Blog - Банка за матични клетки

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.

A recent study published in the Lancet journal has shown that stem cells have been used to bring about significant improvements in vision in what’s being heralded as a world first.

The stem cell treatment focused on the repair of the cornea which becomes damaged in limbal stem-cell deficiency (LSCD), a disease that can lead to blindness.

It’s hoped that these findings could have a huge impact on how those with the disease are treated and that it could underpin future therapy options for those suffering from sight loss. [1]

What is LSCD?

Limbal stem-cell deficiency (or LSCD) is a disease characterised by the loss or deficiency of stem cells in the limbus, the border between the cornea and the sclera. These stem cells are crucial to the maintenance and repair of the limbus, in addition to ensuring the continuation of its barrier function.

Problems with limbal stem cells can lead to the epithelial breakdown of the cornea resulting in inflammation, scarring and potential vision loss.

LSCD has a variety of causes ranging from genetics to acquired causes like inflammation, infection, and trauma and injury. Management of the disease differs depending on the stage of its progression.

At the early stage, managing symptoms can be sufficient to alleviate its impact on quality of life. However, more progressed instances of LSCD require surgery which usually means transplants from key parts of donor eyes. [2]

What did the stem cell study involve?

The study focused on four patients, two men and two women, aged between 39 and 72 who had all been diagnosed with LCSD in both eyes.

Researchers then derived induced pluripotent stem cells (iPSCs) from donated cord blood and used to fabricate corneal epithelial stem-progenitor cells. These were then cultured and transformed into a thin sheet, iPSC-derived corneal epithelial cell sheets (iCEPS).

After removing a layer of scar tissue covering the cornea in one eye in each of the patients, the iCEPS sheet was transplanted on top and covered with a contact lens to protect the graft.

Patients were then monitored continuously to determine safety outcomes for a period of two years. [3]

What were the results of the stem cell study?

Throughout the whole of the two year safety observation period no serious adverse events occurred. The transplant was accepted by the patients without rejection and without tumour formation. In fact, researchers reported that two of the patients even forwent immunosuppressant drugs.

Following the transplant, all four patients saw immediate improvements in their vision during the first year and three out of four patients experienced sustained improvements in their vision and quality of life beyond one year.

These results are hugely encouraging and the research team, based at Osaka University in Japan, hope to move to a larger scale clinical trial to verify their promising findings. [4]

What does this mean for cord blood banking?

As the results of this trial show, stem cells have huge regenerative potential and are at the forefront of medicine, with researchers still coming to terms with the breadth of their applications.

While this study focused on the use of induced pluripotent stem cells derived from cord blood, other types of stem cells can be found in the umbilical cord and placenta, such as haematopoietic and mesenchymal stem cells, that similarly have enormous potential in the field of regenerative medicine.

Unfortunately, the umbilical cord and placenta are often regarded as mere medical waste, meaning that these stem cells get thrown away. But by storing these stem cells in a process called cord blood banking, you can ensure that your baby always has their own stem cells available for use in future therapies.

If you or someone you know is expecting and wants to know more about the power of stem cells and how they can be stored for future use, fill out the form below to request a free Welcome Pack.

References

[1] Soma, Takeshi et al. (2024). ‘Induced pluripotent stem-cell-derived corneal epithelium for transplant surgery: a single-arm, open-label, first-in-human interventional study in Japan’. The Lancet. doi: 10.1016/S0140-6736(24)01764-1. https://doi.org/10.1016/S0140-6736(24)01764-1

[2] Karakus, S. (2024, August 2). Limbal Stem Cell Deficiency. American Academy of Opthalmology. https://eyewiki.org/Limbal_Stem_Cell_Deficiency

[3] Mallapaty, S. (2024, November 8). World-first stem-cell treatment restores vision in people. Nature. https://www.nature.com/articles/d41586-024-03656-z

[4] Mahindra, A. S. (2024, November 10). Biggest Blindness Breakthrough: Japan Performs World’s First Stem Cell-treatment To Restore Vision. Times Now. https://www.timesnownews.com/health/biggest-blindness-breakthrough-japan-performs-worlds-first-stem-cell-treatment-to-restore-vision-article-115144553

A recent story published by The New York Times has highlighted the life-changing potential placenta holds for the treatment of severe burns and wounds.

The story follows Marcella Townsend, who was left unrecognisable by second and third degree burns she suffered after a propane explosion at her mother’s house in Georgia.

With few treatment options available, Marcella underwent a radical, cutting-edge procedure: a human placenta graft.

Incredibly, Marcella’s face now looks nearly identical to how it did before the explosion. [1]

What is a human placenta graft?

A placenta graft involves the application of amniotic membrane – the thin, translucent inner part of the placenta surrounding the foetus in the womb – to an area affected by burns, wounds, or injuries.

Unlike skin grafts using animals or cadavers, placental tissue doesn’t trigger the immune system, meaning it isn’t rejected by the body – a byproduct of the immunological privilege it has between mother and child during pregnancy.

It also stimulates rapid cell regrowth, which can help the body heal with minimal scarring. [2]

What makes the placenta so special?

The placenta provides crucial nutrients and protection to babies during pregnancy, and is filled with proteins and growth factors that make it extremely useful for healing injuries. [3]

Since the early 20th century, the amniotic membrane from placentas has been used in topical treatments for burns, wounds, and ulcers. It’s even been used to treat eye conditions. [4]

And yet, despite their potential, most placentas are discarded as medical waste.

Today in the U.S. only publicly stored donated placentas from elective C-sections are used. [1]

There are also limitations on how they can be used. For instance, amniotic membrane grafts are only sanctioned so long as there is minimal manipulation, meaning that their application is contained to internal and external bandages, along with cases of chronic wounds that take longer than usual to heal, or don’t heal at all.

In the latter context, a placenta graft again proved to be life-changing.

As cited by The New York Times, an 83 year old woman had an infected, chronic wound on her leg that wouldn’t heal after surgery. Having exhausted treatment options – including the use of larvae to eat the infected areas – doctors turned to placenta-derived skin grafts to close the wound.

The woman’s leg healed completely.

While the medical potential of the placenta seems boundless – they have been found to heal burns, wounds, and even restore vision in some cases – their lack of availability, along with the cost of associated procedures, means they remain vastly underused in the medical field. [1]

Why aren’t placentas more widely used?

While it’s clear that placentas have huge potential in a wide range of medical contexts, current therapeutic applications don’t come cheap. It is estimated that the cost of a placental graft ranges between $200 and $3,000 per square centimetre of amniotic membrane in the U.S. [1]

In the UK, amniotic membrane grafts are similarly pricey, with a document from the National Institute for Health and Care Excellence (NICE) showing a 2cm x 3cm graft costing around £1,000 for a single use. [5]

With the street worth of placentas estimated to be around $50,000 – a figure that’s expected to double or even triple over the course of the next decade [6] – it’s no wonder some mothers are looking to privately store their own placentas.

What is private placenta storage?

Private placenta storage is the process by which mothers can choose to store their placenta so that it’s available for their child to use in future therapies, should they need it.

There are hundreds of clinical trials ongoing that are exploring the uses of placenta in treatments for conditions ranging from stroke to diabetes, Crohn’s and osteoarthritis. By saving the placenta, the powerful cells and growth hormones it contains are preserved for future use. [7] [8] [9] [10]

Private placenta storage can be considered to be part of what’s known as cord blood banking – the process by which the perinatal cells and tissues that naturally occur in the umbilical cord and placenta are cryogenically frozen.

Some of these perinatal cells are otherwise known as stem cells, and have many therapeutic benefits. Some are even being used today in over 80 approved treatments.

Saving the umbilical cord and placenta for your baby gives them the best chance to access future therapies using stem cells and other perinatal cells and tissues.

This is because cells from the umbilical cord and placenta are baby’s own perfect match, meaning the risk of rejection in a procedure like a transplant is minimal.

For people like Marcella Townsend, the life-changing potential of the placenta is clear. By storing for your baby, you could ensure that they too have access to future therapies utilising the power of the placenta.

