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Medical Update – September 2026

Medical Update – September 2026

14.09.2026

18 mins of reading

FamiCord Cellular Therapy Products Released in July 2026

TOTAL NUMBER OF PATIENTS TREATED

84

Did You Know?

Highlights from this issue

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For very preterm babies:

A baby’s own cord blood cells showed encouraging five-year signals for fine motor and personal-social development.

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Beyond stem cells:

Cord blood is being explored as a possible source of transfusion products, although storage time remains a key quality challenge.

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For cerebral palsy:

Cord blood treatment is moving closer to carefully controlled clinical
programs, but wider use still needs stronger evidence and safety
pathways.

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In rheumatoid arthritis:

Cord blood-derived MSCs reduced disease activity for several months after one infusion, with larger studies needed to confirm durability.

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For pediatric heart failure:

Wharton’s Jelly MSCs delivered into coronary arteries showed early encouraging signals in two children with dilated cardiomyopathy.

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For psoriasis:

A topical MSC-derived exosome ointment was well tolerated in a first-in-human safety study.

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After severe trauma:

WJ-MSCs showed early safety and kidney recovery signals in three earthquake victims with crush syndrome.

From Birth to Age Five: Encouraging Results for Cord Blood Cell Therapy

A BABY’S OWN CORD BLOOD CELLS MAY SUPPORT HEALTHIER DEVELOPMENT IN VERY PRETERM CHILDREN

Very premature babies have lungs and brains that are still developing, so complications after birth can affect them for years. One major problem is bronchopulmonary dysplasia, a serious lung condition linked with long-term breathing difficulties.
In this study, babies born before 32 weeks of pregnancy received either their own cord blood cells shortly after birth or a placebo. Five years later, children who received the cells had better scores in fine motor and personal-social development.
Put simply, the baby’s own cord blood cells may send helpful repair signals during a very sensitive time after birth. The results are encouraging, but this approach is still experimental and needs larger studies before it can be considered a proven treatment.

Bronchopulmonary dysplasia is one of the most serious complications of very premature birth. It can leave babies with long-term breathing problems and may also affect later neurodevelopment. Because current treatment options are limited, researchers are looking at whether stem cell-based therapies could help protect the fragile lungs of very preterm infants. Early phase I and II trials have shown that this approach appears feasible and safe, with signals suggesting it may reduce the severity of bronchopulmonary dysplasia in babies at highest risk.
In August 2026, Zhuxiao Ren and colleagues reported five-year follow-up results from very preterm infants who had received an infusion of their own cord blood mononuclear cells shortly after birth. The treatment was originally tested as part of a phase II clinical trial in infants born before 32 weeks of gestation. Within the first 24 hours after delivery, babies received either a single intravenous dose of autologous cord blood mononuclear cells (5×10⁷ cells/kg) or a placebo infusion of normal saline.
The original study included 62 infants: 29 in the cord blood cell group and 33 in the control group. Earlier two-year results were already encouraging. Children who received the cell infusion had fewer pneumonia-related hospitalizations and fewer signs of neurodevelopmental delay on the Bayley developmental assessment. The intervention was also linked with a lower rate of moderate-to-severe bronchopulmonary dysplasia among surviving very preterm infants.
At five years of age, the children who had received their own cord blood cells scored significantly higher in the fine motor and personal-social domains of the ASQ questionnaire. In practical terms, this suggests better development in areas such as small hand movements, coordination, and everyday social interaction. Although more research is needed, the follow-up adds an important longer-term signal that early autologous cord blood cell infusion may support both lung-related and neurodevelopmental outcomes in very premature infants.

Key points

  • Very preterm infants face long-term lung and neurodevelopmental risks.
  • Autologous cord blood mononuclear cells were infused within 24 hours after birth.
  • At five years, treated children showed higher fine motor and personal-social scores.

Reference:
Ren Z et al. Five-year follow-up of a phase II trial of autologous cord blood mononuclear cells for bronchopulmonary dysplasia prevention in very preterm infants. Stem Cells Translational Medicine. 2026;15(8):szag045. https://academic.oup.com/stcltm/article/15/8/szag045/8746520

Cord Blood Beyond Stem Cells: A New Look at Transfusion Potential

CORD BLOOD MAY OFFER MORE THAN STEM CELLS AS RESEARCHERS EXPLORE ITS POTENTIAL FOR TRANSFUSION PRODUCTS

Cord blood may be useful not only for stem cell transplantation, but also as a possible source of blood products for transfusion. Its red blood cells contain fetal hemoglobin, which is naturally adapted to newborn life. In a recent study, researchers tested 65 cord blood units and found that they looked promising soon after collection, but storage changed their quality over time. After 30 days, potassium and hemolysis increased, meaning the cells became less stable. This suggests that cord blood could have transfusion potential, but only with strict quality checks, careful storage, and clear clinical standards.

