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CureMedLongevity Medicine

Regenerative medicine: what is approved, what is in trials, and what is only being sold

Four different fields share one marketing word. A narrow set of cell products is genuinely approved — and none of it is approved for ageing.
Reviewed by CureMed LabsUpdated
A laboratory technician handling cell culture plates inside a biosafety cabinet.
The approved products in this field are made under pharmaceutical manufacturing controls for named indications. The products sold for ageing are not.
Simply put

Doctors can now repair a few specific things with living cells — burned skin, a damaged patch of knee cartilage, a scarred cornea, a destroyed bone marrow — and those treatments are approved and they work. Almost nothing sold as “stem cell therapy” for ageing, tiredness or sore joints is one of those treatments, and the published record of what has gone wrong includes patients who went blind, patients hospitalised with blood infections, and a death.

The short answer

Regenerative medicine is not one field with one evidence grade. It is at least four: a small set of genuinely approved cell products for narrow indications; a serious clinical research frontier of stem-cell-derived islets, dopamine neurons and retinal cells that has strong Phase 1/2 data and zero approvals; an experimental surgical frontier of gene-edited pig organs whose longest human success is 130 days; and a large unregulated market selling “stem cell therapy” for ageing that has produced documented blindness, tumours, sepsis and death. A clinic advertising regenerative medicine for longevity is almost never selling anything from the first three.

  • The approved list is short and specific: haematopoietic stem cell transplant, six CAR-T products, MACI and NOVOCART for cartilage defects, StrataGraft and Epicel for burns, Holoclar (EU conditional only), Lantidra, Rethymic, and exactly one mesenchymal stromal cell product — Ryoncil, approved 18 December 2024 for paediatric steroid-refractory acute GVHD. None of these is an anti-ageing indication.
  • Ryoncil, the only FDA-approved MSC product, was approved on a single-arm, open-label pivotal trial of 55 patients with no control group (NCT02336230) — a real approval on a design that cannot separate drug effect from natural history.
  • Alofisel cleared EU review in 2018 and had its marketing authorisation withdrawn on 13 December 2024 after a confirmatory Phase 3 read out negative. It is the field's most useful counterexample to “if it works, it stays approved”.
  • The best 2025 results are Phase 1/2, not approvals: stem-cell-derived islets left 10 of 12 full-dose recipients insulin-independent at one year but included two deaths, and both Parkinson's cell-graft papers in Nature were open-label with no sham control.
  • The harms from unapproved clinic products are documented, not hypothetical: blindness to no light perception after intravitreal injection, a glioproliferative spinal cord lesion, 20 bacterial infections with 19 hospitalisations, and a death from multiorgan failure — all in peer-reviewed journals.
“Regenerative medicine” is one phrase covering at least four different things with wildly different evidence behind them. Some of it is standard hospital care with decades of data. Some of it is the most interesting clinical research being done anywhere right now, published in Nature and the New England Journal of Medicine in 2025, and approved nowhere. Some of it is experimental surgery measured in weeks of graft survival. And some of it is a cash-pay industry selling infusions for ageing with no approval, no controlled evidence, and a peer-reviewed record of serious harm.
The gap between the first three and the fourth is the single most important thing to understand before spending money in this field. Almost every misleading claim you will read works by borrowing credibility from one category and applying it to another — “stem cell therapy is FDA approved” is a true sentence that is almost always used dishonestly.
This page grades each approach at the highest tier that genuinely exists for it, names the trial or the approval behind every claim, and states the harms as plainly as the literature does.

What is actually approved, and for what

Haematopoietic stem cell transplantation is the original and still the only routine curative stem-cell therapy. It works because blood-forming stem cells reliably home to marrow and reconstitute an entire cell lineage — a biological property most other “stem cell” products simply do not have. Omisirge (omidubicel-onlv), an ex vivo–expanded cord blood product, was FDA-approved in April 2023 to shorten neutrophil recovery after cord blood transplant.

CAR-T is the most successful engineered-cell platform: six FDA-approved products for blood cancers — tisagenlecleucel (Kymriah), axicabtagene ciloleucel (Yescarta), brexucabtagene autoleucel (Tecartus), lisocabtagene maraleucel (Breyanzi), idecabtagene vicleucel (Abecma) and ciltacabtagene autoleucel (Carvykti). In January 2024 the FDA notified all six manufacturers of a risk of secondary T-cell malignancies and required a class-wide boxed warning, with labelling updates completed by April 2024. The FDA cited 22 reported T-cell cancer cases as of 31 December 2023, arising 1–19 months after infusion. This is a genuinely curative therapy class that also carries a genuine oncogenic risk, and both halves belong in the same sentence.

The approved products, and their exact indications

Product or procedureWhat it isApproved for
Haematopoietic stem cell transplant · Omisirge (omidubicel-onlv)Blood-forming stem cells rebuild a destroyed marrowEstablished standard of care. Omisirge FDA-approved April 2023 to shorten neutrophil recovery after cord blood transplant
CAR-T (six products)The patient's own T cells re-engineered to attack cancerHaematologic malignancies. Class-wide FDA boxed warning for secondary T-cell malignancy since 2024
MACI · NOVOCART 3D · NOVOCART Inject plusThe patient's own cartilage cells grown in culture and re-implanted on a membraneSymptomatic full-thickness cartilage defects of the knee in adults. MACI Arthro arthroscopic delivery system FDA-approved August 2024
StrataGraft · EpicelLaboratory-grown skinStrataGraft FDA-approved June 2021 for deep partial-thickness thermal burns in adults. Epicel a Humanitarian Use Device since 2007 for burns of 30% or more of body surface
HoloclarCultured limbal stem cells for burn-damaged eyesEMA conditional marketing authorisation, 2015 — the first stem-cell-based ATMP in the EU. Not FDA-approved; a US patient cannot get it domestically
Lantidra (donislecel-jujn)Deceased-donor pancreatic islet cells — donor-derived, not stem-cell-derivedFDA approved June 2023, for adults with type 1 diabetes who cannot reach HbA1c targets because of recurrent severe hypoglycaemia
RethymicAllogeneic cultured thymus tissueCongenital athymia — a genuine engineered-tissue approval for an ultra-rare indication
Ryoncil (remestemcel-L-rknd)Allogeneic bone-marrow mesenchymal stromal cellsFDA approved 18 December 2024 for steroid-refractory acute GVHD in patients 2 months and older. The first and only FDA-approved MSC product
Approved indications are narrow on purpose. MACI treats a discrete cartilage lesion in a knee; it does not treat generalised osteoarthritis. Engineered skin is a burn product, not a cosmetic one.

Where each approach really stands

The regenerative medicine evidence ledger

Ranked on: the highest tier that genuinely exists for each approach as of 31 August 2026 — regulatory approval first, then the most advanced human trial actually running. Each approach is graded on the use being discussed here, not on the most flattering use it has somewhere else in the world.

  1. Haematopoietic stem cell transplant

    Approved

    Blood-forming stem cells reconstitute a destroyed marrow.

    Decades of randomised and registry data. The only routine curative stem-cell therapy, and the benchmark against which everything else in this field should be read.