References

[1] Morgan, K. (2024, October 8). Her Face Was Unrecognizable After an Explosion. A Placenta Restored It. The New York Times. https://www.nytimes.com/2024/10/08/well/placenta-donations-burns-wounds.html

[2] Glat, Paul MD et al. Placental Membrane Provides Improved Healing Efficacy and Lower Cost Versus a Tissue-Engineered Human Skin in the Treatment of Diabetic Foot Ulcerations. Plastic and Reconstructive Surgery – Global Open 7(8):p e2371, August 2019. | DOI: 10.1097/GOX.0000000000002371

[3] Protzman NM, Mao Y, Long D, Sivalenka R, Gosiewska A, Hariri RJ, Brigido SA. Placental-Derived Biomaterials and Their Application to Wound Healing: A Review. Bioengineering. 2023; 10(7):829. https://doi.org/10.3390/bioengineering10070829

[4] Schmiedova I, Dembickaja A, Kiselakova L, Nowakova B, Slama P. Using of Amniotic Membrane Derivatives for the Treatment of Chronic Wounds. Membranes (Basel). 2021 Nov 29;11(12):941. doi: 10.3390/membranes11120941. PMID: 34940442; PMCID: PMC8706466.

[5] (2018, January 30). EpiFix for chronic wounds. National Institute for Health and Care Excellence. https://www.nice.org.uk/advice/mib139

[6] Schweizer, R. (2019, March). What is your Placenta Worth? Parent’s Guide to Cord Blood Banking Foundation. https://parentsguidecordblood.org/en/news/what-is-your-placenta-worth

[7] Mansoureh Barzegar et al. Human Placenta Mesenchymal Stem Cell Protection in Ischemic Stroke is Angiotensin Converting Enzyme-2 and Masr Receptor-Dependent, Stem Cells, Volume 39, Issue 10, October 2021, Pages 1335–1348, https://doi.org/10.1002/stem.3426

[8] Kadam, S., Muthyala, S., Nair, P., & Bhonde, R. (2010). Human placenta-derived mesenchymal stem cells and islet-like cell clusters generated from these cells as a novel source for stem cell therapy in diabetes. The review of diabetic studies : RDS, 7(2), 168–182. https://doi.org/10.1900/RDS.2010.7.168

[9] Mayer L, et al. Safety and tolerability of human placenta-derived cells (PDA001) in treatment-resistant crohn’s disease: a phase 1 study. Inflamm Bowel Dis. 2013 Mar-Apr;19(4):754-60. doi: 10.1097/MIB.0b013e31827f27df. PMID: 23429460; PMCID: PMC4272923.

[10] Gwam C, Ohanele C, Hamby J, Chughtai N, Mufti Z, Ma X. Human placental extract: a potential therapeutic in treating osteoarthritis. Ann Transl Med. 2023 Jun 30;11(9):322. doi: 10.21037/atm.2019.10.20. Epub 2019 Oct 16. PMID: 37404996; PMCID: PMC10316113.

The results reported from Phase II of the first FDA-approved trial for the treatment of multiple sclerosis (MS) using stem cells are highly promising, suggesting that stem cells could be a viable treatment option to help improve the lives of those with the condition. [1]

What is the MS trial?

The Phase II trial, which was carried out by The Tisch MS Research Center of New York, constitutes the next stage in the development of the groundbreaking research signalled by the results from Phase I, the first FDA-approved trial to explore the injection of stem cells for MS.

The Phase II trial involved 54 patients split between a test group and a control group in a randomised, double-blind, placebo-controlled study.

Members of each group received 6 injections of either autologous (meaning their own) mesenchymal stem cell-derived neural progenitors (MSC-NPs) or saline placebo every two months.

These injections were administered into the cerebrospinal fluid of multiple sclerosis patients.

The study utilised a cross-over model by which patients who received the stem cell injections in year 1 were then given the saline placebo in year 2 and vice versa.

How does the stem cell treatment work?

Building on their findings from Phase I, researchers identified MSC-NPs for the treatment having ascertained that they promote tissue regeneration and immunomodulatory effects, in addition to being safe and well tolerated. [2]

MSC-NPs are a subpopulation of mesenchymal stem cells that can upregulate growth factors like hepatocyte growth factor (HGF), and minimise ectopic differentiation – essentially, the abnormal differentiation of cells.

By injecting them into the cerebrospinal fluid – a process called intrathecal (IE) injection – researchers found in an experimental autoimmune encephalomyelitis (EAE – an accepted way of modelling the effects of MS) mouse model that MSC-NPs were associated with increased spinal cord myelination, neurological recovery and reduced immune infiltration into the central nervous system. [3]

What were the results?

The results of the Phase II trial demonstrated huge promise for the use of stem cells in treating MS.

Patients requiring walking assistance saw significant improvements in both a timed 25-foot walk test and a 6 minute walking test than those in the control group.

Additionally, treatment recipients demonstrated improved bladder function, with 69% showing improvements in post-void residual volume.

Amongst the other findings were indicators that these stem cells could be helping to restore neuronal cells and reverse cognitive decline in patients with less advanced disease progression.

Furthermore, the stem cell treatment occasioned notable biomarker changes in cerebro-spinal fluid, especially with regards to the decreased levels of CCL2, a protein associated with inflammation, and the increased levels of MMP9, an indicator of the increased presence of reparative cells. [4]

Overall these findings are extremely promising, indicating that stem cells could be used in future treatments that may help to reverse many of the most debilitating aspects of multiple sclerosis, like disability.

The next stage in the study will be for researchers to investigate the effects of increasing the stem cell dosage.

What does this mean for cord blood banking?

This trial is just the latest example of the regenerative potential stem cells have to help treat life-changing conditions like MS.

As exemplified by this study, mesenchymal stem cells in particular, with their immunomodulatory and tissue repair properties, are especially promising.

Incredibly, the umbilical cord and placenta are one of the richest sources of these stem cells, but are routinely thrown away after birth.

By saving these for your baby, you could be giving them the key to future therapies for everything from cancer and stroke to diabetes and heart disease.

The potential offered by stem cells is vast, but you only get one chance to save baby’s stem cells, and that’s the day they’re born.

To find out more about cord blood banking and how it could protect your family’s health, fill out the form below to request a free Welcome Pack.

References

[1] FDA-Approved Phase II Stem Cell Treatment Trial Shows Significant and Diverse Improvements for Multiple Sclerosis (MS) Patients. Tisch MS Research Centre of New York. https://www.tischms.org/phase-ii-analysis-results

[2] Harris, Violaine K. et al. (2018) Phase I Trial of Intrathecal Mesenchymal Stem Cell-derived Neural Progenitors in Progressive Multiple Sclerosis. eBioMedicine, Volume 29, 23 – 30. https://doi.org/10.1016/j.ebiom.2018.02.002

[3] Harris, Violaine K. et al. (2012) Clinical and pathological effects of intrathecal injection of mesenchymal stem cell-derived neural progenitors in an experimental model of multiple sclerosis. Journal of the Neurological Sciences, Volume 313, Issue 1, 167 – 177. https://doi.org/10.1016/j.jns.2011.08.036

[4] Harris, V.K., Stark, J., Williams, A. et al. (2024) Efficacy of intrathecal mesenchymal stem cell-neural progenitor therapy in progressive MS: results from a phase II, randomized, placebo-controlled clinical trial. Stem Cell Res Ther 15, 151 (2024). https://doi.org/10.1186/s13287-024-03765-6

New research has demonstrated the significant role stem cells play in skin and hair development, which could lead to revolutionary new anti-ageing treatments. [1]

By identifying the ways in which the body grows skin, researchers believe that their findings could lead to the use of artificial skin in treatments for burns, wounds, and age-related degeneration.

The research was conducted as part of The Human Cell Atlas project, which aims to map the genetic instructions contained in every cell in the human body to discover how they work.

New discoveries about skin formation

The study looked at the role immune cells, specifically macrophages, play in the development of skin during pregnancy.

While macrophages – which are a type of white blood cell responsible for killing pathogens – are primarily known for their importance as part of the immune system, researchers sought to identify how they contribute to skin formation in a process known as morphogenesis.

In order to do this, a so called ‘reference atlas’ of prenatal skin (that is, skin that forms between 7-17 weeks after conception) was made using advanced techniques including single cell and spatial transcriptomics – a study of RNA molecules within both one and a population of cells – to detail how elements both within and between skin cells interact.

