NIH inventors have developed the first pharmacologic option for developmental stuttering, which is currently only managed by speech therapy, but addresses a $3 billion underserved global market. This technology uses small-molecule iron chelators – drugs that bind and remove excess iron – for the oral treatment of developmental stuttering in children and adults. Mouse models have shown that using daily low-dose deferiprone reverses speech deficits while normalizing brain-iron MRI signals. Since exemplary chelators deferiprone, deferasirox, and deferoxamine are already marketed for other indications, their safety, dosing, and manufacturing are well characterized, enabling a streamlined regulatory path.

This technology could be used as a stand-alone or adjunctive pharmacotherapy for persistent developmental stuttering in pediatric and adult populations, for MRI-guided precision treatment platform for speech-motor disorders linked to basal-ganglia iron dysregulation, and/or expand into related neurodevelopmental or movement disorders where excess neural iron contributes to pathophysiology.

There are multiple competitive advantages of this potential therapeutic, including: 

• First-in-class, mechanism-based therapy for stuttering, addressing a $3 B underserved global market with no approved drugs.
• Repurposes FDA-approved iron chelators, leveraging extensive safety data and oral formulations for rapid Phase II entry and lower development risk.
• Quantifiable biomarker (MRI R2* brain-iron signal) enables patient stratification and objective efficacy read-outs, enhancing trial efficiency and market differentiation.

NIH inventors at the National Institute of Neurological Disorders and Stroke are seeking a licensee for this technology. If you are interested in learning more or contacting the licensing manager, you can do so via the abstract: Oral Iron-Chelator Therapy for Treating Developmental Stuttering