Studies & Research
on Stem Cells
Stem Cell Research – Knowledge at a Glance
Stem cells are a key topic in modern medicine. They open up new possibilities in the treatment of diseases and in regenerative medicine. Numerous studies are being conducted worldwide to make the best possible use of their potential. On this page, you will find a selection of current scientific works on stem cells – summarised in a compact and easily understandable format.
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New Therapy for Type 1 Diabetes: Islet Cells from Stem Cells
A novel therapy using islet cells derived from stem cells could, for the first time, enable patients to produce their own insulin again – and in many cases even live without insulin injections.
What happens in Type 1 diabetes?
More than eight million people worldwide are affected. In this disease, the immune system destroys the cells in the pancreas that normally produce insulin. Without insulin, the body can no longer properly regulate blood sugar. Patients therefore need to inject insulin for life.
Limitations of previous treatments
Sometimes a transplantation of islet cells or even entire pancreases helps. But there are too few donor organs, quality is inconsistent, and often several transplants are needed.
A new idea: cells from the lab
Researchers have now developed a new method. They artificially produced islet cells from stem cells in the laboratory. These are called zimislecel. In an initial clinical trial in 2025, 14 people with Type 1 diabetes received these cells via an infusion into the liver. All were monitored for at least twelve months.
The results of the study
The transplanted cells engrafted, produced insulin, and functioned similarly to healthy pancreatic cells. In all participants who received the full dose, blood sugar control improved significantly. The important long-term marker HbA1c dropped below seven percent, blood glucose levels remained in the healthy range for most of the day, and severe hypoglycemia no longer occurred. Many patients needed far less insulin, and some none at all. After twelve months, more than 80 percent of the fully treated group were completely independent of insulin injections.
Side effects and safety
Side effects did occur, but were mostly mild to moderate. Participants often reported gastrointestinal complaints, headaches, or skin rashes. In three individuals, the number of certain white blood cells dropped more markedly. Nevertheless, no one had to discontinue the study, indicating an overall acceptable safety profile.
Why this matters
For the first time, it has been shown that beta cells derived from stem cells can not only be produced in the lab but also successfully used in humans. If confirmed in larger studies, this could fundamentally change the treatment of Type 1 diabetes. Many patients would no longer depend on donor organs and could have the chance to live permanently without insulin injections.
Looking ahead
A large five-year follow-up study is already underway. It will investigate how safe and effective the treatment is in the long term and whether the effect persists. Although questions remain, experts see this as a decisive step toward a therapy that could tackle Type 1 diabetes at its root.
Reference:
Reichman TW, Markmann JF, Odorico J, et al.: VX-880-101 FORWARD Study Group. Stem Cell-Derived, Fully Differentiated Islets for Type 1 Diabetes. N Engl J Med. June 20, 2025.
https://www.nejm.org/doi/full/10.1056/NEJMoa2506549 -
Influence of Newborns and Placenta on the Quality of Umbilical Cord Blood
Many parents are not aware that their baby’s umbilical cord blood is full of valuable stem cells. These can later be used in the treatment of serious diseases. However, not all cord blood is equally suitable. A recent study examined which factors related to the mother, child, and placenta influence quality—with fascinating results.
Why Cord Blood Is Important
Over the past 30 years, cord blood has become increasingly significant. It contains valuable stem cells that can help treat many serious diseases, such as certain blood disorders or in the field of regenerative medicine. Compared to other sources of stem cells, cord blood offers many advantages: it can be collected immediately after birth without risk to mother or child, the procedure is painless, and the cells are often easier to use because they require less stringent matching with the recipient.
What Researchers Wanted to Investigate
For cord blood to be stored in a blood bank, its quality must meet certain standards. This includes measuring how many cells the blood contains and how much blood can be collected in total. A study in Egypt investigated which factors in the mother, child, and placenta influence the quality of cord blood.
How the Study Was Conducted
The researchers observed 150 cesarean deliveries. Only mothers between the ages of 18 and 40 were included in the study. Immediately after birth, the cord blood was collected and analyzed. The volume of blood obtained and the number of stem cells it contained were measured.
What the Results Showed
It turned out that the size and weight of the baby and placenta played a major role. Babies with a birth weight of at least three kilograms generally provided more and higher-quality cord blood. A heavier placenta and a longer umbilical cord were also associated with better quality. Another notable finding was that male babies, on average, yielded more cord blood than female babies, although the number of important stem cells was the same. The weight of the placenta proved to be the most reliable indicator of cord blood quality.
