Stem Cell Blog

Употребата на матичните клетки од папочна врвца рапидно се зголемува. Пред 10 години крвта од папочна врвца можеше да лекува околу 40 состојби, но денес таа бројка е над 80. Со нетрпение очекуваме нови терапии за болести и нарушувања како што се дијабет, аутизам и мозочен удар, можете да бидете во тек со најновите случувања во регенеративната медицина на нашиот блог за матични клетки.



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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


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A CAR-T cell therapy for acute lymphoblastic leukaemia[1] has recently been approved by the FDA for use in clinical trials in the United States. The therapy is based on cells derived from allogeneic (donor) umbilical cord blood; it is the very first such treatment to gain FDA approval.[2]

What is acute lymphoblastic leukaemia? 

Acute lymphoblastic leukaemia, or ALL, is a type of blood cancer that starts in the bone marrow. It affects white blood cells (lymphocytes).

In ALL, the bone marrow makes too many of the cells from which lymphocytes derive (lymphoblasts). These lymphoblasts grow and divide quickly; however, they are not fully developed and cannot turn into healthy lymphocytes.

As a result, lymphoblasts build up in the bone marrow and stop all types of healthy blood cells from developing. They may also spread into other parts of the body, including lymph nodes, the liver and spleen, and the brain and spinal cord.[3]

The lack of healthy white blood cells means ALL patients are at a higher risk of infection. ALL can also cause anaemia through the lack of red blood cells, as well as bleeding problems due to lower platelet numbers.[4]

Leukaemia is the most common type of cancer which affects children; of all types of leukaemia, ALL is the most commonly diagnosed in children.[4] It can also affect adults, although this is less common.[3]

What is CAR-T cell therapy?

CAR-T therapy is a type of treatment which uses T cells to fight cancerous cells. The T cells are genetically modified using a viral vector to add a specific protein on their surface, called a chimeric antigen receptor (CAR). This addition makes them able to recognise cancerous cells and destroy them.[5]

In standard CAR-T therapy, the T cells used are the patient’s own, extracted from the blood through a process called apheresis. After extraction, they are taken to a specialised lab to be modified. However, this process is very time-consuming, typically requiring three to five weeks for manufacturing and quality checking. This can be a problem in the case of ALL, as the disease is fast-progressing and could worsen before the CAR-T cells are ready to be reinfused.[6]

What makes this new therapy different?

The new therapy uses T cells derived from allogeneic (someone else’s) cord blood as a starting point instead. These cells are in a more naïve state, meaning they have a higher ability for self-renewal and lower immunogenicity. This means a lower chance to provoke an immune response when used in treatment. As a result, they can be developed into an ‘off-the-shelf’ CAR-T therapy that is ready when needed and does not require a long wait.[2] Following positive results from investigator-initiated trials,[1] the FDA has now approved the therapy for clinical trials in the United States.[2] Furthermore, researchers in China are also testing the therapy in a clinical trial for the treatment of lupus.[7][8]

A similar therapy, also based on T cells derived from donor cord blood, is being developed at the Fondazione Tettamanti research centre in Monza, Italy. Using a special DNA sequence called a transposon, scientists turn the T cells into CAR-enabled cytokine-induced killer cells (CARCIK), making them able to destroy cancer cells. An initial set of clinical trials aimed at determining the feasibility and safety of this treatment showed positive results. Researchers are now continuing to test the therapy in clinical trials for ALL as well as non-Hodgkin lymphoma, and in vitro and animal models for acute myeloid leukaemia.[9]

The benefits of using cord blood for treatments

Being able to derive T cells for CAR treatment from cord blood could pave the way towards more targeted therapies in the future, says Sarah Tettamanti, a researcher at Fondazione Tettamanti. These therapies could be developed from banked cord blood compatible with each specific patient.[9]

For treatments such as these, it could be essential to have ready access to a compatible donor match. Public cord blood banks are available in the UK and worldwide. However, because they rely on donated cords from only a small number of hospitals[10], there is no guarantee that a match will be found when needed.

Conversely, if you store your baby’s cord blood privately at birth, they will always have access to their own perfect match if they ever need it. There is also a 25% chance for the cord blood to be a perfect match for a sibling and 50% chance of a partial match. It is guaranteed to always be a partial match for parents.

It could also prove to be a match for a more distant relative – which thanks to cord blood cells’ naïve state and lower immunogenicity, could still be enough for treatment.

To learn more about the potential uses of cord blood in treatments and how you could preserve this precious resource for your baby and family, fill in the form below and request our free guide.

References

[1] LemanBio (2023). Leman Biotech and Ucello Therapeutics enter into Comprehensive Strategic Collaboration to Advance Universal CAR-T Development. https://www.lemanbio.com/en/news/197.html

[2] BusinessWire. (2025). VectorBuilder Powers FDA IND Approval of World’s First Umbilical Cord Blood-Derived Allogeneic CAR-T Product. https://www.businesswire.com/news/home/20250129313138/en/VectorBuilder-Powers-FDA-IND-Approval-of-World%E2%80%99s-First-Umbilical-Cord-Blood-Derived-Allogeneic-CAR-T-Product

[3] Cancer Research UK (2021). What Is Acute Lymphoblastic Leukaemia (ALL)? https://www.cancerresearchuk.org/about-cancer/acute-lymphoblastic-leukaemia-all/about

[4] Cancer Research UK (2021). About childhood acute lymphoblastic leukaemia (ALL). https://www.cancerresearchuk.org/about-cancer/childrens-cancer/acute-lymphoblastic-leukaemia/about

[5] Cancer Research UK (2021). CAR T-cell therapy. https://www.cancerresearchuk.org/about-cancer/treatment/immunotherapy/types/CAR-T-cell-therapy