Cord blood is best known as a source of stem cells for treating blood cancers, but researchers are also looking at it in a different way: as a potential source of transfusion products. Cord blood red blood cells contain fetal hemoglobin, while cord blood plasma and platelets may provide growth factors useful in wound-healing preparations. Another potential advantage is that cord blood may carry a lower risk of transmitting some infections from mother to baby.
The idea is not new, cord blood transfusion was reported as early as 1939, but it is still rarely used in clinical practice. This is why researchers continue to study whether cord blood can meet the quality and safety standards needed for transfusion.
A 2026 prospective observational study by Madhavi Ambhore and colleagues focused exactly on this question. The team assessed the quality of umbilical cord blood collected from healthy women after normal vaginal deliveries. In total, 65 cord blood units were collected. Their volumes ranged from 25 to 118 mL, with a median of 75 mL.
The collected units were stored at 2–6°C in a blood bank refrigerator. Researchers checked practical quality measures such as collected volume, clots, blood group, complete blood count, sterility culture, plasma hemoglobin, and potassium levels during storage.
On the first day after collection, potassium levels in cord blood and adult blood were within the normal range. After 30 days of storage, potassium increased significantly, and hemolysis also rose. This means that, like adult red blood cells, cord blood red cells change during storage. The study also suggested that cord blood from larger babies may be more stable, probably because larger collected volumes improve the blood-to-anticoagulant ratio.
Overall, cord blood showed comparable early quality parameters to adult blood shortly after collection, but storage had a clear effect on red cell stability by day 30. Changes in plasma hemoglobin suggested that cord blood red cells may be more susceptible to storage-related hemolysis than adult red cells during prolonged storage. Importantly, all 65 collected units remained sterile after 14 days of culture. Because each cord blood unit contains a limited amount of hemoglobin, several units would be needed for an adult transfusion.
The authors concluded that umbilical cord blood may be considered for transfusion soon after collection, provided that strict quality and safety checks are met. The findings are encouraging, but they also show that storage time, unit volume, and standardized quality criteria will be key if cord blood is to become a practical transfusion source.

Key points

  • Cord blood is being explored as a source of transfusion products beyond stem cell banking.
  • A 2026 study assessed 65 cord blood units collected after normal vaginal deliveries.
  • Clinical use would require strict quality checks, short storage, and standardized criteria.

Reference:
Ambhore M et al. Assessment of the Quality Parameters of Umbilical Cord Blood for Transfusion. Indian J Hematol Blood Transfus. 2026 Mar;42(2):573-580 https://pubmed.ncbi.nlm.nih.gov/41728178/

Bringing Cord Blood Treatment for Cerebral Palsy Closer to Practice

CORD BLOOD THERAPY IS MOVING CLOSER TO CLINICAL PRACTICE FOR CEREBRAL PALSY, BUT WIDER USE WILL REQUIRE STRONGER EVIDENCE, SPECIALIST TEAMS, AND CLEAR SAFETY PATHWAYS

Cord blood therapy for cerebral palsy is moving from research toward carefully controlled clinical programs. Early studies suggest that cord blood infusion is generally safe and may help improve movement in some children, especially younger children or those with milder disability. But this is not yet a routine approved treatment. The study shows that successful programs need specialist teams, strong laboratory support, clear safety procedures, long-term follow-up, and realistic evidence of benefit before wider clinical use is possible.

Cord blood infusion is gaining attention as a possible treatment option for children with cerebral palsy. In the United States, it is currently available only in controlled settings, such as clinical trials or expanded access programs. A recent meta-analysis of seven randomized trials suggests that cord blood treatment is generally safe and may improve gross motor skills, especially in children younger than five, those who can already walk, and those receiving higher cell doses. However, cord blood is still not an approved cerebral palsy treatment in any country.
Madison C. B. Paton and colleagues looked at what it takes to move this kind of treatment from research into real-world clinical practice. Their study focused on the cord blood treatment program for cerebral palsy at Duke University and a collaborating site, asking what helps such a program work — and what makes implementation difficult.
The researchers interviewed 16 professionals involved in these programs, including clinical leaders, research coordinators, nurses, allied health professionals, and laboratory staff. The interviews explored practical experience from the inside: how patients are identified, how teams work together, how laboratory and clinical processes are coordinated, and what support is needed to run the program safely.
Five main themes emerged. Successful implementation depended on strong clinical leadership, close cooperation between different professional groups, dedicated resources, community interest, an established safety record, and a research culture able to support careful follow-up. At the same time, teams faced important barriers, including the need for specialist expertise, uncertainty around how strong the efficacy evidence is, funding limits, regulatory requirements, and broader health-system challenges.
Overall, the study shows that cord blood treatment for cerebral palsy can be organized in highly specialized settings, but only when the right infrastructure, governance, trained teams, and ethical oversight are in place. The experience from the United States offers useful lessons for future programs, while also reminding us that wider access will require regulatory approval, sustainable funding, and stronger evidence of clinical benefit.

