In Focus podcast | Can a cancer therapy help treat autoimmune diseases?

CAR-T (Chimeric Antigen Receptor T) cell therapy, which has revolutionised the treatment of certain blood cancers, is now demonstrating significant clinical ...

What Happened

CAR-T (Chimeric Antigen Receptor T) cell therapy, which has revolutionised the treatment of certain blood cancers, is now demonstrating significant clinical promise against severe autoimmune diseases including lupus, myasthenia gravis, and stiff person syndrome.
Kyverna Therapeutics' miv-cel (KYV-101), a CD19-targeting CAR-T therapy, achieved statistically significant clinical benefit across all primary and secondary endpoints in a registrational trial for stiff person syndrome — reversing disability and eliminating immunotherapies after a single dose.
The company announced plans to submit for FDA approval of miv-cel for stiff person syndrome in the first half of 2026, which would make it the first ever FDA-approved CAR-T therapy for an autoimmune disease.
In India, NexCAR19 — developed by ImmunoACT, a spin-off from IIT Bombay — became the first CAR-T therapy designed and approved in India (CDSCO approval: October 2023) and is available at over 30 hospitals across 10+ cities at approximately ₹40 lakh per treatment.
The global pivot of CAR-T from cancer to autoimmune disease represents a potential paradigm shift: using precision immunotherapy to "reset" a dysregulated immune system rather than suppress it broadly.

Static Topic Bridges

CAR-T Cell Therapy — Mechanism and Process

Chimeric Antigen Receptor T (CAR-T) cell therapy is a form of adoptive cell therapy in which a patient's own T lymphocytes (immune cells) are genetically engineered to express a synthetic receptor — the Chimeric Antigen Receptor (CAR) — that can identify and bind to a specific target protein (antigen) on diseased cells. The process is personalised: cells are extracted from the patient, modified in a laboratory, expanded into millions of copies, and reinfused. Once inside the body, the engineered T cells seek out and destroy cells bearing the target antigen. This is distinct from conventional immunotherapy (which boosts the immune system broadly) and from chemotherapy (which targets dividing cells).

Structure of a CAR: Extracellular antigen-binding domain (from lab-made antibody fragments) + transmembrane domain + intracellular signalling domain + co-stimulatory domain (e.g., 4-1BB)
Most common cancer target: CD19 — a protein expressed on B lymphocytes (white blood cells), overexpressed in many B-cell malignancies
Autoimmune disease target: Also CD19 or BCMA (B-cell Maturation Antigen) — since many autoimmune diseases are B-cell driven
Manufacturing: Takes approximately 2–4 weeks from cell extraction to infusion
Unlike small-molecule drugs, CAR-T is a one-time treatment (though relapse can occur)

Connection to this news: The same CD19-targeting mechanism that destroys cancerous B-cells in leukemia and lymphoma is now being applied to eliminate autoreactive B-cells that drive autoimmune diseases. The clinical results suggest a potential "immune reset" — a concept with profound implications for treating otherwise refractory autoimmune conditions.

FDA-Approved CAR-T Therapies — Regulatory Landscape

The United States Food and Drug Administration (FDA) has approved several CAR-T therapies for blood cancers since 2017, marking the first regulatory approvals of gene-modified cell therapies for any disease. All FDA-approved CAR-T therapies, as of 2026, are indicated for haematological (blood) malignancies. The FDA's Centre for Biologics Evaluation and Research (CBER) regulates CAR-T therapies under Biologics License Applications (BLAs), treating them as gene therapy products.

First FDA-approved CAR-T therapy: tisagenlecleucel (Kymriah) — approved August 2017 for acute lymphoblastic leukemia in patients up to age 25
Current indications for FDA-approved CAR-T therapies include: Acute Lymphoblastic Leukemia (ALL), Diffuse Large B-Cell Lymphoma (DLBCL), Follicular Lymphoma, Mantle Cell Lymphoma (MCL), Chronic Lymphocytic Leukemia (CLL), Multiple Myeloma
All current approvals target B-cell cancers (CD19) or multiple myeloma (BCMA)
Side effects: Cytokine Release Syndrome (CRS) — a potentially life-threatening inflammatory response — and neurotoxicity (ICANS) are the primary safety concerns
FDA has signalled a "tailored approach" to CAR-T therapies for autoimmune diseases, given the different risk-benefit profile compared to cancer patients

Connection to this news: Miv-cel's expected FDA submission for stiff person syndrome would be the first BLA seeking approval of a CAR-T therapy in a non-cancer, non-fatal disease context — a regulatory and ethical frontier that requires balancing the therapy's significant risks against chronic autoimmune disease burden.

India's CAR-T Development — NexCAR19 and ImmunoACT

India developed its own indigenous CAR-T therapy through a collaboration between IIT Bombay's Biosciences and Bioengineering Department and ImmunoACT, a company incorporated in 2018 as a spin-off of IIT Bombay research that began in 2013. NexCAR19 — a humanised, second-generation anti-CD19 CAR-T therapy — received CDSCO (Central Drugs Standard Control Organisation) approval in October 2023, making India one of the very few countries to have an indigenously developed and approved CAR-T therapy. The President of India launched NexCAR19 at IIT Bombay, underscoring the national significance of the achievement.

NexCAR19 developer: ImmunoACT (IIT Bombay spin-off; research began 2013, company founded 2018)
CDSCO approval: October 2023 — India's first indigenously approved CAR-T therapy
Target: CD19 (B-cell antigen); "humanised" antibody domains to reduce immune rejection
Cost in India: approximately ₹40 lakh (compared to ₹5 crore+ for imported therapies)
Global cost: USD 350,000–500,000 per treatment in the United States
Availability: 30+ hospitals across 10+ cities in India as of 2024
Tata Memorial Centre (Mumbai) was a key clinical trial partner

Connection to this news: India's NexCAR19 represents the "Make in India" model for advanced gene therapy — developed entirely domestically at a fraction of global costs. As the technology expands from cancer to autoimmune disease applications globally, India's existing indigenous capability positions it to potentially extend NexCAR19 or develop new products for the autoimmune space.

Key Facts & Data

CAR-T therapy: genetically engineered T-cells with chimeric antigen receptors to target specific disease antigens
First FDA-approved CAR-T: tisagenlecleucel (Kymriah), August 2017, for ALL
All current FDA-approved CAR-T therapies target blood cancers (CD19 or BCMA antigens)
Miv-cel (KYV-101) by Kyverna Therapeutics: FDA submission planned H1 2026 for stiff person syndrome — would be first CAR-T approval for an autoimmune disease
Autoimmune diseases in trial: lupus nephritis, myasthenia gravis, stiff person syndrome, systemic sclerosis
NexCAR19: India's first indigenous CAR-T; CDSCO approval October 2023; developed by ImmunoACT (IIT Bombay spin-off)
NexCAR19 cost: ~₹40 lakh in India vs. ₹5 crore+ for imported alternatives
Available in: 30+ hospitals across 10+ Indian cities (as of 2024)
Key safety concern: Cytokine Release Syndrome (CRS) and neurotoxicity (ICANS)
Regulatory body in India: CDSCO (Central Drugs Standard Control Organisation) under Ministry of Health