Why This Matters
The results show that certain characteristics of the child and placenta can predict whether cord blood will be especially suitable for storage. If blood banks use this information, they can make more targeted decisions about which donors’ cord blood to collect. This saves costs, increases efficiency, and ensures that more high-quality blood is available for patients.
Conclusion
The study highlights that the baby’s and placenta’s weight or the length of the umbilical cord can already provide valuable clues about whether the cord blood will later be useful. With this knowledge, blood banks can make better decisions and ultimately improve medical care.
Reference:
Darwish, A., Bassiouny, M.R., Mansour, A.K. et al. Neonatal factors impacting umbilical cord blood unit characteristics. Sci Rep 15, 16776 (2025).
https://www.nature.com/articles/s41598-025-96829-3 -
NK Cell and CAR NK Cell Therapy: Current Advances and Perspectives
In recent years, so-called NK cells—special immune defense cells—have attracted significant attention in cancer research. What makes them especially promising is that they can be expanded in the lab and even genetically engineered to specifically attack tumors. These therapies, often referred to as NK cell or CAR NK cell therapies, are considered highly promising because they are effective and generally cause fewer side effects than other forms of immunotherapy.
Research and Current Evidence
Natural killer (NK) cells are a special type of immune cell that can rapidly attack cancer cells or virus-infected cells. Unlike T cells, they require no prior “training.” For over 30 years, researchers have studied how to expand these cells in the laboratory and return them to patients to treat diseases such as cancer, autoimmune disorders, or infections.
A major breakthrough was the development of genetically modified NK cells, known as CAR NK cells. The first clinical trial began in 2009. Since then, the field has grown enormously: as of April 2025, more than 380 studies testing NK cell infusions are registered on ClinicalTrials.gov, with over 60 of them having already published results.Differences Compared to T Cells
NK cells offer several advantages over T cells. They can recognize tumors both via their natural receptors and via the engineered CAR receptor. This lowers the risk that tumors will “escape” by altering their appearance. Moreover, NK cells rarely trigger rejection reactions such as the feared graft-versus-host disease (GvHD). Even when donor and recipient cells are not perfectly matched genetically, the therapy generally remains safe. To date, GvHD has been observed after NK cell infusion in only a single clinical case—despite thousands of such treatments.
Limitations and Possible Solutions
One drawback is that NK cells survive only briefly in the body. To extend their activity, researchers are working on equipping the cells with additional signaling molecules such as IL-15. Genetic modification of NK cells is also technically more challenging than with T cells, which slows progress.
Medical Applications
NK cells are especially promising for blood cancers in the context of stem cell transplantation. They can improve graft acceptance, lower the risk of GvHD, and prevent relapse. NK cells have also shown effectiveness against neuroblastoma, an aggressive childhood tumor—particularly in combination with anti-GD2 antibodies. Two studies tested haploidentical (partially matched) NK cells together with anti-GD2 chemotherapy in children with relapsed disease.
Manufacturing and Practical Challenges
To make NK cell therapy widely accessible, safe large-scale production methods are needed. One promising approach uses so-called K562-mbIL21-4-1BBL feeder cells. In early-phase (I/II) studies, this method enabled more than 450 infusions in over 150 patients—at doses up to 3 × 10⁸ cells per kilogram of body weight. These treatments demonstrated safety and early signs of efficacy, without dose-limiting toxicities. However, major obstacles remain: production is lengthy, costly, and identifying suitable donors is complex.
Who Makes a Good Donor?
The effectiveness of NK cells also depends on the donor’s genetic makeup. Certain gene combinations make cells more alloreactive—that is, more potent against tumors. Donors carrying KIR genes 2DL1, 2DL2 or 2DL3, and 3DL1 together with HLA features C1, C2, and Bw4 are considered particularly effective for many recipients. However, if a patient already has all three HLA types, no donor can provide a full anti-tumor effect through this pathway—other NK cell mechanisms must then be leveraged. Some genetic profiles are regarded as a kind of “universal donor type” for NK cells, similar to blood group O in blood transfusions.
Outlook
Current evidence demonstrates that NK and CAR NK cell therapies are safe and hold great potential. Nevertheless, much remains to be done: better strategies for cell production, smarter clinical trial designs, and new technical solutions are needed to overcome NK cells’ natural limitations. Advances in manufacturing, donor selection, and enhancement of cell functions will be crucial for these therapies to fully realize their potential.