Key points

  • Cord blood infusion is being studied as a potential option for cerebral palsy.
  • Early trial data suggest general safety and possible motor benefits in selected children.
  • Real-world programs need specialist teams, laboratory support, and careful follow-up.
  • Wider use will require stronger evidence, approval, funding, and clear safety procedures.

Reference:
Paton MCB et al. From evidence to practice: factors influencing the implementation of cord blood treatment for cerebral palsy in the United States. Stem Cells Transl Med. 2026 Jul 20;15(8):szag074.https://pmc.ncbi.nlm.nih.gov/articles/PMC13453308/

Rheumatoid Arthritis and MSCs: Early Benefits, Open Questions

CORD BLOOD-DERIVED MSCS MAY HELP CALM JOINT INFLAMMATION IN RHEUMATOID ARTHRITIS, BUT LONGER-LASTING BENEFIT WILL REQUIRE STRONGER EVIDENCE AND BETTER DOSING STRATEGIES

Rheumatoid arthritis is an autoimmune disease in which the immune system attacks the joints, causing pain, swelling, and damage. Standard medicines can control inflammation, but they do not work for everyone and may raise infection risk. Researchers are testing whether cord blood-derived MSCs could help “rebalance” immunity rather than simply block one inflammatory pathway.
In a clinical trial, one infusion of cord blood-derived MSCs reduced rheumatoid arthritis activity for several months. The strongest improvement appeared after 3–6 months, with some benefit lasting about one year. The treatment was generally well tolerated in the small group followed for five years. However, the effect faded over time, so larger studies are needed to test whether repeated doses could give longer-lasting benefit.

Rheumatoid arthritis is a chronic inflammatory disease in which the immune system attacks the joints, causing pain, swelling, and damage. Although modern drugs have improved disease control, they can increase the risk of serious infections. This is why researchers are exploring mesenchymal stem cells as a way to calm harmful inflammation with a favorable safety profile.
In 2026, Park EH and colleagues reported long-term follow-up from the CURE-IV trial, an early clinical study testing human umbilical cord blood-derived mesenchymal stem cells (hUCB-MSCs) in rheumatoid arthritis. Patients received a single intravenous infusion at one of three cell doses: 2.5×10⁷, 5×10⁷, or 1×10⁸ cells. Nine participants were followed for five years, while continuing standard care chosen by their physicians.
After the infusion, disease activity dropped clearly, with the strongest improvement seen between 3 and 6 months. On average, the DAS28 score fell from 4.76 at baseline to 2.24 after 3 months. This improvement generally lasted for up to one year. Later, symptoms gradually moved back toward baseline, which may reflect the chronic nature of rheumatoid arthritis and the fact that patients received only one MSC dose. Importantly, the single infusion was generally well tolerated during the five-year follow-up.
How could MSCs help in rheumatoid arthritis? Preclinical studies suggest that they may shift the immune system away from joint-damaging inflammation. They can increase regulatory immune cells, reduce pro-inflammatory signals such as TNF-α, IL-17, IL-4, and interferon-gamma, and support anti-inflammatory molecules such as IL-10 and TGF-β. They may also encourage macrophages to adopt a more repair-oriented profile and reduce processes linked with bone loss.
The findings are encouraging, especially because benefits were seen after just one infusion and no major long-term safety signal emerged in this small group. However, the effect was not sustained over the full five years. Larger controlled studies are needed to confirm whether repeated dosing could maintain disease control and to better define long-term safety and real clinical benefit.

Key points

  • Rheumatoid arthritis is driven by chronic joint inflammation.
  • One infusion of MSC improved activity after 3–6 months, with benefit up to one year.
  • Larger studies are needed to confirm durability and optimal dosing.

