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.
NEW Funding Source for FSGS January 9, 2015 by Kylie Karley President Obama signed the appropriations package to fund federal agencies through fiscal year 2015 (ending September 30, 2015). This includes allocations for NIH and other health care funding agencies such as the Department of Defense (DoD), which oversees the Peer-Reviewed Medical Research Program (PRMRP). For the first time, FSGS is listed as a condition eligible to study in the PRMRP. Because FSGS is listed, FSGS researchers have an opportunity to compete for grants from a NEW funding source. This is HUGE. This opportunity is a direct result of NephCure’s professional and volunteer advocacy work in Washington, D.C. and the efforts of our staff to work collaboratively with scientific partners at NIH and the VA to highlight the benefits of studying this disease through the DoD program. The total amount in the PRMRP program is $247.5 MILLION! UPDATE: The Program Announcement was released April 17 for the 2015 cycle. There are five funding mechanisms available. Click here for details.
NIDDK Tenure Track Position Opportunity for Nephrologists December 9, 2014 by Kylie Karley NephCure Kidney International is pleased to announce the development of a new nephrologist position at NIDDK. A tenure track position is available for a nephrologist to establish an independent research program in the Kidney Disease Section, Kidney Diseases Branch, NIDDK, NIH. The search is particularly focused on individuals studying glomerular physiology or glomerular disease. Research in this section focuses on mechanisms and therapy of glomerular disease under the leadership of Jeffrey Kopp, MD. Position Requirements MD degree Demonstrated expertise and a record of achievement in basic or clinical research Current board certification in Nephrology Excellent clinical nephrology skills Have or be eligible to obtain a current US medical license. The position will be supported with independent resources commensurate with experience and programmatic needs, including positions for clinical staff, pre-doctoral and post-doctoral fellows, and a budget for consumables and equipment. Interested applicants should submit a single PDF document containing a Curriculum Vitae, a list of publications, a summary of research accomplishments, a plan for future research including a core research question, the names and contact information of three people who are submitting letters of reference, and copies of no more than 3 publications. All materials should be emailed to: Dr. Mark Knepper, Chair, Kidney Disease Branch Search Committee c/o Lisa Swearinger SwearingerL@mail.nih.gov
Partnering For Cures Conference Stimulates Innovation December 9, 2014 by Kylie Karley In mid-November, Mark Stone, NephCure Kidney International Acting CEO, attended the sixth annual Partnering For Cures Conference in New York City. The Partnering For Cures Conference is a patient-focused event that brings together leaders from all sectors of medical research to foster collaboration – collaboration that will lead to medical discoveries, and, ultimately, treatments and cures. Partnering For Cures is hosted by FasterCures, a Washington, DC-based center of the Milken Institute. FasterCures hosts the conference each year to convene groups like NephCure to identify partnership opportunities and advance outcomes-driven medical research. At the core of Partnering For Cures is the idea that the cultivation of cross-sector relationships will lead to collaborative efforts necessary for the development of new therapies. This year’s conference focused on the importance of patient-reported data and how determined patients are improving and accelerating the search for cures. “Patient empowerment is at the heart of what we do at NephCure,” says Mark Stone. “Conferences like Partnering For Cures help groups like NephCure influence research innovations and remind us of the most important aspect of our research – our patients.”
Seeking Volunteers for NEW Clinical Trials Ambassador Program November 5, 2014 by Kylie Karley Have you ever participated in a clinical research study? Are you willing to share your experience with others? If so, then you would make a fantastic addition to our newest initiative to find better treatments and a cure for Nephrotic Syndrome: the Clinical Trials Ambassador Program! NKI is seeking volunteers from across the country to act as Ambassadors to other patients and the medical community. Ambassadors will spread knowledge and information about the importance of clinical research in the search for better treatment options and a cure. Ambassadors can expect to spend between 5 and 10 hours a month volunteering in various ways that will help spread awareness and knowledge about clinical research. If you are interested in learning more about the Clinical Trials Ambassador Program, contact Chelsey Fix at cfix@nephcure.org.
