PKD Gift of Life: Starbucks; Effects of Coffee on PKD, Australia Lists Jinarc (Tolvaptan)

PKD Treatment

From The Pharma Letter

Australia to list Jinarc on PBS

The Australian government will list a new medicine for adults with genetic kidney disease on the Pharmaceutical Benefits Scheme (PBS), saving patients around A$23,000 ($16,428) a year.

Jinarc (tolvaptan), which was developed and is marketed by Japanese drugmaker Otsuka Pharmaceutical’s (TYO: 4768), will be available through the PBS from January 1, 2019. It is the first effective drug treatment for autosomal dominant polycystic kidney disease (ADPKD) on the PBS.

The disease is a genetic, progressive and painful disease in which cysts develop and grow in the kidneys. Most people with this disease will need dialysis or a transplant by the time they are 60. There are also multiple complications from the disease, which may include hypertension, chronic and acute pain, repeated urinary tract infections, and depression as the cysts grow and quality of life declines.

The PBS listing of Jinarc provides new hope to people diagnosed with this disease that they may be able to have a better quality of life, with potential for improvement for their kidney and overall health.

Around 900 patients each year are expected to benefit from the listing. Instead of A$23,600 per year they will be able to obtain this innovative new medicine for just A$40.30 per script, or A$6.50 for concessional patients. While 10,000 Australians are estimated to have the disease, most are not diagnosed until the disease is advanced.




Gift of Life

From Time Magazine, By MELISSA LOCKER
How a Starbucks Barista Helped a Customer Find a New Kidney

Sometimes a Starbucks barista has something to more offer than a Frappuccinoand a smile.

Nicole McNeil, a barista at the DuPont, Wash., Starbucks knew something was going on with long-time customer Vince Villano when he seemed down during his trips to her store..

So she she sat down with him and asked him what was wrong, KIRO-TV reports. Turns out, Villano, an Army veteran, needed a new kidney. He was suffering from polycystic kidney disease – his kidneys were only functioning at about 4% – and he was facing a life on dialysis. The debilitating condition has no treatment and the only option is a kidney transplant.

The story stuck with McNeil, who went home and shared the heart-breaking tale with her husband, Justin McNeil. The story struck a chord with Justin, too, and he ultimately decided to donate a kidney to Villano. “I said, ‘I’ve got a kidney, you know, we could do this. I think I’m willing to do that,’” Justin McNeil told KIRO. “It didn’t take long.”

The couple gave Villano the news and as they embarked on the long journey of medical testing to confirm the donation would work, the three struck up a close friendship. Villano and Justin McNeil, who is also an Army veteran, spent months traveling back and forth from DuPont, which is between Olympia and Tacoma in Washington State, to the University of Washington Medical Center in Seattle.

“In general, having them as friends, family, I wouldn’t want it to not be this way. I can’t imagine not having them in my life,” Villano told KIRO.

Both Villano and Justin McNeil had their surgeries on Wednesday and, according to an update posted by Starbucks, “The doctors told [Nicole McNeil] the kidney Justin McNeil donated to Villano looks great, and a post-surgery ultrasound confirmed it is functioning well.”

Living with PKD

From BMC Nephrology

Relationship between caffeine intake and autosomal dominant polycystic kidney disease progression: a retrospective analysis using the CRISP cohort

Abstract

Background

Caffeine has been proposed, based on in vitro cultured cell studies, to accelerate progression of autosomal dominant polycystic kidney disease (ADPKD) by increasing kidney size. Since ADPKD patients are advised to minimize caffeine intake, we investigated the effect of caffeine on disease progression in the Consortium for Radiologic Imaging Studies of Polycystic Kidney Disease (CRISP), a prospective, observational cohort study.

Methods

Our study included 239 patients (mean age = 32.3 ± 8.9 ys; 188 caffeine consumers) with a median follow-up time of 12.5 years. Caffeine intake reported at baseline was dichotomized (any vs. none). Linear mixed models, unadjusted and adjusted for age, race, sex, BMI, smoking, hypertension, genetics and time, were used to model height-adjusted total kidney volume (htTKV) and iothalamate clearance (mGFR). Cox proportional hazards models and Kaplan-Meier plots examined the effect of caffeine on time to ESRD or death.

