Watch the Demystifying Research Webinar! July 21, 2015 by Kylie Karley Watch NKI and special guest speaker Dr. Jonathan Hogan from The University of Pennsylvania, as we demystify Nephrotic Syndrome research! Research is important to all members of the NS community- so why not learn about it! We talk about how research works, what is happening in the NS research world, and what patients and their families can do to help. Come with your research questions, and prepare to have them answered! CLICK HERE TO WATCH THE WEBINAR
Test The New ‘My Kidney Buddy’ Mobile App! June 15, 2015 by Kylie Karley Are you buried in paper from all your doctor visits? Ditch the binder- there’s an app for that! As part of the NephCure Kidney Network Patient Registry (www.nephcurekidneynetwork.org) we are developing a mobile app that makes keeping track of your paper medical records a thing of the past! My Kidney Buddy conveniently tracks crucial information such as new symptoms, current medications, and lab values and allows you to easily access them on your smartphone. Additional features include a graphing function so you can better track changes in your lab values over time. The My Kidney Buddy diary feature also allows you to track changes in your diet or if new symptoms occur. Coordinating your care can be complicated and it is important that you and your physician have all your important medical information available. By using My Kidney Buddy, you can be sure that your important health information is recorded and can be easily relayed to your physician… all without the hassle of carrying a heavy binder! We are looking for ‘beta testers’ to try this application and give us feedback on your experience prior to its release to the general public- and we need your help! Help us help you by testing My Kidney Buddy today! Email Chelsey Fix at cfix@nephcure.org for more information.
Jet Food Stores – #Selfies4NephCure Contest May 29, 2015 by Lauren Eva On May 5th, Jet Food Stores hosted their annual NephCure Golf Classic at Twin City Country Club in Sandersville, Georgia. Over the past 12 years, this event has generated more than $350,000 for the NephCure mission. This year’s golf outing saw the introduction of the #Selfies4NephCure contest. Participants took “selfies” of themselves at the event and posted them to their social media pages. Jet Foods and NephCure are hoping that this social media awareness campaign will continue long after the golf outing. Anyone can join the fun and help us spread awareness – so take a picture of yourself, and post it with the hashtag on your Facebook, Twitter and Instagram pages with #Selfies4NephCure. NephCure board member and Jet Food Store President Charles Turner is the driving force behind the event. This cause is important to his family and their goal is to support research to discover and make available better treatments for all families dealing with these kidney conditions. Interested in hosting your own community fundraiser? Whether you’re running in a marathon, giving up your birthday for charity, doing a tribute, starting a walk in your town, or anything else; contact the events team at events@Nephcure.org to raise money for NephCure and make a real-life impact.
Featured Clinical Trial- May Newsletter May 29, 2015 by Kylie Karley Featured Clinical Trial DUET: Sparsentan in FSGS Purpose The DUET trial is an interventional study that will determine how effective the drug Sparsentan is at reducing the urine protein / creatinine ratio over 8 weeks. Who is Eligible? Males and females between the ages of 8 and 75 years old with biopsy proven FSGS, or a proven genetic mutation transplant. Click HERE to see the other criteria that determine who is eligible to participate. How Can I Participate? There are currently over 30 study sites across the United States that are actively recruiting patients! To see that list, click HERE. Don’t see a location near you? No worries! The DUET trial will reimburse you for any travel costs to a study site. All study-related health exams and medications will be provided at no cost. Click HERE to learn more about participating in the DUET Study. Click on the Clinical Trials Finder Map below to see what’s happening near you!
