Uses for Cord Blood Archives - Банка за матични клетки

Key details

Understanding Lesch-Nyhan syndrome: This rare, recessive condition linked to the X chromosome primarily affects boys, and is characterised by a deficiency of the HPRT enzyme, leading to severe motor dysfunction, uric acid overproduction, and compulsive self-injury.
Role of HPRT: Used by the body to process and recycle purines: organic compounds that are a type of building block of DNA and RNA.
Promising transplant results: A recent case study demonstrated that a cord blood transplant (from a 5/6 HLA-matched donor) successfully restored HPRT1 protein levels to normal range and prevented the onset of self-injury behaviour.
Cord blood efficacy: Umbilical cord blood is often considered the preferred stem cell source for treating inborn errors of metabolism because it supports successful engraftment and is safer than other sources when the donor and patient are not a perfect genetic match.

What is Lesch-Nyhan syndrome?

Lesch-Nyhan syndrome is a rare condition which occurs almost exclusively in boys. It is classified as an inborn error of metabolism, meaning it is congenital (present at birth), inherited, and involves a malfunction in the body’s chemical processes (metabolism). It affects a child’s brain and behaviour, as well as causing overproduction of uric acid, a waste product which is normally eliminated through the kidneys.[1][2][3][4] Symptoms include:

Urate crystals: Forming in the urine due to the build-up of uric acid, these orange-coloured crystals can deposit in the diapers of babies with this condition. They are often the first symptom to appear.
Motor issues: These begin to manifest around four months of age, with decreased muscle tone (hypotonia) resulting in a limp, “floppy” appearance and poor head control being one of the earliest symptoms. This is followed by involuntary muscle spasms (dystonia), repetitive movements (chorea) and flailing of the limbs (ballismus), as well as muscle rigidity (spasticity). Opisthotonos, a severe muscle spasm which causes the back to arch and the head and heels to bend backwards, can also occur. Babies can miss developmental milestones such as sitting, crawling or walking; those who had previously learned to sit upright typically regress and lose the ability. Most people with Lesch-Nyhan syndrome cannot walk, are unable to sit without support, and generally use a wheelchair. Speech can be slurred or poorly articulated (dysarthria); problems with swallowing (dysphagia) can also occur.
Gout: The accumulation of uric acid in the joints eventually leads to recurring pain and swelling, similar to what happens in adults with gout. These episodes generally begin in the late teens to early adulthood, and can become progressively more frequent over time.
Intellectual disability: Some cognitive impairment is typically present, although it is hard to gauge accurately due to the motor issues and dysarthria.
Self injury: This compulsive behaviour is a hallmark of Lesch-Nyhan syndrome, occurring in a majority of patients (about 85%), and typically begins in early childhood. It includes biting of the cheeks, lips, fingers or hands, as well as banging the head or limbs against hard objects, scratching the face, or poking at eyes.
Behavioural issues: Patients with Lesch-Nyhan syndrome may also have episodes in which they act aggressive and attempt to injure others, or become verbally abusive.[3][4]

What causes Lesch-Nyhan syndrome?

Lesch-Nyhan syndrome is caused by a genetic mutation, and is inherited in an X-linked recessive pattern. The affected gene, HPRT1, is located on the X chromosome; this means that females are mostly carriers as they’re often protected by an unaffected X chromosome, and males who inherit the defective chromosome from their mothers are affected by the condition. Occasionally, Lesch-Nyhan syndrome can also develop in a family with no history of it. This is known as a spontaneous, or de novo, mutation.

Whether inherited or spontaneous, the mutation results in a severe shortage or complete absence of the HPRT1 protein. The body uses this protein to process and recycle purines, organic compounds which are a type of building block of DNA and RNA. Without this protein, purines are instead broken down but not recycled, resulting in abnormally high levels of uric acid in the blood. When too much uric acid is produced, the kidneys can’t keep up, and uric acid accumulates in the body.[1][2][3][4]

It is still unclear how Lesch-Nyhan syndrome affects the brain, causing the neurological and behavioural issues associated with the condition. Researchers suspect that it may either impact the levels of dopamine, an important chemical messenger required for the brain to function correctly, or reduce the function of dopamine receptors.[2][4]

Is there a cure for Lesch-Nyhan syndrome? What treatment is available?

There is no cure for Lesch-Nyhan syndrome. Treatment primarily aims to treat symptoms, and is generally handled by a team of specialists rather than a single treating physician.[2][3]

Treatment can include:

Allopurinol, a medication normally used for gout, to reduce the amounts of uric acid present in the body and control the symptoms caused by it.
Procedures to break down kidney or bladder stones.
Physical splints or restraints to prevent self-injury, including hip, chest and elbow restraints as well as a mouth guard.
Supportive equipment such as a wheelchair.[2][3]

No standard treatment is available for the neurological and behavioural symptoms of Lesch-Nyhan syndrome, although a variety of medication may help ameliorate some of them.[2][3][5][6]

Could a cord blood transplant help with Lesch-Nyhan syndrome?

If performed early enough, a cord blood transplant could potentially slow or halt the progression of Lesch-Nyhan syndrome. A recently published case study, detailing the results of a cord blood transplant for Lesch-Nyhan syndrome, provides the strongest evidence to date for this.[7]

The patient, a boy, initially presented at six months of age with hypotonia, inability to roll over, and occasional opisthotonos. MRI and EEG results raised the suspicion of Lesch-Nyhan syndrome, which was confirmed with genetic testing. Crucially, the diagnostic confirmation happened before the onset of any self-injury compulsion.

As the condition was progressing and the patient did not have a matched sibling donor, doctors made the decision to proceed with a cord blood transplant from a partially matched (5/6 HLA) donor, performed at 14 months old.

The outcome of the transplant was positive:

Treatment safety: 28 days post-transplant, the patient had mild graft-versus-host disease (GvHD), a condition in which white blood cells remaining in the donated graft attack the cells of the host’s body. This was resolved with corticosteroids, a medication which reduces inflammation and suppresses the immune system. Beyond this, there were no complications, and the boy remained free of GvHD by the time the case report was submitted for publication.
Successful engraftment: Complete donor chimerism, meaning over 95% of the patient’s new blood cells were of donor origin, was achieved on day 32 post-transplant.
HPRT1 protein levels: Prior to the transplant, the patient’s HPRT1 protein levels were low (40.9 pg/ml) compared to those of his father and mother (98.8 pg/ml and 78.9 pg/ml respectively). Post-transplant, levels rose to 91.1 pg/ml by day 32 and remained normal (97.0 pg/ml) at day 126.
Neurological improvement: The patient’s dystonia and spasticity decreased, opisthotonos became more infrequent, and he had improved facial expressions, increased social interaction and developmental progress. At 36 months of age, he still showed no sign of any self-injury behaviour.[7]

This case shows a promising result, but research is still in the very early stages. Including this case, only three haematopoietic stem cell transplants (HSCT) have been performed in children with Lesch-Nyhan syndrome before the onset of the self-injury compulsion:

	Endres et al. (1991)	Kállay et al. (2012)	Weng et al. (2025)
Patient age	16 months	24 months	14 months
Stem cell source	Bone marrow	Cord blood (6/6 match)	Cord blood (5/6 match)
Outcome	Death (day +10)	Survival, full chimerism	Survival, full chimerism
Long-term outcome	N/A	No self-injury at 5yr follow-up. Persistent motor delay.	No self-injury at 36mo. Improved dystonia/social.

Thus, no formal protocol for this treatment has been established yet. Instead, until more evidence is available, the decision on whether a transplant is appropriate should be made individually for each case, by a coordinated multidisciplinary team including ethical oversight, with transparent family counselling so that informed parental consent is possible.[7]

What is known for certain is:

HSCT has been successfully used to treat other conditions in the inborn error of metabolism category, and is the standard of care for some of them, such as Hurler syndrome [8] and Krabbe disease.[9]
Cord blood is generally the preferred stem cell source for such transplants, as it increases the likelihood of full donor chimerism and is safer to use when the donor and the patient aren’t a perfect genetic match.[7][8][10]

In the case of Krabbe disease, specifically, early transplantation is of paramount importance; the sooner a transplant happens, the better the functional results for the child, with differences being noticeable even between children transplanted before 30 days of age and those transplanted after.[11] In the United States, several states have implemented newborn screening for Krabbe disease because of this. Here, too, cord blood presents itself as a superior stem cell source, as families may have previously banked the cord blood from an unaffected sibling and, if not, a matched cord blood unit may be available in a public bank.

