Drug repurposing is the process of developing new uses for existing drugs or drug candidates. This takes advantage of work that has been done for a different treatment use, thereby decreasing development costs and time to launch. A great example of drug repurposing is aspirin. Its original use was to treat pain, but it was discovered to be a great deterrent to heart attacks. Our drug repurposing program aims to foster discovery and development of therapies for PKD and to build a robust clinical trial pipeline in the most cost-effective, least risky and expedient way.
Why is drug repurposing important?
People with PKD can’t wait years – we need to accelerate treatments to patients today. The average development time for a drug is 17 years, with an average cost of $800 million to $1 billion. Drug repurposing shaves years and millions of dollars off of the process. It significantly decreases development costs and can move drug candidates into clinical testing for PKD much faster than traditional drug development.
What progress has been made?
Below are the conversion rates (the number of those who contacted the coordinator after learning about it from the ACT Alert) for the three ACT Alerts sent in fiscal year 2014.
- Identifying Genetic Modifiers in PKD Clinical Research Study in Atlanta
Conversion rate: 7%
- Identifying Genetic Modifiers in PKD Clinical Research Study in Colorado
Conversion rate: 12%
- Study of KD019 in ADPKD
Conversion rate: 0.2%
Phases of drug development
Where progress has been made?
Five drug candidates completed testing since the inception of the program. MSDC-0160, one of the candidates, was shown to slightly slow the disease progression in one animal model. An analog of this compound has undergone further evaluation.
In fiscal year 2014, two additional drug candidates completed initial screening (preclinical testing). One of these candidates showed effectiveness in one animal model and is moving to another animal model. If successful, this will be the first compound to show promise in two animal models.
Additional pharmaceutical companies have contacted us about potential testing of their compounds.
We invested $157,000 in the drug repurposing program in fiscal year 2014.
Accelerating Treatments to Patients (ATP)
The Accelerating Treatments to Patients (ATP) initiative is a comprehensive, integrated research and development program that represents the core of our work. ATP was launched in 2010 to speed up development of treatments which could slow or stop the progression of PKD. Each of the programs within ATP is important and interconnected.