CAR-T Cell Therapy for Autoimmune Diseases: A New Era in Treatment

By SWA

Autoimmune diseases are conditions in which the body’s immune system mistakenly attacks its own cells and tissues. This can lead to chronic inflammation, pain, and damage to organs and systems throughout the body. Diseases like Systemic Lupus Erythematosus (SLE), Systemic Sclerosis, Antiphospholipid Syndrome (APS), Myositis, Primary Immune Thrombocytopenia (ITP), and Myasthenia Gravis are among the most challenging autoimmune disorders to treat.

In recent years, CAR-T cell therapy, originally developed to treat cancer, has emerged as a promising new approach for patients with severe autoimmune diseases who do not respond to conventional treatments.

What Are CAR-T Cells?

CAR-T stands for Chimeric Antigen Receptor T-cell therapy. It involves collecting a patient’s own T-cells from the bloodstream, genetically modifying them in a laboratory to express special receptors (CARs) on their surface, and reinfusing them into the patient. These modified cells can now recognize and eliminate specific immune cells involved in disease activity.

In autoimmune diseases, the goal is to eliminate harmful B-cells or autoreactive immune cells that are attacking the body.

Successful Applications and Current Research

1. Systemic Lupus Erythematosus (SLE)

CAR-T cells targeting specific B-cells—critical players in lupus pathology—have shown highly promising results in early clinical trials. Some patients achieved drug-free remission after treatment.

2. Systemic Sclerosis and Myositis

The University Hospital of Tübingen has reported successful treatments using CAR-T cells in patients with systemic sclerosis and inflammatory muscle disease (myositis). These are chronic, often therapy-resistant conditions that can severely limit quality of life.

3. Primary Immune Thrombocytopenia (ITP)

For the first time, CAR-T cell therapy has been applied successfully to ITP, a condition where the immune system destroys platelets, leading to dangerous bleeding risks.

4. Myasthenia Gravis

This neuromuscular autoimmune disease targets the acetylcholine receptor, impairing communication between nerves and muscles. Initial reports from the University Hospital Dresden indicate that CAR-T therapy could suppress the immune response effectively.

Diagnosis and Treatment – Who’s Involved?

  • Rheumatologists and endocrinologists are primarily responsible for diagnosing and managing autoimmune diseases.

  • General practitioners often play a key role in initial diagnosis and referral.

  • As CAR-T therapy becomes more integrated into practice, immunologists and hematologists will also be crucial in patient selection and monitoring.

CAR-T Therapy: How It Works

  1. T-cell collection: T-cells are taken from the patient's blood via a process similar to a blood donation.

  2. Genetic modification: In a lab, these cells are engineered to express a CAR that recognizes disease-related immune cells.

  3. Expansion and quality control: The modified T-cells are multiplied and tested for purity, functionality, and safety.

  4. Reinfusion: The cells are returned to the patient to begin targeting the harmful immune cells.

  5. Monitoring: Patients are closely observed for side effects and therapy response.

How is CAR-T Cell Therapy Tested?

Several methods ensure the therapy's effectiveness and safety:

  • T-cell counting: Measures the number of T-cells in the blood to assess immune competence.

  • PCR (Polymerase Chain Reaction): Detects and quantifies the presence of CAR sequences in the patient’s genome.

  • Routine blood tests: Monitor organ function (liver, kidney), and cell counts (white/red cells, platelets).

  • Quality control: Ensures the modified cells are free from contamination and are functioning as intended.

  • Monitoring side effects: Special attention is given to CRS (Cytokine Release Syndrome) and ICANS (immune effector cell-associated neurotoxicity syndrome).

Donation and Autoimmune Conditions

  • Blood Donation: Generally not allowed for people with autoimmune diseases due to concerns over immune instability and medications.

  • Organ Donation: Typically not permitted, but exceptions may be made in cases where only certain organs are affected.

  • Stem Cell Donation: May be possible, especially in cases involving blood disorders or congenital immune deficiencies.

Future Outlook

While CAR-T cell therapy is still in its early stages for autoimmune diseases, its success in cancer treatment gives it a strong foundation. Ongoing studies are expanding its use and refining its safety. As more data becomes available, this therapy may represent a paradigm shift in how we treat severe, therapy-resistant autoimmune diseases.