Should a newborn screening targeted at Lesch-Nyhan syndrome be implemented, it would mean the condition could be identified before symptoms develop. The authors of this case study speculate that early transplant could prevent irreversible neuronal loss and preserve neurological potential.[7]

The importance of family banking

Storing your baby’s cord blood privately gives them access to their own perfectly matched stem cells, and a 75% chance they could be a match for a sibling who may need a transplant; comprising a 25% chance of a perfect match, and 50% chance of a partial match.

As the regenerative medicine field continues to advance, researchers are also investigating the potential of autologous stem cell treatments, using patients’ own stem cells. These would involve gene editing techniques such as CRISPR, and have already become available for some inherited conditions, such as sickle cell disease and beta thalassemia; for other conditions, including Lesch-Nyhan[12] and Krabbe disease, they are in pre-clinical or clinical trial stage. For any of these conditions, it is also possible that the most effective treatment would be a combination of cord blood transplant and gene therapy; this is currently being investigated for Krabbe disease.[13]

Should you choose to bank your baby’s cord blood, it is possible that the stem cells it contains could be used for autologous treatments, rather than more invasively collected bone marrow or peripheral blood stem cells. Should your baby prove to be a donor match for one of their siblings or another family member who is in need of a transplant, the cord blood could potentially also be used in that case.

To learn more about banking your baby’s cord blood, as well as about other sources of stem cells that can only be collected immediately after birth, fill in the form below to request your free guide.

References

[1] MedlinePlus. Lesch-Nyhan syndrome. https://medlineplus.gov/genetics/condition/lesch-nyhan-syndrome/

[2] Cleveland Clinic (2022). Lesch-Nyhan Syndrome: Causes, Symptoms & Treatment. https://my.clevelandclinic.org/health/diseases/23493-lesch-nyhan-syndrome

[3] National Organization for Rare Disorders (2015). Lesch Nyhan Syndrome. https://rarediseases.org/rare-diseases/lesch-nyhan-syndrome/

[4] Nanagiri, A. and Shabbir, N. (2020). Lesch Nyhan Syndrome. https://www.ncbi.nlm.nih.gov/books/NBK556079/

[5] Brainfacts.org. (2025). Lesch Nyhan Syndrome. https://www.brainfacts.org/diseases-and-disorders/neurological-disorders-az/diseases-a-to-z-from-ninds/lesch-nyhan-syndrome

[6] Nyhan, W.L. (2005). LESCH-Nyhan Disease. Journal of the History of the Neurosciences, 14(1), pp.1–10. doi:https://doi.org/10.1080/096470490512490

[7] Weng, T.-F., Tin, C.-H. and Wu, K.-H. (2025). Umbilical Cord Blood Transplantation in Lesch-Nyhan Syndrome: A Case Report and Literature Review. Cureus. doi:https://doi.org/10.7759/cureus.97008

[8] Tan, E.Y., Boelens, J.J., Jones, S.A. and Wynn, R.F. (2019). Hematopoietic Stem Cell Transplantation in Inborn Errors of Metabolism. Frontiers in Pediatrics, 7. doi:https://doi.org/10.3389/fped.2019.00433

[9] Wright, M.D., Poe, M.D., DeRenzo, A., Haldal, S. and Escolar, M.L. (2017). Developmental outcomes of cord blood transplantation for Krabbe disease. Neurology, 89(13), pp.1365–1372. doi:https://doi.org/10.1212/wnl.0000000000004418

[10] Aldenhoven, M. and Kurtzberg, J. (2015). Cord blood is the optimal graft source for the treatment of pediatric patients with lysosomal storage diseases: clinical outcomes and future directions. Cytotherapy, 17(6), pp.765–774. doi:https://doi.org/10.1016/j.jcyt.2015.03.609

[11] Allewelt, H., Taskindoust, M., Troy, J., Page, K., Wood, S., Parikh, S., Prasad, V.K. and Kurtzberg, J. (2018). Long-Term Functional Outcomes after Hematopoietic Stem Cell Transplant for Early Infantile Krabbe Disease. Biology of Blood and Marrow Transplantation, 24(11), pp.2233–2238. doi:https://doi.org/10.1016/j.bbmt.2018.06.020

[12] Jang, G., Shin, H.R., Do, H.-S., Kweon, J., Hwang, S., Kim, S., Heo, S.H., Kim, Y. and Lee, B.H. (2023). Therapeutic gene correction for Lesch-Nyhan syndrome using CRISPR-mediated base and prime editing. Molecular Therapy – Nucleic Acids, 31, pp.586–595. doi:https://doi.org/10.1016/j.omtn.2023.02.009

[13] ClinicalTrials.gov (2024). A Phase 1/2 Clinical Study of Intravenous Gene Transfer With an AAVrh10 Vector Expressing GALC in Krabbe Subjects Receiving Hematopoietic Stem Cell Transplantation (RESKUE). https://www.clinicaltrials.gov/study/NCT04693598

Today is World Cord Blood Day! A lot of progress has been made since the very first cord blood transplant was performed in 1988, 37 years ago. Since then, over 60,000 cord blood transplants have been performed worldwide, and cord blood stem cells have become the accepted treatment for over 80 illnesses and conditions, ranging from Fanconi anaemia to Krabbe disease, from SCID to leukaemia, and more.

What’s more, thousands of clinical trials all over the world are investigating the potential of cord blood stem cells to treat conditions which are currently incurable, including cerebral palsy, heart failure, spinal cord injuries and Crohn’s disease, to name but a few.

Despite this, cord blood and other perinatal sources of powerful stem cells, including the cord tissue and the placenta, are still frequently discarded after birth. Thus, World Cord Blood Day was created to highlight cord blood’s therapeutic potential and encourage more families to preserve it for the future.

Cord blood news

Research in the regenerative medicine field continues to advance our knowledge of what’s possible using cord blood. In case you’ve missed them, here are some news articles from the past year:

Early trials in mice have found that a combination therapy involving umbilical cord stem cells and electrical stimulation could be key to treating spinal cord injuries.
An Australian toddler is in remission from a dangerous form of leukaemia following a transplant of his baby sister’s cord blood.
A leukaemia treatment derived from umbilical cord blood has gained FDA approval to begin clinical trials.
A treatment based on umbilical cord blood stem cells has been found to offer superior results for the treatment of osteoarthritis.
A novel therapy for lymphoma that is highly resistant to treatment, based on umbilical cord blood cells, published positive results.
Cord blood therapy has been shown to be an effective treatment for cerebral palsy, improving motor skills.
A six year old girl became the first child in Australia to receive her own cord blood as treatment for cerebral palsy.

Banking your baby’s cord blood

Both public and private banks are an option if you want to save your baby’s cord blood rather than having it discarded.

If you choose a public bank, you are donating your baby’s cord blood. This means that it will be available for use to anyone who might need it. The NHS accepts cord blood donations if you are giving birth at one of three hospitals; the Anthony Nolan charity can collect cord blood donations from five more.

As an alternative, you can choose private cord blood banking. When you do this, your baby’s cord blood stem cells will be stored and reserved solely for your family’s use, ready and waiting should your baby, or another family member, ever need them. In addition to being a perfect genetic match for your baby, they also have a 25% chance of being a perfect match for a sibling, and a 50% chance of being a partial match. They are also guaranteed to be a partial match for parents.

At Cells4Life, we offer a range of services aimed at providing the maximum possible benefit to families who choose to store their baby’s precious stem cells with us. Our proprietary processing technology retains up to 3 times more stem cells than industry-standard processing methods, making cord blood collection fully compatible with delayed and optimal cord clamping. We also offer cord tissue, placenta and amnion banking.

