FDA Approves ADPKD Treatment: JYNARQUE™ (tolvaptan), Avella Pharmacy Selected to Distribute JYNARQUE™

PKD Treatment

From PKD Foundation

BREAKING NEWS: FDA Approves First Treatment for ADPKD

Today is a historic day in providing hope to patients with polycystic kidney disease! We are excited to announce that the U.S. Food and Drug Administration (FDA) granted approval of JYNARQUE™ (tolvaptan) to be the first treatment in the United States for adult patients with autosomal dominant polycystic kidney disease (ADPKD).

For the past 35 years, the PKD Foundation’s goal has been to support PKD patients and families from care to cure. We are proud to not only have supported early studies that led to the development of JYNARQUE™ as a treatment, but also helped guide PKD patients to the JYNARQUE™ clinical trials. It is gratifying to play a part in the discovery of this treatment and to see it come to fruition.

Many thanks to the patients who graciously took the time and resources to participate in the clinical trials to bring JYNARQUE™ to the PKD community.This treatment would not exist without these patients.

To read our full press release and sign up for JYNARQUE™ updates, please visit pkdcure.org/jynarque. We are also hosting a special webinar about the new treatment with Ron Perrone, M.D., next Monday, April 30. To register, please click here.

We hope that this is just the beginning of new treatments on the horizon. Our work to provide access to future studies and to new treatments is more energized than ever.

And, as always, we will continue to stand right beside you, until there is a cure.

Sincerely,



Andy Betts
CEO




From Business Wire

Otsuka’s JYNARQUE™ (tolvaptan) Approved by U.S. FDA as the First Treatment to Slow Kidney Function Decline in Adults at Risk of Rapidly Progressing Autosomal Dominant Polycystic Kidney Disease (ADPKD)

ADPKD, the fourth leading cause of end-stage renal disease,1,2 is a progressively debilitating genetic disease characterized by the development of fluid-filled cysts in the kidneys3

Data from two phase 3 clinical trials showed that JYNARQUE™ (tolvaptan) slowed kidney function decline in adults at risk of rapidly progressing ADPKD

JYNARQUE can cause serious and potentially fatal liver injury. Due to elevations of liver enzymes in the blood associated with JYNARQUE, this medication will be available only through a restricted distribution program and patients will need to be monitored for elevations in these enzyme levels. Please see IMPORTANT SAFETY INFORMATION below for more detailed risk information


(Otsuka) announces that the U.S. Food and Drug Administration (FDA) has approved JYNARQUE™ (tolvaptan) as the first drug treatment to slow kidney function decline in adults at risk of rapidly progressing autosomal dominant polycystic kidney disease (ADPKD).

The efficacy of tolvaptan was demonstrated in two pivotal trials, lasting one year and three years, respectively. In the one-year REPRISE study, the primary endpoint was the treatment difference in the change of eGFR from pretreatment baseline to post-treatment follow-up, annualized by dividing by each subject’s treatment duration. In the randomized period, the change of eGFR from pretreatment baseline to post-treatment follow-up was −2.3 mL/min/1.73 m2/year with tolvaptan as compared with −3.6 mL/min/1.73 m2/year with placebo, corresponding to a treatment effect of 1.3 mL/min/1.73 m2/year (p <0.0001). In the three-year TEMPO 3:4 study, tolvaptan reduced the rate of decline in eGFR by 1.0 mL /min /1.73m2 /year (95 % confidence interval of 0.6 to 1.4) as compared to placebo in patients with earlier stages of ADPKD. In the extension trial, eGFR differences produced by the third year of the TEMPO 3:4 trial were maintained over the next 2 years of JYNARQUE treatment.ADPKD is a genetic disease with consequences that can lead to dialysis or kidney transplantation. It is a progressively debilitating and often painful disorder in which fluid-filled cysts develop in the kidneys over time. These cysts enlarge the kidneys and impair their ability to function normally, leading to kidney failure in most patients.3 ADPKD is diagnosed in approximately 140,000 people in the U.S.,4,5 and impacts families across multiple generations, since a parent with ADPKD has a 50 percent chance of passing the disease on to each of their children.6,7

