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A Call for More Specific Standards of Care

From a new article, published by Nephrology News and Issues, comes a provocative call for more specific standards of care in nephrology. As of now, the standard of care for the majority of kidney-failure patients is to follow a similar treatment plan, regardless of the cause for their kidney failure. Yet, according to research conducted at Stanford University School of Medicine, this approach is not enough. In fact—it could even be dangerous.

Researchers used data from over 84,000 patients, who between 1996 and 2011, suffered end-stage kidney disease due to one of six major glomerular disease types. The results were shocking. Mortality ranged all the way from 4% per year for patients with subtype, IgA nephropathy, to 16% per year for patients with subtype, vasculitis. Furthermore, patients with lupus nephritis were almost 2x as likely to die as those with IgA nephropathy. In other words, the specific type of glomerular disease determined how long a patient lived after developing kidney failure. As one researcher put it, “when you divide patients according to their glomerular disease subtype, you actually see a whole spectrum of outcomes (O’Shaughnessy).” And yet, the current standard of care is to follow a similar treatment plan for most kidney-failure patients, regardless of cause.

“We showed that a patient’s cause of kidney failure is strongly associated with their risk of dying after starting dialysis or receiving a kidney transplant.” Thus, treatment can no longer be generalized and non-specific. Medical professionals cannot ignore the cause of kidney failure and proceed with treatment as though all kidney failures are one and the same. The cause of kidney failure cannot be forgotten. Rather, it should be the stepping point from which treatments are determined, and tailored toward disease-specific risks. More so, further research is necessary to determine why these survival disparities exit from one patient to the next. If medical professionals begin to take into consideration what caused the kidneys to fail in the first place, it could possibly improve the patient’s quality of life and even increase their life span following kidney-failure.

To read more, visit: http://www.nephrologynews.com/study-shows-importance-of-cause-of-kidney-failure-when-planning-future-treatment/

 

Newest NKI-ASN Grant Recipient

NEWEST NKI-ASN GRANT RECIPIENT: DR. AZELOGLU

We would like to officially congratulate Dr. Evren Azeloglu, the newest recipient of the NKI-ASN Grant. Dr. Azeloglu is an Assistant Professor in the Department of Pharmacology and Systems Therapeutics at the Icahn School of Medicine at Mount Sinai. He was originally trained as a mechanical engineer, but later Untitledwent on to receive his Ph.D. in Biomedical Engineering from Columbia University. In 2010, Dr. Azeloglu was awarded the Howard Hughes Medical Institute Fellowship from the Life Sciences Research Institute. His background in biomechanics and systems biology is uniquely positioned to study complex diseases such as hypertension and diabetic nephropathy. He aspires to design transformative therapeutic tools using nanotechnology and tissue engineering.

The title of Dr. Azeloglu’s project is “mechanosensitive control of podocyte cytoskeleton and remodeling.” Hypertension is one of the major drivers of kidney disease. However, the physical mechanisms through which high blood pressure affects nephrotic syndrome are not well known. Dr. Azeloglu plans to utilize engineering methods to investigate the structural properties of the cells of the kidney glomeruli during healthy and diseased conditions. This project will be conducted in hypertensive animal models wit­h a nanotechnology platform. He will explore how kidney cells retain their structural integrity against mechanical injury (i.e. due to high blood pressure) and identify potential genes that may be responsible for maintenance of the healthy cellular structure under hypertension. This project aims to use state-of-the-art bioinformatics and nanotechnology tools to link the structural integrity of glomerular cells to overall kidney function. The results of his research may directly translate into treatable drug targets that can improve the resilience of kidney cells against high blood pressure, and slow down the progression of chronic kidney disease.

Advocacy in Action: More Funding for FSGS Research!

Remember When…Advocacy 2015

Back in January, FSGS was officially added to the list of conditions eligible for research funding through the Department of Defense’s 2015 Peer Reviewed Medical Research Program (PRMRP). This opened up a new $247.5 million funding source for FSGS researchers. When NKI and our patient families visited Washington DC earlier this year, we advocated to keep FSGS on the list and to increase the funding available for research.

