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Meet Central Florida Regional Leader: Juli Millas

September 2, 2020 by Kylie Winkler

Juli Millas is an FSGS patient living in the Central Florida area, who was first diagnosed in July 2002. She has built a strong sense of community within her region and is passionate about supporting others with rare protein-spilling kidney diseases. While she typically meets up with other local families at Tampa Pig Jig every year, she’s getting creative and adapting due to the pandemic cancelling the 2020 event. Learn what she has planned instead for her community!

What’s your personal connection to NephCure?

I discovered NephCure about seven years ago after attending the Tampa Pig Jig. As an FSGS patient, I couldn’t believe it; I had found a community of people just like me that I had no idea existed! It was nirvana! I found my tribe! When NephCure was looking for volunteers, I knew I had to get involved and I jumped at the opportunity to be a part of them. Being the patient engagement and support volunteer, as well as the regional leader, for Central Florida brings me such joy, pride and satisfaction. I know that I’m a part of something that is making a difference for Nephrotic Syndrome patients.

Why do you volunteer with NephCure?

Juli and other Nephrotic Syndrome families at Tampa Pig Jig.

I’m committed to volunteering for NephCure because I don’t want anyone with Nephrotic Syndrome to ever feel as desperate, lonely, and isolated as I did when I first got sick. I want everyone to know that NephCure is here for them with information, support, and most of all community!

What projects are you working on right now?

The two projects I’m currently working on came to fruition because of the Tampa Pig Jig being cancelled due to COVID-19 this year. Pig Jig is a huge fundraiser for NephCure and it’s a place where patient and patient families meet up every year. It just made sense to do a picnic and fundraiser. So, I am planning a one for the patients and patient families who normally meet up at Pig Jig. And since the folks at Pig Jig will match our donations up to $100,000, I am doing a small fundraiser and I am calling it “The Pig Jig Initiative.”

NephCure is local to you. To learn more and get involved with your regional volunteer community, please click here. 

Filed Under: Recent News, Uncategorized

Meet New York Regional Leader: Marlene Botta

September 1, 2020 by Kylie Winkler

Marlene, her daughter, Jacqueline, and husband (L-R).

Marlene Botta is a patient parent in the New York City area. Her daughter, Jacqueline, battles Nephrotic Syndrome. Marlene has done a tremendous amount of work at the state capital in Albany, even securing March 27th as “Nephrotic Syndrome Awareness Day” throughout New York every year. She’s lead the Long Island Walk multiple years and constantly fights for better treatments for those with Nephrotic Syndrome and FSGS. You can contact Marlene at nephcurenewyork@nephcurevolunteer.org.

What’s your personal connection to NephCure?

My daughter was diagnosed with Nephrotic Syndrome in 2016. NephCure has saved my life and my sanity. Without an outlet for my pain, I would have never gotten myself out of bed some mornings. It was a way for me to turn a horrific moment into triumph.

Where do you focus your volunteer efforts with NephCure?

My family and I do a lot of legislative relations in New York state. I speak to large groups about what NephCure does and try to get legislative initiatives to create awareness and raise funds. In addition to that, we do the Long Island NYC walk, which brings together our community and local families and creates a support system for all.

What projects are you working on right now?

I’m currently figuring out how to do my outreach in this post-Covid world. But, I always feel like I’m coming closer to creating a better life for those families afflicted by this insidious disease.

NephCure is local to you. To learn more and get involved within your regional volunteer community, please click here. 

Filed Under: Recent News

Stop Proteinuria, NephCure Provides Resources to Broader Nephrology Community

August 24, 2020 by Kylie Winkler

In recent years, we have seen Patient-Centered Outcomes, also known as Patient-Reported Outcomes (PROs), be implemented more regularly, specifically in the drug development and approval process. In fact, the Food and Drug Administration (FDA) created a Patient-Focused Drug Development Program specifically aimed at learning about how diseases impact patients’ lives and what an ideal treatment would look like from both patient and caregivers’ point of view. NephCure and our Voice of Patient volunteers continually invest their time to participate in various PRO projects, including Externally-Led Patient-Focused Drug Development Meetings with the FDA, patient and caregiver interviews, panels, and advisory boards with industry partners. These projects have led outside stakeholders towards developing deeper insight into patient priorities and have even played a part in making clinical trial protocols more patient-friendly.

