Children with ‘bubble-boy disease’ May Soon Have a Cure, Glaxo’s Potential Treat receives Positive Response
Patients of ‘bubble-boy disease’ may soon have a cure as Glaxo’s potential cure has cleared one more hurdle after a European Union regulatory panel stood into its support. If approved, the treatment will help children born with the rare immune-system condition.
On Friday, an advisory committee of the European Medicines Agency showed its support for the cure, which involves introducing a new gene into the sufferer’s stem cells. Positive attitude of the committee has made path easy for the treatment. There are high chances that it will receive approval in coming months.
The new therapy will be beneficial for kids with Adenosine deaminase deficiency, also called ADA-SCID. It is a disease that targets children and takes their lives if left untreated.
Current method to treat the condition is bone-marrow transplant. It is the best available option, but chances of success purely depends on how well matched the donor is. “For the one in four babies who have a well matched family member, a transplant can be an effective cure. But for the rest, success rates can be as low as 50%”, as per Professor Bobby Gaspar from London’s Great Ormond Street Hospital.
The new cure is called Strimvelis. A team of doctors and health experts from Milan invented it. They claimed that the therapy has been used to treat more than 20 children in last more than one decade, and all of them are still alive. Most of the treated patients do not require any further treatment, the team claimed.
If approved, Strimvelis will become the second to be sold in European region. Before this, Glybera of UniQure NV has been allowed to treat a rare genetic condition where a human body cannot break down fat molecules.