The primary endpoint in TEMPO 3:4 study was the intergroup difference for rate of change of total kidney volume (TKV) normalized as a percentage. The trial met its pre-specified primary endpoint of 3-year change in TKV (p<0.0001). The difference in TKV between treatment groups mostly developed within the first year, the earliest assessment, with little further difference in years two and three. In years 4 and 5 during the TEMPO 3:4 extension trial, both groups received JYNARQUE and the difference between the groups in TKV was not maintained. Tolvaptan has little effect on kidney size beyond what accrues during the first year of treatment. The key secondary composite endpoint (ADPKD progression) was time to multiple clinical progression events of: 1) worsening kidney function (defined as a persistent 25% reduction in reciprocal serum creatinine during treatment (from end of titration to last on-drug visit); 2) medically significant kidney pain (defined as requiring prescribed leave, last-resort analgesics, narcotic and anti-nociceptive, radiologic or surgical interventions); 3) worsening hypertension (defined as a persistent increase in blood pressure category or an increased anti-hypertensive prescription); 4) worsening albuminuria (defined as a persistent increase in albumin/creatinine ratio category). The relative rate of ADPKD-related events was decreased by 13.5% in tolvaptan-treated patients, (44 vs. 50 events per 100 person-years; hazard ratio, 0.87; 95% CI, 0.78 to 0.97; p=0.0095). As shown in the table below, the result of the key secondary composite endpoint was driven by effects on worsening kidney function and kidney pain events. In contrast, there was no effect of tolvaptan on either progression of hypertension or albuminuria. Few subjects in either arm required a radiologic or surgical intervention for kidney pain. Most kidney pain events reflected use of a medication to treat pain such as use of paracetamol, tricyclic antidepressants, narcotics and other non-narcotic agents.

JYNARQUE can cause serious and potentially fatal liver injury, and acute liver failure requiring liver transplantation has been reported.JYNARQUE has been associated with elevations of blood alanine and aspartate aminotransferases (ALT and AST), with infrequent cases of concomitant elevations in bilirubin-total (BT). To ensure the safety of patients taking JYNARQUE, it is necessary to measure ALT, AST and bilirubin before initiating treatment, at 2 weeks and 4 weeks after initiation, then monthly for 18 months and every 3 months thereafter, for as long as the patient is on JYNARQUE (tolvaptan) treatment. Because of the risks of serious liver injury, JYNARQUE is available only through a restricted distribution program supported by a Risk Evaluation and Mitigation Strategy (REMS) Program approved by the FDA. For more information about JYNARQUE, please visit www.JYNARQUE.com.

"The progressive nature of ADPKD means that kidney function gets worse over time, eventually leading to end-stage renal disease. This progression happens more rapidly for some patients than others.” said Michal Mrug, M.D., Associate Professor at the University of Alabama at Birmingham and investigator on the REPRISE trial. “Today’s approval is great news for adults at risk of rapidly progressing ADPKD because by slowing the decline in kidney function, this therapy may give them more time before kidney transplant or dialysis.”

Andy Betts, CEO of the PKD Foundation, observed, “Today is an historic day in providing hope to patients with autosomal dominant polycystic kidney disease, and we are thrilled to be a part of this first milestone in treatment. For the past 35 years, our goal has been to walk with PKD patients every step of the way. It is gratifying to play a part in the inception of the discovery of this treatment, and to see it come to fruition. We hope that this is just the beginning of a new chapter for adults at risk of rapidly progressing ADPKD who suffer from the disease.”

Also, Tatsuo Higuchi, president and representative director of Otsuka Pharmaceutical Co., Ltd., commented, “This approval is important news for many adults at risk of rapidly progressing ADPKD in the U.S., who have had no therapeutic alternatives to delay the eventual end-stage interventions of dialysis or kidney transplantation. We are humbled to be able to offer an earlier, proactive course of action to slow the progression of this disease, which we know means so much to patients, their families and healthcare providers. Simultaneously, we are grateful to the patients and researchers who through their continued commitment made this milestone possible.”

About ADPKD

ADPKD is a progressively debilitating and often painful genetic disorder in which fluid-filled cysts develop in the kidneys over time. These cysts enlarge the kidneys and impair their ability to function normally, leading to kidney failure in most patients. ADPKD can impact quality of life, and is also associated with cardiovascular complications that can cause death.3 ADPKD is diagnosed in approximately 140,000 people in the U.S.,4,5 and is the fourth leading cause of end-stage renal disease.1,2

ADPKD impacts families across multiple generations, since a parent with ADPKD has a 50 percent chance of passing the disease on to each of their children.6,7 Risk factors for rapid disease progression include having a greater TKV than expected for age,8,9 family history of end-stage renal disease before 58 years of age,10 high blood pressure before 35 years of age,11 certain urologic events before 35 years of age,12 a historical decline in eGFR of ≥5 mL/min/1.73 m2 within 1 year,10 certain inherited genetic profiles,12 or male sex.12Visit https://pkdcure.org/what-is-pkd/adpkd/ for more information about ADPKD.

About JYNARQUE™ (tolvaptan)

JYNARQUE is a selective vasopressin V2-receptor antagonist indicated to slow kidney function decline in adults at risk of rapidly progressing ADPKD. The medication has been approved as a treatment for adults with ADPKD in Japan, the EU, Canada, South Korea, Switzerland, Hong Kong, Australia, Turkey and Taiwan. See local prescribing information for specific indications in each country.