P1130101More Good News:  

Last week, the Senate Appropriations Committee approved the 2016 Defense Appropriations budget including $31.2 million in additional funding for the Peer Reviewed Medical Research Program. FSGS made the list of eligible conditions again, so if the bill passes without changes, our researchers will have access to $278.7 million in 2016!

What’s Next?

The bill will have to be passed by the House and Senate and signed by the President before going into effect. We’ll be tracking it and keeping you updated as it moves through the legislative process.

Thank You!P1130058

Increased funding for research and including FSGS on the list of eligible DOD conditions were two of our “Asks” when we visited Capitol Hill. Thank you advocating in person, writing letters and emails, and making phone calls to increase government awareness of Nephrotic Syndrome diseases and the vital role that government funding plays in finding better treatments and cures! Keep it up and stay tuned for more opportunities to be an Advocate!

Genetic Findings from an NKI Funded Researcher

In 201Heon Yung Gee4, NephCure Kidney International and the ASN Foundation for Kidney Research awarded Dr. Heon Yung Gee with a Young Investigator Grant. Dr. Gee is a physician-scientist at Boston Children’s Hospital who focuses on the genetic causes of nephrotic syndrome. With NephCure and ASN support, he is conducting research on how mutations in KANK2 and ARHGAP4 cause defects in the proteins that cells use to communicate. Lack of communication causes problems with podocytes—the part of the kidney that likely plays a big role in causing nephrotic syndrome.

Recently, Dr. Gee published an update on this research in the Journal of Clinical Investigation. Thanks to Dr. Gee’s research, the Hildebrandt group has added KANK 1, 2, and 4 to the genetic test used to influence the treatment of nephrotic syndrome patients. As of now, this test includes approximately 30 genes, which if mutated, are known to cause nephrotic syndrome. To learn more about genetic tests, stay tuned for an upcoming NKI monthly newsletter.

Dr. Gee’s research has also led to new tests, which can be used to screen for drugs that may provide a treatment for some patients with steroid-resistant nephrotic syndrome.

To learn more about Dr. Gee and other NKI-funded researchers, visit: https://nephcure.org/research/nephcure-funded-research/.

To read Dr. Gee’s article in the Journal of Clinical Investigation, click here: http://www.jci.org/articles/view/79504.

NKN Steering Committee: Now Accepting Applications

The most unique aspect of the NephCureKidney Network Patient Registry is that it is governed by a group of patients, patient parents, patient advocates, clinicians, researchers and representatives from industry who work together to ensure that the needs of the NS patient community and the NS research community are met.

Each month this group of 18 individuals meets via conference call to discuss topics focusing on recruitment and retention, patient engagement, data collection, and medical profession engagement. Because each group of stakeholders can voice their perspectives on the call, it is ensured that the NKN registry will remain both patient centered and a valuable tool for the research community.

Want to be involved?

If you are interested, email Joanna Dauber at jdauber@nephcure.orgor call 610-540-0186 x 36.

Applications will be accepted until April 27th, 2015.

$90 Million Offered In Latest Call For Pragmatic Clinical Studies

The Patient-Centered Outcomes Research Institute (PCORI) is now accepting Letters of Intent (LOIs) through their latest funding announcement under the Pragmatic Clinical Studies initiative, which is offering up to $90 million in support for projects evaluating the effectiveness of different care options in “real-world” settings. PCORI will provide up to $10 million in total direct costs per study. For the LOI deadline and other key dates associated with the latest pragmatic studies funding opportunity, visit the full funding announcement.

PCORI awarded $64.1 million in February to fund the first five studies selected through this initiative and their Board of Governors will consider another slate of potential awards at its next meeting on Tuesday, April 21.

