Embarking on Promising Paths: Myelofibrosis Clinical Trials

By reviewing your medical records and utilizing our AI system, we identify clinical trials you may qualify for. We also provide a concierge service to assist with your smooth transition into these trials. Our team of nurses and doctors manages all aspects of your participation from start to finish. All services are completely free for you and your oncologist.

Myelofibrosis, a chronic myeloproliferative disorder, is a rare type of blood cancer in which normal bone marrow tissue is replaced by fibrous scar tissue (fibrosis). Since the bone marrow’s primary role is to produce red blood cells, white blood cells, and platelets, fibrosis disrupts the production of these cells, leading to various symptoms and complications.

Myelofibrosis is classified into two main types:

Primary Myelofibrosis: This form develops independently, without any preceding blood disorder. A genetic mutation in bone marrow cells causes too many cells to grow and inflammation, leading to fibrosis. Scar tissue forms in the bone marrow, reducing its function and leading to fewer blood cells of all types (pancytopenia).

Secondary Myelofibrosis: This is a complication of other blood disorders. One example is essential thrombocythemia (Post ET Myelofibrosis), which causes an excess of platelets. Another example is polycythemia vera (Post Polycitemia Vera Myelofibrosis), which results in too many red blood cells. It evolves from an existing condition, where the bone marrow gradually becomes fibrotic, leading to symptoms and complications similar to those of primary myelofibrosis.

Severe Fatigue: Persistent tiredness because of anemia, affecting up to 50% of patients.

Abdominal Discomfort: A heavy sensation in the left upper abdomen, often because of an enlarged spleen.

Enlarged Spleen (Splenomegaly): The spleen becomes enlarged due to the accumulation of abnormal blood cells and fibrosis. This can lead to pain or a feeling of fullness in the abdomen, especially after eating small amounts.

Unintentional Weight Loss: Significant weight loss without trying, commonly associated with disease progression.

Bone Pain: Pain in the bones, especially in the legs or ribs, because of abnormal marrow activity.

Night Sweats: Excessive sweating during the night, often soaking clothes.
Easy Bruising: Frequent bruising because of a low platelet count.

Recognizing myelofibrosis symptoms, such as fatigue, anemia, and an enlarged spleen, is vital for early diagnosis and effective management. Early detection allows timely treatment, which helps manage symptoms, prevent complications, and improve patient outcomes.

Low Risk: Indicates the mildest form of myelofibrosis.
Intermediate-1 Risk: Suggests moderate disease progression.
Intermediate-2 Risk: Indicates more rapid disease progression, requiring active treatment.
High Risk: Suggests an advanced form of myelofibrosis that requires intensive treatment.

Accurate staging helps tailor options to specific disease risks, optimizing efficacy and safety for myelofibrosis patients.

Untreated Myelofibrosis: Refers to patients who have been diagnosed but have not yet received any specific treatment. Clinical trials for these patients often focus on novel therapies and combinations to improve initial treatment outcomes and long-term prognosis.

Treated Myelofibrosis: Refers to patients who have already undergone treatment but may have experienced disease progression, relapse, or insufficient response. Clinical trials for this group focus on new treatment options for relapsed, refractory, or treatment-intolerant patients.

Clinical trials are available for myelofibrosis patients. This includes both those who have been treated and those who have not. These trials offer new options that may improve disease management and outcomes.

Stem Cell Transplant Eligible: Typically offered to patients under 70 years old without major comorbidities, diagnosed with high-risk myelofibrosis resistant to JAK2 inhibitors or other non-transplant treatments.

Stem Cell Transplant Ineligible: Patients with mild symptoms related to myelofibrosis may not be eligible, as the adverse effects of the transplant could outweigh the benefits compared to existing treatments.

Clinical trials are available for myelofibrosis patients, whether they are eligible or ineligible for stem cell transplants. These trials offer new options and possible strategies to improve transplant outcomes, manage symptoms, and slow disease progression, expanding care options for all patients.

This is a common question, and the answers are continuously evolving. Myelofibrosis has different subtypes, with Primary Myelofibrosis being one of the most common. Treatment approaches vary based on the disease stage, with more advanced stages often requiring a combination of therapies.

