Gene Therapy for Sickle Cell Disease

You will undergo a thorough evaluation with Duke sickle cell disease and stem cell transplant specialists to determine if gene therapy is right for you. You may be a candidate if you are at least 12 years old and experience repeated pain events -- also called vaso-occlusive crises. If you have had a stroke or are at increased risk for stroke, gene therapy may not be recommended. Your evaluation will also include meeting with a financial counselor to determine whether your health insurance covers this new therapy.

If gene therapy is an appropriate option, the first step is to receive infusions of healthy, non-sickling blood for 60 days. This is followed by apheresis. During this procedure, blood is drawn from your body, filtered to collect your stem cells (which are instrumental in creating new blood cells), and returned to your body. In some cases, apheresis may need to be repeated to collect enough stem cells. The cells will be sent to a specialized manufacturing facility, where they will be altered to create your personalized gene therapy.

It may take ten weeks to six months after your cells are collected to manufacture, test, and send your personalized gene therapy treatment back to Duke. It is important that you stay healthy during this time. If you are not receiving care for sickle cell disease at Duke, we will coordinate care with doctors closer to your home.

Once we receive your gene therapy treatment, you will undergo chemotherapy to make room in your blood and bone marrow for your manufactured cells. This treatment will take place in the hospital where you will stay until you have recovered from any side effects of chemotherapy. This will allow your team of providers to monitor your condition and promptly address any issues. 

You’ll receive the gene therapy infusion through an IV placed in your arm. It will take place in the hospital and will last about 10 minutes. Afterward, your doctors will watch for side effects. Because the manufactured cells originated from your own body, you are not at risk for graft-versus-host disease. However, your immune system will need time to recover, so you will stay in the hospital until your medical team determines it is safe for you to go home.  A typical stay is from three to six weeks.

For about one to three months after gene therapy, some people -- mostly children -- will need to stay close to Duke. This ensures easy access to your medical team should you experience any health problems. After discharge, you will visit the outpatient clinic for frequent checkups. Duke Patient Information Services will work with you and your loved ones to find nearby affordable lodging as needed. Staff can also check to see if you’re eligible for financial assistance for housing.

Duke’s FDA-approved Risk Evaluation and Mitigation Strategy Program can address any risks and side effects associated with gene therapy. If you have a medical emergency during your recovery, you’ll use a special ID card to show emergency room staff and providers that you are a gene therapy patient. This will help our team provide you with specialized treatment.

Our providers led clinical trials on this treatment and took part in specialized training to administer gene therapy. This means you and your family can feel confident that your providers are among the country’s leaders in the field.

Our sickle cell disease specialists have a long history of caring for people with sickle cell disease and partnering with other centers to coordinate complex care. We routinely work with diverse teams of providers -- including stem cell transplant and cellular therapy specialists -- to ensure people with sickle cell disease receive the best care possible. We are dedicated to investigating new and effective therapies for sickle cell disease with the ultimate goal of finding a cure accessible to all.

We offer inpatient units designed specifically for people receiving gene therapy, stem cell transplants, cellular therapies, and for other patients with weakened immune systems. These units are some of the largest in the country and are equipped with dedicated ventilation systems to reduce the risk for infections. Our family-friendly pediatric unit includes a large activity room, patient rooms with accommodations for caregivers, a common kitchen, washer and dryer facilities, and more.