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).
The last two days are the reason I do Government Advocacy! We had 37 people there – comprised of 5 staff members, 1 board member, 14 different families, children aged 2-16, a Mayor and even parents whose son passed away from complications of a transplant. These are our constituents, these are the faces of NS/FSGS. I was so proud, excited, energized by these families who traveled (many from the west coast!) to be here, tell their story and STAND UP & BE COUNTED for their loved ones!
We are so grateful and we thank each and every one of the following participants: Tamekia Bernard, Maria Bradley, Justine Byun, Gregory Byun, Betsy Calloway, Dylan Clancy, Pam Duquette, Lindsay Duquette, Matt Duquette, Richard Fissel, Wayne Hall, Ashlee Harrison, Zeke Harrison, Zoey Harrison, Terry Hauk, Steve Hauk, Lisa Hollomon, Matt Hollomon, Geni Hubbard, Jessie Hull, Jeff Hull, Genneia James, Jaylen James, Jordan James, Annmarie Naples, Anthony Naples, Cecilia Naples, Grace Naples, Kimberly Queen, Helga Queen, Kathleen Romanczuk, Rhoda Thompson, Manu Varma
– Jessica Martin, Director of Program Operations, NKI
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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.
Over 400 SoCal residents came out on a picture perfect January day – the type you only see in Southern California in January – to celebrate the work of NephCure Kidney International at the 6th Annual NKI Los Angeles Walk, Brunch & Learn.
This incredible event was organized and chaired by Los Angeles Attorney Sarretta McDonough of Gibson, Dunn & Crutcher LLP and her wonderful volunteer committee that included Janet Hasson, Genneia James, Peter Daenzer and Rich Griswold. As with the past five years, this event was a true labor of love for this group and the dozens of volunteers who also dedicated their personal time on a Sunday to support NKI. With everything from an incredible Kid’s Zone with face painting, games, a photo booth and even a visit from a bonafide “Minion” (courtesy of NBC/Universal), to a delicious brunch and post-Walk educational Lunch and Learn presentation by nephrologists from three of the largest hospitals in the Los Angeles area, this event offered something for the whole family.
Beyond that, the warmth and camaraderie created a welcoming atmosphere for everyone to feel that they were part of a strong, incredible community effort to fight these conditions and a welcome reprieve from the isolation that living with Nephrotic Syndrome/FSGS can often bring. The Annenberg Beach House in Santa Monica provided the perfect venue for the Walk, family activities, lively silent auction, and of course the important Lunch and Learn with Drs. Elaine Kamil (Cedars Sinai), Kevin Lemley (USC/Children’s Hospital LA) and Sergio Infante (Loma Linda), who generously extended the question and answer session until every single person had been heard. With well over $76,000 raised and more funds coming in every day, this event was truly a success for NephCure’s important mission to fund new treatments and find the cure!
In mid-November, Mark Stone, NephCure Kidney International Acting CEO, attended the sixth annual Partnering For Cures Conference in New York City. The Partnering For Cures Conference is a patient-focused event that brings together leaders from all sectors of medical research to foster collaboration – collaboration that will lead to medical discoveries, and, ultimately, treatments and cures.
Partnering For Cures is hosted by FasterCures, a Washington, DC-based center of the Milken Institute. FasterCures hosts the conference each year to convene groups like NephCure to identify partnership opportunities and advance outcomes-driven medical research. At the core of Partnering For Cures is the idea that the cultivation of cross-sector relationships will lead to collaborative efforts necessary for the development of new therapies.
This year’s conference focused on the importance of patient-reported data and how determined patients are improving and accelerating the search for cures. “Patient empowerment is at the heart of what we do at NephCure,” says Mark Stone. “Conferences like Partnering For Cures help groups like NephCure influence research innovations and remind us of the most important aspect of our research – our patients.”
NephCure is a registered non-profit (501c3) tax-exempt charitable organization. EIN # 38-3569922.
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