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 Karley 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 Kylie Karley 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 Karley 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 Karley 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.
Do you have Focal Segmental Glomerulosclerosis (FSGS)? June 1, 2021 by Kylie Karley Have you or your loved one been diagnosed with FSGS? We have resources for you. The rates of severe kidney disease are high in individuals who are African American, Hispanic black, Afro-Caribbean, or of African ancestry. This could be due to differences in the genetic makeup in the APOL1 gene found typically in individuals with recent African, Caribbean, or Latin American descent. These differences in the genetic makeup are associated with increased rates of hypertension-associated kidney failure, FSGS, HIV-associated kidney disease, and other forms of nondiabetic kidney disease. Fast facts about APOL1 FSGS: Black Americans account for 32% of all kidney failure in the US Black Americans are four times more likely to develop kidney failure than White Americans Approximately 4 in 10 Black Americans on dialysis have kidney failure caused by APOL1 gene changes Approximately 1 in 5 people with two copies of the APOL1 gene changes will develop kidney disease The high-risk APOL1 genotype is present in 75% of Black patients with FSGS Below are resources we’ve complied that might be useful for you: This informational flyer on APOL1 FSGS breaks down the basics and helps you better understand this disease. To download the full informational sheet, click here. In June 2020, we hosted a NephCure U session specifically on APOL1-Associated FSGS. Dr. Jeffrey Kopp from the NIDDK lead, “Kidney Disease in Patients of African Descent: APOL1-Associated Disease” and discussed more on the diagnosis, treatment options, and clinical trials. Listen in on the hour-long educational webinar below. NephCure co-hosted a GlomCon Clinical Trial Conference Series session on Advances in APOL1 Therapeutics on February 14, 2021, featuring Dr. Ogo Egbuna, Dr. Opeyemi Olabisi, and Dr. David J. Friedman. Click here to watch the recording of this session. In addition to these resources, there are also clinical trials available for APOL1-Associated FSGS patients. Clinical trials look at the safety and effectiveness of potential new treatments. The main goals of clinical trials are to find new ways to prevent, detect (find) or treat diseases or health conditions, and to make sure potential new treatments or therapies work well and are safe for people. Check out some pre-screener questions below, provided by Vertex, to see if their clinical trial could be a fit for you or your loved one. Eligible participants must meet the following criteria: Be male or female adults between the ages of 18 and 65 (inclusive) Female participants must not be pregnant or breast-feeding Be of African, Caribbean or Latin American descent Have had a kidney biopsy which has found focal segmental glomerulosclerosis (FSGS) Have not had a diagnosis of kidney disease other than FSGS Be willing to complete the investigational apolipoprotein L1 (APOL1) gene test Be willing and able to follow the study instructions To learn more about Vertex’s clinical trial, click here. If you meet the preliminary criteria listed above, find the location closest to you and click the “I’m Interested” button to get in touch with an investigator for additional evaluation of eligibility. You can find a full list of clinical trials for all protein-spilling kidney diseases on KidneyHealthGateway.com.
Chef Sachet’s Kidney-Friendly Granola Recipe May 6, 2021 by Kylie Karley Need an on-the-go snack? Want to switch up the breakfast routine? Try making some of Chef Sachet’s kidney-friendly granola. As a mother and caregiver to a young boy suffering from FSGS, her recipes are tried and true for those following kidney conscious diets. Granola Recipe Prep Time: 5 min Cook Time: 30 min Yield: A LOT! Ingredients: 6 cups oats 3 cups toasted shaved almonds 1 1/2 cups toasted sunflower seeds 1 cup unsweetened toasted coconut flakes 1 cup plumped raisins, dried cranberries 1 cup pure maple syrup 1 cup honey 1/2 cup coconut oil 2 tsp ground cinnamon (optional) Instructions: In a large bowl, toss oats with honey, syrup and oil. Lay onto a non-stick baking sheet and toast in the over at 375 degrees for 20 min (or until oats are toasted and stiff). Be careful as to not over toast the oats as they will burn and turn bitter! Let oats cool completely, then toss with other ingredients. Store in a sealed cereal container or mason jar. Important tips: Nuts (always use unsalted) provide a great source of protein for those trying to stay away from high phosphates in meat. Add some m&m’s and dried apricots and you got yourself a yummy trail mix! Add some yogurt, fresh fruit, and a drizzle of honey and you got yourself a delicious breakfast alternative!
