Pediatric gene therapy for blood disorders

What is pediatric gene therapy for blood disorders?

Gene therapy is a way of treating blood disorders by using altered versions of a child’s own cells.

First, doctors get a special type of blood sample from your child that includes their stem cells (cells that grow into other cells your body needs). Then one or more of your child’s genes are edited in a lab. Before treatment, your child receives chemotherapy, to get their body ready to receive the edited genes. Finally, the stem cells with the edited genes are put back into your child’s body.

The new versions of the genes can help in different ways. Some are designed to replace the genes that originally led to your child’s blood disorder. Some give your child’s body extra help in reducing symptoms and harm caused by their disorder.

What are the benefits of pediatric gene therapy?

Gene therapies have the potential to reduce symptoms and severity of certain blood disorders.

• Sickle cell disease: Gene therapy may help children avoid painful episodes where red blood cells clog blood vessels and stop blood from reaching cells and tissue. These episodes often require treatment right away, so gene therapy may help children with sickle cell disease go to the emergency department less often because of their condition.
• Beta thalassemia: Gene therapy may help children stop needing blood transfusions to manage their condition.

Gene therapies also avoid some risks of allogeneic bone marrow transplant, which is another treatment option for some blood disorders. This type of transplant requires using stem cells from a donor. Sometimes, the donor’s cells attack the child’s body rather than bond with it. Those problems don’t occur with gene therapy because it uses a child’s own cells.

What are the side effects of pediatric gene therapy?

Gene therapy itself doesn’t have many side effects. But the chemotherapy that children receive beforehand can cause some. We watch for side effects closely and have lots of ways to address them, from medication to aromatherapy.

Side effects of chemotherapy may include:

• Nausea
• Diarrhea
• Constipation
• Fatigue
• Hair loss
• Headaches
• Loss of appetite and weight loss
• Mouth or throat sores

What are the risks of pediatric gene therapy?

Children receiving gene therapy for blood disorders may be at higher risk of developing blood or other cancers. Your child will have regular tests for years after therapy to look for signs of cancer. Your child’s health data will also be included in a registry managed by the company that makes their particular gene therapy, to help research this and other possible risks.

The chemotherapy that children receive before gene therapy can affect their ability to have children. The risk varies according to the type of chemotherapy and other factors. Someone from our Fertility Preservation team will meet with you and your child prior to treatment to talk through concerns, goals and options your child has for planning a family.

What to expect with pediatric gene therapy

Gene therapy is made with your child’s own cells. The therapy your child receives has to be manufactured first.

What to expect before pediatric gene therapy

The process of manufacturing your child’s gene therapy can take 3 months or longer.

It starts with collecting your child’s cells. Your child will take medicine that brings their stem cells into their blood. About a week later, doctors will connect your child to a machine that filters their blood and pulls out the stem cells. They may need more than one session to get enough cells.

Finally, your child’s cells are sent to a lab, which uses the cells to make a gene therapy just for them. It takes 7 or more weeks to do this.

What to expect during pediatric gene therapy

Your child will come to the hospital about a week before treatment to get ready. First, they receive chemotherapy for a few days. Then they rest while doctors monitor them and do some final tests.

The treatment itself takes a few hours. Gene therapy is given as an infusion, which means it’s a liquid that goes into your child’s blood through an IV.

What to expect after pediatric gene therapy

Your child will stay in the hospital for 3 to 6 weeks after treatment. This gives them time to rest and recover from chemotherapy, while doctors watch them for side effects and make sure that the gene therapy takes effect. Your child may have blood and other tests during this time.

Your child’s doctor will schedule regular tests and visits for the next several years, to continue monitoring for problems and measuring how well the therapy is working.

What questions should I ask my provider about pediatric gene therapy for blood disorders?

• What is your experience with gene therapies for blood disorders and other conditions?
• How many infusions has your team performed?
• Is your team involved in gene therapy research?
• Is gene therapy the best option for my child? What other options do they have?