Their findings showed that interactions between immune cells like macrophages and non-immune cells were crucial for key processes in morphogenesis, including hair follicle development, scarless wound healing, and the formation of blood vessels in the skin. [2]

The important role of stem cells in determining skin formation

Researchers were able to verify their findings about the significant role played by immune cells in skin morphogenesis by comparing pre-natal and adult skin with a lab-grown skin model derived from stem cells.

The model, known as an organoid, successfully mimicked many aspects of skin and hair follicle development found in prenatal and adult skin.

However, it was only when researchers added macrophages that they found the model created blood vessel networks, confirming that immune cells play a more significant role in the development of skin than previously thought.

This stem-cell derived model of human skin is thought to have a vast array of possible therapeutic applications extending from use in identifying the development of conditions affecting skin and hair to providing a more accurate test material for future skin and hair treatments. [3]

Mesenchymal stem cells – key players in hair and skin formation

Another area researchers looked at during the study was the formation of hair follicles during prenatal development.

In earlier studies with mice, researchers identified special cells called pre-Dc cells that eventually become part of the hair follicle structure (called the dermal condensate, or Dc). [4]

Researchers identified similar cells in human prenatal skin and tracked how they developed into the Dc and another structure called the dermal papilla (Dp), which is important for hair growth.

Using various advanced methods, researchers were able to map out the development of these cells over time and predict their paths.

They found that certain early skin cells (fibroblasts) go down one of two routes: some become specialised for hair follicle formation, while others develop into more general skin cells.

As the hair-specific cells move toward the outer layer of the skin, they turn on genes that help them stick together and form the structures needed for hair growth.

The study also looked at how mesenchymal cells interact with skin’s outer (epithelial) cells during hair follicle formation.

They identified important signalling molecules, such as CXCL12, that help guide cell migration and organisation. Interestingly, immune cells were also predicted to assist with these processes. [5]

Discovering the genetic origins of skin and hair conditions

Having mapped how skin and hair developed in pre-natal skin, researchers turned to investigating the origins of certain skin and hair conditions.

They found that reduced hair growth (hypotrichosis) and abnormal hair shapes (like pili torti) are caused by specific genes that are active during early hair follicle development.

Similarly, they were able to identify that epidermolysis bullosa (EB) – a disorder that makes the skin fragile and prone to blistering – had its roots in the expression of the gene COL7A1 in several types of prenatal skin cells.

Researchers were again able to compare gene expression in prenatal skin with their stem cell-derived lab-grown skin models and found strong similarities, noting that while these diseases often stem from defects in skin structure, they are frequently worsened by immune system involvement. [6]

The importance of stem cells and cord blood banking

As this new research demonstrates, stem cells play a pivotal role in not only helping researchers to better understand the formation of vital organs, like skin, but also in helping to facilitate potential future therapies.

By being able to compare cells from both prenatal and adult skin with artificial lab-grown skin using stem cells, researchers were able to identify the hitherto unknown significance of immune cells in skin formation and accurately map the genetic instructions involved in growing skin.

In their paper published in Nature, The Human Cell Atlas team have achieved something remarkable: essentially providing a set of instructions for creating human skin.

Their research is being heralded as groundbreaking and for good reason. Their findings will help to contribute to the development of a range of therapies for genetic skin conditions, treatments for wounds and burns, and remedies that could help to rejuvenate skin.

While the stem cells utilised in this trial were from embryonic stem cells and induced pluripotent stem cells, the stem cells found in umbilical cord tissue have similarly huge potential in regenerative medicine.

In fact, and as we’ve shown previously, trials utilising mesenchymal stem cells – the same type as found in umbilical cord tissue – are already underway that could aid in the treatment of both hair loss and skin conditions like atopic dermatitis. [7] [8]

What’s clear is that stem cells are pivotal in both cutting edge research and the development of future regenerative therapies.

Unfortunately, the umbilical cord and placenta – both rich sources of stem cells – are routinely thrown away after birth. By saving these powerful stem cells for your baby, you could give them access to future therapies that are currently being developed using stem cell-based research as in this study.

To find out more about saving stem cells for your baby, or the baby of someone you know who’s expecting, fill out the form below to download your free Welcome Pack.

References

[1] Ghosh, P. (2024, October 16). New skin research could help slow signs of ageing. BBC News. https://www.bbc.co.uk/news/articles/c756q166gd9o

[2] Gopee, N.H., Winheim, E., Olabi, B. et al. A prenatal skin atlas reveals immune regulation of human skin morphogenesis. Nature (2024). https://doi.org/10.1038/s41586-024-08002-x

[3] Lee J, Rabbani CC, Gao H, Steinhart MR, Woodruff BM, Pflum ZE, Kim A, Heller S, Liu Y, Shipchandler TZ, Koehler KR. Hair-bearing human skin generated entirely from pluripotent stem cells. Nature. 2020 Jun;582(7812):399-404. doi: 10.1038/s41586-020-2352-3. Epub 2020 Jun 3. PMID: 32494013; PMCID: PMC7593871.

[4] Martisova A, Sommerova L, Krejci A, Selingerova I, Kolarova T, Zavadil Kokas F, Holanek M, Podhorec J, Kazda T, Hrstka R. Identification of AGR2 Gene-Specific Expression Patterns Associated with Epithelial-Mesenchymal Transition. Int J Mol Sci. 2022 Sep 16;23(18):10845. doi: 10.3390/ijms231810845. PMID: 36142758; PMCID: PMC9504245.

[5] Gopee, N.H., Winheim, E., Olabi, B. et al. A prenatal skin atlas reveals immune regulation of human skin morphogenesis. Nature (2024). https://doi.org/10.1038/s41586-024-08002-x

[6] Huitema L, Phillips T, Alexeev V, Igoucheva O. Immunological mechanisms underlying progression of chronic wounds in recessive dystrophic epidermolysis bullosa. Exp Dermatol. 2021 Dec;30(12):1724-1733. doi: 10.1111/exd.14411. Epub 2021 Jun 27. PMID: 34142388; PMCID: PMC9290674.

[7] (2024, January 10). Stem Cells Could Be The Future of Hair Regrowth. Cells4Life. https://cells4life.com/2024/01/stem-cells-could-be-the-future-of-hair-regrowth/

[8] (2024, July 11). Promising Results of Stem Cell Eczema Treatment. Cells4Life. https://cells4life.com/2024/07/promising-results-of-stem-cell-eczema-treatment/

Recent research indicates that umbilical cord exosomes have huge potential in the field of regenerative medicine.

In this blog we’ll explore what exosomes are, where they’re found, and how they could be used in developing new, life changing therapies for a range of conditions.

What are exosomes?

Exosomes are nano-sized particles that contain key biomolecules like proteins and lipids and travel between cells like messengers, regulating numerous biological processes including inflammatory responses.

Initially thought to be responsible for conveying waste away from cells, in recent years numerous trials and studies have explored their intercellular communicatory function and the potential this offers in a range of therapeutic applications.

Where are exosomes found?

Exosomes are produced by virtually every cell in the body, including stem cells.

Excitingly they can be derived from the mesenchymal stem cells that exist within both umbilical cord blood and umbilical cord tissue.

Due to the low immunogenicity (the likelihood of being attacked by the immune system when transplanted) of mesenchymal stem cells derived from the umbilical cord, along with their non-invasive harvest procedure and ease of expansion in vitro, umbilical cord MSC exosomes (UC-MSC-Exo) are well suited for research and potentially therapeutic purposes. [1]

What can umbilical cord exosomes do?

Below are a few examples of recent trials utilising UC-MSC-Exo in the treatment of various conditions.

Heal wounds and repair nerves

A 2022 study investigating the use of UC-MSC-Exo in treating cutaneous nerve damage and wound healing showed that exosomes have the ability to promote skin and nerve regeneration.

Studied in vitro, researchers found that UC-MSC-Exo promoted the migration and proliferation of skin fibroblasts – cells that play a key role in forming connective tissue.

Additionally, UC-MSC-Exo were also found to promote the secretion of nerve growth factors by fibroblasts, suggesting that exosomes may enhance wound healing by promoting nerve repair.

Tested in vivo in a mouse model, the study showed that the wounds of the treatment group who received UC-MSC-Exo healed faster than the wounds of the group who received a control medium.

