Summarized by Daily Strand AI from peer-reviewed source
Researchers at Johns Hopkins have developed tiny biodegradable particles that can reprogram a patient's own immune cells from inside the body, potentially replacing one of the most complex and expensive procedures in modern medicine. The particles, made from a water-degradable polymer, carry a snippet of mRNA — the same type of genetic messenger used in COVID-19 vaccines — that instructs a specific type of immune cell called a T cell to hunt down and destroy B cells. B cells are the culprits behind blood cancers like leukemia and lymphoma, as well as autoimmune diseases like lupus, where the immune system attacks healthy tissue. The engineered T cells that result are called CAR T cells, short for chimeric antigen receptor T cells, which are essentially T cells fitted with a custom targeting system.
In mouse experiments, a single injection of these nanoparticles wiped out 95% of circulating B cells within 24 hours and destroyed about half of the B cells residing in the spleen. After a week, blood B cell levels had recovered to roughly half their original number. The nanoparticles work in stages: they are coated with molecules that help them find and latch onto T cells, stimulate those T cells to become active, slip inside them, and then break apart to release the mRNA cargo. The new design uses only three components, compared to the four or five required by competing lipid-based nanoparticle systems, and can be stored frozen for long periods — a practical advantage that existing lipid-based formulations do not offer.
Current CAR T cell therapies require extracting blood from a patient, shipping it to a specialized facility, engineering the T cells in a lab over several weeks, and infusing them back — a process that can cost hundreds of thousands of dollars and is available only at select medical centers. A ready-made injectable nanoparticle that accomplishes the same reprogramming inside the patient's own body could dramatically lower costs and expand access to treatment for blood cancers and autoimmune diseases that affect millions of people worldwide. The simpler formulation and freeze-storage capability also mean the therapy could potentially be manufactured at scale and distributed more like a conventional drug.
That said, this research is at an early preclinical stage. All data so far come from healthy mice, not from animals or people with actual cancer or lupus. Safety and effectiveness in human disease models have not yet been established, and significant additional research will be required before this approach could reach clinical trials. The team says studies in both mouse disease models and human models are currently underway.
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