Through daily interactions with glomerular disease patients and caregivers, NephCure consistently sees one very concerning trend. Patients don’t always understand, and many nephrologists aren’t consistently explaining, the correlation between proteinuria and decreased kidney function. The article in the May 2020 edition of the Clinical Journal of the American Society of Nephrology, “Identifying Outcomes Important to Patients with Glomerular Disease and Their Caregivers” clearly confirms this concern. The top 3 most important outcomes among patients include:

  1. Kidney function
  2. Mortality
  3. The need for dialysis or kidney transplant.

However, patients’ consideration of proteinuria ranks twenty-third in priority.

Over time, chronic proteinuria leads to kidney failure.1, 2 Proteinuria results from a dysfunction in the filtering units of the kidney. If a medication can treat that dysfunction, protein spilling into the urine will stop and the filtering units of the kidney (or glomeruli) will remain healthy. However, if protein continues to spill, the glomeruli will begin to scar, causing permanent damage and eventually a reduction in kidney function.

In our professional capacity here at NephCure, the “home” for all individuals who are affected by primary protein-spilling kidney diseases, we have heard too many firsthand accounts of adult patients who have been diagnosed with glomerular disease and whose nephrologist does not treat the proteinuria. Instead, tragically, these patients’ labs are monitored once or twice a year, and they are told to come back when it’s time for dialysis and/or transplant.

NephCure is working to change this practice by educating both the nephrology community and patient families that the number one treatment goal of any glomerular disease should be to stop proteinuria or reduce it as much as possible, which will in turn slow down the progression of the kidney disease and hopefully, in some cases, prevent the need for dialysis and/or transplant.

To that end, we have developed a new patient-focused brochure aimed at educating newly-diagnosed patients and families about the importance of reducing proteinuria in order to maintain their kidney function. We are seeking a collaboration with the broader nephrology community to distribute these at point of care and help NephCure provide support and resources to the primary glomerular disease population.

Kelly Helm, Assistant Director, Patient Advocacy

If you are interested in supporting us in this endeavor, you can request patient materials here. Thank you for your interest in helping us reduce proteinuria, save kidneys, and save lives.


Kelly Helm
Assistant Director, Patient Advocacy

 

 

 

1 Troost JP, Trachtman H, Spino C, et al. Proteinuria Reduction and Kidney Survival in Focal Segmental Glomerulosclerosis [published online ahead of print, 2020 Jul 23]. Am J Kidney Dis. 2020;S0272-6386(20)30846-5. doi:10.1053/j.ajkd.2020.04.014 https://www.ajkd.org/article/S0272-6386(20)30846-5/fulltext
2 Troost JP, Trachtman H, Nachman PH, et al. An Outcomes-Based Definition of Proteinuria Remission in Focal Segmental Glomerulosclerosis. Clin J Am Soc Nephrol. 2018;13(3):414-421. doi:10.2215/CJN.04780517 https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5967666/

Filed Under: Recent News, Uncategorized

An Inside Look: Vertex’s Study in APOL1-FSGS

July 29, 2020 by Kylie Winkler

We recently sat down with Dr. Ogo Egbuna, Vertex Pharmaceutical’s clinical development lead for their clinical trial in APOL1-mediated focal segmental glomerulosclerosis (FSGS) to learn a bit more about their program and why it’s exciting for the disease community. Dr. Egbuna is a board-certified nephrologist with a deep interest in FSGS and he provided some insight into the importance of the trial and what potential participants should know if they’re interested.

Dr. Ogo Egbuna

The purpose of the study discussed below is to evaluate the safety, tolerability and effectiveness of an investigational medication in individuals with APOL1-mediated FSGS. The word “investigational” means this medication is not approved for use by the Food and Drug Administration (FDA) in the United States or other regulatory agencies in the UK, Europe or elsewhere.

How is this drug different than other treatments currently in use for FSGS?

As many of you may know, there are many different causes of FSGS. At Vertex, we’re focused on FSGS mediated by variants of the APOL1 gene. With our investigational small molecule treatment, we are aiming to target the underlying cause of disease by inhibiting the APOL1 pathway.

Why is a drug for APOL1-mediated FSGS needed?

A key part of our strategy at Vertex is to work on diseases where there is a high unmet need for treatment. The current treatments do not address the underlying cause of disease.

Let’s assume that you are able to successfully recruit participants in this study and the drug proves to be effective. What is an estimated or typical timeline for when we could see this drug available on the market?