A six-year-old girl named Zara has become the first child in Australia to receive an infusion of her own stored cord blood as treatment for cerebral palsy outside of a clinical trial. Experts hope that this milestone will pave the way towards broader availability, therefore removing the need for Australian children with cerebral palsy to travel elsewhere for treatment.[1]

Zara’s story

Zara was born prematurely, and was diagnosed with cerebral palsy after she began missing milestones. Before she was born, her parents had made the decision to store her cord blood with an Australian stem cell bank, due to her mother having type 1 diabetes. After the cerebral palsy diagnosis, her parents wondered if the stored cord blood might help her.[1]

Initially, they considered travelling overseas, as cord blood treatment for cerebral palsy is not generally available in Australia. In fact, excluding Zara, only 12 other children have received this treatment, and all were part of a clinical trial. Conversely, Australian charity Cerebral Palsy Alliance (CPA) estimates that hundreds of families have had to spend tens of thousands of dollars to seek this treatment abroad.[1][2]

Planning and securing the required approvals for the treatment took more than a year. It was a coordinated group effort, involving not only Zara’s parents and her paediatric neurologist, Professor Michael Fahey, but also the stem cell bank, CPA, Melbourne’s Monash Children’s Hospital and Hudson Institute of Medical Research and several local politicians.[1]

Zara received her cord blood in April of this year, and her progress will be monitored in the months and years ahead. The biggest gains are expected between three and six months after the treatment, but Zara’s parents are already seeing improvements in her balance and movement, as well as reduced muscle stiffness. “At soccer training, she can weave between the cones easier,” her mother said. “At physio the other day, for the first time in her life, she walked up and down four steps without having to hold on to the rail.”[1]

What does cord blood treatment for cerebral palsy entail?

After careful thawing and preparation, the cord blood treatment is given as an infusion, through a drip into the arm. The process takes 20-30 minutes and is very similar to a standard blood transfusion.[3][1] It is believed that the cord blood treatment works by helping the brain form new pathways; these are then strengthened and refined by a cycle of rehabilitation, done after the treatment.[3] A recently-published review paper confirms, through extensive data analysis, that cord blood treatment followed by rehabilitation improves motor skills significantly more than rehabilitation alone.[4][5]

The treatment can use either autologous (the child’s own) cord blood, as in Zara’s case, or allogeneic (a donor’s). In the latter case, a sibling is often the cord blood donor. At Cells4Life, we have released six cord blood samples for treatment of a sibling with cerebral palsy, most recently in 2024; sibling donors were also used in the single clinical trial performed in Australia.[2]

Why is this treatment not more widely available?

The science supports the effectiveness of cord blood treatment for cerebral palsy, particularly in younger children as well as those with less severe forms of the condition.[4][5] However, it is not yet approved in any country, including Australia and the UK, and as such is not readily available as a therapy option. This is because large-scale phase 3 clinical trials are needed for approval, and are expensive and complicated to organise.[5]

In the meantime, outside of clinical trials, cord blood treatment for cerebral palsy is available only through expanded access or compassionate use pathways. This can require either individual approval for the treatment, as in Zara’s case, or participation in an existing expanded access programme, such as the one at Duke University in the USA.

Both of those routes to treatment are likely to require a child having access to either their own banked cord blood, or a sibling’s. This is because while there are public cord blood banks, the cord blood stored there may be available for use only for approved treatments or clinical trials.

Banking your baby’s cord blood at birth could therefore be essential to ensure access to this treatment if they, or a sibling, develop cerebral palsy. To learn more about cord blood banking, as well as other potential uses of cord blood for other diseases and conditions, fill in the form below to request your free guide.

References

[1] Booth, S. (2025). ‘Already improving’: First Aussie child receives top cerebral palsy treatment. https://www.heraldsun.com.au/health/conditions/already-improving-first-aussie-child-receives-top-cerebral-palsy-treatment/news-story/7f5e9b0bbab8bcf802aaa4e9e282d9ab

[2] Hudson Institute of Medical Research. (2025). Hudson Cell Therapies facilitates Australian first UCB stem cell treatment for cerebral palsy. https://www.hudson.org.au/news/hudson-cell-therapies-facilitates-australian-first-ucb-stem-cell-treatment-for-cerebral-palsy/

[3] Cerebral Palsy Alliance. (2025). Umbilical cord blood and cerebral palsy. https://cerebralpalsy.org.au/advocacy/umbilical-cord-blood/

[4] Finch-Edmondson, M., et al. (2025). Cord Blood Treatment for Children With Cerebral Palsy: Individual Participant Data Meta-Analysis. Pediatrics, 155(5), p.e2024068999. doi:https://doi.org/10.1542/peds.2024-068999

[5] Parent’s Guide to Cord Blood (2025). Cord Blood Proven Effective for Cerebral Palsy. https://parentsguidecordblood.org/en/news/cord-blood-proven-effective-cerebral-palsy

A review paper, newly published in the prestigious Pediatrics journal, statistically demonstrates that cord blood therapy for cerebral palsy is an effective treatment to improve the motor skills of children suffering from this condition. The paper is an analysis of data from eleven studies on over 400 children with cerebral palsy.

What is cerebral palsy?

Cerebral palsy is a group of motor disorders that affect movement, posture and coordination. It’s caused by damage to the brain while it is still developing. This happens most often before birth, but sometimes during or immediately after.

The severity of the condition can vary greatly. Symptoms include lack of balance and muscle coordination, muscles that are either too stiff or too floppy, exaggerated reflexes, and issues with walking, eating and speech.[1][2]

There is currently no cure for cerebral palsy. Available treatment includes physiotherapy, occupational therapy, medication and sometimes surgery. This is aimed at helping people with the condition to be as healthy and independent as possible.[3]

What did the analysis find?

According to the analysis results, children who underwent cord blood therapy for cerebral palsy combined with rehabilitation saw a significantly greater improvement in gross motor skills compared to those gained from rehabilitation alone. The improvements reached their peak between 6 and 12 months after the cord blood therapy; the data also indicates that higher cell doses per kg of patient weight result in bigger improvements. This is consistent with the current hypothesis that the treatment works by reducing inflammation in the brain and stimulates tissue repair, leading to improvements in brain connectivity.[4][5]

After six to twelve months, the majority (68%) of children who underwent cord blood therapy for cerebral palsy scored higher on the gross motor function measure scale used for cerebral palsy (GMFM-66) than the entire control group. The analysis did, however, also find that the treatment had better results in younger children, as well as those with less severe cerebral palsy. Overall, the children who saw the best results were those under the age of 5 who had some ability to walk, whether unaided or with support, before the therapy.[4][5]

What does this mean for the treatment of cerebral palsy?

Cerebral palsy remains one of the most common motor disorders in childhood. It can be accurately diagnosed as early as 6 months of age, which is well within the bracket of highest therapy effectiveness identified by the analysis. What’s more, the sooner a child is treated, the better. This is because a single unit of cord blood will result in a higher cell dose per kg when a child is still small, and may even be enough for multiple treatments.[4]

At the present time, cord blood therapy is not approved as a treatment for CP in any country. This is due to it still requiring large-scale, phase 3 trials before it can reach this approval. Therefore, it is only available through clinical trials as well as expanded access or compassionate use programmes.[4]

This lack of approval means that, although there are cord blood units available for use in public banks, it may not be possible to use these for treatment in the immediate without gaining access to a clinical trial. Even then, the clinical trial may require the cord blood used in the treatment to be autologous (the child’s own), or to come from a sibling donor. Expanded access programmes may also have the same requirements.

A child having access to their own banked cord blood, or a sibling’s, could therefore be essential to ensure treatment can happen during the window of opportunity that could achieve the best results. To learn more about how you could preserve this important health resource for your child and your family, fill in the form below to request your free Parents’ Guide to Cord Blood Banking.

References

[1] Mayo Clinic (2023). Cerebral palsy – symptoms and causes. https://www.mayoclinic.org/diseases-conditions/cerebral-palsy/symptoms-causes/syc-20353999

[2] NHS (2023). Overview – Cerebral Palsy. https://www.nhs.uk/conditions/cerebral-palsy/

[3] NHS (2023). Treatment – Cerebral palsy. https://www.nhs.uk/conditions/cerebral-palsy/treatment/

[4] Parent’s Guide to Cord Blood (2025). Cord Blood Proven Effective for Cerebral Palsy. https://parentsguidecordblood.org/en/news/cord-blood-proven-effective-cerebral-palsy

[5] Finch-Edmondson, M., et al. Cord Blood Treatment for Children With Cerebral Palsy: Individual Participant Data Meta-Analysis. Pediatrics 2025; e2024068999. 10.1542/peds.2024-068999.