The study concluded that UC-MSC-Exo produced therapeutic effects by promoting skin and nerve regeneration. [2]

Slow the progress of osteoarthritis

Another study from 2022 demonstrated that exosomes derived from the umbilical cord have the potential to be used as a treatment method for osteoarthritis (OA).

Using a rat model, researchers undertook a surgical procedure designed to recreate the effects of knee osteoarthritis. They then administered injections of either UC-MSC-Exo or saline solution. After 8 weeks, knee samples were taken to assess the progression of the disease.

Researchers discovered that in the knees of rats who had received injections of exosomes the progression of OA had been halted and severe damage to knee cartilage prevented.

One of the key reasons behind this, the researchers found, was that the exosomes promoted the migration and proliferation of chondrocytes – cells that make up cartilage. They also found that the exosomes helped to inhibit the secretion of pro-inflammatory factors, as well as regulate immune responses; contributing factors in cartilage regeneration. [3]

Alleviate liver damage and disease

A study from 2020 showed that exosomes derived from umbilical cord mesenchymal stem cells were able to secrete the molecule miR-455-3p which helped to suppress inflammatory immune responses and inhibit signalling pathways in order to improve liver damage. [4]

Another study showed that exosomes derived from umbilical cord mesenchymal stem cells have antioxidant effects.

A contributing factor to the progression of liver damage, oxidative stress occurs when there is an imbalance in free radicals (unstable oxygen molecules) and antioxidants in the liver. Oxidative stress can lead to what’s known as apoptosis – programmed cell death. [5]

By using a mouse model, researchers were able to ascertain that injections of UC-MSC-Exo inhibited apoptosis in liver injury, reversing the fallout from oxidative stress. [6]

Promote heart repair

There have been several studies indicating that exosomes derived from umbilical cord mesenchymal stem cells have the ability to reduce myocardial injury – damage to heart tissue.

A study from 2018 showed that UC-MSC-Exo may have the ability to promote the expression of Smad7, a protein which aids in myocardial protection by blocking certain signalling pathways.

Researchers believed this could be the mechanism underlying exosomes’ ability to inhibit apoptosis, promote angiogenesis (the formation of new blood vessels) and improve cardiac function following acute myocardial infarction. [7] [8]

Rejuvenate older stem cells

An especially intriguing study from 2020 also showed that exosomes derived from umbilical cord mesenchymal stem cells could help in myocardial repair by rejuvenating the activity and function of mesenchymal stem cells in older patients.

Because heart conditions mainly affect older patients, the risk of immunogenicity associated with allogeneic stem cell transplants in helping to treat heart damage is a potential risk. This leaves autologous stem cell transplants – using stem cells from the patient themselves – as an available treatment option. However, due to age and cardiovascular risk factors, these autologous stem cells can be limited in their usefulness.

What this study found, however, is that UC-MSC-Exo have the ability to ameliorate the senescence of older mesenchymal stem cells and renew their biological activity, such as the potential to differentiate into cartilage, bone, and fat cells.

In effect this means that umbilical cord exosomes could help in restoring the regenerative capacity of mesenchymal stem cells in a range of therapies for patients who need them most. [9]

Exosomes and cord blood banking

As some of these trials demonstrate, exosomes are a hugely exciting field of regenerative medicine, and their therapeutic potential is only just beginning to be understood.

Most exciting is that these exosomes can be found in umbilical cord blood and tissue which, although usually thrown away as medical waste, can be stored so that your baby has access to their own stem cells that they can use in future therapies.

To learn more about the potential of umbilical cord stem cells and exosomes, contact our specialist team on 01444 873 950 – they’ll be more than happy to help answer any questions you may have.

In the meantime, why not fill out your details below for a free Welcome Pack containing everything you need to know about storing stem cells for your baby.

References

[1] Zhang, N., Zhu, J., Ma, Q. et al. (2020) Exosomes derived from human umbilical cord MSCs rejuvenate aged MSCs and enhance their functions for myocardial repair. Stem Cell Res Ther 11, 273. https://doi.org/10.1186/s13287-020-01782-9

[2] Zhu, Z., Zhang, X., Hao, H., Xu, H., Shu, J., Hou, Q., & Wang, M. (2022). Exosomes Derived From Umbilical Cord Mesenchymal Stem Cells Treat Cutaneous Nerve Damage and Promote Wound Healing. Frontiers in cellular neuroscience, 16, 913009. https://doi.org/10.3389/fncel.2022.913009

[3] Li, P., Lv, S., Jiang, W., Si, L., Liao, B., Zhao, G., Xu, Z., Wang, L., Zhang, J., Wu, H., Peng, Q., Li, Z., Qi, L., Chi, G., & Li, Y. (2022). Exosomes derived from umbilical cord mesenchymal stem cells protect cartilage and regulate the polarization of macrophages in osteoarthritis. Annals of translational medicine, 10(18), 976. https://doi.org/10.21037/atm-22-3912

[4] Shao, M., Xu, Q., Wu, Z., Chen, Y., Shu, Y., Cao, X., Chen, M., Zhang, B., Zhou, Y., Yao, R., Shi, Y., & Bu, H. (2020). Exosomes derived from human umbilical cord mesenchymal stem cells ameliorate IL-6-induced acute liver injury through miR-455-3p. Stem cell research & therapy, 11(1), 37. https://doi.org/10.1186/s13287-020-1550-0

[5] Li, S., Tan, H. Y., Wang, N., Zhang, Z. J., Lao, L., Wong, C. W., & Feng, Y. (2015). The Role of Oxidative Stress and Antioxidants in Liver Diseases. International journal of molecular sciences, 16(11), 26087–26124. https://doi.org/10.3390/ijms161125942

[6] Jiang, W., Tan, Y., Cai, M., Zhao, T., Mao, F., Zhang, X., Xu, W., Yan, Z., Qian, H., & Yan, Y. (2018). Human Umbilical Cord MSC-Derived Exosomes Suppress the Development of CCl4-Induced Liver Injury through Antioxidant Effect. Stem cells international, 2018, 6079642. https://doi.org/10.1155/2018/6079642

[7] Wang, X., Zhao, Y., Sun, L., Shi, Y., Li, Z., Zhao, X. … Zhu, W. (2018). Exosomes derived from human umbilical cord mesenchymal stem cells improve myocardial repair via upregulation of Smad7. International Journal of Molecular Medicine, 41, 3063-3072. https://doi.org/10.3892/ijmm.2018.3496

[8] Zhao, Yuanyuan, Sun, Xiaoxian, Cao, Wenming, Ma, Jie, Sun, Li, Qian, Hui, Zhu, Wei, Xu, Wenrong. (2015) Exosomes Derived from Human Umbilical Cord Mesenchymal Stem Cells Relieve Acute Myocardial Ischemic Injury, Stem Cells International, 761643, 12 pages, 2015. https://doi.org/10.1155/2015/761643

[9] Zhang, N., Zhu, J., Ma, Q. et al. (2020) Exosomes derived from human umbilical cord MSCs rejuvenate aged MSCs and enhance their functions for myocardial repair. Stem Cell Res Ther 11, 273. https://doi.org/10.1186/s13287-020-01782-9

Umbilical cord blood has long been recognized as a valuable source of life-saving stem cells.

Since the first cord blood transplant was carried out in 1988 for a patient with Fanconi anaemia, it’s estimated that more than 40,000 cord blood transplants have been delivered worldwide, improving countless lives. [1]

Today, new research is revealing even more potential uses for cord blood, especially in combination with other types of transplants.

One exciting development in this area is the emergence of ‘haplo cord’ transplants, which combine cord blood stem cells with haplo-identical bone marrow, offering promising outcomes and improved survival rates for patients who need stem cell transplants.

What is a haplo cord transplant?

A haplo cord transplant is a procedure that involves the infusion of stem cells from both a cord blood unit and a haplo-identical bone marrow donor.

‘Haplo-identical’ means the donor is a half-match to the patient, typically a parent or sibling.

Both bone marrow and umbilical cord blood contain haematopoietic stem cells (HSCs) which can be used to treat blood cancers like acute myeloid leukaemia (AML) and lymphoma.

HSCs are blood-forming stem cells, meaning that they can differentiate into different kinds of blood cells, including white blood cells, red blood cells, and platelets.

