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.
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.
The 10th Annual International Podocyte Conference Sold-Out in Freiburg, Germany August 8, 2014 by Lauren Eva Three hundred Nephrology researchers and clinicians descended on the university town of Freiburg, Germany to share the latest data on podocyte and kidney disease research. Co-chaired by Dr. Tobias B. Huber of Freiburg, and Dr. Thomas Benzing of Cologne, the 2014 conference presented a record number of abstracts to investigators and students from around the globe. “We are very excited by this participation, and the opportunity to share the latest research with our colleagues on topics ranging from biomarker data research to glomerular function, podocyte regeneration, and other topics. It is only through this research that cures for glomerular diseases will be found,” said Dr. Huber. Organized in collaboration with NephCure Kidney International, demand for the 10th Annual meeting became so high that conference organizers had made live stream video of the presentations available to additional attendees. The conference chairs recognized the expertise of their global scientific advisory committee in bringing a rich and vibrant meeting to life. “It’s powerful to be at the 10th conference, and to see how far research has come in the search for answers which will drive the development of new therapies for FSGS and other Glomerular diseases. The npartia[ation and collaboration amongst the scientists, the bio-pharmaceutical companies and clinicians is exciting to see happen,” said NephCure Kidney International CEO Henry Brehm. “We have more to do, and being among these professionals gives me hope.” Organizers closed the conference with a traditional awards ceremony. Dontscho Kerjaschki received the prestigious Marilyn Farquhar Award for Podocyte Research 2014. Evelyne Huynh Cong, Stefan Porubský,and Hani Suleiman received the Best Abstract Award. A full list of the 2014 International Podocyte Conference schedule and more information is available here. The 2016 International Podocyte Conference will convene in Jerusalem, Israel.
NephCure Joins ASN to Fund 2014 Kidney Research Fellows August 8, 2014 by Lauren Eva NephCure is happy to partner with the American Society of Nephrology and the ASN Foundation for Kidney Research to co-fund one of the 19 new research projects aimed at improving the quality of life for millions of kidney disease patients. The 2014 recipient of NephCure Kidney International-ASN Foundation for Kidney Research Grant is Heon Yung Gee, MD, PhD at Boston Children’s Hospital. “The 2014 grant recipients will propel advances in patient care and outcomes through basic, translational and clinical research,” Chair of the Board of Directors Bruce A. Molitoris, MD, FASN, said. “ASN is delighted to support this research and foster the careers of the next generation of investigators who will advance care for kidney patients.” For more information on all the recipients and their research, read the full article here.