Stem cell therapy and severe combined immunodeficiency (SCID)

SCID occurs in around 1 in 50,000 – 100,000 births [1]

The most common type of SCID only occurs in males (X-linked) [1]

Females can carry the X-linked SCID gene and have a 50% chance of passing it onto their child [2]

If left untreated, children with SCID only live for a year or two [1]

There are at least 13 genetic defects that can cause SCID [2]

Stem cell transplants performed no later than the age of 3 months have up to 95% success rate [3]

SCID occurs in around
1 in
50k – 100K
births

What is SCID?

Severe combined immunodeficiency (SCID) is a group of rare, inherited disorders that target the immune system. SCID patients are defenceless against bacteria and viral infections that would usually be harmless to healthy people, and this can prove life-threatening [4].

SCID is an inherited condition, caused by the malfunctioning of a protein that’s responsible for the development and function of the immune system. This protein deficiency can affect many different genes, causing different types of SCID. Symptoms usually occur within the first three to six months of a baby’s life, in the form of frequent infections, cough and colds, chronic diarrhea and skin rashes that go on for longer than expected.

If left untreated, germs in our surroundings can cause life-threatening illnesses in children with SCID, such as pneumonia and even liver disease. Therefore, it’s very important to recognize the signs as early as possible to receive a diagnosis. This can be achieved with the help of a blood test and cells count.

Stem cell therapy research

The only treatment that offers a permanent cure for SCID is a stem cell transplant. Blood-forming hematopoietic stem cells (HSCs) found in cord blood and bone marrow are infused into the patient’s bloodstream. From here they will become healthy white blood cells, red blood cells and platelets, to effectively replenish immune function.

The child’s recovery rate depends on their overall health at the time of treatment, the child’s age and the closeness of stem cell match. Autologous stem cells (taken from the patient) are an 100% DNA match for them, providing a higher success rate in transplant.

Studies have proven the effectiveness of HSCs treatment, particularly between matched siblings, for immunodeficiency diseases since 1968. Today, it’s typical for infants treated within three to four months of age to have a 90% or higher survival rate after stem cell therapy.[5]

While other treatments are available to SCID patients, blood stem cell transplant remains the primary life-saving route. HSCs are especially effective if administered soon after diagnosis or prior to infection.[6]

Our stem cell releases for SCID

Future Health Biobank released our first cord blood sample for treating SCID in 2014. The sample was for the donor’s 8 weeks old sibling, who was born with severe combined immunodeficiency. The stem cell transplant was successful and the boy’s condition has improved.

Many parents and healthcare professionals approach us seeking information for children that are diagnosed with SCID. Our guidance for therapypage provides useful links to the latest developments in stem cell therapies.

References

1. https://www.seattlecca.org/diseases/severe-combined-immune-deficiency-scid/scid-facts

2. https://primaryimmune.org/about-primary-immunodeficiencies/specific-disease-types/severe-combined-immune-deficiency-and-combined-immune-deficiency

3. http://www.childrenshospital.org/conditions-and-treatments/conditions/s/severe-combined-immunodeficiency/diagnosis-and-treatment

4. https://www.gosh.nhs.uk/conditions-and-treatments/conditions-we-treat/severe-combined-immunodeficiency-scid

5. https://primaryimmune.org/treatment-information/stem-cell-and-gene-therapy

6. https://www.ncbi.nlm.nih.gov

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