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European Child Receives Strimvelis Gene-Therapy Treatment

Posted on: 3 May 2017, source: Rare Disease Report
Before March, only 1 patient had ever been treated via commercial gene therapy. GlaxoSmithKline (GSK) doubled that total with their announcement this week. In 2015, a European patient with familial chylomicromenia received Glybera to restore their LPL enzyme levels. On Tuesday, GSK announced that it treated its first patient in March, almost a full calendar year after their drug, Strimvelis, was approved for sale. Strimvelis is assumed to provide an outright cure for Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency (ADA-SCID), a rare disorder caused by the absence of the essential protein adenosine deaminase (ADA). ADA is required to produce lymphocytes. It leaves babies without a fully-functioning immune system and extremely vulnerable to infections.


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The disorder affects an estimated 15 children in Europe each year, and GSK reportedly never expected Strimvelis to be a big seller. In August, the company released a statement noting their “money-back guarantee” – that if their drug fails to cure the rare disorder frequently referred to as “bubble boy disease,” the patient will be refunded in full.

The drug can revise a patient’s genetic makeup, and while gene therapy has been widely explored in studies, its commercial potential has gone unproven due to difficulty organizing cross-border reimbursement. Currently, Strimvelis is offered at a single center in Milan, Italy. Its lone location for availability can be partly to blame for the delay in commercialization, per GSKs project leader for Strimvelis, Jonathan Appleby.

As an alternative to having patients travel to Italy for treatment, Appleby notes the company is testing ways to cryogenically freeze and transport patients’ cells. The hope is that this will assist in several more patients across Europe being treated with Strimvelis in 2017.

Adenosine deaminase (ADA) deficiency is an genetic disorder that damages the immune system with the effect being severe combined immunodeficiency (SCID). Patients with SCID lack almost all immune protection from bacteria, viruses, and fungi. The severely immunocompromised patients must lead very protective lives, as exemplified in movies such as the Boy in the Plastic Bubble.

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Posted on: 3 May 2017, source: Rare Disease Report
Before March, only 1 patient had ever been treated via commercial gene therapy. GlaxoSmithKline (GSK) doubled that total with their announcement this week. In 2015, a European patient with familial chylomicromenia received Glybera to restore their LPL enzyme levels. On Tuesday, GSK announced that it treated its first patient in March, almost a full calendar year after their drug, Strimvelis, was approved for sale. Strimvelis is assumed to provide an outright cure for Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency (ADA-SCID), a rare disorder caused by the absence of the essential protein adenosine deaminase (ADA). ADA is required to produce lymphocytes. It leaves babies without a fully-functioning immune system and extremely vulnerable to infections.
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