Autoimmune breakthrough holds phenomenol promise
In my 40 years of practicing medicine, I’ve been privileged to have a front-row seat to therapeutic advances surpassed only by the medical revolutions of vaccines, anesthesia and antibiotics.
In the 1980s, I gave patients clot-busting drugs that made massive heart attacks vanish before my eyes. Two decades later, new cancer treatments — especially immunotherapy — brought remissions, and even cures sometimes, to those who previously would have had died rapidly from their tumors.
The incredibly precise monoclonal antibody therapies that came to market in the late 1990s allowed us to tame debilitating illnesses like Crohn’s disease and rheumatoid arthritis. More recently, the holy grail of truly effective treatment for obesity is in our hands with drugs like Ozempic.
For all of these wonders, however, I think we may be on the verge of even more astounding treatments, some of which I never imagined. I’d like to introduce you to one of these therapies, still unproven, yet so full of potential it deserves our attention.
One in eight Americans has an autoimmune disorder. These diseases are highly diverse, and the symptoms they cause can be so confusing that diagnoses are often difficult to secure.
More than 100 autoimmune diseases exist, common ones including lupus, ulcerative colitis, psoriasis, rheumatoid arthritis, celiac disease, type 1 diabetes, Grave’s disease and multiple sclerosis.
Symptoms can vary from mildly annoying to organ-damaging and life-threatening. For patients with severe disease, potent and very expensive drugs are used to suppress parts of the immune system that are running out of control.
Newer medicines have proven a boon to both patients and the pharmaceutical companies, which make billions in profits from them.
Because of the tremendous diversity of autoimmune disorders, I never suspected that a single treatment to rid the body of these various diseases would be possible. But that may be the case.
An article published in The New England Journal of Medicine earlier this year shocked me in a way no previous study had.
The research in this case only evaluated 15 patients, a paltry number that did not allow broad conclusions from the results. Still, the findings were so stunning that I suspect it may portend a revolution in autoimmune disease.
The researchers provided these patients — eight with lupus, three with muscular inflammation and four with systemic sclerosis — with cutting-edge CAR-T cell therapy, which I’ll explain shortly.
All patients suffered severe, progressive disease resistant to at least two standard drugs for their conditions. These were the sickest of the sick.
CAR-T cell therapy was used initially for treating cancers. It involves removing immune T-cells, capable of spurring an immune response in the body, and “training” them to look for certain antigens, often proteins on the surface of the patient’s own cancer cells.
This is truly targeted therapy, because when reinfused, those T-cells summon the patient’s immune system to attack just the specific cancer cells.
In this case, the researchers targeted specific white blood cells — part of the patient’s own immune system — that were responsible for the autoimmune disease. The T cells went to work, and the rogue white blood cells rapidly disappeared.
The part of the study that shocked me was how the patients responded.
I am used to studies that show “moderate improvement” or “decrease in symptoms” from effective medications. This study used the terms “remission,” symptom level “equal to zero” and “disease activity ceased.”
And it wasn’t just a few that improved. As the researchers reported, “All 15 patients successfully discontinued glucocorticoids and all other immunosuppressive medications.”
I had to convince myself there wasn’t something I was missing. It seemed too jaw-droppingly good to be true.
So I read the accompanying editorial, where one can often find caution and skepticism. And while the author was careful to state that more and larger studies are needed, he included this extraordinary question, “Could CAR-T cell therapy cure autoimmunity?” And trust me when I say “cure” is a word that researchers rarely utter.
This is only one study, and much needs to be learned. But we may look back and see this study marking a “penicillin moment” when we found a path for the cure of an entire class of illnesses.
This is even more remarkable because these diseases are typically chronic, lifelong conditions, unlike infections where the body often provides the eventual cure on its own.
Medicine is moving forward at an accelerating pace. New tools are leading to exceptional breakthroughs. I can’t predict what is coming, but I find myself increasingly optimistic and excited.
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