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Blood Disorder Research Is Making Advancements Daily

Alexis Thompson, M.D.

President, American Society of Hematology

From life-long blood disorders like sickle cell disease and hemophilia to sudden, life-altering blood cancer diagnoses like leukemia or multiple myeloma, blood diseases can be the source of so much uncertainty for patients and their caregivers. Fortunately, there is much to be hopeful for in the understanding and treatment of these disorders, and the American Society of Hematology (ASH) is working with scientists, research institutions, pharmaceutical companies and policymakers to accelerate scientific discovery, drug development and deployment of new therapies to conquer blood diseases.

Harnessing immunity to defeat cancer

In 2017, a revolutionary new therapy made the leap from the research bench to the bedside when the U.S. Food and Drug Administration (FDA) approved a groundbreaking CAR T-cell therapy to treat certain leukemia patients. In a process that once would have sounded like science fiction, this therapy involves taking a person’s immune cells from their bone marrow, reprogramming them to target their cancer and returning them to the patient.

CAR T-cell therapy is a prime example of precision medicine’s potential to revolutionize the care we deliver to our patients. CAR-T cell therapy has also been approved for some patients with another blood cancer called lymphoma. Almost every day, clinical trials are reporting further success in treating patients with other types of blood cancers, such as multiple myeloma, using CAR T-cells. ASH is committed to improving the safety, effectiveness and availability of revolutionary cancer therapies, and we are actively promoting research to hasten their delivery.

Sickle cell disease (SCD) is an inherited blood disorder that affects nearly 100,000 Americans, primarily of African, Mediterranean and Middle Eastern descent. SCD causes red blood cells to become rigid and sickle-shaped, leading to reduced oxygen flow to almost every organ, causing crises of severe pain, stroke, organ damage and even death.

While treatment options are currently limited for those with SCD, several research teams from around the globe are using the latest advancements in precision medicine to make cures in SCD possible in the near future. This includes calling on gene therapies and genome editing techniques to correct the genes responsible for this disease. While it’s still too early to deliver many of these therapies and cures to people, scientists are hard at work making them a reality.

Today, tomorrow, and beyond

In the 1960s, the first successful trials in chemotherapy were reported in people with leukemia. Today, with scientific breakthroughs in precision medicine, hematologists are mapping the frontiers of medicine, and ASH is honored to play a key role in fostering this groundbreaking work.

Every day, our 17,000 members and counting are hard at work to improve the lives of millions of patients around the world.

Perhaps you or a loved one has been affected by a blood disorder, and you would like to support new research that will contribute to novel treatments for the disease. By becoming an advocate for hematology, you can help increase public awareness about blood disorders and support state and federal funding for research, which is critically needed to make this exciting science count for patients. 

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