Cell Therapies – From Experiment to Standard of Care

On May 1, we marked World Fanconi Anemia Day, and tomorrow the 3rd Fanconi Anemia Europe Scientific Meeting will take place in Gdańsk. Fanconi anemia is a rare genetic disorder in which impaired DNA repair leads to bone marrow failure. There are several treatment methods for this disease—blood and platelet transfusions, stimulation of blood cell production—but the cornerstone of treatment is bone marrow transplantation.

In 1988, stem cells from umbilical cord blood were used for the first time in the world to treat this disease. This proved that cord blood can be an effective alternative to bone marrow or peripheral hematopoietic stem cells in the treatment of blood disorders. Forty years ago, this was a medical experiment; today, it is not, and umbilical cord blood stem cells are used worldwide in medicine, especially in hematology.

Cell therapies using cord blood are not merely a promise of the future. Their history began with basic research conducted in the 1980s. In 1988, a breakthrough occurred—the first clinical application in a patient. In Europe, then a cradle of pioneering and innovative medical solutions, Prof. Eliane Gluckman decided to perform the first cord blood transplant, saving the life of Matthew Farrow. Matthew was 5 years old at the time and suffered from Fanconi anemia. The transplant was made possible by collecting stem cells from the umbilical cord blood of his younger sister, who had been born four months earlier.

This was the world’s first successful cord blood transplant, performed in 1988—over 35 years ago—and at the time an innovative and experimental medical procedure that initiated a new era in the use of stem cells in therapy. Today, hematopoietic stem cell transplantation is a recognized and widely used treatment for hematological diseases. Since 2006, European guidelines have placed cord blood stem cells on par with those derived from bone marrow or peripheral blood—meaning they are an equivalent source of transplant material.

“Many people are still unaware of how important cord blood stem cell transplants are. My younger sister’s cord blood saved my life when I was five and allowed me to grow up, have my own family, and live a normal life. I was very lucky, because I know many people who unfortunately could not be saved. There is still too little awareness about the importance of cord blood stem cells. I am living proof that this method works,” says Matthew Farrow, the first patient in the world to receive a cord blood transplant.

Stem cells from cord blood have unique properties that translate into specific therapeutic benefits. After transplantation, they engraft in the recipient’s bone marrow and begin producing healthy blood and immune cells, allowing restoration of functions impaired by disease.

“Cord blood cells offer hope in the treatment of cancer, particularly acute myeloid leukemia, as well as many non-cancerous diseases. Importantly, in cord blood stem cell transplantation, full HLA matching is not required, unlike with bone marrow or peripheral blood cells. This means that some degree of mismatch is acceptable, while the chances of a successful transplant remain very high. The risk of immunological complications is also lower,” emphasizes Prof. Krzysztof Kałwak, Head of the Department of Bone Marrow Transplantation, Oncology and Pediatric Hematology at the Medical University of Wrocław and Chair of the EBMT Pediatric Diseases Working Party.

In recent years, the conditions surrounding cord blood transplantation have changed significantly. The development of cord blood banks, improved storage techniques, and advances in donor matching have greatly increased the availability of this therapy. Until recently, one of the concerns and arguments against cord blood banking was the belief that the collected volume was too small and did not contain enough stem cells for treatment, especially in older children or adults. Currently, two technologies for the expansion of hematopoietic stem cells from cord blood are registered, significantly broadening the clinical applications of this unique biological material.

The development of transplantology and increasing availability of cord blood mean that more and more patients—including those with Fanconi anemia—can benefit from a therapy that was once considered a breakthrough innovation and is now a foundation of modern hematology and transplantation medicine.

“In light of current medical knowledge, transplantation of stem cells from cord blood is no longer an experimental method but a proven, life-saving procedure that offers a real chance for cure or long-term remission. This method should be treated as one of the equivalent therapeutic options, selected by the physician depending on the patient’s clinical indications,” adds Prof. Krzysztof Kałwak.

Graft-versus-Host Disease

One of the key issues related to stem cell transplantation, including cord blood transplantation, is graft-versus-host disease (GvHD). This is a complication in which donor immune cells recognize the recipient’s tissues as foreign and begin to attack them.

GvHD can be treated; however, in some cases, available therapies are ineffective. In such situations, mesenchymal stem cells (MSCs), isolated for example from the umbilical cord, may serve as an alternative. They form the basis for so-called advanced therapy medicinal products (ATMPs).

“Prevention and treatment of graft-versus-host disease (GvHD) is a key element of the transplantation process, significantly affecting its success. In some cases, available prophylactic and therapeutic methods are insufficient, which may lead to serious complications, prolonged hospitalization, or even life-threatening situations. Therefore, it is crucial to ensure patients have access not only to the transplant itself but also to comprehensive peri-transplant care, including modern methods for preventing and treating GvHD, which significantly increases the chances of successful therapy and recovery,” emphasizes Prof. Krzysztof Czyżewski from the Department of Pediatrics, Hematology, Oncology, Immunology and Transplantology at Nicolaus Copernicus University in Toruń.

Hematopoietic stem cell transplantation, including from cord blood, has for years been a recognized and widely used standard treatment for many hematological diseases, while therapies using mesenchymal stem cells (MSCs) remain in clinical development and are sometimes described as “experimental.” However, this does not mean they are unproven—on the contrary, they are based on solid scientific foundations and are subject to rigorous regulations, institutional oversight, and bioethical review.

Unfortunately, the complexity of issues related to cell therapies means that the term “experimental therapy” is often misunderstood and unjustly perceived negatively. It is worth remembering that every breakthrough treatment—from antibiotics to transplantation—was once an innovation at the research stage, and medical progress has always relied on the courage to introduce new, responsibly tested solutions that eventually become standards of care.

FamiCord Group is one of the leading stem cell banking groups in Europe and worldwide. The organization operates across multiple international markets, offering comprehensive services related to the collection, processing, and storage of stem cells. Guided by the mission of protecting health and life, FamiCord enables the preservation of biological material derived from umbilical cord blood and other perinatal tissues. As a provider of integrated medical services, the Group ensures the highest standards of quality and safety at every stage of cooperation. With many years of experience and strong scientific and expert background, FamiCord supports the development of modern stem cell-based therapies.