Sunday, March 25, 2018

PKD Foundation Update

PKD Foundation

From PKD Foundation

Washington Summary March 2018


Advocacy Alert

Last week, the PKD Foundation asked its advocates to urge Congress to keep PKD on the list of eligible research programs for Fiscal Year 2018 under the Department of Defense’s Congressionally Directed Medical Research Program (CDMRP). Thank you to everyone who took action! We are continuing to push and monitor this situation and are also actively working to ensure inclusion for Fiscal Year 2019.

Health Research and Other Spending Programs

Congress continues to work on a spending proposal that will keep government departments and agencies (such as NIH) open for the rest of FY18. The latest short-term funding law expires on March 23.

At this writing, neither the House nor Senate Appropriations Committee has released details on how much NIH and other health research programs will receive for this fiscal year, which ends on September 30. That funding level will be the baseline for determining future health research funding.

At the same time, the House Appropriations Committee is beginning to work on the FY19 budget. On March 15, HHS Secretary Alex Azar appeared before the Labor/HHS Subcommittee to explain the administration’s proposal.

Affordable Care Act (ACA) Cost Sharing

Congress continues to discuss ways to stabilize the ACA individual insurance market by providing cost-sharing reduction (CSR) funds. The latest proposal from Sens. Lamar Alexander (R-TN) and Patty Murray (D-WA) would fund the cost-sharing program, give states more waiver flexibility, and fund reinsurance programs. Sens. Susan Collins (R-ME) and Bill Nelson (D-FL) have their own proposals to stabilize the individual insurance market through direct funding and a reinsurance program similar to previous state-based risk pools. Whether any of the efforts succeed will depend on convincing the House of Representatives.

Insurance Coverage for Essential Health Benefits and Pre-Existing Conditions

The administration has announced additional proposals that would revise or replace major ACA provisions. In addition, some states are taking their own actions.

On Feb. 20, the administration announced that it would allow the sale of short-term (less than a full year) health insurance policies. These plans would not have to cover people with certain medical conditions. The plans also could charge higher premiums for people with health issues. Once the 60-day comment period ends, HHS will review the comments and issue a final regulation.

In late January, Idaho announced that it would permit the sale of non-ACA compliant policies to its residents. Under these policies, insurers could charge higher premiums for people with preexisting conditions. On March 8, CMS advised Idaho officials that the state’s plan fails to enforce essential ACA provisions. Idaho officials believe that their proposal can be modified to comply with ACA, and they may submit a modified proposal.

Elsewhere, the Iowa senate has passed a bill that would allow the Iowa Farm Bureau and Wellmark to sell non-ACA-compliant policies.

If you receive your health insurance through a non-ACA plan, you still should monitor activities in your home state.

Bills of Importance to the PKD Community

Living Donor Protection Act (HR 1270/no Senate bill yet) would remove barriers to living organ donation. Rep. Jerrold Nadler (D-NY) and Rep. Jaime Herrera Beutler (R-WA) introduced the bill. PKD and several other patient groups have signed a letter urging House Members to cosponsor HR 1270.
The OPEN Act (HR 1223/ S 1509) would make it easier for companies to repurpose approved drugs for treating rare diseases. Reps. Gus Bilirakis (R-FL), GK Butterfield (D-NC), and Mike McCaul (R-TX) introduced HR 1223. Sens. Orrin Hatch (R-UT) and Robert Menendez (D-NJ) introduced S 1509.

Say Thanks to Supporters

The following Senators and Members of Congress have cosponsored either the OPEN Act or the Living Donor Protection Act since the previous newsletter. If any of them represent you, please say “thank you” the next time that you contact them.

HR 1223, the OPEN Act
Rep. Christopher Smith (R-NJ)
Rep. Alcee Hastings (D-FL)
Rep. Barbara Comstock (R-VA)
Rep. Brian Fitzpatrick (R-PA)
Rep. Michelle Lujan Grisham D-NM

Stay Alert

When the time comes, we will ask PKD advocates to immediately contact their elected officials to protect your interests. Your voice needs to be heard.