    Established standard of care; licensed products include Omisirge (omidubicel-onlv), FDA-approved April 2023.FDA approval, Omisirge (omidubicel-onlv), April 2023

  2. CAR-T cell therapy

    Approved

    The patient's T cells re-engineered to attack their cancer.

    Multiple Phase 3 programmes and a curative signal in previously untreatable disease. The FDA cited 22 reported secondary T-cell cancers as of 31 December 2023, arising 1–19 months post-infusion.

    Six FDA-approved products for haematologic malignancies; class-wide boxed warning for secondary T-cell malignancy since 2024.FDA class-wide boxed warning, requested January 2024, labels updated April 2024

  3. Autologous chondrocyte implantation (MACI, NOVOCART)

    Approved

    The patient's cartilage cells expanded in culture and re-implanted on a scaffold.

    Pivotal Phase 3 against arthroscopic microfracture, N=144. Label-expansion Phase 3s are still running: adolescents (NCT03588975) and talus lesions (NCT06915233, N=309). Treats a defect, not osteoarthritis.

    FDA-approved for full-thickness knee cartilage defects in adults; MACI Arthro delivery system approved August 2024.NCT00719576

  4. Engineered skin (StrataGraft, Epicel)

    Approved

    Laboratory-grown skin for severe burns.

    Two completed Phase 3s for StrataGraft (NCT03005106 randomised vs. autograft, N=71; NCT04123548 open-label, N=52). Epicel's Humanitarian Use Device status means it cleared a rare-disease pathway with a lower efficacy bar than a standard approval.

    StrataGraft FDA-approved June 2021; Epicel a Humanitarian Use Device since 2007.NCT03005106

  5. Holoclar (limbal stem cells)

    Approved

    Cultured corneal limbal stem cells for eyes damaged by burns.

    Conditional means the EMA required further evidence after approval. It is the first stem-cell-based ATMP authorised in the EU, and it is unavailable in the US.

    EMA conditional marketing authorisation, 2015. Not FDA-approved.EMA conditional marketing authorisation, 2015

  6. Lantidra (donislecel) for type 1 diabetes

    Approved

    Deceased-donor pancreatic islets infused for severe recurrent hypoglycaemia.

    Donor-derived rather than stem-cell-derived, and therefore supply-limited by cadaveric pancreas availability — exactly the constraint the stem-cell islet programmes are trying to remove.

    FDA approved June 2023, narrow indication.FDA approval, Lantidra (donislecel-jujn), June 2023

  7. Ryoncil (remestemcel-L) for paediatric steroid-refractory GVHD

    Approved

    Allogeneic bone-marrow mesenchymal stromal cells.

    Pivotal trial NCT02336230: Phase 3, single-arm, open-label, N=55, completed April 2018. Overall response rate at day 28 approximately 70%, with around 30% complete response — but with no control group, the design cannot isolate drug effect from natural history and concomitant care.

    FDA approved 18 December 2024 — the first and only FDA-approved MSC product.NCT02336230

  8. Alofisel (darvadstrocel) for Crohn's perianal fistula

    Phase 3 trials

    Allogeneic adipose-derived MSCs for complex perianal fistula.

    EU-approved in 2018; the confirmatory Phase 3 read out negative in November 2023 and the European Commission withdrew the authorisation at the holder's request. The most instructive data point in the MSC field: regulatory clearance is not the same as confirmed efficacy.

    EU marketing authorisation withdrawn 13 December 2024 after a negative confirmatory Phase 3.European Commission withdrawal of marketing authorisation, 13 December 2024

  9. MSC injection for knee osteoarthritis

    Phase 3 trials

    Adipose, marrow or cord-derived MSCs injected into the joint.

    Meta-analyses show benefit versus saline but not superiority to a cheap corticosteroid or hyaluronic acid injection, from small heterogeneous trials with short follow-up. NCT07106229 (N=573, placebo-controlled) and NCT06570291 (N=520) should settle it; until they report, “proven” is the wrong word.

    No FDA or EMA approval. Large placebo-controlled Phase 3s now running.NCT07106229

  10. IV MSCs or exosomes for ageing, energy, or “systemic rejuvenation”

    Sold · unproven

    Unapproved autologous or allogeneic cell products sold cash-pay.

    No credible evidence of benefit for any ageing indication, and documented serious harm including blindness, spinal cord tumour, sepsis and death. Most infused MSCs are trapped in the lungs and cleared within days; they do not travel to a joint and become new cartilage.

    Unapproved new drugs. FDA warning letters, permanent injunctions, DOJ prosecutions and FTC actions.FDA consumer warning on unapproved human cell and tissue products, updated March 2026

  11. Stem-cell-derived islets for type 1 diabetes (zimislecel / VX-880)

    Phase 3 trials

    Fully differentiated, lab-made insulin-producing islets infused into the portal vein.

    Phase 1/2 published in NEJM 2025: all 14 participants with 12 months of follow-up engrafted; 10 of 12 full-dose recipients insulin-independent at day 365. Two participants died — one of cryptococcal meningitis, one of progression of pre-existing severe neurocognitive impairment.

    Not approved. NCT04786262 now listed as Phase 3, recruiting, N=52, primary completion June 2027.PMID 40544428 · NCT04786262

  12. Stem-cell-derived dopamine neurons for Parkinson's disease

    Phase 3 trials

    Lab-made dopaminergic cells grafted bilaterally into the putamen.

    Two Phase 1 trials published back-to-back in Nature in April 2025 showed graft survival, no tumour formation and no cell-product-attributed adverse events — both open-label, with no sham control, in a disease with famously large placebo effects in surgical trials.

    Not approved in the US or EU. exPDite-2 (NCT06944522), N=102, sham-surgery controlled, primary completion March 2027. A Japanese marketing application was filed in August 2025.PMID 40240592 · PMID 40240591 · NCT06944522

  13. Retinal pigment epithelium replacement for macular degeneration

    Phase 2 trials

    A replacement RPE cell layer placed under the retina.

    Reported 36-month OpRegen data show maintained improvement in RPE/drusen complex area in treated eyes (+2.6 mm² at 24 months, +1.0 mm² at 36 months versus baseline). Structural improvement on imaging is not the same as restored vision.

    Not approved anywhere. OpRegen now in Phase 2a (NCT05626114, N=60); the NEI autologous iPSC-RPE programme is Phase 1/2a (NCT04339764, N=20).NCT02286089 · NCT05626114

  14. Xenotransplantation (gene-edited pig kidney, heart, liver)

    Phase 1 · safety only

    Multi-gene-edited pig organs transplanted into humans.

    Six known living human recipients under expanded access before formal trials. Longest xenokidney function in a living human is 130 days; longest xenoheart 49 days to graft failure. Lifetime zoonotic-infection surveillance is a condition of participation.

    Not approved. FDA INDs cleared in 2025; EXPAND (NCT06878560) recruiting since 29 October 2025, N=50.NCT06878560

  15. Bioengineered whole solid organs

    Preclinical only

    A scaffold seeded with cells and grown into a transplantable organ.

    The tracheal programme, the field's furthest clinical attempt, produced deaths and retracted papers. The binding constraint is vascularisation: cells more than roughly 200 micrometres from a capillary die, and no group has built a hierarchical vascular tree at organ scale.