Drug discovery and development is a long process, but we’re working as quickly as possible to do the things needed to determine whether this potential therapy has an acceptable risk/benefit profile for patients and if so, we’ll work with regulators to bring it to people who need it as soon as possible. At Vertex, we focus on serious diseases where we can have a transformative impact for patients, not just an incremental benefit. Rather than looking for problems we can solve with only the tools we’ve used before, we figure out the problems that need to be solved for the diseases we’re going after and invent the tools to potentially fix them.

Who is this study for? 

We are excited to work with and grow our relationships with the FSGS community. This study is for adults of African or Caribbean descent (ages 18-60) with two APOL1 gene variants and biopsy confirmed FSGS. For more information on this study, please visit FSGSResearchStudy.com

Do I have to live near a study site to participate?

We are committed to designing our trial to be as easy for participants as possible, and we have taken the participant considerations into account each step of the way. With that in mind, we’re excited to incorporate telemedicine and participant choice into our clinical trial process.

You do not need to live near a study site to participate in the Vertex APOL1-mediated FSGS study.  Only your first screening visit needs to be in person; after that, all visits can be done from home, at the study site or a combination of the two.  It is your choice.  For home visits, a home health company will provide a nurse to visit your home to collect information and perform required tests. You’ll be able to complete a telemedicine phone call or video visit with the study doctor, and the study drug can be shipped right to your home. For visits at the study site, Vertex provides travel assistance that will support costs and arrangements.

How will I know if I have an APOL1 gene variant?  

As part of the Vertex APOL1-mediated FSGS study, you will be tested for APOL1 gene variants.

What does it mean if I test positive for the APOL1 gene variant?

This means you have a genetic variant that increases your risk of developing kidney disease and accelerates the progression of kidney disease.

Can I be in remission from proteinuria and still participate in the study?

Patients who are in remission are not eligible for this study.

Can I be on dialysis?

Patients currently on dialysis are not eligible for this study.

If I need a biopsy to participate in this study, will my insurance cover it? Would Vertex cover it?

The study is enrolling participants with a previously confirmed FSGS diagnosis.

This article was developed in partnership with Vertex Pharmaceuticals.

Filed Under: Recent News

NephCure Kidney International Promotes Lauren Lee to Executive Vice President, Stakeholder Engagement

June 23, 2020 by Kylie Winkler

KING OF PRUSSIA, PA (June 23, 2020) – NephCure Kidney International® announced today the promotion of Lauren Lee to Executive Vice President, Stakeholder Engagement, effective immediately. In her new role, Lee will oversee the departments of Research & Engagement, Community Development, and Operations. Lee will continue to report to NephCure Chief Executive Officer Joshua Tarnoff.

Lauren Lee, Executive Vice President, Stakeholder Engagement

In her prior role as Chief Research Officer, Lee transformed the Department of Research & Engagement to reach a broad array of stakeholders, including patients, healthcare providers, researchers, government entities, biopharmaceutical companies, and other like-minded NGOs. She recently led the mission-critical Gateway Initiative that now involves nearly 30 primary glomerular kidney disease clinical trials across the globe and balances a wide number of collaborators. To date, no company has left the space due to recruiting difficulties: the hallmark charge and success gauge of the initiative.

“From day one, Lauren has displayed incredible compassion for patients and tireless dedication to our mission while handling multiple consuming programs,” Tarnoff said. “Her strong leadership and strategic vision in adapting to evolving environmental demands and driving key programmatic successes made her the clear choice both internally and externally when considering candidates for this large and critical new senior level role. I’m thrilled to announce her promotion at a time when our patient community is in urgent need of additional support due to COVID-19 and an influx of clinical research opportunities.”

Lee has worked for NephCure since 2013, starting as Director of Grassroots Operations before spending 6 years directing the Research & Engagement Department. For many years, she led NephCure’s efforts to build a nation-wide network of patient families, clinicians, and researchers, over the years traveling to nearly every major and minor city within the NephCure community. More recently, she has played a key role in effectively steering NephCure into a powerful clinical research facilitator and collaborator within the glomerular kidney disease space.

“It is truly a privilege to be part of an outstanding organization like NephCure,” Lee shared. “We have only just begun to hit our stride in making a material difference in the lives of the patients we serve through meaningful initiatives like Gateway, the NephCure Specialist directory, and our Regional Volunteer Communities. In this new role, I hope to continue the momentum with a keen focus on mobilizing all stakeholders around our mission of bringing new treatments to patients.”

Lee earned a Bachelor of Arts in Anthropology and French from Carleton University in Ontario, Canada and a Master of Arts in Museum Studies from the State University of New York at Oneonta. She will remain based in the Philadelphia area.