Sunday, March 18, 2018

PKD Fundraising Atlanta: Day of the Juice, Bionic Kidney Trials in 2018, Growing Veins for Dialysis

PKD Fundraising

From Paste Magazine, By Jim Vorel
Talking All Things Hoppy With the Organizers of Atlanta’s New “Day of the Juice” Festival

Talking All Things Hoppy With the Organizers of Atlanta’s New “Day of the Juice” Festival



Paste: Who are your charities?

Lowenberg: There’s two charities, the Polycystic Kidney Disease Foundation and the Georgia Transplant Foundation. Eric Levin, one of our founders, has had a kidney transplant, so those charities are pretty close to home for Modern Hops as a whole.


Finding a niche for a first-time beer festival is no easy feat. Organizers need to hit upon a concept that captures some element of the current zeitgeist, while also dealing with a lack of brand recognition for a festival whose name or production company are unfamiliar to attendees. For the same reasons, it’s difficult to get the most hyped and desirable breweries to attend a new festival, just as it’s hard to book popular bands at a first-year music fest. On some level, if you’re trying to start a new festival from scratch (especially in a city with no shortage of fests), the deck is stacked against you.

Enter, Modern Hops and Atlanta’s upcoming (March 31) Day of the Juice charity festival. Rather than simply replicating the format of one of Atlanta’s more established beer festivals (which are numerous), this indie craft beer distributor is doubling down on a more esoteric concept—a showcase of juicy (and mostly hop-forward) beers from a collection of well-curated, buzzworthy, but relatively smaller stature breweries. Yes, it’s a festival of hoppy, hazy and juicy … but without a Tree House or Trillium in sight. Rather, the focus is on younger breweries striving to be the nextTree House or Trillium.

Considering this fest is happening in Paste’s backyard, I was able to sit down for a short chat with one of the organizers, Michael Lowenberg, Modern Hops’ “flavor chaser extraordinaire.” Fun fact: After hearing him say those words, I inquired if that’s the title that appears on his business cards. As it turns out: Yep. It certainly is. Lowenberg is one of four partners in the indie distributor, along with co-founders Eric Levin and Barrett Hoard, and partner Philip Barnes, all of whom are working to make Georgia’s first brewery-hosted festival a reality. Modern Hops also collaborated with the founder of festival sponsor Craft Connect, Elias Spartis, to create the brand direction and artwork.




Artificial Kidney Research

From Snopes, By Alex Kasprak

Will Bionic Kidneys Replace Dialysis by 2020?

Kidneys are responsible for removing harmful chemicals and impurities from our blood — the filter in the hot tub that is our body’s circulatory system. When kidneys fail, a condition known clinically as end stage kidney failure, a patient currently has two options: a kidney transplant, or dialysis.

Kidney transplants are challenging to obtain, due primarily to a massive shortage of living kidney donations. For every person who received a kidney transplant in 2016, five patients did not, and 4000 people died on the waiting list that year.

For those waiting for a transplant, dialysis is the only solution, but it is an imperfect one. Dialysis is a process in which blood is mechanically filtered to remove excess water, solutes and toxins from the blood, mimicking the job of a kidney. Compared to the real deal, it is far from a perfect replacement, as reported in a 2017 Wired feature:

Dialysis does a decent job cleansing blood of waste products, but it also filters out good stuff: salts, sugars, amino acids. Blame the polymer manufacturing process, which can’t replicate the 7-nanometer precision of nephrons — the kidney’s natural filters.

Making dialysis membranes involves a process called extrusion, which yields a distribution of pore sizes — most are about 7nm but you also get some portion that are much smaller, some that are much larger, and everything in between. This is a problem because that means some of the bad stuff (like urea and excess salts) can sneak through and some of the good stuff (necessary blood sugars and amino acids) gets trapped.

Because of these realities, there has been a great deal of Internet buzz over a project out of the University of California, San Francisco named The Kidney Project. Lead by UCSF bioengineering professor Shuvo Roy and professor of medicine at Vanderbilt University William Fissell, it has the ultimate goal of creating an artificial kidney approved by the Food and Drug Administration that could be installed with a minimally invasive surgery and function for an indefinite amount of time.

