Protecting your child: What are the key diseases that stem cell storage could treat?

 In Our Transplants, Stem Cell News

As a parent, it’s only natural to want to do all you can to protect your child, especially when it comes to their health and wellbeing. Collecting and storing your newborn’s stem cells for possible use against future illnesses is one simple step that could have far-reaching benefits. Cord blood banking – the storage of blood from the umbilical cord at birth – is becoming an increasingly popular way to safeguard your children’s health for life.

Umbilical cord blood is collected at birth in a simple procedure that involves no pain whatsoever for mother or baby. The cord blood is tested and then frozen. When it’s needed, stem cells from the cord blood are transplanted into the patient’s bloodstream and set about replacing damaged cells and repairing tissue. The first such transplant in the Arab world was performed in 1998 and this form of therapy is now part of the successful treatment of more than 80 different diseases.

The first cord blood transplant in the Arab world was performed in 1998 and this form of therapy is now part of the successful treatment of more than 80 different diseases.

The known benefits of stem cell storage

So let’s look at some of the key benefits of stem cell storage, and what it could mean for your family.

1. Blood disorders: Umbilical cord blood containing potent ‘ hematopoietic stem cells’ (HSCs), which are responsible for maintaining blood production, is used in transplants to replace damaged, diseased blood cells with healthy new ones. This form of application is particularly effective at helping to treat inherited blood disorders such as sickle cell anaemia, severe aplastic anaemia and thalassemia.

It is estimated that there are over two million people with sickle cell disease worldwide, mostly in Africa, the Middle East and India. The inherited condition is caused by an abnormal form of haemoglobin, which causes the red blood cells to become sickle-shaped. The abnormal cells cannot move freely through the blood vessels and cause blockages, which result in severe pain and chronic progressive organ damage. Both parents may carry the abnormal gene but remain healthy themselves. The anaemia that results from the condition is not present at birth but develops in the first six months.

During a cord blood stem cell transplant, the patient first undergoes chemotherapy to wipe out the damaged blood cells. This is followed by a cord blood transfusion with the new, healthy stem cells, which will produce normal red blood cells. According to a recent study, close to 90% of children receiving a stem cell transplant from a matched sibling were cured of sickle cell disease.

2. Metabolic disorders: Cord blood transplants are also used to treat inherited metabolic disorders – genetic conditions where abnormal chemical reactions in the body alter the metabolic process. Most people with inherited metabolic disorders have a defective gene that results in enzyme deficiency, which can cause a variety of different symptoms.

Stem cell therapy has been successfully used to treat metabolic disorders such as Hurler’s Syndrome. This inherited disease occurs when there is an inability to break down long chains of sugar molecules called glycosaminoglycans (GAGs), which are found throughout the body. Without the enzyme, there is a build-up of GAGs, eventually leading to severe organ damage.

For children who inherit this condition, it is best to treat it as soon as possible because the condition worsens over time. Again chemotherapy is used to remove all the damaged cells and then the healthy stem cells are transfused into the bloodstream, where they form healthy new blood cells, which have the enzyme needed to break down GAGs and stop further damage to the body. Cord blood stem cell transplants have also proven effective for rare metabolic disorders such as Krabbe disease and Sanfilippo syndrome, which could otherwise be fatal for infants.

3. Immune disorders: The immune system is the body’s method of self-defence against harmful substances such as bacteria, viruses, and toxins. However, in some cases the immune system cannot distinguish between healthy, harmless tissue and harmful antigens. As a result, it attacks and destroys normal tissue, resulting in autoimmune disorders such as type 1 diabetes, Crohn’s disease, lupus, multiple sclerosis, and rheumatoid arthritis.

Undergoing a transplant of healthy stem cells from the umbilical cord can help restore a faulty immune system. These stem cells are not only less likely to provoke an immune response but also contain a greater number of the regulatory T cells – a type of white blood cell that helps to maintain the balance of the immune system.

Undergoing a transplant of healthy stem cells from the umbilical cord can help restore a faulty immune system.

Immediately before a transplant of healthy cord blood stem cells takes place, the patient’s own white blood cells, responsible for the immune response, are destroyed using chemotherapy. The new stem cells are then introduced into the bloodstream to repair the immune system.

In the case of type 1 diabetes, the pancreas can’t effectively produce insulin, the hormone required to convert food into energy, because the immune system attacks the insulin-producing cells. The incidence of type 1 diabetes is increasing worldwide, nowhere more so than in the Middle East. According to the 2013 Diabetes Atlas, the incidence of type 1 diabetes per 100,000 individuals per year was 11·4 in Qatar and 22·3 in Kuwait.

