Stem cells: the most frequently asked questions, with answers and sources.

No, not in a general sense. There are specific established clinical uses, especially in hematology. Many other applications are still in research or in trials. Sources: NIH, FDA, ISSCR.

No, there is no evidence. In the largest controlled trial, in 180 children, an infusion of cord blood did not improve socialization or symptoms compared with placebo. The FDA and EMA warn against those who offer these therapies for a fee. Sources: Dawson et al., Journal of Pediatrics 2020, FDA, EMA.

No. For ALS, the phase 3 trial of NurOwn did not meet its goal, and the FDA advisory committee voted against it, 17 to 1. For Alzheimer's disease, there are no approved products. Sources: Cudkowicz et al., Muscle & Nerve 2022, FDA 2023, FDA.

Whether it is authorized for your disease (by AIFA, the Italian Medicines Agency, or by the EMA) or is part of an approved and registered clinical trial; which published studies show that it works; which cells are used and how they are prepared; what the risks are and who will follow up with you afterwards; why you should have to pay for an experimental treatment. Sources: ISSCR, FDA. See Protect yourself from fake cures.

No. iPSCs are essential for modeling diseases and for translational research. On March 6, 2026, Japan approved the world's first two iPSC-derived products, for Parkinson's disease and for heart failure, but on a conditional and time-limited basis: efficacy must be confirmed within 7 years. They therefore remain far from routine use. Source: JST Science Japan.

No. Autologous does not automatically mean safe, appropriate, or effective. What matters is the process, the manipulation, the indication, and the follow-up.

No. An organoid reproduces some aspects of a tissue or organ, but it is not equivalent to a complete organ or to a standard clinical procedure.

Because the language of regenerative medicine can be used to market procedures that have not been adequately validated. The distinction between a trial, a commercial offer, and an approved therapy is essential.

No. It is one of the most loosely used words in marketing. You need to understand exactly which cell product is being described and what its regulatory status is.

Transplantation of hematopoietic (blood-forming) stem cells (from bone marrow, peripheral blood, or umbilical cord blood) for diseases of the blood and immune system, such as leukemias, lymphomas, and some anemias. It has been in use for decades and is considered the standard of care. Sources: NIH, ISSCR, ASH.

Yes. In December 2023, the FDA approved Casgevy (exagamglogene autotemcel), which uses CRISPR to modify the patient's hematopoietic stem cells, for sickle cell disease; the approval was later extended to transfusion-dependent beta-thalassemia. It is the first approved CRISPR-based product. Source: FDA.

Yes, but for a very specific indication. In December 2024, the FDA approved Ryoncil (remestemcel-L) for steroid-refractory acute graft-versus-host disease in children. It is the first MSC product approved by the FDA, and it does not imply efficacy for the generic uses that are advertised. In the EU, the mesenchymal cell product Alofisel was withdrawn from the market on December 13, 2024 at the request of the marketing authorization holder. Sources: FDA, EMA.

Embryonic stem cells come from the blastocyst and are pluripotent; adult (tissue) stem cells have a narrower potential and serve to maintain tissues; iPSCs are adult cells reprogrammed to a state similar to the embryonic one (Yamanaka, 2006; 2012 Nobel Prize in Physiology or Medicine). Sources: NIH, ISSCR.

Generally not. The FDA and ISSCR warn against clinics that sell unapproved treatments for a wide range of conditions: evidence of efficacy is often lacking and there are real risks. Be wary of broad promises made for a fee and outside regulated trials. Sources: FDA, ISSCR.

Umbilical cord blood is a real source of blood stem cells, used in some transplants. In Italy, however, storing it “for yourself” is not allowed and private banks cannot operate: you can donate the cord blood free of charge to a public bank or, with authorization from the regional authorities and at your own expense, ship it to a bank abroad. According to the Centro Nazionale Sangue (Italy's National Blood Center), there is no solid evidence in favor of personal use, and there are very few cases of actual use. Sources: Centro Nazionale Sangue, DM 18 novembre 2009 (Italian ministerial decree of November 18, 2009). See Donate.

People aged 18 to 35 who weigh more than 50 kg and are in good health can sign up; you stay on the registry until age 55. All it takes is an interview with a doctor and a blood or saliva sample. For those who turn out to be a match for a patient, more than 9 out of 10 donations in 2025 were made from the blood, with a procedure similar to plasma donation. Sources: Centro Nazionale Trapianti (Italy's National Transplant Center), IBMDR (the Italian Bone Marrow Donor Registry). See Donate.