The challenge, essentially, is to create a molecular-scale filter that the body can easily push blood through without the need for additional power, that does not cause the blood to clot, and that allows for the passage of “good stuff” while still blocking out the “bad stuff.”

The solution the UCSF team has come up with involves two components: A nano-engineered silica filter to remove dissolved toxins, sugars, and salts, and a bioreactor containing live kidney cells that allow the body to resorb the sugar, salt, and water removed by the filter. A 2018 press release from the NIH’s National Institute of Biomedical Imaging and Bioengineering describes the current conceptual design:

The experimental device is designed to accommodate up to a liter of blood per minute, filtering it through an array of silicon membranes. The filtered fluid contains toxins, water, electrolytes, and sugars. The fluid then undergoes a second stage of processing in a bioreactor of lab-grown cells of the type normally lining the tubules of the kidney. These cells reabsorb most of the sugars, salts, and water back into the bloodstream. The remainder becomes urine that is directed to the bladder and out of the body.

Advances in silicon nanotechnology spurred by electronics manufacturing gave the researchers the ability to manufacture silicon pores that consistently have the precise size and shape necessary to reduce stress on the blood cells and, as a result, clotting, Roy told Wired in 2017. On the cellular side, bioreactors that utilize living kidney cells have been tested successfully in animal studies since 1999.

The project, which aims to combine both of these elements into a single device, received a boost in 2015 when the researchers received a $6 million grant from the NIH, and the FDA included it an initiative aimed at fast-tracking the development, evaluation, and review of certain medical devices. The next steps will involve humans:

Clotting is the biggest concern, so they’ll surgically implant the device in each participant’s abdomen for a month to make sure that doesn’t happen. If that goes well they will do a follow-up study to make sure it actually filters blood in humans the way it’s supposed to. Only then can they combine the filter with the bioreactor portion of the device […] to test the full capacity of the artificial kidney.

While some reports suggest that clinical trials began in 2017, Roy told us via e-mail that their hope was that clinical trials could begin later in 2018. He is optimistic about the prospect of bringing the device to market before the close of the decade, however. “We are hopeful that the first clinical trial will begin this year. If all goes well and funds are available, we could be on the market as early as 2020,” he said.




From Engadget, by Daniel Cooper

Future dialysis patients could grow their own artificial veins


I rarely think about kidney failure, and when I do, it's almost always in the context of a charity appeal from my local hospital. Dialysis machines are the primary way that people with kidney disorders survive until a donor organ can be found. Going to a hospital multiple times a week to have your blood cleaned never seemed like it was a fun way to spend one's time, either. But after talking to Aditlys CEO Silvére Lucquin, I learned these trips to the hospital are not the worst part of the process.

Lucquin's company has been working on a polymer-based implant that can be wired into a person's blood vessels. The implant is, essentially, a hollow scaffold built from a new plastic polymer that encourages tissue growth. Once inserted into a person's veins, their own bodies begin growing a new blood vessel around the artificial one. The implant then dissolves after a couple of months. Leaving behind an entirely new link that can be connected up to a dialysis machine.

The implant itself is harnessing a variety of doctrines, including electrospinning and supramolecular chemistry. These techniques have been married under the new process of endogenous tissue restoration which has been pioneered by a company called Xeltis. That company, however, has used the process to restore damaged heart valves, leaving Aditlys to experiment with its vascular implant.

Our kidneys, you see, are filters that clear out the excess fluid and junk that lingers in our bloodstream, turning it into urine. If a person's kidneys shut down, then the bad stuff in their blood builds up, which can often be fatal. Until a transplant can take place, patients have to visit hospitals every few days to have their blood cleaned. That's where the dialysis machine comes in, which pulls blood out of a vessel, filters it and pushes it back into their bodies.

In emergencies, that can be carried out using a catheter that's been inserted into a blood vessel, but that's not possible on a regular basis. Long-term dialysis users instead need to have a plastic tube implanted into their arm, either an arteriovenous graft or an arteriovenous fistula -- essentially an artificial junction. Regular veins simply can't cope with having thick needles shoved in on a biweekly basis and all that blood taken out. Not to mention that fistulas help improve the blood flow, making dialysis easier.

