The Liposorber- An Innovative Device for FSGS July 22, 2015 by Kylie Karley Not too long ago, the Liposorber LA-15 System was approved by the FDA for use in pediatric FSGS patients that have tricky cases. This unique device offers a different type of treatment for patients: it cleans the blood of LDL’s, or the “bad cholesterol”. According to the FDA’s Press Announcement, “The device works by removing certain lipoproteins from the patient’s blood. The patient’s blood is first passed through a plasma filter where the blood cells are separated from plasma (the liquid component of the blood).” The Press Announcement goes on to cite studies that showed positive outcomes of patients that used the Liposorber System, including a study that showed patients excreted less protein after kidney transplant if they had treatment with the Liposorber System (compared to transplant patients that did not receive Liposorber treatment). Dr. Joshua J. Zaritsky, a pediatric nephrologist at Nemours/ Alfred I. DuPont Hospital in Wilmington, DE, believes in this innovative treatment option, saying, “I am hopeful we may be onto something with the Liposorber. I am beginning to get the feeling that the excess lipids we see in Nephrotic Syndrome are in themselves toxic to the kidney- this may explain why the machine works and provide another yet unexplored mechanism to help treat Nephrotic Syndrome/FSGS”. The Liposorber LA-15 System (is in) a post-approval study. If you are interested in learning more- be sure to ask your doctor and read more about the device HERE.
You’re Changing The Story- The New and Improved NKN! July 22, 2015 by Kylie Karley Announcing the new and improved NephCure Kidney Network (NKN) Patient Registry! Our updated survey helps you to tell your story, and it’s only a click away. www.nephcurekidneynetwork.org With just 30 minutes of your time you can become part of the movement that changes how research of chronic kidney diseases like FSGS and Nephrotic Syndrome is done. How? Step one is to join a patient-powered registry. NephCure has partnered with Arbor research to create a safe, secure, informed network for patients and their loved ones – just like you. We call this registry the NephCure Kidney Network, or NKN. How does it work? You spend about 30 minutes answering questions online. You will need to have some things on hand, like lab results. Remember, researchers and doctors need data to develop better treatment options, so the more detailed information you can provide the better. What happens next? When you complete the survey your data donation, is reviewed for completion and then de-identified, which means your name and other identifying information is removed, and is made available to researchers and clinicians studying and treating FSGS and the conditions that cause Nephrotic Syndrome. Your data donation provides valuable information that can change the face of research. Your donation may make it easier for researchers to determine if you could be eligible to participate in clinical trials, which can be anything from answering a survey to visiting a medical center for more involved studies. Your participation is completely voluntary. It’s been more than a decade since there has been a breakthrough treatment for kidney disease, and there are no FDA-approved treatments for FSGS and NS available today. As cases of chronic kidney disease continue to rise, we need to change that. Your data donation can help. Join the NKN today and join a movement to make a difference.
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
The Evolution of Alternative Medicine July 15, 2015 by Kylie Karley In today’s world, 1 in 2 Americans suffer from a chronic disease. Director of the Center for Functional Medicine, Mark Hymen, describes the current response to health issues as an “acute disease system for a chronic disease population.” This seems especially relevant to patients whose disease can be treated, but not cured, as is the case with patients of Nephrotic Syndrome. Are there better methods to treat chronic health problems? And if so, how can we improve these patients’ quality of life in a holistic manner—physically, mentally, and emotionally? Around the nation, there are currently 50 institutions, ranging from Harvard to Mayo Clinic, that offer integrative, previously known as “alternative,” treatments. These treatments include, but are not limited to, acupuncture, massage, nutrition counseling, and much more. So what exactly is integrative medicine? At the core of integrative medicine lies a central dogma of “wellness.” The idea of wellness is a more holistic approach to medical treatment, and particularly to the medical treatment of chronic diseases. With the term “alternative,” however, comes residual stigma that these methods are only the source of false hope, and false hope alone. Integrative medicine, therefore, hopes to escape from these negative preconceptions, bridging the gap between alternative approaches and scientific validity. In other words, taking therapies that once were known as alternative medicine and subjecting them to the scientific method as with any other evidence-based medicine. On a national level, this change in focus was coupled with a change in name; the National Center of Complementary and Alternative Medicine was transformed into the National Center of Complementary and Integrative Health. The center’s director confirmed this transformation, stating that the NCCIH had in fact redirected, and narrowed, its aim. When the organization first began, it pursued a wide variety of approaches. Now, however, it has begun to focus in on only the approaches that hold some form of promise and scientific worth. For instance, in the 1990s, one doctor was