Page Contents
Gene therapy is an exciting new type of treatment and area of research for sickle cell disease. Researchers are studying ways to modify blood-forming cells so red blood cells no longer sickle, with the goal of preventing pain episodes and organ damage.
Physicians and scientists at Fred Hutch Cancer Center are helping advance gene therapy through clinical trials and research.
Sickle Cell Disease Care Tailored to You
You and your family are our top priority. At Fred Hutch Cancer Center, we offer comprehensive and compassionate care — personalized to you. You'll have access to the latest treatment options, clinical trials and supportive care services.
How Gene Therapy Works
Gene therapy is the name given to several different treatments that involve changing the genetic sequences inside our cells to help cure or treat a disease. For sickle cell disease, researchers are developing gene therapies designed to stop red blood cells from sickling.
The goal of gene therapies is to reduce or eliminate many of the complications caused by sickle cell disease, particularly recurring pain crises and the need for red blood cell transfusions.
At present, gene therapies for sickle cell disease involve the same procedure as a bone marrow or stem cell transplant. In the gene therapy transplant, the patient needs to get their blood-forming cells (stem cells) collected to have the DNA modified in a lab. After that, the patient receives preparation with chemotherapy to allow space in the bone marrow where the modified version of their own stem cells will live. Those modifications to the DNA of the stem cells help the body make red blood cells that no longer sickle.
Fred Hutch oncologist and researcher Hans-Peter Kiem, MD, PhD, and University of Washington researchers are unlocking new ways to cure sickle cell disease by editing mutated DNA right inside the bloodstream, without the need to collect the cells before genetic modifications. There is also research to develop different ways to perform the transplant without the need for chemotherapy. Find out more about how gene therapy works.
Who Can Receive Gene Therapy
The current FDA-approved gene therapy is for patients with sickle cell disease and recurrent pain crises. In deciding if gene therapy is appropriate for you, your physician will consider many factors, including your overall health, your medical history and the characteristics of your disease when determining whether gene therapy may be an option.
If gene therapy is appropriate, your care team will discuss what type of gene therapy is appropriate for you, the potential benefits, risks and what to expect during treatment.
Why Choose Fred Hutch for Sickle Cell Disease Care
The Fred Hutch Sickle Cell Disease Program includes some of the world’s leading hematologists. They can provide you with all the information to help you choose the right therapies for you and they know how to deliver them. Our physicians and researchers also do clinical trials (also called clinical studies). Through these trials, we are able to offer you therapies that aren’t offered everywhere.