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Q&A with Dr. Kopp of the NIH

picture- KidneyWordCloudDr. Jeffrey Kopp is a physician and researcher who focuses on FSGS and related diseases. He currently leads a group in the kidney disease section (officially called the National Institute of Diabetes and Digestive and Kidney Diseases, or NIDDK) of the National Institutes of Health (NIH). Dr. Kopp is also working on a new clinical trial for FSGS, MCD, and MN patients at the NIH headquarters near Washington D.C. We had the awesome pleasure of sitting down and catching up with Dr. Kopp about his fascinating job and new clinical trial. Keep reading to learn more, and read about some of his other research projects here. 

Interview highlights:

  • Dr. Kopp works at the National Institute of Health’s kidney branch, where he studies glomerular diseases such as FSGS and MCD. He also serves as Captain for the United States Public Health Service, and has been deployed to help with medical care during natural disasters.
  • Dr. Kopp is leading a new clinical trial for FSGS, MCD, and MN patients at the NIH studying a compound called ManNAc as a treatment option. 
  • ManNAc is a sugar that occurs naturally in your body. Another researcher at the NIH found that mice without ManNAc developed MCD, and adding ManNAc to their diet was helpful in treating it. Therefore, it may be effective at treating MCD, FSGS, and MN in humans (Dr. Kopp describes the full mechanism below—make sure you read the article!)
  • This study requires people to stay at the NIH for 11 days total, but it can be split up into 2 trips. Luckily, there is a lot to do to pass free time you may have at the NIH, including movie marathons, exercise programs, an art gallery, and an in-house business center.
  • Learn  more about taking part in the study by clicking here or contacting Emily Brede, RN at emily.brede@nih.gov

Full interview:

NKI: What is your job at the NIDDK?

Jeffrey B. Kopp, M.D.
Jeffrey B. Kopp, M.D.

Dr. Kopp: I am fortunate to lead a translational research group at the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), which is part of the National Institutes of Health. Our mission is to develop a better understanding of the disease mechanisms responsible for focal segmental glomerulosclerosis (FSGS) and to develop more effective and less toxic therapies.

I also serve in the United States Public Health Service, with a rank of Captain. My primary mission at NIH is to carry out basic and clinical research in FSGS. I also deploy for public health emergencies, such as natural disasters. Thus, I participated in the medical response to Hurricanes Katrina and Ike.

SIDE NOTE: What is NIH?

Dr. Kopp: The NIH is a federal biomedical research facility located in Bethesda, MD. The campus includes a 240-bed Clinical Research Center and extensive outpatient clinics. Every patient who comes to NIH participates in a research protocol. Some protocols involve novel treatments and other protocols involve giving samples for research. NIH physicians may give advice about standard therapies that can be used. There are no charges for any medical care provided by the NIH Clinical Center.

NKI: What do you enjoy about CKD research?

Dr. Kopp: CKD, and particularly glomerular diseases (such as FSGS), are incompletely understood, and the available therapies are not ideal. I like the challenge of understanding and treating these diseases, and most of all I like the opportunity to improve the lives of patients with these conditions.

NKI: The newest clinical trial for FSGS, MCD, and MN patients at the NIH is looking at MaNAc as a treatment option. Why did you decide to study MaNAc?

Dr. Kopp: A colleague at NIH developed mice unable to make ManNac. She found that these mice developed glomerular disease soon after birth. This disease resembled a human glomerular disease, minimal change disease. Providing extra ManNAc orally to the mice cured the kidney disease. This prompted the question: can we use ManNAc to induce remissions in our patients?

Chemical Structure of ManNAc

NKI: What is ManNAc?

Dr. Kopp: Perhaps the word sounds to you like manna, the food the Israelites found in the desert and that helped sustain them. There is a tree in Europe that exudes a sweet white resin, similar to the sap of the sugar maple, and people who knew the Bible story called the tree the manna tree. A chemist found a distinctive and novel sugar in the manna resin, and he called the new sugar “mannose”.

NKI: Does ManNAc occur naturally in the body? Is it found in food?

Dr. Kopp: ManNAc is a natural product and essential for good health. Our food does not contain much ManNAc. Our bodies make ManNAc, which is converted in our cells to mannose. This in turn is converted to sialic acid, which is put on many proteins. All of these are sugars, but they differ from glucose in that they are not related to diabetes and they are present in very small amounts, so that they do not add calories in the diet.