Reference:
Caporale JR, Naeimi Kararoudi M, Lamb MG, Lee DA. Dark NKnight rising: a current perspective on NK cell and CAR‑NK cell therapy. Cytotherapy. 2025 Jul;27(7):812‑825. DOI/Link: https://www.isct-cytotherapy.org/article/S1465-3249(25)00687-5/fulltext
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Cord Blood Helps Children with Cerebral Palsy
An increasing number of studies show that cord blood can help children with cerebral palsy. A new large-scale investigation now confirms that this treatment significantly improves motor function, offering affected families an important additional opportunity.
New Hope for Children
The Parents Guide to Cord Blood Foundation reports a major advance: cord blood has proven effective in the treatment of cerebral palsy. The basis is a large study published in the journal Pediatrics. Data from more than 400 children across 11 different studies were pooled to provide a clearer picture.
Study Design
Children who received additional medications were excluded from the analysis. This left 170 children who received cord blood and 171 who underwent standard rehabilitation only. The results were clear: children treated with cord blood showed significantly greater improvements in motor skills than those receiving standard therapy alone.
Who Participated
Most of the children (90%) had the most common form of cerebral palsy, known as spastic CP. The average age was just under five years, ranging from infants to adolescents. In nearly all studies, the cord blood was administered intravenously, with only one study using an injection into the spinal canal. On average, children received 56.1 million cells per kilogram of body weight. The majority of treatments (84%) used donor blood rather than the child’s own.
Significant Improvements
After six months, the treated children showed an average improvement of 1.36 points on a standard motor skills test compared to the control group. After twelve months, the advantage was 1.42 points. Both results were statistically significant.
Which Children Benefited Most
The therapy worked especially well in children who received higher cell doses, were under five years of age, and were already attempting to walk before treatment. Researchers emphasize that early diagnosis and intervention are crucial, since the young brain has the highest ability to adapt.
Why Cord Blood Works
Researchers assume that the cells do not directly form new nerve cells but instead release substances that reduce inflammation, support the brain’s self-healing, and strengthen connections between nerve cells. This likely explains the observed improvements in motor function.
A Turning Point in Treatment
The findings show that cord blood therapy can no longer be considered merely experimental. It measurably improves the daily functional abilities of affected children. Experts now hope that more families will gain access to this treatment once additional studies confirm the results.
References:
Parents Guide to Cord Blood Foundation: https://parentsguidecordblood.org/en/news/cord-blood-proven-effective-cerebral-palsy
Finch‑Edmondson M, Paton M, Webb A, Ashrafi M, Blatch‑Williams R, Cox J, Charles, Novak I. Cord Blood Treatment for Children With Cerebral Palsy: Individual Participant Data Meta‑Analysis. Pediatrics. 2025;155(5):e2024068999. https://publications.aap.org/pediatrics/article/155/5/e2024068999/201565/Cord-Blood-Treatment-for-Children-With-Cerebral
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The Hidden Power of the Placenta
The placenta accompanies every life only during pregnancy and is usually overlooked afterward. Yet research in recent years has made it clear: this organ holds great potential for new medical therapies and healing methods.
A Forgotten Organ in the Spotlight
More and more researchers are discovering the placenta as a source of new treatment options. In the July 2025 issue of Bioactive Materials, a comprehensive review highlighted the many ways in which this organ can be used. For a long time, the placenta was regarded as waste after birth, but today it is moving to the forefront of modern medicine.
Why the Placenta Is Special
During pregnancy, the placenta fulfills vital functions for the child. Beyond that, it has valuable properties: its tissue is richly supplied with blood, structurally stable, and abundantly available. This makes it particularly well-suited to support healing processes and to serve as a basis for developing new medical materials.
From Skin Protection to Ophthalmology
The review describes several ways in which placental components are already being used or could be applied in the future. These include creams, injections, and capsules enriched with placental extract that may support skin healing. Freeze-dried placental membranes are also being explored for wound treatment. Moreover, specific placental layers—such as the amniotic and chorionic membranes—are being studied for their regenerative and anti-inflammatory properties.
Particularly exciting is the field of ophthalmology, where so-called cord blood serum is attracting attention. Studies show that it works better than conventional treatments such as artificial tears or blood serum. It may soon play an important role in treating dry eye disease and corneal damage.
A Toolbox for the Future
Beyond existing applications, new approaches have the potential to fundamentally change medicine. These include tiny particles derived from placental cells that can act as carriers for therapeutic agents, as well as tissue scaffolds from the placenta that can serve as natural support structures in transplants.
This diversity makes the placenta a kind of toolbox for research and clinical practice. Already, it is drawing attention in skincare and aesthetic medicine, and in the future, it could play a central role in healing and cell therapies.