The FDA approval of JYNARQUE is supported by data from the TEMPO 3:4 (Tolvaptan Efficacy and Safety in Management of Autosomal Dominant Polycystic Kidney Disease and Its Outcomes) and REPRISE (Replicating Evidence of Preserved Renal Function: an Investigation of Tolvaptan Safety and Efficacy in ADPKD) clinical trials published in the New England Journal of Medicine in November 2012 and November 2017, respectively.

JYNARQUE will be sold in a 28-day treatment pack at a wholesale acquisition cost of $13,041.10.

More information for U.S. Healthcare Providers can be found at www.JYNARQUEhcp.com.

INDICATION and IMPORTANT SAFETY INFORMATION for JYNARQUETM (tolvaptan)

INDICATION:

JYNARQUE is indicated to slow kidney function decline in adults at risk of rapidly progressing autosomal dominant polycystic kidney disease (ADPKD).

IMPORTANT SAFETY INFORMATION:

WARNING: RISK OF SERIOUS LIVER INJURY
JYNARQUE (tolvaptan) can cause serious and potentially fatal liver injury. Acute liver failure requiring liver transplantation has been reported
Measure transaminases (ALT, AST) and bilirubin before initiating treatment, at 2 weeks and 4 weeks after initiation, then monthly for the first 18 months and every 3 months thereafter. Prompt action in response to laboratory abnormalities, signs, or symptoms indicative of hepatic injury can mitigate, but not eliminate, the risk of serious hepatotoxicity.
Because of the risks of serious liver injury, JYNARQUE is available only through a Risk Evaluation and Mitigation Strategy program called the JYNARQUE REMS Program

CONTRAINDICATIONS:
History, signs or symptoms of significant liver impairment or injury. This contraindication does not apply to uncomplicated polycystic liver disease
Taking strong CYP3A inhibitors
With uncorrected abnormal blood sodium concentrations
Unable to sense or respond to thirst
Hypovolemia
Hypersensitivity (e.g., anaphylaxis, rash) to JYNARQUE or any component of the product
Uncorrected urinary outflow obstruction
Anuria

Serious Liver Injury: JYNARQUE can cause serious and potentially fatal liver injury. Acute liver failure requiring liver transplantation has been reported in the post-marketing ADPKD experience. Discontinuation in response to laboratory abnormalities or signs or symptoms of liver injury (such as fatigue, anorexia, nausea, right upper abdominal discomfort, vomiting, fever, rash, pruritus, icterus, dark urine or jaundice) can reduce the risk of severe hepatotoxicity. To reduce the risk of significant or irreversible liver injury, assess ALT, AST and bilirubin prior to initiating JYNARQUE, at 2 weeks and 4 weeks after initiation, then monthly for 18 months and every 3 months thereafter.

Hypernatremia, Dehydration and Hypovolemia: JYNARQUE therapy increases free water clearance which can lead to dehydration, hypovolemia and hypernatremia. Instruct patients to drink water when thirsty, and throughout the day and night if awake. Monitor for weight loss, tachycardia and hypotension because they may signal dehydration. Ensure abnormalities in sodium concentrations are corrected before initiating therapy. If serum sodium increases above normal or the patient becomes hypovolemic or dehydrated and fluid intake cannot be increased, suspend JYNARQUE until serum sodium, hydration status and volume status parameters are within the normal range.

Inhibitors of CYP3A: Concomitant use of JYNARQUE with drugs that are moderate or strong CYP3A inhibitors (e.g., ketoconazole, itraconazole, lopinavir/ritonavir, indinavir/ritonavir, ritonavir, and conivaptan) increases tolvaptan exposure. Use with strong CYP3A inhibitors is contraindicated; dose reduction of JYNARQUE is recommended for patients taking moderate CYP3A inhibitors. Patients should avoid grapefruit juice beverages while taking JYNARQUE.

Adverse Reactions: Most common observed adverse reactions with JYNARQUE (incidence >10% and at least twice that for placebo) were thirst, polyuria, nocturia, pollakiuria and polydipsia.

Other Drug Interactions:
Strong CYP3A Inducers: Co-administration with strong CYP3A inducers reduces exposure to JYNARQUE. Avoid concomitant use of JYNARQUE with strong CYP3A inducers
OATP1B1/3 and OAT3 Transporter Substrates: Caution should be used in patients who take JYNARQUE and OATP1B1/B3 and OAT3 substrates (e.g., statins, bosentan, glyburide, nateglinide, repaglinide, methotrexate, furosemide), as the plasma concentrations of these substrates may be increased.
BCRP Transporter Substrates: Tolvaptan is an inhibitor of BCRP. Patients who take JYNARQUE, should avoid concomitant use with BCRP substrates (e.g., rosuvastatin)
V2-Receptor Agonist: Tolvaptan interferes with the V2-agonist activity of desmopressin (dDAVP). Avoid concomitant use of JYNARQUE with a V2-agonist.

