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New gene therapy helps rebuild immunity in ADA SCID infants allowing them to avoid isolation and repeated treatments while leading healthier lives.
Highlights:
Children born with ADA SCID (Adenosine Deaminase Severe Combined Immunodeficiency) have a genetic defect that prevents their bodies from producing adenosine deaminase, the key enzyme needed for immune cell development. Without it they lack working T cells and B cells. Even minor infections are life threatening (1✔ ✔Trusted Source- 62 ADA SCID infants treated in the largest long-term study to date
- 95 percent developed durable immune reconstitution
- Most stopped requiring continued enzyme or antibody therapy within months
Gene therapy delivers lasting immune protection in children with rare disorder
Go to source). Standard treatment options such as bone marrow transplantation or enzyme replacement therapy provide temporary support but do not always lead to long term immune protection.
Bubble Baby Disease | Immune Deficiency Disorder | Severe Immunodeficiency in Children
Bubble baby disease, or Severe Combined Immunodeficiency (SCID) is a genetic disorder in which there is a severe deficiency of lymphocytes, a type of white blood cells needed for normal functioning of the immune system.
Bubble baby disease, or Severe Combined Immunodeficiency (SCID) is a genetic disorder in which there is a severe deficiency of lymphocytes, a type of white blood cells needed for normal functioning of the immune system.
TOP INSIGHT
Did You Know?
#BubbleBabies once had to live in complete isolation to survive. Today gene corrected #stemcells can help them grow a fully functioning #immunesystem.
#SCID #GeneTherapy #ChildHealth #Medindia
How the Gene Therapy Works
Gene therapy offers a transformative alternative: Doctors collect the infant’s own blood forming stem cells. A healthy ADA gene is added to these cells inside the laboratory using a lentiviral vector. The corrected cells are then infused back into the child. Over time the cells mature and create new working immune cells that can recognize and fight infections (2✔ ✔Trusted SourceLong-Term Safety and Efficacy of Gene Therapy for Adenosine Deaminase Deficiency
Go to source).
Why Is Screening Newborns for Bubble-Baby Disease Important?
Neonatal screening for bubble baby or immunodeficiency disease enhances the survival rate of affected children.
Neonatal screening for bubble baby or immunodeficiency disease enhances the survival rate of affected children.
Strong Long Term Study Results
A total of 62 children with ADA SCID received the therapy between 2012 and 2019. They were followed up for several years with a median follow up time of 7.5 years and a range of 5 to more than 11 years. Out of these, 59 children developed strong and lasting immunity and remain stable without needing repeat treatment (3✔ ✔Trusted SourceLong-term outcomes of lentiviral gene therapy for ADA-deficient severe combined immunodeficiency
Go to source). Most children were able to stop enzyme and antibody replacement therapy within a few months to years after treatment. They also developed protective immune responses to routine childhood vaccines like tetanus and pneumococcal vaccines. No major long term safety concerns have been reported.
More than half of the children received cryopreserved stem cells making this therapy easier to deliver even in places far from specialized centers.
Gene Therapy Brings Hope for Bubble Boy Disease Treatment
Gene therapy by GlaxoSmithKline offers hope for children with bubble boy disease with Phase I and II approved by EU regulation board.
Gene therapy by GlaxoSmithKline offers hope for children with bubble boy disease with Phase I and II approved by EU regulation board.
From Bubble Life to Normal Childhood
Babies with SCID were once known as Bubble Babies because they had to stay in clean room environments to avoid infections. This gene therapy has changed their future. Many treated children are now attending regular school playing outdoors and enjoying normal childhood activities.
GSK’s Gene Therapy, Strimvelis, for “Bubble Baby Disease” Gets NICE’s Approval
The National Institute for Health and Care Excellence has approved GSK’s gene therapy, Strimvelis, as an option to treat a rare immune deficiency condition.
The National Institute for Health and Care Excellence has approved GSK’s gene therapy, Strimvelis, as an option to treat a rare immune deficiency condition.
Takeaway
Gene corrected stem cell therapy represents a major scientific breakthrough for ADA SCID. When children are diagnosed early and treated quickly they can develop their own strong immunity and live long healthy lives.References:
- Gene therapy delivers lasting immune protection in children with rare disorder - (https://newsroom.ucla.edu/releases/gene-therapy-delivers-lasting-immune-protection-children-rare-disorder)
- Long-Term Safety and Efficacy of Gene Therapy for Adenosine Deaminase Deficiency - (https://pubmed.ncbi.nlm.nih.gov/41092330)
- Long-term outcomes of lentiviral gene therapy for ADA-deficient severe combined immunodeficiency - (https://www.contemporarypediatrics.com/view/long-term-outcomes-of-lentiviral-gene-therapy-for-ada-deficient-severe-combined-immunodeficiency)
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