OnCo
trialsTrialCompleted

FMT plus pembrolizumab in anti-PD-1-refractory melanoma (Pittsburgh)

Fifteen melanoma patients whose immunotherapy had failed received a stool transplant from someone whose immunotherapy had worked, then restarted the drug. Six of them benefited, including three with lasting responses.

Single-arm study. Donors were seven melanoma patients with complete or durable partial responses to anti-PD-1. Of 15 recipients, 6 had clinical benefit (3 objective responses, 3 stable disease beyond 12 months); responders showed engraftment of donor taxa, increased CD8 T-cell activation and decreased IL-8-producing myeloid cells, and lower circulating IL-8. A companion Israeli study (Sheba, NCT03353402, 10 patients, 3 responders) was published alongside. Together they were the first human evidence that manipulating the microbiome can reverse checkpoint resistance, and they set off the current wave of randomised FMT and defined-consortium trials. Small, uncontrolled, and with a response rate a sceptic could attribute to re-challenge alone.

Setting
Metastatic melanoma with primary resistance to anti-PD-1: single colonoscopic FMT from a durable responder donor, then pembrolizumab
Phase
Phase 2
Sponsor
UPMC Hillman Cancer Center / NCI
Registry
Headline result
6 of 15 patients with clinical benefit (3 objective responses).
Reported
2021
Enrolled
15
Replication
Sheba study (Science 2021) concordant; randomised phase 2 trials (LND101 NCT06623461; Oslo NCT05286294) in progress.

Outcomes

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In plain words
What these results mean for people, not percentages
15 people took part
Clinical benefit (objective response or SD over 12 months)primaryresponse endpoint
  • 40 out of 100 people had their tumour shrink with FMT + pembrolizumab.
  • There was no comparison group, so this number cannot tell you how much of the effect is due to the treatment.
  • A response rate counts how many people had their tumours shrink by a set amount; shrinking is encouraging but does not by itself show that people live longer or feel better.
  • These results apply to the people the trial enrolled: Metastatic melanoma with primary resistance to anti-PD-1: single colonoscopic FMT from a durable responder donor, then pembrolizumab. People in a different situation may not see the same effect.
  • The trial selected people by a biomarker (PD-1); the result should not be assumed for people whose cancer does not have it.
  • Only 15 people took part, so the numbers are less certain than in a large trial.

Numbers are from the trial as recorded here; see the source links in the table below. This is orientation, not medical advice: ask your team how closely the trial population matches you.

15 participants enrolled.

Clinical benefit (objective response or SD over 12 months)primary
FMT + pembrolizumab40 of 100
n = 15
Source
EndpointArmnValueHR (95% CI)pSource
Clinical benefit (objective response or SD over 12 months)primaryFMT + pembrolizumab1540%link
Replication
Sheba study (Science 2021) concordant; randomised phase 2 trials (LND101 NCT06623461; Oslo NCT05286294) in progress.

Connected

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