Can preserved umbilical cord blood help treat certain hereditary diseases

Cord blood is significant because it contains blood stem cells, which can develop into any type of blood cell in the human body, such as oxygenated red blood cells or white blood cells that are part of the immune system.

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Illustration, Photo: Shutterstock
Illustration, Photo: Shutterstock
Disclaimer: The translations are mostly done through AI translator and might not be 100% accurate.

Pravin Gudipati and Ramja Raz, a couple from Houston, Texas, welcomed their first child, Kian, in 2019.

Before he was born, they were thinking about what to do with the blood from his umbilical cord.

They could throw it away, give it away or keep it privately for some future use.

For Gudipati, an IT professional, and Raza, an artist, there was no dilemma.

"We decided to donate it to a public non-profit bank, which meant that anyone who would benefit from the cord blood could also access it for free," says Gudipati.

His wife talked to several banks before they decided on the Foundation Cord for Life.

Cord blood is significant because it contains blood stem cells, which can grow into any type of blood cell in the human body, such as oxygenated red blood cells or white blood cells that are part of the immune system.

Blood stem cells are used to treat leukemia and other blood diseases.

They are not only in the blood of the umbilical cord, but also in the bone marrow, which is why surgical bone marrow transplantation is necessary in many blood diseases.

When the patient's own bone marrow is affected by the disease, then it needs to be matched with a donor for the transplant, which does not mean that the patient's body will always accept the donated cells.

Cord blood transplantation, on the other hand, is relatively simpler and more similar to a regular blood infusion.

Frozen cord blood is thawed, stem cells are tested for viability, and then injected into veins.

If your parents privately stored cord blood after you were born, you could theoretically access a supply of your own blood stem cells later in life (cord stem cells are viable for 20 years).

If they chose a public bank, it could give them to others as well.

"Thousands of life-saving transplants are performed each year using blood stem cells," says Leonard Zon, professor of pediatrics at Harvard Medical School and director of the Stem Cell Program at Children's Hospital Boston.

In terms of risk of rejection or infection, cells from the umbilical cord have an advantage, Zon adds.

They do not need the surgery associated with transplantation and can self-renew.

"One blood stem cell could replace 3.4 liters of your blood.

"Medical professionals currently use umbilical cord blood to treat blood disorders or to make red blood cells in conditions such as sickle cell anemia or to treat leukemia," he adds, noting that it can also be used to treat rare inherited genetic disorders.

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Around the world, more and more parents are finding that banking their baby's umbilical cord blood is the best way to ensure future access to this potentially life-saving resource, although the popularity of the procedure varies widely from country to country.

According to the database Cell Trials Data, about advanced cell therapy clinical trials, cord blood banking rates are the highest in America—three percent of cord blood is stored each year.

In India, that number is around 0.4 percent, in Great Britain only 0.3 percent, while in France that number is even 0.08 percent.

The value of the global cord blood banking market is estimated at one billion euros in 2020, and is expected to grow to four billion euros in the next decade.

For parents who can afford to privately store their baby's blood, the decision can be easy.

There are no risks during storage, and they would have direct access to perfectly matched blood.

Some, however, argue that as a health resource, cord blood is most powerful when used as a public good rather than a private one.

According to the American Academy of Pediatrics, private banks around the world hold six times more units of cord blood compared to public banks, but they dispense about 30 times more units for therapeutic use.

Professor Zon points out that blood stem cells are an extremely valuable resource, which does not mean that private storage is the only option for access.

"I always tell parents that if they can afford it, they should store cord blood privately, otherwise, it's not necessary," he says.

As long as public banks grow and donations become more diverse, it's possible to find the right match if you need it, he adds.

Zon appeals to parents to be realistic in their expectations of private banks and to choose carefully.

"In any procedure where you store tissue, there's a chance it could get infected.

"The ability of a healthcare worker to save cord blood is important," he points out.

Parents need to be aware that cord blood likely has a limited shelf life and cannot be stored indefinitely, Zon says.


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In some countries, cord blood can indirectly help solve other problems, such as the spread of antibiotic-resistant "superbugs" in hospitals.

