Potential FSGS Treatment Option To Be Tested In Phase 3 Clinical Trial

April 2, 2017 by Chelsey Fix 1 Comment

Early in March, Retrophin, Inc. announced plans to launch a phase 3 clinical trial to evaluate a potential therapy for FSGS patients. The therapy, called Sparsentan, successfully completed a phase 2 clinical trial in 2016 with promising results. The phase 2 clinical trial, known as the DUET Trial, showed a significant decrease in proteinuria for patients that received the therapy, and a greater proportion of patients that received Sparsentan during the trial reached partial remission.

Retrophin plans to launch the phase 3 trial in the second half of 2017. Phase 3 clinical trials are meant to demonstrate the effectiveness of a drug to determine how valuable it may be in clinical practice. It is the last step of research before a drug becomes approved by the FDA for clinical use.

The company is currently working with the FDA to approve the protocol for the clinical trial, which includes using the reduction of proteinuria as an endpoint to demonstrate the therapy’s effectiveness. If approved, Sparsentan would be the first FDA-approved drug for FSGS patients.

NephCure will be working closely with Retrophin to bring awareness to this clinical trial and help bring the patient perspective to their research.

To learn more about Retrophin, Inc. and Sparsentan, click here.

To learn more about ongoing studies, click here.

Matthew Singer, Nephlete

January 17, 2017 by Lauren Eva 2 Comments

Why I Do What I Do: Spotlight On Matthew Singer, Nephlete

Matthew Singer recently took on the Chicago Rock ‘n’ Roll Half Marathon as a Nephlete to raise funds for NephCure and help support his brother, Eric, in his kidney journey. We spent some time with him to learn more about his story and how he was able to raise the most of any individual fundraiser in 2016!

Matthew and Eric on race day.

NKI: Tell us how you’re involved with NephCure.

Matthew: In past years, I’ve donated to NephCure on several occasions. However, in 2016, I decided to make a more substantial commitment by running the Chicago Rock ‘n’ Roll Half Marathon and raising money for kidney research.

NKI: Why do you do what you do? What’s your personal connection to kidney disease?

Matthew: My brother has been living with FSGS for several years, so kidney disease research is obviously a cause I care a great deal about. I had been thinking of running a race, so it was serendipity when I learned that NephCure was raising funds through a [local] Chicago race. After I brainstormed the #SaveOurKidneys fundraising pitch, I had a conversation with my brother, and we were both excited about the idea. We realized that we might be able to raise a pretty significant amount of money for a cause near and dear to both of our hearts!

NKI: What was the hardest part about doing the run and/or fundraiser?

Matthew: The weekly long runs to prepare for the race were definitely the hardest. It is one thing to run 13+ miles when you’ve got the adrenaline of race day, but quite another to run 10 miles on a training run.

Matthew’s hilarious (and very successful!) fundraising page.

NKI: What was the best part?

Matthew: I was absolutely blown away by the the overwhelming response — not only close friends and family, but distant acquaintances were really generous. It is one thing to “like” our posts on social media, but another to actually reach into your wallet to donate. I was awed by how many people supported us, and how generously. Frankly, it wasn’t all that much work to raise the money: a few e-mails and social media posts accounted for most of our donations.

The race day was great as well. The weather was surprisingly cool for July in Chicago — which helped me finish the race with a personal best time.

Congratulations to Matthew and his brother Eric for their hard work to #SaveOurKidneys and fund research to find a cure for FSGS and Nephrotic Syndrome!

Learn more about how you can get involved as a Nephlete and raise funds for research while participating in your local races! Send us an email at events@nephcure.org to get started.

DUET Study Releases Preliminary Results (SPOILER it looks promising!)

September 7, 2016 by Chelsey Fix 11 Comments

big-news On September 7, 2016, Retrophin Inc. released the “Top Line” results from their recently completed DUET study, a Phase 2 clinical trial testing safety and efficacy of Sparsentan for FSGS patients.

Results showed promise for Sparsentan’s effectiveness at reducing proteinuria in patients with FSGS, with one group of patients seeing an average reduction of 44.8%. The DUET study included 96 patients, and only one serious adverse side effect (anemia) was reported. All patients chose to extend their treatment with Sparsentan during the trial’s open label extension period.

