Make your voice heard. The FDA is listening.
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.
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 www.FSGS.APOL1Studies.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.
When schools are no longer session, NephCure Kidney International opens its doors to fresh, new faces for internship opportunities. It’s standard protocol to not only give these students professional work experience, but to make sure they understand the struggles that come along with a Nephrotic Syndrome diagnosis.
But this year, two of our interns already knew about these struggles firsthand. Both Peyton Azar and Beau Gent are patients diagnosed with rare, protein-spilling kidney diseases, and they’re both striving to make a difference in the lives of others.
When Peyton Azar was eight years old, she began waking up with puffy, swollen eyes. At first, doctors believed the swelling was due to allergies, but various allergy treatments and the change of seasons failed to improve her symptoms. After a few months without relief, her doctors ordered a urine test; when protein was detected, they took swift action.
“They took me to the children’s hospital in Pittsburgh the next morning, and then everything after that was kind of, I guess, my new life in a sense,” Peyton said.
She was diagnosed with focal segmental glomerulosclerosis (FSGS), and since then has embarked on the long, up-and-down journey of living with a chronic kidney disease.
“Since FSGS is more of a rare disease, I’ve kind of been a guinea pig for a lot of things. I’ve been in a lot of clinical studies and things of that nature,” she said, adding that her treatments have been on the intense side.
She went into remission for a few years after her diagnosis but relapsed in seventh grade and underwent Prednisone infusions. The steroids helped, but not entirely; though her symptoms weren’t horrible, she wasn’t necessarily in remission.
Then, when Peyton was 15, her kidneys began to fail. She went on dialysis, and in July 2015, the summer after her sophomore year of high school, she received a kidney transplant, with a kidney from a special living donor: her mother.
But soon after the transplant, her kidneys unfortunately failed again—the FSGS was attacking once more.
In yet another effort to combat the disease, Peyton began plasmapheresis, which helped her move into remission after a few months of treatment. In the four and a half years since then, she hasn’t experienced a single relapse.
“I definitely don’t feel as normal as the average person would,” she expressed. “I definitely get fatigued easier, and sometimes my transplant site will hurt. I also get migraines a lot and have stomach issues from my medications. So, it’s definitely not normal, but it’s a lot better than pre-transplant.”
Peyton is a rising senior at West Virginia University, and explains that within the last year, she has developed a much clearer picture of the career path she wants to take: working with a health organization, ideally in a patient advocacy role.
“I would love to be a part of something that makes a difference,” she said, adding although she’s hoping to work in the healthcare field, she’s also open to joining a nonprofit with a focus other than healthcare—just as long as she’s helping people.
Peyton refuses to let her disease dictate her life or dampen her aspirations.
“I feel like so many people with health issues think ‘Why me? Why did it happen? Why do I have this?’” she said. “But I think a big one for me is ‘Okay, I have this. What can I do with it?’ I don’t want to just sit down and take it, I guess. I want it turned to something positive and have it strengthen my character.”
While Peyton is interning with NephCure’s Department of Research and Engagement, Beau Gent, another Nephrotic Syndrome patient and summer intern, is working with the Community Development team. A rising junior at Regis University in Denver, Beau’s kidney health journey has been a winding one.
At 13 years old, Beau was diagnosed with lupus. Although his health was relatively stable throughout his high school career, he experienced harsh flare-ups during his freshmen year of college. His doctor ordered blood work and found a concerning creatinine level. Shortly after, he was hospitalized and received a new diagnosis.
“I was then diagnosed with lupus nephritis because my lupus had then started attacking my kidneys, and then from there it kept on progressing,” Beau explained.
He was 18 years old at the time.
“At one point they thought that they were going to be able to salvage my kidneys,” he said, describing how he underwent a multitude of immunosuppressant treatments, including a round of steroids and then chemotherapy—neither treatment helped.
After his freshmen year, with little improvement, Beau received a third diagnosis: end-stage renal disease.
“I haven’t been through this whole kidney journey as long as many other people, but it came extremely fast, and it was tough,” he explained.