NKI: What is the reason for believing that ManNAc might be useful in treating glomerular diseases?

glovesDr. Kopp: Podocytes are cells on the outside of the kidney glomeruli and serve to prevent plasma proteins from leaking into the urinary space. Many patients with glomerular diseases have lost sialic acid from the proteins on the podocyte. We think that providing extra ManNAc might promote the return of sialic acid to podocyte proteins and that this might improve podocyte function. We see some evidence in mouse models of FSGS that supplemental ManNAc in the diet helps treat these mice.

NKI: What is involved for patients in this study?

Dr. Kopp: Patients will provide their medical records for review by the NIDDK team. We also review the kidney biopsy materials from past kidney biopsy. No kidney biopsy is done as part of this study. If patients appear to qualify for the study, they will come to NIH for an outpatient visit for evaluation and to discuss study participation.

NKI: Is travel to NIH paid for?

Dr. Kopp: Travel to NIH can be arranged and provided by NIH. If overnight accommodation is needed, NIH can provide this also.

NKI: Why are patients required to stay at the NIH during this study?

NIH Headquarters
NIH Headquarters

Dr. Kopp: The study requires being an inpatient for 11 days, either as a single stay or as two stays of five and six days. The reason for the inpatient stay is allow frequent sampling of blood and urine and for safety, to be sure there are no side effects.
NKI: What can patients do with any “free time” during the study? How much free time do you expect patients to have?

Dr. Kopp: During the first five days, there are frequent time points for sample collection. During the second six days, samples are needed at 8 am and 8 pm. There is extensive free time that patients can use as they like.

There are many activities that can help pass the time at NIH

• Patient Computers combination television and computer (with Internet access) at most patients’ bedsides to provide access to games, web browsing, and personal e-mail via the Internet

• Patient Library has more than more than 5,000 books, including a selection of current best-sellers, reference, foreign language, large-print, picture, and audio books

• Clinical Center’s Fine Art Program has more than 2,000 works of art. Most artwork remains on permanent display throughout the hospital, but there are six galleries on the first floor that change every eight weeks. A walking tour is available to assist patients, caregivers and visitors in their enjoyment of the artwork on display.

•Recreation Therapy programs include:
o Arts and crafts
o Music
o Games and sports
o Social events
o Exercise
o A large selection of DVD movies
o Instruction in coping skills such as relaxation, enhanced communication, and stress management

• Spiritual Care Department offers Catholic, Jewish, Islamic, and Protestant services in the interfaith chapel

• Business Center has four PCs and four MACs (all with Internet connection) as well as a combined printer/copier/FAX and telephones are available.

NKI: Who can participate in the ManNAc study?

Dr. Kopp: We are recruiting adults (age ≥18 years) with a primary glomerular disease, including minimal change disease, FSGS, and membranous nephropathy, and with nephrotic range proteinuria (urine protein/creatinine ratio > 2 g/g).

Exclusion criteria include having diabetes mellitus and receiving pulse therapies, such as rituximab. Monetary compensation is provided.

NKI: How do I get more information about the study?

Dr. Kopp: The study, like all clinical research studies, is described at clinicaltrials.gov.
You also contact the study research nurse, Emily Brede, RN at Emily.brede@nih.gov

Watch the Demystifying Research Webinar!

Watch NKI and special guest speaker Dr. Jonathan Hogan from The University of Pennsylvania, as we demystify Nephrotic Syndrome research! Research is important to all members of the NS community- so why not learn about it!

We talk about how research works, what is happening in the NS research world, and what patients and their families can do to help. Come with your research questions, and prepare to have them answered!

CLICK HERE TO WATCH THE WEBINAR 

The Silver Lining Found In Nephrotic Syndrome – A Story by Lauren Bentley (former NKI Intern)

The summer before 8th grade, I began to experience extreme fatigue. No matter how long I slept at night, I could not keep up with my friends. I often woke up with swelling in my neck, face and eyes. Sometimes I vomited for no apparent reason. My parents had been taking me to doctors for years but nobody could pinpoint the cause of my symptoms. I was tested for a variety of diseases and visited almost every unit in the children’s hospital. Finally, we landed in the Rheumatologist’s office. She told us that nothing abnormal showed up in my blood work but to come back if my symptoms grew worse or I experienced swelling in my feet or legs.