Alvin Shih MD, the executive vice-president for Retrophin Inc., said “significant reductions in proteinuria, along with a well-tolerated preliminary safety profile have us excited about being one step closer to providing a new treatment option for patients with FSGS.”

NephCure Kidney International is excited about these results and support Dr. Shih’s hope that we are moving closer to providing a new, effective, and safe treatment option for FSGS patients. Mark Stone, Chief Executive Officer of NephCure Kidney International, remarked “These preliminary results are very exciting for our community. This gives us hope that better treatment options will be available for our families in the near future.”

NephCure Kidney International would like to thank everyone who contributed time, talent, and resources to this study. Thank you, especially, to the patients and families who participated and helped bring effective treatments within reach.

Read the official press release here

White House Organ Summit 2016

June 20, 2016 by Abbey Swan 2 Comments

The Obama Administration met with numerous companies, foundations, hospitals, universities, and patient advocacy groups at the White House’s Organ Summit on Monday, June 13. The goals of the summit include increasing the number of organ transplants by 2,000 each year, improving patient outcomes, facilitating research and developments around organ donation, and closing the gap between Americans who support organ donation and those who are actually registered organ donors.

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Last year, the United States exceeded 30,000 annual organ transplants for the first time, yet 120,000 Americans are still waiting for an organ donation. Today, twenty-two people will die waiting for a life-saving transplant. President Obama, and several government and non-governmental organizations have made many efforts to reduce the organ donation waitlist, support patients, and increase access to organ transplantation.

Announced on Monday, almost $200 million in investments will be made to facilitate research and development related to organ donation. Specifically the Department of Defense (DOD) in a $160 million public-private investment will create an Advanced Tissue Biofabrication Manufacturing Innovation Institute to develop new manufacturing techniques to repair organ damage by replacing cells and tissues and that can hopefully be used one day to replace entire organs. In similar efforts, the DOD will award small businesses working to advance the science behind preserving organs and tissues.

The donor registration system is being re-imagined to seamlessly and effectively increase registrations and transplants. More than a dozen organizations including Facebook and Twitter are finding new tools and developing campaigns to make registering to be an organ donor easier with the intention of signing up 1 million new donors by fall of 2016.

More than 100,000 people on the organ waiting list are awaiting a kidney transplant so kidney-specific projects were a highlight of Monday’s Summit.

The American Society of Nephrology will partner with the XPRIZE Foundation to encourage the development of a new device solution for patients experiencing end-stage renal disease – an improvement on current dialysis methods. This project aims to overcome the decades of stagnation in kidney disease treatment.

In addition, dozens of transplant centers announced a collaboration to share data and best practices for hard-to-match patients, which could help more than 1,000 people gain access to transplants. Johns Hopkins University is currently working with the National Institute of Allergy and Infectious Disease to create HIV-positive donor pools, which could also lead to as many as 1,000 more transplants per year.

Part of the Organ Summit included the publication of letters written by organ donation recipients. One of which was from NBA player Alonzo Mourning, a kidney transplant patient. Mourning, who lost his kidney due to FSGS, went on to win a championship following his transplantation, and today brings awareness to the efforts of the White House and the importance of organ donation registration.

Your Advocacy at Work – New Funding for FSGS Research

May 12, 2016 by Abbey Swan Leave a Comment

Since 2014, NKI and our dedicated community of patient advocates have been encouraging Congress to include FSGS on the Department of Defense’s list of conditions eligible for research funding through the Peer Reviewed Medical Research Program. Learn more about these efforts here.

Directly as a result of our advocacy, FSGS was included on the list in 2015 and 2016. This new stream of grant funding gives FSGS researchers access to up to $278 million in grant awards. Last year was the first year that FSGS researchers were able to submit grant applications for this new funding stream and recently, the DoD announced which of those applications were recommended for funding. We are thrilled that five FSGS projects made the list!

FY 2015 Discovery Award

Suzie Pun – University of Washington

FY 2015 Investigator-Initiated Research Award – Partnering PI Option

Ali Gharavi – Columbia University Medical Center

Simone Sanna-Cherchi – Columbia University Medical Center

Suzie Pun – University of Washington

Stuart Shankland – University of Washington

We can’t wait to tell you more about the winning projects for 2015 and look forward to even more success in the 2016 cycle.

In order to ensure that FSGS continues to be included as an eligible condition each year, we must continue to advocate. Stay tuned for more information about what you can do to help!

