NBA Hall-of-Famer Alonzo Mourning Shares Personal Story to Raise Kidney Disease Awareness November 18, 2021 by Kylie Winkler KING OF PRUSSIA (Nov. 18, 2021) – NBA hall-of-famer Alonzo Mourning was at the height of his basketball career—he had just won gold in the 2000 Summer Olympics when he noticed extreme swelling throughout his body and a lack of energy. A routine physical exam showed abnormalities, and eventually, Mourning received a diagnosis of a rare, protein-spilling kidney disease called focal segmental glomerulosclerosis (FSGS). “I said ‘Doc, am I going to die?’ He paused; he took too long to answer me. And he said, ‘We have no known cure’… ultimately, he said, ‘In about 10-12 months you’ll probably be on dialysis,’” Mourning said as he recalled the jarring day. Mourning, who missed the entire 2002-2003 season due to kidney disease and later went on to win the World Championship with the Miami Heat in 2006, publicly recounted his personal journey with FSGS in a video for NephCure Kidney International’s kidney disease awareness campaign, debuting Nov. 18. In highlighting Mourning’s story, NephCure aims to reach those who are at risk for kidney disease and to educate the public about the signs and symptoms of these conditions. Black Americans are 4-5 times more likely to develop kidney failure than white Americans, and 1 in 8 are at risk for a genetic form of kidney disease. A variation on the APOL1 gene contributes to this disparity. “We are proud to work with Alonzo Mourning in sharing such an important message that will inspire and inform others who fall within this at-risk population. Our goal is to find a cure for this debilitating kidney disease, as well as educate and support those who are affected by it,” said Michael Levine, NephCure Board President and kidney disease patient parent. This awareness campaign comes just months after NephCure launched its Health Equity Initiative, with the goal of ensuring equitable access to advancements in research, treatments, and care, and to reach individuals from communities of color earlier in their disease progression, preventing or delaying their need for dialysis and transplantation. Support for this video and the awareness campaign was provided by Vertex Pharmaceuticals, Travere Therapeutics, and other funders of the Health Equity Initiative. To watch the entire video with Alonzo Mourning, click here. About NephCure Kidney International 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. Founded in 2000 by a group of committed patient parents, NephCure has invested more than $40 million in kidney disease research and helped create a landscape where there are now more than 30 interventional drug trials for primary glomerular kidney diseases. NephCure is a U.S. tax exempt 501(c)(3) public charity. Learn more at NephCure.org. ###
Everything You Need to Know About Travere’s New Clinical Trial for Children, EPPIK September 2, 2021 by Kylie Winkler Travere Therapeutics has recently launched their Phase 2 pediatric clinical trial, EPPIK (Evaluating Problematic Proteinuria in Kids) which is aimed at helping children with FSGS, MCD, IgAN and other rare kidney diseases. We know that with a clinical study specifically designed for kids, parents and caregivers may have some questions. We asked Travere to answer some of these questions and have complied an FAQ list for our community around EPPIK. What is the EPPIK study? EPPIK (Evaluating Problematic Proteinuria in Kids) is a Phase 2 study of children 1-17 years old with focal segmental glomerulosclerosis (FSGS), minimal change disease (MCD), IgA nephropathy (IgAN), also known as Berger’s disease, IgA vasculitis (IgAV), also known as Henoch-Schönlein purpura, or Alport syndrome (AS). These rare kidney diseases are associated with progression to end-stage kidney disease and currently have limited or no treatment options. This study aims to further understand if an investigational therapy, sparsentan, can help the kidneys filter protein and slow the decline of kidney function. The amount of protein found in the urine (called proteinuria) is seen as a marker of kidney function. Lowering proteinuria levels is associated with better kidney health outcomes. What is sparsentan? Sparsentan is a novel Dual Endothelin Angiotensin Receptor Antagonist (DEARA) – this means it is a dual acting, highly selective antagonist of both the endothelin A receptor (ETAR) and the angiotensin II subtype 1 receptor (AT1R). In the EPPIK Study, sparsentan is taken once a day orally. What is a Phase 2 study? A Phase 2 study is a clinical trial that helps researchers determine how safe, tolerable and effective an investigational drug may be in specific patient populations, such as those noted above. While the EPPIK study is a Phase 2 study, sparsentan has been studied in another Phase 2 study as well as two Phase 3 studies involving approximately 880 patients (some of whom were children). The purpose of the EPPIK study is to gain further experience on the use of sparsentan in children. How long does this study take? Patients who meet all eligibility criteria and are enrolled in the study may expect to participate in the study for about 2 years and 3 months. Will you be enrolling children with congenital Nephrotic Syndrome or infantile Nephrotic Syndrome? Yes, children ages 1-17 with one of those syndromes who meet the study criteria will be able to participate in the EPPIK Study. The specific disorders we are looking for are noted above in the answer to question 1. How many children and young people are you looking to enroll in this study? We will be looking to enroll approximately 57 children in the EPPIK study. How many study sites are there available? Where are these