Scientists are trying a revolutionary new approach too treat rheumatoid arthritis, multiple sclerosis, lupus and other devastating autoimmune diseases — by reprogramming patients’ out-of-whack immune systems.
When your body’s immune cells attack you instead of protecting you,today’s treatments tamp down the amiable fire but they don’t fix what’s causing it. Patients face a lifetime of pricey pills, shots, or infusions with some serious side effects — and too ofen the drugs aren’t enough to keep their disease in check.
“We’re entering a new era,” said Maximilian Konig, MD, a rheumatologist at Johns Hopkins university in Baltimore, who’s studying some of the possible new treatments. they offer “the chance to control disease in a way we’ve never seen before.”
How? Researchers are altering dysfunctional immune systems, not just suppressing them, in a variety of ways that aim to be more potent and more precise than current therapies.
They’re highly experimental and, because of potential side effects, so far largely restricted to patients who’ve weary today’s treatments. But people entering early-stage studies are grasping for hope.
“What the heck is wrong with my body?” Mileydy Gonzalez,35,of New York remembers crying,frustrated that nothing was helping her daily lupus pain.
Diagnosed at 24, her disease was worsening, attacking her lungs and kidneys. Gonzalez had trouble breathing, needed help to stand and walk and couldn’t pick up her 3-year-old son when last July, her doctor at NYU Langone Health suggested the hospital’s study using a treatment adapted from cancer.
Gonzalez had never heard of that CAR-T therapy but decided, “I’m going to trust you.” over several months, she slowly regained energy and strength.
“I can actually run, I can chase my kid,” said Gonzalez, who now is pain- and pill-free. “I had forgotten what it was to be me.”
‘Living drugs’ Reset Rogue Immune Systems
CAR-T was developed to wipe out hard-to-treat blood cancers. But the cells that go bad in leukemias and lymphomas — immune B cells — go awry in a different way in manny autoimmune diseases.Some U.S. studies in mice suggested CAR-T therapy might help those diseases. Then in Germany, Georg Schett, MD, at the University of Erlangen-Nuremberg, tried it with a severely ill young woman who had failed other lupus treatment. After one infusion, she’s been in remission — with no other medicine — since march 2021.
Last month, Schett told a meeting of the American College of Rheumatology how his team gradually treated a few dozen more patients, with additional diseases such as myositis and scleroderma — and few relapses so far.
Those early results were “shocking,” Hopkins’ Konig recalled.
They led to an explosion of clinical trials testing CAR-T therapy in the U.S. and abroad for a growing list of autoimmune diseases.
How it works: Immune soldiers called T cells are filtered out of a patient’s blood and sent to a lab, where they’re programmed to destroy their B cell relatives. After some chemotherapy to wipe out additional immune cells, millions of copies of those “living drugs”## New Immunotherapies Offer Hope for Autoimmune Disease Remission
Recent advances in immunotherapy, initially developed for cancer treatment, are showing remarkable promise in treating autoimmune diseases like lupus and rheumatoid arthritis (RA). Researchers are exploring ways to “reset” the immune system, potentially leading to long-term remission and, in some cases, a functional cure. While still in early stages, these developments represent a important shift in the treatment landscape for millions affected by these chronic conditions.
### CAR-T Cell Therapy: A Breakthrough for Severe Autoimmune Disease
Chimeric antigen receptor (CAR) T-cell therapy, a type of immunotherapy where a patient’s own T cells are engineered to target and destroy specific cells, has traditionally been used to fight cancer. However, researchers are now adapting this approach to target the self-reactive immune cells that drive autoimmune diseases.
The therapy works by extracting T cells from the patient, genetically modifying them to recognize and eliminate the immune cells responsible for attacking the body’s own tissues, and then reinfusing them back into the patient. This process effectively “resets” the immune system.
Allie Rubin, a 60-year-old from Boca Raton, Florida, experienced this firsthand. After three decades battling lupus and a subsequent diagnosis of lymphoma, she qualified for CAR-T cell therapy. Despite a temporary side effect that delayed her recovery, Rubin has now been free of both cancer and lupus for nearly two years. “I just remember I woke up one day and thought, ‘Oh my god, I don’t feel sick anymore,'” she recounted. [https://www.npr.org/sections/health/2024/01/26/1226346641/autoimmune-disease-cure-car-t-cell-therapy-lupus-rheumatoid-arthritis](https://www.npr.org/sections/health/2024/01/26/1226346641/autoimmune-disease-cure-car-t-cell-therapy-lupus-rheumatoid-arthritis)
### Targeting Rogue T Cells and Prolonging Insulin production in Type 1 Diabetes
Beyond lupus, CAR-T therapy is being investigated for other autoimmune conditions. Researchers at the University of california, San Francisco, are exploring its use in type 1 diabetes, aiming to modulate rogue T cells that attack insulin-producing cells in the pancreas and, crucially, prolong insulin production. [https://medschool.cuanschutz.edu/centers/adpc/autoimmune-disease-prevention-center/research/stopra-national—for-participants](https://medschool.cuanschutz.edu/centers/adpc/autoimmune-disease-prevention-center/research/stopra-national—for-participants)
### Understanding the Path to Rheumatoid Arthritis
researchers are also applying these principles to rheumatoid arthritis (RA).A study tracking individuals with self-reactive antibodies – present in approximately 30% of those who eventually develop RA – for seven years has revealed crucial immune changes occurring *before* the onset of joint pain and swelling. [https://www.npr.org/sections/health/2024/01/26/1226346641/autoimmune-disease-cure-car-t-cell-therapy-lupus-rheumatoid-arthritis](https://www.npr.org/sections/health/2024/01/26/1226346641/autoimmune-disease-cure-car-t-cell-therapy-lupus-rheumatoid-arthritis)
These early changes represent potential targets for new drugs.Dr. Kevin Deane of the University of Colorado Anschutz Medical Campus is leading the StopRA: National study,which aims to identify and study more individuals at risk of developing RA to better understand the disease’s progression.### Challenges and Future Outlook
Despite the optimism, significant challenges remain. Concerns about the safety of CAR-T therapy and the duration of its effects are ongoing areas of research. However, CAR-T is currently the most advanced immunotherapy in testing for autoimmune diseases.
Dr. Konig of Johns Hopkins University believes the next decade will bring dramatic changes to the field. “We’ve never been closer to getting to – and we don’t like to say it – a potential cure,” she stated.[https://www.npr.org/sections/health/2024/01/26/1226346641/autoimmune-disease-cure-car-t-cell-therapy-lupus-