Reference:
Park EH et al. Long-term safety of intravenous human umbilical cord blood-derived mesenchymal stem cell therapy in rheumatoid arthritis: a 5-year follow-up study. Stem Cells Transl Med. 2026 Jul 20;15(8):szag062. https://pmc.ncbi.nlm.nih.gov/articles/PMC13441169/pdf/szag062.pdf

Supporting Fragile Hearts: WJ-MSCs in Pediatric Cardiomyopathy

WHARTON’S JELLY MSCS MAY SUPPORT FRAGILE PEDIATRIC HEARTS WITH EARLY ENCOURAGING SIGNALS IN DILATED CARDIOMYOPATHY

Pediatric dilated cardiomyopathy is a rare heart disease in which the heart becomes enlarged and too weak to pump blood properly. In the A HEART-WISE study, doctors tested whether Wharton’s Jelly-derived MSCs could be safely delivered directly into the coronary arteries of two children. Both tolerated the procedure well, and early signs suggested better heart function and exercise capacity. These results are encouraging, but they come from only two patients, so much larger studies are needed before this approach can be considered a proven treatment.

Pediatric dilated cardiomyopathy is a rare but serious heart disease in which the heart muscle becomes enlarged and too weak to pump blood effectively. For the sickest children, heart transplantation remains the standard option. Because donor hearts are limited and treatment options are still challenging, researchers are exploring whether mesenchymal stromal cells could help support heart repair and function.
In August 2026, Hoda Madani and colleagues reported results from the A HEART-WISE study, the first case series evaluating intracoronary delivery of allogeneic Wharton’s Jelly-derived MSCs in children with dilated cardiomyopathy. Two pediatric patients received WJ-MSCs at a dose of 1.5×10⁶ cells/kg using a dual-catheter intracoronary technique.
Both children tolerated the procedure without serious treatment-related adverse events. In the first patient, important improvements were seen after 3 months: functional class improved from NYHA III to II, LVEF increased from 33% to 48%, walking distance rose from 300 to 373 m, and NT-proBNP decreased from 3502 to 1734 pg/mL. Unfortunately, the patient died at 6 months due to COVID-19, underlining how vulnerable this group can be.
The second patient was followed for 12 months. Over this period, functional status improved, the NYU Pediatric Heart Failure Index fell from 9 to 5, and walking distance increased from 265 to 380 m. The child also showed better appetite, more participation in school physical activities, and weight gain after 3 months. LVEF rose from 43% to 64%, while NT-proBNP stayed within the normal range.
These early findings suggest that allogeneic WJ-MSC delivery to the coronary arteries may be feasible and well tolerated in pediatric dilated cardiomyopathy, with encouraging clinical signals. However, this is only a two-patient case series, so larger studies are needed before the approach can be considered a proven therapy.

Key points

  • Pediatric dilated cardiomyopathy is a rare but serious heart disease.
  • A HEART-WISE tested intracoronary allogeneic WJ-MSCs in two children.
  • Early signals were encouraging, but larger studies are needed before clinical use.

Reference:
Madani H et al. A HEART-WISE: Allogeneic Wharton’s Jelly-derived Mesenchymal Stromal Cells’ Intracoronary Transplantation in Pediatric Patients with Dilated Cardiomyopathy: The First Case Reports. BMC Cardiovasc Disord. 2026 Apr 27;26(1):509. https://pmc.ncbi.nlm.nih.gov/articles/PMC13270590/

MSC-Derived Exosomes for Psoriasis: Early Safety, Future Potential

MSC-DERIVED EXOSOME OINTMENT SHOWS EARLY SAFETY AS A FIRST STEP TOWARD LOCAL THERAPY
FOR INFLAMMATORY SKIN DISEASE

Psoriasis is a chronic inflammatory skin disease that often requires long-term treatment. In this first-in-human study, researchers tested an ointment containing MSC-derived exosomes on healthy skin to check whether repeated use was safe and well tolerated. The product was applied for 20 days and did not raise major safety concerns. This does not yet prove that it works in psoriasis, but it is an important first step toward developing local, cell-based therapies for inflammatory skin diseases.