In July of 2019, less than a year after he was diagnosed with lupus nephritis, Beau started dialysis.
“I spent my entire sophomore year on dialysis, which was a time and a half, to say the least. I would go to classes in the morning, and then in the afternoon I would go to dialysis. So, I’d start my day around 7:00 in the morning and I’d end my day at 8:00 or 9:00 at night,” he said. “It wasn’t ideal, but it was a necessary thing, and thankfully I was able to make it work.”
In April 2020, Beau underwent a kidney transplant, and, like Peyton, received a kidney from one of his parents. Beau’s living donor was his father, and since the transplant, he’s improved tremendously. Though his doctors haven’t specifically declared he’s in remission, the results of his blood work and other appointments have been encouraging.
“I’ve been leaps and bounds better,” he said. “I feel absolutely amazing.”
For Beau, who enjoys weightlifting, the hardest part about dialysis was being unable to gain any weight or muscle mass.
“Once I got on the dialysis and they were taking all that water weight off, I was like a string bean,” he laughed. Six weeks after his transplant, he was able to begin lifting weights and hiking again, which he describes as ‘a huge blessing.’
Beau’s intern position with NephCure isn’t the first time he’s been involved with the organization. Last year, his mother entered an essay writing contest sponsored by NephCure, and she and Beau were selected to travel with the team to lobby government officials in Washington, D.C.
Like Peyton, Beau also wants to dedicate his career to helping others. His goal is to become a physician’s assistant, specifically in the pediatric field, and he says he might even want to go into nephrology.
“I’ve gotten to talk to a lot of amazing healthcare professionals, and I’ve gotten to talk to some not so amazing healthcare professionals, and kind of learn things that I would like to change in the system, and learn things that I would like to keep in the system,” Beau said. “I think that that is definitely an interesting perspective that you can have as a patient kind of going into a healthcare profession yourself, because you know it firsthand.”
Though he was quickly forced into a life of serious kidney disease on top of an autoimmune disorder, Beau doesn’t let his illnesses define him.
“My goal has always been to make people think that I’ve never been sick,” he said. “I want to work ten times harder than the healthiest person around, being that it’s okay for my health personally.”
Beau and his mother have a motto they live by to help push them through the hard times: “no wasted days.” Living with chronic illnesses means every day has the potential to be a challenge; Beau makes a conscious effort to truly appreciate the days that aren’t so tough.
“If you’re given a day where you don’t feel absolutely terrible and you can do a little bit, or you can push yourself a little bit more, take advantage of it,” he said. “Because you don’t know if the next day you’re going to feel terrible again and can’t get out of bed.”
Both Beau and Peyton have already greatly contributed to the team. Their presence is an appreciated reminder of the strength and resiliency Nephrotic Syndrome patients possess, and NephCure is looking forward to their continued collaboration.
While the past few months have brought a great deal of confusion, pain, and discomfort to the entire world, we understand the thirst for information is just as prevalent now as it was at the start of the COVID-19 pandemic.
As new research is coming out and guidelines are seemingly changing every day, NephCure is here to help you and other patients with protein-spilling kidney diseases navigate the unknown. We continue to advise you to consult with your physician to design your own protection plan, as every patient’s situation is unique.
No matter your age, if you have chronic kidney disease at any stage or have received an organ transplant, the CDC has clearly outlined and our NephCure Specialists agree that you are at an increased risk for complications from the COVID-19 virus.
We recognize that while your physical health is important, your mental, social, and financial health are also a priority. We encourage you to talk to your doctor, family, and support system to devise a plan you feel comfortable with as you decide when and how to re-enter society.
In the words of Dr. Anthony Fauci, “If it looks like you’re overreacting, you’re probably doing the right thing.” Ultimately, until a vaccine is found, we urge you to follow all precautionary steps recommended by the CDC to decrease your chance of getting COVID-19 infection.
We are in regular communication with our NephCure Specialists to help find the answers you need. We will keep you updated as we receive new information in the coming weeks and months.
As you develop your protection plan, here are some key points to remember:
Consult with your personal nephrologist and family to create a plan that is right for you.
