Japan evidence and access guide
Allogeneic Stem Cell Therapy in Japan
Japan has several donor-derived regenerative cell products with defined approvals or conditional approvals — but there’s no single allogeneic treatment that works for every disease. The question that matters is whether your exact diagnosis maps to a specific product, study population and outcome.
What is currently relevant
Do not group every donor-derived cell product together
Japan’s current landscape includes allogeneic mesenchymal stem or stromal cell products, modified donor-derived cells and products made from donor-derived iPS cells. They’re not interchangeable — each one differs in what it’s approved for, how it’s given, what evidence sits behind it, and what risks come with it.
TEMCELL HS Inj.
Allogeneic bone-marrow-derived mesenchymal stromal cells. PMDA approval is for acute graft-versus-host disease after hematopoietic stem cell transplantation, not for unrelated inflammatory or degenerative conditions.
Alofisel
Allogeneic adipose-derived mesenchymal stromal cells for complex perianal fistulas in selected adults with Crohn’s disease after inadequate response to previous therapy and required local preparation.
Akuugo
Human allogeneic bone-marrow-derived mesenchymal stem cells modified with Notch-1 intracellular domain. Japan granted conditional and time-limited approval for chronic motor paralysis after traumatic brain injury. It is implanted by stereotactic brain surgery, not given as a general IV MSC infusion.
AMCHEPRY
Allogeneic iPS-cell-derived dopaminergic neural progenitor cells for motor symptoms in patients who do not respond adequately to existing drug therapy including levodopa-containing products.
RiHEART
Allogeneic iPS-cell-derived cardiomyocyte sheets for severe heart failure associated with ischemic cardiomyopathy under a conditional and time-limited pathway.
AlloStem Sheet
Allogeneic adipose-tissue-derived mesenchymal stem cell sheets for difficult-to-heal or recurrent erosions and ulcers in defined forms of epidermolysis bullosa. The approved use is topical and lesion-specific, not a whole-body infusion.
Regulatory source: PMDA Review Reports: Regenerative Medical Products. Product approval never means the same cells are proven for another disease.
Start with your diagnosis
Four questions decide whether the evidence is relevant to you
Does your confirmed diagnosis match the studied indication?
Similar symptoms are not enough. The disease definition, severity and previous treatment must match the evidence used to support the product.
Were patients like you included?
Age, disease stage, organ function, infection status, previous procedures and current medicines can change eligibility and risk.
What outcome was actually measured?
Remission, motor scores, walking distance, functional class and survival are different endpoints. A positive change in one does not prove improvement in another.
What is the exact access pathway?
Ask whether it is an approved product, conditional product, registered research plan or another regulated route. These categories do not carry the same level of evidence.
Patient-relevant clinical data
Read the result together with the study size and design
A percentage from six patients doesn’t carry the same weight as one from a randomized controlled study. The figures below show the actual endpoint, follow-up and main limitation — so you can judge each one on its own terms.
Response and survival were measured
Complete response had to last at least 28 days. PMDA also reported survival at 180 days and 52 weeks after the first infusion.
60.0% 15/25 alive at 180 days, 95% CI 38.7–78.9
52.0% 13/25 alive at 52 weeks, 95% CI 31.3–72.2What this means: the interval is wide because the study was small and had no concurrent control group. It is not a personal survival forecast.PMDA Table 8.9
Combined remission used clinical closure plus MRI
This is the strongest comparative dataset among these examples. A separate Japanese single-arm study enrolled 22 patients.
54.2% 58/107 vs 37.1% 39/105 at Week 52; difference +17.1 points, 95% CI 3.9–30.3
68.2% 15/22 in the Japanese study at Week 52, without a control groupWhat this means: the controlled global comparison is more informative than the Japanese percentage alone. The result applies to the studied Crohn fistula setting, not to other diseases.PMDA Tables 18–19 and Japanese Study Darvadstrocel-3002
Motor function at Week 24 was the main endpoint
The primary comparison pooled the three SB623 dose groups and compared them with sham surgery.
+6.0 point between-group difference, 95% CI 0.3–11.8, P=.0401What this means: the study showed a difference on a motor scale. It did not establish a survival benefit, and Japan’s approval is conditional and time-limited while more evidence is collected.PMDA Study TBI-01, Table 22
OFF-state motor score was followed for 24 months
A marked response was defined in the study as at least a 5-point improvement in OFF-state MDS-UPDRS Part III at 24 months.
Function and exercise capacity were assessed at Week 52
All eight patients were NYHA Class III before transplantation. The study also measured walking distance and peak oxygen uptake.
4 of 8 increased 6-minute walking distance by at least 45 m at Week 52
4 of 8 increased peak VO₂ by at least 10% at Week 52
75 + 150 planned post-marketing RiHEART and external-control sample sizesWhat this means: PMDA cautions that the eight-patient open-label study cannot separate the product effect from rehabilitation, medicines or other influences well enough to make a firm efficacy estimate.PMDA Study CVSC0005 and post-marketing plan
Ulcer area, not a “cure rate,” was measured
All six enrolled patients had dystrophic epidermolysis bullosa. The study assessed mean ulcer-area change after repeated sheet applications.
