Gene therapy and its impact on rare and fatal childhood diseases

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Gene therapy and its impact on rare and fatal childhood diseases

Dr Paul Zollinger-Read, the Medical Director of Medical Imaging Partnership and Prime Health shares his thoughts on the latest news coming out of the US of research trials using gene therapy for children suffering with fatal diseases.

Staying on the subject of new developments in my final article for May, I am going to briefly touch on an article in last week’s New Scientist concerning gene therapy. The article reports on several genetic conditions in children that used to be fatal and how gene therapy is now starting to give hope to these challenging conditions.

How can gene therapy help?

It reports on how a team at the University of California, Los Angeles, Kohn and his colleagues, are testing out a gene therapy for a rare condition, leukocyte adhesion deficiency type-1 (LAD-1).

This is generally a fatal condition leading to death within two- years. This is because a certain type of immune cell called leukocytes cannot travel around the body to where they are needed, for example, at the site of an infection or injury. This leads to what would typically be simple skin or lung infections becoming chronic. The only current treatment is a stem cell transplant, which is not without considerable risk.

The team are trialling replacing the defective genes with normal versions. These are into the child’s DNA using a virus that has been modified not to cause harm, just to work as a transport vehicle for the new DNA.

Who has received this treatment so far?

Nine children have received gene therapy since 2021 and so far, none of them has developed chronic skin or lung infections; they are all leading normal lives. It is too early to celebrate yet as many years of study need to be done to ensure there are no unintended consequences; however, this looks pretty miraculous. And this is just one of many such approaches. There’s no doubt that gene therapy is starting to significantly impact these rare and fatal childhood diseases.