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!
Genetic Findings from an NKI Funded Researcher June 5, 2015 by Kylie Karley In 2014, NephCure Kidney International and the ASN Foundation for Kidney Research awarded Dr. Heon Yung Gee with a Young Investigator Grant. Dr. Gee is a physician-scientist at Boston Children’s Hospital who focuses on the genetic causes of nephrotic syndrome. With NephCure and ASN support, he is conducting research on how mutations in KANK2 and ARHGAP4 cause defects in the proteins that cells use to communicate. Lack of communication causes problems with podocytes—the part of the kidney that likely plays a big role in causing nephrotic syndrome. Recently, Dr. Gee published an update on this research in the Journal of Clinical Investigation. Thanks to Dr. Gee’s research, the Hildebrandt group has added KANK 1, 2, and 4 to the genetic test used to influence the treatment of nephrotic syndrome patients. As of now, this test includes approximately 30 genes, which if mutated, are known to cause nephrotic syndrome. To learn more about genetic tests, stay tuned for an upcoming NKI monthly newsletter. Dr. Gee’s research has also led to new tests, which can be used to screen for drugs that may provide a treatment for some patients with steroid-resistant nephrotic syndrome. To learn more about Dr. Gee and other NKI-funded researchers, visit: https://nephcure.org/research/nephcure-funded-research/. To read Dr. Gee’s article in the Journal of Clinical Investigation, click here: http://www.jci.org/articles/view/79504.
Q&A With Dyan Bryson from Retrophin May 29, 2015 by Kylie Karley Recently, we got a chance to chat with Dyan Bryson, the Patient Advocacy Director at Retrophin, Inc. – the pharmaceutical company that is sponsoring the DUET study for FSGS patients. We took this opportunity to ask Dyan why clinical research – and the DUET study in particular – is important for Nephrotic Syndrome patients. Keep reading to see our Q&A session with Dyan! NephCure Kidney International: Why do you enjoy/what inspires you about working in the pharmaceutical industry? Dyan Bryson: I first came into this industry thinking I would stay 3 years, that was almost thirty years ago. I simply fell in love with the industry and its potential to help people. I came in rather naively, and at one point I became completely fed up with the lack of focus on patients. I am now back in love as the industry is finally moving towards fulfilling its potential to really help patients, not just support its brands. I love being in an industry where companies that have awakened to putting the patient at the center of its business are also profitable – doing well by doing good. These companies can be examples to others that this can be done. We have realized that we need to support people after they have filled our prescriptions; we have to develop strategies to support their caregivers; we have to support people by realizing that the therapeutic area our drug may be managing is just one of a whole list of things a patient, a person, has to manage in their lives. NKI: What makes participating in DUET a unique opportunity for the NS community? DB: Participating in a clinical trial, especially DUET, is an opportunity to contribute to the knowledge and health of the community. Of course, I am biased, but as a patient advocate I know that if we truly have a drug that can help patients with this rare condition – a condition that has no existing therapy – it can help so many people in the long run. Participation allows us to collect data that may help other people avoid the current long term outcomes of end stage renal disease and transplant. Although we as the drug maker will profit, so will the community through better quality of life. If you participate you can contribute to bettering the health of others. NKI: Why is it so important for rare disease patients and their families to care about research? DB: Many times it is the family of, or person who has, a rare disease that brings that disease to the attention of researchers. Many times if that interaction did not happen there would be no awareness of the disease and, hence, no research. Rare diseases are not like diabetes – researchers, drug companies understand the impact of diabetes. Payers understand the impact diabetes makes on their bottom line as they pay the costs of diabetes management. All stakeholders have clear incentives to manage the disease better. But for a disease where there is low awareness there is little understanding of the impact on a person’s health, hence little understanding of why to do the research. Patients with a rare disease, and their care partners, are the experts on that disease as they manage it 24/7. They are the ones that can drive the understanding of the disease and interest in doing research on that disease like no one else. I still remember the first time that, as a sales representative, my company brought a patient in to talk about the therapeutic area for which we were about to launch a drug. We all had studied and been tested for months to be certified, to