From Waste to Source of Hope
Just a few decades ago, the placenta was typically discarded after birth. Today, scientists see it as a valuable, ethically uncontroversial, and biologically rich resource. With further research and responsible use, it may soon become an integral part of modern treatments.
References:
Moghassemi S, Nikanfar S, Dadashzadeh A, Sousa MJ, Wan Y, Sun F, Colson A, De Windt S, Kwaspen L, Kanbar M, Sobhani K, Yang J, Vlieghe H, Li Y, Debiève F, Wyns C, Amorim CA. The revolutionary role of placental derivatives in biomedical research. Bioact Mater. 2025 Mar 19;49:456-485. https://www.sciencedirect.com/science/article/pii/S2452199X25001136
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Highlights from the 31st ISCT Annual Meeting: Innovations in Cell and Gene Therapy
Each year, the International Society for Cell and Gene Therapy (ISCT) brings together researchers, clinicians, and industry representatives from around the world. At the 31st Annual Meeting, held in May 2025 in New Orleans, the focus was on new developments with the potential to fundamentally change the treatment of serious diseases.
Key highlights of the 31st ISCT Annual Meeting
From May 7–10, 2025, the 31st ISCT Annual Meeting took place in New Orleans, USA. The latest advances in cell and gene therapies were presented. The full program is available online, and all scientific contributions were published in the journal Cytotherapy. Below is a selection of particularly notable findings.
The meeting highlighted how rapidly the field is evolving—from new manufacturing techniques and patient-specific therapies to novel approaches in tissue repair. Key questions for the future include how therapies can be made globally accessible, individually tailored, yet manufactured in a standardized way.
Standardized Quality in Cell Therapies
One contribution addressed the challenge that cell therapies often vary in effectiveness because the cells come from different donors. A new procedure was presented that allows cell quality to be better assessed and standardized. Tests showed that nearly 7,000 genes displayed different activity when cells were specially pre-processed. These findings could help make the production of cell-based products more consistent and reliable in the future.
Genetic Differences in ALS Patients
A large study involving 189 patients with amyotrophic lateral sclerosis (ALS) investigated whether the effectiveness of a specific cell therapy depends on patients’ genes. 62% of participants carried a genetic variant linked to higher ALS risk. This group—particularly those with a certain genotype—responded significantly better to treatment (65% vs. 29% under placebo, p = 0.011). The results suggest that in the future, it may be possible to tailor cell therapies based on genetic profiles.
New Approaches to Wound Healing
An experimental study introduced a special scaffold for use in wounds. Made from natural materials such as alginate and chitosan, it was enriched with specific cells that promote healing. In animal models of chronic wounds, injuries healed faster, and the new tissue was of higher quality. Laboratory tests also showed that the scaffold had anti-inflammatory and antibacterial properties, supporting healing.
One-Day CAR-T Cell Therapy
A research team presented a new platform enabling the production of CAR-T cells in under 24 hours. These engineered cells are used to treat certain cancers. In an initial study of 10 patients, plus validation with 3 additional participants, all 13 production runs met quality standards. The cells remained 94% viable on average, and fewer side effects occurred (15% fever reactions, 8% neurological events) compared to conventional CAR-T therapies. If validated, this approach could significantly accelerate global access to such treatments.
Real-World Results in Severe GvHD
A large analysis examined 242 adults in Germany, Hungary, and France between 2017 and 2024 with severe graft-versus-host disease (GvHD), a life-threatening complication after bone marrow or stem cell transplantation. Patients received therapy with a product derived from donated bone marrow cells. Even in those who did not respond to standard treatments, the therapy showed a favorable safety profile and encouraging efficacy at days 28, 60, and 180 after initiation. While results are not yet final, they form the basis for ongoing international trials assessing whether this treatment can improve survival compared to the best available therapies.
References
Program/Overview:
https://indd.adobe.com/view/bdbe9517-de33-4950-81cb-88e54d9911adCytotherapy-Supplement:
https://www.sciencedirect.com/journal/cytotherapy/vol/27/issue/5/suppl/SFurther abstracts:
https://www.sciencedirect.com/science/article/abs/pii/S1465324925000921
https://www.sciencedirect.com/science/article/abs/pii/S1465324925000933
https://www.sciencedirect.com/science/article/abs/pii/S1465324925000969
https://www.sciencedirect.com/science/article/abs/pii/S1465324925001021
https://www.sciencedirect.com/science/article/abs/pii/S1465324925001124 -
Mesenchymal Stem/Stromal Cells in the Treatment of Chronic Wounds: A Promising Future for Regenerative Medicine
Chronic wounds are a major problem for many people because they often fail to heal for months or even years, severely limiting quality of life. New research shows that certain stem cells can support healing and open up entirely new treatment possibilities.
