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
DUET Study Releases Preliminary Results (SPOILER it looks promising!) September 7, 2016 by Kylie Karley On September 7, 2016, Retrophin Inc. released the “Top Line” results from their recently completed DUET study, a Phase 2 clinical trial testing safety and efficacy of Sparsentan for FSGS patients. Results showed promise for Sparsentan’s effectiveness at reducing proteinuria in patients with FSGS, with one group of patients seeing an average reduction of 44.8%. The DUET study included 96 patients, and only one serious adverse side effect (anemia) was reported. All patients chose to extend their treatment with Sparsentan during the trial’s open label extension period. Alvin Shih MD, the executive vice-president for Retrophin Inc., said “significant reductions in proteinuria, along with a well-tolerated preliminary safety profile have us excited about being one step closer to providing a new treatment option for patients with FSGS.” NephCure Kidney International is excited about these results and support Dr. Shih’s hope that we are moving closer to providing a new, effective, and safe treatment option for FSGS patients. Mark Stone, Chief Executive Officer of NephCure Kidney International, remarked “These preliminary results are very exciting for our community. This gives us hope that better treatment options will be available for our families in the near future.” NephCure Kidney International would like to thank everyone who contributed time, talent, and resources to this study. Thank you, especially, to the patients and families who participated and helped bring effective treatments within reach. Read the official press release here
The Unique Adult Experience Webinar December 1, 2015 by Kylie Karley “As far as I knew, kidney disease didn’t run in my family. Most important, I didn’t have time for kidney or any other kind of disease.” Dine Watson, author of the Washington Post article “Kidney disease? I was only 33 years old, and I felt fine” will be featured on our next webinar, The Unique Adult Experience. Diagnosed with FSGS in 1984, Dine’s story will resonate with all adults living with Nephrotic Syndrome and the related diseases. During this webinar, Dine will offer insight about all aspects of living as an adult with kidney disease, and also how to hope and how to find a community. Register now to reserve your spot! Hope to see you there! Questions? Contact Chelsey at 610-540-0186 ext. 19 or cfix@nephcure.org
2015 Countdown to a Cure – THANK YOU! November 20, 2015 by Lauren Eva We’ve said “hello” and “goodbye” to another Countdown to a Cure… From the beautiful scenery of New York City’s Chelsea Piers, to the heartfelt speeches delivered by NephCure’s beloved family, the Jones’ and honoree, Olympic athlete Aries Merritt, this event was truly the “Chance of a Lifetime” to make a difference in the fight against Nephrotic Syndrome. The success of Countdown has always been measured by the generosity of the many hundreds in attendance who consistently show support for NephCure and this year, our expectations were overwhelmingly exceeded, for which we could not be more grateful. You are changing the story. Energies were high and attendees were excited, lighting the way for one of the most successful galas in NephCure history. “Fund a Cure” donations blew expectations out of the water, bidding was at a high and the second annual game of “heads or tails,” brought a touch of silliness to an evening surrounding a very serious cause. Emcee, Moody McCarthy, along with “Asbury Fever,” a Bruce Springsteen Tribute band, kept the party going all night long with many moments filled with laughter, dancing and mingling. As always, we want to thank the committee who worked so hard to put this event together and the volunteers who generously gave up their time to help this event flourish into a huge success. The 2015 New York Countdown to a Cure raised over $750,000 and many left the event feeling inspired and hopeful. Finally, thanks to YOU. To each of you reading this who’ve decided to join us in this fight. We can’t do this alone. We need you, we’re grateful for you and we thank you.
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/
Featured Clinical Trial- May Newsletter May 29, 2015 by Kylie Karley Featured Clinical Trial DUET: Sparsentan in FSGS Purpose The DUET trial is an interventional study that will determine how effective the drug Sparsentan is at reducing the urine protein / creatinine ratio over 8 weeks. Who is Eligible? Males and females between the ages of 8 and 75 years old with biopsy proven FSGS, or a proven genetic mutation transplant. Click HERE to see the other criteria that determine who is eligible to participate. How Can I Participate? There are currently over 30 study sites across the United States that are actively recruiting patients! To see that list, click HERE. Don’t see a location near you? No worries! The DUET trial will reimburse you for any travel costs to a study site. All study-related health exams and medications will be provided at no cost. Click HERE to learn more about participating in the DUET Study. Click on the Clinical Trials Finder Map below to see what’s happening near you!
