---
title: "Gene Therapy for Fatal Childhood Illness Approved in Europe, And It Has A 100% Survival Rate"
description: "It was quite the moment for scientific research, as the world's first child gene therapy has been approved by the European Commission. GSK will offer treatment for ADA-SCID, a disease known to be fatal at an early age."
date: "2016-06-28"
modified: "2016-06-29"
authors:
  - name: "Neil C. Bhavsar"
    link: "https://futurism.com/authors/neilbhavsar"
url: "https://futurism.com/gene-therapy-with-100-survival-rate-for-childhood-fatal-illness-approved-in-europe"
categories:
  - "Science & Energy"
tags:
  - "approved"
  - "gene therapy"
  - "genetics"
  - "scid"
---

# Gene Therapy for Fatal Childhood Illness Approved in Europe, And It Has A 100% Survival Rate

![](<https://futurism.com/wp-content/uploads/2016/06/A-GlaxoSmithKline.jpg>)
*\<em\>Image: GlaxoSmithKline\</em\>*

## The Fatal Illness

It's only been a short [three months](<https://futurism.com/european-committee-approves-worlds-first-gene-therapy-children>) since talks had erupted about the potential approval of a certain gene therapy. But those three months of deliberations by the European Commission have led to a decision that will grant [days, months, years and maybe even decades ](<http://www.bloodjournal.org/content/early/2016/04/29/blood-2016-01-688226?sso-checked=true>)to children whose parents have felt *lucky* to have spent any time with them at *all*.

The condition at the heart of the discussion is ADA-Severe Combined Immunodeficiency (ADA-SCID), a rare disorder that is found in 15 newborns each year in Europe. It is characterized by the lack of the Adenosine Deaminase (ADA) protein, which leads to a heavily deficient immune system, as lymphocytes, a type of white blood cell, need the protein for production. Without a functioning immune system, children with ADA-SCID aren't expected to live long after birth--that is until now.

## Taking the Fatal out of Fatal Illness

That is where *Strimvelis,* GlaxoSmithKline's (GSK) newest gene therapy technique comes into play. By utilizing the patient's own bone marrow, a normal copy of the ADA gene is inserted into the cells and then the gene-corrected cells are re-introduced to the patient intravenously.

The confidence in this technique stems from the fact that there was a 100% survival rate in trials that followed 18 treated patients for up to 3 to 13 years. That isn't to say there aren't issues. All patients also suffered an adverse effect from the trial, but most resulted with a complication that occurs regularly throughout childhood. To that effect, the European Commission felt safe approving the British firm's treatment for commercial use.

> This is the start of a new chapter in the treatment of rare genetic diseases...

Upon hearing this, the head of GSK's Rare Disease Unit, [Martin Andrews](<http://www.gsk.com/en-gb/media/press-releases/2016/strimvelistm-receives-european-marketing-authorisation-to-treat-very-rare-disease-ada-scid/>) says that: “Today’s approval is the result of many years’ work with our collaborators in Milan and is the next step towards bringing life-changing treatment to patients with ADA-SCID and their families. This is the start of a new chapter in the treatment of rare genetic diseases and we hope that this therapeutic approach could also be used to help patients with other rare diseases in the future.”

A new chapter indeed, one featuring the world of personalized medicine.

A brief video following the story of a boy diagnosed with SCID:

https://www.youtube.com/watch?v=QPtOt2l6-1A

## Author
Science today, science tomorrow, science forever.

### Author social links  
[Facebook](<https://www.facebook.com/Neilogen>)  
[LinkedIn](<https://www.linkedin.com/in/neil-c-bhavsar-033225118>)  
[Twitter](<https://x.com/Neilcogen>)