NephCure Kidney International Launches Revolutionary Clinical Trial Discovery Tool for Kidney Disease: KidneyHealthGateway.com February 19, 2019 by Kylie Karley (KING OF PRUSSIA, PA- FEB. 19, 2019)- NephCure Kidney International (NephCure), a not-for-profit organization focused on accelerating research for effective treatments for rare forms of Nephrotic Syndrome, announced today the launch of a newly designed patient-centric website, KidneyHealthGateway.com; a resource to help approximately 100,000 Nephrotic Syndrome patients nationwide access expert care and cutting-edge research opportunities for rare and chronic kidney diseases. The website is aimed at assisting patients, through a user-friendly platform, connect to appropriate clinical trial opportunities. After answering several questions regarding their health and diagnosis, patients are shown a list of clinical trials fitting their criteria—ultimately showing each patient their best individualized trial options. “We are thrilled to debut our new website and assist patients by providing access to the optimal care and innovative research they need. There’s more interest in glomerular diseases now than ever before, and in 2019 we’ve seen a marked increase in the number of clinical trials for Focal Segmental Glomerulosclerosis (FSGS), IgA Nephropathy, and other forms of Nephrotic Syndrome. The Gateway initiative also represents a true cross collaborative effort and includes active partners from government agencies, patients, physicians, and research organizations, as well as industry. It’s a crucial time to fill these clinical trials and deliver on our promise to usher in better treatments for our patients,” Joshua Tarnoff, NephCure’s CEO said. Because KidneyHealthGateway.com is designed to improve many aspects of a patient’s quality of life, the website will also include a list of ‘Nephrotic Syndrome Specialists’ in order to connect patients to glomerular kidney disease experts earlier in their diagnosis. Delay in finding expert care can result in a lack of access to contemporary treatment approaches and a delay in gaining access to potentially relevant clinical trials. The goal of the Nephrotic Syndrome Specialists program is to improve patients’ quality of life and reduce the potential for irreversible kidney damage by providing earlier access to clinicians who are specialists in and routinely treat Nephrotic Syndrome. The ‘Nephrotic Syndrome Specialists’ program and KidneyHealthGateway.com are two parts of a larger, all-encompassing initiative NephCure has created to grow the glomerular kidney disease community and ultimately fill clinical trials. This ‘Gateway’ initiative also includes six professional working groups addressing barriers to patient recruitment, and the expansion of this patient community through global engagement and the inclusion of all primary glomerular kidney diseases. In service to this initiative, NephCure has formally partnered with the IgA Nephropathy Foundation of America with plans to complete a full organization merger in the future. Aliza Thompson, MD, MS, Deputy Director of the Division of Cardiovascular and Renal Products in the Center for Drug Evaluation and Research of the FDA, announced, “We are hopeful that this initiative will improve outcomes for our patient community and lead us closer to providing safe and effective treatments for primary glomerular kidney diseases.” Support for KidneyHealthGateway.com and the overall initiative to improve access to care and ensure trial recruitment is provided by Retrophin, Inc.; Pfizer Inc.; Mallinckrodt Pharmaceuticals; Reata Pharmaceuticals; Variant Pharmaceuticals, Inc.; Goldfinch Bio; Achillion Pharmaceuticals; and ChemoCentryx. NephCure Kidney International’s mission is to accelerate research for effective treatments for rare forms of Nephrotic Syndrome, and to provide education and support that will improve the lives of those affected by these protein-spilling kidney diseases. Specifically, NephCure is focused on glomerular diseases such as: FSGS, IgA Nephropathy, Minimal Change Disease, Membranous Nephropathy, C3 Glomerulopathy, and Membranoproliferative Glomerulonephritis. NephCure is headquartered in King of Prussia, Pennsylvania. For more information about the organization, visit NephCure.org or call 1-866-637-4287.
