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Embarking on Promising Paths: Myelofibrosis Clinical Trials

Myelofibrosis often raises numerous questions, but there's newfound optimism. Our platform links individuals to the latest clinical trials, particularly emphasizing those related to myelofibrosis. We make it easier for patients to find and take part in new medical studies, giving them access to advanced treatments.

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    You can receive a guidebook with information about myelofibrosis by filling out the form.

      Find Your Treatment Now

      A Patient Relations Coordinator will reach out to you soon.

      Personal data is fully confidential and 100% secure

      You can receive a guidebook with information about myelofibrosis by filling out the form.

      The Latest in Myelofibrosis Treatment Options

      Our matching system bridges the gap to cutting-edge clinical trials, unlocking a realm of possibilities for individuals confronting myelofibrosis. Within our platform, you’ll discover comprehensive details about clinical trials catering to all phases of myelofibrosis. These trials have new treatments not for everyone, made to target the specific cause of your myelofibrosis diagnosis. This presents you with an opportunity to proactively combat the disease alongside us.

       

      How our system works

      After checking your medical records and using our AI system, we find clinical trials that you might qualify for. We also offer a concierge service to help you smoothly transition into the clinical trial.

      Our team of nurses and doctors will handle all aspects of your involvement in the trial, from start to finish.

      All the services provided are completely free for you and your treating oncologist.

       

      Understanding the disease

       

      What is Myelofibrosis?

      Myelofibrosis is a rare blood disorder where too many blood cells grow in the bone marrow. Here’s a breakdown of the key aspects:

      1. Bone Marrow Dysfunction: In myelofibrosis, there is a disruption in the normal bone marrow function. The bone marrow undergoes infiltration with fibrous tissue, a process known as fibrosis, instead of producing blood cells efficiently. This fibrosis replaces the healthy bone marrow and impairs its ability to produce an adequate number of blood cells.

      2. Bone marrow products: this organ produces three types of blood cells. Red blood cells carry oxygen. White blood cells fight infections. Platelets assist in blood clotting. In myelofibrosis, the fibrous tissue crowds out the space required for the production of these blood cell types.

      3. Symptoms: Due to reduced red blood cell production (anemia), patients may experience fatigue, weakness, and pallor. Having fewer white blood cells can increase the risk of infections. Additionally, a deficiency in platelets can lead to bruising, bleeding, or longer clotting times.

      4. Spleen Enlargement: Many individuals with myelofibrosis develop an enlarged spleen (splenomegaly). When the bone marrow doesn’t produce enough blood cells, the spleen can become overactive. This can lead to stomach pain and a sensation of feeling full quickly.

      5. Causes: Myelofibrosis can arise as a primary disorder, known as primary myelofibrosis, or as a secondary condition associated with other bone marrow disorders like polycythemia vera or essential thrombocythemia. These conditions are collectively known as myeloproliferative neoplasms (MPNs).

      6. Genetic Mutations: Certain genetic mutations, such as those involving the JAK2, CALR, or MPL genes, are commonly associated with myelofibrosis. These mutations play a role in driving abnormal cell growth in the bone marrow.

       

      NGS testing and clinical trials

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

      The genetic mutations are also known as biomarkers, and many are known to be related to the origin of the disease. Some of the more common biomarkers found to cause myelofibrosis are the following:

      1. JAK2 Mutations: One of the most prevalent biomarkers associated with myelofibrosis is mutations in the Janus kinase 2 (JAK2) gene, particularly the V617F mutation. These mutations lead to an aberrant activation of the JAK2 signaling pathway, resulting in the excessive production of blood cells and contributing to the development of myelofibrosis. JAK2 mutations are found in a substantial percentage of myelofibrosis cases.

      2. CALR Mutations: Mutations in the CALR (Calreticulin) gene are another common biomarker in myelofibrosis. CALR mutations alter the production of the CALR protein, which plays a role in cell signaling. These mutations are often detected in cases where JAK2 mutations are absent, providing an alternative genetic basis for the disease.

      3. 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. They can contribute to the pathogenesis of the disease.

      4. Triple-Negative Status: In some cases of myelofibrosis, 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 be responsible for driving the development of myelofibrosis in these individuals. Research continues to uncover additional biomarkers that may explain these cases.

       

      What is the treatment for
      Myelofibrosis?

      This is a question that many people have, and the responses are continuously advancing. Myelofibrosis comprises different subtypes, and one of the most common is Primary Myelofibrosis. Treatment approaches can differ based on the disease’s stage, with more advanced stages often necessitating a combination of therapies. Detecting the initial indicators of myelofibrosis can be challenging, but early detection can lead to a quicker diagnosis and potentially enhance treatment outcomes.

      • Usual medication:

      1. JAK Inhibitors: Medications like ruxolitinib (Jakafi) and fedratinib (Inrebic).

      2. Hydroxyurea (Droxia, Hydrea)

      3. IMID (Immunomodulatory drugs): Lenalidomide (Revlimid)

      • Blood Transfusions: For individuals with severe anemia due to myelofibrosis, regular blood transfusions may be necessary to maintain healthy hemoglobin levels.
      • Stem Cell Transplantation: A hematopoietic stem cell transplant (also known as a bone marrow transplant) may be considered for eligible patients, especially in younger individuals with advanced disease. This procedure involves replacing the patient’s unhealthy bone marrow with healthy stem cells from a compatible donor.
      • Splenectomy: In some cases, surgical removal of the enlarged spleen (splenomegaly) may be considered if it causes severe discomfort or complications.

       

      The Role of Clinical Trials in
      Myelofibrosis

      Clinical trials play a crucial role in the treatment of myelofibrosis for several reasons:

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

      2. 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.

      3. 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.

      4. 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.

      5. Promoting Research Advancements: Clinical trials play a pivotal role in advancing research, aiding scientists in understanding the effectiveness of new treatments and identifying which patients benefit the most. This can pave the way for the development of more effective therapeutic approaches and personalized medicine strategies.

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      How Massive Bio Helps Patients With Myelofibrosis

      Massive Bio specializes in finding clinical trials of new treatments for MF and related conditions. If you’ve been diagnosed with MF or any other myeloproliferative neoplasm, we’re here to help. That’s true even if you require advanced myelofibrosis treatment or advanced blood cancer treatment for any other type of myeloproliferative disorder. If your MF has progressed, let us help you find the best treatment options. Using our artificial intelligence-based platform, we can match you to clinical trials of advanced myelofibrosis treatment options today. If you aren’t sure if you have MF or a related condition, that’s okay. Additional testing can help determine your exact diagnosis.

       

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      What is a Myelofibrosis clinical trial?

      Clinical trials test the latest scientific advancements in Myelofibrosis treatment. Patients who choose to enroll in trials can receive cutting-edge treatment and high-quality care under the direction of scientists, doctors, and researchers. Myelofibrosis patients might gain access to promising drugs and innovative treatments long before they're made available to the public.

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      Dr. Arturo explains:
      What Are Clinical Trials?

      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 treatments. 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 treatment 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.

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      • Where can I find clinical research studies in my area?

        Where can I find clinical research studies in my area?

        Your doctor may know of a clinical research study being conducted in your area that’s recruiting participants and is right for you. However, Massive Bio uses its artificial intelligence-powered platform to match patients to clinical research studies of treatments that give you the best chance of a positive outcome and are being conducted in a geographical location that makes sense for you.

      • Can I continue seeing my doctor or oncologist while also taking services of Massive Bio?

        Can I continue seeing my doctor or oncologist while also taking services of Massive Bio?

        Yes, Massive Bio keeps your doctor up to date on your status throughout your participation.