Annual Report

Advancing Research

Our focus is Accelerating Treatments to Patients (ATP), an initiative dedicated to finding treatments for PKD as soon as possible.

In the fiscal year ending in June 2013,
$1.5 MILLION was invested in the ATP
initiative, including the highlights below.

Choose an ATP program below to learn more about the PKD Foundation's research efforts.

Tissue Donation

Coordinating donations of discarded PKD kidneys to research labs, allowing individuals to actively participate and support basic science.

Research Grants

Supporting services and facilities for PKD scientists so that Foundation dollars can be leveraged across the PKD research community. Supports discoveries in basic PKD science.

Core Labs

Providing grants to support facilities or services to PKD scientists at either no cost or a greatly discounted cost.

  • PKD Electron Microscopy Core for PKD Research
  • ADPKD Mutation Database (PKDB) 
  • PKD Research Biomaterials and Cellular Models Core

Scientific Meetings

Funding PKD-related meetings that bring experts together and provide continuing education for health professionals.

Drug Repurposing

Finding potential drug candidates or new uses for existing drugs to treat PKD.

PKDOC (PKD Outcomes Consortium)

Working with the Food and Drug Administration (FDA) for regulatory approval to use total kidney volume (TKV) as an outcome measure in PKD drug trials.

CTAP (Clinical Trials Awareness Program)

Supporting clinical trials by raising awareness through PKD Foundation patients, families and health professionals.



Tissue Donation Program17 potential kidney donations

We invested $10,000 to coordinate donation of discarded human PKD kidneys to research labs around the country. The tissue is highly valuable for researchers to learn about how PKD progresses through the kidneys. The program also provides an important way for individuals to participate in basic science and feel empowered by directly supporting research.

Last year, we contacted researchers on behalf of patients 17 times for potential kidney donations.

If you are interested in donating your kidney, please visit our Tissue Donation page on pkdcure.org.


Research Grant Funding

In June, we released a Request for Application (RFA) for research grant proposals. Therapy development was specified as the preferred focus, but basic science proposals were also accepted. Grants awarded will be announced in early 2014. The Foundation anticipates that 12, two-year grants will be awarded for $80,000 per year (per grant).


Core Labs

We invested $180,000 to provide grants to support facilities or services to PKD scientists at either no cost or a greatly discounted cost. In the fiscal year 2013, we went from funding two core labs to three.

$180,000

 

PKD Electron Microscopy Core for PKD Research led by Vincent Gattone, Ph.D., Indiana University School of Medicine, Electron Microscopy Center

ADPKD Mutation Database (PKDB) led by Peter Harris, Ph.D., Mayo Clinic

PKD Research Biomaterials and Cellular Models Core led by Darren Wallace, Ph.D., University of Kansas Medical Center


Scientific Meetings5 meetings, $7,000

We invested $7,000 to fund PKD-relevant scientific and clinical meetings that bring together PKD scientists from around the world to discuss current findings and encourage research collaboration. We have also funded medical education programs at national clinical meetings to educate health professionals in the basic science, diagnosis, disease management and potential treatments for PKD.

In the last year, the Foundation funded and had a presence at the following conferences:

  • Federation of American Societies for Experimental Biology Summer Research Conference
  • National Kidney Foundation Spring 2013 Clinical Meetings
  • ARPKD Clinical Consensus Meeting

Drug Repurposing4 drugs, $600,000 invested

We invested $600,000 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.

Four drug candidates completed testing during the fiscal year. MSDC-0160, one of the candidates, was shown to slow the growth of cysts in an animal model.


PKD Outcomes Consortium Project (PKDOC)

We invested $698,000 in the PKD Outcomes Consortium Project (PKDOC). PKDOC is critical to the PKD Foundation because FDA approval of total kidney volume (TKV) as an indicator of disease progression in PKD provides a clear regulatory pathway for future clinical trial development.

Total Kidney Volume (TKV)Significant progress was made during the year as discussions occurred between the FDA and PKDOC regarding the creation of a briefing book (preliminary submission package) and the submission process for TKV. There was an in-person meeting held in November 2012 between the FDA and PKDOC to discuss the information in the briefing book. In June 2013, the updated PKDOC briefing book was submitted to the FDA for review.

The briefing book prepared for the FDA was modified for submission to the European Medicines Agency (EMA) in March 2013. It is also currently under review. The EMA is the European version of the FDA in the United States.

PKDOC has been solely funded by PKD Foundation fundraising efforts, an investment of more than $3 million, since 2009.


Clinical Trial Awareness Program (CTAP)

We invested $25,000 to inform patients about current clinical studies so they can make educated decisions about participating. We send emails, Accelerated Clinical Trials (ACT) Alerts, about studies that are being conducted in their area.

2 clinical trials, 36,000 emailsIn the last year, we sent ACT Alerts focused on two clinical trials that were enrolling ADPKD patients. Thirty-six thousand emails were sent six times over a three-month period, with an average of 14 percent of recipients opening the emails.

The organization conducting one of the trials implemented a call center questionnaire to gain insights from patients, and nearly 80 percent of the 416 callers learned about the trial from our ACT Alerts.

If you would like to sign up to receive ACT Alerts, click here.


TolvaptanOtsuka Logo

A major announcement was made by Otsuka Pharmaceutical Co. Ltd. in November 2012 at the American Society of Nephrology (ASN) meeting regarding the results of the Tolvaptan Efficacy and Safety in Management of Autosomal Dominant Polycystic Kidney Disease Outcomes (TEMPO) Trial.

In April, Otsuka Pharmaceutical Co. Ltd. announced that the U.S. Food and Drug Administration (FDA) accepted for priority review the company’s new drug application (NDA) for the potential use of tolvaptan to treat ADPKD.

It was the first time that a drug specifically targeted to alter ADPKD progression in humans was shown to be of benefit, which was an exciting milestone for PKD patients and the Foundation. The concept of using vasopressin blockade as a way of slowing ADPKD progression was originally developed by Vincent Gattone, Ph.D., whose research has been in part supported by the PKD Foundation.

*In late August Otsuka Pharmaceutical Development & Commercialization, Inc. received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding tolvaptan to treat adult patients with ADPKD. The FDA issues CRLs to convey that their initial review of  an NDA is complete and they cannot approve it in its present form. After a follow-up meeting between Otsuka and the FDA, next steps are being determined by Otsuka.


Kaplan Award

Dr. Dorien PetersDorien Peters, Ph.D., accepts the Lillian Jean Kaplan Prize.

Dr. Vincent GattoneVincent Gattone, Ph.D., was one of two Lillian Jean Kaplan Prize winners.

In June, Vincent Gattone, Ph.D., and Dorien Peters, Ph.D., were awarded the Lillian Jean Kaplan International Prize for Advancement in the Understanding of Polycystic Kidney Disease (PKD). The award is a partnership between the PKD Foundation and International Society of Nephrology (ISN) and recognizes a medical professional or researcher exhibiting excellence and leadership in PKD research and whose work demonstrates tangible achievement toward improving knowledge and treatment of PKD. The Kaplan award was established in 2002 by the PKD Foundation and the ISN through the generosity of Thomas Kaplan in memory of his mother, Lillian Jean Kaplan, who had PKD and passed away in 2002.