    Nothing approved. No whole solid organ has ever been successfully engineered and transplanted.PMID 28544662 · PMID 31035858

  16. Organoids

    Preclinical only

    Self-organising 3D tissue models, typically 0.5–5 mm across, grown from stem cells.

    Real value in disease modelling, drug screening and toxicity testing, and in testing patient-derived tumour organoids against drug panels. They lack vasculature, immune and neural components, and cannot exceed a few millimetres — they are not a step on a path to a transplantable organ.

    Not a therapy and not regulated as one. Genuine, high-value laboratory science.

  17. Partial epigenetic reprogramming (ER-100, Life Biosciences)

    Phase 1 · safety only

    A cellular reprogramming candidate aimed at partially resetting cell state.

    A Phase 1 first-in-human dosing event with no efficacy data of any kind. It will be heavily promoted to a longevity audience; a first dose is a regulatory milestone, not a result.

    First participant dosed 9 June 2026 — the first cellular-reprogramming candidate cleared by the FDA for human trials.First dosing reported 9 June 2026

Mesenchymal stromal cells: the widest gap between the pitch and the data

Mesenchymal stromal cells — the field has largely stopped calling them stem cells — are adherent connective-tissue cells isolated from marrow, fat, or umbilical cord. They are the product behind most of what is sold commercially as “stem cell therapy”.

After intravenous infusion, the great majority are trapped in the lungs and cleared within days. Their plausible mechanism is paracrine and immunomodulatory: they secrete factors that damp inflammation. It is not engraftment and differentiation into new tissue. The clinic pitch — that the cells travel to the damaged area and become new cartilage, neurons or heart muscle — is not what the evidence supports.

1
MSC product with FDA approval, for one paediatric indication (Ryoncil, 18 December 2024)
FDA approval, Ryoncil (remestemcel-L-rknd), 18 December 2024
N=55
single-arm, open-label pivotal trial that approval rests on — no control group
NCT02336230 (MSB-GVHD001), Mesoblast, Phase 3, completed April 2018
13 Dec 2024
date the EU withdrew Alofisel's authorisation after its confirmatory Phase 3 failed
European Commission withdrawal of marketing authorisation for darvadstrocel
0
approved MSC indications for ageing, joints in general, fatigue or neurological disease
FDA consumer warning on unapproved human cell and tissue products, updated March 2026

What the knee osteoarthritis evidence actually shows

Meta-analysisSizeFinding
Rahmadian et al. 2025, J Orthop Surg Res (PMID 40886001)8 treatment arms from 6 RCTs, N=300Pooled WOMAC SMD at 12 months −1.35 (95% CI −1.97 to −0.74). A moderate-to-large effect — from a small total N, with I² = 49.8%.
Han et al. 2025, Arthroscopy (PMID 39914607)9 Level I RCTs, N=671Stromal vascular fraction beat saline and hyaluronic acid but was inferior to corticosteroid at 3 months. Adipose MSCs beat saline and conservative care but were not superior to hyaluronic acid at any timepoint.
Tian et al. 2024, Front Endocrinol (PMID 38915896)18 studiesMSCs superior to placebo for pain and function at 12 months, with no significant difference in treatment-related adverse events. Authors flag high heterogeneity and short follow-up.
Three meta-analyses, one honest read: MSC injection probably does something for pain and function at 12 months under trial conditions, and has not been shown to beat a cheap corticosteroid or hyaluronic acid injection, or to regenerate cartilage.

Two large placebo-controlled Phase 3 trials should settle the question: NCT07106229 (MAG200, Magellan Stem Cells, N=573) and NCT06570291 (AlloJoin, N=520). Until they report, anyone using the word “proven” about MSC injection for osteoarthritis is ahead of the data — and the clinics selling it are charging today for the answer those trials have not yet produced.

The real research frontier: strong Phase 1/2 data, zero approvals

This is the genuinely exciting part of the field, and the part where careful framing matters most. Stem-cell-derived islets, dopamine neurons and retinal cells produced top-journal results in 2025. Not one of them is approved for these uses anywhere in the US or EU.

Type 1 diabetes produced the strongest single result. Reichman et al. (NEJM 2025, PMID 40544428) reported the Phase 1/2 FORWARD study of zimislecel (VX-880, NCT04786262): allogeneic stem-cell-derived, fully differentiated islets infused into the portal vein with glucocorticoid-free immunosuppression. C-peptide was undetectable at baseline in all 14 participants with at least 12 months of follow-up, and all 14 engrafted with detectable C-peptide afterwards. Of the 12 who received the full single dose, all 12 were free of severe hypoglycaemic events with HbA1c under 7% and more than 70% time in range, and 10 of 12 were insulin-independent at day 365. NCT04786262 is now listed as Phase 3, recruiting, N=52, with primary completion in June 2027.

Parkinson's disease produced two Phase 1 papers published back-to-back in Nature in April 2025. Bemdaneprocel (hESC-derived, BlueRock/Bayer; Tabar et al., PMID 40240592, NCT04802733) grafted 12 patients bilaterally in the putamen with one year of immunosuppression. It met its primary safety objectives, with no adverse events attributed to the cell product and no graft-induced dyskinesias. At 18 months, putaminal ¹⁸F-DOPA PET uptake had increased, and MDS-UPDRS Part III OFF improved by an average of 23 points in the high-dose cohort.

The Kyoto University allogeneic iPSC product (Sumitomo Pharma; Sawamoto et al., PMID 40240591) transplanted 7 patients aged 50–69 with 24-month follow-up. There were no serious adverse events across 73 mild-to-moderate events, and — critically for the main theoretical iPSC risk — MRI showed no graft overgrowth and no tumour formation. Among the 6 evaluated for efficacy, 4 improved on MDS-UPDRS Part III OFF and 5 on ON, with mean changes of 9.5 points (20.4%) OFF and 4.3 points (35.7%) ON, and putaminal F-DOPA Kᵢ up 44.7%. Sumitomo Pharma filed a Japanese manufacturing and marketing application in August 2025; if approved, it will be approved in Japan, not the US or EU.

Retinal cell replacement — real, early, and not approved anywhere

  • OpRegen / RG6501 (hESC-derived RPE, Lineage Cell Therapeutics with Genentech-Roche): Phase 1/2a NCT02286089, N=24, with 36-month data showing maintained improvement in RPE/drusen complex area in treated eyes (+2.6 mm² at 24 months, +1.0 mm² at 36 months versus baseline). Now in Phase 2a GAlette (NCT05626114, N=60).
  • NCT04339764 — National Eye Institute, autologous iPSC-derived RPE on a PLGA scaffold, Phase 1/2a, N=20, recruiting. The flagship US autologous iPSC ocular programme.
  • Other active early-phase programmes include NCT04627428 (Luxa RPESC-RPE-4W, N=18), NCT01691261 (PF-05206388 at Moorfields, N=9, completed, with long-term follow-up under NCT03102138), NCT02590692 (CPCB-RPE1, N=16) and NCT05445063 (autologous iPSC-RPE, Beijing Tongren, N=10).
  • NCT03167203 is an Astellas long-term safety surveillance study of hESC-RPE recipients running to 2029 — long-tail safety monitoring is standard and appropriate for these products, and its existence is a sign of a serious programme, not a red flag.
  • NCT02563782 and NCT02122159 were both withdrawn with zero enrolment — a reminder that a trial registry entry is not evidence that anything happened.
  • The honest gap: no RPE cell therapy is approved anywhere, and the best available evidence is anatomical improvement on imaging in small, mostly uncontrolled early-phase studies. Structural improvement on OCT is not restored vision and should never be presented as such.

