Myeloproliferative neoplasms (MPNs) are caused by genetic mutations in bone marrow stem cells that disrupt the body’s ability to produce healthy blood cells.
Approximately 300,000 people in the United States live with MPNs today. For many, the disease gradually worsens over time. Around 20-25% of patients progress to advanced disease, including Myelofibrosis (MF), a severe condition marked by progressive scarring of the bone marrow. MF causes debilitating symptoms, including a swollen spleen that can grow from the size of a closed fist to the size of a rugby ball, and is associated with a median survival of 5-7 years. In some cases, MPNs transform into secondary leukaemia, where average life expectancy is less than 12 months.
Current treatments, such as JAK inhibitors, can help manage symptoms, but they are not truly disease modifying, so the prognosis remains relatively unchanged. They do not eliminate the cells that drive the disease and can often cause significant side effects. As a result, there is an urgent need for therapies that go deeper and address the root cause of MPNs.
