Technology ID
TAB-5056

Oral Iron-Chelator Therapy for Treating Developmental Stuttering

E-Numbers
E-186-2023-0
E-186-2023-1
Lead Inventor
SheikhBahaei, Shahriar
Lead IC
NINDS
Applications
Therapeutics
Therapeutic Areas
Psychiatry/Mental Health
Neurology
Development Stages
Pre-clinical (in vivo)

This technology discloses the use of small-molecule iron chelators—drugs that bind and remove excess iron—for the oral treatment of developmental stuttering in children and adults. Mouse models carrying human stuttering mutations show both elevated striatal iron and impaired vocalization; daily low-dose deferiprone reverses these speech-like deficits while normalizing brain-iron MRI signals. Because 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. The approach offers the first disease-targeted pharmacologic option for a disorder currently managed only by speech therapy.

Commercial Applications
  • Stand-alone or adjunctive pharmacotherapy for persistent developmental stuttering in pediatric and adult populations.
  • MRI-guided precision treatment platform for speech-motor disorders linked to basal-ganglia iron dysregulation.
  • Expansion into related neurodevelopmental or movement disorders where excess neural iron contributes to pathophysiology.
Competitive Advantages
  • 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.
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