One night while I was at camp, our counselors told us to get ready to play capture the flag. I ran to put on my sneakers but no matter how hard I pushed my foot into my sneaker, it would not slip in. I looked down and realized the entire bottom half of my body, from my toes to knees, had swelled up like a balloon. The counselors and doctor on hand attributed the swelling to being out in the heat, walking around campus and eating salty foods. When I mentioned the swelling to my mom, she thought the same thing.

However, after I returned home from camp, the swelling did not disappear. In fact, it stayed exactly the same. My mom made an appointment with my Rheumatologist, who ordered blood work immediately. Two weeks later, I found myself sitting on the examination table in a Nephrologist’s office. The blood work showed I had signs of protein spillage and kidney damage. The team of doctors suspected I had Nephrotic Syndrome, a rare form of kidney disease. I could not believe this was happening. I felt as though I did not belong to my body, and instead, was watching this unfold on a movie screen. I can only describe it as an out of body experience.10394043_10152537606535806_2643722050799595988_n

I spent two days in the hospital after undergoing a kidney biopsy. The results confirmed I did in fact have a form of Nephrotic Syndrome known as Membranous Nephropathy. In everyday words, this meant I had a kidney disease and damage as a result of Lupus that went undetected for years. Lupus is an autoimmune disorder in which the body cannot tell the difference between good and bad cells. As a result, it attacks healthy tissues, such as joints and major organs.

It is difficult to describe the feelings I had while receiving this diagnosis. Part of me was confused over all the medical terminology. Part of me was sad. Part of me was overwhelmed and angry that my body had failed me. But after hearing how the doctors would treat my disease, almost all of me became scared. Along with diet and activity restrictions, I was prescribed several heavy-duty medications. Prednisone was one of the medications that would give me the most side effects. As the doctor rambled off the side effects I grew increasingly frightened. She said I would have mood swings, low energy, an upset stomach, grow “chipmunk cheeks,” and gain as much as thirty pounds – all in the course of just a few weeks after starting treatment.

I remember this like it was yesterday. After my doctor’s appointment, my mom dropped me back off at school. I did not want to show I was upset or worried, so I told her I wanted to go back to class. As I sat in my desk in the back of Mrs. LaFave’s English classroom, I stared straight ahead of me. She rambled on and on about the Masque of the Red Death and all these other Edgar Allan Poe pieces, but all I could hear was my doctor’s voice rattling off the symptoms of Prednisone. What would people think?

I was on a heavy dose of Prednisone throughout my 8th grade year. Without fail, the Prednisone showed its nasty side effects. By November, I gained about 15 pounds, was always exhausted and had round, chubby, chipmunk cheeks. As I stood in front of the mirror, I did not recognize the person staring back at me. Going to school everyday was challenging. As I walked down the hall I heard whispers. Rumors circulated about why my appearance drastically changed in such a short amount of time. “Did you have some kind of weird surgery?” random students asked me. “What’s wrong with Lauren’s face?” they would ask my friends.

I did not want to share my health problems with anyone. I was worried people would treat me differently if they knew about my disease. I did not want my teachers to feel bad for me and give me special privileges. I also wanted to appear strong for myself and my family. The last thing I wanted was my parents to worry about me.

One afternoon, I sat in the cafeteria eating lunch with three of my girl friends. I looked up and noticed the girls sitting in the next booth were staring at me. One of them leaned over and whispered something in her friend’s ear. It was clear they were gossiping about me. This was probably my breaking point. I had experienced other incidents like this before since being on Prednisone. Girls, who never cared to give me the time of day before, asked me what happened to my face. A group of kids in my math class whispered about it constantly, as if I could not hear them. When I left class every second period to go take my medicine at the nurse’s office, kids asked me why I always left class. But it was something about this one particular incident that broke me. I held in my feelings all day. By the time I walked through the door at home, I could not take it anymore. I burst into tears. My mom held me and rubbed my back as I lay in bed.