Replacing organs: pig transplants, engineered organs, and organoids

Two very different things get filed under “lab-grown organs”. One of them — gene-edited pig organs — is a real, regulated, first-in-human clinical programme with named patients and published outcomes. The other — building a solid organ from a scaffold and cells — has never worked.

Xenotransplantation is the fastest-moving area in regenerative medicine and the one that most demands exact numbers. Six known living human recipients have received gene-edited pig organs to date, most under expanded access rather than a formal trial.

Every reported living human recipient of a gene-edited pig organ

CaseOrganOutcome — exactly as reported
University of Maryland, January 2022 (Bennett)10-gene-edited pig heartWeaned from ECMO; the xenograft initially functioned without apparent rejection. Sudden diastolic thickening and graft failure on day 49; life support withdrawn on day 60. Autopsy found the graft nearly doubled in weight with myocyte necrosis and interstitial oedema — not typical rejection. (PMID 35731912)
University of Maryland, September 2023 (Faucette)Gene-edited pig heartPatient died approximately 6 weeks post-transplant.
Massachusetts General Hospital, March 2024 (Slayman)Pig kidney with 69 genomic editsXenograft functioned immediately; creatinine fell and dialysis was no longer needed. T-cell-mediated rejection on day 8, reversed with intensified immunosuppression. Patient died on day 52 of sudden cardiac causes; autopsy showed severe coronary artery disease and ventricular scarring, with no evident xenograft rejection. (PMID 39927618)
NYU Langone, November 2024 (Looney)10-gene-edited pig kidneyFunctioned 130 days — the longest xenograft function in a living human to date. Kidney removed on 4 April 2025 after signs of rejection; patient recovered, was discharged on day 5, and returned to dialysis.
Massachusetts General Hospital, January 2025 (Andrews)eGenesis EGEN-2784 pig kidneyReported off dialysis and discharged approximately one week post-transplant.
Massachusetts General Hospital, June 2025 (Stewart)eGenesis EGEN-2784 pig kidneyReported gradually resuming work.
These durations are the whole story. Reporting “a successful pig kidney transplant” without the number of days it functioned would be dishonest.

The formal trials, and the honest limits

  • EXPAND (NCT06878560, United Therapeutics): Phase 1/2 of a 10-gene-edited “UKidney” in end-stage renal disease, N=50, recruiting since 29 October 2025 at NYU Langone and Northwestern. First surgery under protocol performed at NYU Langone in November 2025. Endpoints include 24-week patient and xenograft survival, measured GFR, quality of life — and incidence of zoonotic infection. Part B follow-up runs for the participant's lifetime, with a listed completion date of 2075.
  • EXTEND (NCT07224763, GGTA1-knockout thymokidney, N=50) opened in June 2026; NCT07696962 (ChoironeX GalKO porcine thymokidney, N=4) is due to start July 2026; NCT07429838 tests an eGenesis EGEN-5784 porcine liver used extracorporeally via the OrganOx metra cross-circulation device for acute-on-chronic liver failure, N=20 — an external bridge device, not an implanted liver. NCT05340426, an earlier UAB porcine kidney study, was withdrawn with zero enrolment.
  • The primate data is not a promise. Non-human primate xenokidney survival with the latest multi-gene-edited donors has exceeded 750 days in the best reported cases (PMID 42104578, PMID 40026598). Human experience is 130 days at best.
  • In highly allosensitised primates, even the best 3-knockout / 7-transgene donors are rejected via antibody-mediated rejection once immunosuppression is withdrawn (PMID 38865482).
  • Lungs are far behind kidneys. The first pig lung xenotransplant into a brain-dead human recipient showed technical feasibility without hyperacute rejection, but also early inflammatory injury and progressive dysfunction; consistent multi-month lung xenograft survival has not been achieved even in primates (PMID 41583874).
  • Gene-edited pig kidneys studied in brain-dead decedents showed clinically silent mesangial immune-complex deposits in the pre-implant biopsies of all three xenografts — a donor-derived process nobody had characterised before (PMID 40675320). Doppler resistive-index elevation preceded biopsy-confirmed T-cell-mediated rejection in two MGH recipients (PMID 41088519).

The binding constraint is vascularisation. Cells more than roughly 200 micrometres from a capillary die, so building an organ means building a hierarchical vascular tree down to capillary scale and then getting the recipient's circulation to anastomose to it. No group has solved that at organ scale. 3D bioprinting has produced perfusable vascular geometries in small constructs; it has not produced a transplantable solid organ. Tissue-engineered bladder augmentation in small paediatric case series from the mid-2000s remains the most-cited “engineered organ” success — it has never been reproduced in a controlled trial, was never commercialised, and applies to a hollow, low-metabolic-demand organ. It is not a template for a kidney.

Organoids are a separate thing again, and they are frequently misreported. They are self-organising 3D tissue structures grown from stem cells, typically 0.5–5 mm across, that reproduce some architecture and function of a real organ. Their value is real and large: disease modelling for genetic conditions that are hard to model in animals, drug screening and toxicity testing, and testing patient-derived tumour organoids against drug panels to guide treatment. Their limits are equally consistent across reviews — high variability of self-organising growth, restricted experimental access, and absent vasculature, immune and neural components. The same vascularisation ceiling applies: an organoid without a blood supply cannot exceed a few millimetres. “Scientists grew a mini-brain” is true, and it describes a laboratory model, not a step towards a transplantable organ.

Two recent bioengineering preprints illustrate where the laboratory work is heading — vascularised cartilage microtissues for endochondral bone grafts (Kronemberger et al., bioRxiv, DOI 10.64898/2026.07.13.738124) and engineered endothelial cell grafts forming functional anastomoses (Hartel et al., bioRxiv, DOI 10.64898/2026.07.17.739058). Both are preprints. Neither has been peer reviewed, and neither should be cited as evidence for any clinical claim.

The realistic near-term routes to reducing transplant demand are therefore cell therapies that restore a single function — islets, retinal pigment epithelium, dopamine neurons — and xenotransplantation. Not engineered organs.