A phase 1 trial of a novel cell therapy for lymphoma that is highly resistant to standard treatment (highly refractory lymphoma) has published very positive results in the Nature Medicine journal. The therapy, which uses natural killer (NK) cells derived from cord blood, resulted in complete remission in two thirds of the treated patients.

What are natural killer cells?

NK cells are a specific type of white blood cell, a part of the immune system which targets and destroys infected and cancerous cells. These cells patrol the body, determining which cells should be destroyed based on whether they receive the correct signals from them or not.[1]

Their ability to kill cancerous cells makes NK cells ideal for the development of cell therapies to treat cancer. These typically hinge on modifying the NK cells, granting them the ability to recognise and destroy cancerous cells they would otherwise ignore.[2] Such is the case for this novel cord blood cell therapy for lymphoma, a type of blood cancer which affects lymphocytes, white blood cells which are part of the immune system. Normally, lymphocytes help the body fight off infection. With lymphoma, however, they do not work correctly and can multiply out of control, accumulating quickly in lymph nodes and other organs in the lymphatic system, such as the spleen and liver.[3][4]

Thanks to advances in medicine, lymphoma is generally considered a very treatable type of cancer, with 5-year survival rates averaging between 74% and 89% depending on the type of lymphoma.[5][6][7] However, sometimes the cancer still resists treatment or reoccurs afterwards, leaving patients with refractory or relapsed lymphoma with few options and a poor prognosis.[8] This can happen in between 10%-40% of cases, depending on the type of lymphoma, the patient age at diagnosis, and how advanced the disease was at diagnosis.[9][10] This is the need this cord blood cell therapy for lymphoma is attempting to fill.

What does the new therapy entail?

Researchers first isolated NK cells from cord blood and then activated them using a combination of cytokines.[8] Cytokines are proteins which the immune system uses to send signals;[11] using them to activate NK cells stimulates their cancer-fighting abilities.[12]

Then, the antibody AFM13 was added to the NK cells, enabling them to target the CD30 protein. This protein is found on the surface of cancerous cells in specific types of lymphoma, primarily in Hodgkin lymphoma but also other varieties.[8]

Following chemotherapy, doctors infused a dose of the treated NK cells into patients. This was then followed by three doses of the AFM13 antibody, administered once a week.

Why is this therapy so important?

The 42 patients who took part in the trial all had highly refractory lymphoma, having received a median of seven prior treatment courses. In spite of this, the therapy achieved an overall response rate of 92.9%, with 66.7% of patients experiencing complete remission. In patients with Hodgkin lymphoma specifically, the rates were even higher, with a complete remission rate of 73% and an overall response rate of 97.3%.

Eleven patients remained in complete remission for at least 14 months, with this lasting, for some, up to 40 months after receiving the therapy. Five patients remained in complete remission without any further treatment, and six went on to receive a stem cell transplant.

At a median follow-up of 20 months, the therapy led to complete disease remission in 26.2% patients, or about one in four patients. The two-year overall survival rate was 76.2%.[8] These are encouraging and very positive results, particularly considering how resistant their cancer had been to previous treatment. Moreover, the therapy proved to be safe and well-tolerated, with no adverse side effects beyond those caused by the chemotherapy.

This trial was a small-scale one, primarily aimed at confirming the safety of the treatment and determining an optimal dose. Larger trials are therefore needed before this therapy can become available to patients.

Still, trials like this, as well as many others currently ongoing, highlight the strong curative potential of cord blood cells. To discover more about cord blood cells, and find out how you could preserve them for your baby and family’s potential future use, fill in the form below to request our free guide.

References

[1] Cleveland Clinic (2023). What Are Natural Killer Cells (NK Cells)? https://my.clevelandclinic.org/health/body/24898-natural-killer-cells

[2] Eissmann, P. (2016). Natural Killer Cells | British Society for Immunology. https://www.immunology.org/public-information/bitesized-immunology/cells/natural-killer-cells

[3] Blood Cancer UK. Lymphoma. https://bloodcancer.org.uk/understanding-blood-cancer/lymphoma/

[4] Mayo Clinic (2024). Lymphoma – Symptoms and Causes. https://www.mayoclinic.org/diseases-conditions/lymphoma/symptoms-causes/syc-20352638

[5] LLS (2025). Lymphoma Survival Rate By Age. https://lls.org/blog/lymphoma-diagnosis-survival-rate-age-prognosis-and-treatment

[6] Cancer Research UK (2012). Survival | Hodgkin Lymphoma | Cancer Research UK. https://www.cancerresearchuk.org/about-cancer/hodgkin-lymphoma/survival

[7] Cancer Research UK (2011). Survival | non-Hodgkin lymphoma | Cancer Research UK. https://www.cancerresearchuk.org/about-cancer/non-hodgkin-lymphoma/survival

[8] Nieto, Y., et al. (2025). Allogeneic NK cells with a bispecific innate cell engager in refractory relapsed lymphoma: a phase 1 trial. Nature Medicine. doi:https://doi.org/10.1038/s41591-025-03640-8

[9] Radhakrishnan Ramchandren (2012). Advances in the Treatment of Relapsed or Refractory Hodgkin’s Lymphoma. The Oncologist, 17(3), pp.367–376. doi:https://doi.org/10.1634/theoncologist.2011-0258

[10] Harrysson, S., et al. (2021). Incidence of relapsed/refractory diffuse large B-cell lymphoma (DLBCL) including CNS relapse in a population-based cohort of 4243 patients in Sweden. Blood Cancer Journal, 11(1). doi:https://doi.org/10.1038/s41408-020-00403-1

[11] Cleveland Clinic (2023). What are Cytokines? Types and Function. https://my.clevelandclinic.org/health/body/24585-cytokines

[12] Romee, R., et al. (2016). Cytokine-induced memory-like natural killer cells exhibit enhanced responses against myeloid leukemia. Science Translational Medicine, [online] 8(357), p.357ra123. doi:https://doi.org/10.1126/scitranslmed.aaf2341

Osteoarthritis, a chronic degenerative joint disease, affects millions of people worldwide and is a leading cause of disability.[1][2] Currently available treatments can offer relief from pain and other symptoms. However, they fail to address the root of the disease, as they cannot effectively restore the damaged joint.[3] A novel stem cell therapy based on cord blood mesenchymal stem cells (UCB-MSCs) is now emerging as a promising regenerative approach. The therapy, called Cartistem, has been shown to regenerate strong, elastic cartilage in the joint, rather than the fibrous, weaker cartilage created by an alternative treatment.[4]

What is osteoarthritis?

Osteoarthritis is the most prevalent form of arthritis, and primarily affects middle-aged and older adults. It involves the deterioration of the cartilage that protects the ends of bones within a joint. Because cartilage does not contain blood vessels, it has a limited ability to self-repair[5]; with continued deterioration, it disappears entirely, leading to damage and changes to the bones themselves. Symptoms include joint stiffness and pain, as well as reduced range of motion, and can increase over time. Eventually, they can become severe enough to make day-to-day activities more difficult.[1][6]

The risk of osteoarthritis increases with age, although there are other risk factors which can contribute. These include joint injuries, overuse of the joint in work or sports, obesity and genetics.[1] Osteoarthritis can affect any joint in the body, but most frequently appears in the hands, knees, hips and spine.[1] Knee osteoarthritis, in particular, often involves both knees. It can cause pain during walking, especially going uphill or downhill, as well as difficulty straightening the legs and a sensation of the knee “giving way”.[7]

Treatments for knee osteoarthritis

The current standard of care for knee osteoarthritis includes a range of treatments that aim to manage pain, reduce stiffness and improve range of motion. They include both surgical and non-surgical options.

Non-surgical treatment is typically tried at first. This includes pain medication as well as lifestyle changes, such as weight loss and exercise, and assistive devices, such as special footwear, braces, or walking aids.[8][9] If non-surgical treatments have not been effective or the patient’s daily life is seriously affected, doctors then look at surgical treatments. This could be an operation called an osteotomy, where a small section of bone on one side of the joint is removed to realign the joint and shift weight away from damaged parts of the cartilage.[10] Knee replacement, either partial or total, is also an option.[8][9]

Another possibility is a surgical treatment called microfracture or microdrilling. This treatment involves doctors making small holes into the surface of the bone where cartilage is absent. The holes cause blood and bone marrow to coat the surface of the bone, then forming into a clot. Thanks to the stem cells contained in blood and bone marrow, this clot eventually develops into a new layer of cartilage.[11] However, this new cartilage is not as strong as the original cartilage, and can break down more easily,[5][12] meaning any improvement is temporary.