Results

Caffeine-by-time was statistically significant when modeling ln(htTKV) in unadjusted and adjusted models (p <  0.01) indicating that caffeine consumers had slightly faster kidney growth (by 0.6% per year), but htTKV remained smaller from baseline throughout the study. Caffeine consumption was not associated with a difference in mGFR, or in the time to ESRD or death (p > 0.05). Moreover the results were similar when outcomes were modeled as a function of caffeine dose.

Conclusion

We conclude that caffeine does not have a significant detrimental effect on disease progression in ADPKD.

PKD Treatment: Concern for New Medicare Policy, Lixivaptan in Phase 2 Testing

PKD Treatment Policy

From Commercial Appeal, Karyn Waxman, Guest columnist

New Medicare policy will harm patients like me | Opinion

Every day, 12.5 million Americans struggle with polycystic kidney disease, a disease that causes numerous cysts to grow in the kidneys, slowly reducing kidney functionality and possibly leading to kidney failure. I am one of them.

As the Memphis chapter founder and coordinator for the PKD Foundation, I work to help provide a forum for patients who want to find treatments and cure this disease.

That’s why I was alarmed by the Centers for Medicare and Medicaid Services’ new Medicare Advantage Part B step therapy policy, which would begin implementation next year.

Under step therapy, or “fail first,” health insurers require patients to use more cost-effective drugs before using more complex drugs, if the first drug doesn’t work. While this method may work for other parts of Medicare where generic and name brand drugs are indistinguishable, Medicare Part B drugs are not compatible with step therapy drugs.

Part B covers treatment for many serious conditions like PKD and relies on treatment that is tailored to the biology of each patient.

If health insurers forced doctors to prescribe less effective Part B treatments in the name of cost cutting, their zeal for cutting corners may backfire, leading to an increase in visits to the emergency room by Medicare Part B patients who respond poorly to the less effective treatment. As a result, the cost of treatment would rise exponentially, impacting the entire health care system and increasing costs for consumers.

This method also violates the trusted relationship between a doctor and patient. Doctors who treat patients with PKD often forge relationships with those patients that last years, forming a partnership to find the right combination of drugs that will allow each patient to manage their condition and retain a normal quality of life.


When an insurance company steps in on this years-long process in order to dictate which drugs it will allow, it throws years of hard work and collaboration out the window, jeopardizing both the doctor-patient relationship and the health of the patient.

This issue is incredibly personal for me. As someone who struggles with PKD, I anticipate undergoing a kidney transplant from a living donor in the next year. In order to make sure my body doesn’t reject the kidney, I’ll need to undergo a specific regimen of medications, tailored to my own body, for the rest of my life. I’ll be immunosuppressed, meaning I’ll be more susceptible to infections and certain types of cancer.

I can’t afford to “fail first,” or to gamble with drugs that an insurance company might choose for me based off of their own bottom line. This backwards approach takes away the rights of the doctor and the patient, and too often leads to increase in costs on the entire healthcare system.

Patients who battle PKD require timely care and should be permitted to opt out of step therapy, if their health or financial situations change. We simply can’t afford to try a cheaper, riskier treatment for a prolonged period with little success when there is an existing treatment that works. While health insurance companies want to pinch pennies, ultimately, we are the ones who may pay the price.

Sen. Lamar Alexander has been a champion for improving access to health care in this country. He understands that patients deserve to be taken seriously, and a doctor’s expertise should not be overrun by the will of an insurance company. That is why I urge Sen. Alexander to stand up for patients and doctors’ rights and reverse this harmful policy.

Karyn Waxman is the Memphis chapter founder and coordinator for the PKD Foundation.

From Business Wire

Palladio Biosciences Announces Dosing of First Patients with Lixivaptan in ELiSA, a Phase 2 Clinical Study in Patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Palladio Biosciences, Inc. (Palladio) http://palladiobio.com/, a privately held biopharmaceutical company founded to develop medicines that make a meaningful impact on the lives of patients with orphan diseases of the kidney, today announces the dosing of the first patients with lixivaptan in ELiSA, a phase 2 clinical study in patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD).