Early detection of myelofibrosis can be challenging, but it can lead to a quicker diagnosis and potentially improve treatment outcomes.

JAK Inhibitors: These drugs target and inhibit the overactive JAK protein in myelofibrosis, reducing inflammation, spleen size, and abnormal blood cell production.

Hydroxyurea: This drug reduces the overproduction of blood cells and controls symptoms like splenomegaly in patients who are ineligible for stem cell transplantation or JAK inhibitors.

Splenectomy is surgery to remove the spleen. This procedure is done for enlarged spleen treatment when causes serious pain or when other treatments have not been effective.

Hematopoietic Cell Transplantation: Typically reserved for younger patients or those with high-risk disease due to the procedure’s risks.

EPO-stimulating Agents help the bone marrow produce more red blood cells. They are used to treat anemia due to Myelofibrosis.

Blood Transfusions: For individuals with severe anemia because of myelofibrosis, regular blood transfusions may be necessary to maintain healthy hemoglobin levels.

Next-Generation Sequencing (NGS) is essential for linking patients with specific genetic mutations in their cancers to clinical trials for targeted options. This approach, part of personalized medicine, increases the effectiveness and success rates of these trials by ensuring patients receive options most likely to work for their particular cancer type.

The genetic mutations, also known as biomarkers, are often related to the disease’s origin. Some common biomarkers associated with myelofibrosis include:

JAK2 Mutations: Changes in the JAK2 gene, especially V617F, are common in myelofibrosis, seen in about half of patients. These mutations result in the excessive production of blood cells, contributing to the development of myelofibrosis.

CALR: A mutated protein that interacts with the MPL receptor on hematopoietic stem cells, activating the JAK-STAT pathway and leading to cell proliferation and inflammation. It is found especially in patients without the JAK2 mutation.

MPL Mutations: Mutations in the MPL (Myeloproliferative Leukemia Virus Oncogene) gene are less common but are still associated with myelofibrosis. MPL mutations affect the MPL receptor, which regulates blood cell production, contributing to the disease’s pathogenesis.

Triple-Negative Status: In some myelofibrosis cases, none of the above-mentioned mutations (JAK2, CALR, or MPL) are present. These cases are termed “triple-negative.” Despite the absence of these mutations, other genetic alterations or factors may drive myelofibrosis development in these individuals. Research continues to uncover additional biomarkers to explain these cases.

ASXL1: A mutated gene involved in DNA expression, associated with a poorer prognosis and higher risk of disease progression.

EZH2: A mutation involved in gene expression regulation, linked to a more aggressive disease course and poor prognosis.

SRSF2: Mutations that impair RNA splicing, leading to abnormal protein production and poor outcomes in high-risk disease.

Clinical trials play a crucial role for several reasons:

Access to Innovative Options: Clinical trials offer individuals the opportunity to access novel possibilities, particularly crucial for serious conditions like myelofibrosis, where conventional therapies may be less effective.

Enhancing Outcomes: Participation in clinical trials allows patients to contribute valuable insights that may enhance survival rates and overall well-being for future myelofibrosis patients.

Comprehensive Medical Attention: Patients enrolled in clinical trials typically receive comprehensive care, with a dedicated team of healthcare providers and researchers closely monitoring their progress.

Hope for the Future: For many individuals dealing with myelofibrosis, involvement in a clinical trial instills hope, both for their own recovery and for the potential positive impact on future patients.

Promoting Research Advancements: Clinical trials play a pivotal role in advancing research, aiding scientists in understanding the effectiveness of new options and identifying which patients benefit the most.

Cancer is an unfortunate reality that touches most of us at some point in our lives. If you or a loved one has cancer, you may have heard or read that clinical trials could offer access to innovative new options. But what exactly is a clinical trial? In this video, Massive Bio co-founder Arturo Loaiza-Bonilla, MD, explains how clinical trials work, what to expect if you enroll in one, and why a clinical trial can be an important option for many cancer patients.

We dream of the day when cancer disappears from our lives. Massive Bio is working tirelessly on achieving that goal.