International Podocyte Conference in Montreal August 1, 2018 by Rebecca Cook NephCure hosted the 12th International Podocyte Conference in Montreal from May 31 to June 2, 2018. With over 309 attendees, including researchers, study coordinators, pharmaceutical representatives, physicians, and trainees, the conference was a huge success and one of the largest in the conference’s history. New this year was a deliberate focus on “bench to bedside” research, concentrating on applying research uncovered in the lab towards improving treatments for patients. The crowd of 309+ participants! The biannual conference – most recently also held in Germany, Miami, and Israel – focuses on a specific cell in the kidney called a podocyte. These cells have “foot processes” that look like tentacles, which wrap around capillaries to help the kidney filter blood. The tentacles and general shape of the podocyte resemble an octopus, which is also the conference’s mascot! Podocytes wrapping around a capillary. The banner for this year’s Podocyte Conference. NephCure CEO Josh Tarnoff introduced the conference on a positive note: While 20 years ago little was known about the function of the podocyte and how it related to chronic kidney disease, today, in part because of the connections made at past Podocyte Conferences, we have huge developments in treatments. There are now many pharmaceutical companies interested in Nephrotic Syndrome, and they are seeking to develop treatments as a result of discussions at these biannual conferences. NephCure CEO Josh Tarnoff introducing the conference. As in past years, NephCure hosted a patient education workshop for families in conjunction with the conference. The one-day program featured talks on treatment options, updates in research, and dietary recommendations, and brought together 25 patient families and more than 75 people impacted by Nephrotic Syndrome. Dr. Tomoko Takano of McGill University speaking on Cytoskeleton Regulators in Health and Disease. In Nephrotic Syndrome, the kidney’s podocytes are not functioning properly. This loss of function is one of reasons that protein leaks into the urine. It was once thought that when podocytes stop functioning, they could not be restored. However, Podocyte Conference Steering Committee Member Stuart Shankland discussed new research in regenerating these cells in animal models with the intention of developing treatments for human patients during his presentation, “Developmental origin of podocyte in injury.” Shankland has been researching how parietal epithelial cells may be able to repair or regenerate injured podocytes. Both parietal epithelial cells (PECs) and podocytes are a part of the Bowman’s capsule, a membrane that surrounds the glomerulus of each nephron in a kidney. The Bowman’s capsule, featuring the tubular pole (TP), the podocytes (Pod), the parietal epithelial cells (PEC), the vascular pole (VP), and the Bowman’s space (BS). Ryuichi Nishinakamura of Japan’s Kumamoto University spoke on “Dissecting podocyte development and disease in kidney organoids.” An organoid is an group of artificially-grown cells that resemble an organ. Researchers use organoids to learn more about how organs and organ cells like podocytes function. Anna Greka, a researcher and physician at Harvard’s Broad Institute, delivered a talk in the Advances in Therapeutics for Glomerular Nephropathies portion of the conference. Her lecture, “Mechanism-based precision therapies for progressive kidney disease,” in addition to many other presentations, emphasized the importance of personalized treatments for patients. Dr. Peter Mathieson of the University of Edinburgh at the question mic. The feedback we received on the conference was overwhelmingly positive, and many participants said it will impact their practice and research. One participant said, “More collaboration with basic scientists will lead to find[ing a] cure for podocytopathy,” meaning all diseases that affect podocytes. Other participants commented that the conference helped them in “Recognizing novel pathways of disease that will provide targets for ongoing and upcoming clinical trials.” The researchers were grateful for a space to share their developments and forward the field. Another idea common to the evaluations was the gift of hope for the future many of these talks provided. One responder explained that the conference “raises optimism for future care.” The multi-faceted concept of research, from basic science to clinical trials to cures, is ingrained in NephCure’s DNA. We are invigorated by the discussions at this year’s Podocyte Conference and look forward to hosting the next conference in Manchester, England in 2020. (Photography by Photos FERA – www.photosfera.ca)
Early Career Investigator Conference on Clinical Research for Rare Diseases July 27, 2018 by Rebecca Cook