confirm we knew the area well enough to talk about it with the physicians we called on. Now, finally, we were at the launch meeting to get our marching orders and celebrate the launch – fun and work at the same time. So, the patient gets on the stage, we had no idea what we are in for. The patient told their story. Soon we were ALL in tears. That one patient helped us understand why were really there. We understood their day-to-day and now we were on a mission to help. Twenty-five years later I can still see that patient on that stage; that experience informs me every single day. That is the impact of a patient telling their story, motivating and inspiring those who can do something about the disease take action. NKI: What is your suggestion for patients that want to learn more and be more involved in clinical research? DB: I know it can be daunting even to think about participating in a clinical trial. You think you are going to be a guinea pig, I understand and so do many others. There is a tidal wave of information out there that can help understand how trials work and help people to understand the process. The US government has even issued grants to researchers all over the country to figure out how to better inform the public about clinical trial participation (http://www.nih.gov/health/clinicaltrials/index.htm) Certainly on the NephCure site (https://nephcure.org/wp-content/uploads/2014/08/Clinical-Research-one-sheet-9162014.pdf) Also, several healthcare advocacy groups have sites that contain materials in several languages to discuss clinical trial involvement: National Minority Quality Forum “Are you in” Campaign – http://www.nih.gov/health/clinicaltrials/index.htm National Medical Association – Project IMPACT – http://www.impact.nmanet.org/about NKI: What do you want a participant to know before they decide to volunteer for a clinical trial? DB: The managers of clinical trials are usually very good about providing access to information about their trial. Scour the site provided, read the materials you are given before you make a decision. Know that the managers of that trial have had to put the trial protocol, all trial materials through a review of a professional board that understands the therapeutic area for which the trial was designed. This board is called an Institutional Review Board (IRB). The purpose of the IRB is to ensure that all human subject research be conducted in accordance with all federal, institutional, and ethical guidelines. This IRB oversees the entire trial, meets at points throughout the trial and makes independent assessments to ensure the safety and well-being of the study participants. In other words, the drug company or other researchers are running the trial with considerable oversight. The study participant is not on their own.
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
NephCure Kidney Network Annual Report Release May 8, 2015 by Kylie Karley We are pleased to provide you with a copy of the first Annual Data Report of the NephCure Kidney Network Patient Registry. The 2015 Annual Data Report summarizes the data provided by 350+ Registry participants collected from March 2014 to March 2015. Click here for the full report Please contact registry@nephcure.org with any questions.
NKN Steering Committee: Now Accepting Applications April 17, 2015 by Kylie Karley The most unique aspect of the NephCureKidney Network Patient Registry is that it is governed by a group of patients, patient parents, patient advocates, clinicians, researchers and representatives from industry who work together to ensure that the needs of the NS patient community and the NS research community are met. Each month this group of 18 individuals meets via conference call to discuss topics focusing on recruitment and retention, patient engagement, data collection, and medical profession engagement. Because each group of stakeholders can voice their perspectives on the call, it is ensured that the NKN registry will remain both patient centered and a valuable tool for the research community. Want to be involved? If you are interested, email Joanna Dauber at jdauber@nephcure.orgor call 610-540-0186 x 36. Applications will be accepted until April 27th, 2015.
$90 Million Offered In Latest Call For Pragmatic Clinical Studies April 10, 2015 by Kylie Karley The Patient-Centered Outcomes Research Institute (PCORI) is now accepting Letters of Intent (LOIs) through their latest funding announcement under the Pragmatic Clinical Studies initiative, which is offering up to $90 million in support for projects evaluating the effectiveness of different care options in “real-world” settings. PCORI will provide up to $10 million in total direct costs per study. For the LOI deadline and other key dates associated with the latest pragmatic studies funding opportunity, visit the full funding announcement. PCORI awarded $64.1 million in February to fund the first five studies selected through this initiative and their Board of Governors will consider another slate of potential awards at its next meeting on Tuesday, April 21.
