ideasIdea
Public reference standards and potency assays for CAR-T so every lab measures alike
There is no agreed ruler for measuring how strong a CAR-T product is. Shared reference materials would let hospitals, companies and regulators compare products fairly.
Potency is the release attribute regulators worry about most for cell therapies, and every developer uses a different in-house assay, making comparability, decentralised manufacturing and biosimilar-like competition impossible. The proposal is a programme, led by a metrology institute such as NIST or NIBSC with the ISCT and regulators, to produce certified reference CAR-T cell materials, reference target cells and standardised cytotoxicity and cytokine assays with published acceptance criteria, made available at cost.
Hypothesis
Adoption of reference standards reduces inter-laboratory coefficient of variation in CAR-T potency measurement from above 40% to below 15% and is accepted by FDA and EMA as the basis for site-to-site comparability without clinical bridging.
Rationale
Reference standards underpinned the biosimilar revolution and the standardisation of flow cytometry for CD34 counting. Cell-therapy potency is technically harder but not different in kind.
What would test it
Produce pilot reference materials, run an international ring trial across 20 laboratories, and publish variance before and after standardised protocols.
Maturity
early clinical
Who has to act
research
Cost to try
Medium ($1M to $50M)
Years to first evidence
3
Bottlenecks it attacks
- Manufacturing cost and time for living and radioactive medicines · Cell therapies take weeks to make for one patient and cost hundreds of thousands of dollars. Isotopes run short.
- Preclinical results do not reproduce · Fewer than half of landmark cancer biology findings reproduce when someone else tries.