You Want to Put What in Me? New Treatment for XSCID

I have two sisters, each with the remarkable talent to walk into a thrift store with only a few bucks and walk out with a brand new wardrobe. Growing up in Oklahoma, a lot of what they left with was made of denim. “Jean therapy,” they called it.

Well, Harriet and Beth, your jean therapy ain’t got nothin’ on this:

This isn’t exactly what we mean either when we talk about “gene therapy.” Source:

Researchers at the National Institute of Allergy and Infectious Diseases, in partnership with St. Jude Children’s Research Hospital, have been studying the effectiveness of gene therapy in children and adolescents with X-linked severe combined immunodeficiency (XSCID).

Say what?

XSCID affects the development of immune cells (think “white blood cells”), so those who have the condition also have an incredibly hard time fighting off infections.

As a result, they experience chronic illness and sometimes early death. The gold standard in treatment has so far been bone marrow transplants. Basically, doctors wipe out the defective immune system of the person with XSCID and then replace it with a normal immune system derived from the bone marrow of a donor.

Bone marrow transplants have been most effective when the donor is a sibling…but sometimes that’s not always an option. In those instances, donor bone marrow may come from a parent or someone who’s unrelated. Unfortunately those transplants aren’t always effective. Enter Lentiviral Gene Transfer.

Now, in order to understand this we first must ask ourselves, “What the heck is a lentivirus?”

robert downey jr question excellent question iron man

A lentivirus, as its name suggests, is a virus–a microorganism adept at infiltrating the human immune system. In this case, however, the virus has been modified to actually carry corrected genes.

Similar approaches to gene therapy have been used effectively before, but this is the first time it’s being looked at to treat people living with XSCID.

Once the virus infiltrates the immune system of someone with XSCID, it actually helps in producing normal, healthy immune cells. At least, that’s the goal and what researchers have been studying since October 2010 when this clinical trial kicked off.

So far, there’s promise.

Early results (and I’ve got to stress that it is still early) have showed “substantial improvements in immunity […] with one patient continuing to improve three years after therapy.”

A crucial component of the therapy seems to be early intervention, before organ damage associated with XSCID becomes irreversible.

The study of gene therapy to treat children and adolescents with XSCID isn’t scheduled to be completed until the end of 2025, which admittedly is a long way off. But researchers actually hope to have all their data collected by the end of 2018, so updates should be trickling in over the next few years. You can be sure we’ll keep you updated.

James Ernest Cassady

James Ernest Cassady

Though "Ernest" is a family name that's been passed down for generations, James truly earned his middle moniker when, at the age of five, he told his mother that "laughing is stupid unless EVERYBODY is happy." Since then, the serious little bastard has been on a mission to highlight the world's shortcomings (and hopefully correct them). In addition to his volunteer work at hospitals and animal shelters, James also enjoys documentaries and the work of William Faulkner. He is originally from Oklahoma.

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