putting in around 750 stents a year for patients with high blood pressure. With the help of Dean Ornish, however, a leading physician in lifestyle medicine, the same doctor began taking very ill heart patients and teaching them yoga and meditation. The results were astonishing. There was a 91% reduction in chest pain in her heart patients. More so, this lifestyle change actually began to reverse plaque in her patient’s arteries. Still not convinced? In a five-year research study on Transcendental Meditation, there was a 48% reduction in heart attack, stroke, and sudden death. As one doctors puts it, “to me that’s medicine—meditation is medicine.” This relationship between mind and body can go both ways: physical health impacts mental health, and the reverse is true as well. When a patient’s mind influences a certain health outcome, this is typically known as the placebo affect. Even without a physical stimulus producing a certain response, there is a response nonetheless. In that regard, the placebo affect can, and maybe should, play an important role in the medical world. As told by Lauren Atlas, director of the NCCIH’s neuroimaging lab, “we’ve known for decades that the placebo affect can engage endogenous opioids—your body’s own pain-relieving substances—to fight pain even without any treatment.” This is another area of promise, worthy of future research, within the field of integrative medicine. With the help of the NCCIH, further research will be done on integrative medicine and its role in the future standards of care for those suffering from chronic illnesses. Want to read more? Visit: http://www.theatlantic.com/health/archive/2015/06/the-evolution-of-alternative-medicine/396458/
INSIDE LOOK: 21st Century Cures July 15, 2015 by Kylie Karley What is the 21st Century Cures bill? Co-sponsor of the bill, Fred Upton, describes our current situation as such: patients who need “the next generation of treatments and cures, but they do not have until the next generation to wait.” This bill was designed to update, and effectively, accelerate the drug development process. It is funded by the federal sale of oil from the national reserve. What does this bill actually do? Guarantees 8.75 billion dollars to the National Institute of Health (NIH) AND, 550 million dollars to the Food and Drug Administration (FDA) over the course of the next five years In that regard, 21st Century Cures will be a huge win for the rare disease community by: Ensuring mandatory funding for the NIH Providing funds to the FDA to streamline the clinical trials process Incentivizing the development of “orphan products,” which are medical products intended for the diagnosis, prevention, or treatment of rare diseases Paving the way for the development of new cures and treatments for the 7,000 rare diseases—including NS—which currently have no approved treatment Now that I know what the bill does, what can I do? The legislative initiative, 21st Century Cures, was passed in the House on July 10, 2015. HOWEVER, our work is not finished. Before this bill can become a reality, it must also pass the Senate. Stay tuned for advocacy alerts from NephCure to see how you can get involved!
5th Grade Class Celebrates World Kidney Day! July 10, 2015 by Kylie Karley On World Kidney Day, a NephCure Kidney International staff member traveled to Stratford Landing Elementary School in Alexandria, VA to speak with a 5th grade classroom about Nephrotic Syndrome. They learned all about kidneys, Nephrotic Syndrome, and the brave patients that are part of the NephCure family. The kids are currently writing to “Pen Pals” to learn more about the journey that our patients experience. (If you are interested in having your child be a pen pal with one of the students, email Chelsey Fix at cfix@nephcure.org) The kids did some wonderful work decorating kidneys to help raise kidney awareness! Check out some of the kidneys below!
Major Breakthrough Against Rare Kidney Disease July 6, 2015 by Kylie Karley In recent news, there has been what could be a huge discovery regarding membranous nephropathy. MN is a rare kidney disease that falls under the umbrella term of “Nephrotic Syndrome.” At this point in time, there are still no successful, first-line treatments for this disease. Now, however, researchers have been able to identify the site at which antibodies bind to the kidney and cause damage in patients with MN. These findings were published in the Journal of the American Society of Nephrology. Researchers were able to create a three-dimensional model of the protein, known as PLA2R. PLA2R is a transmembrane glycoprotein, previously identified as the surface protein that allows antibodies to bind on to the kidney. By creating this 3-D model, researchers were able to narrow in and determine the specific area of the PLA2R protein that antibodies bind to. Furthermore, researchers found that small molecules, acting as competitive inhibitors, could block this site and prevent antibodies from actually attaching. So what does this mean? By knowing where antibodies bind to on the kidney, this open up new possibilities for MN treatments. There are two exciting methods that the article mentions. The first is removing antibodies from the PLA2R binding site once they are present, and the second is preventing antibodies from binding altogether. Ultimately, this article provides insight into a new path from which treatments, and even cures, for MN may come to fruition. To read more, visit: http://www.sciencedaily.com/releases/2015/05/150521091747.htm
