CDM1 Clinical Study Yields Encouraging Results
Discoveries made by the research lab of Lubov Timchenko, PhD, at Cincinnati Children’s Medical Center led to the first clinical trial for congenital myotonic dystrophy type 1 (CDM1). Results from the phase IIa trial, completed in 2017, showed positive results in patients, confirming Timchenko’s academic findings spanning the last two decades.
CDM1 causes muscle weakness and cognitive dysfunction and affects young patients; there is no known cure. The developing treatments for myotonic dystrophy type 1 (DM1) are mainly based on the use of antisense oligonucleotides to degrade toxic RNA in patients’ tissues. Timchenko proposed a different therapeutic approach, which is based on the correction of a signaling molecule, GSK3β kinase, which alters activity of RNA-binding proteins in patients with CDM1 and DM1.
AMO Pharma initiated a drug development program examining the therapeutic effects of the clinical-trial-ready inhibitor of GSK3β, AMO-02 in vivo models of DM1 and CDM1. This program included an initial Phase IIa clinical trial in a small group of patients with CDM1. Timchenko is a principal investigator of AMO Pharma’s pre-clinical studies investigating the benefits of AMO-02 in mouse models of DM1 and CDM1.
Phase IIa of the clinical trial tested safety and efficacy of AMO-02 in adolescent and adult patients with congenital and juvenile onset DM1, treated once daily with either 400mg or 1000mg AMO-02 orally over 12 weeks. Earlier this year AMO Pharma announced that the study revealed that AMO-02 is safe, and the treatment improves patients’ daily living activities and reduces CNS symptoms. The trial is currently in transition to Phase II/III for pediatric CDM1 in a multi-center international study.
“We are excited about these encouraging results in a small group of patients with CDM1,” Timchenko says. “The most critical step will be next phase of the trial in a large group of pediatric patients with CDM1. Since DM1 is a very complex and variable disease, more studies are needed to address specific challenges in children with CDM1.”
Timchenko says that at Cincinnati Children’s, a tight connection with the patient and family community motivates research studies toward the development of therapeutic approaches for CDM1 and DM1. Cincinnati Children’s is planning its second regional DM1 conference to provide an update of academic and clinical research for patients with DM1 and their families.
To learn more, contact Lubov.Timchenko@cchmc.org.