Advocacy Alert: Oppose Tax Reform November 27, 2017 by Kylie Karley Advocacy Alert: Your Action Needed Tell your Members of Congress to oppose tax reform provisions that harm individuals and families facing rare or chronic medical conditions Take Action Now Congress is currently working on a comprehensive overhaul of the U.S. tax code. The House has already passed a measure and the Senate hopes to consider its proposal early in December. The goal is for both chambers to pass their own bills before going to a conference to negotiate a final proposal. At this time, both the House and Senate tax reform bills include provisions that would harm those affected by rare and/or chronic medical conditions: House Bill Eliminates the Orphan Drug Tax Credit and provides no new alternative to continue to incentivize and facilitate the development of therapies for rare diseases. Eliminates the Deduction for Medical Expenses which many patients and their families claim to help offset the costs of medical care. Senate Bill Eliminates Affordable Care Act’s individual mandate to purchase insurance and the Congressional Budget Office has estimated that this action will result in 13 million Americans losing health coverage. With fewer Americans (especially healthy individuals) buying into the insurance market, costs for insurance could drastically increase as the market becomes flooded with individuals that use health insurance frequently Drastically restructures the Orphan Drug Tax Credit to make it much less generous and applicable in a way that would drastically reduce rare disease therapy development. As Congress continues to consider tax reform, and which provisions will be included in any final measures, please contact your Senators and House Representative and ask them to oppose provisions undermining the Orphan Drug Tax Credit, the Medical Expense Deduction, and the individual mandate. How To Take Action: Visit www.senate.gov and identify the contact information for your two Senators by selecting your state. Use the contact information for the DC office, which will include a phone number starting with 202 Visit www.house.gov and identify the contact information for your House Representative by entering your zip code in the upper right corner. Call the offices and ask for the Health Legislative Assistant’s voicemail box or e-mail address. Use the script below to leave or send a message. Script for email or phone calls: Dear _______, My name is _________ and I am a constituent from [home town]. As the legislative process for tax reform continues to move forward, please oppose any provisions eliminating or diminishing the Orphan Drug Tax Credit, the Medical Expense Deduction, and the individual mandate to purchase insurance. As an advocate for patients and commonsense tax policy, the aforementioned provisions are essential to maintaining medical innovation and promoting comprehensive care. [Explain a little about your particular situation] Thank you for time and for your consideration of my request. Sincerely, [Name] [Address]
Advocacy Alert! Action Required: Tell Congress to Preserve Protections for Chronic Disease Patients April 10, 2017 by Kylie Karley Advocacy Alert! Action Required: Tell Congress to Preserve Protections for Chronic Disease Patients Contact your House Member to ask them to support critical patient protections during the current healthcare reform effort Tell Congress to Preserve Protections for Chronic Disease Patients The leadership of the House of Representatives is continuing to work with conservative and moderate Republicans in an effort to repeal and replace the Affordable Care Act (ACA). Their proposal, the American Health Care Act (AHCA), was pulled from consideration a few weeks ago when it could not muster the votes to pass. However, House leaders continue to try and find common ground in order to modify the House leadership bill to make it passable. The emerging House leadership plan includes a number of provisions that would be devastating for patients with chronic, complex, and costly medical conditions. The bill would remove protections for individuals with pre-existing health conditions. It would also eliminate the ACA’s Essential Health Benefits—federal quality standards for health insurance policies. In place of these protections, the bill would expand health savings accounts and tax credits, establish state risk sharing subsidies, and leave it to states to determine which essential health benefits they will offer—likely leading to lower quality benefits for patients with costly diseases. For patients with costly health conditions, they could likely never put enough money in a health savings account, nor would they be able to take advantage of a tax break associated with not utilizing healthcare services. Further, segregating costly patients into high risk pools has not worked in the past and would jeopardize access for the most vulnerable. Elimination of the federal mandate that insurers offer a minimum level of benefits and allow states the flexibility to decide these benefits would likely mean that many states would have the incentive to not recommend comprehensive benefits to those with pre-existing health conditions. Insurers could also dramatically hike premiums for those with expensive chronic health care needs. The House of Representatives could vote on this bill when it returns on April 24th from the Easter recess. Grassroots outreach and educating Members of Congress about the needs of chronic disease patients continues to influence the overall debate. At this time, please reach out to your House member and ask them to protect patients and oppose discriminatory and dangerous provisions. Take Action Secure the contact information for your House representative by visiting House.gov and using the “Find Your Senator/Representative” query tool