Dallas
214-456-2382
Fax: 214-456-6133
Ste C6403
Children's Health℠ is one of the first pediatric centers certified to provide new gene therapies for sickle cell disease and beta thalassemia. We embrace and help develop innovative therapies like these so that children with complex conditions can manage their symptoms with minimal risk and need for future treatment.
214-456-2382
Fax: 214-456-6133
Ste C6403
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.
Gene therapies have the potential to reduce symptoms and severity of certain blood disorders.
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.
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:
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.
Gene therapy is made with your child’s own cells. The therapy your child receives has to be manufactured first.
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.
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.
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.
A Child Life specialist will meet with you before therapy to cover all the details you need, including any diet restrictions and how to plan for missing school.
Our team is on the forefront of this exciting new area of treatment. We actively contribute to research and guidelines that help improve how gene therapy is given so that it has maximum benefit for the children who need it.
Gene therapy uses altered versions of a child’s own cells to treat a disorder. Scientists alter or add specific genes inside the child’s cells. Then they put the cells back into the child’s body, where they embed and make more versions of themselves. As the child grows, they keep producing the new or altered genes, helping them manage or reduce symptoms of their disorder.
Children stay in the hospital to recover from the chemotherapy they get prior to treatment. Chemotherapy clears away the stem cells inside a child’s bones so that the gene therapy can take their place. But it also makes children feel tired and sick, and their bodies need time to rest and rebuild.
Texas Medicaid has established guidelines for covering multiple ex vivo gene therapies. However, providers are in active discussion with Texas Medicaid on how these important therapies will be obtained, billed, and reimbursed. Unfortunately, this has resulted in considerable delays in the initiation of treatment, leaving hospitals without a clear process to move forward.
We know this can be a frustrating waiting process as insurance provider guidelines are created. Children’s Health will continue to stay updated on new information so that we can support your child.