A new therapeutic option

Cartistem is an off-the-shelf stem cell therapy developed in South Korea, where it has been approved for knee osteoarthritis treatment since 2012.[13] It consists of a combination of allogeneic UCB-MSCs and hyaluronic acid hydrogel. Applied to the area of damaged cartilage during surgery, the stem cells stimulate cartilage regeneration.[14]

Although Cartistem can be considered comparable to microdrilling in terms of the underlying process, a recent study has shown that this therapy can regenerate cartilage that is stronger and more resilient. The study, the results of which were recently presented at the 2025 annual meeting of the American Academy of Orthopaedic Surgeons, directly compared the results of Cartistem to those of microdrilling.[4]

According to researchers, Cartistem repaired a greater surface area than microdrilling, producing stronger cartilage which contained more collagen and had improved stiffness and elasticity. In contrast, microdrilling produced weaker, more fibrous cartilage, and in one out of five patients resulted in no cartilage regeneration at all.[14]

On the strength of the results from this and other studies, the FDA granted approval to proceed directly to a phase 3 clinical trial in the United States. A phase 3 trial is also underway in Japan.[4]

The regenerative power of cord blood stem cells

The strong potential of UCB-MSCs for the treatment of osteoarthritis is well-documented, with several recent studies highlighting their positive effects.[15][16][17]

Cartistem is a proprietary, off-the-shelf therapy, using allogeneic (donor) UCB-MSCs. However, as therapies are still being discovered and developed, it is entirely possible that a patient having access to their own cord blood stem cells could make the difference in terms of effectiveness. This could be the case not only for osteoarthritis, but for also for many other illnesses and diseases.

To discover more about cord blood stem cells, and how you could preserve them for your baby for potential future treatment, fill in the form below to request your free guide to cord blood banking.

References

[1] Mayo Clinic (2021). Osteoarthritis. https://www.mayoclinic.org/diseases-conditions/osteoarthritis/symptoms-causes/syc-20351925

[2] Neogi, T. (2013). The Epidemiology and Impact of Pain in Osteoarthritis. Osteoarthritis and Cartilage, 21(9), pp.1145–1153. doi:https://doi.org/10.1016/j.joca.2013.03.018

[3] Mayo Clinic (2021). Osteoarthritis – Diagnosis and treatment – Mayo Clinic. https://www.mayoclinic.org/diseases-conditions/osteoarthritis/diagnosis-treatment/drc-20351930

[4] Ji-hye, K. (2025). MEDIPOST’s stem cell therapy regenerates stronger cartilage than microdrilling, clears path to US phase 3. KBR. https://www.koreabiomed.com/news/articleView.html?idxno=26972

[5] Tuan, R.S., Chen, A.F. and Klatt, B.A. (2013). Cartilage Regeneration. Journal of the American Academy of Orthopaedic Surgeons, 21(5), pp.303–311. doi:https://doi.org/10.5435/jaaos-21-05-303

[6] Johns Hopkins Medicine (2024). Osteoarthritis. https://www.hopkinsmedicine.org/health/conditions-and-diseases/arthritis/osteoarthritis

[7] NHS (2019). Symptoms – Osteoarthritis. https://www.nhs.uk/conditions/osteoarthritis/symptoms/

[8] NHS (2023). Treatment – Osteoarthritis. https://www.nhs.uk/conditions/osteoarthritis/treatment/

[9] Cleveland Clinic (2021). Osteoarthritis of the Knee: Symptoms, Causes and Treatments. https://my.clevelandclinic.org/health/diseases/21750-osteoarthritis-knee

[10] Cleveland Clinic. (2022). Osteotomy (Bone Cutting): What It Is, Procedure & Recovery. https://my.clevelandclinic.org/health/articles/22688-osteotomy

[11] Royal Orthopaedic Hospital. Microfracture/Chondroplasty of the Knee. https://roh.nhs.uk/services-information/knees/microfracture-chondroplasty-of-the-knee

[12] MedlinePlus. Knee microfracture surgery: MedlinePlus Medical Encyclopedia. https://medlineplus.gov/ency/article/007255.htm

[13] MEDIPOST. (2022). CARTISTEM®. https://en.medi-post.co.kr/cartistem/

[14] Jung, S., et al. (2024). Implantation of hUCB‐MSCs generates greater hyaline‐type cartilage than microdrilling combined with high tibial osteotomy. Knee Surgery Sports Traumatology Arthroscopy, 32(4), pp.829–842. doi:https://doi.org/10.1002/ksa.12100

[15] Jung, S.-H., et al. (2024). Allogeneic umbilical cord blood-derived mesenchymal stem cell implantation versus microdrilling combined with high tibial osteotomy for cartilage regeneration. Scientific Reports, [online] 14(1). doi:https://doi.org/10.1038/s41598-024-53598-9

[16] Zhang, P., Dong, B., Yuan, P. and Li, X. (2023). Human umbilical cord mesenchymal stem cells promoting knee joint chondrogenesis for the treatment of knee osteoarthritis: a systematic review. Journal of Orthopaedic Surgery and Research, 18(1), p.639. doi:https://doi.org/10.1186/s13018-023-04131-7

[17] Prodromos, C., Finkle, S., Rumschlag, T. and Lotus, J. (2020). Autologous Mesenchymal Stem Cell Treatment is Consistently Effective for the Treatment of Knee Osteoarthritis: The Results of a Systematic Review of Treatment and Comparison to a Placebo Group. Medicines, 7(8), p.42. doi:https://doi.org/10.3390/medicines7080042

A new study on cell therapy for cerebral palsy has, for the first time, directly compared the efficacy of two different treatments. Although more research is needed, the study’s results offer valuable insights for future clinical trials tackling this challenging disorder.

The challenges of cell therapy for cerebral palsy

Over the past two decades, cell therapy has emerged as a promising treatment for cerebral palsy. In particular, mononuclear cells from cord blood (UCB-MNCs) and mesenchymal stem cells from cord tissue (UCT-MSCs) have proven to be a safe and effective therapy. Many clinical trials have obtained results showing improvements in patients’ motor function through these cells’ anti-inflammatory, neuroprotective and regenerative properties.

However, efforts to pinpoint the best treatment method have been stymied by significant variations in trial parameters. Differences from study to study can include the type and severity of cerebral palsy, the age of the patients, the cell type, dose and delivery method, whether the study is open-label or blinded, and even the methods and time periods used to monitor improvement. Consequently, no two studies are alike, and results cannot be directly compared.[1][2]

Study methods and goal

The new study, conducted in Iran, is a pooled analysis of the results of two studies, one on UCB-MNTs and one on UCT-MSCs. Both studies were conducted in the same research centre; moreover, to aid in the comparison, the research methodology and other variables were kept the same as much as possible.[3][4][5]

The study patients were aged 4-14 and had spastic cerebral palsy with white matter lesions. After eligibility screening, 108 patients were randomly assigned to the two treatment arms or a control group. Treatment was done via intrathecal (into the spinal fluid) injection, with the control group receiving a sham procedure instead. Researchers then assessed patients’ motor function, quality of life, disability and spasticity after 1, 3, 6 and 12 months.

Both individual studies were double-blinded. Furthermore, all statistical analysis, both for individual studies and for the final pooled comparison, was performed by a blinded statistician.

The individual studies aimed to confirm that UCB-MNCs and UCT-MSCs are a safe and effective treatment for cerebral palsy. Following that, the pooled study analysis compared the effects of the UCB-MNC treatment to those of the UCT-MSC treatment across the study period.

