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Stem cell therapy cures two-year-old of HLH


In a story reported internationally as a medical ‘breakthrough’, a pioneering stem cell treatment has”cured” a toddler after he was given weeks to live.

Alex was born with haemophagocytic lymphohistiocytosis (HLH), a rare autoimmune disease in which the body reacts “inappropriately to a trigger”

He was being treated with an experimental drug at Great Ormond Street Hospital but his parents Paolo and Cristina were told it may become ineffective after five weeks.

Stem cell collection

Alex’s parents, who are originally from Italy but met and live in London, had initially set up a desperate worldwide appeal for a compatible bone marrow donor.

They wrote on Facebook that their son had “fought against an endless series of complications to still be here”.

His parents explained that the condition affects “only 0.002 per cent of children” and that “50 per cent of children affected by this disease die within the first month”.

They decided to take him to the Vatican-run hospital, where doctors used specially treated stem cells from his dad Paolo’s blood to replace Alex’s affected white blood cells.

Hematopoietic stem cell (HSC) transplant is the only cure for HLH. HSCs can be collected from bone marrow, peripheral blood and from umbilical cord blood.

Learn more about cord blood banking

Children with HLH have over-activated white blood cells, known as T-cells, which cause severe inflammation and damage to tissues including the liver, spleen and bone marrow.

‘Ready to resume life’

Franco Locatelli, head of the department that developed the treatment, told the Times: “We can say that Alex is completely cured and that his immune system is now healthy.

“The donor’s cells have completely replaced the sick ones, which would not have allowed Alex to survive. It’s an enormous achievement for the type of transplant.”

We can say that Alex is completely cured and that his immune system is now healthy

Dr Franco Locatelli, Bambino Gesu Children’s Hospital In Rome

He added that Alex, who is the seventh child to benefit from the treatment at the hospital, was now “ready to resume the life that all children of his age should have”.

He is now the seventh child with HLH to benefit from the procedure at the Italian hospital.

What is haemophagocytic lymphohistiocytosis (HLH)?

Haemophagocytic lymphohistiocytosis (HLH) is a rare immune disorder where the body reacts inappropriately to a ‘trigger’, usually an infection.

HLH is a rare disease and it can be difficult to diagnose because many of the symptoms can mimic severe infection or other conditions.

They include a skin rash, raised temperature and swollen liver, spleen and lymph glands. There may be anaemia, infection or bruising and bleeding. If the brain is affected, a child may show symptoms such as seizures, ataxia (wobbliness) or drowsiness.

It’s usually diagnosed through blood tests but more specialist tests will check liver function and check for signs and causes of infection.

Family stem cell storage

You can store your baby’s umbilical cord blood and cord tissue after they are born. The procedure is non-controversial and compatible with a natural third stage and delayed cord clamping.

Sources: 

PID UK, GOSH and Great North Children’s Hospital

https://www.bbmt.org/article/S1083-8791(15)01443-3/abstract

https://www.thetimes.co.uk/article/boy-2-cured-by-stem-cell-breakthrough-d69kbsqst

https://www.thesun.co.uk/fabulous/8913094/boy-cured-rare-disease-stem-cell-treatment/

https://www.bbc.co.uk/news/world-europe-45976480

stem cell preservation

BSc (Hons) Microbiology

Chief Executive Officer | Biovault Family

Biovault Family CEO, Kate Sneddon, joined Biovault in July 2009 and became Chief Executive Officer in 2016. As health industry professional her experience includes working as a microbiologist and leader at GSK for over 10 years. Her expertise in cord blood banking has been recognised in her awards, features in Parliamentary Review and Parents Guide to Cord Blood, as well as contributions to research with UCL and others.


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