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Autologous T Regulatory Cells Expressing An Hla-A2-Specific Chimeric Antigen Receptor

This article discusses the ongoing clinical trials of QEL-001, an innovative drug designed to prevent liver transplant rejection. QEL-001 is a type of cell therapy using autologous T regulatory cells that have been modified to express a specific chimeric antigen receptor targeting HLA-A2. The trials aim to evaluate the safety, tolerability, and clinical activity of this groundbreaking treatment in patients who have received a liver transplant with an HLA-A2 mismatch.

Table of Contents

What is QEL-001?

QEL-001 is an innovative medical treatment being developed to prevent liver transplant rejection[1]. It is classified as an advanced therapy, specifically a type of cell therapy[1]. The full name of this treatment is “Autologous T Regulatory Cells Expressing an HLA-A2-Specific Chimeric Antigen Receptor,” which is quite a mouthful! Let’s break this down:

  • Autologous: This means the treatment uses the patient’s own cells.
  • T Regulatory Cells: These are a type of immune cell that helps control the immune system’s response.
  • HLA-A2-Specific Chimeric Antigen Receptor: This is a special protein added to the T cells that allows them to recognize and target specific molecules related to transplant rejection.

How Does QEL-001 Work?

QEL-001 works by modifying a patient’s own immune cells to help prevent rejection of a liver transplant[1]. Here’s a simplified explanation of the process:

  1. Cells are collected from the patient’s blood through a process called leukapheresis.
  2. These cells are then modified in a laboratory using a lentiviral vector, which is a tool that introduces new genetic material into the cells[1].
  3. The modified cells are grown and multiplied.
  4. The patient receives the modified cells back through an intravenous infusion (into a vein)[1].

The goal is for these modified cells to help regulate the immune system and prevent it from attacking the transplanted liver, which could lead to rejection.

Who is QEL-001 For?

QEL-001 is being developed for a specific group of liver transplant patients[1]:

  • Patients who are HLA-A2 negative but have received a liver from an HLA-A2 positive donor.
  • Patients aged 18 to 75 years.
  • Patients who received their liver transplant between 12 months and 5 years ago.
  • Patients who have not had any episodes of rejection in the past 12 months.

HLA stands for Human Leukocyte Antigen, which is a type of molecule on cells that helps the immune system recognize what belongs in the body and what doesn’t. The mismatch in HLA-A2 status between the patient and the donor liver is what puts these patients at risk for rejection.

Clinical Trial Details

QEL-001 is currently being studied in a Phase I/II clinical trial[1]. This means it’s still in the early stages of research. The main goals of this study are:

  • To evaluate the safety and tolerability of QEL-001
  • To assess how well QEL-001 works in preventing liver transplant rejection
  • To determine if patients can safely reduce or stop their other immunosuppressive medications after receiving QEL-001

Potential Benefits

If successful, QEL-001 could offer several benefits to liver transplant patients[1]:

  • Reduced need for long-term immunosuppressive medications, which can have significant side effects
  • Lower risk of transplant rejection
  • Improved long-term outcomes for liver transplant patients

Safety Considerations

As with any new medical treatment, safety is a top priority in the study of QEL-001[1]. The clinical trial is designed to carefully monitor for any side effects or complications. Some key safety considerations include:

  • Monitoring for infections, as the treatment involves modifying immune cells
  • Checking for any unexpected immune reactions
  • Ensuring the modified cells don’t cause any unintended effects

It’s important to note that QEL-001 is still in the research phase, and more studies will be needed to fully understand its safety and effectiveness.

Aspect Details
Drug Name QEL-001
Drug Type Autologous T Regulatory Cells Expressing an HLA-A2-Specific Chimeric Antigen Receptor
Administration Intravenous injection
Target Condition Prevention of liver transplant rejection
Trial Phase Phase I/II
Primary Objectives Evaluate safety and tolerability of a single dose
Key Eligibility Criteria Adults 18-75, HLA-A2/A28 negative, received HLA-A2 positive liver transplant 12 months to 5 years prior
Primary Endpoints Incidence of dose-limiting toxicities and treatment-emergent adverse events
Key Secondary Endpoints Successful withdrawal from immunosuppression, incidence of infections, achievement of operational tolerance

FAQ

  • What is QEL-001? QEL-001 is a novel cell therapy treatment that uses a patient's own T regulatory cells, which have been genetically modified to express a chimeric antigen receptor targeting HLA-A2. It is designed to prevent liver transplant rejection in patients who have received a liver from an HLA-A2 positive donor.
  • Who is eligible for the QEL-001 clinical trial? Eligible participants are adults aged 18-75 who have received an HLA-A2 positive liver transplant 12 months to 5 years prior to the study. They must be HLA-A2/A28 negative themselves and meet specific health criteria, including stable immunosuppression and adequate organ function.
  • What are the main objectives of the QEL-001 clinical trial? The primary objectives are to evaluate the safety and tolerability of a single dose of QEL-001. Secondary objectives include assessing its clinical activity, monitoring safety-related events, and determining the presence or absence of replication-competent lentivirus.
  • How is QEL-001 administered to patients? QEL-001 is administered as a suspension for injection via intravenous use. Before receiving the treatment, patients undergo leukapheresis to collect their own cells, which are then modified to create the personalized therapy.
  • What are the potential benefits of QEL-001 for liver transplant patients? If successful, QEL-001 could potentially allow patients to reduce or eliminate their need for immunosuppressive medications. The trial aims to assess whether patients can achieve 'operational tolerance,' meaning they can maintain stable liver function without ongoing immunosuppression, which could significantly improve quality of life and reduce long-term complications.

Ongoing Clinical Trials on Autologous T Regulatory Cells Expressing An Hla-A2-Specific Chimeric Antigen Receptor

  • Study of QEL-001 (CAR T regulatory cell therapy) to prevent liver transplant rejection in patients with HLA-A2 positive donor organ

    Not recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    Belgium Spain

Glossary

  • Autologous: Derived from the patient's own cells or tissues.
  • T Regulatory Cells: A type of white blood cell that helps control the immune system's response and prevent it from attacking the body's own tissues.
  • Chimeric Antigen Receptor (CAR): A specially engineered receptor that can be added to immune cells to help them target specific proteins on other cells.
  • HLA-A2: A specific type of human leukocyte antigen (HLA), which is a protein found on the surface of cells that helps the immune system identify foreign substances.
  • Leukapheresis: A medical procedure that separates and collects white blood cells from the blood, while returning the rest of the blood components to the body.
  • Immunosuppression: The intentional reduction of the body's immune response, often used to prevent organ rejection after transplantation.
  • Operational Tolerance: A state where a transplant recipient can maintain stable organ function without the need for ongoing immunosuppressive medications.
  • Replication-Competent Lentivirus (RCL): A virus capable of reproducing itself, which is a safety concern in gene therapy treatments.
  • Dose-Limiting Toxicity (DLT): Side effects of a treatment that are severe enough to prevent an increase in dosage or require a reduction in dose.
  • Biopsy Proven Acute Rejection (BPAR): Rejection of a transplanted organ that has been confirmed through examination of a tissue sample under a microscope.

References

  1. http://clinicaltrials.eu/trial/study-on-qel-001-for-preventing-liver-transplant-rejection-in-patients-with-hla-a2-mismatch/