But "when you leave plastic implants in the body," explained Lucquin, "they tend to occlude [block] quite easily." His research claims that these blockages take place around 50 percent of the time, often within the first year. That's not the only problem because, according to a paper out of Bayer College of Medicine, these implants are a hotbed of germs. Researchers George Nassar and Juan-Carlos Ayus say that the pipes have "repeatedly shown to be a risk factor for bacteremic and nonbacteremic infections."

Worse still, even in a best-case scenario, fistulas fail in almost 40 percent of implantations for a variety of reasons. Even if it doesn't, they normally have to be replaced once every two years or so, and that means regular trips back to the vascular surgeon. Those patients, explained Lucquin, "after a decade of dialysis and all that decay, means that it's really complicated to find a clean place to put a new graft or fistula."

Now, the hope is that with Aditlys' new device, many of these issues will go away and most patients will need only one implant. Or, at the very worst, a couple, but that's not something that Lucquin could comment on publicly. After all, things are at such an early stage that it's not clear if the theory and the practice really match up. But the CEO did point out that even if the vessels do fail, those failures won't nearly be as frequent as they are right now.

Of course, it's going to be several years worth of clinical testing before the company can market the implant to patients. And it'll take a while before vascular surgeons decide to attempt to use the tool in place of what currently works. But if successful, some of the many additional pains that people with kidney failure have to deal with may be a thing of the past.

Sunday, March 4, 2018

PKD Clinical Trial: bardoxolone, Dialysis: Self-Serve, PKD Fundraising, Waiting for a Kidney

PKD Research

From Global Newswire

Reata Provides Program Update on Phase 2 Rare Renal Clinical Trials


First patient enrolled for all cohorts of PHOENIX

First data from PHOENIX expected 2H18

Retained benefit analysis from Phase 2 portion of CARDINAL expected 3Q18


IRVING, Texas, Feb. 27, 2018 (GLOBE NEWSWIRE) -- Reata Pharmaceuticals, Inc. (Nasdaq:RETA) (Reata or Company), a clinical-stage biopharmaceutical company, today provided guidance on the timing of data announcements from the ongoing Phase 2 PHOENIX and CARDINAL trials of bardoxolone methyl (“bardoxolone”) in rare forms of chronic kidney disease (“CKD”).

The Phase 2 PHOENIX program is studying bardoxolone in patients with autosomal dominant polycystic kidney disease (“ADPKD”), IgA nephropathy, focal segmental glomerulosclerosis (“FSGS”), and CKD associated with type 1 diabetes. Approximately 25 patients per cohort will receive bardoxolone open-label, orally, once-daily for 12 weeks. The purpose of this study is to determine the safety and efficacy of bardoxolone, and the primary efficacy endpoint is change from baseline in eGFR after 12 weeks of treatment. Each cohort of patients is being independently enrolled and analyzed, and each has now enrolled at least one patient. The Company anticipates that initial data from one or more PHOENIX cohorts will be released during the second half of 2018.

CARDINAL is a Phase 2/3 study of bardoxolone in patients with CKD caused by Alport syndrome. The Phase 2 portion of CARDINAL enrolled a total of 30 patients to assess the safety and efficacy of once-daily, oral administration of bardoxolone, and its primary efficacy endpoint was change from baseline in estimated glomerular filtration rate (eGFR) at week 12. Full primary endpoint results from the study were reported in November 2017 after all patients had reached week 12. Patients remain in the study for up to two years, and eGFR will be measured at 52 weeks following 48 weeks of treatment and 4 weeks of drug withdrawal (“retained benefit”). The Company expects to report the week 52 retained benefit analysis from this study in the third quarter of this year.

Results from the 52-week retained benefit analysis are relevant to the ongoing Phase 3 portion of CARDINAL, a double-blind, placebo-controlled trial enrolling up to 150 patients worldwide. This Phase 3 study can support accelerated approval by the FDA based upon an improvement in eGFR following 48 weeks of once-daily treatment and 4 weeks of drug withdrawal. After this retained benefit analysis, patients will continue on their original study treatment for another 48 weeks, and full approval can be supported by a retained benefit at 104 weeks following a second 4-week drug withdrawal. Prior trials in patients with other forms of CKD have demonstrated that improvements in eGFR are durable for up to two years, and the change in eGFR after 12 weeks correlates with changes at one year on-treatment and post-withdrawal.