Q&A With Dyan Bryson from Retrophin May 29, 2015 by Kylie Karley Recently, we got a chance to chat with Dyan Bryson, the Patient Advocacy Director at Retrophin, Inc. – the pharmaceutical company that is sponsoring the DUET study for FSGS patients. We took this opportunity to ask Dyan why clinical research – and the DUET study in particular – is important for Nephrotic Syndrome patients. Keep reading to see our Q&A session with Dyan! NephCure Kidney International: Why do you enjoy/what inspires you about working in the pharmaceutical industry? Dyan Bryson: I first came into this industry thinking I would stay 3 years, that was almost thirty years ago. I simply fell in love with the industry and its potential to help people. I came in rather naively, and at one point I became completely fed up with the lack of focus on patients. I am now back in love as the industry is finally moving towards fulfilling its potential to really help patients, not just support its brands. I love being in an industry where companies that have awakened to putting the patient at the center of its business are also profitable – doing well by doing good. These companies can be examples to others that this can be done. We have realized that we need to support people after they have filled our prescriptions; we have to develop strategies to support their caregivers; we have to support people by realizing that the therapeutic area our drug may be managing is just one of a whole list of things a patient, a person, has to manage in their lives. NKI: What makes participating in DUET a unique opportunity for the NS community? DB: Participating in a clinical trial, especially DUET, is an opportunity to contribute to the knowledge and health of the community. Of course, I am biased, but as a patient advocate I know that if we truly have a drug that can help patients with this rare condition – a condition that has no existing therapy – it can help so many people in the long run. Participation allows us to collect data that may help other people avoid the current long term outcomes of end stage renal disease and transplant. Although we as the drug maker will profit, so will the community through better quality of life. If you participate you can contribute to bettering the health of others. NKI: Why is it so important for rare disease patients and their families to care about research? DB: Many times it is the family of, or person who has, a rare disease that brings that disease to the attention of researchers. Many times if that interaction did not happen there would be no awareness of the disease and, hence, no research. Rare diseases are not like diabetes – researchers, drug companies understand the impact of diabetes. Payers understand the impact diabetes makes on their bottom line as they pay the costs of diabetes management. All stakeholders have clear incentives to manage the disease better. But for a disease where there is low awareness there is little understanding of the impact on a person’s health, hence little understanding of why to do the research. Patients with a rare disease, and their care partners, are the experts on that disease as they manage it 24/7. They are the ones that can drive the understanding of the disease and interest in doing research on that disease like no one else. I still remember the first time that, as a sales representative, my company brought a patient in to talk about the therapeutic area for which we were about to launch a drug. We all had studied and been tested for months to be certified, to confirm we knew the area well enough to talk about it with the physicians we called on. Now, finally, we were at the launch meeting to get our marching orders and celebrate the launch – fun and work at the same time. So, the patient gets on the stage, we had no idea what we are in for. The patient told their story. Soon we were ALL in tears. That one patient helped us understand why were really there. We understood their day-to-day and now we were on a mission to help. Twenty-five years later I can still see that patient on that stage; that experience informs me every single day. That is the impact of a patient telling their story, motivating and inspiring those who can do something about the disease take action. NKI: What is your suggestion for patients that want to learn more and be more involved in clinical research? DB: I know it can be daunting even to think about participating in a clinical trial. You think you are going to be a guinea pig, I understand and so do many others. There is a tidal wave of information out there that can help understand how trials work and help people to understand the process. The US government has even issued grants to researchers all over the country to figure out how to better inform the public about clinical trial participation (http://www.nih.gov/health/clinicaltrials/index.htm) Certainly on the NephCure site (https://nephcure.org/wp-content/uploads/2014/08/Clinical-Research-one-sheet-9162014.pdf) Also, several healthcare advocacy groups have sites that contain materials in several languages to discuss clinical trial involvement: National Minority Quality Forum “Are you in” Campaign – http://www.nih.gov/health/clinicaltrials/index.htm National Medical Association – Project IMPACT – http://www.impact.nmanet.org/about NKI: What do you want a participant to know before they decide to volunteer for a clinical trial? DB: The managers of clinical trials are usually very good about providing access to information about their trial. Scour the site provided, read the materials you are given before you make a decision. Know that the managers of that trial have had to put the trial protocol, all trial materials through a review of a professional board that understands the therapeutic area for which the trial was designed. This board is called an Institutional Review Board (IRB). The purpose of the IRB is to ensure that all human subject research be conducted in accordance with all federal, institutional, and ethical guidelines. This IRB oversees the entire trial, meets at points throughout the trial and makes independent assessments to ensure the safety and well-being of the study participants. In other words, the drug company or other researchers are running the trial with considerable oversight. The study participant is not on their own.