The ELiSA study (Evaluation of Lixivaptan in Subjects with ADPKD), which is being conducted at several clinical sites in the United States, will look at how well two different doses of lixivaptan work in preserving kidney function. It will also look at how well adult subjects with ADPKD tolerate the study drug, which will be given by mouth for 7 days. The study is enrolling male or female ADPKD patients between 18 and 60 years of age and estimated Glomerular Filtration Rate (eGFR) greater than 30 mL/min/1.73m2. Completion of the ELiSA study will pave the way for the initiation of a Phase 3 registration study in 2019.

“Dosing the first ADPKD patients with lixivaptan is a major milestone for Palladio Biosciences,” said Lorenzo Pellegrini, CEO of Palladio. “The ELiSA Study is a key component of our clinical development program and will be used to inform the design of our upcoming pivotal Phase 3 study. We believe that lixivaptan has the potential to meet some of the many unmet medical needs of ADPKD patients.”

David Baron, Chief Scientific Officer of the PKD Foundation, said, “PKD is part of America’s under-recognized kidney disease epidemic. We at the PKD Foundation continue to support the development of new drug therapies for patients with PKD, in part by increasing awareness of PKD clinical studies. We look forward to seeing results of the ELiSA trial and the continued development of lixivaptan for ADPKD.”

To learn more about the trial, visit the Clinicaltrials.gov website at https://www.clinicaltrials.gov/ct2/show/study/NCT03487913.Additional details can also be found at http://palladiobio.com/clinical-trials/ and https://pkdcure.org/clinical-study/elisa-evaluation-lixivaptan-subjects-adpkd/.

About Lixivaptan:

Lixivaptan was granted Investigational New Drug (IND) clearance to proceed with a Phase 2 clinical trial of lixivaptan capsules in patients with ADPKD in the first half of 2018. It had previously received orphan designation by FDA for the treatment of ADPKD. It is a potent, selective vasopressin V2 receptor antagonist, a mechanism of action that has clinical proof of concept to slow kidney function decline in adults at risk of rapidly progressing ADPKD. Lixivaptan was previously administered to more than 1,600 subjects across 36 clinical studies as part of a prior clinical development program for the treatment of hyponatremia. Palladio expects to leverage lixivaptan’s large body of data generated in the hyponatremia clinical program to accelerate the development of lixivaptan for the treatment of ADPKD.

About Polycystic Kidney Disease (PKD) – Key Facts and Figures:

PKD is an inherited genetic disease that affects thousands of people in the United States and millions globally. ADPKD is the most common type of PKD. A person with ADPKD has a 50 percent chance of passing the disease on to each of his or her children. The disease is characterized by uncontrolled growth of fluid-filled cysts in the kidney, which can each grow to be as large as a football. Symptoms often include kidney infections and chronic pain. The continued enlargement of cysts and replacement of normal kidney tissue causes irreversible loss of renal function. In the United States, approximately 2,500 new people with PKD require dialysis or a kidney transplant every year, making PKD the 4th leading cause of kidney failure. There is no cure for PKD.

About Palladio Biosciences, Inc.:

Palladio Biosciences is a privately-owned, clinical stage biopharmaceutical company developing medicines for orphan diseases of the kidney and is located in Newtown, PA. For more information, please visit www.palladiobio.com.

About the PKD Foundation:

The PKD Foundation is the only organization in the U.S. solely dedicated to finding treatments and a cure for PKD to improve the lives of those it affects.

Our vision is to #endPKD. To learn more about PKD, please visit the PKD Foundation website: https://pkdcure.org/resources/category/advocacy/#

Please note that lixivaptan is for investigational use only.

Contacts
Palladio Biosciences, Inc.
Linda Hogan, +1 908-294-8728
lhogan@palladiobio.com

PKD Research: Gene Therapy, Gift of Life: Life Changing Phone Call

PKD Research: Gene Therapy

iNews, United Kingdom, by Tom Bawden

Why are cure for obesity could be on the horizon

A cure for severe obesity could be on the horizon after scientists used a new kind of gene therapy to stop mice getting fat. They say the same technique could potentially be used to treat some cases of epilepsy, cancer, kidney disease and mental and physical developmental delays in children – although it is likely to be around a decade before any are adopted by the health service. Many cases of severe obesity in mice and in humans are driven by a genetic defect which leaves afflicted individuals with an insatiable appetite. This means that they keep on eating and become very overweight.