An Inside Look: Precision Medicine, Podocytes, and Goldfinch Bio April 30, 2021 by Kylie Karley Goldfinch Bio is a biotechnology company based in Cambridge, MA which focuses on advancing kidney precision medicine. Its mission is to deliver disease-modifying precision medicine that brings hope and renewed quality of life to people living with kidney diseases. We recently spoke with Goldfinch Bio’s Dr. Liron Walsh, Vice President Clinical and Translational Nephrology, who explains more about the company’s precision medicine approach, breaks down what podocytes are, and speaks more on its Phase 2 clinical research trial, TRACTION-2, which is currently enrolling patients. Dr. Liron Walsh, Vice President Clinical and Translational Nephrology, Goldfinch Bio What is precision medicine? The causes of kidney disease are unique among groups of individuals. In diseases such as focal segmental glomerulosclerosis (FSGS), the causes of disease can vary from genetic to environmental. However, current treatments for FSGS are the same regardless of the cause. With these “one-size-fits-all” approaches, some patients respond to treatment, while others do not. Many patients experience harmful side effects from these treatments with little to no benefits. The goal of precision medicine is to develop treatments which are specifically designed to treat the underlying cause of an individual’s kidney disease. In this way, patients can receive the right medicine and avoid treatments which are not going to be helpful or may even be harmful. Today, precision medicine is often used to treat cancer. By first understanding the genetic makeup of a patient’s tumor, a doctor can then choose the best medicine to specifically treat that patient’s cancer. This approach has greatly improved the lives of many people with cancer. Precision medicine is now on the horizon for people with kidney diseases. Patients, nephrologists, and kidney researchers are working together to better understand the causes of kidney diseases. Through this deeper understanding, precision medicine will help develop treatments that target the specific causes of kidney diseases like FSGS and minimal change disease (MCD). Precision medicine holds promise to improve treatment outcomes and quality of life for many people living with kidney disease. Goldfinch Bio and Precision Medicine Goldfinch Bio is developing a novel experimental medicine, GFB-887, with the hope of preventing the progression of kidney disease in patients with FSGS, treatment-resistant MCD (TR-MCD), and diabetic nephropathy. While these kidney diseases may seem very different, they have something very important in common– very specialized cells of the kidneys called podocytes are damaged. GFB-887 is designed to specifically target these podocytes and prevent damage. What are podocytes? Podocytes are very specialized cells that wrap around the blood vessels of the kidneys. They form a network with other nearby podocytes to prevent the body from losing important proteins, such as albumin, while at the same time, filtering out waste. Normal functioning podocytes are very critical to our health. When podocytes are damaged, gaps form in the barrier and important proteins are lost in the urine. This is commonly referred to as ‘spilling protein in the urine,’ and is an early sign of kidney disease. Patients with damaged podocytes experience swelling, weight gain, and fatigue. Unfortunately, damaged podocytes cannot heal, and they cannot be replaced by new, healthy podocytes. The kidney tries to repair the damage by creating a scar, much like the scar from a cut. Over time, more damage and more scarring will lead to kidney failure. By protecting the podocytes from being damaged in the first place and ensuring that the podocytes can function normally, it may be possible to prevent or delay kidney failure. What makes the Goldfinch Bio team different than other pharmaceutical companies? First, we are focused on advancing treatments for people with kidney diseases. Second, we are pioneering precision medicine for kidney diseases. While precision medicine is often used in cancer, and some rare genetic diseases as well, they are an entirely new area of drug research and development for kidney diseases. We are deeply committed to bringing innovative treatments to people with kidney diseases and working very closely with the patient community as partners. Goldfinch Bio’s tagline is ‘Saving Kidneys, Ending Dialysis.’ How did you come up with this? Our tagline– Saving Kidneys, Ending Dialysis– is a shortened version of our vision, which is “We aspire to save kidneys and end dialysis.” We know that for many patients, kidney transplant and dialysis are the only treatment options. Unfortunately, kidney transplant may not be an option for many patients and is associated with significant long-term complications, and the experience of dialysis is very difficult. Through our precision medicine approach, we want to bring new, effective treatment solutions to patients so that we can give them hope and a renewed quality of life. We finalized our vision by hosting a focus group with people living with kidney disease to really understand what was important to them. A resounding theme emerged: these individuals expressed to us that they would give anything to avoid dialysis. And, from there, our new vision emerged. Can you tell us more about your ongoing clinical trial? The TRACTION-2 clinical research trial is evaluating our investigational precision medicine, GFB-887, for the potential treatment of FSGS, TR-MCD, and diabetic nephropathy. The purpose of the trial is to determine if GFB-887 is safe and may help people who have high levels of protein in their urine due to kidney diseases caused by podocyte injury. For more information on the trial, you can visit tractionclinicaltrial.com or at Kidney Health Gateway. What are you most looking forward to at Goldfinch Bio? We are very excited about the recent creation of our FSGS Patient Advisory Board. We have created this board to foster a close working partnership with the patient community. For National Kidney Month, we held a virtual panel discussion with our board members. You can learn more about our Patient Advisory Board and watch the panel discussion here. We are very excited to continue to collaborate with this Board to ensure we have a patient-centric approach and mindset throughout our company. To learn more about the TRACTION-2 clinical trial, click here. To see a full list of clinical research opportunities, and to find the trial best for you or your loved one, visit KidneyHealthGateway.com. This article was developed in partnership with Goldfinch Bio, Inc.