sites located? Sites will be in the United States, UK, and various countries in the EU (France, Germany, Italy, Spain, Netherlands, Poland, Sweden). The final list of sites has not been fully determined yet. Because this study is for children, how is it different than a study for adults? The main difference is how much blood is drawn from the children to do all the tests necessary to evaluate sparsentan in the study. Patient safety is always our number one priority, and, with children, we won’t sample as much blood as we would from an adult. We narrowed the number of tests and worked with the central lab to be able to get results from a smaller sample size. We also must ensure that we adequately inform both the child who might participate in the study and the child’s parents/guardians about all the requirements the study has as well as any risks that may be associated with participating in the study. We do this by developing different study assents or ways to communicate what the study entails so that participating families understand the purpose of the study and what is involved. Are you asking kids about the taste, smell, etc. of the study drug? Yes, we will be asking the children to answer simple questions on how the drug tastes, smells, feels, etc. How do you determine which participants will be required to have pregnancy testing or take birth control? We realize this is a delicate subject for some children and their parents, but it is very important that any female child who has had her first menstruation period take birth control according to the protocol. This class of drugs is known to potentially cause harm to a developing fetus, and therefore we do not want any female participant who is able to have a child to become pregnant. Is there flexibility where lab samples can be collected for my child? Is there an option for home care visits to collect these samples? Yes, there is some flexibility in where labs are taken. If the child can’t make it to the clinical site to have the labs drawn, which is always the preferred way, we have contracted with a company that will come to the patient’s home or other convenient location, to draw the labs and send them in for testing. Does my child have to stop the medications they’re on? What drugs can they remain on? That depends, if they are enrolled in this study, they will need to stop any drug that functions similarly to the investigational drug. That is, if they are on an ACE inhibitor or ARB, they will need to stop those drugs at least 2 weeks before they begin taking the experimental drug and will not be able to take them while they are participating in the study. They will be able to continue with certain blood pressure medications if not in the same drug class, as well as many other medications they may be on. The Investigator will have a complete list of drugs that are allowed and not allowed. What will happen to the results of this clinical study? After all the patients have been enrolled and have completed the study, the data will be analyzed to see if it was successful in reducing the amount of protein in the urine and if it slowed the decline in kidney function as measured by estimated glomerular filtration rate, or eGFR. How well the drug was tolerated, that is, the safety profile, will also be analyzed. Those results will, in all likelihood, be put into a document known as a manuscript and published in a scientific journal. Data will also be added to the study listing on the clinicaltrials.gov website at the end of the study. If my child is in another trial studying sparsentan, do they have to enroll in this new study? If so, will their study site change? No, if your child is participating in another trial with sparsentan they would not be eligible to join the EPPIK study. They would stay in the study they are currently in. For more information about EPPIK, contact medinfo@travere.com
Get to Know Dr. Matt Sampson August 26, 2021 by Delaney Geraghty NephCure is proud to honor Dr. Matthew Sampson as our 2021 Boston Countdown to a Cure Medical Honoree, recognizing his outstanding contributions to nephrology and the NephCure community. Dr. Sampson is a Pediatric Nephrologist at Boston Children’s Hospital and holds the Warren E. Grupe Endowed Chair in Nephrology. He is also an Associate Professor of Pediatrics at Harvard Medical School, and an Associate Member of the Broad Institute, where he’s involved in the Kidney Disease Initiative. Learn more about Dr. Sampson’s research on the Sampson Lab website. The second annual Boston Countdown to a Cure will be held on Saturday, September 18th, 2021 — click here for event tickets. Why did you choose to study nephrology, and specifically rare kidney diseases? Dr. Sampson: During my training, I took care of a lot of children with kidney disease. I was struck by how little we knew about the underlying causes of these conditions. The treatments we had for them were oftentimes not specific and created as many side effects as they did opportunities to help their condition. It was this combination of many children being quite sick and not exactly knowing why they were sick, and then recognizing the current medications that we had for them were insufficient to help treat or cure the disease that really drove me to study these rare kidney conditions, specifically Nephrotic Syndrome. Can you tell us about some of your more recent work? Dr. Sampson: My group’s general focus is to map genetic causes and contributors to Nephrotic Syndrome and to understand how these genetic risk factors are contributing to this disease. If we understand how the genes contribute to disease, then we can work with collaborators to develop drugs for these genetic forms of