Scientists Explore New Paths for Autoimmune Treatment

Experimental therapies aim to reprogram immune systems

Researchers are investigating novel approaches to treat autoimmune diseases such as lupus, rheumatoid arthritis, and multiple sclerosis by reprogramming the immune system rather than suppressing it. Current therapies often require lifelong medication with limited effectiveness and significant side effects. Patients frequently rely on costly infusions or pills that manage symptoms but do not address the underlying immune dysfunction. The new strategies seek to offer more targeted and lasting solutions for those who have exhausted conventional options.

CAR-T Therapy and Immune Reset

One promising method involves CAR-T therapy, originally developed for blood cancers, now being tested for autoimmune conditions. The treatment uses modified T cells to eliminate malfunctioning B cells, which play a role in many autoimmune disorders. Early results from clinical trials in Germany and the United States show sustained remission in some patients, including those with lupus and scleroderma. Researchers believe the deep depletion of B cells may allow the immune system to reboot and regenerate healthy cells.

The process includes filtering T cells from a patient’s blood, programming them in a lab, and reinfusing them after chemotherapy. While effective, CAR-T is complex and expensive, with treatments costing up to $500,000. Some companies are developing off-the-shelf versions using donor cells to reduce cost and complexity. Despite its potential, CAR-T remains limited to patients with severe disease due to safety concerns and the intensity of the procedure.

Alternative Cellular and Molecular Strategies

Beyond CAR-T, scientists are exploring other cell-based therapies, including regulatory T cells that suppress inflammation. These “peacekeeper” cells are being engineered to calm autoimmune responses rather than destroy other cells. Another approach involves T cell engagers—lab-made antibodies that redirect existing T cells to target harmful B cells without custom engineering. Early trials with drugs like teclistamab have shown promising results, with several patients entering drug-free remission.

Precision targeting is also a focus, with researchers aiming to eliminate only the specific B cells responsible for disease. At Johns Hopkins, teams are developing T cell engagers that recognize biological markers unique to faulty B cells. Meanwhile, mRNA technology is being adapted to instruct immune cells to regulate themselves. Using biodegradable nanoparticles, scientists hope to deliver genetic instructions that encourage the growth of healthy regulatory cells.

Predicting and Preventing Disease

Efforts are underway to identify individuals at risk of developing autoimmune diseases before symptoms appear. In type 1 diabetes, the drug teplizumab has been approved to delay onset by modulating rogue T cells. Similar strategies are being explored for rheumatoid arthritis, where certain antibodies signal increased risk. Long-term studies are mapping immune changes that precede joint damage, offering potential targets for early intervention.

Dr. Kevin Deane leads a national study to better understand these preclinical stages and develop preventive treatments. While much research remains, the goal is to shift from reactive care to proactive disease management. Scientists caution that these therapies are still experimental and may not work for everyone. Nonetheless, the progress has sparked optimism among researchers and patients alike.