Stem cell transplants, which have the potential to save lives, come with risks for both donors and recipients. However, advancements in stem cell therapies may offer solutions to mitigate these risks. Stem cell transplants are used to treat conditions such as leukemia, myeloma, and lymphoma, where the bone marrow is damaged and unable to produce healthy blood cells. In Europe alone, 40,000 stem cell transplants occur yearly, and this number is increasing despite the disruptions caused by the COVID-19 pandemic.
One challenge in stem cell transplants is finding a suitable donor. While siblings from the same biological parents offer the best chance of a match, 70% of patients do not have a full match within their families and require external donors. Another complication is graft-versus-host disease (GVHD), where the donor cells attack the healthy cells in the recipient’s body. Risk factors for GVHD are mainly associated with the donor, such as age, sex, and human leukocyte antigen (HLA) mismatch. Addressing the risks involved in stem cell transplants, the European Parliament proposed an update to regulate substances of human origin (SoHO), emphasizing the need to monitor and protect donor health proportionate to the levels of risk involved.
In the future, stem cell therapies are moving towards advanced therapies (ATMPs), which minimize the risks associated with traditional transplants. These new therapies eliminate the need for external donors by allowing the editing of a patient’s own cells or genes without introducing foreign agents. The European Medicines Agency (EMA) has recommended the approval of Casgevy, a cell-based gene therapy that utilizes CRISPR/Cas9 technology. Casgevy is indicated for treating transfusion-dependent beta-thalassemia and severe sickle cell disease, both debilitating and life-threatening conditions caused by genetic mutations. While these new therapies bring significant advancements, there is a need to ensure their safe, fair, and accessible implementation. The full potential of these treatments will require cautious progress and coordinated efforts in the coming years.
