Is there a cure for sickle cell disease?

Many kids and families long for a cure for sickle cell disease. Also called sickle cell anemia, this blood disorder causes red blood cells to become hard and crescent shaped instead of round and flexible. These cells can block blood flow, leading to pain, fatigue and damage to organs over time.

For a long time, stem cell transplant was the only cure for sickle cell disease – and was typically only an option for kids who have a sibling who is a perfect donor match. But now, there’s a new option: gene therapy.

“Gene therapy is exciting because now we can offer a curative option to more patients,” says Tiffany Simms-Waldrip, M.D., Pediatric Hematologist/Oncologist at Children’s Health℠ and Clinical Professor at UT Southwestern. “While both gene therapy and stem cell transplant are curative, they’re also both intensive treatments that require many months of care and hospitalization. I always encourage families to talk to their hematologist about the best option for them.”

Stem cell transplant for sickle cell disease

A stem cell transplant gives kids new cells that can make regular red blood cells instead of sickle-shaped red blood cells. This can cure sickle cell disease.

What happens during a stem cell transplant for sickle cell disease?

First, kids with sickle cell disease and their families meet with their hematologist and the transplant team to discuss all the steps of a transplant procedure and if it might be right for their child. The procedure includes:

• Finding a donor. Ideally, the donor is a sibling. Although it is uncommon is sickle cell patients, an unrelated donor who is a perfect match may be used if the patient does not have a sibling who is a match.

• Doing a thorough health evaluation. Doctors will check organ function and may do other tests to make sure a child is healthy enough for the procedure.

• Receiving chemotherapy. Chemotherapy removes a child’s own bone marrow and helps prevent their body from rejecting donor cells.

• Removing bone marrow from the donor’s hip. During this surgical procedure, the marrow is removed from the donor and infused into the child with sickle cell disease, usually the same day.

• Taking immunosuppressive medicine. These medicines help to keep the new donor cells from reacting to the patient and are typically taken for several months after the transplant.

• Staying in the hospital for a few weeks. From start to finish, kids are usually in the hospital for 6-8 weeks for a stem cell transplant.

What are the benefits of a stem cell transplant for sickle cell disease?

For children with a fully-matched sibling donor, a stem cell transplant offers:

• Around a 90-95% cure rate.

• Lower risk of long-term complications.

• No need for lifelong sickle cell treatments.

What are the risks of a stem cell transplant for sickle cell disease?

People who have a stem cell transplant may develop:

• Side effects of chemotherapy. The chemotherapy used before transplant can cause side effects like nausea, vomiting, hair loss, and mouth sores. It may also affect your child's ability to have biological children later in life. Our care team will walk you through the risks and benefits and talk with you about options to preserve fertility before treatment starts.

• Weakened immune system. Being more likely to get infections in the weeks after transplant and for as long as they are taking medicine to suppress the immune system.

• Graft-versus-host-disease (GVHD). GVHD is when the new donor cells do not recognize the patient/patient’s body and react causing inflammation and damage.

Can a sibling who has sickle cell trait be a stem cell donor?

Yes, siblings with sickle cell trait can be stem cell donors.

Sickle cell disease is caused by a change in the genes that make hemoglobin. Children get one hemoglobin gene from each parent.

• Sickle cell trait means a child has one sickle hemoglobin gene and one normal hemoglobin gene. People with sickle cell trait don’t usually experience symptoms of sickle cell disease.

• Sickle cell disease means a child has two copies of the sickle hemoglobin gene, one from each parent.

“The goal of transplant is to reduce the amount of sickle hemoglobin the body produces,” Dr. Simms-Waldrip says. “So with a donor who has sickle cell trait, the recipient may go from making 100% sickle hemoglobin to 30-50% sickle hemoglobin – and that can greatly improve their health.”

Gene therapy for sickle cell disease

Gene therapy is a new curative option for sickle cell disease. It was first approved by the FDA for certain kids 12 and older in 2023. And a gene therapy option for kids ages 2 and older was FDA approved in 2026.

Gene therapy changes a child’s stem cells so they make less sickle hemoglobin, helping reduce sickle cell symptoms.

“Once you infuse the modified stem cells, ideally they take over the majority of the blood-making process,” Dr. Simms-Waldrip says.

What happens during gene therapy for sickle cell disease?

The process of getting gene therapy for sickle cell disease can take about a year from start to finish and includes multiple hospital admissions, including a hospital stay of 4-8 weeks.

First, the child and family have a consultation to discuss what the process looks like and the potential risks or side effects. Then, they can decide if this is the right treatment for them and which type of gene therapy is the best option. Gene therapy treatment for sickle cell includes:

• Making sure a child is healthy enough to undergo gene therapy.

• Getting insurance authorization, which can take several months.

• A procedure to collect stem cells. They may need to do this more than once.

• Sending the stem cells to the manufacturer, who genetically modifies the cells. (This can take 2-3 months for some therapies and up to 6 months for others.)

• Getting chemotherapy to reduce cells that make sickle hemoglobin and make room for the modified cells.

• Getting an infusion with the modified stem cells.

• Staying in the hospital for a few weeks after the infusion waiting for the new cells to grow.

What are the benefits of gene therapy for sickle cell disease?

• Gene therapy is considered a curative option for kids with sickle cell disease, meaning they generally do not need treatments for sickle cell disease after gene therapy.

• Kids who do gene therapy are not at risk for GVHD, because they're infused with their own cells.

What are the risks of gene therapy for sickle cell disease?

• The chemotherapy used before transplant can cause side effects like nausea, vomiting, hair loss, and mouth sores. It may also affect your child's ability to have biological children later in life. Our care team will walk you through the risks and benefits, and talk with you about options to preserve fertility before treatment starts.

• Kids are more likely to get infections in the weeks after transplant because their immune system is weak.

“Gene therapy is the first new curative option for sickle cell in a long time and it's exciting to see patients benefit from it,” Dr. Simms-Waldrip says. “Right now, it's only available for some kids with sickle cell disease. But I believe over time, it'll be available to a larger group of kids and families.”

Explore treatment options for sickle cell disease

Children's Health comprehensive care for children with sickle cell disease, including access to advanced treatments such as stem cell transplant and gene therapy. Our multidisciplinary team works closely with families to help them understand their options and determine the best path for their child. Learn more about treatment options and our program.