Myelofibrosis (MF)
Myelofibrosis (MF) is an uncommon form of bone marrow cancer that disrupts the regular production of blood cells in the body. Our company is committed to developing cutting-edge diagnostics and therapeutics for the management of MF. As your reliable partner in MF research, we offer streamlined and comprehensive solutions to meet all your scientific research requirements.
Overview of MF
MF is a rare and intricate form of blood cancer. It triggers the development of extensive fibrous tissue in the bone marrow, resulting in severe anemia characterized by weakness and fatigue. The scarring of the bone marrow can also lead to a reduction in the number of platelets, thereby raising the risk of bleeding.
The pathogenesis of MF primarily involves mutations in the JAK2, CALR, or MPL genes within the hematopoietic stem/progenitor cells residing in the bone marrow. The incidence of MF is extremely low, with a global annual incidence of approximately 0.47 cases per 100,000 individuals.
Fig. 1 Comparison of healthy bone marrow and bone marrow from MF individuals. (Veletic, Ivo, et al., 2019)Targets of MF Therapy
| Target | Description |
|---|---|
| JAK-STAT Signaling Pathways | JAK2 mutations leading to dysregulation of the JAK-STAT signaling pathway are a characteristic feature of MF. Targeted JAK inhibitors hold potential for MF therapeutics. Ruxolitinib, a JAK1/JAK2 inhibitor, was the first FDA-approved drug for intermediate- and high-risk MF, demonstrating notable spleen size reduction and symptom improvement. |
| Epigenetic Regulation | Epigenetic modifications play a key role in the development and progression of MF. CPI-0610 targets dysregulated epigenetic regulation in MF, modulating gene expression and potentially reversing the fibrotic process. The drug is currently being studied further. |
Types of MF Therapy
- Immunotherapy
Immunotherapy approaches aim to utilize the immune system of MF individuals to identify and eliminate malignant cells. Immune checkpoint inhibitors, such as pembrolizumab and nivolumab, have shown promising results in early-stage trials, highlighting their potential as future therapeutic options for MF.
- Stem Cell Transplant Therapy
Allogeneic stem cell transplantation (SCT) remains the only potentially curative option for eligible MF individuals. SCT involves the infusion of healthy stem cells from a compatible donor, which replaces the diseased bone marrow of MF individuals, leading to the restoration of normal hematopoiesis.
Our Services
Our company has established comprehensive platforms for developing rare disease diagnostics and therapies, encompassing small molecule drug, cell therapy, gene therapy, therapeutic antibody, therapeutic peptide, and therapeutic protein. Through our dedicated platforms, we are fully devoted to advancing the development of innovative diagnostic tools and therapies for MF.
Recognizing the significance of animal disease models in the therapy development for MF, we offer our expertise in establishing animal models specifically tailored for MF. These models serve as invaluable tools to facilitate the safety evaluation and pharmacokinetics study of your drug candidates.
Animal Models of MF
| Xenograft Models | |||
| Xenograft models involve the transplantation of human MF cells or patient-derived xenografts (PDX) into immunodeficient animals. These models allow the examination of human MF cells within a living organism and enable the evaluation of disease progression, response to therapy, and the study of the MF microenvironment. | |||
| Genetically Engineered Models | |||
| Our scientists have achieved success in generating transgenic animal models by introducing genetic modifications in MF through techniques like pronuclear injection or gene targeting. Additionally, we have also obtained gene knockout and knock-in models using technologies such as CRISPR/Cas9 or homologous recombination. These models hold significant importance for studying the pathology and therapeutics of MF. | |||
| Optional Models |
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| Optional Species | Mice, Rats, Zebrafish, Non-human Primates (Baboons and Macaques), Others | ||
If you are interested in our services, please don't hesitate to contact us for more information and a detailed quotation regarding the specific services you require.
References
- Veletic, Ivo, et al. "Myelofibrosis osteoclasts are clonal and functionally impaired." Blood, The Journal of the American Society of Hematology 133.21 (2019): 2320-2324.
- Jacquelin, Sebastien, et al. "Murine models of myelofibrosis." Cancers 12.9 (2020): 2381.
All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.
