Myelofibrosis (MF) is a rare type of myeloproliferative neoplasm (MPN) with notable areas of medical need. Protheragen has a wonderful team of researchers and scientists who have considerable experience in MF. They are dedicated into helping develop novel therapies for MF which care for the unguarded medicinal needs of the disease and try to progress the field of MF therapeutics.
Myelofibrosis (MF) is a rare and serious bone marrow disorder characterized by abnormal blood cell production that causes gradual scarring (fibrosis) of the bone marrow. This condition disrupts the normal rhythm of blood cell production, often leading to manifestations like tiredness, weakness, the presence of bruises or minor bleeds, along with splenomegaly. MF may manifest independently, which is referred to as primary MF, or may develop from other blood disorders like polycythemia vera or essential thrombocythemia, in which case it is termed secondary MF.
Fig. 1 Targets for novel therapies in myelofibrosis (MF). (Tremblay D, Mascarenhas J., 2021)
Myelofibrosis (MF) is the result of several genetic mutations, including JAK2, CALR and MPL, within the hematopoietic stem cells which leads to pathway deregulation, in particular to the JAK-STAT cascade with resultant production of pathologic blood cells in excess. This causes chronic inflammation and in addition the secretion of cytokines, notably TGFβ which stimulates fibrosis of the bone marrow by activating fibroblasts and excessive collagen deposition. At the same time, splenomegaly is often observed due to extramedullary hematopoiesis. Further, the altered microenvironment of the marrow greatly reduces the normal cell production accelerating the progression of the disease along with the accompanying symptoms.
Fig. 2 Pathological mechanisms of myelofibrosis (MF). (Gangat N, Tefferi A., 2020)
Despite being a rare blood cancer, myelofibrosis (MF) is a blockbuster market with sales expected to grow from $2.39 billion in 2021 to $2.89 billion in 2031, at a compound annual growth rate (CAGR) of 1.9%. However, only 10-15% of affected individuals achieve long-term disease control, and many experience relapse, anemia, thrombocytopenia, or progression to acute myeloid leukemia (AML). The development of new therapies is urgent.
Table. 1 Drug development pipeline for myelofibrosis (MF).
| Drug Names | Mechanism of Action | Targets | NCT Number | Research Phase |
|---|---|---|---|---|
| Ruxolitinib | Reduces inflammatory cytokines and splenomegaly. | JAK1/JAK2 | NCT00934544 | Approved |
| Pomalidomide | Inhibits angiogenesis and modulates TNF-α/IL-6. | TNF-α/IL-6 | NCT00463385 | Phase II |
| Zoledronic Acid | Inhibits osteoclast activity. | Bone resorption factor | NCT00287261 | Phase II |
| Obatoclax Mesylate | Induces apoptosis in malignant cells. | BCL-2, MCL-1, BCL-XL | NCT00360035 | Phase II |
| Tasquinimod | Immunomodulator and anti-angiogenic. | HDAC4, HIF-1α | NCT06605586 | Phase I/II |
Disclaimer: Protheragen focuses on providing preclinical research services. This table is for information exchange purposes only. This table is not a treatment plan recommendation. For guidance on treatment options, please visit a regular hospital.
At Protheragen, our unwavering commitment lies in delivering state-of-the-art diagnostic and therapeutic development services tailored specifically for myelofibrosis (MF). Our expertise is centered around the development of innovative therapies spanning multiple molecular classes, meticulously studied in intricately crafted disease models during the preclinical phase.
Animal models provide researchers with valuable tools to study the underlying mechanisms of myelofibrosis (MF) and test potential therapies. These models bridge the research gap from bench to bedside, accelerating therapeutic breakthroughs for MF.
| Types | Optional Models | |
|---|---|---|
| Genetically Engineered Models |
|
|
| Induced Model |
|
|
Specializing in preclinical research for drug development, Protheragen offers a comprehensive solution that includes pharmacodynamics (PD), pharmacokinetic (PK) and toxicology studies to thoroughly validate and optimize therapies for myelofibrosis (MF). If you are interested in our services, please feel free to contact usfor more details and quotation information of related services.
References
Myeloproliferative Disorders (MPDs)
Bone Marrow Failure Syndromes (BMFS)