Several successful trials have found that cord blood stem cell therapy has helped patients produce their own insulin, which has significantly reduced the need for insulin from outside sources.

4. Cancer: Stem cells derived from cord blood are now a viable treatment for people diagnosed with certain types of blood cancer, such as leukaemia, lymphoma (cancer of the lymphatic system), myeloma and myelodysplastic syndromes (MDS), also called pre-leukaemia.

When the body develops leukaemia, one of the most common forms of cancer in infants, abnormal white cells in the blood grow too quickly, pushing the entire system out of balance, weakening and completely blocking the immune system. The cancerous white blood cells can spread into the lymphatic system and from there move throughout the body.

A 2010 study published in the Asia Pacific Organisation for Cancer Prevention revealed that cancer is the third leading cause of death in the UAE. Up to 60 new cases of cancer in children are reported in Dubai each year, of which the majority are leukaemia cases.

When using cord blood stem cell transplantation, chemotherapy is used to destroy the leukaemia cells before giving a transfusion of healthy cord blood stem cells to replace the diseased blood cells. The stem cells are injected directly into the veins and make their way to rebuild the depleted bone marrow. 

 

Future stem cell therapies

Ongoing medical research continues to reveal exciting new ways in which cord blood stem cell therapy could have a positive effect. Let’s look at some of the main areas currently being studied. 

Regenerative medicine

Research is currently looking at whether stem cells, which can transform into other types of cells found in tissue, blood and organs, can further the cause of regenerative medicine – where cells regenerate or facilitate the repair of cells damaged by disease, genetics, injury or ageing. One example is the bone-thinning disease osteoporosis, with which mineral abnormalities cause bone loss to occur faster than new bone growth, leading to a thinning of the bones, which become porous, brittle and prone to fracture.

According to the US National Osteoporosis Foundation, 10 million Americans have the disease and 44 million more have low bone density, putting them at risk. Studies from the Third International Conference on Vitamin D Deficiency also showed that 78% of the UAE population has this deficiency, one of the known causes of osteoporosis, and the condition is highly prevalent among Emirati women.

Stem cell therapy for osteoporosis could reduce susceptibility to fractures and boost lost mineral density by increasing the number of effective bone-forming cells.

Neurological conditions

Research is now moving on to the possible use of cord blood stem cells in treating neurological disorders, such as autism and cerebral palsy, where damage to the brain results in a lack of full control or physical movement. Some studies suggest that the mesenchymal stem cells (MSCs) found in the umbilical cord blood release substances that promote natural brain repair. For example, in a 2017 trial carried out by Duke Research University, 75% of autistic children treated with cord blood stem cells showed an improvement in their symptoms in the first six months.

Research is now moving on to the possible use of cord blood stem cells in treating neurological disorders, such as autism and cerebral palsy, where damage to the brain results in a lack of full control or physical movement.

Cardiovascular

Stem cells have shown a promising role in preclinical studies for treating ischemic stroke, where a blood clot blocks the flow of blood and oxygen to the brain. Other conditions where the use of stem cell therapy is currently being researched include treatment for congenital heart disease, a range of birth defects that affect the workings of the heart and cardiovascular disease.

New breakthroughs on the horizon

With ground breaking research continuing to unlock the full potential of stem cell therapy, there is no doubt that collecting and storing your baby’s umbilical cord blood now could potentially be a life-saver in years to come. Whatever your family’s medical history, a stem cell transplant could prove your child’s best chance of ensuring they receive the most effective treatment or even a cure.

Banking your baby’s precious stem cells at birth is one of the greatest gifts you can give to your newborn – an investment that will last a lifetime.

About the author: Shamshad Ahmed, CEO and Founder of Smart Cells International.
Shamshad Ahmed is CEO and Founder of Smart Cells International Ltd. Opening in 2000, Smart Cells became the UK’s first private cord blood company – its goal to give parents more access to potentially life-saving treatment for their families. It is one of the UK’s largest private banks, operating across the globe and storing over 50,000 cord blood samples from people in over 70 countries. Shamshad started his career in finance and foreign exchange at Citibank before moving over to the world of clinical trials. He holds a BA from Nottingham Trent University, and he has been a member of the Young President’s Organization since 2008 – having served on the board for a number of those years.

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