in the upper right corner. Call the office and ask for the Health Legislative Assistant. You can either leave a voicemail or request their e-mail address and send them a message using the template below. Politely and occasionally follow up on your request. You should have an expectation that the office will respond to your specific concerns. If you would like to do more, you can request a brief meeting with the staff at your members’ local offices (the location information is on their websites). +++++++++++++++++ Dear _______, My name is _________ and I am a constituent from _________. I am also an advocate for the community of individuals impacted by _________ (condition). I write to urge you to maintain stability for chronic disease patients as you and your colleagues consider healthcare reform and changes to the American Health Care Act (AHCA). The AHCA in its current form would be devastating for my community. Segregating high cost patients into high risk programs has not worked in the past, even with a federal subsidy. Additionally, eliminating the federal mandate that insurers offer a minimum level of benefits would likely mean that many states could offer substandard benefits for those with pre-existing health conditions or hike premiums for the most vulnerable Americans in desperate need of essential healthcare. Please make sure any proposal maintains crucial patient protections that promote access and prevent financial hardships. Specifically, please ensure any future proposal: maintains essential health benefits prohibits pre-existing condition discrimination prohibits lifetime and annual caps on benefits allows young adults to stay on family coverage until they are 26 limits out-of-pocket costs for patients in a meaningful way [Add a paragraph of brief information about the medical condition you are concerned about. Tell your story.] Thank you for your time and your consideration of this letter. Please tell me how you have responded to my request. Sincerely, [Name] [Address]
Potential FSGS Treatment Option To Be Tested In Phase 3 Clinical Trial April 2, 2017 by Kylie Karley Potential FSGS Treatment Option To Be Tested In Phase 3 Clinical Trial Early in March, Retrophin, Inc. announced plans to launch a phase 3 clinical trial to evaluate a potential therapy for FSGS patients. The therapy, called Sparsentan, successfully completed a phase 2 clinical trial in 2016 with promising results. The phase 2 clinical trial, known as the DUET Trial, showed a significant decrease in proteinuria for patients that received the therapy, and a greater proportion of patients that received Sparsentan during the trial reached partial remission. Retrophin plans to launch the phase 3 trial in the second half of 2017. Phase 3 clinical trials are meant to demonstrate the effectiveness of a drug to determine how valuable it may be in clinical practice. It is the last step of research before a drug becomes approved by the FDA for clinical use. The company is currently working with the FDA to approve the protocol for the clinical trial, which includes using the reduction of proteinuria as an endpoint to demonstrate the therapy’s effectiveness. If approved, Sparsentan would be the first FDA-approved drug for FSGS patients. NephCure will be working closely with Retrophin to bring awareness to this clinical trial and help bring the patient perspective to their research. To learn more about Retrophin, Inc. and Sparsentan, click here. To learn more about ongoing studies, click here.
Q&A with Dr. Kopp of the NIH December 1, 2016 by Kylie Karley Dr. Jeffrey Kopp is a physician and researcher who focuses on FSGS and related diseases. He currently leads a group in the kidney disease section (officially called the National Institute of Diabetes and Digestive and Kidney Diseases, or NIDDK) of the National Institutes of Health (NIH). Dr. Kopp is also working on a new clinical trial for FSGS, MCD, and MN patients at the NIH headquarters near Washington D.C. We had the awesome pleasure of sitting down and catching up with Dr. Kopp about his fascinating job and new clinical trial. Keep reading to learn more, and read about some of his other research projects here. Interview highlights: Dr. Kopp works at the National Institute of Health’s kidney branch, where he studies glomerular diseases such as FSGS and MCD. He also serves as Captain for the United States Public Health Service, and has been deployed to help with medical care during natural disasters. Dr. Kopp is leading a new clinical trial for FSGS, MCD, and MN patients at the NIH studying a compound called ManNAc as a treatment option. ManNAc is a sugar that occurs naturally in your body. Another researcher at the NIH found that mice without ManNAc developed MCD, and adding ManNAc to their diet was helpful in treating it. Therefore, it may be effective at treating MCD, FSGS, and MN in humans (Dr. Kopp describes the full mechanism below—make sure you read the article!) This study requires people to stay at the NIH for 11 days total, but it can be split up into 2 trips. Luckily, there is a lot to do to pass free time you may have at the NIH, including movie marathons, exercise programs, an art gallery, and an in-house business center. Learn more about taking part in the study by clicking here or contacting Emily Brede, RN at emily.brede@nih.gov Full