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.
Los Angeles Walk a Huge Success! February 10, 2015 by Lauren Eva Over 400 SoCal residents came out on a picture perfect January day – the type you only see in Southern California in January – to celebrate the work of NephCure Kidney International at the 6th Annual NKI Los Angeles Walk, Brunch & Learn. This incredible event was organized and chaired by Los Angeles Attorney Sarretta McDonough of Gibson, Dunn & Crutcher LLP and her wonderful volunteer committee that included Janet Hasson, Genneia James, Peter Daenzer and Rich Griswold. As with the past five years, this event was a true labor of love for this group and the dozens of volunteers who also dedicated their personal time on a Sunday to support NKI. With everything from an incredible Kid’s Zone with face painting, games, a photo booth and even a visit from a bonafide “Minion” (courtesy of NBC/Universal), to a delicious brunch and post-Walk educational Lunch and Learn presentation by nephrologists from three of the largest hospitals in the Los Angeles area, this event offered something for the whole family. Beyond that, the warmth and camaraderie created a welcoming atmosphere for everyone to feel that they were part of a strong, incredible community effort to fight these conditions and a welcome reprieve from the isolation that living with Nephrotic Syndrome/FSGS can often bring. The Annenberg Beach House in Santa Monica provided the perfect venue for the Walk, family activities, lively silent auction, and of course the important Lunch and Learn with Drs. Elaine Kamil (Cedars Sinai), Kevin Lemley (USC/Children’s Hospital LA) and Sergio Infante (Loma Linda), who generously extended the question and answer session until every single person had been heard. With well over $76,000 raised and more funds coming in every day, this event was truly a success for NephCure’s important mission to fund new treatments and find the cure!
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.
Precision Medicine Initiative February 4, 2015 by Kylie Karley President Barack Obama announced recently the Precision Medicine Initiative to be included in the 2016 federal budget. This is a bold new enterprise to revolutionize medicine and generate the scientific evidence needed to move more personalize medicine into every day clinical practice. Precision Medicine can be defined as getting the right treatment, at the right time – every time – to the right patient. It is about tailoring treatments “for a person” based on how they experience a medical condition rather than focusing on treating “the disease.” The President’s budget includes support for the FDA to develop new approaches for evaluating next generation genetic screening and the NIH to create research that embraces volunteer participation. There is significant emphasis in this Initiative on engaging people experiencing a health problem in the development of targeted treatments for their condition. Patients should be active partners in the search for new and better therapies and not merely passive recipients of investigational treatments. Dr. Francis Collins, Director of the National Institutes of Health, in a small luncheon following President Obama speech, spoke with great enthusiasm of the need to gain better insights into the biology of all diseases to make a difference for the millions of Americans who suffer from them. He was clear that this initiative must be led by us, the people. He stated, “The word ‘patients’ is not the right one. This is about participants and really about partners.” Dr. Kathy Hudson, Deputy Director for Science, Outreach and Policy at NIH commented, “The Initiative will set the foundation for new ways of engaging research participants, sharing health data and information, and employing technology advances to mine the information for comprehensive results.” NephCure Kidney International is very excited to already be a part of this movement as the NephCure Kidney Network (Patient Registry) is the ONLY kidney disease Patient Powered Research Network in another key government initiative — the Patient-Centered Outcomes Research Network (PCORnet). The NephCure Kidney Network is poised to be at the forefront of integrating new opportunities from the President’s Initiative with new technology and “big data” solutions that will propel new therapies for our patient community. STAND UP & BE COUNTED. Visit https://www.nephcurekidneynetwork.org/ to learn more about participating in the NephCure Kidney Network (Patient Registry) so your voice can be heard and to share your experience to accelerate new therapies and, one day, a cure for FSGS and Nephrotic Syndrome. Contact research@nephcure.org to connect with a member of NephCure’s research team.