New Options for Treating Chronic Wounds
Chronic wounds are injuries that do not heal over a long period of time. They remain a serious challenge in medicine. Patients suffer greatly from the limitations, and healthcare systems are heavily burdened. Estimates suggest that in Europe about 1.5–2 million people, and in the United States around 5 million people, are affected by such wounds.
What Are Stem Cells and Why Are They Interesting?
A specific type of stem cells—called mesenchymal stem/stromal cells (MSCs)—offers new opportunities for wound healing. Stem cells are unique cells that can differentiate into various tissue types, thereby supporting natural healing processes. Scientific studies describe their important role at multiple stages of wound healing. They also help maintain skin homeostasis.
How Stem Cells Support Healing
MSCs aid the body in repairing damaged tissue by reducing inflammation, regulating immune responses, and stimulating the growth of new cells. They release signaling molecules that trigger several processes: attracting skin cells, forming new tissue, regenerating blood vessels, and covering wounds with new skin.
Recent studies also show that these cells can act against pathogens. They enhance the body’s immune defense against bacteria and can produce specific proteins that kill microbes. Some laboratory research even suggests that they may directly attack bacteria.
Research Findings
There are now many scientific studies testing MSCs for the treatment of chronic wounds, including diabetic ulcers, burns, pressure ulcers, bone-healing disorders, and rare diseases such as epidermolysis bullosa.
According to the U.S. National Library of Medicine database, as of 20. September 2024, four clinical trials of MSC therapies for chronic wounds were planned or ongoing, one specifically targeting patients with epidermolysis bullosa. At that time, 28 studies had already been completed, including five on this rare condition.
Opportunities and Open Questions
Most of these studies show positive results: patients experienced improved wound healing. However, experts emphasize that larger trials are still needed to determine the best treatment strategies. Different delivery methods have been tested so far: directly applying the cells to the wound, injecting them into the bloodstream, or combining them with carriers such as gels, membranes, or skin grafts.
Looking Ahead
New technologies such as 3D tissue printing and tissue engineering are particularly exciting, as they may significantly enhance the effectiveness of stem cell therapies. The ultimate goal is to transform the management of chronic wounds and improve healing outcomes for millions of patients.
References:
Nasadiuk K, Kolanowski T, Kowalewski C, Wozniak K, Oldak T, Rozwadowska N: Harnessing Mesenchymal Stromal Cells for Advanced Wound Healing: A Comprehensive Review of Mechanisms and Applications. Int. J. Mol. Sci. 2025, 26, 199.
https://www.mdpi.com/1422-0067/26/1/199 -
New Treatment for Knee Osteoarthritis: Umbilical Cord Cells More Effective than Cortisone
Knee osteoarthritis affects millions of people worldwide. Standard therapies such as painkillers or cortisone often provide only temporary relief. Researchers have therefore been exploring new approaches—and a recent study shows that umbilical cord cells may offer longer-lasting benefits.
Background
Knee osteoarthritis is the most common joint disease. It causes pain, restricted mobility, and a significant reduction in quality of life. In recent years, researchers have investigated whether cells from the umbilical cord—so-called stem cells—can provide more sustained symptom relief.
Study Design
In February 2025, the journal Cytotherapy published a clinical pilot study involving 30 patients with knee osteoarthritis. Participants received either a single injection of umbilical cord cells or a cortisone injection (triamcinolone, 10 mg/ml). The number of umbilical cord cells administered was around five million—lower than in many other studies.
Results
Effectiveness was measured using the WOMAC score, which assesses pain, stiffness, and mobility. After three months, patients in the cord cell group reported more than double the improvement compared to baseline, while those in the cortisone group experienced only minor relief. After six months, the positive effects in the cord cell group persisted nearly unchanged, while the cortisone group’s condition had returned close to baseline. By nine months, cortisone’s effect had almost completely worn off, whereas patients treated with cord cells continued to report significant pain relief and improved mobility.
Conclusion
The results indicate that a single treatment with umbilical cord cells for knee osteoarthritis can be both safe and effective. Compared to cortisone, this method provides longer-lasting pain relief and improves mobility for many months.