New technique

Now, scientists have found a way to correct that defect, putting a brake on consumption after a reasonable amount of food has been eaten. Mice with corrected versions of the mutated genes were up to 40 per cent lighter than their untreated counterparts and the effects were long lasting, they found. “The results were dramatic. This technique provides a potential cure for certain forms of obesity as well as hundreds of other diseases such as epilepsy, developmental delay, cancer and polycystic kidney disease,” said lead researcher Navneet Matharu, of the University of California in San Francisco.

The technique could also potentially be used to treat prostate, breast, colon, ovarian and lung cancer, as well as Parkinson’s and inflammatory bowel disease, he said. Severe obesity – where your body mass index is 40 or higher – is one of numerous diseases that can be caused by a mutation in one of the two copies we have of every gene, one from each parent. That dysfunctional gene leaves the second copy to do all the work – which it may not be strong enough to do on its own. In the case of severe obesity, the disease can be caused by a mutation of the gene that regulates hunger or of the gene that regulates satiety.

Pumping up the volume

This treatment worked by ‘turning up the volume’ of the remaining functional gene so that it can do its job effectively. This was done by taking one of the functional copies of the gene, boosting it to amplify its power and then inserting it into the hunger-control regions of the brains of mice by injecting it in a virus that has been rendered harmless.

The researchers are confident that the same technique can be applied to numerous other diseases where a mutation in just one copy of the gene – known as haploinsufficiency – can lead to diseases. “We believe our system could be applied to any situation in which having only one functional copy of a gene leads to disease. Our method demonstrates tremendous therapeutic potential for numerous diseases,” said Dr Matharu. Sometimes the mutation is a fairly minor cause of a disease and in others it can be the primary cause, researchers said. They point out that at least 660 genes – and potentially more than 3,000 – human genes are haploinsufficient, acting as a cause hundreds of diseases. The research is published in the journal Science.

ANALYSIS

It’s still early days but this obesity breakthrough in mice could have the potential to revolutionise the healthcare system for humans. Assuming that the new gene therapy technique also keeps down the weight in humans, it has the potential to significantly cut cases of severe obesity – which lead to numerous health problems and often early death. It also costs the NHS a fortune to treat the associated diseases. Ultimately, despite the potential to avert early death, medics and patients may baulk at the prospect of an injection to the brain to cure obesity. Time will tell on that one. But there are other cases where an injection to the brain would be well worth it – with extreme epilepsy being an obvious example.

Gift of Life

From Woodward News, Woodward, OK, By Elise Solloway Area Correspondent

The life-changing phone call: The generosity of a friend and a successful surgery

Kidney donor Randy Arrington and recipient Elise Solloway, Oct. 30, 2018 (one day after the transplant surgeries).

Editor's Note: Elise Solloway is a correspondent for the Woodward News. She recently received a kidney transplant after fighting a genetic kidney disease. She underwent successful transplant surgery in October and is telling her story this week in the Woodward News.

After finding a donor and being accepted for the transplant, the NZTI staff wanted us both to come to Oklahoma City the very next day to get pre-registered for surgery at Integris Baptist Medical Center, get pre-op lab and EKGs done, get surgery preps at the pharmacy, and for me to get my first batch of immunosuppressant (anti-rejection) medications to take in preparation for the surgery so my body would not reject Randy’s kidney.

Weeks of prayers by family, friends, and my church family were answered. In just a few months, I went from being a patient on the national and NZTI kidney transplant list to being scheduled to have a transplant from a healthy, generous, living donor who responded to my Facebook posts.

Having been a chronic worrier most of my life and dealing with many health problems over the years, I learned as an adult to put things in perspective when approaching life’s challenges. None of my previous fears had ever come true, so I gradually learned to not worry, just put things in God’s hands and leave them there.