NEW!! Clinical Studies Map! October 14, 2014 by Kylie Karley NephCure Kidney International Introduces Clinical Research Map October 14, 2014 We are excited to introduce a new user-friendly, interactive clinical research map. This map includes current clinical research sites and studies available to patients affected by FSGS and other Nephrotic Syndrome diseases. The map, which can be found here, will help patients sort through studies available to them. Categories include studies specific to adults, pediatrics, Nephrotic Syndrome, and FSGS, to name a few. “Patient participation is key to advancing research and treatment options available to patients,” says Marilyn Hailperin, the National Director of Research at NephCure. “This site map allows patients to view studies underway in their area.” This map is another way NephCure is living out our mission to support research seeking the causes of Nephrotic Syndrome diseases, to improve current treatments and, ultimately, to find a cure. Here at NephCure, we value the supportive role we play for our patients. Spreading the word about clinical research to our patients is a crucial aspect of this role, because, after all, patients power research! Link to the map: here. For more information about the risks and benefits of participating in clinical research, click here.
NephCure Kidney International Announces NKN Steering Committee Co-Chairs October 2, 2014 by Kylie Karley We are pleased to share that Kathleen Broderick and Randall Snyder were recommended and have accepted their nominations as Steering Committee Co-Chairs for the NephCure Kidney Network (NKN). Kathleen was considered a strong candidate due to her dual experience as a patient caregiver as well as a patient advocacy representative. Similarly, Randy’s dual perspective as a patient caregiver and clinical provider give him unique insight relevant to the NKN. Kathleen is a member of the NephCure Board of Directors and Research Committee and an editor at DeGruyter Publishing, a Science, Technology, and Medicine (STM) publisher of professional books and scholarly journals in Boston, Massachusetts. Additionally, Kathleen cares for her 16-year-old son, who was diagnosed with Nephrotic Syndrome at 20 months. Randy is an interventional radiologist with his master’s in molecular biology living in Medford, New Jersey, who cares for his son who was diagnosed with focal segmental glomerulosclerosis. Kathleen and Randy will be facilitating Steering Committee calls and communication moving forward. Please join us in welcoming Kathleen and Randy as the inaugural NKN Steering Committee Co-Chairs!
Claritas Genomics Launches Convenient Genetic Testing Option For Nephrotic Syndrome Patients September 24, 2014 by Lauren Eva On September 11, 2014, Claritas Genomics in Cambridge, MA, announced the creation of its newest genetic screening panel, which tests gene variants commonly associated with steroid resistant Nephrotic Syndrome in children. The test uses next-generation gene sequencing techniques to detect mutations in the 28 most common gene variants associated with Nephrotic Syndrome, making it the largest genetic panel for any pediatric kidney disease. Typical candidates for this genetic test include: 1) pediatric patients less than 12 months old; 2) pediatric patients that do not respond to steroid therapy; 3) patients with a family history of Nephrotic Syndrome; 4) pediatric patients with features of FSGS; and 5) patients who have congenital malformations associated with Nephrotic Syndrome. In a time where healthcare is becoming more patient centric, as opposed to disease centric, genetic screening may lead to more ‘personalized’ treatment based on the characteristics of a patient’s disease profile. Information about genetic underpinnings of Nephrotic Syndrome may inform what drugs are used for initial treatment, provide improved information for patients and families about the likely clinical course of disease, could improve the selection of appropriate transplant donors and provide opportunities for genetic counseling for other family members. “This is the start of an exciting new era in understanding genetic factors that play a role in the onset and progression of at least some cases of Nephrotic Syndrome. NephCure Kidney International is proud to have supported the research at Boston Children’s Hospital that led to the development of this commercial application,” says Marilyn Hailperin, National Director of Research for NephCure. “Clear guidelines for when to seek genetic testing and the impact on patient treatment still need to be developed, but the availability of tests such as ClariFocus will help to advance scientific knowledge that will lead to better therapies.” Patients and their family members should talk with their nephrologist about this new test. It is only available with a physician order at this time.
