. Titmarsh G, Duncombe A, McMullin M, et al. How Common are Myeloproliferative Neoplasms? A Systematic Review and Meta-analysis. Am J of Hematol. 2014:1-7.
12. “Immune Thrombocytopenia.” U.S. National Institutes of Health website. U.S. National Institutes of Health. Web. 2 August 2016.
13. Saleh, Mansoor N., et al. "Safety and efficacy of eltrombopag for treatment of chronic immune thrombocytopenia: results of the long-term, open-label EXTEND study." Blood 121.3 (2013): 537-545.
14. Fogarty, Patrick F., and Jodi B. Segal. "The epidemiology of immune thrombocytopenic purpura." Current opinion in hematology 14.5 (2007): 515-519.
- Blood cancers and serious blood disorders affect many people across Europe. In 2016 165.000 people died from blood cancers, equating to almost 9% of all cancer deaths in Europe1.
- Novartis is committed to transforming the lives of people living with blood cancers and serious blood disorders. Believing that anyone living with these conditions has the right to a life free from pain, free from symptoms and free from disease.
- More than +20 years Novartis and its research partners led the era of Targeted Therapies in cancer, transforming Chronic Myeloid Leukemia (CML) into a chronic, rather than life-limiting, condition16. Continued research opened the possibility of treatment free remission (TFR) for CML patients2and investigation is ongoing to potentially further expand care in CML3.
- Today we are working to transform treatment for many pediatric and young adult ALL and DLBCL patients with first-in-class, single infusion CAR-T cell therapy that uses the patient’s own, genetically modified, T cells to fight cancer4,5. Our focus is to broaden the impact of cell therapy in oncology by going deeper in B-cell malignancies and reaching more patients in other hematological cancers.
References
1. Institute for Health Metrics Evaluation GBD results 2016
2. Tasigna CDS, Novartis, 15 July 2016. AMN107_CDS v1.7 (clean) 15Jul2016
3. DOF_Mauro M Asciminib Abstract_2018
4. Novartis receives first ever FDA approval for a CAR-T cell therapy, Kymriah™ (tisagenlecleucel, CTL019), for children and young adults with B-cell ALL that is refractory or has relapsed at least twice, Novartis, 30 August 2017. Kymriah FDA approval press release_Aug 2017
5. Kymriah Prescribing Information. May 2018