A Call for More Specific Standards of Care July 6, 2015 by Kylie Karley From a new article, published by Nephrology News and Issues, comes a provocative call for more specific standards of care in nephrology. As of now, the standard of care for the majority of kidney-failure patients is to follow a similar treatment plan, regardless of the cause for their kidney failure. Yet, according to research conducted at Stanford University School of Medicine, this approach is not enough. In fact—it could even be dangerous. Researchers used data from over 84,000 patients, who between 1996 and 2011, suffered end-stage kidney disease due to one of six major glomerular disease types. The results were shocking. Mortality ranged all the way from 4% per year for patients with subtype, IgA nephropathy, to 16% per year for patients with subtype, vasculitis. Furthermore, patients with lupus nephritis were almost 2x as likely to die as those with IgA nephropathy. In other words, the specific type of glomerular disease determined how long a patient lived after developing kidney failure. As one researcher put it, “when you divide patients according to their glomerular disease subtype, you actually see a whole spectrum of outcomes (O’Shaughnessy).” And yet, the current standard of care is to follow a similar treatment plan for most kidney-failure patients, regardless of cause. “We showed that a patient’s cause of kidney failure is strongly associated with their risk of dying after starting dialysis or receiving a kidney transplant.” Thus, treatment can no longer be generalized and non-specific. Medical professionals cannot ignore the cause of kidney failure and proceed with treatment as though all kidney failures are one and the same. The cause of kidney failure cannot be forgotten. Rather, it should be the stepping point from which treatments are determined, and tailored toward disease-specific risks. More so, further research is necessary to determine why these survival disparities exit from one patient to the next. If medical professionals begin to take into consideration what caused the kidneys to fail in the first place, it could possibly improve the patient’s quality of life and even increase their life span following kidney-failure. To read more, visit: http://www.nephrologynews.com/study-shows-importance-of-cause-of-kidney-failure-when-planning-future-treatment/
Newest NKI-ASN Grant Recipient June 29, 2015 by Kylie Karley NEWEST NKI-ASN GRANT RECIPIENT: DR. AZELOGLU We would like to officially congratulate Dr. Evren Azeloglu, the newest recipient of the NKI-ASN Grant. Dr. Azeloglu is an Assistant Professor in the Department of Pharmacology and Systems Therapeutics at the Icahn School of Medicine at Mount Sinai. He was originally trained as a mechanical engineer, but later went on to receive his Ph.D. in Biomedical Engineering from Columbia University. In 2010, Dr. Azeloglu was awarded the Howard Hughes Medical Institute Fellowship from the Life Sciences Research Institute. His background in biomechanics and systems biology is uniquely positioned to study complex diseases such as hypertension and diabetic nephropathy. He aspires to design transformative therapeutic tools using nanotechnology and tissue engineering. The title of Dr. Azeloglu’s project is “mechanosensitive control of podocyte cytoskeleton and remodeling.” Hypertension is one of the major drivers of kidney disease. However, the physical mechanisms through which high blood pressure affects nephrotic syndrome are not well known. Dr. Azeloglu plans to utilize engineering methods to investigate the structural properties of the cells of the kidney glomeruli during healthy and diseased conditions. This project will be conducted in hypertensive animal models with a nanotechnology platform. He will explore how kidney cells retain their structural integrity against mechanical injury (i.e. due to high blood pressure) and identify potential genes that may be responsible for maintenance of the healthy cellular structure under hypertension. This project aims to use state-of-the-art bioinformatics and nanotechnology tools to link the structural integrity of glomerular cells to overall kidney function. The results of his research may directly translate into treatable drug targets that can improve the resilience of kidney cells against high blood pressure, and slow down the progression of chronic kidney disease.
Advocacy in Action: More Funding for FSGS Research! June 16, 2015 by Kylie Karley Remember When… Back in January, FSGS was officially added to the list of conditions eligible for research funding through the Department of Defense’s 2015 Peer Reviewed Medical Research Program (PRMRP). This opened up a new $247.5 million funding source for FSGS researchers. When NKI and our patient families visited Washington DC earlier this year, we advocated to keep FSGS on the list and to increase the funding available for research. More Good News: Last week, the Senate Appropriations Committee approved the 2016 Defense Appropriations budget including $31.2 million in additional funding for the Peer Reviewed Medical Research Program. FSGS made the list of eligible conditions again, so if the bill passes without changes, our researchers will have access to $278.7 million in 2016! What’s Next? The bill will have to be passed by the House and Senate and signed by the President before going into effect. We’ll be tracking it and keeping you updated as it moves through the legislative process. Thank You! Increased funding for research and including FSGS on the list of eligible DOD conditions were two of our “Asks” when we visited Capitol Hill. Thank you advocating in person, writing letters and emails, and making phone calls to increase government awareness of Nephrotic Syndrome diseases and the vital role that government funding plays in finding better treatments and cures! Keep it up and stay tuned for more opportunities to be an Advocate!