The unregulated market, and what it has done to patients

no light perception
worst one-year visual acuity among 3 patients injected in both eyes at a US clinic for macular degeneration
Kuriyan et al., NEJM 2017 · PMID 28296617 · DOI 10.1056/NEJMoa1609583
19 of 20
patients hospitalised after infection from cord-blood products marketed as stem cell therapy, across 8 US states
Hartnett et al., JAMA Netw Open 2021 · PMID 34618037 · DOI 10.1001/jamanetworkopen.2021.28615
65%
of unopened, undistributed vials tested were contaminated (22 of 34), with 16 bacterial species identified
Hartnett et al., JAMA Netw Open 2021 · PMID 34618037
22
secondary T-cell malignancy cases reported to the FDA across approved CAR-T products as of 31 December 2023
FDA class-wide boxed warning, requested January 2024, labels updated April 2024

Documented harms, with citations

HarmSettingDocumented outcome
Bilateral blindness (PMID 28296617)US clinic; intravitreal autologous adipose cells for age-related macular degeneration, both eyes injected at once3 patients. Ocular hypertension, haemorrhagic retinopathy, vitreous haemorrhage, combined traction and rhegmatogenous retinal detachment, lens dislocation. Baseline acuity 20/30–20/200 → 20/200 to no light perception at one year.
Spinal cord tumour (PMID 27331440)Stem cell tourism; intrathecal infusion after ischaemic strokeGlioproliferative lesion of the spinal cord causing paraplegia — the canonical documented case of a tumour arising from an unregulated stem cell product.
Bacterial bloodstream and joint infection (PMID 34618037)Umbilical cord blood products marketed as stem cells; 8 US states, Aug 2017–Sep 201820 patients, 19 hospitalised. Isolates were common enteric organisms — E. coli in 14, Enterobacter cloacae in 7. Sterility testing of unopened, undistributed vials found 65% contaminated. Whole-genome sequencing matched patient isolates to product isolates across states: one contaminated source.
Death from multiorgan failure (PMID 33422096)Unproven stem cell treatmentA 48-year-old with disseminated skin ulcers, hepatitis and cardiomyopathy; fatal outcome. The authors note how few such cases reach the literature with causality that cannot be refuted — the published harm count is a floor, not a ceiling.
Death after an engineered organ (PMID 28544662)GMP stem-cell-seeded tracheal graft, compassionate useRecipient died of sudden airway obstruction three weeks post-transplant. Published by the authors as a negative result and a warning about translating from preclinical models.
Secondary T-cell malignancyApproved CAR-T products — a harm that exists on the approved side too22 cases reported to the FDA as of 31 December 2023, onset 1–19 months post-infusion. Class-wide boxed warning requested January 2024.
The last row matters as much as the others: honest grading means reporting the risks of the approved products, not only of the unapproved ones.

An international review (PMID 34415026) identifies the USA, China, India, Thailand and Mexico as the leading destinations for stem cell tourism — note that the US is on that list, so this is not solely an offshore problem. It found that clinic websites generally provide no basic travel-health information, and that beyond ineffectiveness the treatments are associated with infection, rejection and tumorigenesis.

The FDA's position is that autologous and allogeneic cell products that are more than minimally manipulated, or used for a non-homologous function, are unapproved new drugs and unlicensed biologics requiring a BLA. That position has now been tested and upheld: the FDA won on appeal at the 9th Circuit against California Stem Cell Treatment Center / Cell Surgical Network over adipose stromal vascular fraction products, and in October 2025 the Supreme Court declined to review the case, letting the ruling stand. Enforcement also runs through permanent injunctions, DOJ criminal prosecutions, FTC deceptive-advertising actions and FDA debarment orders. Warning letters continue: recipients from late 2024 into 2026 include Evolutionary Biologics, Chara Biologics, Supreme Rejuvenation, New Life Medical Services, Innate Healthcare Institute, and Dynamic Stem Cell Therapy on 11 February 2026. Exosome products marketed as regenerative therapies fall under the same logic and have drawn their own letters.

What changed in 2025 and 2026

The developments that actually moved the evidence

  1. April 2025

    Two Parkinson's cell therapies published back-to-back in Nature

    First high-quality safety data showing that grafted pluripotent-stem-cell-derived dopamine neurons survive, produce dopamine, and do not form tumours. Both trials were open-label with no sham control.

    PMID 40240592 · PMID 40240591

  2. 4 April 2025

    The longest human xenograft function on record ends at 130 days

    Towana Looney's gene-edited pig kidney was removed after 130 days following signs of rejection. She recovered and returned to dialysis.

    NYU Langone, April 2025

  3. June 2025

    Stem-cell-derived islets published in NEJM

    10 of 12 full-dose recipients insulin-independent at one year, with two deaths reported among participants and lifelong immunosuppression still required.

    PMID 40544428 · DOI 10.1056/NEJMoa2506549

  4. 17 June 2025

    First sham-controlled Phase 3 of a stem-cell-derived neuronal therapy begins

    exPDite-2 opened with N=102 across 43 sites, sham-surgery controlled, primary completion March 2027. This is the trial that tests whether the open-label Parkinson's signal is real.

    NCT06944522

  5. August 2025

    Sumitomo Pharma files in Japan

    A manufacturing and marketing application for the allogeneic iPSC-derived dopaminergic progenitor product. If approved it would be the first such approval anywhere — and it would be a Japanese approval, not a US or EU one.

  6. October 2025

    The Supreme Court declines to review the FDA's 9th Circuit win

    The ruling against California Stem Cell Treatment Center / Cell Surgical Network stands. The FDA's authority to regulate adipose stromal vascular fraction products as drugs is now unchallenged at the appellate level — the field's most consequential legal outcome to date.

  7. 29 October 2025

    EXPAND opens — the first regulated clinical xenotransplantation trial

    United Therapeutics' 10-gene-edited UKidney in end-stage renal disease, N=50, at NYU Langone and Northwestern. The first surgery under the protocol was performed at NYU Langone in November 2025.

    NCT06878560

  8. 2025

    FDA grants a record 48 RMAT designations

    Regenerative Medicine Advanced Therapy designation speeds development and signals plausible promise on preliminary clinical evidence. It is not an approval, and it is routinely misrepresented as one in marketing.

  9. 11 February 2026

    FDA warning letter to Dynamic Stem Cell Therapy

    One more entry in an unbroken enforcement stream against unapproved cell products, following letters to Evolutionary Biologics, Chara Biologics, Supreme Rejuvenation, New Life Medical Services and Innate Healthcare Institute.

    FDA warning letter, ref. 712579

  10. March 2026

    FDA updates its consumer warning on unapproved cell and tissue products

    The warning states that entities violating the FD&C Act or PHS Act may be subject to legal action without further notice, including seizure and injunction.

  11. March–June 2026

    Second and third eGenesis pig kidney transplants reported

    eGenesis reports the second EGEN-2784 transplant as successful. Durations for these cases have not been reported at the length of the Looney case.

  12. 9 June 2026

    First participant dosed with ER-100, a partial epigenetic reprogramming therapy

    Life Biosciences' candidate is the first cellular-reprogramming therapy cleared by the FDA for human trials. This is a Phase 1 first dose with no efficacy data of any kind — expect it to be promoted to longevity audiences as though it were more.

  13. June 2026

    Two more xenotransplantation trials open

    EXTEND (GGTA1-knockout thymokidney, N=50) begins, and NCT07429838 — an eGenesis EGEN-5784 porcine liver used extracorporeally through the OrganOx metra device, N=20 — is scheduled to start. Also in June, satri-cel received Chinese NMPA approval as the first CAR-T for a solid tumour, a China approval rather than an FDA or EMA one.

    NCT07224763 · NCT07429838

  14. July 2026

    The pipeline, counted

    Three regenerative medicine products had received FDA approval in 2026 year-to-date, six more were under FDA review for a first US approval, and 43 were in Phase 3.

Frequently asked questions

What is stem cell therapy actually used for?