Debunking Clinical Trials Myths May 29, 2015 by Kylie Karley MYTH: Participating in a clinical trial will just make me a “human guinea pig” FACT: A common fear is that those giving you care in a clinical trial won’t treat you like a person or a patient. However, the opposite is true. Participating in a clinical trial gives you access to some of the best care available to patients. Also, strict ethical and regulatory guidelines are in place to ensure that each participant is treated fairly and respectfully, not to mention that all drugs in clinical trial phase go though a vigorous testing process before they are approved for use in a clinical trial. MYTH: I like my doctor, and if I participate in a trial, I won’t be able to see them anymore FACT: This is not true. Participants are encouraged to continue seeing their regular doctor in addition to any appointments they may have with the trial staff. A physician on the clinical trial staff does not replace your regular doctor. MYTH: If I participate in a clinical trial, I’ll have to go off of the medications that keep me in remission FACT: Each trial has a different protocol, or set of rules. Some trials have a protocol that allows participants to stay on current medicines. Or, if a trial asks participants to stop taking their current medications, participants are closely monitored and immediately given their original medicine and dosage if their symptoms worsen. The health of each participant is top priority for every clinical trial team. MYTH: There are no trials happening near me, therefore I can’t participate FACT: There are currently 17 clinical trials that are recruiting patients with Nephrotic Syndrome diseases at nearly 300 sites throughout the United States. If there is not a site close to you, many trials will pay for your travel (like the DUET study!). MYTH: If a clinical trial will help me, my doctor will tell me about it FACT: While we all love our doctors and respect their opinion, the truth is that they are extremely busy. We can’t expect them to save lives everyday and remember every clinical trial going on. Sometimes doctors just don’t know about clinical trials, or are unsure if their patients are interested. The best approach to participating in research is to do some research yourself and then take questions to them. If your doctor is unaware of a trial, they can find out more information and help you make a decision. However, please remember that only YOU can make the decision to advance research to help find better treatment options by participating in a clinical trial. Click HERE to learn more about clinical drug trials
2014 Research In Review February 20, 2015 by Kylie Karley What a Year We Had in 2014! Thank you to all those who collaborate with NephCure Kidney International (NKI) to advance its mission to support research leading to better treatments and cures for those suffering with FSGS and other Nephrotic Syndrome diseases. 2014 saw several major successes in the research world that move us closer to achieving our goals. Below is a summary of NKI’s 2014 research-related accomplishments. NEPHCURE KIDNEY NETWORK PATIENT REGISTRY Hundreds of people with primary FSGS and related glomerular diseases enrolled in the newly launched NephCure Kidney Network Patient Registry. These patients and care givers of patients are at the forefront of a movement called “patient powered research”. By sharing and comparing data contributed by patients about their health and quality of life, we can accelerate the development of new treatments, including new drugs. It is important for people with a rare disease to STAND UP & BE COUNTED to ensure research focuses on the issues of greatest importance to patients and their families. To learn more or enroll, click HERE. RESEARCH GRANTS In 2014, the NKI grant program committed over $2.6 MM in support for scientific investigations that advance knowledge about the cause of NS disease, evaluate possible drug targets and help physician/scientists track the natural history of these rare diseases. Click HERE for more information about grants awarded to investigators at Washington University Medical Center, Boston Children’s Hospital and Rush University. For more information about the research consortia we support, click HERE for the Nephrotic Syndrome Study Network and HERE to learn more about CureGN. CLINICAL TRIALS Patient participation in clinical research is essential to advance new treatments. In 2014, NKI launched several new