Proof of safety versus proof of concept27 Oct 2025 10:46
DSMB ultimately provides the authority to continue the trial and escalate the dose of CAR T cells in Cohort two. By doing so, primarily they will provide external verification of the safety data gathered by MDA Hemogenyx - the focus of phase one trials. We have seen three RNS's confirm clean safety profiles. A well recognised group of excellent early results first up.
While DSMBs are safety-first (stopping trials for harm or futility), oncology trials, particularly like ours at HEMO focussing on a high-unmet-need area R/R AML, include clear secondary endpoints for assessing preliminary efficacy. The trial is collecting data on anti-leukemic activity (e.g., reduction in blast cells, durable responses), pharmacokinetics, and persistence of the CAR-T cells.
In the September 2025 half-year report, the company described dose escalation as "a critical milestone in defining both the safety and therapeutic potential of HG-CT-1." This phrasing suggests the DSMB review incorporates early signals of benefit, not just the absence of toxicity. Contrary to much FUD commentary on this site.
Early CAR-T data (even at these low doses) can show Proof Concept if there's evidence of target engagement, T-cell expansion, or tumour killing—validating the therapy's mechanism before Phase II. For HG-CT-1, which uses a novel "chimeric T-cell" approach to improve persistence and reduce exhaustion, positive early efficacy would act to de-risk the program and likely attract partners / major investment. And we know p1 passed 6 months, p2 3 months and p3 MRD negative within weeks.
So you see, dose escalation corrobrates the proof of safety (very significant imo in its own right as i've said before). But I believe with efficacy data gathered by MDA to date, and likely shared with the DSMB, early proof of concept is creeping up on us a lot quicker than many envisage.
Very excited to see the next RNS land on dose escalation.
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