Director of Research attends RDCRN Face to Face Meeting

MH-PAG
Marilyn with Dr. Pam McInnes – Deputy Director, National Center for Advancing Translational Sciences (NCATS)

1/1/2015 – This week, Marilyn Hailperin, NKI’s National Director of Research attended the Rare Disease Clinical Research Network (RDCRN)’s Coalition of Patient Advocacy Groups (CPAG) face-to-face meeting in Rockville, MD. The meeting included patient advocacy groups that are associated with the rare disease consortia funded by the RDCRN (a program of the NIH), including the Nephrotic Syndrome Study Network (NEPTUNE).

Legislative Action Alert

On the heels of our very successful Advocacy Day, comes this Alert. Please take action before end of day on Friday, March 20

Request that House and Senate Members sign on to letters supporting FSGS research

Members of the House and Senate have drafted letters urging their colleagues in Congress to support medical research on focal segmental glomerulosclerosis (FSGS) and nephrotic syndrome (NS) through the National Institutes of Health and the Department of Defense.

Contact your two Senators and your House Representative to ask them to sign on to the letters. If a large number of Members of Congress express support for research on FSGS/NS, federal agencies will respond by continuing to prioritize their FSGS/NS research portfolios.

To determine your two Senators: Visit www.senate.gov, and select your state in the drop-down menu on the top-right corner of the webpage. Click ‘go.’ Your two Senate offices will appear with their DC office phone numbers and webpages.

To determine your House Representative: Visit www.house.gov, and enter your zip code in the box on the top-right corner of the webpage. Click ‘go’ and a link to the webpage of your House office will appear. You may need to enter your full address if more than one Representative is listed.

Action: 

  1. Call your two Senate offices and your Representative’s office.
  2. Tell the person that answers the phone that you are a constituent.
  3. Ask to speak with the health staffer and deliver the message below. If you can’t reach the health staffer, it is OK if you deliver the message to someone else or to a voicemail.
  4. Ask if you can send a follow-up email and request an email address.
  5. Send a follow-up email based on the message below.

Recommended Message – [brackets] indicate where to insert specific information:

  • “I am a resident of [CITY]. [Cite any meetings you may have had with the office.]
  • I am contacting you to request that your office sign on to a letter supporting research on FSGS. The closing date is March 23rd.

[In the Senate: “The letter is led by Alex Graf in Senator Stabenow’s office”] or [In the House: “The bipartisan letter is led by Dante Cutrona in Rep. Costello’s (R-PA-6) office and Joel Richard in Rep. Deutch’s (D-FL-21) office.”]

  • FSGS and the diseases that cause nephrotic syndrome are devastating kidney diseases. FSGS is a leading primary glomerular cause of end-stage renal disease.
  • [Briefly tell your story as a patient, parent, caregiver, etc.]
  • I hope you will sign on to the letter supporting FSGS research before March 23rd.
  • Please keep my posted on the actions you take. You can reach me at [phone and/or email].

Thank you! Feel free to forward this information to friends and family members as well. Every call helps!

2014 Research In Review

What a Year We Had in 2014!

2014Thank you to all those who collaborate with NephCure Kidney International (NKI) to advance its mission to support research leading to better treatments and cures for those suffering with FSGS and other Nephrotic Syndrome diseases. 2014 saw several major successes in the research world that move us closer to achieving our goals.

Below is a summary of NKI’s 2014 research-related accomplishments.

NEPHCURE KIDNEY NETWORK PATIENT REGISTRY

nknpageHundreds of people with primary FSGS and related glomerular diseases enrolled in the newly launched NephCure Kidney Network Patient Registry. These patients and care givers of patients are at the forefront of a movement called “patient powered research”. By sharing and comparing data contributed by patients about their health and quality of life, we can accelerate the development of new treatments, including new drugs. It is important for people with a rare disease to STAND UP & BE COUNTED to ensure research focuses on the issues of greatest importance to patients and their families. To learn more or enroll, click HERE.