kidney disease. In addition, classifying patients with the genetic form of this disease can be helpful in explaining to parents and children why they have their condition. Even if we don’t yet have a treatment or cure, we may be able to tell them how the disease can progress for children who have the specific genetic form of the condition, and we may be able to suggest certain treatments or set expectations based on that. A couple of areas that we’re really focusing on right now are APOL1-associated kidney disease, which is a disease that primarily affects patients of African ancestry. We know that about 60-70% of self-reported Black patients who have FSGS have the APOL1 form of their condition. If we can understand and find mechanisms of APOL1 disease, we can hopefully help create medicines for treatments and cures. Additionally, we are focusing on finding the genetic contributors to immunosuppressive sensitive Nephrotic Syndrome [i.e., Steroid Sensitive Nephrotic Syndrome (SSNS)]. I think a lot of times doctors think that if patients are immunosuppressive sensitive, meaning they respond to medication, then they are fine, but what they don’t recognize is how poisonous some of these medications are and how desperately families are seeking treatments that don’t come with all the side effects. What work of yours are you most proud of? Dr. Sampson: I think what I’m most proud of is that over the past decade, since I established my lab in Michigan, our group has been comprised of hardworking, motivated individuals who’ve collectively gone after the genomics of Nephrotic Syndrome. We’ve created a group of researchers who can contribute meaningfully to these efforts, deliver results or ideas when called upon, and lead efforts together in a collaborative, collegiate way. Why is the genetic side of kidney disease so important to study and learn more about? Dr. Sampson: Genetic forms of this condition, whether they’re a cause or contributor, really represent a root cause and let us know what makes this disease start or relapse. In doing that, genetic mapping can point us toward specific molecular classifications of disease, which allows us to be more precise in our care of patients. Secondly, by figuring out the genetic causes, it can point us toward specific treatments. Rather than treating the symptoms themselves, we can try to cut the disease off at its origin. What does it mean to you to be named this year’s Boston Countdown to a Cure Medical Honoree? Dr. Sampson: It really means the world to me to be recognized and honored by an organization that has been so meaningful to me since the beginning of my career. NephCure has been instrumental in supporting my efforts through grants and kind words. I found NephCure during my fellowship, and they’ve been supporting me with a lot of energy and enthusiasm throughout my career. They’ve also helped to really connect me with patients. Being involved with NephCure through patient days, patient presentations, and meeting families at other NephCure-sponsored events, it’s clear to see who we’re fighting for here and why I’m going to work every day. It’s a wonderful group to be associated with, and to be honored this year for a contribution that we’re making as a team feels amazing. What has it been like to work with NephCure over the years? Dr. Sampson: It’s great to have a patient advocacy group that is so focused on these rare diseases. There are not many people or organizations in this world that are speaking and specifically advocating for patients with Nephrotic Syndrome in such a professional way. As much as I would like to do that, I have so much work to do in terms of research, papers, grants, and managing my team that I don’t have the opportunity. To know there’s a highly competent group of professionals and volunteers that are coming together on this front is incredibly special and important.
Encouraging interim results from Travere’s PROTECT trial for IgAN patients August 20, 2021 by Kylie Winkler Interim results have been announced from Travere Therapeutic’s PROTECT study, and although preliminary, the data look promising for IgA Nephropathy (IgAN) patients. Travere’s ongoing Phase 3 trial is studying the drug sparsentan in IgAN patients. On average, patients who received sparsentan experienced a nearly 50% reduction of proteinuria after 36 weeks. That’s roughly three times more than the active control. Active protein-spilling (proteinuria) leads to kidney damage. The priority for every patient should be to stop or reduce protein in their urine. To date, sparsentan has been generally well tolerated by those in the study. Unlike steroids and other commonly prescribed off-label drugs for progressive kidney diseases like IgAN, sparsentan is not an immunosuppressant. This news comes just a few months after the interim data from Travere’s Phase 3 DUPLEX study evaluating sparsentan in FSGS, also achieved its interim proteinuria endpoint. The PROTECT and DUPLEX studies are some of the largest studies to date for IgAN and FSGS patients, respectively, to reach this level of trial data. It’s possible that these results, could ultimately lead to a potential approval of sparsentan. To read more about Travere’s interim results from the PROTECT Study in IgAN, click here. Want to learn more about this research and what it could mean for patients with IgAN and other progressive, protein-spilling kidney diseases? Join NephCure’s Nurse Kristen on Facebook Live on August 25th where she discusses this news in further detail — breaking down the study results, who could be impacted by this research, and what the timeline could be to see new treatments for IgAN, FSGS, and other rare, protein-spilling kidney diseases.