−82.26% to −3.09% range of individual ulcer-area changes across the six patientsWhat this means: responses varied widely and there was no control group. The percentage describes ulcer-area change in six patients, not a cure rate or whole-body disease response.MHLW 2026 product summary
When your diagnosis doesn’t match
Do not borrow evidence from another disease
The product, disease, timing and endpoint have to line up. A positive result in one indication doesn’t prove anything about a general donor-cell infusion.
Spinal cord injury
Japan’s Stemirac pathway uses autologous bone-marrow-derived MSCs. That evidence cannot be used as proof for an allogeneic product.
Spinal cord injury evidenceAcute ischemic stroke
TREASURE randomized 206 patients. Excellent outcome was 11.5% vs 9.8% at Day 90 (P=.90) and 15.4% vs 10.8% at Day 365 (P=.43); neither comparison was statistically significant.
Cerebrovascular evidenceKnee joint disorders
Source, preparation, dose and outcome vary between MSC studies. A result from a different cell product should not be presented as the expected result for your knee.
Knee evidenceDiabetes, liver or kidney dysfunction
None of the listed Japanese product approvals above establishes that a general allogeneic infusion restores pancreatic, liver or kidney function.
Diabetes evidenceLiver and kidney evidence
TREASURE source: JAMA Neurology 2024 randomized Phase 2/3 trial. Internal condition pages are background resources, not statements that a specific allogeneic product is suitable for you.
Survival and recovery
First ask what the study actually measured
Patients often want one answer for “success,” “recovery” or “survival.” The published studies above measure very different things — so the endpoint has to match your real goal.
TEMCELL acute GVHD study
Survival was directly reported because acute GVHD after transplantation can be life-threatening. This is the only example above where the quoted study result is a survival percentage.
52.0% at 52 weeks
Alofisel and AlloStem Sheet
Alofisel measured fistula remission using clinical closure plus MRI. AlloStem measured change in ulcer area. Neither endpoint is a general measure of whole-body recovery.
−54.242% mean AlloStem ulcer-area change in n=6
Akuugo and AMCHEPRY
Akuugo used the Fugl-Meyer Motor Scale after traumatic brain injury. AMCHEPRY used Parkinson motor scores. These results do not establish longer survival.
4/6 AMCHEPRY marked motor response at 24 months
RiHEART severe ischemic cardiomyopathy study
The eight-patient study used NYHA class, walking distance and peak VO₂. PMDA says the open-label study is too limited to estimate a reliable treatment-effect rate.
4/8 met the 45 m walking threshold at Week 52
Tell us the one result you care about most — mobility, daily function, remission, wound healing, avoiding further decline or survival. We can help organize the evidence around that question.
Check What AppliesCost references in Japan
Use official product prices as references, not as a personal quote
The figures below are product or reimbursement references. They don’t include every procedure, test, medicine, inpatient stay, follow-up item or international self-pay arrangement — so keep that in mind when you see a number.
How to read the figures
Product reference
The listed amount may refer to one bag, vial set, sheet or treatment unit. It is not automatically the total cost of care.
Procedure and monitoring
Surgery, tests, medicines, inpatient care, monitoring, rehabilitation and follow-up can sit outside the product figure.
International self-pay
Domestic Japanese reimbursement should not be assumed for a non-resident. A written scope is needed before comparing quotes.
Official reference register
TEMCELL HS Inj.
The approved dose is weight-based and can involve repeated infusions, so one bag is not a course price.
Alofisel
Fistula preparation, local procedures and other care are separate.
Akuugo
The figure does not include the complete stereotactic neurosurgical care pathway.
AMCHEPRY
Surgery, tacrolimus monitoring and other care are not represented by the product price alone.
RiHEART
As of 11 August 2026, this is a pending listing rather than an already effective price.
AlloStem Sheet
The study allowed multiple sheets per application, so the unit price is not a total course estimate.
Official price sources: MHLW 2026 price list for TEMCELL and Alofisel; MHLW May 2026 notice for Akuugo and AMCHEPRY; MHLW July 2026 listing for AlloStem Sheet. RiHEART’s ¥53.2 million figure and planned 1 September 2026 listing reflect the Central Social Insurance Medical Council decision announced 22 July 2026.
Risk and treatment burden
The cell source is only one part of the risk
The route, surgery, immune management and repeat dosing can matter just as much as the word “allogeneic.” Ask about the full pathway — not only the cells.
TEMCELL: repeated IV infusions
The approved schedule is twice weekly for 4 weeks, with up to four additional weekly infusions depending on symptoms. PMDA requires monitoring during and after infusion.