RESEARCH GRANTS

In 2014, the NKI grant program committed over $2.6 MM in support for scientific investigations that advance knowledge about the cause of NS disease, evaluate possible drug targets and help physician/scientists track the natural history of these rare diseases. Click HERE  for more information about grants awarded to investigators at Washington University Medical Center, Boston Children’s Hospital and Rush University. For more information about the research consortia we support, click HERE  for the Nephrotic Syndrome Study Network and HERE to learn more about CureGN.

CLINICAL TRIALS

Clinical Trials MapPatient participation in clinical research is essential to advance new treatments. In 2014, NKI launched several new programs to educate patients and increase awareness of opportunities to participate in research.

  • The NEW Clinical Trial Finder website lists 16 studies actively recruiting participants in the US and Canada and another 18 around the world.
  • We launched a Clinical Trial Ambassadors Program  to recruit and train volunteers to help educate patients about the benefits (and risks) to participate in clinical trials and educate the medical community about the need for more research into treatments for NS.
  • We posted, tweeted, emailed and sent letters about NEW FSGS drug trials. Our activities have raised awareness for potential participants and increased the interest of biopharmaceutical companies in working with NKI to advance new drug trials.

CATALYZE, COLLABORATE AND CONVENE

NKI is dedicated to being a catalyst along the research continuum: from support for basic science research to working with the biopharmaceutical industry and the FDA to understand how we might increase the numbers of new drugs moving into the drug approval pipeline and, finally, how to rapidly integrate new treatments into health care practice.

2014 was a busy year as we joined forces with our partners in healthcare and research to promote better treatments and faster cures.

The advocacy work of NKI volunteers, staff and our professional partners in Washington, D.C. was instrumental in significant NEW FUNDING for FSGS and NS research in 2014.

  • Advocacy Day 2013-Rep DeutchThe National Center for Advancing Translational Sciences (NCATS) at the National Institute of Health (NIH) awarded $10 MM in new funding to continue the Nephrotic Syndrome Study Network (NEPTUNE), a consortium of research centers in the US and Canada collecting and analyzing data about patients newly diagnosed with primary NS diseases. NKI was a principal funder of the first phase of this study and continues to support and participate in its expansion.
  • A second institute in NIH, the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) awarded a new $20 MM, 5- year grant to follow patients with FSGS, Minimal Change Disease, Membranous Nephropathy and IgA Nephropathy. NKI was instrumental in collaborating with NIH to get this funding. Click HERE.
  • For the first time, FSGS is listed as a condition eligible to receive research grants through the Department of Defenses’ Peer-Reviewed Medical Research Program. Because FSGS is listed, FSGS researchers have an opportunity to compete for grants from a NEW funding source. The total amount in the DoD’s program is $247.5 Million!

NKI is a leader in assembling, facilitating and promoting professional meetings that bring together partners from industry and the academic world along with patients and their advocates to promote NS health research. In 2014, NKI:

  • Organized the 10th International Podocyte Conference in Freiburg, Germany. This conference brings together Podocyte2014-2researchers from a variety of scientific disciplines and from around the world to promote interactions and new collaborations and to investigate emerging directions in podocyte and glomerular biology research.
  • Convened the PRO in Glomerular Disease Scientific Conference with representatives from academic medical centers, the biopharmaceutical industry, the NIH and the FDA to pursue the development of Patient Reporting Outcome (PRO) instruments to advance new drug trials for FSGS and NS. (A PRO is any report of the status of a patient’s health condition that comes directly from the patient.) An FDA-accepted PRO instrument can accelerate the number of applications to the FDA for new drug trials. NKI continues to lead the development of a PRO development consortium.
  • Convened the third NephCure Research Accelerator Meeting that brought together physicians and scientists with representatives from more than a half dozen biopharmaceutical companies with an interest in drug development for kidney disease. Research Accelerator Meetings were initiated by NephCure to engage these two scientific groups in an exchange of knowledge that will accelerate translation of basic science discoveries to new treatments for people with NS diseases.
  • Presented a session on the NephCure Kidney Network and patient-powered research to the US Summit of Kidney Organizations hosted by the America Society of Nephrologists at its 2014 conference in Philadelphia. NKI continues to be a leader in bringing the voice of patients to the forefront of advocacy for new treatments for chronic kidney diseases such as FSGS, Minimal Change Disease and Membranous Nephropathy.NIA