Study results

Both the UCB-MNC treatment and the UCT-MSC treatment reported positive results over time when compared to the control group. Patients in both groups showed improvement in gross motor function and quality of life, as well as reduction in disability and spasticity.[4][5]

A graph showing outcomes of cell therapy for cerebral palsy.
Figure 1. A comparison graph of the two different treatment groups and the control group.[3]

As the figure above shows, researchers found that the UCB-MNC treatment group showed stronger improvements in motor function early on. However, both the UCB-MNC and UCT-MSC groups achieved the same level of improvement at 6 months post treatment. At twelve months after treatment, there was some gradual deterioration of the improvements; however, researchers noted that UCT-MSCs did seem to result in more sustainable changes, with patients seeing less deterioration compared to the UCB-MNC group.[3]

Due to this deterioration, researchers posit that repeated doses at regular intervals may be the best route to continued improvement.[1] They also suggest that future trials should investigate treatments combining both UCB-MNCs and UCT-MSCs, as it may prove more effective than individual ones.[3]

The future of medicine

Researchers stress that this study is only a start, and further comparative trials and research are needed. Although both UCB-MNCs and UCT-MSCs offer positive results, there is still no certainty on which cell type and source will prove most effective treatment. This is true not only for cerebral palsy, but also for other illnesses and diseases for which an effective treatment is still being sought.

This uncertainty highlights the importance of comprehensive stem cell banking. By storing as many stem cell sources as possible, you could equip your baby and family with the broadest range of options for future regenerative therapies.

To find out how you could preserve both cord blood and tissue, along with amnion and placenta, for your baby’s potential future use, fill in the form below to request your free guide.

References

[1] Parent’s Guide to Cord Blood. (2025). Cerebral Palsy Response to Cell Therapy as a Function of Time. https://parentsguidecordblood.org/en/news/cerebral-palsy-response-cell-therapy-function-time

[2] Qu, J., et al. (2022). Efficacy and safety of stem cell therapy in cerebral palsy: A systematic review and meta-analysis. Frontiers in Bioengineering and Biotechnology, 10. doi:https://doi.org/10.3389/fbioe.2022.1006845

[3] Nouri, M., et al. (2025). Cell-Based Therapy for Cerebral Palsy: A Puzzle in Progress. PubMed, 26(9), pp.569–574. doi:https://doi.org/10.22074/cellj.2024.2032098.1600

[4] Amanat, M., et al. (2021). Clinical and imaging outcomes after intrathecal injection of umbilical cord tissue mesenchymal stem cells in cerebral palsy: a randomized double-blind sham-controlled clinical trial. Stem Cell Research & Therapy, 12(1). doi:https://doi.org/10.1186/s13287-021-02513-4.

[5] Zarrabi, M., et al. (2022). The safety and efficacy of umbilical cord blood mononuclear cells in individuals with spastic cerebral palsy: a randomized double-blind sham-controlled clinical trial. BMC Neurology, 22(1). doi:https://doi.org/10.1186/s12883-022-02636-y.

After two planned transplants from unrelated donors fell through at the very last minute, Australian three-year-old Tommy Bacon is now in remission from a rare, dangerous form of leukaemia following a transplant of the stem cells from his baby sister’s cord blood.[1]

Tommy’s story

Tommy fell ill not long after his parents discovered they were expecting a second child – a baby girl. When he first started showing signs of illness, his parents and their doctor were not immediately alarmed. They assumed it was just a case of the usual germs picked up at daycare, which he had recently begun attending.

After he developed tonsillitis during a family trip to the UK in May 2023, however, his parents took him to the hospital and insisted he should be admitted. Tests eventually revealed that he had leukaemia. More specifically, he had a form of the disease called juvenile myelomonocytic leukaemia (JMML). What’s worse, he had one of the most high-risk, aggressive variants.

JMML is incredibly rare, with only 1-2 children out of one million being diagnosed with it every year[2]. A stem cell transplant is the only curative treatment option. Without a transplant, however, a child with an aggressive variant of JMML could survive for less than a year.[3]

The search for a donor

Australia’s donor registry had no donor compatible with Tommy, so his parents started a donor drive. Eventually, an international donor was found. Unfortunately, however, the donor pulled out of the donation process a week before Tommy was due to start his pre-transplant chemotherapy.

By then, Tommy’s baby sister’s due date was fast approaching, and his parents booked a date for the induction. On the day they were going into hospital, a phone call came that a second donor had been found for Tommy. Still, they decided to have their baby girl’s cord blood collected and stored with an Australian cord blood bank, just in case – although they knew it wasn’t guaranteed that she would be a match for Tommy.

Not long after baby Aria’s birth, the second donor, too, pulled out. The family was heartbroken.

A search for a third donor got underway, but Tommy didn’t have long. Because of this, a decision was made to prepare Tommy’s dad as a half-matched (haploidentical) donor. Such a transplant would not have been ideal, since haploidentical transplant recipients are at higher risk of developing post-transplant complications[4]. Absent a perfect match, though, this was Tommy’s last hope.

Then, the cord blood bank called: they had tested Aria’s cord blood, and she was a perfect match for Tommy.

Within a few weeks, Tommy received his transplant. Four months later, he was in remission.

The importance of family cord blood banking

Tommy’s story highlights the importance of family stem cell banking. By choosing to bank your baby’s cord blood stem cells, they will always be ready and waiting should your baby, or another family member, need them.

Their cord blood stem cells are guaranteed to be their own perfect genetic match. There is also a 25% chance they will be a perfect match for a sibling, and a 50% chance of a partial match. Moreover, they are always a partial match for both parents. This is why it can be so important to bank cord blood for every baby in the family, rather than just one.

Stem cells are being heavily investigated in the field of regenerative medicine to treat a wide variety of illnesses and injuries that are currently considered incurable. There are over 7500 clinical trials currently investigating both autologous (a patient’s own stem cells) and allogeneic (donor stem cells) uses of stem cells, in the hopes of developing new therapies.

These therapies aim to take advantage of the regenerative qualities of stem cells to aid in healing injuries such as spinal cord damage, heart disease, brain injury, arthritis and type 1 diabetes.

By saving your baby’s cord blood stem cells, you can give your baby and family a better chance of accessing these therapies, should they need one in the future.

“I would strongly recommend that if you’re thinking about getting cord blood collected, do it!” says Tommy’s mum, Kylie. “If it can change a life in such a huge way, why would you not?”[5]

To find out more about how cord blood banking works, and how it could safeguard your family’s health, fill in the form below to request a free welcome pack.

References

[1] Gannon, G. (2025). Aria was a miracle stem cell transplant donor for her brother. The Australian Women’s Weekly. https://www.womensweekly.com.au/news/real-life/stem-cell-transplant-donor/

[2] St. Jude Care & Treatment. Juvenile Myelomonocytic Leukemia Treatment. https://www.stjude.org/care-treatment/treatment/childhood-cancer/leukemia-lymphoma/juvenile-myelomonocytic-leukemia-jmml.html

[3] Lls.org. JMML treatment outcomes. https://www.lls.org/leukemia/juvenile-myelomonocytic-leukemia/treatment/treatment-outcomes

[4] Anthony Nolan. Haploidentical stem cell transplants. https://www.anthonynolan.org/patients-and-families/understanding-stem-cell-transplants/haploidentical-stem-cell-transplants

[5] The Project (2024). Baby Girl’s Stem Cells Save Big Brother’s Life. YouTube. https://www.youtube.com/watch?v=JHwWgqEu_Hs

A new study on the effectiveness of treatment for spinal cord injuries indicates that a combination therapy, including epidural electrical stimulation (EES) as well as neural stem cells and umbilical cord stem cells, could offer better results than any one treatment on its own.

What is spinal cord injury?

Spinal cord injury is a debilitating, disabling neurological condition resulting from damage to the spinal cord, or to the nerves at the end of the spinal canal. This is most frequently caused by traumatic occurrences such as vehicle accidents, falls, sport injuries or violence, but may also be caused by tumours, infections or degenerative conditions.

Spinal cord injury causes partial or complete loss of sensation and function below the level of the injury, commonly resulting in paralysis (paraplegia or quadriplegia), loss of bladder and bowel control, and breathing issues.[1]

What therapies are currently available?

At present, there are no known therapies that would reverse the initial injury and return an injured spinal cord to full function.[2] Current treatments for spinal cord injury focus on limiting what is called the secondary injury cascade, ideally preventing further damage and thus further loss of feeling and motor function.[3] These methods include surgery, medication, physical therapy and rehabilitation, and assistive devices such as braces or wheelchairs.

What is epidural electrical stimulation for spinal cord injury?