After reading the pros and cons of a kidney transplant vs dialysis, and all the potential side effects of the various anti-rejection drugs and steroids, I simply entrusted it all to God. I pledged not to be a worrier.

On Monday, October 29, 2018, Randy underwent surgery to safely remove his healthy right kidney which was then transplanted into the left side of my abdomen to replace my two kidneys with advanced Polycystic Kidney Disease. We each spent four days on the 10th floor of Integris Baptist Medical Center where we received exceptionally good care.

Randy and I visited each other’s rooms and walked laps together on our floor beginning the day after our transplant surgeries. On our walks we also met another transplant patient and sometimes all three of us were walking laps and chatting in the hall.

While in the hospital I received about five hours of education related to warning signs of organ rejection, what each of my medications were for, and how to follow the daily schedule for taking the medications and recording my vitals. I also received information on what foods were safe and which ones were not safe to eat due to my new kidney and my various medications.

One day my surgeon asked if I had any children and if they had been tested for PKD. A new medicine approved by FDA has proven to slow down or even stop the progression of PKD and our son would need to start it as soon as possible.

My husband called our son to encourage him to get tested and start the medication. He was surprised to find out he had been tested 2-3 months ago and had no sign of PKD. He just had not notified us for some reason.

Because he did not inherit PKD from me, his children could not inherit it from him. We were overjoyed with relief. PKD ends with me and my sister in our family.

Since coming home from the hospital, Randy and I keep in touch via telephone and the Internet. We are both recovering nicely from our transplant surgeries. He will go back to work Dec. 10.

Randy is literally a part of my life now, and always will be. He gave me the gift of life by the ultimate gift of one of his healthy kidneys to replace my seriously diseased kidneys. I am eternally indebted to him and thankful to God for this gift.

For the rest of my life I will have a medically-necessary schedule to follow. I now live from 8 a.m. to 8 p.m. with a to-do list of medically necessary procedures. Every 8 a.m. I must monitor my weight, temperature, blood pressure, fluid in-take and out-put, and take multiple medications necessary to keep my new kidney working well so it will not be rejected by my body. Then at 8 p.m. I do some of the same things all over again. I must follow this schedule, even when traveling, by pulling over on the side of the road to record my vitals and take my medications at the designated times.

Everywhere I go I must take with me my backpack that contains my daily health log, all my medications sorted in an organizer, a thermometer, a blood pressure machine. It must also include my3-ring binder filled with my medical exam/lab summaries with diagnoses and medication/diet changes based on the lab work results.

For awhile, I will have weekly lab work drawn to monitor my new kidney’s function and my general health, then meet with my medical team to monitor the healing of my incision, then adjust medications and diet as needed. As my system stabilizes with the new kidney, medications will be reduced, but there will always be one or more immunosuppressants/anti-rejection medications to take.

Extra precautions are now necessary everywhere I go and whenever I am around other people. Hand sanitation and/or gloves plus face masks are necessary in some settings to reduce the chances of me catching a virus, infection, or bacteria since I have a weakened immune system and could have an organ rejection.

I must limit my exposure to cats, animal feces, reptiles, ponds, mulch, certain foods, and anyone who has had a live culture immunization within three weeks. Anytime I am exposed to sunlight I must wear sun-blocking clothes, sun screen, and wear a sun-blocking hat, due to my risk of skin cancers from the transplant medications I take. I must also watch my diet because I am now at higher risk of getting diabetes due to my transplant medications and medical history.

PKD Life: Family Business; Combat Human Organ Black Market; PKD Fundraising

PKD Life

From 2Paragraphs

The Profit: NYC Bagel Deli Owner Lost 2 Children To PKD Kidney Disease

Corey Kaplan on The Profit (CNBC)

On the Season 6 premiere of The Profit, small business investor Marcus Lemonis visits NYC Bagel Deli, a bagel chain with three locations in downtown Chicago which sells over 1,000 bagels daily. On The Profit, according to CNBC, “the overbearing owner” Corey Kaplan “ignores his wife’s business ideas and hangs on to the outdated look of his cluttered storefronts.” Marcus tries to help Cory re-brand the company and help develop new products. But not a lot of small business owners like change.

