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 our immune system 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 those cells 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—From the first discovered to the first conquered molecular disease

 

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.

 


For those interested in learning more about blood health, the importance of funding biomedical research, and how you can get involved, please visit www.hematology.org/patients. If you would like to donate to our efforts to conquer blood diseases, please visit www.hematology.org/foundation.