Bala’s Battle with FSGS August 19, 2021 by Rodrigo Campos-Sánchez At the age of 20 years old, Bala Krishnalal’s life was forever changed by the results of a kidney biopsy. In 2014, after suffering from recurring fevers for two years, Bala Krishnalal was diagnosed with focal segmental glomerulosclerosis (FSGS). Like many FSGS patients, he was the first in his family to ever develop the rare kidney disease. “The doctor I was seeing at the time told me that something was wrong with my kidneys. The doctor needed to do a biopsy test on me to understand what was truly happening to me and what damage was possibly being done also,” Krishnalal said. After his kidney disease diagnosis, started what Krishnalal calls the real challenge — finding a doctor who specializes in rare, protein-spilling kidney diseases. He switched doctors multiple times until he found one who listened to him, was knowledgeable about his rare disease, and ultimately made him feel comfortable. “I changed doctors so many times because they did not treat me as a human being. They all had different opinions and treatment plans, which I did not really appreciate,” Krishnalal said. “But my current doctor is so much better. This person gives me so much confidence and listens to my opinions when we talk about prescriptions.” Although Krishnalal never experienced any swelling, one of the more common symptoms of FSGS, he did have a constant runny nose and frequent flu. He stresses the importance of maintaining a kidney-friendly diet and keeps a close eye on his salt and sugar intake. Krishnalal’s diagnosis came at the height of his college career. Being a student, he was also fully dependent on his parents. While Krishnalal’s mother and father were helping him pay for his education, there was not much left to put toward medical bills. “It affected me mentally and emotionally. The diagnosis only added more to what we thought was already hard to pay for,” Krishnalal said. His father, who worked as a taxi driver for 20 years, had to turn to other family members and close friends to borrow money in order to cover his son’s medical bills. “It was a very rough phase in my life, but with God’s grace, currently we are doing well economically. Things are better since I received a job after college,” Krishnalal added. Since graduating, Krishnalal secured a position as an automotive product certification engineer. Thankfully, FSGS does not affect his work ability. Despite his kidney disease, he feels completely fit, comfortable and enthusiastic at work. Seven years after his diagnosis, Krishnalal sees himself as a fighter, a warrior and as a man with FSGS who still has a million dreams ahead of him. “Now that I am on my own and am thinking about building a family with my significant other, I feel as if I am under a lot of pressure. I have many responsibilities I need to take on and always remember the fact that I do have FSGS no matter where I am,” he said. “It is challenging, yet the day will come when I overcome my disease, and many others will also.” Krishnalal is just weeks away from his wedding on September 10, 2021. “I’m overly excited. It is incredible that someone looked past the fact that I have FSGS. It is all about a positive mindset. One should not worry about their medical conditions yet worry about what is to come the next day. The future will be amazing,” Krishnalal said. Additionally, Krishnalal volunteers as a NephCure advocate. He actively connects with new patients, provides them with educational resources, and helps spread awareness for FSGS and other Nephrotic Syndrome related diseases. “I am here to support anyone in case of anything concerning FSGS. I do reach out to a lot of people, and I understand that the ones I do talk with already know a lot about the disease. Yet, there is still so much that can be done,” Krishnalal said.
¿Que debo saber de síndrome nefrótico?: What should I know about Nephrotic Syndrome? July 20, 2021 by Rodrigo Campos-Sánchez Dr. Sergio Infante Durante nuestro webinar de NephCure U: síndrome nefrótico 101 en español, dirigido por el Dr. Sergio Infante, MD, recibimos varias preguntas de pacientes y cuidadores acerca del síndrome nefrótico en sí, ensayos clínicos, y cómo COVID-19 afecta a aquellos con enfermedades renales raras que derraman proteínas. A continuación, encontrarás las respuestas del Dr. Infante a estas preguntas. Para ver el seminario web completo sobre NephCure U (en español) que explica los fundamentos del síndrome nefrótico y cómo gestionar su atención a través de la era COVID-19, presiona aquí. ¿Cómo puede un paciente inmunosuprimido protegerse contra COVID-19? Uno no solo debe tener cuidado contra el COVID-19. Sino, cualquier otra enfermedad. Con COVID-19, si tenemos que poner un poco más de atención. Uno está más predispuesto a tener el COVID-19 cuando uno está deprimido. Uno debe utilizar doble máscara y tener distancia de las personas. No se sabe cuando uno está infectado o ha recibido la vacuna. ¿Crees que me debo poner la vacuna? Si, y porque la vacuna funciona muy bien. Pero también existen ciertas dosis de medicamentos o ciertos tiempos entre los medicamentos en donde la vacuna no va ser efectiva. El tiempo para colocar la vacuna es muy importante. Es muy importante que uno se comunique con el equipo médico para poder saber cuándo hay que ponerse la vacuna. La vacuna no representa riesgos para personas inmunosuprimidas. ¿Existen ensayos clínicos para pacientes con síndrome nefrótico? Sí y también hay varios estudios que se están siendo desarrollados alrededor del mundo con diferentes terapias. Uno de los propósitos más importantes es disminuir la exposición hacia los esteroides. Eso es