In approved medicine: rebuilding a destroyed bone marrow after chemotherapy, treating blood cancers with CAR-T, repairing a defined cartilage defect in a knee, covering severe burns with lab-grown skin, restoring a cornea damaged by a chemical burn (in the EU only), treating congenital athymia, and one paediatric indication for steroid-refractory graft-versus-host disease. That is close to the complete list. Everything else you will see advertised — ageing, fatigue, general joint pain, autism, MS, COPD, long COVID — has no approved cell therapy behind it anywhere in the US or EU.

Are there FDA-approved stem cell treatments for growing or repairing organs?

No. There is no FDA-approved therapy that grows an organ. The approvals that exist replace a cell population or repair a defect: haematopoietic stem cell transplant, CAR-T, MACI and NOVOCART for knee cartilage defects, StrataGraft and Epicel for burns, Lantidra for donor islets, Rethymic for congenital athymia, and Ryoncil — approved 18 December 2024 and the only FDA-approved mesenchymal stromal cell product — for paediatric steroid-refractory acute GVHD. Holoclar, for the cornea, is EU-conditional only and is not FDA-approved.

Are there real risks with stem cell therapy?

Yes, on both sides of the approval line. From unapproved clinic products the peer-reviewed record includes three patients who went from useful vision to as bad as no light perception after intravitreal injection, a spinal cord glioproliferative lesion causing paraplegia after intrathecal infusion abroad, 20 bacterial infections with 19 hospitalisations traced by whole-genome sequencing to one contaminated cord-blood product, and a death from multiorgan failure. From approved products: the six CAR-T therapies carry a class-wide boxed warning for secondary T-cell malignancy, with 22 cases reported to the FDA as of 31 December 2023. The stem-cell-derived islet trial reported two deaths among its participants, one from an immunosuppression-related opportunistic infection.

How do lab-grown organs differ from a traditional organ transplant?

In practice they do not differ, because lab-grown solid organs do not exist. No kidney, liver, heart or lung has ever been successfully bioengineered and transplanted. The furthest clinical attempt, tissue-engineered tracheas, ended in deaths and retracted papers. The constraint is blood supply: cells more than roughly 200 micrometres from a capillary die, so an organ needs a hierarchical vascular tree down to capillary scale that then connects to the recipient's circulation — a problem no group has solved at organ scale. Traditional transplant, and now gene-edited pig organs, remain the only routes to a replacement organ.

Can stem cells be used to grow a functional organ in the lab?

They can grow organoids — self-organising 3D tissue models typically half a millimetre to five millimetres across that reproduce some of an organ's architecture and function. These are genuinely valuable for modelling genetic disease, screening drugs, testing toxicity, and testing a patient's own tumour cells against drug panels. They are not organs and are not regulated as therapies. They have no vasculature, no immune or neural components, and cannot grow past a few millimetres. A headline about a “mini-brain” or “mini-liver” is describing a laboratory model.

What are the success rates of pig organ transplants into humans?

Reported in days, not years, and every number should be stated exactly. The longest xenokidney function in a living human is 130 days, at NYU Langone, and the kidney was removed on 4 April 2025 after signs of rejection. The Massachusetts General Hospital kidney recipient in March 2024 came off dialysis, had a rejection episode on day 8 that was reversed, and died on day 52 of sudden cardiac causes with no evident xenograft rejection at autopsy. The first gene-edited pig heart failed on day 49 and life support was withdrawn on day 60. The first formal trial, EXPAND (NCT06878560, N=50), only began recruiting on 29 October 2025.

How effective is stem cell therapy for neurological disease compared with standard treatment?

Unknown, because the controlled trials have not reported. The two Parkinson's cell-graft trials published in Nature in April 2025 were open-label with no sham control — one reported a 23-point average improvement in MDS-UPDRS Part III OFF in its high-dose cohort, but placebo and expectation effects in unblinded Parkinson's surgical trials are notoriously large. The field has been burned before: the 1990s double-blind placebo-surgery trial of foetal dopamine neurons (NCT03347955) did not confirm early open-label enthusiasm and some recipients developed disabling graft-induced dyskinesias. exPDite-2 (NCT06944522), sham-controlled with N=102, reports in 2027.

How much does stem cell therapy cost, and why is it always cash-pay?

Unapproved clinic treatments typically cost US$5,000–$50,000 out of pocket. The reason no insurer covers them is the reason not to buy them: they are unapproved new drugs, so there is no coverage determination to make. The approved products work the other way round — hospital-administered, insurance-adjudicated, and restricted to a named indication, meaning you qualify for them rather than purchase them. Immediate availability to anyone who can pay, for a condition no regulator has approved a cell therapy for, is itself the answer to whether the treatment is proven.

How do regulations affect what stem cell therapies you can actually get?

Heavily, and differently in each jurisdiction. The FDA treats cell products that are more than minimally manipulated, or used for a non-homologous function, as unapproved new drugs requiring a licence — a position upheld by the 9th Circuit and left standing when the Supreme Court declined review in October 2025. Enforcement runs through warning letters (most recently to Dynamic Stem Cell Therapy on 11 February 2026), permanent injunctions, DOJ prosecutions and FTC actions. Jurisdiction also determines availability of legitimate products: Holoclar is authorised in the EU but not the US, CARTISTEM is approved in South Korea only, and the Kyoto iPSC Parkinson's product, if approved, would be approved in Japan.

Can stem cells treat age-related degeneration?

Not today, and it is worth separating the two halves of that question. Age-related conditions with serious cell-therapy research behind them — macular degeneration, Parkinson's, type 1 diabetes — are in Phase 1 to Phase 3 trials with no approvals for these uses. Generalised “anti-ageing” infusions have no controlled evidence at all. The most instructive fact is Alofisel: an MSC product that cleared full EU review in 2018 and had its authorisation withdrawn on 13 December 2024 when the confirmatory Phase 3 failed. If a licensed MSC product could not confirm efficacy for a specific, measurable indication, an unlicensed one sold for ageing has no basis whatsoever.

Autologous versus allogeneic stem cell therapy — what's the actual difference?

Autologous means the cells come from your own body (usually bone marrow or fat), processed and re-injected — the main advantage is a very low risk of immune rejection since your immune system recognises them as self. Allogeneic means the cells come from a donor, which allows a manufactured, off-the-shelf product that doesn't require an extraction procedure first, but carries a real (if generally low, for the mesenchymal stromal cells typically used) risk of immune reaction and requires more extensive donor screening under most regulators. Neither is categorically "better" — it depends on the specific condition, product, and what's actually been tested for it. Clinics that only offer one type and market it as inherently superior are simplifying a regulatory and manufacturing tradeoff into a marketing claim.

Does stem cell therapy work for back and spine pain?

There's more randomised evidence here than in most stem-cell indications, and it's cautiously positive rather than definitive. A meta-analysis of controlled trials found mesenchymal stem cell injections into degenerated discs produced clinically meaningful reductions in pain and disability scores versus control. A 2025 phase IIb trial of autologous bone-marrow MSCs in 52 patients with chronic low back pain found the injections well-tolerated with no major adverse events, and a separate 36-month trial of allogeneic cells reported durable improvement over controls. This is a genuinely more evidenced use than most stem-cell marketing claims — but it remains off-label, cash-pay, small-trial evidence, not an approved treatment, and results vary by how advanced the disc degeneration is.