programs to educate patients and increase awareness of opportunities to participate in research. The NEW Clinical Trial Finder website lists 16 studies actively recruiting participants in the US and Canada and another 18 around the world. We launched a Clinical Trial Ambassadors Program to recruit and train volunteers to help educate patients about the benefits (and risks) to participate in clinical trials and educate the medical community about the need for more research into treatments for NS. We posted, tweeted, emailed and sent letters about NEW FSGS drug trials. Our activities have raised awareness for potential participants and increased the interest of biopharmaceutical companies in working with NKI to advance new drug trials. CATALYZE, COLLABORATE AND CONVENE NKI is dedicated to being a catalyst along the research continuum: from support for basic science research to working with the biopharmaceutical industry and the FDA to understand how we might increase the numbers of new drugs moving into the drug approval pipeline and, finally, how to rapidly integrate new treatments into health care practice. 2014 was a busy year as we joined forces with our partners in healthcare and research to promote better treatments and faster cures. The advocacy work of NKI volunteers, staff and our professional partners in Washington, D.C. was instrumental in significant NEW FUNDING for FSGS and NS research in 2014. The National Center for Advancing Translational Sciences (NCATS) at the National Institute of Health (NIH) awarded $10 MM in new funding to continue the Nephrotic Syndrome Study Network (NEPTUNE), a consortium of research centers in the US and Canada collecting and analyzing data about patients newly diagnosed with primary NS diseases. NKI was a principal funder of the first phase of this study and continues to support and participate in its expansion. A second institute in NIH, the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) awarded a new $20 MM, 5- year grant to follow patients with FSGS, Minimal Change Disease, Membranous Nephropathy and IgA Nephropathy. NKI was instrumental in collaborating with NIH to get this funding. Click HERE. For the first time, FSGS is listed as a condition eligible to receive research grants through the Department of Defenses’ Peer-Reviewed Medical Research Program. Because FSGS is listed, FSGS researchers have an opportunity to compete for grants from a NEW funding source. The total amount in the DoD’s program is $247.5 Million! NKI is a leader in assembling, facilitating and promoting professional meetings that bring together partners from industry and the academic world along with patients and their advocates to promote NS health research. In 2014, NKI: Organized the 10th International Podocyte Conference in Freiburg, Germany. This conference brings together researchers from a variety of scientific disciplines and from around the world to promote interactions and new collaborations and to investigate emerging directions in podocyte and glomerular biology research. Convened the PRO in Glomerular Disease Scientific Conference with representatives from academic medical centers, the biopharmaceutical industry, the NIH and the FDA to pursue the development of Patient Reporting Outcome (PRO) instruments to advance new drug trials for FSGS and NS. (A PRO is any report of the status of a patient’s health condition that comes directly from the patient.) An FDA-accepted PRO instrument can accelerate the number of applications to the FDA for new drug trials. NKI continues to lead the development of a PRO development consortium. Convened the third NephCure Research Accelerator Meeting that brought together physicians and scientists with representatives from more than a half dozen biopharmaceutical companies with an interest in drug development for kidney disease. Research Accelerator Meetings were initiated by NephCure to engage these two scientific groups in an exchange of knowledge that will accelerate translation of basic science discoveries to new treatments for people with NS diseases. Presented a session on the NephCure Kidney Network and patient-powered research to the US Summit of Kidney Organizations hosted by the America Society of Nephrologists at its 2014 conference in Philadelphia. NKI continues to be a leader in bringing the voice of patients to the forefront of advocacy for new treatments for chronic kidney diseases such as FSGS, Minimal Change Disease and Membranous Nephropathy.