The transplant works by infusing a patient with these blood-forming stem cells so that the patient’s body can begin to produce healthy, rather than diseased, blood cells.

In a haplo cord transplant, the patient is coinfused with both bone marrow stem cells from a related, partially matched donor, along with donated cord blood stem cells.

Haplo cord transplants are not a new concept—they have been under study for over a decade. [2]

In 2014, a Spanish clinical trial demonstrated that the outcomes of haplo cord transplants were comparable to those of traditional matched unrelated donor transplants, putting haplo cord transplants among the frontline for allogeneic stem cell transplant options. [3]

More recently, research led by oncologist Dr. Depei Wu from Soochow University in China has provided more detailed evidence on this transplant approach.

Across trials for AML, B-ALL (B Cell Acute Lymphoblastic Leukaemia) leukaemia and aplastic anaemia it was found that the addition of cord blood to a haploidentical transplant resulted in significant improvements in overall survival. [4] [5] [6]

Why are haplo cord transplants important?

The most exciting and important results demonstrated by studies into haplo cord transplants have been the effect on overall survival rates.

A recent multicentre, phase 3 trial, conducted on patients with acute myeloid leukaemia (AML), showed that those who received a haplo cord transplant had a significantly better three-year overall survival rate compared to those who only received a haploidentical transplant.

Specifically, 80.5% of patients in the haplo cord group survived after three years, compared to 67.8% in the haplo-only group. These results are encouraging for both patients and doctors, suggesting that haplo cord transplants may offer a better chance of long-term survival. [7]

In addition to the improved survival rates, other trials have found that haplo cord transplants also offer other potential advantages.

The addition of cord blood stem cells in transplants has been shown to improve immune responses by accelerating neutrophil (a type of white blood cell that helps the body fight infection) engraftment and platelet recovery. [8] [9]

These outcomes are critical for patients undergoing stem cell transplants, as it means reductions in the risks of infections and complications, including AML relapses.

Additionally, cord blood showed improvements in GvL (graft-versus-leukaemia) outcomes – where donor cells attack leukaemia residing in the host patient – without significantly increasing graft-versus-host-disease (GvHD) rates. [10]

The future of haplo cord transplants

While the early results are promising, there is still much to learn about haplo cord transplants.

Oncologists need to explore various factors, such as the ideal chemotherapy regimen, the role of each donor source, and strategies to minimise complications like graft-versus-host disease.

Additional randomised clinical trials are necessary to better understand the full potential of this transplant approach, however, current research suggests that for patients without a fully matched donor, the combination of cord blood and haplo-identical bone marrow could offer a new hope for survival rates and long-term outcomes.

If you’re interested in learning more about cord blood banking and how it can help in life-saving treatments like haplo cord transplants, fill out the form below to receive a free guide to cord blood banking.

References

[1] Gupta, A. O., & Wagner, J. E. (2020). Umbilical Cord Blood Transplants: Current Status and Evolving Therapies. Frontiers in pediatrics, 8, 570282. https://doi.org/10.3389/fped.2020.570282

[2] (2021, January 28). Haplo-Cord Transplants. Parents’ Guide to Cord Blood Foundation. https://parentsguidecordblood.org/en/news/haplo-cord-transplants

[3] Kwon, Mi et al. (2014, December 1). Haplo-Cord Transplantation Using CD34+ Cells from a Third-Party Donor to Speed Engraftment in High-Risk Patients with Hematologic Disorders. Biology of Blood and Marrow Transplantation. Volume 20, Issue 12. 1083-8791. doi: 10.1016/j.bbmt.2014.08.024

[4] Zhou, B., et al. (2021, January 28). Haploidentical Hematopoietic Cell Transplantation Combined with an Unrelated Cord Blood Unit for Adult Acute Myeloid Leukemia Results in Improved Survival Compared to Haploidentical Hematopoietic Cell Transplantation: Results of a Multicenter, Randomized, Phase III Trial. American Society of Hematology. https://ash.confex.com/ash/2023/webprogram/Paper180993.html

[5] Zhou, B., Xu, M., Lu, S., Liu, Y., Qi, L., Liu, T., Tian, H., Chen, J., Wu, D., & Xu, Y. (2022). Clinical Outcomes of B Cell Acute Lymphoblastic Leukemia Patients Treated with Haploidentical Stem Cells Combined with Umbilical Cord Blood Transplantation. Transplantation and cellular therapy, 28(3), 173.e1–173.e6. https://doi.org/10.1016/j.jtct.2021.12.010

[6] Lei, M., Zhang, Y., Jiao, W., Li, X., Zhou, H., Wang, Q., Qiu, H., Tang, X., Han, Y., Fu, C., Jin, Z., Chen, S., Sun, A., Miao, M., Liu, L., & Wu, D. (2022). Comparison of Haploidentical Hematopoietic Stem Cell Transplant With or Without Unrelated Cord Blood Infusion in Severe Aplastic Anemia: Outcomes of a Multicenter Study. Frontiers in immunology, 13, 912917. https://doi.org/10.3389/fimmu.2022.912917

[7] Zhou, B., Chen, J., Liu, T. et al. (2024). Haploidentical hematopoietic cell transplantation with or without an unrelated cord blood unit for adult acute myeloid leukemia: a multicenter, randomized, open-label, phase 3 trial. Sig Transduct Target Ther 9, 108. https://doi.org/10.1038/s41392-024-01820-5

[8] Kent, Andrew., et al. (2024, August 16). Outcomes of Haplo-Cord Versus Dual Cord Transplants: A Single-Center Retrospective Analysis. Transplantation and Cellular Therapy, Official Publication of the American Society for Transplantation and Cellular Therapy. https://doi.org/10.1016/j.jtct.2024.07.021

[9] van Besien, K., Artz, A., Champlin, R. E., Guarneri, D., Bishop, M. R., Chen, J., Gergis, U., Shore, T., Liu, H., Rondon, G., Mayer, S. A., Srour, S. A., Stock, W., & Ciurea, S. O. (2019). Haploidentical vs haplo-cord transplant in adults under 60 years receiving fludarabine and melphalan conditioning. Blood advances, 3(12), 1858–1867. https://doi.org/10.1182/bloodadvances.2019000200

[10] van Besien, K., Liu, H., Margevicius, S., Fu, P., Artz, A., Chaekal, O. K., … Kwon, M. (2024). Haplo-cord transplant. Realizing the potential of umbilical cord blood grafts. – A review of techniques and analysis of outcomes. Leukemia & Lymphoma, 65(10), 1384–1397. https://doi.org/10.1080/10428194.2024.2361353

Last month researchers at The Parent’s Guide to Cord Blood Foundation compiled a report about how perinatal stem cells were being employed in clinical trials from 2019 through to the end of 2023. [1]

Perinatal stem cells are those which are collected in and around the perinatal period: the period immediately before and after birth.

These include stem cells from cord blood, cord tissue, the placenta and amnion.

In this blog, we’ll summarise some of their key findings and discuss what they mean in deciding to store stem cells for your baby.

Increase in the number of perinatal stem cell trials, despite COVID-19

In their report, researchers led by Dr Frances Verter point out how clinical trials using umbilical cord mesenchymal stem cells in particular reached a peak in 2020 and 2021, the result of accelerated research to help combat the COVID-19 pandemic.

They estimate that during the pandemic (2020-2022), nearly 41% of all advanced cellular therapy trials for COVID-19 relied on perinatal stem cells.

While the number of trials declined by almost half in the years that followed – to be expected, considering the impact of the pandemic on trial numbers – the researchers found that during the two years since their last report in 2021, the cumulative number of trials for advanced cellular therapies using perinatal stem cells increased by 17%.

This shows that the number of clinical trials using perinatal stem cells has not only returned to pre pandemic levels, but has actually increased, demonstrating a sustained interest in the clinical and therapeutic application of perinatal stem cells.

(graph courtesy of The Parents Guide to Cord Blood Foundation, https://parentsguidecordblood.org/en/news/2024-update-how-many-clinical-trials-employ-perinatal-sources-stem-cells)

Trials for stem cells derived from placenta and amnion

Dr Verter and her team also point out that there are increasingly prominent roles for perinatal stem cells derived from a variety of sources beyond cord blood and cord tissue, namely: placenta and amnion.