Advocacy in Action – Nephrotic Syndrome Research Funding Support Letters

March 25, 2016 by Abbey Swan Leave a Comment

P1130101 Thank you to all of you who wrote to your Senators and Representatives asking them to support funding for Nephrotic Syndrome and FSGS research! Through your efforts, appropriations letters were sent to relevant subcommittee chairs in the House and Senate encouraging them to increase funding for these diseases. Four Senators signed the Senate letter and 27 Representatives signed the House letter.

Background

Each year, Congress decides how much federal funding should be applied to medical research activities and provides guidance to the National Institutes of Health (NIH) on what conditions legislators are particularly interested in. Recently, Members of Congress have been deciding on the level of NIH funding for fiscal year (FY) 2017 and crafting the accompanying list of research recommendations. As a result of grassroots outreach, the community of individuals affected by glomerular diseases has educated many Member of Congress who have become champions on research and patient care issues.

House Letter

On behalf of the community, Congressmen Ryan Costello, Ted Deutch, and Alcee Hastings recently circulated a “Dear Colleague” letter on Capitol Hill that voices strong support for advancing research into FSGS and related conditions at NIH. We asked the entire NephCure community to encourage their representatives to sign on to the letter and the response was overwhelming!

Click here to see the letter and all 27 signatories.

Senate Letter

On the Senate side, Senator Debbie Stabenow lead a letter to the Defense Appropriations Subcommittee asking them to include FSGS as a condition eligible for study in the Peer-Reviewed Medical Research Program in the Fiscal Year 2017 Defense Appropriations Bill. Click here to read the letter and see the signatories.

Advocacy Alert! Ask your Senators to sign FSGS funding letter

February 29, 2016 by Abbey Swan Leave a Comment

Contact both your Senators and ask that they sign on to Senator Debbie Stabenow’s (D-MI) letter supporting the inclusion of “focal segmental glomerulosclerosis (FSGS)” as condition eligible for study through the Department of Defense Peer-Reviewed Medical Research Program during the Fiscal Year (FY) 2017 appropriations process (Urgent – Deadline for Signatures is March 11)

Background

Each year, the United States Senate crafts an annual Department of Defense (DoD) appropriations bill, which includes a list of conditions that are deemed “eligible for study” through the Peer-Reviewed Medical Research Program (PRMRP). In order for a condition to be included, Senators need to support the condition and officially ask for its inclusion. Senators have many competing appropriations priorities and in order for them to support a condition-specific request, they need to be educated and asked to do so by their constituents. (You)

As a result of grassroots outreach, the Senate has recognized FSGS as a condition eligible for study annually for a number of years. This support allows FSGS researchers to compete for nearly $278 million in federal research funding each year.

Senators are currently working on the FY 2017 DoD appropriations bill and deciding which conditions will be included on the next PRMRP eligible conditions list. Being included on the list one year is no guarantee of being included again in the next year. At this critical time, please reach out to the offices of your Senators and ask that they “sign on to Senator Debbie Stabenow’s letter supporting the inclusion of “focal segmental glomerulosclerosis (FSGS)” in the DOD PRMRP’s eligible conditions list for FY 2017.” Click here to read the letter.

Take Action

Recommended: Copy and paste the email template below, add your info (where prompted) and send it to NKI’s Washington Representative Phil Goglas at goglas@hmcw.org . Phil will forward your e-mail on to the appropriate staff person in your Senators’ offices on your behalf.

Reach out yourself to the Health Legislative Assistant in the Washington, DC, offices of both your U.S. Senators and ask for their support.
To identify the contact information for your Senators’ Health LAs, simply go to Senate.gov and select your State. If you call the office (the 202 number), the staff will tell you the name of the Health LA and let you leave a voicemail (you can use the message below as a script). E-mail is more effective, but the receptionist will not likely provide you with the Health LA’s e-mail address.
If you would like to e-mail the Health LAs in the offices of your Senators, please contact Phil at goglas@hmcw.org. He will provide you with their name and e-mail address.

Congressional deadlines are fast approaching, so you must reach out to your Senators this week (the first week of March) or early next week to have an impact.