interview: NKI: What is your job at the NIDDK? Jeffrey B. Kopp, M.D. Dr. Kopp: I am fortunate to lead a translational research group at the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), which is part of the National Institutes of Health. Our mission is to develop a better understanding of the disease mechanisms responsible for focal segmental glomerulosclerosis (FSGS) and to develop more effective and less toxic therapies. I also serve in the United States Public Health Service, with a rank of Captain. My primary mission at NIH is to carry out basic and clinical research in FSGS. I also deploy for public health emergencies, such as natural disasters. Thus, I participated in the medical response to Hurricanes Katrina and Ike. SIDE NOTE: What is NIH? Dr. Kopp: The NIH is a federal biomedical research facility located in Bethesda, MD. The campus includes a 240-bed Clinical Research Center and extensive outpatient clinics. Every patient who comes to NIH participates in a research protocol. Some protocols involve novel treatments and other protocols involve giving samples for research. NIH physicians may give advice about standard therapies that can be used. There are no charges for any medical care provided by the NIH Clinical Center. NKI: What do you enjoy about CKD research? Dr. Kopp: CKD, and particularly glomerular diseases (such as FSGS), are incompletely understood, and the available therapies are not ideal. I like the challenge of understanding and treating these diseases, and most of all I like the opportunity to improve the lives of patients with these conditions. NKI: The newest clinical trial for FSGS, MCD, and MN patients at the NIH is looking at MaNAc as a treatment option. Why did you decide to study MaNAc? Dr. Kopp: A colleague at NIH developed mice unable to make ManNac. She found that these mice developed glomerular disease soon after birth. This disease resembled a human glomerular disease, minimal change disease. Providing extra ManNAc orally to the mice cured the kidney disease. This prompted the question: can we use ManNAc to induce remissions in our patients? Chemical Structure of ManNAc NKI: What is ManNAc? Dr. Kopp: Perhaps the word sounds to you like manna, the food the Israelites found in the desert and that helped sustain them. There is a tree in Europe that exudes a sweet white resin, similar to the sap of the sugar maple, and people who knew the Bible story called the tree the manna tree. A chemist found a distinctive and novel sugar in the manna resin, and he called the new sugar “mannose”. NKI: Does ManNAc occur naturally in the body? Is it found in food? Dr. Kopp: ManNAc is a natural product and essential for good health. Our food does not contain much ManNAc. Our bodies make ManNAc, which is converted in our cells to mannose. This in turn is converted to sialic acid, which is put on many proteins. All of these are sugars, but they differ from glucose in that they are not related to diabetes and they are present in very small amounts, so that they do not add calories in the diet. NKI: What is the reason for believing that ManNAc might be useful in treating glomerular diseases? Dr. Kopp: Podocytes are cells on the outside of the kidney glomeruli and serve to prevent plasma proteins from leaking into the urinary space. Many patients with glomerular diseases have lost sialic acid from the proteins on the podocyte. We think that providing extra ManNAc might promote the return of sialic acid to podocyte proteins and that this might improve podocyte function. We see some evidence in mouse models of FSGS that supplemental ManNAc in the diet helps treat these mice. NKI: What is involved for patients in this study? Dr. Kopp: Patients will provide their medical records for review by the NIDDK team. We also review the kidney biopsy materials from past kidney biopsy. No kidney biopsy is done as part of this study. If patients appear to qualify for the study, they will come to NIH for an outpatient visit for evaluation and to discuss study participation. NKI: Is travel to NIH paid for? Dr. Kopp: Travel to NIH can be arranged and provided by NIH. If overnight accommodation is needed, NIH can provide this also. NKI: Why are patients required to stay at the NIH during this study? NIH Headquarters Dr. Kopp: The study requires being an inpatient for 11 days, either as a single stay or as two stays of five and six days. The reason for the inpatient stay is allow frequent sampling of blood and urine and for safety, to be sure there are no side effects. NKI: What can patients do with any “free time” during the study? How much free time do you expect patients to have? Dr. Kopp: During the first five days, there are frequent time points for sample collection. During the second six days, samples are needed at 8 am and 8 pm. There is extensive free time that patients can use as they like. There are many activities that can help pass the time at NIH • Patient Computers combination television and computer (with Internet access) at most patients’ bedsides to provide access to games, web browsing, and personal e-mail via the Internet • Patient Library has more than more than 5,000 books, including a selection of current best-sellers, reference, foreign language, large-print, picture, and audio books • Clinical Center’s Fine Art Program has more than 2,000 works of