Top 5 Conditions You Didn’t Know Could Benefit From Stem Cell Therapy

Five lesser-known conditions where stem cell-based treatment is already approved, an established transplant option, or in late-stage trials are graft-versus-host disease, sickle cell disease, type 1 diabetes, corneal blindness from limbal stem cell deficiency, and severe systemic sclerosis (scleroderma). In each case the benefit comes from a specific, regulated cell treatment for a specific condition, not from a general “stem cell injection”. The evidence level differs for each, so this article labels it.

1. Steroid-refractory acute graft-versus-host disease (GVHD) in children: Ryoncil, the first FDA-approved mesenchymal stem cell therapy, approved December 2024.
2. Sickle cell disease: gene-edited stem cell therapy exa-cel (Casgevy); 29 of 30 evaluable patients were free of pain crises for at least 12 months in the pivotal study.
3. Type 1 diabetes: stem cell-derived islet cells, in late-stage clinical trials.
4. Corneal blindness from limbal stem cell deficiency: cultured limbal stem cell grafts restored a stable corneal surface in 76.6% of eyes in a 112-patient study.
5. Severe systemic sclerosis (scleroderma): autologous stem cell transplant lowered long-term mortality in a randomized trial, with a real early risk.

The label “stem cell therapy” covers very different treatments, and only some are proven. Each condition below is tagged with one of three evidence levels so you can tell an approved treatment from an experiment.

• Blood stem cell (hematopoietic) transplants, which replace or reset the blood and immune system. Read Viezec’s explainers on different types of stem cell transplants and hematopoietic stem cell transplantation.
• Mesenchymal stem cell (MSC) products, which calm inflammation and immune attack.
• Gene-edited stem cells, where a patient’s own blood stem cells are corrected before being returned. See Viezec’s page on gene therapy.
• Cells grown from stem cells, such as insulin-producing islet cells, or cultured tissue stem cells for the cornea.

For wider context, see stem cell-based therapy for human diseases and how effective stem cell therapy is for treating diseases.

• Evidence level: approved product (children). Graft-versus-host disease is a serious complication of donor blood stem cell transplants, in which the donor’s immune cells attack the patient’s own tissues. When the disease does not respond to steroids, treatment options have been limited, and mesenchymal stem cells now have an approved role.

  On 18 December 2024 the US FDA approved Ryoncil (remestemcel-L-rknd) for steroid-refractory acute GVHD in children aged 2 months and older, and called it “the first FDA-approved MSC therapy.” It is made from mesenchymal stromal cells taken from the bone marrow of healthy adult donors (FDA).

In a single-arm study of 54 children, 16 (30%) had a complete response and 22 (41%) a partial response 28 days after starting treatment. Infusions are given twice weekly for four weeks, with more weekly doses for partial responders. Common side effects were infections, fever, bleeding, swelling, abdominal pain and high blood pressure, and infusion reactions can occur (FDA).

The approval is narrow: it covers children with a specific complication, and the study had no comparison group. It shows that mesenchymal cells can be a regulated, tested medicine when the product, dose and patient group are precisely defined. Availability differs by country, so ask your transplant team.

Related reading on Viezec: bone marrow transplant, haploidentical bone marrow transplant and autoimmune disease management with mesenchymal stem cell treatment.

Evidence level: approved product. Sickle cell disease (SCD) is an inherited blood disorder in which misshapen red cells block blood vessels and cause severe pain crises (vaso-occlusive crises, or VOCs), organ damage and hospital stays. A transplant of blood stem cells from a matched donor can cure some patients, but many have no suitable donor. Gene-edited stem cell therapy offers a donor-free option.

Exa-cel (Casgevy) is a one-time therapy in which a patient’s own blood stem cells are collected, edited outside the body with CRISPR-Cas9 to switch fetal hemoglobin production back on, and returned by infusion after chemotherapy (busulfan) clears the bone marrow (AJMC). Fetal hemoglobin does not sickle, so crises can stop.

In the phase 3 study, 44 patients aged 12 to 35 received exa-cel. Of the 30 evaluable for the main endpoint, 29 (97%) had no VOCs for at least 12 months and none was hospitalized for a VOC over a 12-month period. Side effects were mostly from the busulfan conditioning, including mouth sores, low blood counts and infection risk, and one patient died of COVID-19 respiratory failure after lung injury linked to conditioning (AJMC).

In July 2026 the FDA expanded Casgevy’s approval to patients aged 2 years and older, for sickle cell disease with recurrent VOCs and for transfusion-dependent beta thalassemia (HealthDay).

The treatment is intensive and requires weeks in a specialist centre. Cost and access are major barriers, and the long-term follow-up is still limited. Discuss options with a haematology or transplant team.

Related reading on Viezec: stem cell therapy for sickle cell disease, stem cell transplant in thalassemia, gene therapy and the blood stem cell transplant procedure.

Evidence level: late-stage trial. In type 1 diabetes the immune system destroys the insulin-producing beta cells of the pancreas. Researchers can now grow islet cells from stem cells in the laboratory and infuse them into patients, aiming to let the body make its own insulin again.