Email Template

Dear Senator ____________,

Senator Debbie Stabenow is currently circulating a Senate sign on letter in support continuing to include “Focal Segmental Glomerulosclerosis” (FSGS) in the list of conditions deemed eligible for study through the Department of Defense Peer-Reviewed Medical Research Program (PRMRP) during the FY 2017 appropriations process. On behalf of FSGS impacted families across the state, please contact Sam Schuiteman in Senator Stabenow’s office at Sam_Schuiteman@stabenow.senate.gov or 4-4822 to join this important letter by COB Friday, March 11^th.

FSGS is a rare and devastating kidney disease that is a leading cause of end-stage renal disease (ESRD). Nearly 30,000 veterans suffer from ESRD and an additional 3,000 veterans are expected to reach ESRD each year with significant health disparities among African American due to variants of the APOL1 gene. In addition, researchers suggest there are new opportunities for investigating FSGS in the military population with respect to environmental exposures. More needs to be done to improve our understanding of the impact of FSGS among our military personnel and veterans. FSGS has been part of the PRMRP for some time, including FY 2016, and continued participation will lead to further scientific progress.

[OPTIONAL: Briefly tell your FSGS story in 2-4 sentences]

Thank you for your time and your consideration of this request.

Sincerely,

[Your Name]

[Your Address]

Every gift has a story.

November 23, 2015 by Lauren Eva Leave a Comment

Use new eCards to tell someone you love that you care about them.

2015 Countdown to a Cure – THANK YOU!

November 20, 2015 by Jessica Martin Leave a Comment

We’ve said “hello” and “goodbye” to another Countdown to a Cure…

From the beautiful scenery of New York City’s Chelsea Piers, to the heartfelt speeches delivered by NephCure’s beloved family, the Jones’ and honoree, Olympic athlete Aries Merritt, this event was truly the “Chance of a Lifetime” to make a difference in the fight against Nephrotic Syndrome. The success of Countdown has always been measured by the generosity of the many hundreds in attendance who consistently show support for NephCure and this year, our expectations were overwhelmingly exceeded, for which we could not be more grateful. You are changing the story.

Energies were high and attendees were excited, lighting the way for one of the most successful galas in NephCure history. “Fund a Cure” donations blew expectations out of the water, bidding was at a high and the second annual game of “heads or tails,” brought a touch of silliness to an evening surrounding a very serious cause. Emcee, Moody McCarthy, along with “Asbury Fever,” a Bruce Springsteen Tribute band, kept the party going all night long with many moments filled with laughter, dancing and mingling.

As always, we want to thank the committee who worked so hard to put this event together and the volunteers who generously gave up their time to help this event flourish into a huge success. The 2015 New York Countdown to a Cure raised over $750,000 and many left the event feeling inspired and hopeful.

Finally, thanks to YOU. To each of you reading this who’ve decided to join us in this fight.

We can’t do this alone.
We need you, we’re grateful for you and we thank you.

NKI Grand Rapids Walk October 18th

October 15, 2015 by Jayne Drew Leave a Comment

Grand Rapids, MI – The NephCure Kidney Walk returns for the 4th year to the Grand Rapids area, Sunday, October 18^th. The walk brings together area patients, friends, and family dedicated to raising awareness and vital funds to find improved treatment options and a cure for primary Chronic Kidney Diseases, including FSGS and nephrotic syndrome.

The walk will be held at Millennium Park, 1415 Maynard Ave SW in Walker, MI. Registration opens at noon and the walk begins at 1 p.m. There is no registration fee, but teams are encouraged to raise at least $1,000, with a total event goal of $10,000. The event is open to the public, but registration is encouraged in advance at support.nephcure.org/GrandRapids.

The NephCure Kidney Walk is especially meaningful to local residents and co-chairs Heather Luchies and Tressa Hollinger. Both of whose lives and families are impacted by these conditions.

“Being involved in the planning of this walk is my way of giving back to NephCure, who are working hard to find a cure for my disease”, says Heather. “Plus I find it helps “embrace” my disease rather than sit on the couch and do nothing about it.”

Both Heather & Tressa will be on their local Fox 17 station this Saturday morning at 7:40 to do a live interview about the NephCure Kidney International Walk this Sunday.

“This is what I’ve wanted since we partnered with NephCure and started the walk” says Tressa. “More awareness to find a CURE! Let’s rock this Heather Luchies!”

To register or donate to the NephCure Kidney Walk, please visit support.nephcure.org/GrandRapids or contact Jayne Drew at jdrew@nephcure.org