Myelofibrosis (MF) is a rare blood cancer that develops when a genetic mutation occurs in blood stem cells, resulting in excessive scar tissue formation (fibrosis) in the bone marrow, where most blood cells are produced. To help compensate for abnormal blood counts, splenomegaly (enlargement of the spleen) occurs in most patients, which can cause pain or fullness on the left side below the ribs.[i]
A patient’s life expectancy following an MF diagnosis depends on their risk level, which doctors determine using prognostic scoring systems, and patients with high-risk disease often have a life expectancy of two years or less.[ii]
MF is not curative in most cases and there are very few treatment options available especially among an older, high-risk patient population. Recognizing the need for new treatment options in MF, AbbVie researchers are studying a compound through clinical trials, called navitoclax, an investigational therapy for MF. Navitoclax is not approved by the U.S. Food and Drug Administration (FDA). Its safety and efficacy are under evaluation as part of ongoing studies.
Navitoclax is an investigational oral BCL-XL
/BCL-2 inhibitor that may induce apoptosis, a form of regulated cell death that eliminates damaged or unneeded cells from the body. Patients with MF often have high expression of BCL-XL
and BCL-2, where the BCL-2 family proteins prevent apoptosis from occurring.[iii]
By inducing apoptosis, this may cause an anti-fibrosis, or reversal of fibrosis, effect on the bone marrow.[iv]
Current clinical trials are evaluating navitoclax’s impact on the most common features of MF including splenomegaly, bone marrow fibrosis and anemia responses, as well as quality of life. Patients, their treating physicians and caregivers can learn more about AbbVie’s MF clinical trials at myelofibrosisresearch.com
“We are investigating if navitoclax may be an option that offers myelofibrosis patients a new level of disease management including improved symptom control and potentially better treatment outcomes,” said Mohamed Zaki, M.D., Ph.D., vice president and global head of oncology clinical development, AbbVie. “The company’s anti-fibrosis efforts build on AbbVie’s history of transforming standards of care in blood cancers with significant unmet needs.”
Our goal at AbbVie is to deliver a meaningful impact on patients’ lives and one way to do so is through clinical trial research. Clinical trials are an important component of driving new treatments, especially in areas of high unmet needs. For more information on how clinical trials work and to find one that is best suited for you or a loved one, please visit ClinicalTrialsandMe.com
Tsujimoto Y. (1998). Role of Bcl-2 family proteins in apoptosis: apoptosomes or mitochondria?. Genes to cells : devoted to molecular & cellular mechanisms, 3(11), 697–707. https://doi.org/10.1046/j.1365-2443.1998.00223.x
Lagares D, Santos A, Grasberger PE, Liu F, Probst CK, Rahimi RA, Sakai N, Kuehl T, Ryan J, Bhola P, Montero J, Kapoor M, Baron M, Varelas X, Tschumperlin DJ, Letai A, Tager AM. Targeted apoptosis of myofibroblasts with the BH3 mimetic ABT-263 reverses established fibrosis. Sci Transl Med. 2017 Dec 13;9(420):eaal3765. doi: 10.1126/scitranslmed.aal3765. PMID: 29237758.