Sources

Every figure and claim on this page traces to one of these. Where a source is a company announcement rather than peer-reviewed research or a regulator, it is labelled as such.

  1. 01Kuriyan et al. Vision Loss after Intravitreal Injection of Autologous “Stem Cells” for AMD — New England Journal of Medicine, 2017 · PMID 28296617 · DOI 10.1056/NEJMoa1609583
  2. 02Berkowitz et al. Glioproliferative Lesion of the Spinal Cord as a Complication of “Stem-Cell Tourism” — New England Journal of Medicine, 2016 · PMID 27331440 · DOI 10.1056/NEJMc1600188
  3. 03Hartnett et al. Investigation of Bacterial Infections Among Patients Treated With Umbilical Cord Blood-Derived Products Marketed as Stem Cell Therapies — JAMA Network Open, 2021 · PMID 34618037 · DOI 10.1001/jamanetworkopen.2021.28615
  4. 04Večerić-Haler et al. Multiorgan failure with fatal outcome after stem cell tourism — European Journal of Medical Research, 2021 · PMID 33422096 · DOI 10.1186/s40001-020-00477-4
  5. 05Lyons et al. International stem cell tourism: a critical literature review and evidence-based recommendations — International Health, 2022 · PMID 34415026 · DOI 10.1093/inthealth/ihab050
  6. 06Reichman et al. Stem Cell-Derived, Fully Differentiated Islets for Type 1 Diabetes — New England Journal of Medicine, 2025 · PMID 40544428 · DOI 10.1056/NEJMoa2506549
  7. 07Tabar et al. Phase I trial of hES cell-derived dopaminergic neurons for Parkinson's disease — Nature, 2025 · PMID 40240592 · DOI 10.1038/s41586-025-08845-y
  8. 08Sawamoto et al. Phase I/II trial of iPS-cell-derived dopaminergic cells for Parkinson's disease — Nature, 2025 · PMID 40240591 · DOI 10.1038/s41586-025-08700-0
  9. 09Griffith et al. Genetically Modified Porcine-to-Human Cardiac Xenotransplantation — New England Journal of Medicine, 2022 · PMID 35731912 · DOI 10.1056/NEJMoa2201422
  10. 10Kawai et al. Xenotransplantation of a Porcine Kidney for End-Stage Kidney Disease — New England Journal of Medicine, 2025 · PMID 39927618 · DOI 10.1056/NEJMoa2412747
  11. 11Fatima et al. Immunofluorescence and Electron Microscopy in Genetically Engineered Pig-to-Human Kidney Xenotransplantation — American Journal of Kidney Diseases, 2025 · PMID 40675320 · DOI 10.1053/j.ajkd.2025.04.021
  12. 12Bodard et al. Conventional and Contrast-enhanced Ultrasound Imaging in 2 Human Kidney Xenotransplant Recipients — Transplantation, 2026 · PMID 41088519 · DOI 10.1097/TP.0000000000005538
  13. 13Manook et al. Prolonged xenokidney graft survival in sensitized NHP recipients — Science Translational Medicine, 2024 · PMID 38865482 · DOI 10.1126/scitranslmed.adk6152
  14. 14Takemoto et al. Pig Lung Xenotransplantation: Barriers on the Road to Clinical Translation — Transplant International, 2026 · PMID 41583874 · DOI 10.3389/ti.2025.15542
  15. 15Guo et al. Porcine kidney xenotransplantation: From primate models to clinical reality — Animal Models and Experimental Medicine, 2026 · PMID 42104578 · DOI 10.1002/ame2.70195
  16. 16Le Bas-Bernardet & Blancho. Progress in Porcine Kidney Transplantation to Non-Human Primates — Transplant International, 2025 · PMID 40026598 · DOI 10.3389/ti.2025.14003
  17. 17Rahmadian et al. Efficacy of a single intra-articular injection of MSCs for knee osteoarthritis: dose-focused meta-analysis — Journal of Orthopaedic Surgery and Research, 2025 · PMID 40886001 · DOI 10.1186/s13018-025-06190-4
  18. 18Han et al. Intra-articular SVF and MSC Injections Show Variable Efficacy and Higher Potential Complications Compared to Corticosteroid and Hyaluronic Acid — Arthroscopy, 2025 · PMID 39914607 · DOI 10.1016/j.arthro.2025.01.050
  19. 19Tian et al. Relative efficacy and safety of MSCs for osteoarthritis: systematic review and meta-analysis — Frontiers in Endocrinology, 2024 · PMID 38915896 · DOI 10.3389/fendo.2024.1366297
  20. 20Elliott et al. Tracheal Replacement Therapy with a Stem Cell-Seeded Graft — Stem Cells Translational Medicine, 2017 · PMID 28544662 · DOI 10.1002/sctm.16-0443
  21. 21Pepper et al. Factors Influencing Poor Outcomes in Synthetic Tissue-Engineered Tracheal Replacement — Otolaryngology–Head and Neck Surgery, 2019 · PMID 31035858
  22. 22Kronemberger et al. Integrating vascular and hypertrophic cartilage microtissues for endochondral bone tissue engineering — PREPRINT, not peer reviewed — bioRxiv, 2026 · DOI 10.64898/2026.07.13.738124 · preprint
  23. 23Hartel et al. Engineered endothelial cell grafts form functional anastomoses and enable recruitment of intravenously delivered human T cells in CAM tumor models — PREPRINT, not peer reviewed — bioRxiv, 2026 · DOI 10.64898/2026.07.17.739058 · preprint
  24. 24FORWARD — zimislecel (VX-880) stem-cell-derived islets, Phase 3, N=52, recruiting — ClinicalTrials.gov, 2026 · NCT04786262
  25. 25VX-880 in type 1 diabetes with a prior kidney transplant, Phase 3, N=10, recruiting — ClinicalTrials.gov, 2026 · NCT06832410
  26. 26Bemdaneprocel (MSK-DA01) Phase 1 in Parkinson's disease, N=12, completed — ClinicalTrials.gov, 2025 · NCT04802733
  27. 27exPDite-2 — bemdaneprocel vs. sham surgery, Phase 3, N=102, 43 sites — ClinicalTrials.gov, 2026 · NCT06944522
  28. 28Foetal dopamine neuron transplantation vs. sham surgery, University of Denver, N=40, completed — ClinicalTrials.gov, 1999 · NCT03347955
  29. 29OpRegen hESC-derived RPE, Phase 1/2a, N=24 — ClinicalTrials.gov, 2026 · NCT02286089
  30. 30GAlette — OpRegen Phase 2a, N=60, recruiting — ClinicalTrials.gov, 2026 · NCT05626114
  31. 31Autologous iPSC-derived RPE on a PLGA scaffold, National Eye Institute, Phase 1/2a, N=20 — ClinicalTrials.gov, 2026 · NCT04339764
  32. 32EXPAND — 10-gene-edited UKidney xenotransplant, Phase 1/2, N=50, recruiting since 29 October 2025 — ClinicalTrials.gov, 2026 · NCT06878560
  33. 33EXTEND — GGTA1-knockout porcine thymokidney, Phase 1/2, N=50 — ClinicalTrials.gov, 2026 · NCT07224763
  34. 34EGEN-5784 porcine liver via OrganOx metra extracorporeal cross-circulation, Phase 1, N=20 — ClinicalTrials.gov, 2026 · NCT07429838
  35. 35MSB-GVHD001 — remestemcel-L pivotal trial, Phase 3, single-arm, N=55, completed — ClinicalTrials.gov, 2018 · NCT02336230
  36. 36MAG200 allogeneic adipose MSC for knee osteoarthritis, Phase 3, N=573, placebo-controlled — ClinicalTrials.gov, 2026 · NCT07106229
  37. 37AlloJoin allogeneic adipose MSC for knee osteoarthritis, Phase 3, N=520, recruiting — ClinicalTrials.gov, 2026 · NCT06570291
  38. 38MACI vs. arthroscopic microfracture, Vericel, Phase 3, N=144, completed — ClinicalTrials.gov, 2016 · NCT00719576
  39. 39MASCOT — MACI for chondral and osteochondral lesions of the talus, Phase 3, N=309 — ClinicalTrials.gov, 2026 · NCT06915233
  40. 40StrataGraft vs. autograft, Phase 3, N=71, completed — ClinicalTrials.gov, 2021 · NCT03005106
  41. 41FDA approval — Ryoncil (remestemcel-L-rknd), first and only FDA-approved MSC product — US Food and Drug Administration, 2024 · FDA approval, 18 December 2024 — paediatric steroid-refractory acute GVHD
  42. 42FDA approval — Lantidra (donislecel-jujn), deceased-donor islet cell therapy for type 1 diabetes — US Food and Drug Administration, 2023 · FDA approval, June 2023
  43. 43FDA approval — Omisirge (omidubicel-onlv), ex vivo expanded cord blood product — US Food and Drug Administration, 2023 · FDA approval, April 2023
  44. 44FDA approval — StrataGraft for deep partial-thickness thermal burns; Epicel Humanitarian Use Device (2007) — US Food and Drug Administration, 2021 · FDA approval, June 2021
  45. 45FDA class-wide boxed warning for secondary T-cell malignancy on all approved CAR-T products — US Food and Drug Administration, 2024 · Requested January 2024; labels updated April 2024; 22 cases reported as of 31 December 2023
  46. 46FDA consumer warning on unapproved products derived from human cells and tissues — US Food and Drug Administration, 2026 · Updated March 2026
  47. 47FDA warning letter — Dynamic Stem Cell Therapy — US Food and Drug Administration, 2026 · Ref. 712579, 11 February 2026
  48. 48FDA warning letter — Genetech Inc., over the Liveyon-distributed cord blood products (ReGen5, ReGen10, ReGen30) — US Food and Drug Administration, 2018 · 29 November 2018
  49. 49EMA conditional marketing authorisation — Holoclar, the first stem-cell-based ATMP approved in the EU — European Medicines Agency, 2015 · Conditional marketing authorisation, 2015
  50. 50European Commission withdrawal of the Alofisel (darvadstrocel) marketing authorisation after a negative Phase 3 — European Medicines Agency, 2024 · Withdrawn 13 December 2024; confirmatory Phase 3 negative November 2023
  51. 51US Supreme Court denial of certiorari, leaving in place the 9th Circuit ruling for the FDA against California Stem Cell Treatment Center / Cell Surgical Network — Supreme Court of the United States, 2025 · Certiorari denied, October 2025

