Alex’s parents have moved heaven and earth to give their son a chance to beat the rare life-limiting condition he was born with.
We will most likely lose our beloved little Alex while he is still young. The typical life expectancy for those with Hunter Syndrome is between 10 and 20 years. He is already 4 and a half……I can’t stomach the thought of how quickly his life is going by…of how little he has left.Crystal, Alex’s Mum
What is Hunter’s Syndrome?
Hunter’s Syndrome or mucopolysaccharidosis type II (MPS II), is a rare genetic condition in which large sugar molecules called glycosaminoglycans (AKA GAGs, or mucopolysaccharides) build up in body tissues. Hunter syndrome causes abnormalities in many organs, including the skeleton, heart, and respiratory system. In severe cases, this leads to death during the teenage years.
Is there a cure for Hunter’s Syndrome?
Until recently, there was no effective therapy for MPS II. Because of this, palliative care was used.
But recent advances have led to medications which can improve survival and well-being in people with MPS II. These include enzyme replacement therapy, which has been proven to improve many signs and symptoms of MPS II. However, this treatment is not expected to lead to cognitive improvement in patients with severe central nervous system symptoms.
Cord blood stem cell transplantation
Bone marrow transplantation and hematopoietic stem cell transplantation (HSCT) have also been trialled for the treatment of MPS II. Cord blood stem cell transplantation has shown particular promise in the treatment of other MPS disorders, leading doctors and parents of young patients to describe it as “our first choice” of therapy.
Doctors decide cord blood is Alex’s best option
Alexander’s parents and doctors have decided that a stem cell transplant is the best option. If successful, a cord blood transplant will allow Alex to produce the missing enzyme on his own.
“He would no longer need to get weekly enzyme replacement therapy.” Writes mum, Crystal, “Not only would that save him from having to be in the hospital one day, all day, each and every week. But it would allow his body a steady level of enzyme to break down the mucopolysaccharides 24/7 which the synthetic enzyme can’t do (each day after enzyme replacement therapy there is less and less enzyme in the body). This could mean a higher quality of life for Alex, as well as a longer life!!”
Help Alex enjoy a long healthy life
Meeting the cost of travel and recuperation is a challenge for Alex’s family, who live a long distance away from his therapy centre in Minnesota. Alex needs his parents with him, so the family are raising money via his Go Fund Me page. You can help support Alex here.
In her most recent update, Crystal writes, “Thank you, everyone, for your love and support! We have been here in Minnesota for almost two weeks. Alex has had a large number of appointments including an overnight hospital stay last week. He has two new accessories, a tube and central line. Both required as prep for the transplant. He is doing well overall and handling everything like the rockstar he is.”
Store your child’s cord blood for treatments like Alex’s
Cord blood isn’t just a life-saver for children suffering from Hunter’s Syndrome. The cells that are left in your baby’s cord blood after birth can treat leukaemia, lymphoma, diabetes and other conditions of the blood and immune system.
Biovault Family are proud to be the UK’s ethical cord blood. Our scientists are contributing to our mission: stem cell therapies and technologies that make next-generation medicine available to every family.
- Wraith JE, Scarpa M, Beck M, et al. (March 2008). “Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy”. Eur. J. Pediatr. 167 (3): 267–77. doi:10.1007/s00431-007-0635-4. PMC2234442. PMID18038146.
- Help Alex get a cord blood transplant by Alexander’s Army
BSc (Hons) Microbiology
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.