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.

www.NephCure.org

Contact: Lauren Eva

610-540-0186 x21

Lauren.Eva@NephCure.org

Filed Under: Recent News

Finding the Beauty in Kidney Disease: Keyaira Sanders’ Story

June 8, 2020 by Kylie Winkler

“When I was 10 years old, I remember being at a family gathering and feeling absolutely horrible. For the life of me, I couldn’t figure out why I wasn’t feeling well, but I just wanted to feel better. The first sign that I noticed as a ten-year-old little girl was the puffiness around my eyes.

My parents started treatment with allergy medications for approximately a week, but as the days progressed, I began to feel worse. I noticed swelling all over my body, and I also noticed that I couldn’t urinate as much as I usually would. My mother then took me to my pediatrician; I still can picture the puzzled look upon her face when she examined me. She explained to my mother that this was far beyond allergies, and that she should take me to Le Bonheur Children’s Hospital in downtown Memphis.

After seemingly endless tests and blood and urine samples, one of the pediatric nephrologists came in and explained I had Nephrotic Syndrome.

He told us that this was a very rare disease and that I would need a kidney biopsy to determine what form I had. As a little girl, I had no clue what any of this meant.

The doctor made sure to immediately start me on a high dose of Prednisone (steroids) and Lasix (water pills) to help my kidneys get into remission and drain some of the fluid out of my body. The Lasix worked—I was up the majority of the night urinating!

The next day, I was scheduled to have my kidney biopsy. I was terrified—all I could think of was the huge needle going into my back. As the time drew nearer, my heart felt as if it was about to explode through my chest. Teddy bear in hand, I was taken back to the operating room.

The doctor told me to relax and that I was going to be given some “happy medicine.” The next thing I knew, I woke up back in my hospital room. I was in a bit of pain, but it wasn’t as bad as I had imagined.

After being in the hospital for almost five days, I was still receiving steroids daily. I began to notice my appetite increasing, and I later learned that this was one of the common side effects of Prednisone.

A few more days went by, we discovered I had Minimal Change Disease. Nephrotic Syndrome can be a very tricky disease, because everyone is different, and we all respond to medications differently. For me, the steroids were a go! I was able to take the high-dose steroids partnered with blood pressure medications (due to the steroids increasing my blood pressure as another side effect) and Lasix for the fluid.

After being in the hospital for a month and a half, I was finally able to go home and continue my treatment. I was so happy to finally be going home and going back to normal. Little did I know, this was only the beginning.

Fast forward to my sophomore year of high school. I remember being at after school band practice and being in so much pain. My upper back was hurting me on the left side, and I was very short of breath. I managed to get through the entire practice, but I knew something wasn’t right. I got home that night and I didn’t even want to eat; I sat straight up the entire night, just breathing. I remember crying all alone, thinking “I don’t know what this is, but I need to seek some kind of help.”

My mom took me to the emergency room the following day, and they told me that I had pneumonia. They didn’t check anything concerning my disease at all and sent me home with absolutely nothing. When we got home, I still couldn’t even hold down water!

My mother had had enough and called my father from out of town to take me back to the hospital in Memphis for another opinion. They rushed me to the back and began testing immediately. One test I couldn’t even do, because it required me to lie down flat, and I was in too much pain.

I cried all the way there and while we were there. I really broke down when a team of doctors came swarming into the room, asking my father to leave and saying that they had to start treatment right away because my case was very severe. I was only 15 at the time.

I remember hearing the words ‘pulmonary embolism.’ I was puzzled, but later found out that I had a blood clot in my left lung.

They had to begin treatment right away because this was life-threatening. I was placed on a ventilator for about a week, because one day I was trying to get up and blacked out.

There’s not much more that I remember about that experience, but the doctors told my parents that I was very lucky to be alive. Looking back, I am beyond grateful and I understand my purpose.

More time has passed. I am now 22 years old, and I look back at all of the experiences that I have had with this disease—the good and the bad. It may sound crazy, but I wouldn’t change anything that has happened to me. Having Nephrotic Syndrome has not only helped me to grow into the person I am, but it’s also helped me to appreciate life. I know from experience this disease is NOT easy at all; it can be painful and take a huge toll on you and those around you mentally, physically and emotionally.

Often times people assume that just because you may be in remission or back to normal physically, you must also be okay mentally and emotionally. But I’ve battled with depression because I felt as if I wasn’t normal.