Of the 402 clinical trials focusing on the use of mesenchymal stem cells derived or expanded from perinatal tissues, 9% utilised either placenta, amniotic membrane, or a mixture of a number of different perinatal stem cell sources. For reference, only 4% of trials derived MSCs from cord blood.

These numbers suggest that the placenta and amnion are becoming increasingly pivotal to the development of advanced cellular therapies using perinatal sources of stem cells, with the majority of these MSC trials focusing on COVID-19, neurological conditions, orthopaedic conditions and auto-immune disorders.

Cord tissue trials outpace cord blood trials

Since their last report on the cumulative number of trials through to the end of 2021, Dr Verter and her team found that in the years since, through to the end of 2023, there were 117 new trials utilising cord tissue and 40 new trials utilising cord blood. [2]

A key source of mesenchymal stem cells – which have the ability to differentiate into other specialised cell types such as muscle, nerve, and cartilage cells – cord tissue has once again shown itself to be at the forefront of trials utilising perinatal stem cells.

Of the 402 perinatal stem cell trials using MSCs, 87% of these utilised MSCs derived from cord tissue alone.

Considering that during the period from 2019-2023, over 75% of all perinatal stem cell trials were focused on the application of mesenchymal stem cells, cord tissue continues to be a vital source of the kind of stem cells that are driving current therapeutic interest in the application of stem cells from perinatal tissues.

Wide array of medical uses for perinatal mesenchymal stem cells

Another significant finding in this report is the sheer array of diagnosis categories that perinatal mesenchymal stem cells are being trialled for.

While around 45% of trials using perinatal mesenchymal stem cells focused on how they might be applied to treat COVID-19 and other respiratory illnesses, as well as neurological and orthopaedic conditions, another 46% of the total number of perinatal mesenchymal stem cell trials were divided across a wide range of medical diagnoses.

These medical diagnoses spanned trials for auto-immune disorders, the treatment of different forms of wounds, liver conditions, metabolic and genetic conditions (such as Krabbe disease and Hurler’s syndrome), cancers and blood cancers, and cardio-vascular conditions.

These figures suggest not only an increase in the number of perinatal mesenchymal stem cell trials, but also a broad spectrum of possible treatment applications, making them invaluable in the development of future advanced cellular therapies.

(graph courtesy of The Parents Guide to Cord Blood Foundation, https://parentsguidecordblood.org/en/news/2024-update-how-many-clinical-trials-employ-perinatal-sources-stem-cells)

What does this mean for cord blood banking?

If you’re considering storing stem cells for your baby, the findings of this report are encouraging.

The sustained growth in clinical trials using perinatal stem cells, even beyond the pandemic, highlights the continued and expanding interest in their therapeutic potential.

Cord tissue, in particular, stands out as a critical source of mesenchymal stem cells, which are increasingly being used in a wide range of medical applications, from COVID-19 and neurological conditions to auto-immune disorders and orthopaedic treatments.

The growing use of placental and amniotic stem cells further emphasises the value of preserving these additional perinatal tissues, providing a great chance to maximise your baby’s ability to access future treatments.

With ongoing advancements in cellular therapies and the broad spectrum of diseases being targeted, banking stem cells from your baby’s umbilical cord and placenta could offer valuable opportunities for future medical treatments that go beyond what is available today.

Your baby’s stem cells may play a crucial role in the next generation of medical breakthroughs.

If you’re interested in storing stem cells for your baby, fill out the form below to request a free Welcome Pack. It contains information that will help to shed light on the collection process, our storage services, and the future potential offered by umbilical cord stem cells.

References

[1] Verter, F., Bersenev, A., & Couto, P. S. (2024, September 10). 2024 Update: How Many Clinical Trials Employ Perinatal Sources of Stem Cells? Parents Guide to Cord Blood Foundation. https://parentsguidecordblood.org/en/news/2024-update-how-many-clinical-trials-employ-perinatal-sources-stem-cells

[2] Verter, F. (2022, September 15). 2022 Update: How many clinical trials use cord blood or cord tissue? Parents Guide to Cord Blood Foundation. https://parentsguidecordblood.org/en/news/2022-update-how-many-clinical-trials-use-cord-blood-or-cord-tissue

A new therapy, recently granted a UK Innovation Passport Designation, could help to improve treatment outcomes for Krabbe disease patients post-stem cell transplant.

What is Krabbe disease?

Krabbe disease is a rare genetic disorder affecting around 1 in every 100,000 births and is caused by a deficiency of the enzyme galactocerebrosidase (GALC), leading to the breakdown of the myelin in the nervous system.

The most common variant of Krabbe disease, Infant Krabbe Disease, affects children under the age of 1, and can cause muscle stiffness, seizures, and developmental delays. It is often fatal if untreated.

Approximately 85% of Krabbe disease cases are the infantile subtypes. [1]

What are the current treatment options for Krabbe disease?

Currently, the only effective treatment option for Krabbe disease is a transplant of haematopoietic stem cells (HSCs), which are found in bone marrow, peripheral blood, and umbilical cord blood. [2]

By transplanting HSCs from a donor to a Krabbe disease patient, the patient’s unhealthy cells lacking in the GALC enzyme are eventually replaced by healthy red blood cells, white blood cells and platelets derived from HSCs.

These healthy blood cells can then work to populate the brain with GALC enzyme activity, reducing the breakdown of myelin and thereby stabilise cognitive function. [3]

Stem cell transplants using haematopoietic stem cells from cord blood, specifically, have been shown to be highly effective in improving neurological outcomes if the transplant is performed before the development of symptoms. [4]

While HSCT remains the only viable treatment option for Krabbe disease, it is not a cure.

It also does not combat the peripheral neuropathy occasioned by Krabbe disease, a condition affecting the nerves beyond the brain and spinal cord, leading to decline in motor function. [5]

What is the new gene therapy and how does it improve stem cell transplant outcomes?

Developed by Forge Biologics, the FBX-101 therapy works by delivering a copy of the GALC gene to cells in the nervous system, which improves myelination (the process by which the myelin sheath forms) and, crucially, motor function.

It is designed to be administered intravenously after the current standard of care, a haematopoietic stem cell transplant.

In its early phase trial, REKLAIM, FBX-101 was shown to improve motor function in all five of the patients who underwent treatment.

Building on these promising results, the Innovation Passport designation means that FBX-101 will be able to enter the Innovative Licensing and Access Pathway (ILAP), which accelerates both market and regulatory access in the UK. [6]

Why is newborn screening for Krabbe disease so important?

Newborn screening for Krabbe disease is crucial because the condition progresses rapidly, especially in its infantile form, and early intervention is the key to preventing severe neurological damage.

Ideally, the patient would receive a transplant within 30 days in order to have the best chance of improved neurological and transplant outcomes. [7]

Symptoms of Krabbe disease often appear within the first few months of life, and once they start, the deterioration of the nervous system is fast and irreversible.

By the time symptoms are noticeable, significant damage has already occurred, limiting the effectiveness of available treatments.

Newborn screening allows for early diagnosis before symptoms develop, enabling early intervention through HSCT, which is at its most effective if administered before significant damage to the nervous system. [8]

Could cord blood banking help?

Umbilical cord blood is a vital source of haematopoietic stem cells which can differentiate into various kinds of blood cells. These cells are crucial in the treatment of Krabbe disease, but rely on finding a donor match in order for a transplant to be successful.

If Krabbe disease runs in your family, saving cord blood for every child is probably a worthwhile investment.

If your baby does end up developing Krabbe disease, having their sibling’s stem cells in storage could make the difference in being able to access a life-saving transplant as a sibling has a 75% chance of being a partial donor match.

For more information about how cord blood banking could help with the treatment of Krabbe disease, visit our Krabbe Disease and Stem Cells page, here.

If either you or a family member or someone you know is expecting, why not download our free Welcome Pack to learn more about the benefits of cord blood banking. Simply fill out the form below.