Debunking Clinical Trials Myths May 29, 2015 by Kylie Karley MYTH: Participating in a clinical trial will just make me a “human guinea pig” FACT: A common fear is that those giving you care in a clinical trial won’t treat you like a person or a patient. However, the opposite is true. Participating in a clinical trial gives you access to some of the best care available to patients. Also, strict ethical and regulatory guidelines are in place to ensure that each participant is treated fairly and respectfully, not to mention that all drugs in clinical trial phase go though a vigorous testing process before they are approved for use in a clinical trial. MYTH: I like my doctor, and if I participate in a trial, I won’t be able to see them anymore FACT: This is not true. Participants are encouraged to continue seeing their regular doctor in addition to any appointments they may have with the trial staff. A physician on the clinical trial staff does not replace your regular doctor. MYTH: If I participate in a clinical trial, I’ll have to go off of the medications that keep me in remission FACT: Each trial has a different protocol, or set of rules. Some trials have a protocol that allows participants to stay on current medicines. Or, if a trial asks participants to stop taking their current medications, participants are closely monitored and immediately given their original medicine and dosage if their symptoms worsen. The health of each participant is top priority for every clinical trial team. MYTH: There are no trials happening near me, therefore I can’t participate FACT: There are currently 17 clinical trials that are recruiting patients with Nephrotic Syndrome diseases at nearly 300 sites throughout the United States. If there is not a site close to you, many trials will pay for your travel (like the DUET study!). MYTH: If a clinical trial will help me, my doctor will tell me about it FACT: While we all love our doctors and respect their opinion, the truth is that they are extremely busy. We can’t expect them to save lives everyday and remember every clinical trial going on. Sometimes doctors just don’t know about clinical trials, or are unsure if their patients are interested. The best approach to participating in research is to do some research yourself and then take questions to them. If your doctor is unaware of a trial, they can find out more information and help you make a decision. However, please remember that only YOU can make the decision to advance research to help find better treatment options by participating in a clinical trial. Click HERE to learn more about clinical drug trials
Levine Family is STANDING UP TO BE COUNTED! April 10, 2015 by Lauren Eva The Levine family just took great steps to STAND UP & BE COUNTED. Meeting with the Congressman, they took just another step for the best representation and advocation for those with Nephrotic Syndrome and FSGS. Sydney Levine, who recently started a viral social media campaign with the #SUBCselfie project, caught the attention of the Congressman as well as many famous, powerful voices across the country. As a result of the Levine family, who never stop trying to find ways to beat FSGS for son and brother, Matthew, the right people are becoming aware and joining in the fight. The Congressman will be taking this to appropriations on the hill next week!
Why YOU Should Attend a Community Cafe April 6, 2015 by Kylie Karley We know that spring can be extremely busy for everybody, but we wanted to share with you the top 10 reasons why YOU and your family should attend a local Community Cafe Patient Workshop! REGISTER for one in your area! THE TOP 10 REASONS TO ATTEND A COMMUNITY CAFE: 10. Access to leading experts in the Nephrology world 9. Enjoy kidney friendly, low sodium food 8. Learn what 3 questions to ask your nephrologist at your next appointment 7. Find out what events are happening in your area 6. Get the inside scoop on research 5. Meet others and stop feeling alone 4. Hear the do’s and don’ts from a renal dietitian 3. Empower yourself with scientific knowledge 2. Become part of a global community working towards a cure 1. You owe it to yourself to know everything about what’s happening UPCOMING COMMUNITY CAFE SCHEDULE Ann Arbor- May 14
Check out our Peer to Peer Programs! March 19, 2015 by Lauren Eva One of the most important things we do at NephCure is connect people to others experiencing similar challenges of living with chronic kidney disease. Whether it be understanding the complexity of Nephrotic Syndrome/FSGS, the side effects of medications, frequent trips to the nephrologist or navigating your way through labs and diets, it can be incredibly overwhelming. At NephCure, we understand the value of connecting patients and caregivers with others to share experiences, frustrations and, better yet, good news! That’s why we’re expanding our Peer to Peer Support Program. In addition to our online support community, NephSpace, we offer our Patient to Patient Connections (P2PC) program. P2PC is a worldwide organized network of patients and caretakers whose lives have been affected by the diseases causing Nephrotic Syndrome and FSGS. It is designed to connect individuals via email or phone based upon any or all of the following: diagnosis, symptoms, and complications, age of individuals or sometimes specific geographical area. Our volunteer patient/caretaker ambassadors are committed to offering support and sharing their experiences with others who are facing similar challenges. You can learn more or be connected with a patient/caretaker ambassador here https://nephcure.org/?p=1390. We encourage you to take time to make connections with others in similar situations as we know you will benefit greatly from the support and sharing! If you are interested in becoming Volunteer Patient/Caretaker Ambassador please visit https://nephcure.org/get-involved/become-a-volunteer/ or contact Kelly Helm at khelm@nephcure.org.