A Novel Drug Candidate for Treatment of Diabetic Retinopathy
Diabetic retinopathy (DR) is a common complication of diabetes. Diabetic macular edema (DME) is a major pathological feature of DR and a leading cause of blindness. Cystoid macular edema (CME) represents a common cause of vision impairment following cataract surgery. High incidence of CME secondary to cataract surgery occurs in diabetic patients. Breakdown of the blood-retinal barrier or retinal vascular leakage is believed to be responsible for DME and CME. Laser photocoagulation has remained the gold standard for the treatment of DME/CME over the past two decades. However, laser therapy is not always effective, may destroy normal retinal tissues and often is accompanied by side effects. Intravitreal steroids and vascular endothelial growth factor (VEGF) inhibitors have been developed for other ocular diseases. Clinical trials have shown that both therapies confer benefit to some patients with DME and CME, but many patients show only a partial response, and the therapies result in side effects. As there is no FDA-approved drug for DME and CME, the development of effective therapies for these sight-threatening conditions is in demand.
Through a high-throughput screen of over 200 small molecule compounds, we have identified CLT-003 with promising efficacy on DME. CLT-003NP is a promising drug candidate because it has new and multiple targets and can achieve a sustained efficacy on retinal vascular leakage. The ultimate goal of this project is to combine CLT-003 with nano-technology to develop a new and sustained-release drug for DME and CME.
The University of Oklahoma Health Sciences Center
Jian-xing Ma, M.D., Ph.D.
Ying Chen, M.D., Ph.D.
Public Health Impact
Currently, there is no FDA-approved pharmacotherapy for the treatment of diabetic retinopathy. The goal of this project is to combine nanotechnology and a novel promising small molecule drug candidate to develop a sustained-release drug treatment of diabetic retinopathy.
Approved studies are ongoing.
- Synthesis of GMP and non-GMP material
- Formulation development
- PK/ADME studies
- IND-directed toxicology