References

[1] Isabel C. Yoon, Nicholas A. Bascou, Michele D. Poe, Paul Szabolcs, Maria L. Escolar; Long-term neurodevelopmental outcomes of hematopoietic stem cell transplantation for late-infantile Krabbe disease. Blood 2021; 137 (13): 1719–1730. doi: https://doi.org/10.1182/blood.2020005477

[2] Isabel C. Yoon, Nicholas A. Bascou, Michele D. Poe, Paul Szabolcs, Maria L. Escolar; Long-term neurodevelopmental outcomes of hematopoietic stem cell transplantation for late-infantile Krabbe disease. Blood 2021; 137 (13): 1719–1730. doi: https://doi.org/10.1182/blood.2020005477

[3] (2022, August 13). Krabbe Disease (Globoid Cell Leukodystrophy). Cleveland Clinic. https://my.clevelandclinic.org/health/diseases/6039-krabbe-disease-globoid-cell-leukodystrophy

[4] Wright, M. D., Poe, M. D., DeRenzo, A., Haldal, S., & Escolar, M. L. (2017). Developmental outcomes of cord blood transplantation for Krabbe disease: A 15-year study. Neurology, 89(13), 1365–1372. https://doi.org/10.1212/WNL.0000000000004418

[5] Beltran-Quintero, M.L., Bascou, N.A., Poe, M.D. et al. Early progression of Krabbe disease in patients with symptom onset between 0 and 5 months. Orphanet J Rare Dis 14, 46 (2019). https://doi.org/10.1186/s13023-019-1018-4

[6] (2024, March 19). Forge Biologics’ Novel AAV Gene Therapy FBX-101 for Patients with Krabbe Disease is Granted UK’s Innovation Passport Designation. Forge Biologics. https://www.forgebiologics.com/forge-biologics-novel-aav-gene-therapy-fbx-101-for-patients-with-krabbe-disease-is-granted-uks-innovation-passport-designation/

[7] Page, K. M., Ream, M. A., Rangarajan, H. G., Galindo, R., Mian, A. Y., Ho, M. L., Provenzale, J., Gustafson, K. E., Rubin, J., Shenoy, S., & Kurtzberg, J. (2022). Benefits of newborn screening and hematopoietic cell transplant in infantile Krabbe disease. Blood advances, 6(9), 2947–2956. https://doi.org/10.1182/bloodadvances.2021006094

[8] Page, K. M., Ream, M. A., Rangarajan, H. G., Galindo, R., Mian, A. Y., Ho, M. L., Provenzale, J., Gustafson, K. E., Rubin, J., Shenoy, S., & Kurtzberg, J. (2022). Benefits of newborn screening and hematopoietic cell transplant in infantile Krabbe disease. Blood advances, 6(9), 2947–2956. https://doi.org/10.1182/bloodadvances.2021006094

Advancements in stem cell research are transforming how we approach some of the most critical health conditions.

One particularly exciting area is the use of umbilical cord stem cells in treating acute liver failure (ALF).

ALF is a life-threatening condition with limited treatment options, but new research suggests that umbilical cord-derived mesenchymal stem cells (UC-MSCs) could offer a promising solution.

What is Acute Liver Failure (ALF)?

Acute liver failure is a rare, though severe, condition comprising a rapid decline in liver function that can lead to severe health complications and even death if not treated promptly.

In the UK there are around 400 emergency admissions for ALF per year. Mortality rates for ALF are considerable: 60-90%. [1]

ALF is typically caused by viral infections, drug toxicity, or autoimmune diseases.

Most admissions in the UK were for paracetamol overdoses.

Current treatment options are limited to liver transplants or supportive care—both of which come with significant obstacles, including lack of available organs and financial expense. [2]

As a result, there is a growing need to explore alternative therapies, and stem cells from the umbilical cord show huge potential for application in treatment.

How could umbilical cord stem cells help?

The umbilical cord is a rich source of mesenchymal stem cells. These are cells that have the ability to differentiate into other specialised cell types in the body, as well as having immunomodulatory properties and the ability to secrete growth factors. [3]

While MSCs can be derived from various sources, including bone marrow and adipose tissue, numerous studies have shown that the use of mesenchymal stem cells derived from the umbilical cord in the treatment of ALF is associated with lower mortality rates and improved liver function. [4] [5]

It’s also been suggested that UC-MSCs are better positioned to both differentiate into hepatocyte-like cells (i-Heps) and promote the proliferation of hepatocyte cells within the liver. [6]

Hepatocyte cells are responsible for various processes within the liver, and cause inflammation when they become damaged, a contributing factor to ALF. [7]

The ability to either replace damaged hepatocyte cells or promote their proliferation using UC-MSCs could be hugely important in mitigating acute liver failure.

What’s the latest research into using umbilical cord stem cells for ALF?

A new clinical trial is currently underway exploring the use of mesenchymal stem cells derived from the umbilical cord (UC-MSCs) as a treatment option for ALF.

The trial, which is scheduled to conclude in 2028, will assess the safety and efficacy of UC-MSCs in cases of acute-on-chronic liver failure wherein patients experience sudden organ failure as a result of an underlying chronic liver disease.

A multi-centred, double blind and randomised placebo controlled trial, the new study will seek to validate previously promising findings about the use of MSCs in acute liver failure treatment.

Participants will be split into a control group and a treatment group, with those in the treatment group receiving either 3 or 5 intravenous infusions of mesenchymal stem cells depending on whether they are members of the short or prolonged treatment group.

Follow-ups will occur weekly up to 5 weeks and then at 8, 12, 24, and 53 weeks.

The trial will also seek to definitively determine whether umbilical cord-derived mesenchymal stem cells are more effective than other sources of MSCs in the treatment of acute liver failure. [8]

Cord blood banking and ALF treatment

This study is just one of many showing the therapeutic potential of umbilical cord stem cells, not just for ALF, but for a variety of conditions.

There are currently hundreds of clinical trials exploring how umbilical cord stem cells could help in the treatment of conditions ranging from cancer to stroke, heart disease to Parkinson’s disease.

For patients with acute liver failure, UC-MSC-based therapy could one day become a life-saving alternative to liver transplants.

As this research progresses, cord blood banking becomes even more valuable as it means having your own perfectly matched stem cells available for use in future therapies, eliminating the risk of rejection.

If you or someone you know is expecting, download our free Welcome Pack below to find out more about saving baby’s stem cells. It could be the best decision you ever make for their health.

References

[1] Kerslake, D. A. (2024, August 5). Acute Liver Failure. RCEM Learning. https://www.rcemlearning.co.uk/reference/acute-liver-failure/#1567604193379-1229443a-872e

[2] Wang, Y. H., & Chen, E. Q. (2023). Mesenchymal Stem Cell Therapy in Acute Liver Failure. Gut and liver, 17(5), 674–683. https://doi.org/10.5009/gnl220417

[3] Hu, C., Wu, Z., & Li, L. (2020). Mesenchymal stromal cells promote liver regeneration through regulation of immune cells. International journal of biological sciences, 16(5), 893–903. https://doi.org/10.7150/ijbs.39725

[4] Xu, W. X., He, H. L., Pan, S. W., Chen, Y. L., Zhang, M. L., Zhu, S., Gao, Z. L., Peng, L., & Li, J. G. (2019). Combination Treatments of Plasma Exchange and Umbilical Cord-Derived Mesenchymal Stem Cell Transplantation for Patients with Hepatitis B Virus-Related Acute-on-Chronic Liver Failure: A Clinical Trial in China. Stem cells international, 2019, 4130757. https://doi.org/10.1155/2019/4130757

[5] Shi, M., Zhang, Z., Xu, R., Lin, H., Fu, J., Zou, Z., Zhang, A., Shi, J., Chen, L., Lv, S., He, W., Geng, H., Jin, L., Liu, Z., & Wang, F. S. (2012). Human mesenchymal stem cell transfusion is safe and improves liver function in acute-on-chronic liver failure patients. Stem cells translational medicine, 1(10), 725–731. https://doi.org/10.5966/sctm.2012-0034

[6] Zhou, R., Li, Z., He, C., Li, R., Xia, H., Li, C., Xiao, J., & Chen, Z. Y. (2014). Human umbilical cord mesenchymal stem cells and derived hepatocyte-like cells exhibit similar therapeutic effects on an acute liver failure mouse model. PloS one, 9(8), e104392. https://doi.org/10.1371/journal.pone.0104392

[7] Gong, J., Tu, W., Liu, J., & Tian, D. (2023). Hepatocytes: A key role in liver inflammation. Frontiers in immunology, 13, 1083780. https://doi.org/10.3389/fimmu.2022.1083780

[8] Wang, Y., Li, M., Yang, T., Xie, Y., Wang, F. S., Hu, J., & Shi, M. (2024). Human umbilical cord mesenchymal stem cell transplantation for the treatment of acute-on-chronic liver failure: protocol for a multicentre random double-blind placebo-controlled trial. BMJ open, 14(6), e084237. https://doi.org/10.1136/bmjopen-2024-084237

Older Posts

Stem Cell Blog

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?