Psoriasis affects around 2–3% of people worldwide and is much more than a cosmetic skin problem. It is a chronic inflammatory disease that can require long-term treatment. Current systemic medicines can be effective, but prolonged use may bring important side effects, including kidney or liver toxicity, high blood pressure, tremor, malignancy risk, electrolyte disturbances, and loss of response over time. This is why researchers are looking for better-tolerated treatment options, especially approaches that act locally on inflamed skin.
In Cytotherapy, Chandran NS and colleagues reported results from a phase 1, single-center, open-label clinical trial carried out in Singapore. The study tested PTD 2021P, an ointment containing exosomes derived from mesenchymal stem/stromal cells, as a potential topical treatment platform for psoriasis. The first goal was simple but essential: to check whether repeated application of the ointment was safe and well tolerated in healthy volunteers aged 25–45 years.
This is an important first step because it is the first clinical study of a topical MSC-based product designed for inflammatory skin disease. Previous human studies had already explored umbilical cord-derived MSCs given intravenously for psoriasis, with an acceptable safety profile. In laboratory and animal models, MSC-derived exosomes applied directly to the skin reduced inflammatory signals involved in psoriasis, including C5b-9, IL-17, and IL-23.
In the trial, volunteers applied the ointment daily from Day 1 to Day 20, with a 4-hour interval between applications, on healthy skin. The product was well tolerated throughout the 20-day study. These early safety results do not yet prove efficacy in psoriasis, but they support further development of MSC-derived topical therapies for psoriasis and other inflammatory skin disorders.

Key points

  • Psoriasis is a chronic inflammatory skin disease requiring long-term treatment.
  • A phase 1 study tested topical MSC-derived exosome ointment in healthy volunteers.
  • Repeated use over 20 days was well tolerated, with no major safety concerns.
  • Efficacy is not yet proven, but the results support further psoriasis studies.

Reference:
Chandran NS et al. A phase 1, open-label study to determine safety and tolerability of the topical application of mesenchymal stem/stromal cell (MSC) exosome ointment to treat psoriasis in healthy volunteers. Cytotherapy. 2025 May;27(5):633-641. https://pubmed.ncbi.nlm.nih.gov/39918488/

Wharton’s Jelly MSCs in Earthquake Victims: Early Recovery Signals

WHARTON’S JELLY MSCS MAY SUPPORT RECOVERY AFTER SEVERE CRUSH INJURIES WITH EARLY SAFETY AND KIDNEY RECOVERY SIGNALS IN EARTHQUAKE VICTIMS

After severe crush injuries, damaged muscles can release substances that overload the kidneys and trigger serious whole-body complications. In this small case series, three earthquake victims received Wharton’s Jelly-derived MSCs together with standard intensive care. The treatment was well tolerated, and early signals suggested better kidney recovery and wound healing support. However, the results come from only three patients, so they should be seen as an encouraging first signal, not proof that the therapy works.

Major earthquakes can leave survivors with devastating crush injuries, sometimes leading to crush syndrome, a dangerous condition in which damaged muscles release substances that can harm the kidneys and other organs. In May 2026, Deniz Ozel Bilgi and colleagues described how Wharton’s Jelly-derived mesenchymal stem cells (WJ-MSCs) were used alongside standard multidisciplinary care in three critically ill earthquake victims. The goal was to explore whether MSC therapy could support systemic recovery, tissue repair, and wound healing in this extreme clinical setting.
The cases came from the February 2023 earthquakes in Kahramanmaraş, Türkiye, with magnitudes of 7.8 and 7.5. The patients, one 17-year-old female and two males aged 22 and 26, were at high risk of permanent organ damage based on McMahon and APACHE II scores. Each received three WJ-MSC transplants: about two-thirds of the cells were given intravenously and one-third subcutaneously.
The outcomes were encouraging. None of the patients died or lost a limb because of crush injuries, and no allergic reactions or serious MSC-related adverse events were reported. During 12 months of follow-up after discharge, no permanent organ damage or psychological distress was reported. Kidney function and kidney injury scores improved, while markers linked with muscle breakdown and kidney stress — including myoglobin, creatinine, urea, potassium, phosphorus, and CK, generally decreased by day 8.
The report suggests that WJ-MSC therapy may help support wound healing, tissue regeneration, immune balance, and kidney recovery in patients with severe crush syndrome. However, this was only a three-patient case series, so the results should be seen as an early signal rather than proof of effectiveness. Larger studies will be needed to understand whether this approach can reliably improve outcomes after major trauma.

Key points

  • Severe crush injuries can trigger kidney damage and systemic complications.
  • Three earthquake victims received WJ-MSCs alongside standard multidisciplinary care.
  • No serious MSC-related safety issues were reported during follow-up.
  • Kidney and muscle-injury markers improved, but larger studies are needed.

Reference:
Bilgi DO et al. The Role of Mesenchymal Stem Cell Therapies in the Treatment of Crush Injuries and Crush Syndrome after an Earthquake. Bratisl. Med. J. 127, 2577–2590 (2026). https://doi.org/10.1007/s44411-026-00637-8

Total FamiCord Cellular Therapy Products Released till 31.07.2026

TOTAL

8595

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