I’ve had to miss tons of school days, my parents have had to miss work, I’ve had to try different medications through trial-and-error, I’ve had to give myself blood-thinner injections, and recently I’ve had to start Rituxan infusions—this has been a rollercoaster.

Living with this disease is very hard because you always have that “what if,” or that fear that you’ll relapse and have to go through it all again.

It’s a shame to say, but I’ve had doctors not even know how to treat me properly, and this is one of many issues that needs to be highlighted. This is why I am taking a stand: to be a voice to help others who may not feel comfortable sharing their stories. Hopefully me taking this stand inspires so many others to share their stories as well.

I hope whoever reads this finds some kind of positive message and holds on to that for hope. We are all in this together! A saying that I came up with myself and live by is this:

“While we all have something ugly that we go through, we must find the beauty in it and allow it to shape us into the person we are destined to be in life.”

So, no matter what you may be going through, just remember that we all have something ugly, but it’s up to us to bring out the beauty. What’s the beauty in your story?”

– written by Keyaira Sanders

Filed Under: Recent News, Uncategorized

The Trauma of Transplant: Jeremy’s Path to Wellness

May 26, 2020 by Kylie Winkler

“At 14, I had just finished my first year of high school. I played varsity soccer as a freshman, and I was getting ready for one of the most important summers of my young soccer career. But my life took a different course, and from ages 14-24, I had to completely focus on my health. After my first blood draw–I received my diagnosis of FSGS.

During those initial years with FSGS, I did multiple different treatments, including high-dose IV steroids and chemotherapy. I’ve had two kidney transplants and had both my native kidneys removed. I had a staph infection that caused my first kidney to fail, and I have done a total of 4 years of different kinds of dialysis, as well as at least 20 other procedures. Through that time, managing my chronic kidney illness as part of everyday life, my family wondered, “How are you handling this so well?” I would unknowingly shrug and give a cliché line about needing to move on to the next thing.

Two years later, after numerous treatments and six months of dialysis, I received my first transplant. By 21, my body had rejected the kidney, and after three and half years on dialysis, I received my second kidney at 24 from a family member. I was a rock–in survival mode–for those ten years. Nothing fazed me. In times of pain or frustration, I would be upset but subdued. Once those moments would pass, so would the thoughts about them. It always looked and felt like I was doing great, and I was so strong that not even this illness was going to weigh on me.

Everyone’s perception of me was that I was handling this difficult part of my life well. I didn’t realize until about a year ago that the pain, although I wasn’t thinking about it, was still affecting me.

Jeremy and his kidney donor.

Three and a half months after my second transplant, I traveled for the first time in four years without a dialysis machine. I was ecstatic! My brother was playing in his first away college soccer game for Sacramento State, and I was going to get to see him play. For my brain, it was the first quiet moment it had in my adult life. I was sitting in my friend’s bedroom in LA the day before the game, and nobody was home. I couldn’t stop thinking about the pain of my second transplant. I felt trapped.

Then, as I sat on the bed with my legs stretched out and a blanket over them, TV in front of me, the way I had in the hospital, the bedroom seemingly turned into a hospital room; the house became the 8th floor transplant ward at UC Davis. I stayed in this state for almost eight hours until my friend walked in the door. I was numb. I had relived the entire day in a hospital room. I now realize that this was my first time experiencing severe Post-Traumatic Stress Disorder symptoms.

This incident confused me, and when I got back home, I started to do some research. It didn’t take long to find out that post-transplant patients, while having lower rates of depression than pre-transplant patients, have significantly higher rates of depression than the general population.

Was it possible I could be depressed? A wave of guilt washed over me. I was depressed after receiving one of the greatest gifts of my life.

Sure, I had three surgeries: transplant, peritoneal dialysis catheter removal, and a biopsy. I was not able to return to work or school. I was in pain most of the time from the recovery from the surgeries and muscle atrophy. I knew it was going to be difficult, but now it felt as though I had lost control over my mind. That mind, my support group, and my grandma’s home-cooked meals were what got me through all of this. Now, it felt as though I was trapped. I understood this amazing thing had happened to me, yet I was simultaneously reliving the most difficult moments of my past ten years – the moments that I never would wish on anyone.

Since I had discovered it’s so common for transplant recipients to experience PTSD, depression and anxiety, I thought surely my transplant team at the hospital would have a plethora of resources available to help me. This, unfortunately, turned out not to be the case.