porque sabemos los efectos secundarios que existen en los esteroides. En la página de NephCure, existen tipos de recursos y respuestas de cuáles son los estudios que se están desarrollando alrededor del mundo en los grandes centros médicos sobre esta enfermedad. Mi médico no me ha hablado sobre ensayos clínicos. ¿Qué debo hacer? Uno debe utilizar recursos como NephCure. Muchas veces la limitación del tiempo, no nos deja que tengamos una conversación sobre estos tipo de recursos. Es muy importante armar una lista de preguntas. Yo digo esto porque generalmente cuando uno va al médico, se le olvida todo. Cuando existe la oportunidad de hablar con el médico o con alguien del equipo, luego se puede contestar esas preguntas. ¿Deberíamos preguntarle al doctor sobre otro tipo de medicamento para una persona que tiene muchas recaídas? Afortunadamente, tenemos los esteroides. Es uno de los medicamentos más fantásticos que existen. Es capaz de disminuir una gran cantidad de problemas que tenemos. Si tienen efectos secundarios, no hay duda. La utilización de los esteroides es en ciertas ocasiones indispensable porque no tenemos otros recursos. Dependiendo el tipo de enfermedades, si están de buscada por más medicamentos para poder disminuir la utilización de esteroides. Dependiendo del tipo de enfermedad qué hay, pueden existir otro tipos de recursos que podemos utilizar para disminuir la utilización de los esteroides. Pero yo pienso que debemos estar muy agradecidos con que tenemos los esteroides. ¿Existen actividades que las personas con síndrome nefrótico deben evitar, cómo hacer ejercicios o viajar? Mientras que la enfermedad esté activa y que uno tenga una gran cantidad de líquido, uno debe que tener cuidado con la piel, sobre todo. La piel es la barrera más grande que uno tiene. Es el órgano más extenso del cuerpo también. Cuando uno está reteniendo mucho líquido, es frecuente que uno tenga problemas en la piel. Es una de las cosas que uno tiene que evitar. Con viajando, no hay problemas. Poder viajar es importante y también es una parte de nuestras vidas. No hay una contraindicación con viajando. Pero con respecto a los deportes de contacto, hay que tener cuidado con tener traumas y problemas en la piel. During our NephCure U: Síndrome Nefrótico 101 en español webinar, led by Dr. Sergio Infante, MD, we received several questions from patients and caregivers regarding Nephrotic Syndrome itself, clinical trials, and how COVID-19 affects those with rare, protein-spilling kidney diseases. You can find Dr. Infante’s answers to these questions below. To watch the full NephCure U webinar (entirely in Spanish) that explains the basics of Nephrotic Syndrome and how to manage your care through the COVID-19 era, click here. How can an immunosuppressed patient protect oneself against COVID-19? One should not only be careful against COVID-19. But also, with any other disease. With COVID-19, we do have to pay a little more attention. One must double mask and keep distance from people. It is not known when one is infected or has received the vaccine. Do you think I should get the vaccine? Yes, because the vaccine works very well. But there are also certain doses of medicines or certain times in the medicines where the vaccine will not be effective. The time to get the vaccine is very important. It is very important that one contacts one’s health care team to be able to determine when to get the vaccine. The vaccine does not pose a risk to immunosuppressed people. Are there clinical trials for patients with nephrotic syndrome? Yes, and there are also several studies that are being developed around the world with different therapies. One of the most important purposes is to decrease exposure to steroids. That’s because we know the side effects that exist with steroids. On the NephCure’s Kidney Health Gateway webpage, there are types of resources and answers to which studies are being developed around the world in large medical centers about this disease. My doctor has not told me about clinical trials. What should I do? One must use resources like NephCure. Many times, the time limitation does not let us have a conversation about these types of resources. It is important to build a list of questions. I say this because generally when one goes to the doctor, one may forget everything. When there is an opportunity to talk to the doctor or someone on the team, then one can have questions answered. Should we ask the doctor about another type of medication for a person who has a lot of relapses? Fortunately, we have steroids. It is one of the most fantastic medicines available and it can reduce a lot of problems. They have side effects, there is no doubt. Steroid use is sometimes indispensable because we do not have other resources depending on the type of disease. If one is looking for more medicines to be able to decrease steroid use, that is. Depending on the type of disease, there may be other types of resources that can be used to decrease the use of steroids. But I think we should be very grateful that we have steroids. Are there activities that people with Nephrotic Syndrome should avoid, like exercise or travel? If the disease is active and one has a large amount of fluid, one must be careful with the skin. The skin is the largest barrier one has. It is the largest organ of the body as well. When one is holding a lot of fluid, one may often have skin problems. It is one of the things one must avoid. With traveling, there are no problems. Being able to travel is important and is a part of our lives. There is no contraindication with traveling. About contact sports, one must be careful about having traumas and skin problems.
