Approved does not mean “for everyone”
Each authorization applies to one disease and to specific patients. Casgevy, for example, is reimbursed in Italy for thalassemia and sickle cell disease only for patients aged 12 to 35. Source: AIFA.
Treatments
What stem cells really treat today.
A map that is up to date as of October 2, 2026: which diseases have approved therapies, where serious clinical trials are under way, and where the promises have no evidence behind them. Each row links to its sources: medicines agencies, guidelines, and published studies.
Last updatedOctober 2, 2026
SourcesEMA, FDA, AIFA, guidelines, clinical trials
LimitationDoes not replace a doctor's advice
In brief
- The most solidly established treatment is the blood stem cell transplant (often called a “bone marrow transplant”), used for decades for leukemias, lymphomas, myeloma, and other blood diseases.
- There are about twenty authorized stem cell-based medicines worldwide, almost all for rare and very specific diseases. In Italy, the reimbursed ones include Holoclar, Strimvelis, Libmeldy, and Casgevy.
- For Parkinson's disease, heart disease, type 1 diabetes, and spinal cord injuries there are serious clinical trials, and in Japan the first conditional approvals, but no routine treatments in Europe.
- For autism, ALS, Alzheimer's, or aging, there are no approved stem cell medicines in Europe or the United States. Before paying for a treatment, read how to protect yourself.
Disease by disease
What the evidence says today
- Standard treatment: used in clinical practice, with guidelines
- Approved for specific cases: authorized only for narrow indications
- In clinical trials: used only within clinical trials
- Not proven: not enough evidence; be wary of anyone selling it
| Disease | Status | What exists today | Sources |
|---|---|---|---|
| Leukemias, lymphomas, myeloma, myelodysplastic syndromes, severe aplastic anemia | Standard treatment | The blood stem cell transplant has been an established treatment for decades for many blood cancers and for severe aplastic anemia. In Italy, there were 5,258 transplants in 2025. | NCI · EBMT 2022 · CNT 2025 |
| Thalassemia and sickle cell disease | Standard treatment and new therapies | A transplant from a matched sibling is the treatment of choice for young people with transfusion-dependent thalassemia. For those without a donor there is Casgevy, a gene therapy performed on the patient's own stem cells: authorized in the EU and reimbursed in Italy for ages 12 to 35. In the US, there are also Lyfgenia and Zynteglo. | EBMT 2022 · EMA · AIFA |
| Severe immunodeficiencies and some inherited metabolic diseases | Approved for specific cases | Transplantation is the reference treatment for conditions such as SCID or metachromatic leukodystrophy in selected patients. There are approved gene therapies for specific diseases: Strimvelis (ADA-SCID), Libmeldy (metachromatic leukodystrophy), Waskyra (Wiskott-Aldrich syndrome). | EBMT 2022 · EMA Strimvelis · EMA Libmeldy · EMA Waskyra |
| Eye burns with loss of corneal stem cells | Approved for specific cases | Holoclar, grown from the patient's limbal stem cells, has been authorized in the EU since 2015 for adults with chemical or physical eye burns, and is reimbursed in Italy. | EMA |
| Macular degeneration and other retinal diseases | In clinical trials | Only phase 1 and 2 trials with retinal cells derived from stem cells; no approved treatment. Injections of supposed “stem cells” into the eyes, sold by an American clinic, caused severe vision loss in three patients. | Mandai 2017 · FDA · Kuriyan 2017 |
| Major skin burns | Approved for specific cases | Skin grown in the laboratory from the patient's cells has been used since the 1980s in patients with major burns. | Gallico 1984 · FDA |
| Epidermolysis bullosa (“butterfly children”) | In clinical trials in Europe | In 2017, genetically corrected skin, obtained from the patient's stem cells, regenerated almost all of the epidermis of a child with the junctional form: a single case, followed for years. In the US, a similar therapy (Zevaskyn) has been approved since 2025 for the recessive dystrophic form; in Europe it has not. | Hirsch 2017 · Kueckelhaus 2021 · FDA |
| Multiple sclerosis | Approved for specific cases | The autologous blood stem cell transplant is used for the highly active relapsing-remitting form that does not respond to drugs. It works by resetting and rebuilding the immune system, not by repairing nerves, and it carries greater risks than drugs. | EBMT 2020 · ECTRIMS-EBMT 2025 · AISM |
| Type 1 diabetes | In clinical trials | With pancreatic islet cells derived from stem cells (zimislecel), 10 of 12 patients on the full dose no longer used insulin after one year, but they were taking immunosuppressive drugs; among the 14 participants there were 2 deaths. A phase 3 trial is under way; there is no approval. | Reichman 2025 · FDA list |
| Parkinson's disease | In clinical trials (Japan: conditional approval) | In Japan, since March 2026, AMCHEPRY (made from iPS cells) has had a conditional and time-limited approval, based on 7 patients. In Europe and the US there are only trials, one of which is in phase 3. | Sumitomo Pharma · Sawamoto 2025 · Tabar 2025 |