In epidural electrical stimulation, an array of electrodes is implanted along the spinal cord through a surgical procedure called a laminectomy. The stimulation provided by the electrical pulses generated by these electrodes could help in the recovery of functionality following spinal cord injury, improving patients’ ability to walk and stand as well as aiding with bladder and bowel control.[3] [4]

How could stem cells help?

By leveraging the regenerative properties of stem cells, it is hoped that a stem cell treatment could repair and regenerate damaged spinal cord tissue.

This could mean protecting what neurons remain intact, repairing the protective myelin sheath on damaged ones, thus restoring their ability to conduct nerve signals, and replacing lost ones. Stem cells also have the ability to modulate the body’s immune response, and could reduce inflammation and mitigate the secondary damage that follows the initial injury.[5]

There are currently several clinical trials studying the application of stem cells for spinal cord injury. Although more research is needed, results so far are promising, including the high-profile case study of a man who has regained the ability to walk.[6]

What has the new study found?

Researchers at Xi’an Jiaotong University, China, set out to test the effectiveness of a therapy combining both epidural electrical stimulation and stem cell injections, using a mouse model of spinal cord injury.[7]

The study involved four different groups of mice: a group which was treated with EES alone, a group which was treated with a mix of mouse neural stem cells (NSCs) as well as human umbilical cord mesenchymal stem cells (hUCMSCs), a group which received both treatments and a control group in which the spinal cord injury was left untreated. The mice in all groups were monitored and assessed for a period of two months.

Following the injury, all mice had complete loss of function in their hind limbs; a week post-injury, mice in the treatment groups underwent their respective treatments. At the end of the monitoring period, mice in the control group were still unable to support themselves on their hind limbs. Conversely, some mice in the EES group were able to achieve paw standing; mice in the hUCMSC group also achieved this milestone, in a more frequent and sustained manner. Mice in the combined treatment group not only achieved paw standing, but also showed improved motor coordination. Swimming and gait analysis tests corroborated these findings, with the hUCMSC group doing better than the EES group, both doing better than the control and the combined treatment group doing best of all.

What’s next?

Both EES and stem cell transplants are currently the subject of clinical trials to test their effectiveness in the treatment of spinal cord injury. As the new study shows, it is entirely possible that the best treatment will be a combination of both, but more research is required on each individual treatment before the combination therapy can be tested in humans.

What is undeniable is that the number of studies and clinical trials examining the regenerative power of stem cells and their potential for treating currently incurable diseases and injuries continues to grow.

To find out more about what stem cells could do, and how you could preserve a rich source of them for your baby so that they could gain access to future regenerative treatments, fill in the form below to request your free welcome pack.

References

[1] University Hospitals Sussex NHS Foundation Trust. (2023). Spinal cord injury. https://www.uhsussex.nhs.uk/sussex-trauma-network/rehabilitation/conditions/spinal-cord-injury/

[2] NIH (2016). What are the treatments for spinal cord injury (SCI)? https://www.nichd.nih.gov/health/topics/spinalinjury/conditioninfo/treatments

[3] Dorrian, R.M., Berryman, C.F., Lauto, A. and Leonard, A.V. (2023). Electrical stimulation for the treatment of spinal cord injuries: A review of the cellular and molecular mechanisms that drive functional improvements. Frontiers in Cellular Neuroscience, 17. doi:https://doi.org/10.3389/fncel.2023.1095259

[4] Royal National Orthopaedic Hospital. (2024). New research offers quality of life hope for many paralysed after spinal cord injuries. https://www.rnoh.nhs.uk/news/new-research-offers-quality-life-hope-many-paralysed-after-spinal-cord-injuries

[5] Zeng, C.-W. (2023). Advancing Spinal Cord Injury Treatment through Stem Cell Therapy: A Comprehensive Review of Cell Types, Challenges, and Emerging Technologies in Regenerative Medicine. International Journal of Molecular Sciences, 24(18), p.14349. doi:https://doi.org/10.3390/ijms241814349

[6] Stem Cells Help Injured Surfer to Walk Again.

[7] Mu, Z., Qin, J., Zhou, X. et al. (2024.) Synergistic effects of human umbilical cord mesenchymal stem cells/neural stem cells and epidural electrical stimulation on spinal cord injury rehabilitation. Sci Rep 14, 26090. doi:https://doi.org/10.1038/s41598-024-75754-x

Today marks World Cord Blood Day—a day dedicated to highlighting the medical breakthrough that began 36 years ago, in 1988, with a single transplant. That cord blood transplant opened the door to a new era in medicine. Since then, stem cells from cord blood have transformed countless lives, offering hope to those battling over 80 serious diseases including leukaemia, sickle cell anaemia, lymphoma, and more.

Once dismissed as medical waste, cord blood’s potential to heal is becoming more and more recognised, with over 60,000 transplants since 1988. Today, stem cells are the key players in regenerative medicine, studied in thousands of labs and clinical trials around the world. Scientists are exploring their potential to treat conditions as varied as bone fractures, spinal cord injuries, arthritis, and even Crohn’s disease.

Yet, despite this remarkable potential, too often cord blood is still discarded after birth, lost forever. This is why World Cord Blood Day was created: to shine a light on the therapeutic power of cord blood and to encourage more families to preserve this incredible resource for the future.

As research has continued though, it is no longer just cord blood that holds such promise, but also the cord tissue and placental stem cells which hold immense potential in regenerative medicine.

Cord blood news

A lot has happened in the cord blood field since last year. Here are some news you may want to catch up on if you’ve missed them…

Researchers in China found that umbilical cord stem cells could aid in healing intestinal ulcers caused by Crohn’s disease.
A study found that umbilical cord stem cell therapy could improve ovarian function, a potentially important step towards overcoming the ‘biological clock’ of waning fertility.
A five-year-old boy with a rare genetic disorder received a life-changing transplant of cord blood stem cells that had been stored by the NHS for 15 years.
A clinical trial investigating the use of umbilical cord stem cells to treat chronic heart failure is underway.
Umbilical cord stem cells are being researched as a treatment for acute liver failure.
A new therapy could improve treatment outcomes for patients with Krabbe disease following a stem cell transplant.
A recent report by the Parents’ Guide to Cord Blood Foundation highlighted continued interest in treatments based on umbilical cord stem cells in the medical research field.
Haplo cord blood transplants, combining stem cells from a cord blood unit and a bone marrow donor, improve survival rates post-transplant and reduce the risk of graft-versus-host disease.
Umbilical cord exosomes could be used in developing life-changing therapies for a variety of conditions.

How can I bank my baby’s cord blood?

If you want to save this precious resource rather than having it thrown away, both public and private banking are available options.

Public banking means the cord blood stem cells are stored in a cord bank that makes them accessible to anyone who might need them. The NHS public cord bank accepts donations if you are giving birth at one of three hospitals; the Anthony Nolan charity public cord bank accepts donations from a further five hospitals.

Alternatively, you can choose to bank your baby’s cord blood privately, storing the stem cells solely for your family’s own use. They will be ready and waiting should your baby ever need them for treatment, and could be useful for family members, too – there is a 25% chance they would be an exact match for a sibling, or 75% chance of a partial match. They are also a guaranteed partial match for parents.

Everything about our services has been tailored to provide the maximum potential benefit to parents who store their baby’s stem cells with us, such as our advanced processing technology that retains up to three times more stem cells than industry-standard processing methods, meaning full compatibility with delayed and optimal cord clamping. We are also the only UK cord blood bank to offer placenta and amnion storage.

To learn more about cord blood banking and why most UK parents choose to store with us, simply fill in the form below for a free welcome pack.

Older Posts

Stem Cell Blog

Key details

Understanding Lesch-Nyhan syndrome: This rare, recessive condition linked to the X chromosome primarily affects boys, and is characterised by a deficiency of the HPRT enzyme, leading to severe motor dysfunction, uric acid overproduction, and compulsive self-injury.

Role of HPRT: Used by the body to process and recycle purines: organic compounds that are a type of building block of DNA and RNA.

Promising transplant results: A recent case study demonstrated that a cord blood transplant (from a 5/6 HLA-matched donor) successfully restored HPRT1 protein levels to normal range and prevented the onset of self-injury behaviour.

Cord blood efficacy: Umbilical cord blood is often considered the preferred stem cell source for treating inborn errors of metabolism because it supports successful engraftment and is safer than other sources when the donor and patient are not a perfect genetic match.