The Liposorber Through the Eyes of Nephrologists July 1, 2021 by Kylie Winkler Kaneka’s LIPOSORBER® LA-15 System is a blood processing machine used outside of the body. This LDL apheresis device removes certain lipoproteins (cholesterol) from the blood. It’s used for pediatric and adult patients diagnosed with primary Focal Segmental Glomerulosclerosis (FSGS) either before transplant or who experienced FSGS recurrence after transplant. Dr. Joshua Zaritsky, MD, PhD Several doctors across the country have been treating patients with the LIPOSORBER®, including Dr. Joshua Zaritsky, a pediatric nephrologist, and Dr. Vasil Peev, an adult nephrologist. We asked both doctors to share their experience with the LIPOSORBER® system and recommendations for patients considering this treatment option. What is your experience with LDL apheresis, and what kind of patients do you treat? ZARITSKY: I’ve had experience with LDL apheresis over the last six years. There are two classes of patients I treat. One is patients who have had a renal transplant and have had recurrence — they’re probably the most common patients I treat. The other group of patients are those who have FSGS or another sort of steroid-resistant nephrotic syndrome. Those patients haven’t yet undergone transplantation. PEEV: I’ve treated two patients so far with LDL apheresis who have had recurrent FSGS and are post-transplant. What has the patient’s response been to the treatment? ZARITSKY: I’ve had very good luck with those patients who have had recurrence after transplant. We actually published that data; we had seven back-to-back cases that were 100% successful. Since then, we’ve had some failures to treatment, but we’ve had very good luck post-transplant. In the pre-transplant area, we see response rates anywhere from 30-50%. But that being said, we’ve had some incredible responses. Other centers were about to take out both patients’ kidneys, and we’ve been able to rescue them and put them into remission. Dr. Vasil Peev, MD PEEV: Both patients have had an outstanding response to LDL apheresis. One of them had recurrenceafter receiving a living-donor kidney from his mother. He’s now almost three years post-transplant with very stable renal function and no proteinuria. I just saw this patient recently and he’s doing fantastic! His kidney function remains somewhat impaired as his LDL apheresis treatment was initially delayed leading to some progression of CKD from recurrent FSGS after his transplant. When treating him after transplant, I initially gave him what is considered to be standard of care, i.e. plasmapheresis (and various other medications). He unfortunately failed these despite very aggressive and numerous efforts to control his proteinuria. The LDL apheresis was basically a Hail Mary treatment that really saved his kidney and kept him off dialysis. Are patients comfortable during the treatment? ZARITSKY: What I’ve noticed as a doctor is that this is very well-tolerated treatment. There’s not a lot of side effects. For the most part in my clinical experience, there hasn’t been a lot of side effects. PEEV: From what I’ve heard patients say, it’s very comfortable and much smoother than dialysis treatment. The blood flows are much lower (50-100 CCs per hour). The treatments last for few hours and are very well tolerated. Patients don’t feel drained, which is something that’s frequently described with dialysis. What is your recommendation for other nephrologists? ZARITSKY: I think it’s important to reach out to other physicians who have some experience using the equipment, because we can also put our nurses in touch with their nurses who have experience. There’s some tricks and tips that we’re willing to share and, for the most part, it’s a nice community of doctors who are always willing to help out. PEEV: I think that it’s definitely worth a try. Again, in the field of nephrology, unfortunately, we still are working with a handful of very old, adopted drugs from other medical specialties — mainly from oncology. I would convey the message to other nephrologists that the complication rates with this device are zero to none. Although it may require initially some efforts by the nephrologists to get patients treated with the LIPOSORBER® device (in terms of getting access to the device), these efforts will likely be eventually rewarded. In general, I think we should not spend much time with the other treatments if we see that they are not working or failing. Doctors should consider embarking on this treatment modality rather sooner, as sooner they expose their patients on this treatment (before more significant damage is done to the kidney), more likely the patients will have a good response. How early do you suggest starting the treatment? ZARITSKY: FSGS is one of these diseases that can aggressively decrease kidney function, and once that kidney function is gone, there’s no getting it back. The LIPOSORBER® is really one of these treatments that after patients have tried other treatment modalities, you want to treat them as soon as possible. PEEV: Ideally within the first 8-12 weeks after presentation. If you see the patients not responding to first-line therapies in 8-10 weeks following the initial presentation with recurrent FSGS, I would recommend initiating this treatment as soon as possible. When left untreated this condition is frequently associated with allograft loss, despite the best efforts of the transplant team. For more information about Kaneka’s LIPOSORBER® Trial, click here and here.
