A Diabetes Drug Targeted IBM's Failing Muscle Energy in a New Clinical Trial
Last week we reported on a Mayo Clinic study showing that the energy-making machinery inside IBM muscle cells — the mitochondria — is damaged in multiple ways. A natural question follows: what happens if you try to fix it? A clinical trial published in March 2026 in Nature Communications did exactly that, testing a drug called pioglitazone that is designed to boost mitochondrial function.
What the Researchers Did
The trial, led by Dr. Brian Adler and colleagues at Johns Hopkins School of Medicine, was a small phase 1 study in 16 people with IBM. After a 16-week observation period to measure each person's natural trajectory, participants took pioglitazone — a pill already approved for type 2 diabetes — for 32 weeks. Muscle samples, blood tests, and functional measures were compared before and during treatment. The trial was shortened by the COVID-19 pandemic, and 13 people completed treatment.
What They Found
Before treatment, IBM muscle showed broad metabolic abnormalities compared with healthy muscle. During pioglitazone treatment, the muscle's metabolic profile shifted back toward a healthier pattern, with measurable increases in the pathways mitochondria use to make energy (oxidative phosphorylation).
Overall, clinical measures did not clearly change. But in an exploratory analysis, the subset of patients whose muscle metabolism improved the most showed slower decline on the IBM-FRS — a standard measure of daily function — and on the timed up-and-go test. The authors are careful to call this preliminary and correlative, not proof that the drug helps.
What This Means for IBM Patients
Two points matter here. First, this is one of the first trials to show that a drug can actually shift energy metabolism inside IBM muscle — suggesting muscle energy is a targetable problem, not just something to measure. Second, it is a small, early-phase study: pioglitazone did not clearly improve function, and it is not a recommended IBM treatment. It also carries real risks — one participant had a heart-failure exacerbation — so nobody should consider taking it without medical supervision.
The bigger picture: taken together with the Mayo Clinic study published this month in the journal Brain, a consistent story is emerging — mitochondrial damage is a core feature of IBM, and researchers are beginning to test whether repairing muscle energy could slow the disease. Larger, controlled trials would be needed before any of this reaches patients.
Source
• Adler BL, Bene MR, Zhang C, et al. "Modifying muscle metabolic dysregulation in inclusion body myositis with pioglitazone: a single-arm trial." Nature Communications. 2026 Mar 15. doi: 10.1038/s41467-026-70262-0.
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