The PKD Foundation understands the urgency of finding a treatment for polycystic kidney disease. Patients have waited long enough – and that’s why we’re focusing on Accelerating Treatments to Patients (ATP), an initiative dedicated to finding a treatment as soon as possible.
Each program within ATP touches one of the following areas: new discoveries in basic PKD science, the development of potential clinical candidates, development of registration endpoints for clinical development, support of clinical trials and scientific meeting support.
Please use the links below to learn more about the ATP initiative:
PKD Outcomes Consortium Project (PKDOC)
Core Research Grants
Tissue Donation Program
Clinical Trial Awareness Program
What is it?
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.
This program is focused on assisting biotech and pharmaceutical companies or other research organizations in evaluating whether their drug candidates have potential as treatments for PKD.The goal is to foster the discovery and development of new therapies for PKD and to build a robust clinical trial pipeline in the most cost effective, least risky and expedient way.
People with PKD can’t wait years – we need to accelerate treatments to patients today. The average development time for a drug is 13 years, with an average cost of $750 million. 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
By using drug repurposing, we hope to move the successful compounds into clinical trials quickly. Our goal is to uncover compounds currently in use for other medical conditions that could successfully repurposed as a treatment for PKD.
While time is saved by using a repurposed drug, the studies are still a lengthy and complex process. It costs approximately $600,000 and takes two and a half years to move a drug candidate preclinical evaluation from start to finish. Of that amount, it costs $240,000 annually ($20,000 per month) to care for and manage the animals the drugs are tested on.
Since the beginning of this program we have invested $1.73 million and accomplished the following:
- Established the Drug Development Advisory Group
- Established a relationship with a Contract Research Laboratory (CRL) to conduct the tests.
- Five drug candidates in various stages have been evaluated in three animal models with renal cystic disease. One of those candidates shows promise.
- Through the Drug Advisory group’s network within the biotech and pharmaceutical industries we have identified many drug candidates that the group believes are worth pursuing and meet our criteria for selection.
123 of ATP – (pdf) A quick, easy-to-understand explanation of our ATP program.
PKD Outcomes Consortium Project (PKDOC)
What is it?
PKDOC is a significant collaboration between the PKD Foundation, Critical Path Institute, members of the pharmaceutical industry, PKD researchers and clinicians, and the Food and Drug Administration (FDA). The goal of the program is to facilitate clinical trial development for PKD therapies by establishing regulatory endpoints (or biomarkers) to measure early disease progression. Current traditional endpoints only show changes late in the course of the disease when kidneys are approximately five times normal size and normal tissue has been destroyed by hundreds of cysts and fibrotic tissue. At this stage, is may be too late for treatment.
The PKDOC has identified Total Kidney Volume (TKV) as an imaging biomarker that is most relevant for following the natural course of the disease. (A biomarker is a characteristic that can be measured reliably as an indicator of disease processes.)
Currently, glomerular filtration rate (GFR) is used to measure kidney function. Your doctor calculates it from creatinine levels in your blood and urine, your age, gender and other factors. The PKDOC Project believes Total Kidney Volume (TKV) – a measurement of the volume of the kidney - is a more appropriate measure of disease progression in ADPKD. Not only that, but it may indicate kidney damage sooner than GFR.
GFR remains relatively steady in PKD patients until late in the disease, when it falls rather suddenly. At this point, drug interventions are unlikely to improve kidney function since there is little normal tissue left. However, because TKV measures kidney volume which increases gradually over time, it can be used as a measure of disease progression early in the disease, when drug interventions are more likely to be effective.
Also, with GFR as the current outcome measurement, “time to transplant” is essentially the clinical read-out for a clinical trial. In other words, a clinical trial could run for over twenty years if GFR was the endpoint that had to be used. With TKV as the outcome measurement, clinical trials would have a new end point to follow based on volume rather than time.
Data from three patient registries and the HALT PKD clinical trial are being gathered into one database for analysis of the relationship between clinical factors associated with PKD, such as hypertension, hematuria and end stage renal disease, and Total Kidney Volume. The results of the data analysis will be used to prepare a biomarker qualification package to submit to the FDA. If approved, TKV could be then be used to facilitate clinical trial development initially as a prognostic biomarker.
This work has been solely funded by the PKD Foundation, an investment of more than $2.3 million.