Alofisel: local fistula procedure
The cell injection follows required local preparation of the fistula tract. Recovery therefore includes the procedure and wound-related care, not only the cell product.
AMCHEPRY: brain surgery plus tacrolimus
Cells are transplanted stereotactically into both putamina. PMDA’s approved regimen generally continues tacrolimus for about 1 year, followed by a 12-week taper; the period may be extended when necessary.
RiHEART: thoracotomy plus 90-day immunosuppression
In the initial n=8 study, serious adverse events occurred in 5/8, but PMDA ruled out a causal relationship to RiHEART for those events. Procedure-related events could not be ruled out in 5/8, and immunosuppressant-related events in 7/8.
Akuugo: stereotactic intracranial implantation
The pivotal study compared cell implantation with sham surgery. PMDA highlights risks related to the intracranial procedure and requires further evidence because the clinical dataset remains limited.
AlloStem Sheet: repeated wound application
The Japanese study used weekly application for up to 12 weeks, with twice-weekly application allowed after three weeks without clear ulcer-area reduction. Treatment burden depends on wound area and number of sheets.
These figures describe the reported studies and approved use conditions — they’re not a complete list of what could go wrong for one person.
How we help
Turn scattered records into a clear Japan case plan
We organize your records, map your diagnosis to the relevant Japanese evidence, show what’s still uncertain, and prepare the questions that need a clear answer — so you aren’t spending time or money on assumptions.
One concise summary
Diagnosis, stage or severity, previous care, current medicines, key test results and your main goal organized in a review-friendly format.
What applies and what does not
Approved or conditional product evidence is separated from research findings and unrelated disease claims.
A focused record checklist
We identify missing imaging, laboratory data, pathology, functional scores or treatment history that may block a meaningful review.
Questions before a quote is accepted
Product, procedure, testing, monitoring, medicines, follow-up and other expected items are separated so the quote is easier to compare.
Language and document support
We help keep the case questions, document versions and next steps organized during your Japan planning process.
We don’t diagnose, prescribe, manufacture cells, provide a regenerative medicine product, or promise acceptance or results. Final suitability and care decisions must be made by appropriately qualified medical professionals after reviewing the individual case.
Prepare these records first
A useful review needs more than a diagnosis name
Confirmed disease and severity
Diagnosis report, stage or grading when relevant, date of diagnosis, major symptoms and current functional limits.
What you already tried
Previous medicines, procedures, rehabilitation and other care, including dose or date where important and whether it helped.
Recent objective data
Laboratory results, imaging, pathology, endoscopy, cardiac or neurological testing, and functional scores when relevant to the condition.
Factors that change risk
Current medicines, allergies, infections, immune problems, cancer history, major organ dysfunction and previous transplant history.
What outcome matters most
State whether your priority is symptom control, mobility, daily function, remission, avoiding further decline or another measurable goal.
What changed and when
A short chronology makes it easier to see progression, treatment response and whether published study timing is relevant.
You do not need to send every file at once. Start with the diagnosis summary and the most recent objective results; missing items can be identified from there.
Start With What I Have
Before you commit money or travel
Get six answers in writing
Exact product and cell source
Product name, donor source, cell type, dose and route of administration.
Exact regulatory pathway
Approved indication, conditional approval, registered research or another regulated route.
Evidence for your diagnosis
Study size, patient type, comparator if any, follow-up length and the endpoint that improved.
What result is realistic
Ask for a measurable expected outcome and what would count as no response. Avoid guarantees.
Full cost scope
Separate the product price from tests, procedures, monitoring, medicines, follow-up and travel-related changes.
Follow-up after you return home
Know which tests are needed, at what time points, and who will receive the results.
Patient questions
Questions that often change the decision
Is allogeneic therapy better than using my own cells?
Can a positive stem cell study for one condition support my condition?
Why can the same “stem cell” label have very different costs?
Does conditional approval mean the treatment is proven?
How should I read a high percentage from a small study?
How do I check whether a study or regenerative medicine plan is registered in Japan?
Should I stop my current care while exploring regenerative medicine?
Evidence sources
Use the regulatory report before the marketing claim
The figures above are tied to named products and published or regulatory evidence. Approval status and prices can change, so the date of the source matters.
PMDA optimal-use guideline index and MHLW 2026 price and product summary
Current drug price list, Akuugo and AMCHEPRY price notice, and AlloStem Sheet July 2026 listing
Medical information here supports patient education and preparation — it isn’t a diagnosis, prescription, individual prognosis or guarantee of access or outcome.
Send your actual case first
Know what applies before you spend time or money
Send a short diagnosis summary and your latest key results. We can help you organize what’s relevant, what’s still missing and which questions need a clear answer for your Japan plan.
WhatsApp +81-8070161366 LINE +81-8075357788. No outcome can be promised — suitability depends on individual records, the exact product or pathway, and professional assessment.