References
Pico OA, Espinoza F, Cádiz MI, Sossa CL, Becerra-Bayona SM, Salgado MCC, Rodríguez JER, Cárdenas OFV, Cure JMQ, Khoury M, Arango-Rodríguez ML: Efficacy of a single dose of cryopreserved human umbilical cord mesenchymal stromal cells for the treatment of knee osteoarthritis:a randomized, controlled, double-blind pilot study. Cytotherapy. 2025 Feb;27(2):188-200.
https://www.isct-cytotherapy.org/article/S1465-3249(24)00883-1/abstractSadlik B, Jaroslawski G, Gladysz D, Puszkarz M, Markowska M, Pawelec K, Boruczkowski D, Oldak T: Knee Cartilage Regeneration with Umbilical Cord Mesenchymal Stem Cells Embedded in Collagen Scaffold Using Dry Arthroscopy Technique. Adv Exp Med Biol. 2017;1020:113-122.
https://link.springer.com/chapter/10.1007/5584_2017_9 -
Early Isolation and Cryopreservation of NK Cells from Umbilical Cord Blood Increases Their Effectiveness
Research increasingly shows that umbilical cord blood is a valuable resource for medicine. Of particular interest are certain immune cells that may play an important role in treating diseases such as cancer. New studies provide insights into how these cells can best be harvested and preserved to maximize their future therapeutic potential.
Umbilical Cord Blood as a Source of Immune Cells
Umbilical cord blood contains special immune cells known as natural killer (NK) cells, which play an important role in the immune system. To use them in therapy, however, it is necessary to obtain large numbers of these cells while ensuring they remain functional.
Differences Between Donors
At an international conference in 2024, researchers showed that the performance of these immune cells varies significantly between individuals. They also differ in how well they can be expanded. Compared to immune cells from peripheral blood, cord blood NK cells show greater variability in growth but display superior properties when it comes to homing to the bone marrow—a crucial step for their activity in the body.
Importance of Timing
The team led by M. Kennedy demonstrated that the timing after collection is critical. The longer the delay before processing, the worse the cells can later be expanded. When cord blood is frozen immediately after collection, the NK cells expand significantly better (statistically confirmed, p<0.05). Importantly, early freezing does not impair the cells’ function; they retain their therapeutic properties.
Conclusion
Immediate cryopreservation of umbilical cord blood after birth could be a decisive step in enabling these immune cells to become even more effective in future cancer and immunotherapy applications.
References:
Kennedy, W. Patterson, S.T. Cox, R. Danby, D. Hernandez. Early isolation and cryopreservation of NK cells from fresh cord blood, enhances their subsequent performance. Abstracts of the 30th Annual ISCT Meeting May 29-June 1, 2024. Cytotherapy 2024, 26 (6), S176.
https://www.sciencedirect.com/science/article/abs/pii/S1465324924004353Maddineni S, Silberstein JL, Sunwoo JB. Emerging NK cell therapies for cancer and the promise of next generation engineering of iPSC-derived NK cells. J Immunother Cancer. 2022 May;10(5):e004693. doi: 10.1136/jitc-2022-004693. Erratum in: J Immunother Cancer. 2022 Sep;10(9).
https://pmc.ncbi.nlm.nih.gov/articles/PMC9115029/ -
Review of the Virtual World Cord Blood Day Conference
Cord blood contains valuable cells that can save lives. Every year on 15. November, World Cord Blood Day draws attention to this fact. In 2024, a virtual conference was held where experts from around the world shared the latest developments and inspiring success stories.
On 15 November, World Cord Blood Day is celebrated each year. On this day, global awareness is raised about the great potential of cord blood. In 2024, a virtual conference took place on this occasion, presenting the latest insights into transplants and new treatment methods. The event was supported by international organizations such as the Cord Blood Association and the Save the Cord Foundation.
Cord Blood in the Classroom
The conference began with a session titled “Cord Blood in the Classroom.” According to the Cord Blood Association, more than 60,000 cord blood transplants have been performed worldwide since 1988. What makes it special: cord blood contains cells that are now used as a standard treatment for more than 80 blood and metabolic diseases. But research goes further. Studies are currently underway to investigate whether cord blood could also help in conditions such as autism, spinal cord injuries, diabetes, HIV, or strokes.
From the Beginnings to Today
A highlight of the event was the lecture by Dr. Joanne Kurtzberg of Duke University. She looked back on the history of cord blood transplants. In the 1980s, Hal Broxmeyer discovered the unique cells in cord blood. The first successful transplant took place in 1988 in France between siblings, followed in 1993 by the first unrelated donor transplant in the United States.
Today, there are more than 160 cord blood banks worldwide. They store over 800,000 samples for public use and more than 6 million for private use. Each year, around 2,000 to 3,000 transplants are performed. Dr. Kurtzberg emphasized that the success of treatment largely depends on the number of cells contained in a sample.