art. Most artwork remains on permanent display throughout the hospital, but there are six galleries on the first floor that change every eight weeks. A walking tour is available to assist patients, caregivers and visitors in their enjoyment of the artwork on display. •Recreation Therapy programs include: o Arts and crafts o Music o Games and sports o Social events o Exercise o A large selection of DVD movies o Instruction in coping skills such as relaxation, enhanced communication, and stress management • Spiritual Care Department offers Catholic, Jewish, Islamic, and Protestant services in the interfaith chapel • Business Center has four PCs and four MACs (all with Internet connection) as well as a combined printer/copier/FAX and telephones are available. NKI: Who can participate in the ManNAc study? Dr. Kopp: We are recruiting adults (age ≥18 years) with a primary glomerular disease, including minimal change disease, FSGS, and membranous nephropathy, and with nephrotic range proteinuria (urine protein/creatinine ratio > 2 g/g). Exclusion criteria include having diabetes mellitus and receiving pulse therapies, such as rituximab. Monetary compensation is provided. NKI: How do I get more information about the study? Dr. Kopp: The study, like all clinical research studies, is described at clinicaltrials.gov. You also contact the study research nurse, Emily Brede, RN at Emily.brede@nih.gov
August is Advocacy Month August 1, 2016 by Kylie Karley Be an advocate now! Meet with your legislators locally during the upcoming congressional recess to educate them about Nephrotic Syndrome and FSGS and to ask for their assistance with key legislative and policy issues. Background Members of Congress will be in their districts throughout the month of August. Legislators use this time away from Capitol Hill to meet with their constituents in their local offices and learn about the issues impacting the people they represent. Advocates for NephCure Kidney International can use this opportunity to schedule meetings with the local offices of their members of Congress to educate legislators about Nephrotic Syndrome and FSGS and how they can assist and support affected individuals. You do not need to be an expert in government to be effective, you just need to be willing to tell your story. NephCure will assist you with the rest. Taking action is easy: Inform NephCure of your interest in making a local congressional visit by emailing cfix@nephcure.org. NephCure can help you locate the contact information for the local congressional office near you and assist you with scheduling a meeting. Consider making a local visit as a group, with other patient families from your area or with your friends, family, colleagues, or neighbors. Review NephCure’s legislative agenda, and ask us any questions you have about the issues by emailing cfix@nephcure.org. Meet with the local office, tell your story, ask them for their assistance on key issues, provide them with the leave behind materials, and then report back to NephCure by completing this evaluation form. About the Issues Each year, NephCure outlines a federal advocacy agenda that is focused on advancing medical research, facilitating treatment development, and improving healthcare. This year, NephCure has outlined the following issues as part of our legislative agenda: Provide $38.1 billion in fiscal year 2019, a $2 billion increase Provide a proportional increase for the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), the branch of the NIH that would most directly support NS and FSGS research. Provide a proportional increase for the National Institute of Minority Health and Health Disparities (NIMHD). Minorities are disproportionately affected by NS and FSGS, and this branch of the NIH could provide specific funding opportunities and training programs to benefit the community. Support rare disease research at the National Center for Translational Sciences (NCATS). Continue to include FSGS as a disease eligible for study through the Department of Defense’s Peer Reviewed Medical Research Program As a result of this funding opportunity, over $2 million has gone to FSGS research The NKI community must continue to advocate in order for FSGS to be included every year Co-Sponsor the “Chronic Kidney Disease Improvement in Research and Treatment Act of 2017 (H.R. 2644)”. This bill is aimed at improving the lives of individuals with chronic kidney disease by supporting research that identifies and eliminates barriers for transplantation This bill allows individuals to retain access to private insurance This bill is promotes access to home dialysis, which is associated with higher quality of life and better health Finally, this bill pays particular attention to chronic kidney disease in minority populations and aims to improvement access to treatment for underserved areas This agenda is comprised of contemporary legislative and public policy issues impacting the kidney community. However, to be successful in your meetings you will not need to remember complicated political information, you just need to tell your story. The materials below can help you have a productive and effective meeting with your Senators and Representatives. Toolkit for Successful Meetings How To Guide for Meeting With Your Legislators NKI 2018 Legislative Agenda Advocacy Letter Template