Articles on this topic

  • Which regenerative medicine treatments work best for joint pain?

    Regenerative treatments for joint pain ranked against what actually works: exercise therapy and weight loss, GLP-1 agonists for knee OA, corticosteroid and hyaluronic-acid injections, PRP, bone-marrow and adipose stem-cell injections, autologous chondrocyte implantation, exosomes and clinic 'stem cells' — with a pharmacist's advice on what to try first.

  • What is the most effective regenerative medicine for knees?

    Knee treatments ranked on randomised evidence — exercise, weight loss and semaglutide, MACI for cartilage defects, steroid, PRP, hyaluronic acid, bone-marrow and adipose cell injections, exosomes — separated by what is wrong with the knee: osteoarthritis, a cartilage defect, a meniscal tear or a ligament injury.

  • Which regenerative medicine options help avoid joint replacement?

    Options for delaying or avoiding joint replacement ranked on evidence: exercise and weight loss, GLP-1 agonists, bracing and offloading, osteotomy, MACI for cartilage defects, steroid and PRP injections, stem-cell injections, exosomes — and when delaying a replacement does more harm than the operation.

  • What regenerative medicine is best for chronic back pain?

    Chronic back pain treatments ranked on randomised evidence: exercise and cognitive-behavioural approaches, multidisciplinary rehabilitation, staying active, epidural and facet injections, radiofrequency ablation, intradiscal PRP and cell injections, basivertebral nerve ablation, spinal fusion — and the regenerative injections that have not beaten placebo.

  • Which regenerative medicine therapy is safest for arthritis patients?

    Arthritis therapies ranked on safety for the arthritis patient: exercise and weight loss, topical NSAIDs, PRP, hyaluronic acid, corticosteroid injections, MACI, GLP-1 agonists, oral NSAIDs, bone-marrow and adipose cell injections, exosomes and clinic 'stem cells' — with the documented harms, the interactions and what an inflammatory-arthritis patient must know.

  • Best regenerative medicine treatments for osteoarthritis relief without surgery.

    Non-surgical osteoarthritis treatments ranked on relief and durability: exercise, weight loss and semaglutide, topical NSAIDs, braces and aids, steroid injections, PRP, hyaluronic acid, duloxetine, stem-cell injections and exosomes — assembled into a twelve-week programme a pharmacist would prescribe.

  • Best regenerative medicine options for tendon and ligament injuries.

    Tendon and ligament treatments ranked by tendon and by trial: progressive loading and eccentric exercise, shockwave, PRP (works for some tendons, fails for others), corticosteroid (worse at a year), needle tenotomy, surgery, stem-cell injections and exosomes — with the evidence stated for Achilles, patellar, elbow, rotator cuff and ligament injuries.

  • Best non-surgical regenerative medicine therapies for knee cartilage damage.

    Non-surgical therapies for knee cartilage damage ranked on evidence: loading and quadriceps strengthening, weight loss and semaglutide, offloading, PRP, hyaluronic acid, steroid, cell injections, exosomes — with the MRI truth that no injection regrows cartilage, and when MACI or osteotomy is the real regenerative answer.

  • Best regenerative medicine solution to speed up sports injury recovery.

    Sports injury recovery options ranked on return-to-play and re-injury evidence: early progressive loading, criteria-based rehabilitation, blood-flow restriction, PRP by injury (muscle, tendon, ligament), stem-cell injections, exosomes, peptides, hyperbaric oxygen — and what actually shortens the calendar without raising the re-injury rate.

  • Best evidence-based regenerative medicine therapy for chronic joint pain.

    Chronic joint pain therapies ranked strictly on the trial evidence — named trials, populations and outcomes: exercise, weight loss and semaglutide, topical NSAIDs, MACI, corticosteroid, PRP, hyaluronic acid, duloxetine, cell injections and exosomes — with a pharmacist's verdict on what 'evidence-based regenerative' actually leaves.

Regenerative medicine · 10 →

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