Exploring New Horizons: Promising Clinical Trials for Myelodysplastic Syndrome

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.

Myelodysplastic Syndrome (MDS) can lead to several complications that may impact your daily life. Understanding these complications is crucial for managing the disease effectively and improving your quality of life. Knowing what to watch for can help you and your healthcare team take proactive steps to manage these challenges.

Acute Myeloid Leukemia (AML): As normal bone marrow function decreases, 40-50% of MDS cases may deteriorate into AML, a form of cancer. This transition occurs when blasts (immature blood cells) in the bone marrow exceed 20%.

• Severe Anemia: A significant reduction in red blood cells, leading to chronic fatigue, weakness, and shortness of breath.
• Thrombocytopenia: A condition characterized by a low platelet count, which can cause easy bruising, frequent nosebleeds, and prolonged bleeding from cuts due to impaired blood clotting.
• Neutropenia: A reduction in white blood cells, resulting in an increased risk of infections.
• Pancytopenia: A reduction in all three types of blood cells (red blood cells, white blood cells, and platelets).

Once MDS is diagnosed, determining the risk classification is crucial. Patient risk status is assessed based on complete blood counts, the proportion of immature cells in the bone marrow, and the type of genetic abnormalities present in bone marrow cells. This classification helps predict disease progression, guides treatment decisions, and provides insight into prognosis.

Very Low Risk: Mild disease with only minor abnormalities in blood counts, and a low chance of developing leukemia.

Low Risk: Blood cell counts are low, causing mild symptoms. The risk of disease progression or developing leukemia is still low, but some treatment may be needed to manage symptoms.

Moderate-Low Risk: More noticeable abnormalities in blood counts, with a moderate risk of disease worsening.

Moderate-High Risk: Significantly low blood counts leading to notable symptoms, with a higher risk of progressing to leukemia. More intensive treatments may be required.

High Risk: Very low blood cell counts with serious abnormalities in the bone marrow, with a high likelihood of progression to AML. Aggressive treatments, such as chemotherapy and bone marrow transplant, are often necessary.

Very High Risk: Severe disease with extremely low blood counts and a high level of abnormal cells in the bone marrow. There is a very high risk of rapidly developing AML, requiring intensive treatment.

Accurate risk classification helps tailor treatments to specific disease risks, optimizing efficacy and safety for myelodysplastic syndrome patients.

Untreated Myelodysplastic Syndrome: 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 Myelodysplastic Syndrome: 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.

Relapsed/Refractory MDS: Refers to MDS that is difficult to control, either returning after treatment (relapsed) or not responding to initial treatment (refractory). Patients with relapsed/refractory MDS typically require different or more aggressive options.

Non-Relapsed/Refractory MDS: Refers to MDS that responds well to initial approach and does not return or worsen afterward. Patients can manage their symptoms and maintain better disease control with standard treatment options.

Treatment for MDS depends on the patient’s clinical risk status, including the likelihood of developing leukemia or remaining stable. By identifying the risk level, physicians can tailor treatments to ensure personalized care and better disease management.

• Erythropoiesis-Stimulating Agents: Drugs that stimulate the bone marrow to produce more red blood cells, helping to treat anemia and reduce the need for blood transfusions. These are typically used in patients with low-risk MDS.
• Hypomethylating Agents: Medications that inhibit DNA methylation, promoting the production of normal blood cells and reducing abnormal cell growth. These belong to the group of lower-intensity therapies for MDS.
• Immunosuppressive Therapy: Drugs that enhance blood cell production by reducing the immune system’s attack on the bone marrow.
• IDH Inhibitors: Target mutated IDH proteins, helping to decrease the production of abnormal cells and slow disease progression. These are considered low-intensity therapies.
• Chemotherapy: A systemic therapy that targets and kills rapidly dividing abnormal cells, aiming to reduce the number of blasts and prevent progression to leukemia.
• Hematopoietic Cell Transplantation: A procedure that replaces diseased bone marrow with healthy donor stem cells, offering a potential cure for MDS, particularly in younger patients.
• Supportive Care: Includes treatments like blood transfusions and antibiotics to manage symptoms, improve quality of life, and prevent complications without directly targeting MDS.
• Clinical Trials: Research studies that test new treatments or drugs for MDS, providing patients with access to innovative therapies not yet widely available.

Next-Generation Sequencing (NGS) plays a crucial role in connecting patients with specific genetic changes in their cancer to clinical trials that offer targeted treatments. This personalized approach helps match you with therapies that are more likely to be effective for your specific type of cancer, increasing the chances of success in these trials. By using NGS, we can better understand your cancer and find the best treatment options for you.

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

• TP53: A mutated gene that leads to the loss of regulatory functions controlling cell growth, allowing abnormal cells to survive and proliferate. It is associated with poor prognosis and a higher risk of disease progression to leukemia.
• SF3B1: A mutated gene that results in defective production of functional proteins. It is linked to a favorable prognosis.
• ASXL1: Mutations in this gene cause uncontrolled cell growth and are associated with a worse prognosis and a higher risk of progression to AML.
• IDH1/2: Mutations in these genes lead to the production of an abnormal substance that promotes leukemic transformation. These mutations can be targeted by specific IDH inhibitors for treatment.

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