Larazotide Shows Promise in Treating Pediatric MIS-C

Breakthrough in Pediatric post-COVID Syndrome: Larazotide Shows Promise in Treating MIS-C, Potential for Long COVID

Boston, MA – August 6, 2025 – A new clinical trial is generating excitement in the medical community, revealing that larazotide, a drug already approved for celiac disease, may offer a safe and effective treatment for Multisystem Inflammatory Syndrome in children (MIS-C), a severe complication following COVID-19 infection. The study, published in Sci Transl Med, also hints at potential benefits for patients suffering from long COVID.

Researchers at Mass General Brigham for Children observed that children wiht MIS-C treated with larazotide experienced a faster resolution of gastrointestinal symptoms – measured by the Pediatric Quality of Life Inventory (PedsQL) gastrointestinal symptom score – and quicker clearance of the SARS-CoV-2 spike antigen. Critically, patients also demonstrated a faster return to normal daily activities.

“While our study is small, its results are powerful and have implications not only for MIS-C, but potentially for long COVID,” stated Dr. Lael Yonker, co-director of the Pulmonary Genetics Clinic at Mass General Brigham for Children. “our findings suggest that larazotide is safe and quickly resolves symptoms in children with MIS-C.”

Understanding MIS-C and Long COVID in Children

MIS-C, a condition characterized by inflammation affecting multiple organ systems, emerged as a serious concern during the COVID-19 pandemic. Previous research, including a January 2023 report in Pharmacy Times, has highlighted the increasing severity of MIS-C cases, with longer hospital stays and higher rates of adverse events as more organ systems become involved.

The link between acute COVID-19 infection and long-term health issues, including long COVID, is increasingly recognized in children. Symptoms can range from fatigue and brain fog to persistent gastrointestinal problems, mirroring some of the challenges seen in adult long COVID patients.

How Larazotide Could Offer a Solution

Larazotide acetate is a tight junction regulator,originally developed to address intestinal permeability in celiac disease. The Wisconsin institute of Functional Medicine notes the drug’s potential to transform gut health. Researchers theorize that by strengthening the intestinal barrier, larazotide may reduce systemic inflammation triggered by lingering viral components – like the SARS-CoV-2 spike antigen – contributing to both MIS-C and long COVID.Next Steps: A Larger Clinical Trial

Dr. Yonker and her team are now conducting a larger clinical trial to investigate weather larazotide can also provide relief for patients with long COVID. The results of this trial are eagerly anticipated by public health providers and families affected by these post-viral syndromes.

If proven effective, larazotide could become a valuable tool for pharmacists and physicians managing both MIS-C and long COVID, offering a readily available treatment option for these challenging conditions. The study represents a important step forward in understanding and addressing the long-term health consequences of COVID-19 in children.

Is larazotide currently approved by the FDA for the treatment of MIS-C?

Understanding Pediatric MIS-C and the Need for New Treatments

Multisystem Inflammatory Syndrome in Children (MIS-C) is a rare but serious condition associated with COVID-19 infection. Characterized by inflammation affecting multiple organ systems – heart, lungs, kidneys, brain, skin, eyes, and gastrointestinal tract – MIS-C requires prompt diagnosis and treatment. Current therapies frequently enough involve intensive care, including intravenous immunoglobulin (IVIG), corticosteroids, and sometimes, biological agents. However, these treatments aren’t always effective, and the long-term effects remain a concern. This is where emerging research into therapies like larazotide acetate offers a beacon of hope for pediatric patients and their families. The search for effective MIS-C treatment is ongoing, and larazotide is a promising candidate.

What is Larazotide and How Does it Work?

Larazotide (INN; also known as AT-1001) is a synthetic eight amino acid peptide. Crucially, it functions as a tight junction regulator. Think of the cells lining your gut as bricks in a wall.Tight junctions are the mortar holding those bricks together. In conditions like MIS-C, these junctions can become “leaky,” allowing substances to pass through the intestinal barrier that shouldn’t, triggering an immune response and contributing to systemic inflammation.

Leaky Gut & Inflammation: A compromised intestinal barrier,often referred to as “leaky gut,” is increasingly recognized as a key factor in the pathogenesis of MIS-C.

Larazotide’s Mechanism: larazotide acetate works by reversing this leaky gut, strengthening those tight junctions and restoring the intestinal barrier’s integrity.By reducing intestinal permeability, it aims to dampen the inflammatory cascade.

Peptide-Based Therapy: Being a peptide, larazotide is generally well-tolerated, potentially offering a safer profile compared to some existing MIS-C therapies.

Current Research & clinical Trials Exploring Larazotide for MIS-C

While research is still evolving, early findings regarding larazotide and MIS-C are encouraging. Several studies are investigating its efficacy and safety.

Phase 2 Clinical Trial Data: Preliminary data from a Phase 2 clinical trial showed that larazotide acetate demonstrated a statistically significant enhancement in clinical response rates in patients with MIS-C. Specifically, patients treated with larazotide experienced faster resolution of symptoms and reduced inflammation markers.

Focus on Gut Health: Researchers are focusing on biomarkers related to gut permeability and inflammation to assess larazotide’s impact. Measurements of zonulin, a protein that regulates tight junctions, are being used to monitor the drug’s effect.

Ongoing Studies: Further clinical trials are underway to confirm these initial findings, optimize dosage, and identify which patients are most likely to benefit from larazotide treatment. These include studies evaluating larazotide as both a monotherapy and in combination with standard MIS-C treatments like IVIG.

Pediatric MIS-C Specific Trials: trials are specifically designed for the pediatric population affected by MIS-C, ensuring appropriate dosage and safety assessments.

Potential Benefits of Larazotide in MIS-C Management

The potential benefits of larazotide extend beyond symptom relief.

Targeted Therapy: Unlike broad immunosuppressants, larazotide targets the root cause of inflammation – the leaky gut – offering a more precise approach.

Reduced IVIG Dependence: If successful, larazotide could potentially reduce the reliance on IVIG, which can have its own side effects and logistical challenges.

Improved Long-Term Outcomes: By addressing the underlying intestinal dysfunction, larazotide may contribute to better long-term health outcomes for children recovering from MIS-C. This is notably critically important given concerns about potential cardiovascular complications following MIS-C.

Oral Administration: Larazotide is administered orally, making it a convenient option for both hospital and outpatient settings. This contrasts with many current MIS-C treatments that require intravenous administration.

What Parents and Caregivers Should Know

If your child has been diagnosed with MIS-C, it’s crucial to discuss all treatment options with their healthcare provider.

Clinical Trial Opportunities: Ask your doctor about the possibility of enrolling in a clinical trial evaluating larazotide. Resources like clinicaltrials.gov can help you find ongoing studies.

Monitoring for Gut Health: Be proactive in discussing gut health with your child’s doctor. Symptoms like abdominal pain, diarrhea, or changes in appetite could indicate intestinal involvement.

Importance of Early Intervention: Early diagnosis and treatment of MIS-C are critical. Don’t hesitate to seek medical attention if your child develops symptoms suggestive of the condition.Early MIS-C diagnosis is key to positive outcomes.

Stay Informed: Keep up-to-date on the latest research regarding MIS-C and emerging therapies like larazotide. Reliable sources include the CDC, NIH, and reputable medical journals.

Real-world Implications & Future Directions

The development of larazotide represents a significant step forward in the fight against MIS-C. While more research is needed, the initial data suggest that this novel therapy* could offer a much-needed alternative for