LCAT Enzyme Delivery of Cholesteryl Ester to Steroidogenic Tissues

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Solution Overview

Problem

Patients with disorders such as adrenal insufficiency and systemic inflammatory response syndrome experience reduced levels of HDL-cholesterol ester, leading to impaired steroid production and exacerbated conditions, as existing treatments like artificial HDL lack cholesterol esters necessary for steroidogenesis.

Innovation Solution

Administering an effective dose of lecithin-cholesterol acyltransferase (LCAT) or a combination of LCAT with a cholesterol delivery particle (CDP) to normalize or increase HDL-cholesterol ester concentrations, thereby enhancing steroid production in steroidogenic tissues.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Object-affected harmful factors

If artificial HDL consisting of phospholipid and apolipoprotein A-1 is administered to remove excess cholesterol from tissues, then cholesterol removal from arteries is improved, but cholesterol delivery to steroidogenic tissues is worsened because artificial HDL lacks cholesterol esters

Engineering Contradiction:
Improveexcess cholesterol in arteriesVSAvoidcholesterol ester content in HDL
Core Design Contradiction:
Object-affected harmful factorsVSQuantity of substance

Solution Approach 1:

The patent introduces LCAT enzyme as an intermediary that catalyzes the conversion of free cholesterol to cholesteryl esters in HDL particles. This mediator enables the transformation of artificial HDL into a functional form that can deliver cholesterol esters to steroidogenic tissues while maintaining the ability to remove excess cholesterol from arteries.

Inventive Principle:
Principle #24Intermediary (Mediator)

Solution Approach 2:

The patent changes the chemical state of cholesterol in HDL by administering LCAT to convert free cholesterol into cholesteryl esters. This parameter change transforms artificial HDL from a cholesterol-removing agent into a cholesterol-delivery vehicle capable of replenishing steroidogenic tissues.

Inventive Principle:
Principle #35Parameter changes

2Quantity of substance

If HDL-CE levels are low in patients with adrenal insufficiency or systemic inflammatory response syndrome, then steroid production capacity is reduced, but the body cannot replenish CE stores quickly enough during periods of stress

Engineering Contradiction:
ImproveHDL-cholesterol ester concentrationVSAvoidsteroid production rate
Core Design Contradiction:
Quantity of substanceVSProductivity

Solution Approach 1:

The patent applies preliminary action by administering LCAT enzyme in advance to build up HDL-CE stores before periods of physiological stress. This preparatory measure ensures that steroidogenic tissues have adequate cholesterol ester reserves available when suddenly needed, preventing adrenal insufficiency and maintaining steroid production capacity during stress.

Inventive Principle:
Principle #10Preliminary action

Solution Approach 2:

The patent enables the body's own HDL particles to serve the dual function of both cholesterol removal and cholesterol ester delivery by activating LCAT. This self-service mechanism allows the endogenous HDL system to replenish steroidogenic tissues without requiring external cholesterol ester supplementation.

Inventive Principle:
Principle #25Self-service

3Quantity of substance

If LCAT is administered to increase HDL-CE levels, then cholesterol ester delivery to steroidogenic tissues is improved, but the complexity of the treatment regimen increases

Engineering Contradiction:
ImproveHDL-cholesterol ester concentrationVSAvoidtreatment protocol complexity
Core Design Contradiction:
Quantity of substanceVSDevice complexity

Solution Approach 1:

The patent employs a self-service approach where the administered LCAT enzyme utilizes the patient's own endogenous cholesterol and HDL particles to generate HDL-CE. This eliminates the need for complex exogenous cholesterol ester formulations or multiple complementary treatments, simplifying the therapeutic regimen to a single enzyme administration.

Inventive Principle:
Principle #25Self-service

Solution Approach 2:

The patent uses LCAT to create copies of the beneficial HDL-CE phenotype seen in CETP-deficient patients, where HDL particles are naturally enriched with cholesteryl esters. By catalyzing the formation of HDL-CE in treated patients, the therapy replicates the protective effect without requiring genetic modification or complex molecular engineering.

Inventive Principle:
Principle #26Copying

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

This approach effectively increases HDL-cholesterol ester levels, improving steroidogenesis and reducing the risk of life-threatening conditions by maintaining or elevating plasma steroid levels, particularly in critical care scenarios.

Implementation Method 1

the enzyme lecithin: cholesterol acyltransferase (LCAT) enhances the cholesterol-carrying capacity of HDL by catalyzing the transesterification of a fatty acid from lecithin in HDL to cholesterol to form cholesteryl ester (CE)

Methodology Applied
Scientific EffectTransesterification:

Implementation Method 2

The same HDL receptor-mediated process of CE delivery occurs in steroidogenic tissues to maintain a supply of cholesterol for the production of steroids. The HDL receptor for this process is currently thought to be the scavenger receptor B1 (SR-B1).

Methodology Applied
Scientific EffectReceptor-mediated transport:

Implementation Method 3

Cholesteryl ester transfer protein (CETP) is a plasma protein responsible for the net transfer of CE from HDL to the beta-lipoproteins (VLDL and LDL).

Methodology Applied
Scientific EffectCholesteryl ester transfer:

Data Source

PatentEP2566505B1Delivery of cholesteryl ester to steroidogenic tissues
Publication Date: 2019.10.16 ALPHACORE PHARMA LLC

AI summary

Disclosed are compositions and methods for treating conditions characterized by low HDL-CE which can lead to decreased delivery of cholesteryl ester to steroidogenic tissues, reducing the organ's ability to produce steroids especially during periods of demand, stress and or systemic inflammatory response syndrome.