Important Studies and New Insights
Dr. Kurtzberg recalled groundbreaking scientific work. A 2014 study (Wagner et al.) showed that a single cord blood sample with the right dosage can achieve better results in treating blood cancer than combining two samples. Another study from 2020 compared transplants from partially matched donors with those from cord blood. The result: the former are on the rise but carry a higher risk of relapse, while cord blood transplants are associated with higher treatment-related mortality.
Successes in Rare Diseases
Particularly impressive were the long-term results of patients with rare genetic diseases such as Hurler syndrome, Krabbe disease, or metachromatic leukodystrophy. Dr. Kurtzberg reported on two girls who received cord blood transplants for Hurler syndrome at the age of just one. Both are now healthy, live normal lives, and are close to completing their university studies.
More Than Just Cells
Dr. Kurtzberg emphasized that a cord blood sample has far more potential than just the well-known transplant cells. Additional products are now being developed from it. These include special immune cells that could be used in brain injuries, or so-called macrophage products such as DUOC-01, which are intended to support nerve cell repair. Particularly exciting: certain cells from cord blood can rescue damaged brain cells after oxygen deprivation – an effect that does not occur with comparable adult cells.
Looking Ahead
The conference concluded with presentations of current clinical trials on the treatment of cerebral palsy and autism. All these developments show that cord blood is no longer used solely for transplants. It is evolving into a versatile tool that offers new hope for many diseases and opens up exciting prospects for the future.
References:
https://www.worldcordbloodday.org/
Wagner JE et al. Blood and Marrow Transplant Clinical Trials Network. One-unit versus two-unit cord-blood transplantation for hematologic cancers. N Engl J Med 2014 Oct 30;371(18):1685-94. https://pubmed.ncbi.nlm.nih.gov/25354103/
Wagner, John E. et al. Comparison of Haploidentical Related Donor with Post-Transplant Cyclophosphamide (PTCy) and Umbilical Cord Blood (UCB) Transplantation after Myeloablative Conditioning for Hematological Malignancy. Biology of Blood and Marrow Transplantation, Volume 26, Issue 3, S291 www.astctjournal.org/article/S1083-8791(19)31495-8/fulltext
Escolar ML et al. Transplantation of umbilical-cord blood in babies with infantile Krabbe’s disease. N Engl J Med. 2005 May 19;352(20):2069-81. www.nejm.org/doi/full/10.1056/NEJMoa042604
Kwon JM et al. Consensus guidelines for newborn screening, diagnosis and treatment of infantile Krabbe disease. Orphanet J Rare Dis. 2018 Feb 1;13(1):30. https://pubmed.ncbi.nlm.nih.gov/29391017/
Dumont-Lagacé M, Feghaly A, Meunier MC, Finney M, Van’t Hof W, Masson Frenet E, Sauvageau G, Cohen S. UM171 Expansion of Cord Blood Improves Donor Availability and HLA Matching For All Patients, Including Minorities. Transplant Cell Ther. 2022 Jul;28(7):410.e1-410.e5. https://pubmed.ncbi.nlm.nih.gov/35311667/
Saiyin T, Kirkham AM, Bailey AJM, Shorr R, Pineault N, Maganti HB, Allan DS. Clinical Outcomes of Umbilical Cord Blood Transplantation Using Ex Vivo Expansion: A Systematic Review and Meta-Analysis of Controlled Studies. Transplant Cell Ther. 2023 Feb;29(2):129.e1-129.e9. https://pubmed.ncbi.nlm.nih.gov/36396108/
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20 Years of Treating Ischemic Cardiomyopathy with Mesenchymal Stem/Stromal Cells
Developing new therapies for severe heart disease has been a major research goal for many years. In ischemic cardiomyopathy, a form of heart failure, physicians have long sought ways to improve damaged heart function. For two decades, researchers have been investigating whether special cells could help.
The Disease
Ischemic cardiomyopathy is a common and very serious heart condition. Reduced blood supply damages the heart, leaving it unable to pump enough blood through the body. The heart has only a very limited capacity for self-repair.
The Treatment Approach
For about 20 years, researchers have been studying whether certain cells—known as stem or stromal cells—can help repair damage to the heart muscle. These cells are derived from bone marrow, umbilical cord, or adipose (fat) tissue. The goal is to strengthen heart function and reduce patient symptoms.
The Evidence from Studies
A large review of 49 clinical studies with a total of 1,408 participants has summarized the findings to date. Follow-up times ranged from 1 to 10 years, depending on the study. Bone marrow–derived cells were used most frequently (59.2%), followed by umbilical cord cells (16.3%) and adipose tissue cells (12.2%). In most cases, the cells came from the patients themselves (59.2%). Donor cells were used in 38.8% of cases, and in 2% a combination of both.