In my next appointment with UC Davis, I talked to a social worker. That was the most UC Davis had to offer. When I asked for the opportunity to talk to a therapist, they told me that they didn’t have one for me to see, and none to recommend outside of the hospital. The social worker then informed me they wouldn’t be able to see me regularly. I was on my own to find a therapist who was qualified to help in what felt like a very specific need.

I was astonished that one of the top kidney transplant facilities in the nation didn’t offer any mental health services post-transplant. This started a year-long journey looking for somebody with experience in chronic illness– someone who could help with the mental health struggles I was facing. I set up one appointment with a trauma specialist that my family recommended. During our visit, they told me they believed I had PTSD. At first, it was hard to accept. I did not believe them. I thought that was only for war vets and people who had gone through something truly traumatic. That wasn’t me.

While my search for support continued, my mental health became more of a challenge. About one month after my first incident in Los Angeles, I learned that a player I coached in soccer was diagnosed with a rare auto-immune disease (unrelated to the kidneys). This news sent me into a severe depression that I was not equipped to get out of. When I finally overcame it, I knew–yet again–that I was in need of professional help right away.

I set up a series of initial appointments with different therapists and went through a frustrating time. I had a hard time finding someone who took my insurance and who also had experience working with patients that had chronic health conditions and experienced the same mental turmoil that I had. My diagnosis of PTSD took a long time for me to accept, and even longer for me to begin meaningful treatment.

I felt a lot of shame about the idea of my kidney transplant being labeled as a trauma. This was meant to be the solution to years of battling a difficult illness and now it felt like an extended part of the illness, which left me with a feeling of failure.

What I didn’t know for the first ten years was that the “rock” that I had been was a defense mechanism–a way for me to survive the trauma in my life. I had just simply avoided processing it. This is why, after my second transplant, the pain that I hadn’t processed before was emerging in a way that I had very little control over.

I got my first transplant eight years earlier, when I was 16 years old, but I lost it to a staph infection that put me in the hospital for three weeks and nearly killed me. I’d done a total of four years of dialysis before the two transplants (six months before the first transplant, and three and a half years before the second). I have done chemotherapy, high-dose IV prednisone, and a number of other treatments over the past ten years.

The second kidney transplant was the tip of my PTSD iceberg, which I had ignored for all too long. I received the transplant on July 3, 2017. I stayed in the hospital for seven days after and in a hotel close by for another week after that. For four days in the hospital I had high fevers because of the immune suppressants that I was on. I had eight different IVs because of the iron treatments I was receiving. I had blood drawn every morning at 5 a.m. They removed a nerve block in my side while I was awake. It gave me terrible leg twitches.

When I got to the hotel, I learned that the surgery had damaged a nerve near the incision, causing shooting pain in my groin anytime I tried to lay down past a 45-degree angle. Six weeks later, right when I was walking normally and getting into a regular sleeping routine, I had a surgery to remove my peritoneal dialysis catheter. It left me in bed for three days post-surgery. Another four weeks after that, I had my first kidney biopsy. This was the most minor procedure, but it still left me with some discomfort for about a week. My point is this: transplants are hard, long and invasive. Surgeries in and of themselves are traumatic, regardless of their purpose. Dialysis and treatments for kidney diseases are inherently traumatic for the entire support group, not just the person experiencing it directly.

I’ve been so fortunate to have a comprehensive and almost global support group: my entire extended family, my inner circle, my two donors, friends, work colleagues, NephCure, COTA, nurses and doctors (some of whom I still stay in contact with), and so many people that I have only met once or twice. All of them have been incredible and they are the reason why I am the person I am today (and as mentally fit as I am). I have seen the toll this takes on them. My entire immediate family and I are all in therapy now because of the difficulty of this process–not just because of the second transplant. I think we all wish that we started therapy earlier, and together. I had very little therapy in the eight years leading up to my second transplant.

The therapist I see now once told me, “People avoid or stop going to therapy when they need it the most.” For a long time, I turned a blind eye to therapy.

I did as much as I could to avoid processing the extreme difficulty I was facing in my life. I did this until I was in so much mental pain that I was forced to take action.

Don’t get to that point. Wherever you are in this journey, and quite frankly, if you’ve read this far, simply talking to somebody who has no connection to you and an expertise in mental health is something you need. You cannot and do not need to handle this on your own. Your support group cannot and does not need to handle this on their own.

Even if you do not have PTSD, depression, anxiety, or anything of the sort, therapy can help you process the difficulties we all face from being forced to be kidney warriors. Caring for your mental health can help you live a more balanced life–a life filled with joy.”