This was one of the first times in my life I felt completely shattered. It boggled my mind to think that my peers could be so critical of someone’s appearance when I had no control over it. This may seem weird to say, but during this time, I also realized I had been so blessed. My condition could have been so much worse than it actually was. I could have needed a transplant. My doctors could have put me on dialysis. I could have been admitted to the hospital for weeks instead of a few days. As I went to the hospital for my monthly checkup and blood work, I saw children who were hooked up to a million machines. Newborn babies were being poked for blood work. Children were battling cancer and fighting for their lives. I was still able to sleep in my own bed at night. I didn’t need to have different IVs pump fluids into my body. Instead, I just had to take some medicine. I could run around outside and go to a real school. My friends and I still hung out on the weekends. During this time, I was also showered by so much love and support. My parents were amazing. They made me special low-salt dinners. When I cried, they listened and wiped away my tears. My doctors went above and beyond their job description. They provided me with emotional support and hugged me after every appointment. My friends at school stood up for me when other students gossiped and spread rumors. My church family visited me in the hospital, sent cards and spoiled me with special treats.

Looking back now as an adult, I am extremely grateful for this experience. It taught me so much about myself. I learned how to be disciplined to eat healthy, get enough sleep and take my medicine on time. I learned to listen to my body and identify when it told me to slow down. As of today, I still have Lupus but my kidney disease is in remission. I am thankful everyday for a healthy body that allows me to lead a happy, normal life. It also taught me life lessons. I never judge a person based on the outside or first impressions. Everyone has a story that has shaped who they are today. Until you have walked in their shoes, it is unfair to place judgment. During the teenage years where gossip spread like wild fire, I learned just how hurtful rumors can be. I learned how important it is to be kind to everyone, regardless of how they dress, speak, or who they hang out with.

Today, I intern for NephCure Kidney International. I love interacting with patients, hearing their stories, and sharing my own personal experience with them. I realized this struggle was put in my life so I could share my story with other people.

For those who are still struggling with Nephrotic Syndrome or FSGS, there IS light at the end of the tunnel. It may not seem like it at the time, but things do get better. Those chipmunk cheeks don’t stay round forever. The kids who may be gossiping or picking on you, won’t mean a thing in a few years. Nothing is ever as bad as it may seem at the time. You will realize this experience made you a stronger person.

NEW!! Clinical Studies Map!

NephCure Kidney International Introduces Clinical Research Map

October 14, 2014

Screen Shot 2014-10-14 at 2.00.19 PMWe are excited to introduce a new user-friendly, interactive clinical research map. This map includes current clinical research sites and studies available to patients affected by FSGS and other Nephrotic Syndrome diseases. The map, which can be found here, will help patients sort through studies available to them. Categories include studies specific to adults, pediatrics, Nephrotic Syndrome, and FSGS, to name a few.

“Patient participation is key to advancing research and treatment options available to patients,” says Marilyn Hailperin, the National Director of Research at NephCure. “This site map allows patients to view studies underway in their area.”

This map is another way NephCure is living out our mission to support research seeking the causes of Nephrotic Syndrome diseases, to improve current treatments and, ultimately, to find a cure. Here at NephCure, we value the supportive role we play for our patients.  Spreading the word about clinical research to our patients is a crucial aspect of this role, because, after all, patients power research!

Link to the map: here.

For more information about the risks and benefits of participating in clinical research, click here.

NephCure Kidney International Announces NKN Steering Committee Co-Chairs

We are pleased to share that Kathleen Broderick and Randall Snyder were recommended and have accepted their nominations as Steering Committee Co-Chairs for the NephCure Kidney Network (NKN). Kathleen was considered a strong candidate due to her dual experience as a patient caregiver as well as a patient advocacy representative. Similarly, Randy’s dual perspective as a patient caregiver and clinical provider give him unique insight relevant to the NKN.

Kathleen is a member of the NephCure Board of Directors and Research Committee and an editor at DeGruyter Publishing, a Science, Technology, and Medicine (STM) publisher of professional books and scholarly journals in Boston, Massachusetts. Additionally, Kathleen cares for her 16-year-old son, who was diagnosed with Nephrotic Syndrome at 20 months.

Randy is an interventional radiologist with his master’s in molecular biology living in Medford, New Jersey, who cares for his son who was diagnosed with focal segmental glomerulosclerosis.

Kathleen and Randy will be facilitating Steering Committee calls and communication moving forward. Please join us in welcoming Kathleen and Randy as the inaugural NKN Steering Committee Co-Chairs!