Director of Research attends RDCRN Face to Face Meeting April 1, 2015 by Kylie Karley Marilyn with Dr. Pam McInnes – Deputy Director, National Center for Advancing Translational Sciences (NCATS) 1/1/2015 – This week, Marilyn Hailperin, NKI’s National Director of Research attended the Rare Disease Clinical Research Network (RDCRN)’s Coalition of Patient Advocacy Groups (CPAG) face-to-face meeting in Rockville, MD. The meeting included patient advocacy groups that are associated with the rare disease consortia funded by the RDCRN (a program of the NIH), including the Nephrotic Syndrome Study Network (NEPTUNE).
Legislative Action Alert March 17, 2015 by Lauren Eva On the heels of our very successful Advocacy Day, comes this Alert. Please take action before end of day on Friday, March 20 Request that House and Senate Members sign on to letters supporting FSGS research Members of the House and Senate have drafted letters urging their colleagues in Congress to support medical research on focal segmental glomerulosclerosis (FSGS) and nephrotic syndrome (NS) through the National Institutes of Health and the Department of Defense. Contact your two Senators and your House Representative to ask them to sign on to the letters. If a large number of Members of Congress express support for research on FSGS/NS, federal agencies will respond by continuing to prioritize their FSGS/NS research portfolios. To determine your two Senators: Visit www.senate.gov, and select your state in the drop-down menu on the top-right corner of the webpage. Click ‘go.’ Your two Senate offices will appear with their DC office phone numbers and webpages. To determine your House Representative: Visit www.house.gov, and enter your zip code in the box on the top-right corner of the webpage. Click ‘go’ and a link to the webpage of your House office will appear. You may need to enter your full address if more than one Representative is listed. Action: Call your two Senate offices and your Representative’s office. Tell the person that answers the phone that you are a constituent. Ask to speak with the health staffer and deliver the message below. If you can’t reach the health staffer, it is OK if you deliver the message to someone else or to a voicemail. Ask if you can send a follow-up email and request an email address. Send a follow-up email based on the message below. Recommended Message – [brackets] indicate where to insert specific information: “I am a resident of [CITY]. [Cite any meetings you may have had with the office.] I am contacting you to request that your office sign on to a letter supporting research on FSGS. The closing date is March 23rd. [In the Senate: “The letter is led by Alex Graf in Senator Stabenow’s office”] or [In the House: “The bipartisan letter is led by Dante Cutrona in Rep. Costello’s (R-PA-6) office and Joel Richard in Rep. Deutch’s (D-FL-21) office.”] FSGS and the diseases that cause nephrotic syndrome are devastating kidney diseases. FSGS is a leading primary glomerular cause of end-stage renal disease. [Briefly tell your story as a patient, parent, caregiver, etc.] I hope you will sign on to the letter supporting FSGS research before March 23rd. Please keep my posted on the actions you take. You can reach me at [phone and/or email].” Thank you! Feel free to forward this information to friends and family members as well. Every call helps!