Precision Medicine Initiative

President Barack Obama announced recently the Precision Medicine Initiative to be included in the 2016 federal budget. This is a bold new enterprise to revolutionize medicine and generate the scientific evidence needed to move more personalize medicine into every day clinical practice. Precision Medicine can be defined as getting the right treatment, at the right time – every time – to the right patient. It is about tailoring treatments “for a person” based on how they experience a medical condition rather than focusing on treating “the disease.” The President’s budget includes support for the FDA to develop new approaches for evaluating next generation genetic screening and the NIH to create research that embraces volunteer participation.

There is significant emphasis in this Initiative on engaging people experiencing a health problem in the development of targeted treatments for their condition. Patients should be active partners in the search for new and better therapies and not merely passive recipients of investigational treatments. Dr. Francis Collins, Director of the National Institutes of Health, in a small luncheon following President Obama speech, spoke with great enthusiasm of the need to gain better insights into the biology of all diseases to make a difference for the millions of Americans who suffer from them. He was clear that this initiative must be led by us, the people.  He stated, “The word ‘patients’ is not the right one. This is about participants and really about partners.”

Dr. Kathy Hudson, Deputy Director for Science, Outreach and Policy at NIH commented, “The Initiative will set the foundation for new ways of engaging research participants, sharing health data and information, and employing technology advances to mine the information for comprehensive results.”

NephCure Kidney International is very excited to already be a part of this movement as the NephCure Kidney Network (Patient Registry) is the ONLY kidney disease Patient Powered Research Network in another key government initiative — the Patient-Centered Outcomes Research Network (PCORnet). The NephCure Kidney Network is poised to be at the forefront of integrating new opportunities from the President’s Initiative with new technology and “big data” solutions that will propel new therapies for our patient community.

STAND UP & BE COUNTED. Visit https://www.nephcurekidneynetwork.org/ to learn more about participating in the NephCure Kidney Network (Patient Registry) so your voice can be heard and to share your experience to accelerate new therapies and, one day, a cure for FSGS and Nephrotic Syndrome.

Contact research@nephcure.org to connect with a member of NephCure’s research team.

NIDDK Tenure Track Position Opportunity for Nephrologists

NIDDK Pic

NephCure Kidney International is pleased to announce the development of a new nephrologist position at NIDDK. A tenure track position is available for a nephrologist to establish an independent research program in the Kidney Disease Section, Kidney Diseases Branch, NIDDK, NIH. The search is particularly focused on individuals studying glomerular physiology or glomerular disease. Research in this section focuses on mechanisms and therapy of glomerular disease under the leadership of Jeffrey Kopp, MD.

Position Requirements

  • MD degree
  • Demonstrated expertise and a record of achievement in basic or clinical research
  • Current board certification in Nephrology
  • Excellent clinical nephrology skills
  • Have or be eligible to obtain a current US medical license.

The position will be supported with independent resources commensurate with experience and programmatic needs, including positions for clinical staff, pre-doctoral and post-doctoral fellows, and a budget for consumables and equipment.

Interested applicants should submit a single PDF document containing a Curriculum Vitae, a list of publications, a summary of research accomplishments, a plan for future research including a core research question, the names and contact information of three people who are submitting letters of reference, and copies of no more than 3 publications.

All materials should be emailed to:

Dr. Mark Knepper, Chair, Kidney Disease Branch Search Committee
c/o Lisa Swearinger
SwearingerL@mail.nih.gov