“Diverse forms of CKD are driven by a common final set of inflammatory pathways that bardoxolone targets,” said Colin Meyer, M.D., Chief Medical Officer of Reata. “Treatment with bardoxolone has resulted in clinically meaningful increases in kidney function in patients with Alport syndrome, CKD caused by type 2 diabetes, and CKD associated with pulmonary hypertension, and we hope to demonstrate similar efficacy in these additional types of CKD being studied in PHOENIX. We anticipate that bardoxolone may complement commonly used therapies that modestly affect progression in these diseases, which have no FDA-approved treatments.”

About Bardoxolone Methyl

Bardoxolone methyl is an experimental, oral, once-daily activator of Nrf2, a transcription factor that induces molecular pathways that promote the resolution of inflammation by restoring mitochondrial function, reducing oxidative stress, and inhibiting pro-inflammatory signaling. In addition to CARDINAL and PHOENIX, bardoxolone methyl is currently being studied in CATALYST, a Phase 3 study for the treatment of connective tissue disease associated pulmonary arterial hypertension. The FDA has granted orphan designation to bardoxolone methyl for the treatment of Alport syndrome and pulmonary arterial hypertension.



PKD Fundraising

From Auburn Journal, by Aurora Sain

Corks for a cure

Four families volunteering for the Sacramento Chapter of the PKD Foundation, who are emotionally and personally touched by polycystic kidney disease, are hosting the third annual Corks for a Cure.

The events mission is to raise funds and awareness for polycystic kidney disease (PKD), in which there is no treatment and no cure.

“This is such a fun and meaningful event and I hope you'll help us raise awareness and encourage our community to attend,” said Auburn resident Valen Keefer.

One Placer County family, the Lusby’s, are helping to double the impact of the event this year by matching dollar for dollar raised up to $25,000.

Kari Lusby’s 9-year-old son, Honor, has been enduring chronic pain since the age of 2 and it was not until 2013 that they discovered it was polycystic kidney disease. After Kari and her family learned that there is no treatment or cure for PKD, they established Crusade for Cures Foundation. The Lusbys are determined to end PKD so that Honor and millions of others battling this disease can live happy and healthy lives. They are excited to support this year’s Corks for a Cure event and help double the impact with all donations going to fund life-saving medical research.

Polycystic kidney disease is one of the most common, life-threatening genetic diseases. PKD is when fluid-filled cysts develop and enlarge in both kidneys, eventually leading to kidney failure. The average size of a normal kidney is a human fist, polycystic kidneys can get much larger, some getting as large as a football and weighing up to 30 pounds each.

More than 50 percent of people with PKD will develop kidney failure by the age of 50. Once a person has kidney failure, dialysis and transplantation are the only options to treat the damage the disease has caused. Parents have a 50 percent chance of passing the disease to each of their children.

PKD can also develop spontaneously; about 10 percent of the people diagnosed have no family history of the disease. The Sacramento Chapter Coordinator of the PKD Foundation and Corks for a Cure organizer, Julia Adams of Fair Oaks, knows this firsthand. Her daughter, Elizabeth, developed PKD spontaneously and was diagnosed when she was 2 years old and faced kidney failure in her early 20s. Elizabeth’s dad, Rick, was her living kidney donor.

“I started volunteering for the PKD Foundation and raising funds so other families don't have to hear, "Your child has PKD, there is no treatment and no cure,” Adams said.

The event will feature a silent auction, wine, hors d’ oeuvres and dancing.




Living with PKD

From Goldendale Sentinal, By Max Erikson


Goldendale resident Joanne Davenport has been struggling with Polycystic Kidney Disease (PKD) for many years and is currently one of the 100,000 people nationwide on a waiting list that one day could provide a donated kidney to save her life.

PKD is a hereditary disease that causes numerous fluid-filled cysts to grow in the kidneys. As the cysts grow, it damages the kidney function to the point of kidney failure, also known as renal disease. Kidneys are the organs in the body that filter blood, maintain healthy fluid levels, help make red blood cells, and help keep blood pressure under control.