Key Takeaways

  • CAR-T therapy offers targeted and personalized treatment for autoimmune diseases by resetting or modulating the immune system.

  • Early studies in SLE, systemic sclerosis, ITP, and myasthenia gravis are encouraging.

  • The therapy requires rigorous testing, patient monitoring, and specialized medical teams.

  • While still experimental in autoimmune conditions, CAR-T has the potential to transform care for many patients who previously had limited options.

References:

Antiphospholipid Syndrome (APS) https://swaresearch.blogspot.com/2025/01/diagnosis-and-treatment-of.html

EULAR recommendations for the non-pharmacological management of systemic lupus erythematosus and systemic sclerosis https://ard.bmj.com/content/83/6/720


© 2000-2025 Sieglinde W. Alexander. All writings by Sieglinde W. Alexander have a fife year copy right. Library of Congress Card Number: LCN 00-192742

Comments

Post a Comment

Popular posts from this blog

Schnitzler Syndrome: A Rare Autoinflammatory Disorder

By SWA
Schnitzler syndrome is a rare, chronic autoinflammatory disease characterized by chronic urticaria (hives), recurrent fever, joint pain, and monoclonal gammopathy (abnormal proteins in the blood). First described by Dr. Liliane Schnitzler in 1972 , this condition is often misdiagnosed, leading to years of untreated symptoms before a correct diagnosis is made. The case report, " A 58-Year-Old Woman With Urticaria, Fever, and Joint Pain " , describes a case that closely aligns with my own experience with this challenging disorder. Key Features of Schnitzler Syndrome Schnitzler syndrome presents with a characteristic set of symptoms, though not all patients experience them in the same way: Chronic Urticaria (Hives) – Persistent, non-itchy or mildly itchy red patches that do not respond to antihistamines. Monoclonal Gammopathy (IgM or IgG type) – Abnormal monoclonal immunoglobulin detected in the blood (most commonly IgM ). Recurrent Fever – Unexplained fever episodes, often...
Read more

Dysferlin Protein: Key Roles, Genetic Locations

By SWA
Image
The purpose of this article is to share and discuss the details of a new discovery, which is highlighted within: CRISPR-Cas9: A Breakthrough Tool to Repair the DYSF Gene The dysferlin protein plays an essential role in maintaining the integrity of muscle cell membranes, particularly in tissues that endure significant mechanical stress, such as skeletal and cardiac muscles . Encoded by the DYSF gene , dysferlin is indispensable for repairing damaged cell membranes after injury caused by muscle contraction or external forces. This article explores the main locations where dysferlin is produced, its role in muscle repair, and supportive nutritional and therapeutic strategies for individuals with dysferlin deficiency, such as those affected by dysferlinopathies (e.g., Limb-Girdle Muscular Dystrophy Type 2B or Miyoshi Myopathy). Currently, there is no definitive answer as to why the severity of dysferlin protein expression or depletion varies. Do pathogens play a role in the methylation o...
Read more

Very Long-Chain Fatty Acids (VLCFAs) X-ALD and Spinal Muscular Atrophy (SMA): Exploring the Connection

By SWA
Update Oct 28th 2024:  A Novel Mouse Model for Cerebral Inflammatory Demyelination in X-Linked Adrenoleukodystrophy: Insights into Pathogenesis and Potential Therapeutic Targets https://onlinelibrary.wiley.com/doi/abs/10.1002/ana.27117 Very Long-Chain Fatty Acids (VLCFAs) are a specific type of fatty acid that are broken down in the body by specialized cellular structures called peroxisomes. These organelles play a critical role in maintaining metabolic balance by degrading VLCFAs. When this process is disrupted, VLCFAs accumulate in various tissues, which can lead to serious metabolic and neurological complications. Elevated VLCFA levels are characteristic of several peroxisomal disorders, most notably X-linked Adrenoleukodystrophy (X-ALD) and Zellweger Syndrome. In X-ALD, for example, a genetic defect impairs the peroxisomal breakdown of VLCFAs, resulting in their accumulation in the brain, spinal cord, and adrenal glands. This buildup can cause progressive neurological damag...
Read more