Drug repurposing is the process of developing new uses for an existing drug or candidate compounds. This approach takes advantage of work that has been done for a different therapeutic use, therefore decreasing development costs and time to launch. Repurposing involves convincing someone who is developing a drug for a different indication that they should investigate PKD.
If a drug under development is at or near candidate stage, we already know a lot about how it can be delivered, what its pharmacokinetic properties are, what doses hit the intended target reliably, what safety issues exist, if any, and the candidate can be prepared and formulated.
Where We Are Now
Our mission says it best – discover treatments and a cure for PKD; improve the lives of those it affects. Continuous progress has been made toward these goals, thanks to the PKD Foundation’s efforts to harness the best minds in science, recruit the finest research talent and foster understanding of PKD within the clinical setting.
The PKD Foundation’s significant and productive investments in basic PKD research are now ready for harvest. The field of PKD is ripe for a significant growth in translational research and development that will more effectively harness basic discoveries and spawn new therapies for PKD.
Aligning For A Promising Future
To begin this journey into drug repurposing and translational research, we hired our first Chief Science Officer, Jill Panetta, PhD, and recruited a Drug Development Advisory Group.
Dr. Panetta took the reins of the PKD Foundation’s drug development efforts in spring 2010 with the directive to turn PKD basic science into clinical advances and, hopefully, treatments for PKD. Her experience in matching scientists to research and development challenges is unprecedented. She brings with her 20 years in drug development research at Eli Lilly and Company as well as being the co-founder of InnoCentive , a drug research and development organization. In short, Dr. Panetta has the experience and leadership needed to drive PKD drug development efforts toward potential PKD treatments.
Where We Are Headed
Many pharmaceutical and biotech organizations have compounds that may prove effective in slowing disease progression in ADPKD but are unaware of this potential new application for their drugs. To help open their eyes to this possibility, the PKD Foundation hosted a meeting lead by PKD experts to educate attendees from pharmaceutical and biotech industries, as well as representatives from the Food and Drug Administration and other key partners on the science of ADPKD, about the molecular and cellular mechanisms involved in the cyst formation and enlargement and cellular and animal models of renal cystic disease used in drug evaluation and preclinical testing.
The meeting format was designed to mix PKD researchers and pharmaceutical and biotech researchers, to build new alliances and to build interest in moving drugs forward in PKD.
From that meeting came strong confirmation we are on the right path to launch the new Accelerating Therapies to Patients initiative.
Dr. Panetta then assembled a team to help create a roadmap on how to use what we know to find a treatment for PKD. A list of drug targets/mechanisms/pathways that are implicated in the pathogenesis of PKD was compiled by select members of the PKD research community. Our new Drug Development Advisory Group, including highly-respected leaders in the pharmaceutical and biotech industries, is charged with priotizing drug targets, identifying potential compounds and regularly evaluating results to ensure success.
Most recently, the PKD Foundation contracted with PreClinOmics and PharmOptima, two Contract Research Organizations (CROs), to provide preclinical evaluation of several compounds in three animal models of renal cystic disease. There are three rodent strains with renal cystic disease being used as the animal models; one for ARPKD, one for slowly progressive ADPKD and one for rapidly progressive ADPKD. Currently, validation of the two rodent models is ongoing: Tolvaptan and Octreotide, drugs tested earlier in these models and found to slow cyst growth, are being tested again in this new setting to make sure that their effects can be reproduced. Once validation is complete, new compounds can be tested.
Through these five initiatives, the PKD Foundation aims to maximize the number of innovative compounds heading into clinical trials, thus increasing the odds a successful treatment is found. PKD patients and their loved ones have waited long enough and need medications to reduce the effects of PKD until a cure can be found. We are excited to take this next step in fulfilling our mission and improving the lives of all those PKD affects!
The PKD Foundation has created several resources to help better explain drug development and drug repurposing. Please choose from the links below.
Drug Development Timeline
Drug Development Machine