SKYSONA

What is SKYSONA?

Skysona is a gene therapy treatment for boys with early signs of the cerebral form of ALD. This disease causes serious damage to the brain and nervous system. If it isn’t treated early, the damage can be permanent — and the disease can be life-threatening.

Skysona works by using your child’s own blood stem cells. Our doctors collect some of your child’s stem cells, fix the gene that’s causing the problem, and then return the repaired stem cells to your child’s body. These new, healthy cells can help slow or stop brain damage.

Who qualifies to get SKYSONA?

Skysona gene therapy is approved for boys ages 4 to 17 who have early-stage cerebral ALD.

The treatment must be done early, before serious brain changes appear on a magnetic resonance imaging test (MRI). Our doctors will perform regular MRI tests to see if your child is eligible. They’ll also make sure your child is healthy enough to receive the treatment.

What are the benefits of SKYSONA?

Skysona has several benefits. Treatment with Skysona gene therapy

• Uses your child’s own cells, so there's no need to find a matching donor.

• Can stop the progression of CALD before it causes permanent brain damage. In a recent study, 81% of treated boys were alive after 6 years – without major functional disabilities.

• Does not have a risk of graft versus host disease (when new donor cells attack the body), which is the most serious risk of donor stem cell transplant.

What are the risks and side effects of SKYSONA?

Skysona involves a type of chemotherapy to get your child’s body ready to receive the healthy cells. This treatment can cause:

• Hair loss, nausea and mouth sores

• A higher risk of infections for about 2 weeks

• Rare but serious (and usually temporary) liver problems

• A chance of developing a type of blood cancer called leukemia (about 10%)

• Future fertility issues, which we will help you understand and prepare for, through our fertility preservation services

Even with these risks, many families choose Skysona because the cerebral form of ALD is so serious and there are few other options.

What to expect with SKYSONA

Our cellular therapy and neurology experts will answer all your questions about Skysona – including how it compares to stem cell transplant. They’ll also review what you can expect before, during and after the treatment.

Our financial services team is also here to help you get insurance approval for Skysona. Plus, our social workers and pediatric psychologists can provide the important logistical or emotional support you may need. We’re here to support your whole family, not just your child.

When you’re considering Skysona, it’s important to know that the entire process typically takes about 4 to 5 months.

What to expect before SKYSONA

• Your child receives medication over 4 days to help stimulate blood cells to move from their bone marrow into their bloodstream.

• Our ALD experts collect your child’s blood stem cells over 2 to 3 days in the hospital and send them to a lab.

• The lab “fixes” the faulty gene. This takes a few months.

What to expect during SKYSONA

• About 10 days before receiving Skysona, your child will start chemotherapy in our hospital. Chemotherapy kills diseased cells to make room for your child’s body to receive new, healthy cells.

• Then, the new healthy stem cells are put back into their body through a tube inserted into a vein (IV). This takes about 20 minutes.

• Your child will remain in the hospital for 2 to 4 weeks, until the new cells are established in your child’s bone marrow and your child’s immune system is stronger.

What to expect after SKYSONA

• Your child must stay in or near the Dallas area for about 100 days. This allows us to keep a close watch on them. It also allows you to easily receive any post-therapy care from our expert team that your child may need. To help make things easier on your family, our social work team can help your family find lodging in the area.

• Your child will have checkups every 3 to 4 months to make sure the new cells are working and to make sure your child is not having any side effects.

• We’ll continue to help provide long-term, follow-up care for at least 10 years.

How do I prepare my child for SKYSONA?

Is my child allowed to eat or drink before Skysona Gene Therapy?

Yes, your child will be able to eat and drink before Skysona treatment although they may need to stop eating and drinking a few hours before chemotherapy treatment.

And because the chemotherapy part of treatment can weaken a child’s immune system, eating healthy, nutritious foods – and drinking a lot of filtered water – is very important. Our doctors and clinical dietitians will help you know what your child can eat and drink to keep them as healthy as possible during the entire treatment process.

Child Life services for Skysona Gene Therapy

Our caring Child Life specialists are available to help your child anytime during the treatment process. These specially trained professionals help kids of all ages cope with medical procedures by explaining things to them in language they can understand.

Child Life specialists can also help distract kids with fun toys or games when they need it, which can help them feel calmer and more relaxed in the hospital.

What questions should I ask my provider about SKYSONA?

• How many Skysona procedures have you and your team performed?

• Is gene therapy the best option for my child? What other options do they have?

• What experience does your team have managing Skysona-related chemotherapy complications?

• What is the long‑term outlook for children who receive Skysona gene therapy?

• Can you explain the short-term and lifelong risks, such as potential cancer?

• How is fertility affected, and what preservation options are available?

• What financial, housing, or insurance support is provided?

• Food and Drug Administration (FDA): Skysona

• Skysona.com treatment page

• Medline Plus: X-linked adrenoleukodystrophy

• Stop ALD Foundation

• ALD Alliance, formerly known as the Aidan Jack Seeger Foundation

• ALD Connect

• United Leukodystrophy Foundation