On Nov. 9, 2012, an in-person meeting with the FDA took place to review the PKDOC summary and analysis. The steering committee believed it was a productive meeting and that good progress is being made. The analysis will continue to be refined for another submission. A second in-person meeting is anticipated for 2013, which will be followed by the formal submissions of the biomarker qualification package.
Core Research Grants
This program is focused on providing grants that support facilities and services PKD scientists at no cost or at a greatly discounted cost. This allows Foundation dollars to be leveraged across the PKD research community.
In the past year, $160,000 has been distributed among these core labs. They include:
- Electron Microscopy Core Facility for PKD Researchers, Dr. Vince Gattone, Indiana University School of Medicine
- This core is dedicated to facilitate research on polycystic kidney disease (and other hepatorenal fibrocystic diseases) and is well equipped to handle most electron microscopy needs. Services are provided at a significant discount because the costs are underwritten by the PKD Foundation.
- ADPKD Mutation Database (PKDB), Dr. Peter Harris, Mayo Clinic
- The PKDB has been established to facilitate the characterization of ADPKD gene variants in PKD1 and PKD2, the two genes known to have mutations causative of ADPKD. It is a repository for all variants in these genes, whether likely disease-causing or not. This database displays the most comprehensive listing of all published PKD1 and PKD2 mutations and includes the silent polymorphisms described to date. This information is available to PKD researchers at no cost because of the support of the PKD Foundation.
- PKD Research Biomaterials and Cellular Models Core, Dr. Darren Wallace, Kansas University Medical Center
- The PKD Biomaterials Core provides human PKD materials, including fixed and frozen tissues, cysts fluids and primary cells isolated from the cysts of human PKD kidneys retrieved during surgery.
- The Core also generates biological materials from animal models of PKD which can be used for high through-put drug screening, validation of drug targets and biomarker discovery.
- With this grant, a website for patients and researchers has been created, www.kumc.edu/pkdbiomaterials. Patients can learn about donating their kidneys to research, and researchers can learn how to get tissue to test hypotheses and evaluate drug effects.
This program is focused on funding PKD-relevant scientific and clinical meetings that bring together PKD scientists from around the world to discuss current findings and encourage research collaborations. The PKD Foundation has also funded medical education programs at national clinical meetings to educate medical professionals in the basic science, diagnosis, disease management and potential treatments for PKD.
We have provided funding through educational grants to support scientific conferences and meetings that support the Foundation’s mission. The funding has ranged from $5,000 to $50,000 per conference.
Many partnerships are being cultivated to support collaboration with other key organizations, including the National Kidney Foundation (NKF) and the International Society of Nephrology (ISN).
Tissue Donation Program
The PKD Foundation coordinates donations of discarded human PKD kidneys to research labs around the country. These kidneys are living tissue and come from patients who have had their kidneys removed (a procedure called a nephrectomy).
This tissue is highly valuable for researchers to learn about how PKD progresses through kidneys. The program also provides an important way for individuals to actively participate and support the basic science.
If you are scheduled to have a polycystic kidney(s) removed, we hope you’ll consider donating it to research.
The PKD Foundation will work with you to facilitate donating your discarded kidneys to research labs. If your kidney(s) will be removed and you’d like to donate them to PKD research, please contact the Integrated Research and Development Department at the PKD Foundation at 1-800-PKD-CURE or by email at least 10 business days prior to the kidney removal with the following information:
- Date of surgery
- Number of kidneys being donated
- Hospital name and location (address, city, state, zip)
- Name and contact information of the donor
- Name and contact information of the transplant coordinator
- Name of the surgeon
- How long the donor has been on dialysis (if applicable)
- Date of past transplant(s) (if applicable)
Frequently Asked Questions about Discarded Kidney Donation (link)
Clinical Trial Awareness Program (CTAP)
The PKD Foundation believes empowered patients are the key to accelerating the development of a robust clinical trial pipeline. Without patient participation in medical research, medical breakthroughs cannot happen. The Clinical Trial Awareness Program is focused on educating patients about current studies so they can make educated decisions about participating.
This program was launched in 2010. We are increasing awareness about trials by providing alerts to patients and physicians. These are called ACT (accelerating clinical trial) alerts.There are around 30,000 people who are on the email list to receive alerts about trials.
If you’d like to participate in a clinical trial to help science get closer to a treatment, you can find specific information about the trials here. To participate in clinical trial, you'll need to have a formal diagnosis of PKD.
You can also learn more about PKD Foundation's Accelerating Clinical Trials (ACT) program here.