Dasatinib, developed by BMS and marketed as Sprycel©, is FDA-approved for some children with CML (November 2017). This on-market tyrosine kinase inhibitor (TKI) demonstrates better toxicological parameters than Rapamycin. Palliative effects on HCM and cardiac function were observed at dosages far below those employed in cancer therapy. This implies that a regiment of low-dose Dasatinib may improve HCM and cardiac function in NS, and reverse HCM in NSML (80% of cases).
IGIA Pharmaceuticals is repurposing dasatinib (IG-100), an orally bioavailable inhibitor of multiple signaling kinases, for the treatment of Noonan Syndrome (NS) and Noonan Syndrome with Multiple Lentigines (NSML) with associated cardiac dysfunction in infants. Infants presenting with hypertrophic cardiomyopathy (HCM) during the first 6 months of life have a 69% risk of dying during the first year of life. Once HCM is established, and particularly after development of Congestive Heart Failure (CHF), it may be too late for effective pharmacological intervention.