According to a story from Targeted Oncology, recent data suggests that the experimental therapy IMG-7289 could be a useful treatment for patients with myelofibrosis, a rare type of cancer that affects the bone marrow. Currently, there are only two approved treatments for this disease; these are the JAK inhibitors fedratinib (marketed as Inrebic) and ruxolitinib (marketed as Jakafi).
Myelofibrosis is considered a rare type of bone marrow cancer. The disease is characterized by the excessive accumulation of abnormal stem cells in the bone marrow which trigger a process called fibrosis, or scarring. Over time, the bone marrow is replaced with scar tissue. While the exact cause of myelofibrosis is not known, genetic mutations affecting the MPL, JAK2, and CALR genes are known risk factors. Symptoms of myelofibrosis include enlarged spleen, anemia, shortness of breath, easy bruising and bleeding, greater risk of infection, bone pain, gout, fatigue, weight and appetite loss, and increased blood cell volume. As a cancer that affects stem cells, stem cell transplant can cure the disease. However, this process carries many significant risks. Most other forms of treatment are symptomatic and supportive and do not alter the course of myelofibrosis. There is a dire need for safer and more effective therapies for the disease. To learn more about myelofibrosis, click here.
Last August the US Food and Drug Administration (FDA) granted Fast Track designation to IMG-7289 as a treatment for the disease. The drug may have utility in other, somewhat related diseases, such as polycythemia vera. The drug was tested in a phase 1/2a clinical trial of myelofibrosis patients that had become intolerant to JAK inhibitors. 66 percent of patients who received IMG-7289 saw a reduction in spleen size, and 56 percent saw a reduction of at least 50 percent on other symptom assessments.
This trial is expanding into phase 2b and is recruiting new patients in the EU, the UK, and the US.
IMG-7289 is an inhibitor of the enzyme LSD1, which plays a role in the differentiation of hematopoietic cells. These cells include a type called megakaryocytes, which have been linked to mechanism of the disease. LSD1 tends to be overexpressed in myelofibrosis and related diseases, which are called myeloproliferative neoplasms.