-Jeremy Bedig

Filed Under: Recent News

You Can Change FSGS Treatment Options: EL-PFDD 2020

April 28, 2020 by Kylie Winkler

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. The goal is to hear from patients who have the disease, in order for the FDA and pharmaceutical companies to understand the patient experience.

This year, NephCure Kidney International and the National Kidney Foundation are coming together to conduct an EL-PFDD meeting, on August 28, 2020, to inform the FDA about the patient’s perspective of living with focal segmental glomerular sclerosis (FSGS). The meeting will be held online. We invite anyone who has FSGS, lives with someone affected by it, or is interested in it to attend this meeting. 

This year’s EL-PFDD meeting on FSGS will be co-chaired by two NephCure Specialists, Drs. Laura Mariani and Suneel Udani. The two of them answer the following questions regarding the importance of patient attendance at the meeting in August.

 

As a patient with FSGS, why should I consider attending this meeting? 

EL-PFDD co-chair and NephCure Specialist, Dr. Suneel Udani

Because FSGS is rare, most people designing and evaluating the results of clinical trials, that test new medications, have not met anyone with FSGS.  While clinicians taking care of patients with FSGS understand the impact the disease has on your lives, this impact is not something that can always be captured on a blood or urine tests, and therefore regulators who determine which medicine becomes available 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 not only what you would not only like, but also what you would expect from new treatments in terms of how they are administered, for how long the medication must be taken, the side effects, or anything else you think would be relevant to new treatments.  Each segment of the medical community (clinicians, researchers, industry leaders) benefits from hearing your perspective so that the new treatments available truly meet the needs of the intended population—you.

The EL-PFDD meeting is a unique setting where all these groups—patients, clinicians, scientists, industry, and regulators—are gathered together to listen. Therefore, your attendance and insight are dependent and vital to this meeting’s success.

 

How does this meeting contribute to putting new medications for FSGS on the pharmacy shelf?

EL-PFDD co-chair and NephCure Specialist, Dr. Laura Mariani

The comments from the meeting can inform the FDA about the urgent need for new, more effective, and less toxic treatment options for FSGS. The FDA tries to bridge communication between patients and the medical community. The FDA aims to develop criteria that not only represents the effectiveness of therapies, but also determines if these therapies truly help people feel better. In gathering patient feedback, this meeting will help companies design better studies to assess the symptoms most relevant to patients and help the FDA interpret the results of those studies with the patient’s needs in mind.


Why does the Food and Drug Administration want to hear from patients?

The FDA recognizes that patients and their family members are the experts on what it is like to live with FSGS and to take the currently approved medications.  This meeting can help the FDA better understand the impact of the disease and its treatments on patients.

 

As a doctor, why do you believe the EL-PFDD is important?

“As a physician, I have been frustrated with the treatment options for Nephrotic Syndrome since I learned what they were, how effective (or not) they are, and the side effects we ask patients to tolerate. However, for the first time in our history, there are more candidates for treatments and studies to investigate their impact. The potential for multiple new therapies has changed my perspective. Whereas in the past our bar for being excited for a new treatment was simply if it made an impact on abnormal lab tests, now we have to raise the bar to not only look at improving laboratory tests, but also how effective a treatment is at helping someone with the disease live better. We can never have that insight without the patient voice. Rather than hearing about patients’ experience after a new medicine has been approved, the EL-PFDD provides an opportunity for patients’ voices to truly shape the narrative and make sure that the approval process includes the perspective of those living with the disease and what is important to them.” -Dr. Suneel Udani

“I want to be able to offer patients more effective and less toxic therapies for FSGS.  To do that, we need to lower the barriers at every step of the drug development and approval process.  Hearing directly from patients is the best way for our partners in this process to understand the urgency of need in this area and to help all of us improve the lives of patients living with FSGS.” – Dr. Laura Mariani

 

To register for this year’s EL-PFDD meeting focusing on FSGS on August 28, 2020, please click here. 

Filed Under: Recent News

I Stay Home For: Keith

April 23, 2020 by Kylie Winkler

Keith, a 4-year-old from New Hampshire, was diagnosed with Nephrotic Syndrome one year ago, in April 2019. Originally, Keith was responsive to steroids, but relapsed each time doctors tried to taper him off. In October 2019, he stopped responding to steroids altogether. Keith is currently on his third medicine and his family is hopeful this one will work. His mother, Jackey, shares their COVID-19 story.