Last Week to Enter! February 9, 2015 by Lauren Eva Cookin’ for Kidneys Recipe Contest NephCure is thrilled to announce the release of our first-ever kidney-friendly cookbook, released in partnership with A.I. DuPont Hospital. Recipes included have been created and tested by certified nutritionists with the needs of kidney patients in mind. In recognition of the AMAZING NKI community, the cookbook will also feature recipes created by patients and their families who are living with Nephrotic Syndrome and FSGS. Cookbook recipes will be selected through an online contest in which patients and their families submit their favorite healthy recipes. Submissions are reviewed and winners are selected by a panel of nutritionists, physicians, patients and family members. SEE GUIDELINES BELOW. Several recipes will be included for breakfast, lunch, dinner and snack categories. In addition, nutrition facts and tips for making healthy eating both convenient and affordable will be given. Forwards by Nemours Nephrology, NKI and A.I. DuPont Hospital will be included as well. Contest Guidelines SUBMISSION DEADLINE: Feb 15 (midnight) To qualify for this contest, participants must: Submit original recipes Submit recipes that are kidney friendly and/or healthy Fill out the submission form provided online Enter submissions by midnight on February 15, 2015 Participants should keep in mind the following: Recipes will be judged based on nutritional value, taste, ease of preparation, etc. No more than one recipe submission per category Upon completing the form and submitting a recipe, participants agree their recipe can be published in the cookbook Winners will receive the following: One free cookbook A profile/about me of themselves in the cookbook SUBMIT YOUR RECIPE HERE The fine print Sponsor: NephCure Kidney International (“Sponsor”): 150 Warner Road., King of Prussia, PA. To enter: Complete the entry form and follow on-screen instructions to register and submit your original kidney-friendly recipe, including step by step instructions, ingredients, prep instruction, and cook time (“Creation”), recipe title and an optional photo of your chocolate recipe (Creation, recipe title and optional photo submitted together shall be deemed an “Entry”). All entrants must have a valid email address and complete the entry form in full. In case of dispute as to identity of the winner who enters, Entry will be declared made by the registered user (meeting eligibility criteria) of the e-mail account, and if a prize is won, it will be awarded to that registered user. Contest ends and entries must be received by 11:59 p.m. EST, 2/14/15. Limit one Entry per person per recipe category during the Entry Period. Entry must meet all of the following requirements or it will be disqualified. Proof of Entry submission does not equate to proof of receipt. Entry must be completely edible: Non-edible items — such as craft sticks, doilies, etc. — may be used as an element of the Entry, provided any such elements that come in contact with the food be of food grade, and all such elements can be easily removed prior to consumption, and do not affect the consumption or the safety of the Creation. The optional photo should be of the completed Creation only (no people, animals or other items should be included in the photograph). Optional photo must be in a png, jpeg or gif file format. Optional photo must be in color. Creation must include ingredients, recipe prep instructions, cook time and temperature. Entry must be original and created by the entrant, unpublished, the sole property of the submitter, and not previously submitted in any other contest. Entry must not violate any 3rd party rights including, but not limited to copyrights, trademark or right of publicity. Entrant agrees and understands that Sponsor may edit or recreate their Entry in any way, at its sole discretion, including, without limitation, substitutions of products named in the entry. Entry must be in English. Ownership and Use of Entry: By submitting an Entry, you (your parent/legal guardian if you are deemed a resident of a jurisdiction that deems you to be a minor) agree that your Entry, including your Creation, recipe, and photograph, and all rights of ownership and publication of same will become the property of Sponsor and will not be returned. In addition, by submitting an Entry, you (your parent/legal guardian if you are deemed a resident of a jurisdiction that deems you to be a minor) agree that NKI shall have the full right to use your name and Entry, commercially or non-commercially, both in a perpetual and worldwide manner, without compensation or notice, unless prohibited. This includes possible use of your name and Entry – whether it becomes a winner or not – in NKI advertising or promotions, including but not limited to print and Internet (may include not only NKI sites but other Internet sites and social networking sites, such as Facebook, Twitter or Pinterest, at the sole discretion of NKI). Entrant agrees and understands that Sponsor may edit or recreate their Entry in any way, at its sole discretion. Voting and Judging: Following the close of the contest, beginning one (1) month on or about 2/15/15 to 3/15/15, A group of qualified judges will vote for their favorite entries using the criteria set forth below. At the conclusion of the contest period, Judges will select 2 winning recipes per category based on the following criteria: Taste Nutritional Value Creativity Ease of Preparation Visual Appeal On or about 4/1/15, judges will announce one (2) Prize Winners per recipe category based on the judging criteria. All decisions of the judges are final and shall be at their sole discretion. Winners will be notified by e-mail on or about 4/5/15. If an