May 22, 2024

New Treatment Option Being Researched for Severe Blood Disorders of the Bone Marrow

A recent international clinical trial has provided hope for patients suffering from severe forms of myelodysplastic neoplasms (MDS), a condition in which the healthy maturation of blood cells is impaired. Higher-risk MDS, which is characterized by rapid progression and severe symptoms, often leads to acute leukemia and has limited treatment options for patients who are unable to undergo curative or intensive treatments such as stem cell transplantation or high-dose chemotherapy.

Led by Professor Uwe Platzbecker from the University of Leipzig Medical Center, the trial involved a collaboration with a large international team of researchers and tested a new treatment combination for higher-risk MDS. MDS affects approximately 4,000 people in Germany each year and can cause anemia, infections, and an increased risk of bleeding. Patients with higher-risk MDS have a lower life expectancy compared to individuals of the same age.

Typically, stem cell transplantation or chemotherapy are used to treat higher-risk MDS. However, these treatments are not suitable for all patients, highlighting the need for alternative therapeutic approaches. In the international trial, conducted at 54 sites in 17 countries, researchers tested a combination of hypomethylating agents (HMA), the current standard of care for this patient population, with the new active ingredient sabatolimab.

Sabatolimab is an intravenously administered immunotherapeutic agent that targets specific domains on myeloid immune and tumor cells. It belongs to a class of drugs known as immune checkpoint inhibitors, which override tumor survival mechanisms by targeting areas of the immune system. This approach prevents the cancer from evading the immune system, which would otherwise attack it.

The study, detailed in The Lancet Haematology journal, represents the first randomized trial involving immunotherapy in combination with the standard of care for myeloid neoplasms. Professor Uwe Platzbecker, a renowned hematologist and the director of the Department for Hematology, Cell Therapy, Hemostaseology, and Infectious Diseases at the University of Leipzig Medical Center, has been conducting research on myelodysplastic neoplasms for over two decades.

Although the addition of sabatolimab did not significantly improve response rates or progression-free survival in this study, it demonstrated a manageable safety profile. This is particularly significant for patients who have limited treatment options available to them. Common side effects included a reduction in white blood cells (neutropenia) and platelets (thrombocytopenia), which occurred in both the sabatolimab and placebo groups.

Despite the initial results, researchers remain hopeful about combining sabatolimab with the standard treatment. A randomized phase 3 trial, the final stage of drug development, is already underway. This follow-up study aims to evaluate the potential benefits of combining sabatolimab with azacitidine, the most commonly used HMA, in terms of overall patient survival.

The research conducted in this international clinical trial represents a significant step towards finding improved treatment options for patients with severe blood disorders of the bone marrow. By exploring the potential of immunotherapy in combination with existing standards of care, researchers hope to improve patient outcomes and increase survival rates for those affected by higher-risk MDS.

1. Source: Coherent Market Insights, Public sources, Desk research
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