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.

A recent study published in the Lancet journal has shown that stem cells have been used to bring about significant improvements in vision in what’s being heralded as a world first.

The stem cell treatment focused on the repair of the cornea which becomes damaged in limbal stem-cell deficiency (LSCD), a disease that can lead to blindness.

It’s hoped that these findings could have a huge impact on how those with the disease are treated and that it could underpin future therapy options for those suffering from sight loss. [1]

What is LSCD?

Problems with limbal stem cells can lead to the epithelial breakdown of the cornea resulting in inflammation, scarring and potential vision loss.

LSCD has a variety of causes ranging from genetics to acquired causes like inflammation, infection, and trauma and injury. Management of the disease differs depending on the stage of its progression.

At the early stage, managing symptoms can be sufficient to alleviate its impact on quality of life. However, more progressed instances of LSCD require surgery which usually means transplants from key parts of donor eyes. [2]

What did the stem cell study involve?

The study focused on four patients, two men and two women, aged between 39 and 72 who had all been diagnosed with LCSD in both eyes.

Researchers then derived induced pluripotent stem cells (iPSCs) from donated cord blood and used to fabricate corneal epithelial stem-progenitor cells. These were then cultured and transformed into a thin sheet, iPSC-derived corneal epithelial cell sheets (iCEPS).

After removing a layer of scar tissue covering the cornea in one eye in each of the patients, the iCEPS sheet was transplanted on top and covered with a contact lens to protect the graft.

Patients were then monitored continuously to determine safety outcomes for a period of two years. [3]

What were the results of the stem cell study?

Throughout the whole of the two year safety observation period no serious adverse events occurred. The transplant was accepted by the patients without rejection and without tumour formation. In fact, researchers reported that two of the patients even forwent immunosuppressant drugs.

Following the transplant, all four patients saw immediate improvements in their vision during the first year and three out of four patients experienced sustained improvements in their vision and quality of life beyond one year.

These results are hugely encouraging and the research team, based at Osaka University in Japan, hope to move to a larger scale clinical trial to verify their promising findings. [4]

What does this mean for cord blood banking?

As the results of this trial show, stem cells have huge regenerative potential and are at the forefront of medicine, with researchers still coming to terms with the breadth of their applications.

While this study focused on the use of induced pluripotent stem cells derived from cord blood, other types of stem cells can be found in the umbilical cord and placenta, such as haematopoietic and mesenchymal stem cells, that similarly have enormous potential in the field of regenerative medicine.

If you or someone you know is expecting and wants to know more about the power of stem cells and how they can be stored for future use, fill out the form below to request a free Welcome Pack.

References

A recent story published by The New York Times has highlighted the life-changing potential placenta holds for the treatment of severe burns and wounds.

The story follows Marcella Townsend, who was left unrecognisable by second and third degree burns she suffered after a propane explosion at her mother’s house in Georgia.

With few treatment options available, Marcella underwent a radical, cutting-edge procedure: a human placenta graft.

Incredibly, Marcella’s face now looks nearly identical to how it did before the explosion. [1]

What is a human placenta graft?

A placenta graft involves the application of amniotic membrane – the thin, translucent inner part of the placenta surrounding the foetus in the womb – to an area affected by burns, wounds, or injuries.

Unlike skin grafts using animals or cadavers, placental tissue doesn’t trigger the immune system, meaning it isn’t rejected by the body – a byproduct of the immunological privilege it has between mother and child during pregnancy.

It also stimulates rapid cell regrowth, which can help the body heal with minimal scarring. [2]

What makes the placenta so special?

The placenta provides crucial nutrients and protection to babies during pregnancy, and is filled with proteins and growth factors that make it extremely useful for healing injuries. [3]

Since the early 20th century, the amniotic membrane from placentas has been used in topical treatments for burns, wounds, and ulcers. It’s even been used to treat eye conditions. [4]

And yet, despite their potential, most placentas are discarded as medical waste.

Today in the U.S. only publicly stored donated placentas from elective C-sections are used. [1]

In the latter context, a placenta graft again proved to be life-changing.

As cited by The New York Times, an 83 year old woman had an infected, chronic wound on her leg that wouldn’t heal after surgery. Having exhausted treatment options – including the use of larvae to eat the infected areas – doctors turned to placenta-derived skin grafts to close the wound.

The woman’s leg healed completely.

While the medical potential of the placenta seems boundless – they have been found to heal burns, wounds, and even restore vision in some cases – their lack of availability, along with the cost of associated procedures, means they remain vastly underused in the medical field. [1]

Why aren’t placentas more widely used?

While it’s clear that placentas have huge potential in a wide range of medical contexts, current therapeutic applications don’t come cheap. It is estimated that the cost of a placental graft ranges between $200 and $3,000 per square centimetre of amniotic membrane in the U.S. [1]

In the UK, amniotic membrane grafts are similarly pricey, with a document from the National Institute for Health and Care Excellence (NICE) showing a 2cm x 3cm graft costing around £1,000 for a single use. [5]

With the street worth of placentas estimated to be around $50,000 – a figure that’s expected to double or even triple over the course of the next decade [6] – it’s no wonder some mothers are looking to privately store their own placentas.

What is private placenta storage?

Private placenta storage is the process by which mothers can choose to store their placenta so that it’s available for their child to use in future therapies, should they need it.

There are hundreds of clinical trials ongoing that are exploring the uses of placenta in treatments for conditions ranging from stroke to diabetes, Crohn’s and osteoarthritis. By saving the placenta, the powerful cells and growth hormones it contains are preserved for future use. [7] [8] [9] [10]

Private placenta storage can be considered to be part of what’s known as cord blood banking – the process by which the perinatal cells and tissues that naturally occur in the umbilical cord and placenta are cryogenically frozen.

Some of these perinatal cells are otherwise known as stem cells, and have many therapeutic benefits. Some are even being used today in over 80 approved treatments.

Saving the umbilical cord and placenta for your baby gives them the best chance to access future therapies using stem cells and other perinatal cells and tissues.

This is because cells from the umbilical cord and placenta are baby’s own perfect match, meaning the risk of rejection in a procedure like a transplant is minimal.

For people like Marcella Townsend, the life-changing potential of the placenta is clear. By storing for your baby, you could ensure that they too have access to future therapies utilising the power of the placenta.

References

The results reported from Phase II of the first FDA-approved trial for the treatment of multiple sclerosis (MS) using stem cells are highly promising, suggesting that stem cells could be a viable treatment option to help improve the lives of those with the condition. [1]

What is the MS trial?

The Phase II trial, which was carried out by The Tisch MS Research Center of New York, constitutes the next stage in the development of the groundbreaking research signalled by the results from Phase I, the first FDA-approved trial to explore the injection of stem cells for MS.

The Phase II trial involved 54 patients split between a test group and a control group in a randomised, double-blind, placebo-controlled study.

Members of each group received 6 injections of either autologous (meaning their own) mesenchymal stem cell-derived neural progenitors (MSC-NPs) or saline placebo every two months.

These injections were administered into the cerebrospinal fluid of multiple sclerosis patients.

The study utilised a cross-over model by which patients who received the stem cell injections in year 1 were then given the saline placebo in year 2 and vice versa.

How does the stem cell treatment work?

Building on their findings from Phase I, researchers identified MSC-NPs for the treatment having ascertained that they promote tissue regeneration and immunomodulatory effects, in addition to being safe and well tolerated. [2]

MSC-NPs are a subpopulation of mesenchymal stem cells that can upregulate growth factors like hepatocyte growth factor (HGF), and minimise ectopic differentiation – essentially, the abnormal differentiation of cells.

What were the results?

The results of the Phase II trial demonstrated huge promise for the use of stem cells in treating MS.

Patients requiring walking assistance saw significant improvements in both a timed 25-foot walk test and a 6 minute walking test than those in the control group.