You Can Change MN Treatment Options: EL-PFDD 2021 June 30, 2021 by Delaney Geraghty Externally led patient-focused drug development (EL-PFDD) meetings bring together patients and care partners, US Food and Drug Administration (FDA) representatives, pharmaceutical companies, and doctors who are experts in the particular disease to discuss the path toward new treatment options. These meetings serve as a platform for patients who have the disease to make their voices heard and provide the FDA and pharmaceutical companies insight into the patient experience. This year, NephCure Kidney International and the National Kidney Foundation are coming together to conduct an EL-PFDD meeting on August 27, 2021, to inform these key groups about the patient perspective of living with membranous nephropathy (MN). The meeting will be held online. We invite anyone who has MN, lives with someone affected by it, or is interested in it to attend this critical meeting. This year’s EL-PFDD meeting on MN will be co-chaired by Drs. Laurence Beck and J. Ashley Jefferson. Read on to hear from them about the importance of patient attendance at this EL-PFDD meeting. Dr. Laurence Beck Dr. J. Ashley Jefferson As a patient with membranous nephropathy, why should I consider attending this meeting? Dr. Jefferson: MN is a rare disease, but one that has a major impact on those who suffer from it. Although nephrologists taking care of MN patients understand the effects of this disease, many of the people designing and evaluating the results of clinical trials and testing new medications haven’t met anyone with MN and may not recognize the issues that patients face each day. This meeting is your chance to explain not only the impact of the disease, but also the limitations of our current therapies. Each patient’s experience is unique, so hearing from as many different people as possible is incredibly helpful. We encourage you to share what you would like to see from new treatments for MN in terms of how they are administered, how long the medication must be taken, the side effects, or anything else you think would be relevant to the development of new treatments. The EL-PFDD meeting is a unique setting where patients, clinicians, scientists, industry leaders, and regulators are all gathered together to listen. Therefore, your attendance and insight vital is vital. How does this meeting contribute to putting new medications for membranous nephropathy on the pharmacy shelf? Dr. Beck: All new medications must go through a rigorous process to make sure they work effectively to treat the disease in the safest manner possible before these new therapies can be used by your doctors. This process involves clinical trials, often designed by the pharmaceutical company, and ultimate approval by the FDA if the medication shows success in the clinical trial. By sharing your views on which factors are most important to you as a patient, both the pharmaceutical companies and the FDA can make sure to focus on these factors when designing and running the clinical trial and when evaluating the results of the trial for the medication’s final approval for use in treating MN. Why does the FDA want to hear from patients? Dr. Beck: It’s important for both the FDA and the companies making these medications to understand from you, the patient, what it’s like to live with MN, what the most troubling symptoms are, what side effects or inconveniences you’ve had from prior therapies, and what you would like to see in terms of future treatments for this condition. There are many factors that the FDA considers when deciding whether or not to approve a medication for clinical use, and patient-reported outcomes are becoming increasingly important in this decision-making process. As a doctor, why do you believe the EL-PFDD is important? Dr. Jefferson: This is an exciting time in the management of MN. We’ve made major advances in the science underlying this disease in the last 10-15 years, and we now have a much better understanding of what causes MN in many patients. The key now is to translate this knowledge into the most effective treatments with the fewest side effects. To do this, we need the help of patients at an early stage in the design of clinical studies to help us understand what is and isn’t acceptable in a study and study medication, and make sure the approval process for new treatments includes the perspective of people living with the disease. In my clinic, I want to see new, evidence-based, effective, and safe treatments become available to treat my patients and alleviate the burden that this disease places on patients and their families. Dr. Beck: As a physician who treats this rare disease, the EL-PFDD gives me the opportunity to hear directly from you, the patient, about your experiences with the disease and its therapy, as well as your goals and hopes for different, better, and safer therapies. I’ve been interested in MN since I became a kidney specialist and have learned a lot from patients like you. There have been a number of exciting advances in the past few years, but it’s also clear we need to keep thinking hard and creatively about this disease and how to develop novel and more effective ways to safely treat it, keep it from causing further kidney damage, and prevent it from coming back once treated. Doctors, researchers, pharmaceutical companies, and the FDA highly value your input about these matters and your willingness to help us move forward together to meet these common goals. Register for the EL-PFDD Meeting