“I stay home for Keith. Our lives didn’t change as drastically as yours when COVID-19 came around. For the last several months we’ve already been wearing masks, secluding ourselves and working to stay healthy. Keith has Nephrotic Syndrome, a chronic kidney disease and has been in relapse since October. He hasn’t responded to a couple of treatments and is now on an immunosuppresant drug which puts him at greater risks for complications from something like COVID-19 but will *hopefully* finally put him into remission.

You want to know something that will make you grateful? Spending time at Boston Childrens Hospital. We have spent a lot of time inpatient and outpatient and have met families going through unimaginable pain and suffering. Having to stay home should be a luxury! We still have to go to the hospital a lot but will continue to be careful and are SO thankful for our BCH family and for NephCure for bringing awareness to families like ours. #IStayHomeForNephCure.”

Through her personal Facebook page, Jackey Bennett was able to raise more than $1,600 to support NephCure Kidney International’s COVID-19 Emergency Fund!

Filed Under: Recent News

Clinical Trials and COVID-19: FAQs from Patients to Sponsors

March 31, 2020 by Kylie Winkler

In response to the COVID-19 crisis, NephCure Kidney International has prepared a list of FAQs, solicited from patients in our community, to pharmaceutical companies sponsoring clinical trials. We urge sponsors, with patients currently enrolled in studies, to be proactive in your communications. This may be addressing these questions via email, phone conversations, orthrough in-person discussions.

For our patient community, we are doing our best to provide these answers to you and/or direct you to the appropriate resources. As you review these questions, you will see that many of these require answers specific to your studies (i.e. the introduction of virtual clinic visits or offering reimbursement for alternative travel arrangements). 

I am worried about leaving my house to go to my scheduled appointment. What should I do?  

 

Assuming you are not exhibiting symptoms of the virus and are able to get to your appointment, we encourage you to keep all previously scheduled appointments. Should there be a change in operating procedures at your clinic, your healthcare provider should notify you in advance. Some clinics are offering virtual visits. Please contact your study coordinator for more information about your specific site. 

  

My state/city/county has placed restrictions on ‘non-essential’ travel. Do my study visits fall into this category?  

 

Like grocery stores and pharmacies, medical facilities and hospitals remain open. Clinical study appointments are essential to your overall health so you should plan to attend your scheduled appointments unless otherwise directed. Be sure to contact the study team immediately should you experience any symptoms of the virus at any time. 

  

Can I avoid coming to the clinic for study appointments during the COVID–19 outbreak?  

 

We encourage you to try to attend your study visits. These appointments will occur unless something changes at your clinic, in which case you will be notified in advance. Some Sponsors may move to virtual visits to protect the health of study participants. You can contact your study coordinator to further discuss your options. 

  

I am afraid to take public transportation given the number of people and concern over being exposed to the COVID-19 virus. Are there alternate ways for me to get to my appointments?  

 

This feeling is perfectly understandable during this time of uncertainty. If you can avoid public transportation, we strongly encourage you to do so. Should you choose to take an Uber, Lyft, or other mode of transportation to attend your appointments, the company conducting your trial will most likely reimburse you for the full amount. Be sure to contact your study coordinator before you make this decision so you can to fill out the appropriate forms for changing your mode of transportation.  

  

 

What happens if I miss a study visit? Can I just pick up at the next one?   

 

It is important for you to notify your study coordinator if you are unable to make an appointment. The coordinator can assist you with scheduling another appointment and/or help with travel arrangements if needed. However, we strongly encourage you to try to attend all of your regularly scheduled study visits.  

Why is it important for me to continue participating in the trial as scheduled?  

We are thankful to all the volunteers who are participating in our study. Clinical studies are developed with scheduled procedures and visits in mind from the beginning. For this reason, we hope that you will do your best to adhere to your study visit schedule to the best of your abilities. 

 

Questions best addressed by individual sponsors  

 

If I need to change my travel arrangements to my appointments, who do I contact? 

 

Is the study medication I am taking an immunosuppressant? In other words, am I immunocompromised right now? OR Does the trial medication put me at greater risk of contracting Coronavirus?  

  

What type of financial compensation can I receive if the Coronavirus affects my travel plans?  

  

If I am under quarantine, can I still participate in the trial?  

  

Who should I contact with questions?  

  

Can I have labs drawn and/or medication administered at home?  

 

Can I complete my study visits virtually through telemedicine visits?  

 

If I can get to my study visit, will face masks be available when I arrive? 

Filed Under: Recent News

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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.

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