A Day in the Life of a Nephrologist: 2021 NephCure Patient Summit April 20, 2021 by Kylie Karley Have you ever wondered what it might be like to walk in a kidney specialists’ shoes for a day? Get a rare peek ‘behind the curtain’ and hear from actual nephrologists about their perspective on treating rare kidney patients, some of their daily challenges, and tips for making the most of your appointments. Join us Sunday, May 16th from 12-4pm ET for the 2021 NephCure Patient Summit. During this FREE virtual gathering of patients from around the globe, you’ll hear about the latest in research and treatments and learn about your disease. You’ll also have the opportunity to directly ask our experts questions and participate in interactive community roundtables. The “Day in the Life of a Nephrologist” panel will feature Drs. Jason Cobb, Michelle Rheault, and Suneel Udani. The Patient Summit will also offer you the chance to hear from other NephCure specialists, NephCure staff, and regional volunteer leaders. Meet the Panelists: Dr. Jason Cobb is an assistant professor of medicine in the Emory University Division of Renal Medicine. Dr. Cobb is a graduate of Morehouse College with a BS in biology. He holds a medical degree from Emory University School of Medicine and completed his internal medicine residency and nephrology fellowship at Emory University. He is board-certified in internal medicine and nephrology. He sees patients in nephrology clinic at Emory University Hospital Midtown in the Medical Office Tower seeing patients with nephrology complaints such as chronic kidney disease due to diabetes, hypertension, vascular disease, and glomerulonephritis. Also, Dr. Cobb takes care of hemodialysis patients at Emory Dialysis Greenbriar and Emory Dialysis Northside, and home dialysis patients at Emory Dialysis Northside. He also rounds on inpatient services at Emory University Hospital Midtown and Emory University Hospital. Research and teaching interests include quality improvement and nephrology fellow clinic at Grady Memorial Hospital. Dr. Michelle Rheault is a board certified pediatric nephrologist and Associate Professor of Pediatrics. She completed her Pediatric Nephrology fellowship at the University of Minnesota in 2006 followed by a research fellowship at Mount Sinai School of Medicine in New York City. She joined the faculty at the University of Minnesota in 2008 and is currently the Director of the Division of Pediatric Nephrology and Medical Director of the Pediatric Dialysis unit. She is on the steering committee of the Pediatric Nephrology Research Consortium, a clinical research network that aims to facilitate collaboration in pediatric nephrology. Her clinical and research interests include Alport syndrome and other genetic kidney diseases, pediatric glomerular disease, and pediatric end stage kidney disease. Dr. Suneel Udani is a native of Chicago’s western suburbs. He joined Advanced Renal Care in July 2011. He completed his undergraduate, Master’s in Public Health, and medical school degrees at Northwestern University and went on to do his Internal Medicine training at the University of Chicago and the University of Pittsburgh. He served as a Chief Medical Resident at Cook County Hospital prior to returning to the University of Chicago for his fellowship in Nephrology. Dr. Udani is the author of multiple peer-reviewed journal articles and has presented his research at national conferences and is the author of multiple text book chapters on diagnosis and management of kidney disease. His focus is on glomerular disease and heart-kidney interactions. He is also a clinical investigator for community-based renal research program and a dedicated educator and advocate for patients with kidney disease. To learn more about the 2021 NephCure Patient Summit, including a full list of speakers, and full event agenda, click here.