According to the National Kidney Foundation, 600,000 people in the United States are currently fighting the disease, and one in three American adults are at risk for PKD. Risk factors include having diabetes, high blood pressure, family history of kidney failure, and being age 60 or older.

Davenport has lived in Goldendale for 26 years and was a License Practical Nurse (LPN) at Klickitat Valley Health for 14 years before she got sick. For Davenport, and many others, a kidney transplant is their best hope for living a longer healthier life, but finding a donor with the correct blood type is difficult.

“I have known I’ve had the disease since I was about 30,” Davenport says. “But when I turned 40, it really started to progress. After the last five years of fighting it, I’m at the point where if I don’t find a donor, I will need to start dialysis.”

There are two ways a person with PKD can receive a kidney for transplant. One way is to be on the waiting list to receive a kidney from a deceased organ donor. However, the average wait time for a kidney from a deceased person is three to five years. Many with PKD don’t live long enough to exercise that option.

The other option is to receive a kidney from a person who is alive and willing to donate. That is known as a living donation. Living organ donation programs were developed as a direct result of the critical shortage of deceased donors. Living donations give the best chance at survival for individuals waiting for a transplant, and oftentimes it can be a donation from a close friend or family member. In 2015 6000 living donations were made in the United States.

Living donations are a faster alternative if the right match can be found, which requires matching the correct blood type with the donor and the recipient. Davenport has recently made a public request to the Goldendale community to see if there is anybody interested in becoming a living donor or willing to help find someone who will.

“I’m hoping that there will be someone with a gracious heart willing to help and give me a chance to live,” Davenport says.

Davenport has been working with the Virginia Mason Medical Center in Seattle that has been providing kidney transplants since 1972. Virginia Mason has a living donation program, and for those who might be interested in learning more about donating, Davenport encourages people to visit their website at http://www.virginiamason.org/living-donation.




From Lynn Journal, Boston

John Nucci Faces the Biggest Battle of His Life:Respected Former Suffolk County Clerk Magistrate in Need of a Kidney Transplant

Throughout his political career, whether it was running for Boston School Committee, City Council or Suffolk County clerk magistrate, John Nucci has relied on the help of others to help him win.

Now, Nucci needs the help of others more than ever to help him win the biggest battle of his life.

In his 30s, after the passing of his father following complications of Polycystic Kidney Disease, Nucci found out that he had inherited the same genetic disorder where the renal tubules become structurally abnormal, resulting in the development and growth of multiple cysts within the kidney.

The diagnosis was grim and for the last three decades Nucci lived knowing that someday his kidneys would begin shutting down.

“I was tested in my 30s to find out if I had inherited the disease and I was told then that I did in fact have cysts on my kidneys,” said Nucci. “As these cysts form over the years your kidney function gets lower and lower. My doctors at MassGeneral have been watching it every year since I was in my 30s and in the past year my levels have been dropping fast. That’s the nature of this disease. Once you’re diagnosed you’re never at full kidney function but you can live at 30 percent kidney function for years. Once it drops down to about 15 percent is when they say ‘it’s time.'”

For Nucci ‘it’s time’ meant starting the process of finding a living kidney donor in order to potentially avoid dialysis treatments and the further deterioration of his failing kidneys.

“I’m months away from dialysis,” said Nucci. “It’s inevitable, it’s going to come. But while dialysis is a life saver it’s not a good quality of life. The other problem is that if I have to go that route the longer I’m on dialysis waiting for a donor the less likely a kidney transplant will work or last.”

No stranger to rallying the troops in Eastie and citywide, Nucci, his wife, Peggy, and their three sons, John, Michael and Danny have launched a campaign in local Boston newspapers, on social media and through the relationships Nucci has cultivated over the years to find a living donor match.

Nucci’s wife and his boys have been quarterbacking the campaign to find a living donor as soon as possible. His three boys all tested positive for Polycystic Kidney Disease and were automatically eliminated as donors so they switched gears and began reaching out to other friends and family to find a match.

