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!
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 
went 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.
In 2014, 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.
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.
What a Year We Had in 2014!
Thank 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
Hundreds 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
Patient 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.
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.
The 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.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:
researchers 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.
President Obama signed the appropriations package to fund federal agencies through fiscal year 2015 (ending September 30, 2015). This includes allocations for NIH and other health care funding agencies such as the Department of Defense (DoD), which oversees the Peer-Reviewed Medical Research Program (PRMRP).
For the first time, FSGS is listed as a condition eligible to study in the PRMRP. Because FSGS is listed, FSGS researchers have an opportunity to compete for grants from a NEW funding source. This is HUGE. This opportunity is a direct result of NephCure’s professional and volunteer advocacy work in Washington, D.C. and the efforts of our staff to work collaboratively with scientific partners at NIH and the VA to highlight the benefits of studying this disease through the DoD program. The total amount in the PRMRP program is $247.5 MILLION!
UPDATE:
The Program Announcement was released April 17 for the 2015 cycle. There are five funding mechanisms available. Click here for details.
NephCure is happy to partner with the American Society of Nephrology and the ASN Foundation for Kidney Research to co-fund one of the 19 new research projects aimed at improving the quality of life for millions of kidney disease patients.
The 2014 recipient of NephCure Kidney International-ASN Foundation for Kidney Research Grant is Heon Yung Gee, MD, PhD at Boston Children’s Hospital.
“The 2014 grant recipients will propel advances in patient care and outcomes through basic, translational and clinical research,” Chair of the Board of Directors Bruce A. Molitoris, MD, FASN, said. “ASN is delighted to support this research and foster the careers of the next generation of investigators who will advance care for kidney patients.”
For more information on all the recipients and their research, read the full article here.
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