| Heart failure and heart attack | Not proven in the EU and US | Twenty years of studies with bone marrow cells have produced uncertain or negative results. In 2018, Harvard requested the retraction of 31 papers from one laboratory on “cardiac stem cells” because of falsified data. In Japan, since 2026, RiHEART cardiac cell sheets have had a conditional approval; an earlier product, HeartSheet, was withdrawn in 2024. | Cochrane 2016 · DREAM-HF 2023 · STAT 2018 · Cuorips |
| Spinal cord injuries | In clinical trials | No approved treatment in the EU or US. In Japan, Stemirac has had a conditional approval since 2018, criticized because it was based on limited data and had no control group. The first trials with nerve cells made from iPS cells have only just begun. | Kawaguchi 2026 · Sugai 2026 |
| Stroke | In clinical trials | The TREASURE trial, in 206 patients, was negative; according to the Cochrane review, the certainty of the evidence is low. | TREASURE 2024 · Cochrane 2019 |
| Autism | Not proven | In the largest controlled trial, in 180 children, an infusion of cord blood did not improve socialization or symptoms. The FDA and EMA warn against those who sell these therapies. | Dawson 2020 · FDA · EMA 2020 |
| ALS | Not proven | The phase 3 trial of NurOwn did not meet its goal, and the FDA advisory committee voted against it, 17 to 1. | Cudkowicz 2022 · FDA 2023 |
| Alzheimer's disease, cerebral palsy, aging | Not proven | No approved product. For cerebral palsy, a controlled trial showed no difference in the primary outcome. | FDA · EMA 2020 · Sun 2017 |
| “Plant stem cell” cosmetics | Not proven | They contain extracts of cultured plant cells, not living human stem cells, and there is no evidence that they regenerate the skin. In the EU, the use of cells and tissues of human origin in cosmetics is prohibited. | EU Regulation 1223/2009 · Trehan 2017 (review) |
Table compiled on October 2, 2026. Knowledge changes: decisions about your own health always require the advice of a specialist doctor.
Authorized medicines
The list of stem cell-based medicines
This list includes medicines whose active substance is made of stem or progenitor cells, or is derived from them, authorized in the European Union, the United States, or Japan up to October 2, 2026: blood stem cells corrected with gene therapy, cord blood, mesenchymal cells, eye stem cells, and products derived from iPS cells. It does not include CAR-T cell therapies, chondrocytes, or pancreatic islets from donors.
| Medicine | What it contains | For which disease | Authorization | Notes |
|---|---|---|---|---|
| The patient's blood stem cells, corrected with gene therapy | ||||
| Strimvelis | The patient's blood stem cells, to which the ADA gene is added | ADA-SCID, a severe inherited immunodeficiency, in patients without a matched family donor | EU, May 26, 2016 | Marketing authorization holder: Fondazione Telethon. Reimbursed in Italy since 2016. EMA |
| Libmeldy (Lenmeldy in the US) | The patient's blood stem cells with the ARSA gene added | Early-onset metachromatic leukodystrophy | EU, December 17, 2020 · US, March 18, 2024 | Reimbursed in Italy since 2022. EMA |
| Casgevy | The patient's blood stem cells modified with CRISPR (gene editing) | Severe sickle cell disease and transfusion-dependent beta-thalassemia | UK, November 16, 2023 · US, December 8, 2023 and January 16, 2024 (for ages 2 and up since July 1, 2026) · EU, conditional, February 9, 2024 | Reimbursed in Italy (AIFA, the Italian Medicines Agency, September 30, 2025) for patients aged 12 to 35. EMA · FDA |
| Waskyra | The patient's blood stem cells with the WAS gene added | Wiskott-Aldrich syndrome | US, December 9, 2025 · EU, January 9, 2026 | Marketing authorization holder: Fondazione Telethon. Price and reimbursement under review by AIFA (September 2026). EMA |
| Kresladi | The patient's blood stem cells with the ITGB2 gene added | Severe leukocyte adhesion deficiency type I, in children without a matched sibling | US, March 26, 2026 (accelerated approval) | Not authorized in the EU. FDA |
| Zynteglo | The patient's blood stem cells with a beta-globin gene added | Beta-thalassemia | EU, conditional, May 29, 2019 · US, August 17, 2022 | Withdrawn from the EU on March 24, 2022 at the request of the marketing authorization holder, for commercial reasons. EMA |
| Skysona | The patient's blood stem cells with the ABCD1 gene added | Cerebral adrenoleukodystrophy | EU, July 16, 2021 · US, September 16, 2022 (accelerated approval) | Withdrawn from the EU on November 18, 2021 for commercial reasons. In the US: blood cancers in 10 of 67 trial participants (15%); since 2025, restricted to patients without a matched donor. |
| Lyfgenia | The patient's blood stem cells with a beta-globin gene added | Sickle cell disease | US, December 8, 2023 | Warning about blood cancers. Not authorized in the EU. FDA |
| Umbilical cord blood | ||||
| Omisirge | Cord blood cells from a donor, multiplied in the laboratory | Blood cancers requiring a transplant (ages 12 and up); severe aplastic anemia (ages 6 and up) | US, April 17, 2023 and December 8, 2025 | FDA |