“I’m not social media savvy so my wife and the boys have been doing a tremendous job getting the word out on Facebook, making phone calls and reaching out to anyone and everyone,” said Nucci. “Finding a living donor is not the hard part and I have had dozens of people step up and get tested and for that I’m beyond grateful. A lot of people have already gone through the process of extensive testing at Mass General but for one reason or another they were eliminated. The problem isn’t so much finding someone who wants to donate it is finding a match. A direct donor has to be a direct blood type (Nucci is Type O) and Mass General is very diligent making sure that the donor is healthy and their kidneys are healthy.”

Nucci said waiting for a deceased donor could take seven to nine years and at his age the direct donor is really the only viable option.

“It’s a race against time,” he said. “I’m ready in terms of my own health. I am a healthy candidate for the transplant it’s just finding the match.”

In the meantime, his son, John, is running the Hyannis Sprint Triathlon to raise money and awareness for Polycystic Kidney Disease.

“In addition to raising money for this great cause, I’m also hoping to get the word out to as many potential donors as possible,” said John. “I know that it’s a huge ask, and it’s not something I would put out there if it wasn’t absolutely necessary, but we’re hoping that someone out there is willing and able to help him. I didn’t even know what a reasonable amount to target would be, and I’m honestly overwhelmed by the support. We’re raising money and getting the word out to so many more people and potential donors than my family and I possibly could’ve on our own, and it seriously means the world to all of us.”

Nucci’s wife Peggy has also been overwhelmed by the amount of support so far.

“I really want everyone to know how much we appreciate everyone’s willingness to help out with our efforts to find the matching kidney donor which John truly needs right now,” said Peggy.

For now, Nucci is trying not to think to much of what lies ahead and focus solely on coming out of this battle victorious, like he did so many times in his political career.

“I’m convinced I’m going to beat this,” said Nucci. “I have never won anything in my life without the help of my friends. My entire political life was spent relying on friends and supporters to win, and this is no different. In many ways it’s like another campaign and I’m going to need that support to win.”

If you or anyone you know is interested in becoming a donor, there is a quick and confidential online screening form that can be found at www.mghlivingdonors.org.




Dialysis Technology

From Medical Design & Outsourcing, By Danielle Kirsh

How self-serve medical devices could make patients feel in control

Intimidating medical devices can cause anxiety for patients — in the same way that people can experience “white coat hypertension” when seeing a doctor.

Devices that are smaller and more patient-centered could help patients feel better about their treatment and make them feel like they’re in charge, according to Leslie Trigg, CEO at Outset Medical (San Jose, Calif.).

“Because you are in control of it, it’s a different experience, even if it takes the same amount of time. It’s really about control. I think when you strip that all away, what really matters to us as human beings is the value we place on being independent and in control of our own destiny. And that’s really on a human level what the driving philosophy is behind Tablo,” Trigg said.

The Tablo is Outset Medical’s 3-foot-high kidney dialysis machine. It makes clean water, produces dialysate, takes blood pressure and delivers medication — all in one compact design. The machine is no taller than a desk and features a touchscreen interface meant to make treatment easier in clinics and hospitals. Its low-profile design is much smaller than other dialysis machines that require special filtration centers and large machines in the office.

“Tablo was launched in some of the existing 6,500 dialysis clinics [in the U.S.] to enable patients to do dialysis on their own. And this idea – we call in-center self-care – is really akin to self-serve dialysis. We kind of just borrowed on the concept of self-serve in retail,” Trigg said. “We are borrowing from that retail concept of self-care and bringing it into the dialysis setting because the system is so easy to use for the average patient. This is an opportunity for the patient to come in and set up Tablo on their own and manage their treatment.”

While Tablo is not currently available for home use, personalizing treatment and making it convenient helps patients feel at ease with their conditions. There are certain chemotherapy treatments that can be done at home, and the National Institutes of Health reports that it can improve patient outcomes and improve the quality of care.

Bringing the treatment closer to the patient is important since people are living longer and it provides a cost-effective solution to different treatments.

“People are living longer,” Trigg said. “If people are able to manage cancer as a chronic disease rather than an acute terminal episode, you have an opportunity – and obviously a responsibility – to make those sorts of chronic [therapies] that are maximumly cost-effective for patients and maximumly cost efficient for payers and providers.”