Vertex’s zimislecel (formerly VX-880) is given as a single infusion into the liver. In the phase 1/2 study, 12 patients with at least one year of follow-up all reached an HbA1c below 7% with time in range above 70%. Ten of 12 (83%) no longer needed injected insulin at month 12, mean daily insulin dose fell by 92%, and all 12 were free of severe low blood sugar events from day 90. There were no serious adverse events related to the treatment, and two deaths were unrelated to it (Vertex, June 2025).

The company reported that the study had moved into its phase 3 portion, in adults with severe hypoglycemic events and impaired hypoglycemia awareness, and planned to submit marketing applications to regulators in 2026 (Vertex, March 2025). Check Vertex’s latest announcements for the current regulatory status.

• Patients need lifelong immunosuppressive drugs, as in an organ transplant, to stop rejection and a repeat immune attack (Beyond Type 1).
• The first studies are small and target people with the most severe disease, not everyone with type 1 diabetes.
• Gene-edited cells designed to avoid immunosuppression are still at an early stage (Beyond Type 1).
• This is not the same as clinic “stem cell injections for diabetes”, which have no comparable evidence.

Related reading on Viezec: type 1 diabetes breakthrough: stem cell therapy with islet cells, stem cell treatment for type 1 diabetes, type 1 diabetes treatment and type 1 vs type 2 diabetes.

Evidence level: established graft. The clear front surface of the eye, the cornea, is renewed by stem cells that live at its edge (the limbus). A chemical or thermal burn can destroy them, leaving the cornea cloudy and covered with blood vessels, and an ordinary corneal transplant tends to fail because there are no stem cells to renew it. Replacing the stem cells themselves can restore the surface.

In a long-term study of 112 patients, mostly with chemical or thermal burns, a small biopsy of limbal tissue from the patient’s healthy eye was grown in the laboratory and transplanted. It permanently restored a clear, self-renewing corneal surface in 76.6% of eyes, and restored eyes stayed stable for up to 10 years (Healio, on the NEJM study).

A later comparison of four approaches estimated five-year graft survival at 71% for the patient’s own cultured cells and 75% for their own limbal tissue, but only 33% for donor limbal tissue and 0% for donor cultured cells. Vision improved only in the groups that used the patient’s own cells (EuroTimes).

Patients with one healthy eye can donate the cells for the other, which is why many cases are one-sided burns. If both eyes are affected, options are more limited and often use donor tissue with immune-suppressing drugs, which have poorer results. Treatment is done by a corneal specialist, and a later corneal transplant may still be needed once the surface is stable.

Related reading on Viezec: corneal stem cell transplant procedure, stem cell transplant for eyes, eye injuries stem cell treatment in Delhi and the eye diseases hub.

Evidence level: established transplant, for selected patients. Systemic sclerosis is an autoimmune disease in which the skin thickens and the lungs, heart and kidneys can be damaged. In its rapidly progressing diffuse form, an autologous hematopoietic stem cell transplant, using the patient’s own blood stem cells, has been compared head-to-head with chemotherapy in randomized trials.

• SCOT (US and Canada): 75 adults with diffuse disease were randomized to myeloablative stem cell transplant or 12 monthly cyclophosphamide infusions. At 54 months, 91% of transplant patients were alive versus 77% on cyclophosphamide, and 79% were free of death or organ failure versus 50% (HCPLive). Treatment-related mortality was 3% with transplant and 0% with cyclophosphamide.
• ASTIS (Europe): 156 patients with early diffuse disease. The transplant group had eight treatment-related deaths in the first year versus none in the control group, but a clear long-term benefit: events (death or organ failure) at 4 years were 19% versus 26%, with a hazard ratio of 0.34 (ASTIS, JAMA 2014).

The transplant is intensive and carries a real early risk of death and of serious infection, low blood counts and shingles, so it is offered mainly to people with severe, fast-moving disease and adequate heart, lung and kidney function (HCPLive). It is not a treatment for mild or stable scleroderma, and it should happen only in an experienced transplant centre.

Related reading on Viezec: stem cell therapy for scleroderma in India, stem cell transplant success rate for scleroderma, systemic sclerosis stem cell transplant and autologous hematopoietic stem cell transplantation benefits.

The table compares what is used, how strong the evidence is, and the main caution for each condition.

Sources: FDA, AJMC, Vertex, Healio, HCPLive and ASTIS.

A convincing stem cell claim names the exact condition, the exact cell product and the study behind it. Every success above is a specific product or procedure tested in a defined patient group, and none supports the idea that any stem cell injection can treat any disease.

• Which condition and which cells? Ask for the cell source, how cells are processed and tested, and how they are given.
• Where is the evidence? Ask for the published study or registered trial for your exact condition, not a different disease.
• Is it approved or experimental? The FDA warns that stem cell products offered outside approved uses or regulated trials may be unapproved and unsafe (FDA).
• What are the written risks? Serious treatments such as transplants carry real mortality risk, and a trustworthy team says so.
• Who will follow me up? Choose a team with a named specialist and a follow-up plan.

• A promise of a cure, or of the same cells for many unrelated conditions.
• No named study, trial number or regulator approval.
• Pressure to pay before an examination and your medical records are reviewed.

Read Viezec’s pages on safety and adverse effects of stem cell treatment, stem cell therapy safety, quality control tests and stem cell success rate. To discuss your own reports, contact the Viezec team.