CAR-T Cell Adduct Modification for Cytokine Release Syndrome Control

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Solution Overview

Problem

Current CAR-T cell therapies face challenges with cytokine release syndrome (CRS), a potentially lethal complication due to uncontrolled expansion and immune activation, particularly in patients with large tumor burdens, where severe CRS can lead to multiorgan system failure.

Innovation Solution

Introducing adducts into the genomic nucleic acids of CAR-T cell-derived effector cells to inhibit further division and proliferation, while maintaining immunologic function, thereby reducing the severity of cytokine release syndrome and ensuring targeted disease treatment.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If CAR-T cells are expanded and activated to mediate clinical benefit, then antitumor efficacy is improved, but cytokine release syndrome severity increases

Engineering Contradiction:
Improveantitumor efficacyVSAvoidcytokine release syndrome severity
Core Design Contradiction:
ReliabilityVSObject-affected harmful factors

Solution Approach 1:

The patent applies preliminary action by introducing nucleic acid targeting compounds into CAR-T cells before infusion to pre-limit their proliferation capacity. This preventive measure ensures that while cells maintain full antitumor activity, they cannot undergo uncontrolled expansion that would trigger severe CRS, thus resolving the contradiction between efficacy and safety

Inventive Principle:
Principle #10Preliminary action

Solution Approach 2:

The patent changes the proliferative parameter of CAR-T cells by introducing nucleic acid adducts that specifically inhibit cell division without affecting cytolytic function. This parameter modification allows cells to exert therapeutic effect while preventing the excessive proliferation that causes cytokine release syndrome

Inventive Principle:
Principle #35Parameter changes

2Productivity

If high-levels of CAR-T cell expansion are achieved, then clinical benefit is improved, but cytokine release syndrome incidence increases

Engineering Contradiction:
Improveclinical benefitVSAvoidcytokine release syndrome incidence
Core Design Contradiction:
ProductivityVSObject-affected harmful factors

Solution Approach 1:

The patent applies partial action by allowing sufficient CAR-T cell expansion to achieve clinical benefit while intentionally limiting excessive expansion through nucleic acid targeting compounds. This controlled partial proliferation prevents CRS incidence while maintaining enough cell numbers for therapeutic effect

Inventive Principle:
Principle #16Partial or excessive action

3Reliability

If CAR-T cells are infused to treat cancer, then disease treatment is achieved, but multiorgan system failure risk increases

Engineering Contradiction:
Improvedisease treatmentVSAvoidmultiorgan system failure risk
Core Design Contradiction:
ReliabilityVSObject-generated harmful factors

Solution Approach 1:

The patent applies preliminary anti-action by pre-treating CAR-T cells with nucleic acid targeting compounds that counteract the potential for uncontrolled proliferation before infusion. This preemptive measure eliminates the root cause of multiorgan failure risk while preserving the cells' ability to treat cancer effectively

Inventive Principle:
Principle #9Preliminary anti-action

Applied Scientific Principles

This section explains which scientific principles are used to turn an abstract innovation direction into a practical engineering solution.

Function Achieved in This Case

The approach effectively limits CAR-T cell proliferation in vivo, reducing the severity of cytokine release syndrome and maintaining the cells' ability to target cancer cells, thus enhancing the safety and efficacy of CAR-T cell-based therapies.

Implementation Method 1

modifying the nucleic acid of the T cells in the expanded T cell population by reaction with a nucleic acid targeting compound that reacts directly with the nucleic acid

Methodology Applied
Scientific EffectDNA adduct formation: Chemical Bonding

Implementation Method 2

the adducts are introduced with a frequency necessary to prevent cell division

Methodology Applied
Scientific EffectDNA crosslinking: Chemical Bonding

Data Source

PatentEP3215139B1Compositions and methods for improved car-t cell therapies
Publication Date: 2020.08.19 CERUS CORP
  • EP3215139B1 patent drawingFigure 1A~1B
  • EP3215139B1 patent drawingFigure 2
  • EP3215139B1 patent drawing

AI summary

The present invention relates to the preparation and use in recipients of CAR-T cell-derived effector cells which are modified to limit their proliferation within the recipient. This is accomplished through the introduction of adducts into the nucleic acids of CAR-T cell-derived effector cells following expansion in vitro to provide expanded and activated CAR-T cell-derived effector cells that retain immunologic function, including the expression of one ore more cytokines.