GSK Paved Personalized Gene Therapy – Strimvelis Path for ‘Bubble Boy’ Syndrome
GlaxosmithKline’s (GSK)‘s Strimvelis (‘Bubble Boy’ gene therapy) after Committee for Medicinal Products for Human Use (CHMP) recommendation is on its way to become the first gene therapy to treat this extremely rare disease.
GSK announcement regarding the marketing approval of Strimvelis by Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) along with the Committee for Advanced Therapies (CAT) shown a sudden leap of advancement for the treatment of a very rare genetic disorder Severe Combined Immunodeficiency (SCID) or ‘Bubble Boy’ Syndrome’.
‘Bubble Boy’ Syndrome is a congenital disorder caused due to the impaired adaptive immune system due to numerous genetic mutations. It is known to be the most severe form of primary immunodeficiency. Due to the highly compromised or almost lack of immune system these children become prone to almost all types of fatal diseases and infections (bacterial, viral, fungal). Babies are prone to die within 1 year unless undergone successful hematopoietic stem cell transplantation.
Strimvelis (‘Bubble Boy’ gene therapy) developed and reached this stage as a result of partnership between GSK and its Italian research partners SanRaffaele Telethon Institute for Gene Therapy and the Milanese medical biotech company MolMed. For 13 years it remained in its struggling period of trials, which after CHMP recommendation is on its way to become the first gene therapy to treat this extremely rare disease.
Strimvelis is an ex-vivo gene therapy for the treatment of adenosine deaminase deficiency-SCID (ADA-SCID). In this form of SCID there is a lack of enzyme adenosine deminase (ADA) in the victim which results in accumulation of those substrates in the cell which are responsible for this enzyme. As a result of the toxic effects of these unused substrates immature lymphoid cells of the immune system fails to mature resulting in the absence of immunity.
In the gene therapy treatment Strimvelis functions by utilizing a virus as a vector to insert copies of ADA gene into the faulty hematopoietic stem cells of the patient extracted from their bone marrow. After this the stem cells are restored back into the patient making the patient capable of generating its own gene which is responsible for the production of ADA and hopefully evoking the patient’s immune system if shown immune compatibility.
We can say GSK has shown a new dawn to the bubble boy syndrome patients by opening a personalized treatment option for them and for further development of a new gene therapies platform for personalized medicine development. Currently MolMed is the only approved site for the manufacturing of these type of personalized medicine making it the sole option for the treatment of ‘Bubble Boy Syndrome’. The company is hopeful that with the CHMP recommendation they can expect the final European commission approval in three months then leading for the goal of FDA approval.
Patrick Vallance, President, R&D, GSK said, “We welcome this opinion from the CHMP which is an important step towards making Strimvelis available to the children living with this incredibly rare and fatal condition. Going forward, we hope to apply this gene therapy platform technology across other diseases, enabling many more patients to benefit from this innovative treatment approach.”
‘Bubble Boy’ syndrome have been interestingly featured in a 2001 release American Drama/Teen movie Bubble Boy. In the movie Jimmy Livingston (played by Jake Gyllenhaal) sufferer of ‘Bubble Boy’ syndrome was forced to live in his sterile dome of the bedroom. His life took some interesting and adventurous turns when a girl named chloe (played by Marley Shelton) moved in his neighborhood. (Courtesy: Movieclips Trailer Vault)
Featured image credit: Ultrasound Baby. ZF foto © Hriana (Stock Photo ID: 104498354)