2014 Research In Review February 20, 2015 by Kylie Karley What a Year We Had in 2014! Thank you to all those who collaborate with NephCure Kidney International (NKI) to advance its mission to support research leading to better treatments and cures for those suffering with FSGS and other Nephrotic Syndrome diseases. 2014 saw several major successes in the research world that move us closer to achieving our goals. Below is a summary of NKI’s 2014 research-related accomplishments. NEPHCURE KIDNEY NETWORK PATIENT REGISTRY Hundreds of people with primary FSGS and related glomerular diseases enrolled in the newly launched NephCure Kidney Network Patient Registry. These patients and care givers of patients are at the forefront of a movement called “patient powered research”. By sharing and comparing data contributed by patients about their health and quality of life, we can accelerate the development of new treatments, including new drugs. It is important for people with a rare disease to STAND UP & BE COUNTED to ensure research focuses on the issues of greatest importance to patients and their families. To learn more or enroll, click HERE. RESEARCH GRANTS In 2014, the NKI grant program committed over $2.6 MM in support for scientific investigations that advance knowledge about the cause of NS disease, evaluate possible drug targets and help physician/scientists track the natural history of these rare diseases. Click HERE for more information about grants awarded to investigators at Washington University Medical Center, Boston Children’s Hospital and Rush University. For more information about the research consortia we support, click HERE for the Nephrotic Syndrome Study Network and HERE to learn more about CureGN. CLINICAL TRIALS Patient participation in clinical research is essential to advance new treatments. In 2014, NKI launched several new programs to educate patients and increase awareness of opportunities to participate in research. The NEW Clinical Trial Finder website lists 16 studies actively recruiting participants in the US and Canada and another 18 around the world. We launched a Clinical Trial Ambassadors Program to recruit and train volunteers to help educate patients about the benefits (and risks) to participate in clinical trials and educate the medical community about the need for more research into treatments for NS. We posted, tweeted, emailed and sent letters about NEW FSGS drug trials. Our activities have raised awareness for potential participants and increased the interest of biopharmaceutical companies in working with NKI to advance new drug trials. CATALYZE, COLLABORATE AND CONVENE NKI is dedicated to being a catalyst along the research continuum: from support for basic science research to working with the biopharmaceutical industry and the FDA to understand how we might increase the numbers of new drugs moving into the drug approval pipeline and, finally, how to rapidly integrate new treatments into health care practice. 2014 was a busy year as we joined forces with our partners in healthcare and research to promote better treatments and faster cures. The advocacy work of NKI volunteers, staff and our professional partners in Washington, D.C. was instrumental in significant NEW FUNDING for FSGS and NS research in 2014. The National Center for Advancing Translational Sciences (NCATS) at the National Institute of Health (NIH) awarded $10 MM in new funding to continue the Nephrotic Syndrome Study Network (NEPTUNE), a consortium of research centers in the US and Canada collecting and analyzing data about patients newly diagnosed with primary NS diseases. NKI was a principal funder of the first phase of this study and continues to support and participate in its expansion. A second institute in NIH, the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) awarded a new $20 MM, 5- year grant to follow patients with FSGS, Minimal Change Disease, Membranous Nephropathy and IgA Nephropathy. NKI was instrumental in collaborating with NIH to get this funding. Click HERE. For the first time, FSGS is listed as a condition eligible to receive research grants through the Department of Defenses’ Peer-Reviewed Medical Research Program. Because FSGS is listed, FSGS researchers have an opportunity to compete for grants from a NEW funding source. The total amount in the DoD’s program is $247.5 Million! NKI is a leader in assembling, facilitating and promoting professional meetings that bring together partners from industry and the academic world along with patients and their advocates to promote NS health research. In 2014, NKI: Organized the 10th International Podocyte Conference in Freiburg, Germany. This conference brings together researchers from a variety of scientific disciplines and from around the world to promote interactions and new collaborations and to investigate emerging directions in podocyte and glomerular biology research. Convened the PRO in Glomerular Disease Scientific Conference with representatives from academic medical centers, the biopharmaceutical industry, the NIH and the FDA to pursue the development of Patient Reporting Outcome (PRO) instruments to advance new drug trials for FSGS and NS. (A PRO is any report of the status of a patient’s health condition that comes directly from the patient.) An FDA-accepted PRO instrument can accelerate the number of applications to the FDA for new drug trials. NKI continues to lead the development of a PRO development consortium. Convened the third NephCure Research Accelerator Meeting that brought together physicians and scientists with representatives from more than a half dozen biopharmaceutical companies with an interest in drug development for kidney disease. Research Accelerator Meetings were initiated by NephCure to engage these two scientific groups in an exchange of knowledge that will accelerate translation of basic science discoveries to new treatments for people with NS diseases. Presented a session on the NephCure Kidney Network and patient-powered research to the US Summit of Kidney Organizations hosted by the America Society of Nephrologists at its 2014 conference in Philadelphia. NKI continues to be a leader in bringing the voice of patients to the forefront of advocacy for new treatments for chronic kidney diseases such as FSGS, Minimal Change Disease and Membranous Nephropathy.