Your risk may vary depending on the community spread within your region. Search for your county using this map and check with your local health department for information relevant to your location.
This statement was prepared with input from NephCure Specialists:
Ambarish Athavale, MBBS, MD
Lawrence Holzman, MD
Elaine Kamil, MD
Ali Poyan Mehr, MD
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.
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.
Contact: Lauren Eva
We are deeply horrified by the recent violent deaths of George Floyd in Minnesota and Breonna Taylor in Kentucky. They are the latest in a vast chain of Black men, women, and children in the United States whose lives have been tragically and wrongfully cut short.
Their lives mattered.
We stand in solidarity with those fighting for justice, peace, and equity for Black people and other communities of color in this country.
Systemic racism and deeply entrenched inequalities have plagued our country since even before its founding. Our healthcare system continues to deny equitable access to research and care that could significantly improve the lives of Black people and other people of color at all walks of life.1, 2, 3, 4, 5
At NephCure, we are focused on providing support and education to all who are affected by chronic, rare, and progressive protein-spilling kidney diseases, in addition to pushing forward research to better treat and cure these diseases.
Black Americans are disproportionately affected by chronic kidney disease, and uniquely affected by an aggressive form of a protein-spilling kidney disease called Focal Segmental Glomerulosclerosis (FSGS).6, 7 Working to improve the options and availability of treatments for Black individuals affected by kidney disease falls squarely within the scope of our organization.
NephCure serves ALL people who are affected by protein-spilling kidney diseases, but we know we can do better for our communities of color.
We have been working with some collaborators over the past few months on a Health Equity project, focused on increasing diversity in this field, and we’re excited to share more about that soon. But we recognize that this moment is not about us. With that in mind, we are providing space and time throughout this month (and beyond) specifically aimed at highlighting Black voices within our community.
There is a great deal of work to be done, and we are dedicated to learning, growing, and constantly improving. If you are so inclined, we welcome your feedback on how we’re doing in this regard.
We are not so audacious to expect that we can end the consequences of systemic inequities that have negatively shaped life for Black people in the United States for hundreds of years. But we believe we can make change in our community. And we will.
1 Hamel LM et al., (2016). Barriers to clinical trial enrollment in racial and ethnic minority patients with Cancer. Cancer Control. 2016 Oct; 23(4): 327–337. DOI: 10.1177/107327481602300404 https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5131730/
2US Food and Drug Administration. (2017, February 13). Drug Trials Snapshots Summary Report (2015 and 2016) https://www.fda.gov/drugs/drug-approvals-and-databases/drug-trials-snapshots-summary-report-2015-and-2016
3D. K. Ginther et al., (2011). Race, ethnicity, and NIH research awards. Science. 333, 1015–1019 (2011). DOI: 10.1126/science.1196783 https://science.sciencemag.org/content/333/6045/1015.full
4The Commonwealth Fund. (2008, April 1). Do Primary Care Physicians Treating Minority Patients Report Problems Delivering High-Quality Care? https://www.commonwealthfund.org/publications/journal-article/2008/apr/do-primary-care-physicians-treating-minority-patients-report
5Hayanga, AJ et al., (2009). Residential Segregation and Access to Surgical Care by Minority Populations in US Counties. Journal of the American College of Surgeons, 208(6), 1017-1022. DOI: https://doi.org/10.1016/j.jamcollsurg.2009.01.047 https://www.journalacs.org/article/S1072-7515%2809%2900207-5/abstract
6National Institute of Diabetes and Digestive and Kidney Diseases. Race, Ethnicity, & Kidney Disease. Retrieved June 8, 2020, from https://www.niddk.nih.gov/health-information/kidney-disease/race-ethnicity
7Rosenberg AZ, Kopp JB (2017). Focal segmental glomerulosclerosis. Clin J Am Soc Nephrol 2017;12:502–17.doi:10.2215/CJN.05960616 https://cjasn.asnjournals.org/content/12/3/502
“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
“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.
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.”
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?
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?
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.
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!