Parents’ Place – Back to School 101 Webinar July 30, 2015 by Lauren Eva Please register for Parents’ Place-Back to School 101 Webinar on Aug 05, 2015 8:00 PM EDT at: https://attendee.gotowebinar.com/register/8489982755633437953 Going back to school can be exciting yet stressful. Add chronic illness and special considerations into the mix and it can be overwhelming! Join us for a FAQ session with other parents who have become “experts” at sending their children back to school and ensuring they have proper care. After registering, you will receive a confirmation email containing information about joining the webinar.
Innovations for End-Stage Renal Patients July 23, 2015 by Kylie Karley As many researchers continue to search for ways to minimize the damage caused by chronic kidney disease, The Kidney Project is exploring an innovative way to improve the lives of patients who have experienced kidney failure. The lab at UCSF is heading a project on a small, implantable artificial kidney, and NephCure had the chance to hold a Q&A with The Kidney Project to hear all about this project. It could be a game-changer for patients in need of dialysis! 1. What interests you about this area of the body? Why did you choose to work with kidneys? Chronic kidney disease has reached epidemic proportions. An astounding 26 million Americans live with this disorder. As incidences of diabetes, high blood pressure, and obesity rise – some of the key causes of chronic kidney disease – so do the numbers of people diagnosed with kidney failure. These patients will either need dialysis or a kidney transplant. Neither option is ideal. Only 20 percent of patients on a kidney transplant wait list in any given year receive a new kidney, and individuals receiving dialysis are often unable to work, eat normally, or live full lives. Our team is developing a surgically implanted, bioartificial kidney to perform the vast majority of the filtration, balancing, and other biological functions of the natural kidney. The two-part device combines a membrane hemofilter and a bioreactor of human renal tubule cells to mimic many of the metabolic, endocrine, and immunological functions of a healthy kidney. It is powered by the body’s own blood pressure without the need for external tubes and tethers or immunosuppressant drugs. 2. Where is the artificial kidney at in the development process? When do you hope to have it available for patients? We need to complete animal studies to demonstrate safety and functionality of the bioartificial kidney. Through our collaboration with the Food and Drug Administration (FDA), we have developed a pathway that will allow us to arrive at the first clinical trials with a pre-negotiated set of milestones. In this pathway, we must first show that the hemofilter will successfully operate in vivo for 30 days. We are currently working on this task. Given our budgetary constraints, we have taken the approach of constructing small-scale hemofilters and evaluating their performance inside animals as a function of filter arrangement, vascular connections, and surface coatings. We have successfully shown filtration for 10 days and vascular patency for 30 days. As funding becomes available, we will increase the filtration to 30 days with full-scale devices. The completion of this key task will provide the necessary data for us to approach the FDA for approval of the first clinical trial. We expect to have a device released for clinical trials in the year 2017 if we successfully raise all of the necessary funding and we do not encounter any unanticipated development challenges. Typically, there are at least two cycles of clinical testing required for all medical devices. The nature of the results of the first round of clinical trials will largely influence the timing of release and industrial-scale manufacturing. That being said, we estimate that the clinical trials will be complete by the year 2020. During the clinical trials, we will be working with manufacturers to discuss and manage the details of production. Once the clinical trials are complete, the device will be immediately available for patients. 3. Is there anything that our patients can do to assist with your efforts? The Kidney Project is an ambitious project and is not without its challenges. In the short term, our primary challenge is funding the research. A significant hurdle is procuring enough money in order to proceed through the preclinical studies, which would allow us to build full-scale prototypes to generate data for the first round of in-human studies. NKI’s note- Eventually, patients will be needed for these studies, so be sure to check our website, and The Kidney Project’s website often to stay up to date! 4. What other innovative projects should we be aware of? What’s exciting about kidney research right now? Several teams have developed the idea behind a wearable artificial kidney (WAK), which translates the traditional dialysis machine into something that can be worn on a belt around the waist. It works by filtering a patient’s blood through a membrane. While it is much more convenient than conventional dialysis, it is does not perform all the functions of a healthy kidney. We are excited about The Kidney Project and the promise it holds for patients. By building on proven scientific successes in cell biology and silicon nanotechnology, we are engineering an implantable and self-regulating bioartificial kidney that does not require the patient to be tethered to a dialysis machine or take immunosuppressive medications. NKI will continue to monitor for any further developments of these projects. For more information on The Kidney Project, visit their website HERE.
Live Your Best Life With Nephrotic Syndrome! July 22, 2015 by Kylie Karley Coming this fall to a city near you: our freshened up patient workshop about “Living Your Best Life with Nephrotic Syndrome”! These FREE half-day seminars feature experts in Nephrotic Syndrome that can help you, and your family, live YOUR best life! Added bonus: a free kidney healthy lunch! Click on your city below to reserve your spot before it’s too late! Albuquerque, NM – Oct. 24 REGISTER Ann Arbor, MI – Oct. 24 REGISTER St. Louis, MO – Nov. 14 REGISTER Philadelphia, PA – Nov. 14 REGISTER RECENTLY COMPLETED WORKSHOPS: Charlotte, NC- Sept. 12 Cleveland, OH – Sept. 19 Austin, TX – Oct. 3 Baltimore, MD/ D.C. – Oct. 3
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