NephCure in the News September 22, 2014 by Lauren Eva PatientCrossroads Launches New Patient Registries The new rare-disease registries arose from the NIH Global Rare Diseases Registry (GRDR) pilot program San Mateo, Calif. (PRWEB) September 04, 2014 PatientCrossroads announces the launch of five new patient registries on behalf of leading rare disease advocacy organizations. The new registries are sponsored by organizations originally selected by the NIH to participate in its Global Rare Diseases Registry and Repository (GRDR) pilot program. Five New Patient Registries Launch Through the PatientCrossroads Program include: The Barth Syndrome Foundation, Foundation for Sarcoidosis Research, Lymphangiomatosis and Gorhams Disease Alliance, NephCure Kidney Network and PSC (Primary Sclerosing Cholangitis) Partners Seeking a Cure registries have been launched using the PatientCrossroads registry platform. Read more>> ‘A rare young lady’: Despite illness, girl handles life like a champ By Isaac Groves / Times-News Published: Wednesday, September 10, 2014 at 05:31 PM. GRAHAM — At age 6, Brandi Chrisp is a lively, sweet, smart, talkative, bold girl without a shy bone in her body and, honestly, one of the cutest kids you will ever meet. “I really like to play and sing,” she said. “And my favorite thing is going out to eat with my family.” She has also been hospitalized several times in the past year with a rare kidney problem, and she and her family are raising money to fund research to help people like her. “I have nephrotic syndrome,” she said. Read more>> Iowa-ISU backers running for bragging rights and charity Posted Online: Sept. 10, 2014, 9:39 pm Roughly 100 participants – most as part of relay teams – will run a 125-mile rout from Ames to Iowa City beginning today, September 10, and ending on tomorrow. Along with the bragging rights aspect, the event is also a fundraiser that benefits NephCure Kidney International. Read more>> ‘We Have a Long Road Ahead of Us’ 10-Year-Old Union Boy Lives With Rare Kidney Disease Posted: Wednesday, September 17, 2014 6:32 pm By Karen Cernich, Feature Writer Sitting on the living room floor of his Union home building a toy car out of Legos, Bradley Scott looks like an average 10-year-old, but to doctors treating him for the rare kidney disease FSGS, he’s something of a medical mystery. “He’s been rewriting the medical books since he was a baby,” Bradley’s mom, Amanda Scott, remarked. “I think every specialist that we’ve seen says he’s rewriting the medical books.” Read more>> Northern VA Home Improvement Company Supports Fairfax County Public Schools PRWeb | September 20, 2014 Moss Building and Design, a family-owned remodeling company in Fairfax County, is always looking for ways to give back to the community they work and live in. Giving back to the local community is an important goal of the remodeling company whose efforts include school sponsorships as one of the many ways they give back. Moss’ community involvement also includes sponsoring local golf tournaments, supporting the local NephCure foundation, building the haunted house structure for Bradley Farm Haunted House, and even rebuilding the roof of the dug-outs at Chantilly Park for Chantilly Little League. Read more>>
Rival Game Relay Raises Money and Awareness for NephCure September 19, 2014 by Lauren Eva RGR Organizers Kate and Mike Maurer Mike and Kate Maurer are runners who came up with an inspired idea and organized a great event for a great cause, but they’re more than that: they’re parents to a wonderful little boy named Alex. Five-year-old Alex is active and energetic, he loves climbing and running…when he’s feeling well enough to play like other five year olds. Alex has Nephrotic Syndrome, a condition neither of his parents had even heard of before he was diagnosed. Ultra-runner Troy Thompson Mike and Kate know the trials of managing and living with NS, and like tens of thousands of others rely on breakthroughs in medical research to find better treatments and a cure for their son. They decided they needed to do something. As avid runners having completed numerous races of distances ranging from 5k to marathon, and team relays of 212 miles, they decided to blend their love of running with their desire to fundraise and created a 124-mile race called the Rival Game Relay. Capitalizing on a centuries-old Iowa rival between the University of Iowa Hawkeyes and the Iowa State Cyclones, they timed their race to coincide with the annual Iowa/ISU football game. Eleven teams registered to complete the course beginning in Ames, IA, Cyclone territory, and finishing just outside of Iowa City, IA, land of the Hawkeyes. Several teams were comprised of eight or more runners who ran various legs, “ultra” teams contained 5 runners or less, and ultra runner Troy Thompson completed 102 of the 124 mile course on his own! NephCure Kidney International thanks the Maurers and all of the runners who participated in the inaugural Rival Game Relay event. The 2014 RGR trophy went to the ISU Cyclones NephCure and the RGR team up to fight kidney disease! Post-race festivities under the RGR tent