While getting to know Corey and his wife Candis, Lemonis learns that the couple had five children but “two of them are in heaven,” says Corey. The two children they lost, Corey and CorriAngel, both died extremely young of PKD, polycystic kidney disease. It is a chronic, genetic disease causing uncontrolled growth of cysts in the kidney, often leading to kidney failure. Lemonis encouraged Corey to change the name of NYC Bagel Deli to Corey’s NYC Bagel Deli in honor of his children.

After filming The Profit, Corey Kaplan wrote of Lemonis’ huge heart and “the fact that he is helping us bring awareness to PKD (the disease that our children died from), I will never forget him and his open-hearted kindness.” The Profit airs Tuesdays at 10 pm on CNBC.

Organ Transplants

From Australian Broadcast Corp., By political reporter Stephanie Dalzell

Australian transplant waiting list contributes to human organ black market, committee says

Key Findings:

Committee finds if Government doesn't act, organ market will flourish

About 1,400 Australians waiting for transplant, 11,000 on renal dialysis

Average waiting time for replacement kidney is 3 years


The criminal masterminds behind the illegal trade of human body parts raked in $2.3 billion around the globe last year.

About 12,000 organs were sold on the black market, and while the majority of those exchanges involved kidneys, 654 hearts and 2,615 livers were sold for up to $394,000 each.

That illegal trade will continue to grow if the Australian Government does not do more to deter human organ trafficking, according to a unanimous report handed down by a parliamentary committee.

About 1,400 Australians are currently waiting for an organ transplant, while a further 11,000 are on kidney dialysis, and the committee found if the government failed to address the gap between the number of people requiring organ transplants and the limited supply of freely donated organs, the black market would keep flourishing.

Commercial organ market

Organ	Global illicit transplants (per annum)	Price range (AUD)
Kidney	7,995	$68,000 - $163,000
Liver	2,615	$134,000 - $197,000
Heart	654	$176,000 - $394,000
Lung	469	$203,000 - $394,000
Pancreas	233	$149,000 - $190,000

The chairman of the Human Rights Sub-Committee of the Joint Standing Committee on Foreign Affairs, Defence and Trade, Liberal MP Kevin Andrews, told Parliament the average waiting time for a kidney in Australia was three years.

"Desperate people often facing certain death without a transplant may travel far from their own countries to places such as Egypt, the Philippines or Pakistan, paying tens of thousands of dollars or more, for an organ transplant, where the donor is most likely in dire financial straits, possibly exploited, and unable to give free and informed consent to donation," Mr Andrews said.
Cutting down demand

The committee's report recommended the Australian Government pursue a range of measures to strengthen its involvement in international efforts to combat human organ trafficking, collect data on Australians involved in illegal organ trafficking overseas and also tighten criminal laws around organ harvesting.

It also concluded the Government should seek to improve organ-donation rates through ongoing funding of programs, education awareness campaigns, and the investigation of other international programs — such as opt-out organ donation.

Mr Andrews said the committee heard from many people who argued protections against the practice needed to be strengthened.

PKD Fundraising

From MLive, By Greg Chrapek  chrapek@mlive.com

West Michigan Aviation Academy hosting `Teal Out' basketball game to raise awareness of polycystic kidney disease

The West Michigan Aviation Academy boys basketball team is capping a week of fundraising by the school by hosting a 'Teal Out Night' game against Hopkins this Friday.

The Teal Out event is part of a week-long effort by the students that serves as a fundraiser and awareness activity to end polycystic kidney disease (PKD). Student activities during the week include Penny Wars, t-shirt sales and an auction. Teal is the official color for PKD. East year the students at West Michigan Aviation Academy select a non-profit organization to work with and PKD was chosen as this year's charity.

Speaking at the game even will be Dr. Greg Vanden Heuvel from Western Michigan University and Kim Ahrens, a Detroit PKD representative. The event is taking place to bring awareness of PKD to West Michigan and generate funds for research to find a cure.

Friday's basketball game with Hopkins is schedule for 7 p.m. this at the school located at 5363 44th St. SE.