| Zemcelpro | Cord blood stem cells from a donor, partly multiplied in the laboratory | Adults with blood cancers who need a transplant and have no other suitable donor cells | EU, conditional, August 25, 2025 | EMA |
| Cord blood (“HPC, Cord Blood”) | Cord blood units from donors, not multiplied | Blood stem cell transplant | US, 9 products authorized between 2011 and 2024 | FDA list |
| Mesenchymal (stromal) cells | ||||
| Temcell | Mesenchymal cells from a donor's bone marrow | Acute graft-versus-host disease | Japan, September 18, 2015 | Full authorization. |
| Ryoncil | Mesenchymal cells from a donor's bone marrow | Steroid-refractory acute graft-versus-host disease, in children aged 2 months and older | US, December 18, 2024 | Not approved for adults. FDA |
| Stemirac | Mesenchymal cells from the patient's bone marrow | Traumatic spinal cord injury | Japan, December 28, 2018 (conditional and time-limited, 7 years) | In November 2025 the manufacturer applied for full approval; the decision is not known. |
| Akuugo | Modified mesenchymal cells | Chronic motor paralysis after traumatic brain injury | Japan, July 31, 2024 (conditional and time-limited) | Still under conditional approval. |
| Seibiscus | Mesenchymal cells from the patient's synovial membrane | Knee meniscus injury | Japan, May 2026 | Non-conditional authorization. |
| Alofisel | Mesenchymal cells from a donor's fat tissue | Perianal fistulas in Crohn's disease | EU, March 23, 2018 | Withdrawn from the EU on December 13, 2024 at the request of the marketing authorization holder. EMA |
| Stem cells of the eye (limbal) | ||||
| Holoclar | The patient's limbal stem cells, grown in the laboratory | Limbal stem cell deficiency due to chemical or physical eye burns, in adults | EU, conditional, February 17, 2015; standard authorization since February 22, 2024 | Reimbursed in Italy since 2017. The manufacturer, Holostem (Modena), has been in liquidation since July 2026. EMA |
| Nepic | Limbal cells grown in the laboratory | Limbal stem cell deficiency | Japan, March 2020 | |
| Derived from iPS (reprogrammed) cells | ||||
| AMCHEPRY | Dopaminergic neuron progenitors derived from donor iPS cells | Parkinson's disease, motor symptoms not controlled by medication | Japan, March 6, 2026 (conditional and time-limited) | Efficacy must be confirmed within 7 years. Sumitomo Pharma |
| RiHEART | Sheets of heart muscle cells derived from donor iPS cells | Severe ischemic heart failure | Japan, March 6, 2026 (conditional and time-limited) | Cuorips |
| Other | ||||
| Tregzi (Orca-T) | Blood stem cells from a donor together with selected immune cells (regulatory and conventional T lymphocytes) | Transplant from a matched donor in adults with blood cancers | US, June 30, 2026 | FDA |
Outside the EU, the US, and Japan, some mesenchymal cell products have been authorized in South Korea, China, and India: for example, Cartistem (Korea, 2012) for knee cartilage defects. These are national authorizations, in some cases conditional, that are not valid in Europe. Sources: FDA list (September 17, 2026), the EMA's EPAR documents, and, for Japan, announcements from the companies holding the authorizations.
How to read an approval
Three things to know
Conditional means provisional
In the EU, a conditional marketing authorization is granted on less complete data than usual, when the benefit of having the medicine available right away outweighs the risk; it is valid for one year and can be renewed. In Japan, conditional and time-limited approval requires efficacy to be confirmed within a few years: HeartSheet did not manage this and was withdrawn. Sources: EMA, Hakariya 2025.
Authorized elsewhere does not mean available in Italy
An approval in the US or Japan is not valid in Europe. In the EU, advanced therapy medicinal products are assessed by the EMA; in Italy, price and reimbursement are decided by AIFA. Some products, such as Zynteglo and Skysona, have also been withdrawn from the European market for commercial reasons. Sources: EMA, EMA Zynteglo.
Sources
Main references
- FDA: list of approved cellular and gene therapy products (updated September 17, 2026)
- EMA: advanced therapy medicinal products
- EMA: conditional marketing authorization
- FDA: consumer alert on regenerative medicine products
- FDA: information for patients on regenerative medicine therapies
- EMA: warning against unproven cell-based therapies (April 28, 2020)
- EBMT: indications for blood stem cell transplantation (Snowden et al., Bone Marrow Transplantation, 2022)
- National Cancer Institute: stem cell transplants
- Centro Nazionale Trapianti: 2025 activity report (in Italian)
- AIFA: reimbursement decision for Casgevy, September 30, 2025 (in Italian)
- Dawson et al., Journal of Pediatrics 2020: cord blood in autism
- Sharrack et al., Bone Marrow Transplantation 2020: autologous transplant in autoimmune diseases, including multiple sclerosis
- Reichman et al., New England Journal of Medicine 2025: stem cell-derived pancreatic islets in type 1 diabetes
- ISSCR: nine things to know about stem cell treatments
The other sources are given row by row in the two tables.