NephCure Kidney International Elects Longtime Volunteer Michael Levine as Board of Directors President June 15, 2021 by Kylie Winkler Levine succeeds NephCure co-founder Irving Smokler in the position Michael Levine, NephCure Board President, at Countdown to a Cure 2019 KING OF PRUSSIA, Pa. (June 15, 2021) — NephCure Kidney International (NephCure) announced today the election of longtime volunteer and board member Michael Levine as the new Board of Directors President. Levine, who joined NephCure’s Board of Directors in 2009, succeeds Irving Smokler, NephCure’s co-founder who has been with the organization since its inception in 2000. NephCure is one of the only organizations dedicated to finding better treatments and a cure for Nephrotic Syndrome and other rare, protein-spilling kidney diseases. “I’m beyond proud of all we have accomplished over the past 21 years,” said Smokler. “We’ve gone from knowing relatively nothing about Nephrotic Syndrome and FSGS to now a much better understanding of genetic causes and pathways, and we’re likely only a couple years away from having our first approved therapies. Some of this is due to the $40 million we’ve raised for research. It’s been a pleasure to be a part of this organization’s creation and success. I am confident in our future and in Michael’s leadership and passion for NephCure’s mission.” Levine has a special connection to NephCure — his son, Matthew, was diagnosed with focal segmental glomerulosclerosis (FSGS) in 2006, when he was only 2 years old. Shortly after Matthew’s diagnosis, Levine and his wife, Dana, joined NephCure in the pursuit of support and information. Before long, Levine became a dedicated volunteer, later advancing to the Board of Directors’ Executive Committee, which he’s served on since 2009. Levine takes a leading role in planning and running both Countdown to a Cure New York, NephCure’s largest annual fundraising event, and the yearly All In for a Cure golf tournament and banquet. These events have helped raise millions of dollars to support NephCure’s mission of saving kidneys and saving lives. Michael Levine with son, Matthew Levine Professionally, Levine has served as the president of L&L Painting for more than 14 years. The New York native’s deep passion for charity and helping others has also led him to join the Board of The Curetivity Foundation and actively contribute to various other charitable organizations throughout his life. “My mission has always been to help find a cure for all the warriors battling FSGS, Nephrotic Syndrome, and other chronic kidney diseases,” Levine said. “They are waiting for us to deliver a cure. We will not fail them under any circumstances — failure is not an option.” About NephCure Kidney International 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. Founded in 2000 by a group of committed patient parents, NephCure has invested more than $40 million in kidney disease research and helped create a landscape where there are now nearly 30 interventional drug trials for primary glomerular kidney diseases. NephCure is a U.S. tax exempt 501(c)(3) public charity. ###
Regulatory Update on Travere’s DUPLEX Study: Continuing on Accelerated Track June 4, 2021 by Kylie Winkler We know that many of you may have questions on the recent regulatory update from Travere Therapeutics on their sparsentan program for FSGS. We’re posting this to help you understand what it means: Travere reported that despite the DUPLEX Study achieving its interim endpoint, the FDA indicated in recent interactions that the data from the DUPLEX Study are not yet adequate enough to support accelerated approval Travere noted that FDA has acknowledged the high unmet need and the limited treatment options for people living with FSGS For anyone participating in or following the DUPLEX Study, there are no changes to report at this time. The study will continue as planned to the final endpoint after 108 weeks of treatment Travere is continuing to engage with regulators about generating additional data from DUPLEX in the first half of next year with the gold of submitting for accelerated approval in 2022 This means that sparsentan could potentially gain accelerated approval in the U.S. in 2023 Patient retention in the study is critical to providing the FDA with most robust data package possible to support a potential approval at the appropriate time Travere remains confident in the potential for sparsentan to become a new treatment standard for FSGS, if approved Travere also recently announced that their Phase 3 PROTECT Study in IgAN has completed enrollment and interim data from the trial are expected in August of this year The interim data could also lead to an accelerated approval submission for IgAN To read Travere Therapeutics’ original press release, click here. Still want to learn more about how a drug gets approved by the FDA? Here is an FAQ list to help you find the information you’re seeking. How does the FDA determine which study drugs are eligible for accelerated approval and which follow the normal regulatory timeline? The purpose of the FDA’s accelerated approval program is to get important new drugs to patients in need earlier. Generally speaking, the drug must have an impact on factors like survival, day-to-day functioning, or the likelihood that the condition, if left untreated, will progress from a less severe condition to a more seriousone. The drug must fulfill an unmet medical need, which the FDA defines as providing a therapy where none exists or providing a therapy which may be potentially better than an available therapy. How does this news impact my participation in the trial? This news doesn’t change anything about your participation in this study. In fact, it’s very important that you remain in the study. Your continued participation is the only way Travere’s sparsenten will retain the potential to be granted accelerated approval. Please continue to do everything you are currently doing and comply with all study protocol/requirements. Be sure to talk to your study doctor if you have questions. The FDA’s response to Travere’s request for accelerated approval was, ‘the available data from the interim assessment of the DUPLEX Study would not be adequate to support an accelerated approval at this time.’ Will they be able to submit for accelerated approval at a later time? Although Travere was not granted accelerated approval at this time, they may still be able to submit for accelerated approval once additional data accrue in the DUPLEX Study. Subject to further discussions this summer, those data may become available to FDA in the first half of 2022. What does this mean for Europe? Travere continues to work through the conditional marketing authorization process and anticipates meeting with European regulators this summer to discuss next steps. Subject to their upcoming discussions with EMA, Travere is still planning for a submission in Europe this year. They expect to provide an update in the coming months.