[6] Leukemia & Lymphoma Society. Chimeric Antigen Receptor (CAR) T-Cell Therapy. https://www.lls.org/treatment/types-treatment/immunotherapy/chimeric-antigen-receptor-car-t-cell-therapy

[7] https://mp.weixin.qq.com/s/t7tQTUwpl5vmr78cSRXGhQ

[8] https://mp.weixin.qq.com/s/rVx9bVuU3-2pKio_LbO7ZQ

[9] Cerati, F. (2025). Dal cordone ombelicale le cellule modificate per trattare la leucemia. Il Sole 24 Ore. https://www.ilsole24ore.com/art/dal-cordone-ombelicale-cellule-modificate-trattare-leucemia-AGD2eidC

[10] Cord Blood Bank – NHS Blood and Transplant. Where can you donate? https://www.nhsbt.nhs.uk/cord-blood-bank/donate/where-can-you-donate/


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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


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This year’s World Sickle Cell Day is on the 19th June, a day to recognise and raise awareness of the disease, the millions around the world who suffer from it, and ongoing research into how treatment is advancing.

With this year’s theme being ‘we are STRONGER TOGETHER’, we thought it would be a good opportunity to highlight how cord blood transplants between siblings have emerged as a viable treatment alternative for those suffering from the condition.

Making use of sibling cord blood necessitates having it in storage and so in celebration of World Sickle Cell day, we wanted to highlight how cord blood banking could provide a lifeline for those suffering from the disease, as well as how storing cord blood for your baby could help safeguard the health of your family.

What is sickle cell disease?

Sickle cell disease is the name for a group of inherited blood disorders that are passed down from parents to children.

Particularly common amongst those who have African or Caribbean heritage, sickle cell disease inhibits the haemoglobin in red blood cells from carrying oxygen.

This can lead to the red blood cells in those with the condition to become misshapen, inflexible and liable to sticking together and blocking blood flow, causing tremendous pain – known as sickle cell crises – in addition to an increased risk of stroke, lung problems, eye problems and infection.

The disease gets its name from the shape of the red blood cells affected by the condition: crescent or ‘sickle’ shaped rather than discoid. [1]

What treatments are available for sickle cell disease?

Currently, treatments for sickle cell disease focus primarily on the alleviation of symptoms.

While there have been significant steps forward in recent years, including the development of the Casgevy therapy which utilises genetically engineered bone marrow stem cells from the patients themselves in lieu of a donor transplant, medicines like antibiotics and painkillers remain the most prevalent way of combating sickle cell.

There is only one known cure for sickle cell disease: a stem cell or bone marrow transplant. In these treatments, healthy red blood cells are produced by the donated stem cells, replacing the ones that are affected by sickle cell. [2] 

However, difficulty in locating an unrelated donor match, in addition to the risks posed by graft-versus-host disease, hinders the ready availability of transplantation as a treatment option.

The benefits of cord blood banking for sickle cell disease

Within the last decade, sibling cord blood transplants have emerged as a viable alternative to bone marrow transplants as a treatment option for sickle cell disease.

A comprehensive study in 2017 following the success rates of sibling cord blood transplants over a period of 20 years found that of the 28 patients with sickle cell who received cord blood from a sibling, all but one are both alive and free from sickle cell disease. [3]

With a reduced risk of graft-versus-host disease, in addition to a 25% chance of a perfect match and a 50% chance of a partial match, using the stem cells from a sibling’s umbilical cord blood alleviates many of the current obstacles to obtaining a transplant to treat sickle cell disease.

Moreover, because of the way sickle cell is passed down between parents and children, if one child is born with sickle cell disease then there’s a 75% chance that a subsequent child will not have the disease, making a cord blood transplant between siblings possible. [4]

Should I store my baby’s cord blood?

Underlying the breakthroughs in treating sickle cell through a sibling cord blood transplant is one crucial detail: whether or not that sibling has cord blood samples in storage. There’s only one opportunity to save their cord blood: in the minutes after they’re born.

Without cord blood samples in storage, availability to stem cell transplant treatments for sickle cell, and other blood or inherited conditions, becomes more difficult as it’s harder to find a suitable match.

Ensuring that you save cord blood for every child maximises opportunities for treatment, particularly in instances where a sibling transplant could provide a cure for conditions requiring an HLA match, like sickle cell disease.

Additionally, with the emergence of the aforementioned Casgevy therapy and ongoing trials exploring the possibility for autologous stem cell treatments for sickle cell disease, saving your baby’s stem cells means that they have improved access to cutting edge therapies using their own cord blood samples. [5]

Storing cord blood for every child is the only way to ensure that they have improved access to the benefits of cord blood banking.

If you want to learn more about how storing cord blood for your baby could provide protection for their health and the health of their siblings, fill out the form below for a free Welcome Pack.

References

[1] (2024, April 22). What Is Sickle Cell Disease? National Heart, Lung and Blood Institute. https://www.nhlbi.nih.gov/health/sickle-cell-disease

[2] (2022, November 30). Overview: Sickle Cell Disease. NHS. https://www.nhs.uk/conditions/sickle-cell-disease/

[3] Rafii, Hanadi et al. “Family cord blood banking for sickle cell disease: a twenty-year experience in two dedicated public cord blood banks.” Haematologica vol. 102,6 (2017): 976-983. doi:10.3324/haematol.2016.163055

[4] Autosomal Recessive: Cystic Fibrosis, Sickle Cell Anemia, Tay-Sachs Disease. University of Rochester Medical Center. https://www.urmc.rochester.edu/encyclopedia/content.aspx?ContentID=P02142&ContentTypeID=90

[5] (2024, March 19). Clinical Study of BRL-101 in the Treatment of Sickle Cell Disease. ClinicalTrials.gov. https://clinicaltrials.gov/study/NCT06287099