In India, there are concerns about the rise of antimicrobial resistance, and avoiding surgery would be of significant benefit, experts say.

"Multidrug-resistant (antibiotic-resistant) bacterial infections are a huge problem in pediatric bone marrow transplant patients in India," says Hariharan Periasami, a scientist at the Research Center. Wockhardt for drug discovery in Aurangabad, Maharashtra.

As a result, the patient spends longer in the hospital, as a result of which the costs also increase, he adds.

Cord blood infusions, on the other hand, are not as risky.

Some experts warn that the power of cord blood should not be overestimated.

One study of claims made by private cord blood storage companies in Canada found there was "significant hype" in their online marketing material, from exaggerating the chance that a child would need cord blood to promising how it could be used.

Canadian Blood Services, which funded the study, says the odds of a baby needing cord blood are between one in 20.000 (0.005 per cent) and one in 250.000 (0.0004 per cent).

In India, the probability of needing stored cord blood in the first 20 years of life is estimated at 0.04 percent to 0.005 percent.

"It's not a magic pill that cures everything, and yet it's being marketed as 'biological insurance,' which is wrong," says Somya Gupta, an obstetrician and gynecologist at the Cloud Nine Hospitals chain in the northern Indian city of Gurugram.

"If you save your baby's cord blood, it's a 100 percent match for them, but if you're trying to treat a rare disease, their cord blood would still have disease-causing factors, so it can't be used."

Gupta is an advocate of public banks, of which there are very few in India.

"We urgently need to establish public cord blood banks," he adds.

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Parents in India currently pay huge sums to store umbilical cord blood in private banks or discard it.

Data from the Indian Society for Blood and Marrow Transplantation registry shows that between 2012 and 2020, only 60 unrelated cord blood transplants were performed.

In the US, where public banks abound, there is a somewhat different debate over cord blood.

Can it be used to treat certain hereditary diseases?

A new hope?

When Bertrand Might was two years old, he received an infusion of his own blood cells from the umbilical cord.

He suffered from a condition that affected his brain, says Matthew Might, Bertrand's father and director of the Institute for Precision Medicine Hugh Kaul at the University of Alabama at Birmingham.

The exact situation was determined much later.

Bertrand was the first patient diagnosed with NGLI1 deficiency, a complex neurological disorder that lacks the enzyme N-glycanase 1 (NGLI1).

"If the mutation doesn't affect cord blood, we thought these stem cells would repair some of the damage in the brain, creating a functional version of the missing enzyme.

"Even if it didn't produce the enzyme, we hoped it might temporarily stop or reverse the loss of white matter in the brain," he adds.

The infusion was given as part of an experimental study by Joanne Kurtzberg, a professor of pediatrics at Duke University in 2009, testing the effects of stem cell infusions on children with conditions that affect the brain.

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The infusions really seemed to help Bertrand, at least temporarily.

Over a period of two years, an MRI scan showed no further loss of white matter in the brain.

Majt also noticed the improvement of his son's motor skills.

“I remember he could hold a spoon again.

"His babbling became clearer and more understandable even though he is not speaking, the period of relentless deterioration was stopped, which bought us time to get an official diagnosis," he says.

The diagnosis helped Bertrand get the right care and treatment, and it extended his life.

However, the boy died at the age of 12 in 2020.

However, the injected cells could not cure him, says Might, because they had the same mutations that caused his disorder.

Although using the patient's cord blood is unlikely to work in these situations, as they will carry the same genetic errors that cause the disease, matching them with donated cord blood is more promising.

A smaller trial conducted at the Children's Hospital of the University of Pittsburgh Medical Center used donated umbilical cord blood from public banks to treat 44 children with a range of 20 non-cancerous genetic disorders, such as sickle cell anemia, metabolic disorders, Hunter syndrome and immune deficiencies.

The study offered hope that infusions of donated umbilical cord blood could be used to treat a wide range of patients with inherited diseases.

For Gudipati and Raju, a couple in Texas, this hope of helping another child prompted them to donate their son's umbilical cord blood.

"We believed that Kian came into the world to make a significant difference in someone's life.

"So we didn't hesitate and we didn't want this precious resource to end up as bio-waste," adds Gudipati.


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