The cells were administered in different ways: through the vein, via the coronary arteries, or injected directly into the heart muscle. In some cases, administration was combined with heart surgery, such as bypass surgery.
The Results
The studies showed clear improvements. The pumping ability of the heart—measured as left ventricular ejection fraction—increased by an average of 5.75 percentage points (p < 0.0001). Quality of life also improved: in nearly two-thirds of patients, the walking distance in the six-minute walk test increased, and in more than half, the severity of heart failure improved according to the NYHA classification.
Limitations of Current Research
Despite these encouraging results, there are limitations. In nearly a quarter of the studies (22.4%), comparison groups were missing, which reduces the strength of the findings. Experts therefore emphasize the urgent need for larger, well-controlled studies. Only then can it be determined whether this treatment truly has the potential to enter widespread clinical use.
Reference:
Seyihoglu B, Orhan I, Okudur N, Aygun HK, Bhupal M, Yavuz Y, Can A. 20 years of treating ischemic cardiomyopathy with mesenchymal stromal cells: a meta-analysis and systematic review. Cytotherapy. 2024 Dec;26(12):1443-1457. https://pubmed.ncbi.nlm.nih.gov/39078351/
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A new diagnostic panel ensures the safety of mesenchymal stem/stromal cells
Stem cell therapies are considered a promising approach for treating numerous diseases. However, concerns have repeatedly been raised that stem cells could pose a cancer risk. A new study now provides clarity and shows: mesenchymal stem/stromal cells (MSCs) are safe – thanks to a newly developed testing procedure.
New testing method confirms stem cell safety
In the December issue of the journal Human Genetics, a study by the team at the Polish Stem Cell Bank (PBKM, FamiCord Group) was published. It addresses a frequently cited concern about stem cells: the potential danger that they might cause cancer. The results, however, clearly demonstrate that so-called mesenchymal stem/stromal cells (MSCs) are safe and do not pose a cancer risk.
Why these cells are important
MSCs are considered particularly valuable because they can influence the immune system and suppress inflammation in the body. This is why they play a central role in many medical therapies. Nevertheless, there have long been concerns about whether these cells could give rise to tumors.
Existing guidelines and their limitations
The International Society for Cellular Therapy (ISCT) has already established rules for how MSCs should be cultivated and tested for medical purposes. These include methods for detecting changes in the genetic material. While such procedures provide important insights, they are not sufficient to definitively prove whether the cells are safe. This is precisely where the new PBKM team’s testing method comes in.
A new procedure for greater safety
Until now, various methods have been available to test the safety of these cells, but none were widely accepted or particularly easy to apply. To address this, the PBKM team developed a special diagnostic procedure. This method examines the molecular characteristics of MSCs and reliably shows that they are clearly distinct from cancer cells.
How the study was conducted
The research team analyzed cells from Wharton’s Jelly, a tissue found in the umbilical cord. They studied 100 carefully selected genes and compared the activity levels of these genes in three different cell types: umbilical cord MSCs, cancer cells, and induced pluripotent stem cells (iPSCs). Using established markers and a dedicated gene panel, the researchers were able to clearly demonstrate that MSCs display a distinct pattern compared to the two reference groups. From this work, a robust diagnostic panel was developed that reliably distinguishes MSCs from potentially dangerous cells.
Implications for future therapies
For the study, umbilical cord stem cells were first isolated and expanded in the laboratory. They were then analyzed in a multi-step process, including genetic profiling. The new protocol makes it possible to clearly differentiate the gene expression profiles of MSCs from those of iPSCs and various cancer cell lines.
This diagnostic panel complements the existing guidelines of the International Society for Cellular Therapy (ISCT), which sets standards for the clinical use of MSCs. It now provides a standardized way to verify the safety of these cells.
Building trust in stem cell therapies
With this development, the PBKM team has closed an important gap. The procedure increases safety for patients, clinicians, and manufacturers alike. It strengthens confidence in stem cell therapies and paves the way for their broader and more reliable application in regenerative medicine.
Reference:
Anna M. Różycka‑Baczynska, Igor M. Stepaniec, Marta Warzycha, Izabela Zdolinska‑Malinowska, Tomasz Oldak, Natalia Rozwadowska, Tomasz J. Kolanowski: Development of a novel gene expression panel for the characterization of MSCs for increased biological safety. J Appl Genetics (2024).
https://link.springer.com/article/10.1007/s13353-024-00917-5#citeas