entrant changes his/her email address after he/she enters the Contest, it is his/her sole responsibility to notify the Sponsor by (postal) mailing notice to the address listed above to be received by the end of the Contest. Winner and winning recipe will be posted on www.Facebook.com/NephCureFoundation other outlets at its sole discretion. Prizes: All winning entries will be included in the to-be published Kidney-Friendly Cookbook, receive a copy of the Kidney Friendly Cookbook (scheduled to be published early Summer 2015 and have the opportunity to be featured in the cookbook. Total ARV for all prizes is $9.95. Where applicable, no cash substitutions for prizes will be permitted and prizes are non-refundable and non-transferable except at sole discretion of Sponsor. NKI reserves the right to substitute prize of equal or greater value at its sole discretion. Federal, state and local taxes are the sole responsibility of the winners. Eligibility: Contest open to legal residents internationally, 12 years or older, except employees and the families (parents, children, siblings, spouses) of NKI, Facebook, their affiliates, subsidiaries, promotion and advertising agencies. NKI, Facebook, their affiliated companies, subsidiaries, promotion and advertising agencies, representatives, agents or employees are not responsible for, and shall be indemnified by, the prize winners against any claims, injuries, losses or damages of any kind resulting from acceptance, use, misuse, possession, or loss of the prizes. Subject to all federal, state and local laws. Void where prohibited or restricted by law. General: By entering the Contest, entrants (their parents/legal guardians if deemed residents of a jurisdiction that deem them to be minors) accept and agree to these rules and the decision of the judges, which shall be final. Potential winners (his/her parent/legal guardian if deemed a resident of a jurisdiction that deems him/her to be a minor) will be required to sign a Winner’s Agreement and Release and return it by date specified in notification letter or prize will be forfeited in its entirety and another potential winner may be selected. Potential. Release Of Liability & Publicity: All winners (their parents/legal guardians if deemed residents of a jurisdiction that deem them to be minors) consent to the use of their names, photographs or likenesses and limited biographical information (city, state) for publicity or advertising purposes without further notice or compensation, where permitted by law. All entrants release Sponsor, and each of its parents, affiliates, subsidiaries, officers, directors, shareholders, agents, employees, and all others associated with the development and execution of this Contest, from any and all liability with respect to, or in any way arising from, this Contest and/or acceptance or use of the prize, including liability for personal injury, damages, death or monetary loss. Sponsor assumes no responsibility for any injury, loss or damage to entrants’ or to any other person’s computer relating to, or resulting from, entering or downloading any information or software in connection with this contest. Sponsor and its agents are not responsible for technical, hardware, software or telephone malfunctions of any kind, lost or unavailable network connections, or failed, incorrect, incomplete, inaccurate, garbled or delayed electronic communications caused by the user, or by any of the equipment or programming associated with or utilized in this Contest, or by any human or other errors that may occur in connection with this Contest. Other Conditions: Contest Parties are not responsible for: (i) entry submissions or other documentation received after the deadline, or that is lost, stolen, illegible, misdirected, incomplete, or damaged; (ii) human error; (iii) incomplete transmission defaults or faulty network connections, telephone, computer server/system/software failure of any kind; (iv) delayed, garbled or corrupted data; and/or (v) any problems or technical malfunction of any telephone network or lines, computer on-line systems, servers, or providers, computer equipment, software, failure of any e-mail or players on account of technical problems or traffic congestion on the Internet or at any website, or any combination thereof, including any injury or damage to Entrant’s or any other person’s computer related to or resulting from participation in, uploading or downloading any materials for this Contest. Winners List: Winners will be posted on www.facebook.com/NephCure Kidney International on or about 4/1/15.
Become an Advocate This March January 29, 2015 by Lauren Eva Breaking News! NephCure will be accompanying patients and their families to Washington, D.C. on March 11-12 (World Kidney Day!) to speak directly with your legislators in Congress and ask for more government funding for FSGS and NS research. Would YOU like to STAND UP & BE COUNTED in Washington, DC.??As one of the millions of Americans affected by chronic kidney disease, your voice and your story matters. Please email jmartin@nephcure.org to express your interest and get on our list so we can set up meetings with your legislator! TO REGISTER: http://support.nephcure.org/advocacyday
Community Cafe Kick-off! January 21, 2015 by Kylie Karley NKI is kicking off its Community Café tour! Visiting cities like Stamford, CT, Birmingham, AL, and Miami, FL., there is sure to be a café near you. Click here to see when and where these FREE patient education seminars are happening. Don’t miss this chance to get up close and personal with leading experts in the field while enjoying kidney-healthy food. REGISTER for Stamford Cafe on Feb. 4th REGISTER for Birmingham Cafe on Feb 28th REGISTER for Miami Cafe on March 7th