What is Lesch-Nyhan syndrome?

Urate crystals: Forming in the urine due to the build-up of uric acid, these orange-coloured crystals can deposit in the diapers of babies with this condition. They are often the first symptom to appear.

Gout: The accumulation of uric acid in the joints eventually leads to recurring pain and swelling, similar to what happens in adults with gout. These episodes generally begin in the late teens to early adulthood, and can become progressively more frequent over time.

Intellectual disability: Some cognitive impairment is typically present, although it is hard to gauge accurately due to the motor issues and dysarthria.

Behavioural issues: Patients with Lesch-Nyhan syndrome may also have episodes in which they act aggressive and attempt to injure others, or become verbally abusive.[3][4]

What causes Lesch-Nyhan syndrome?

Is there a cure for Lesch-Nyhan syndrome? What treatment is available?

There is no cure for Lesch-Nyhan syndrome. Treatment primarily aims to treat symptoms, and is generally handled by a team of specialists rather than a single treating physician.[2][3]

Treatment can include:

Allopurinol, a medication normally used for gout, to reduce the amounts of uric acid present in the body and control the symptoms caused by it.

Procedures to break down kidney or bladder stones.

Physical splints or restraints to prevent self-injury, including hip, chest and elbow restraints as well as a mouth guard.

Supportive equipment such as a wheelchair.[2][3]

No standard treatment is available for the neurological and behavioural symptoms of Lesch-Nyhan syndrome, although a variety of medication may help ameliorate some of them.[2][3][5][6]

Could a cord blood transplant help with Lesch-Nyhan syndrome?

If performed early enough, a cord blood transplant could potentially slow or halt the progression of Lesch-Nyhan syndrome. A recently published case study, detailing the results of a cord blood transplant for Lesch-Nyhan syndrome, provides the strongest evidence to date for this.[7]

As the condition was progressing and the patient did not have a matched sibling donor, doctors made the decision to proceed with a cord blood transplant from a partially matched (5/6 HLA) donor, performed at 14 months old.

The outcome of the transplant was positive:

Successful engraftment: Complete donor chimerism, meaning over 95% of the patient’s new blood cells were of donor origin, was achieved on day 32 post-transplant.

HPRT1 protein levels: Prior to the transplant, the patient’s HPRT1 protein levels were low (40.9 pg/ml) compared to those of his father and mother (98.8 pg/ml and 78.9 pg/ml respectively). Post-transplant, levels rose to 91.1 pg/ml by day 32 and remained normal (97.0 pg/ml) at day 126.

Neurological improvement: The patient’s dystonia and spasticity decreased, opisthotonos became more infrequent, and he had improved facial expressions, increased social interaction and developmental progress. At 36 months of age, he still showed no sign of any self-injury behaviour.[7]

This case shows a promising result, but research is still in the very early stages. Including this case, only three haematopoietic stem cell transplants (HSCT) have been performed in children with Lesch-Nyhan syndrome before the onset of the self-injury compulsion:

What is known for certain is:

HSCT has been successfully used to treat other conditions in the inborn error of metabolism category, and is the standard of care for some of them, such as Hurler syndrome [8] and Krabbe disease.[9]

Cord blood is generally the preferred stem cell source for such transplants, as it increases the likelihood of full donor chimerism and is safer to use when the donor and the patient aren’t a perfect genetic match.[7][8][10]

Should a newborn screening targeted at Lesch-Nyhan syndrome be implemented, it would mean the condition could be identified before symptoms develop. The authors of this case study speculate that early transplant could prevent irreversible neuronal loss and preserve neurological potential.[7]

The importance of family banking

Storing your baby’s cord blood privately gives them access to their own perfectly matched stem cells, and a 75% chance they could be a match for a sibling who may need a transplant; comprising a 25% chance of a perfect match, and 50% chance of a partial match.

To learn more about banking your baby’s cord blood, as well as about other sources of stem cells that can only be collected immediately after birth, fill in the form below to request your free guide.

References

What’s more, thousands of clinical trials all over the world are investigating the potential of cord blood stem cells to treat conditions which are currently incurable, including cerebral palsy, heart failure, spinal cord injuries and Crohn’s disease, to name but a few.

Cord blood news

Research in the regenerative medicine field continues to advance our knowledge of what’s possible using cord blood. In case you’ve missed them, here are some news articles from the past year:

Early trials in mice have found that a combination therapy involving umbilical cord stem cells and electrical stimulation could be key to treating spinal cord injuries.

An Australian toddler is in remission from a dangerous form of leukaemia following a transplant of his baby sister’s cord blood.

A leukaemia treatment derived from umbilical cord blood has gained FDA approval to begin clinical trials.

A treatment based on umbilical cord blood stem cells has been found to offer superior results for the treatment of osteoarthritis.

A novel therapy for lymphoma that is highly resistant to treatment, based on umbilical cord blood cells, published positive results.

Cord blood therapy has been shown to be an effective treatment for cerebral palsy, improving motor skills.

A six year old girl became the first child in Australia to receive her own cord blood as treatment for cerebral palsy.

Banking your baby’s cord blood

Both public and private banks are an option if you want to save your baby’s cord blood rather than having it discarded.

Zara’s story

What does cord blood treatment for cerebral palsy entail?

Why is this treatment not more widely available?

Both of those routes to treatment are likely to require a child having access to either their own banked cord blood, or a sibling’s. This is because while there are public cord blood banks, the cord blood stored there may be available for use only for approved treatments or clinical trials.

References

What is cerebral palsy?

Cerebral palsy is a group of motor disorders that affect movement, posture and coordination. It’s caused by damage to the brain while it is still developing. This happens most often before birth, but sometimes during or immediately after.

The severity of the condition can vary greatly. Symptoms include lack of balance and muscle coordination, muscles that are either too stiff or too floppy, exaggerated reflexes, and issues with walking, eating and speech.[1][2]

There is currently no cure for cerebral palsy. Available treatment includes physiotherapy, occupational therapy, medication and sometimes surgery. This is aimed at helping people with the condition to be as healthy and independent as possible.[3]

What did the analysis find?

What does this mean for the treatment of cerebral palsy?

References

What are natural killer cells?

NK cells are a specific type of white blood cell, a part of the immune system which targets and destroys infected and cancerous cells. These cells patrol the body, determining which cells should be destroyed based on whether they receive the correct signals from them or not.[1]

What does the new therapy entail?

Researchers first isolated NK cells from cord blood and then activated them using a combination of cytokines.[8] Cytokines are proteins which the immune system uses to send signals;[11] using them to activate NK cells stimulates their cancer-fighting abilities.[12]

Then, the antibody AFM13 was added to the NK cells, enabling them to target the CD30 protein. This protein is found on the surface of cancerous cells in specific types of lymphoma, primarily in Hodgkin lymphoma but also other varieties.[8]

Following chemotherapy, doctors infused a dose of the treated NK cells into patients. This was then followed by three doses of the AFM13 antibody, administered once a week.

Why is this therapy so important?

Eleven patients remained in complete remission for at least 14 months, with this lasting, for some, up to 40 months after receiving the therapy. Five patients remained in complete remission without any further treatment, and six went on to receive a stem cell transplant.

This trial was a small-scale one, primarily aimed at confirming the safety of the treatment and determining an optimal dose. Larger trials are therefore needed before this therapy can become available to patients.

References

What is osteoarthritis?

Treatments for knee osteoarthritis

The current standard of care for knee osteoarthritis includes a range of treatments that aim to manage pain, reduce stiffness and improve range of motion. They include both surgical and non-surgical options.

A new therapeutic option

On the strength of the results from this and other studies, the FDA granted approval to proceed directly to a phase 3 clinical trial in the United States. A phase 3 trial is also underway in Japan.[4]

The regenerative power of cord blood stem cells

The strong potential of UCB-MSCs for the treatment of osteoarthritis is well-documented, with several recent studies highlighting their positive effects.[15][16][17]

To discover more about cord blood stem cells, and how you could preserve them for your baby for potential future treatment, fill in the form below to request your free guide to cord blood banking.

References

A new study on cell therapy for cerebral palsy has, for the first time, directly compared the efficacy of two different treatments. Although more research is needed, the study’s results offer valuable insights for future clinical trials tackling this challenging disorder.