As a parent, you’ll do anything to ensure that your child has the safest, healthiest, and happiest future possible. Parents of children with cerebral palsy know this all too well—from various types of therapy, to medications for pain, seizures, and spasticity, to acupuncture and chiropractic care, these parents are eager to try anything that will help their children lead as normal a life as possible.

Although there’s no guarantee that cerebral palsy can be cured with gene therapy, we support treatments of any kind that are safe and aim to improve the lives of children living with CP. If you’ve been curious about gene therapy for your child, here’s what you need to know.

What Is Gene Therapy, and How Does it Work?

Our genes are what contain our DNA, which controls the form and functions of our bodies—which is why genes that don’t work properly can cause disease. Put simply, gene therapy is a technique that scientists use to modify a person’s genes to try and treat or prevent a medical condition or disease. This experimental technique can be used in a few different ways, including:

• Replacing a mutated (disease-causing) gene with a healthy copy of the gene
• Doing away with a disease-causing gene that isn’t functioning properly
• Introducing a new or modified gene into the body to help fight a disease or disorder

Currently, gene therapy is being used to study only diseases that have no other cure. According to the Mayo Clinic, it’s a promising technique for treatment of cancer, heart disease, cystic fibrosis, diabetes, AIDS, and hemophilia. In fact, clinical trials have demonstrated success in treating severe combined immune deficiency, hemophilia, blindness caused by retinitis pigmentosa, and even leukemia.

However, it’s critical to understand that it also comes with potential risk. Because a gene cannot be easily embedded into your cells, it has to be inserted using a carrier, also known as a vector. This technique introduces certain risks, including an adverse immune system reaction or possible infection or tumor. However, to ensure absolute safety, all US gene therapy clinical trials are closely monitored by both the Food and Drug Administration as well as the National Institutes of Health.

How Does Gene Therapy Relate to Cerebral Palsy?

As it stands, there is no definitive cure for cerebral palsy. CP is the most common childhood motor disability, with 1 in every 323 children being affected. Scientists are optimistic that continued research will lead to an eventual cure. Some of the current applications being tested include brain cell repair, interventions to help present brain damage, and stem cell research.

In fact, stem cell therapy has shown hope for a way to prevent or repair the damaged brain cells that cause cerebral palsy. Harvard Medical School conducted an experiment by injecting animals with stem cell implants, which resulted in the damaged brain cells being replaced with newer nerve cells that functioned properly. While much is still unknown, scientists continue their research, carrying the hope that gene therapy may be the key to unlocking an eventual cure.

Supportive Cerebral Palsy Family Lawyers Who Care About Your Family

If you believe your child’s cerebral palsy was caused by medical neglect or error, we’re here to offer the educational and legal resources you need to help your child seek an improved quality of life. Contact us for a free, no-obligation consultation to discuss the next steps in your case, so you and your family can start down the road to the brightest future possible.

Reviewed by:
Trish Fletcher, MS, BSN, CRNP, NNP-BC, ALNC
Neonatal Nurse Practitioner | Birth Injury Legal Nurse Consultant

Tricia is a dedicated, focused, Birth Injury Legal Nurse Consultant and Neonatal Nurse Practitioner with more than 25 years of experience. Her strong clinical and critical thinking skills, paired with expertise caring for neonates in a Level III Neonatal Intensive Care Unit (NICU), ensures meticulous medical records review. READ FULL BIO

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