Pregnancy and Lactation: Based on animal data, JYNARQUE may cause fetal harm. In general, JYNARQUE should be discontinued during pregnancy. Advise women not to breastfeed during treatment with JYNARQUE.

To report SUSPECTED ADVERSE REACTIONS, contact Otsuka America Pharmaceutical, Inc. at 1-800-438-9927 or FDA at 1-800-FDA-1088 (www.fda.gov/medwatch).

Please see FULL PRESCRIBING INFORMATION, including BOXED WARNING

From PR NewsWire

Avella Selected by Otsuka to distribute JYNARQUE


Avella announced that it has been selected by Otsuka America Pharmaceutical, Inc. (Otsuka) to distribute JYNARQUE™ (tolvaptan), the very first FDA-approved therapy for patients diagnosed with autosomal dominant polycystic kidney disease (ADPKD). Avella is one of just three pharmacies providing access to the medication.

ADPKD is one of the most common, life-threatening genetic diseases, diagnosed in 140,000 Americans. In patients with this condition, fluid-filled cysts develop and enlarge in both kidneys and eventually leading to kidney failure. ADPKD is the fourth leading cause of kidney failure and more than 50 percent of people with this condition will develop kidney failure by age 57.

JYNARQUE is a selective vasopressin V2-receptor antagonist. This means it blocks vasopressin, an anti-diuretic hormone secreted by the pituitary gland, which plays a key role in the fluid balance of the kidneys. By blocking this hormone, the drug can decrease cyst-cell proliferation and fluid secretion, which ultimately can reduce cyst growth. JYNARQUE was shown to reduce the rate of kidney function decline by 35 percent over a 12-month period when compared to a placebo, in patients with ADPKD.

"Avella is proud to be among a very select group of pharmacies extending access to this important therapy, the first of its kind available to treat this condition," said Leslie Yendro, Director of Business Development for Avella. "Since ADPKD can be a painful and debilitating disease for patients, it is exciting to see a new medication is finally available to help support these individuals."

Reusing Transplanted Kidneys, Transplant Medical Leave, XORTX & PKD Foundation Collaboration

Kidney Transplants

From Modern HealthCare, By Steven Ross Johnson

Could 'reused' transplanted kidneys solve the waitlist problem? Some docs think so

Las Vegas resident Vertis Boyce has always liked to travel to visit friends and family. But for the past 9½ years, her travel has been limited by her need for dialysis treatments. Boyce experienced renal failure in 2008 due to a combination of high blood pressure and a genetic disposition.

"Traveling was an inconvenience because I always had to set up appointments for dialysis in each city that I would visit," Boyce said.

Things changed for the better however last July, when Boyce, 70, received a kidney transplant after being on the waiting list for nine years. Boyce's procedure was one of more than 34,700 organ transplants performed in the U.S. in 2017, marking a new record.

But what made Boyce's transplant nearly unique was the fact that she was the third person to have the same kidney, thanks to a procedure that had been performed only a few times since it was first introduced in the U.S. nearly a decade ago.

Boyce's kidney came from a 25-year-old man who had received it in a transplant in 2015. A 17-year-old girl who had died was the source of the kidney, which was functioning well inside the first recipient until he died in a car accident in 2017.

Such transplants have not been widely performed because of the procedure's complexity, as well as the fact that the damage a kidney usually sustains after an initial transplant makes it a poor candidate for a second procedure.

But the team that performed Boyce's surgery is hopeful the technique can reveal the potential for reusing viable kidneys to become a standard medical practice at transplantation centers and help reduce the shortage of available organs.

Normally if a transplant recipient unexpectedly dies or when the organ is rejected, the donated kidney is discarded. In 2013 more than 16% of all kidneys recovered from deceased donors were discarded, according to the U.S. Renal Data System.

"I didn't want to see a high-functioning kidney get wasted," said Dr. Jeffrey Veale, a transplant surgeon at Ronald Reagan UCLA Medical Center. Veale has performed three procedures during which a donor's kidney was reused in another patient.

He said as many as a quarter of kidney transplant recipients die with a functioning kidney.

After a kidney is transplanted, scar tissue can form around the organ; that makes reusing it more difficult because the surgeon can't attach it to vital blood vessels.

Veale addressed that problem by removing the second donor's iliac blood vessels that go into the leg and sewing them into Boyce.

"I sewed the first donor's kidney along with the second patient's vessels, and they all kind of work together to help the third person get off dialysis," Veale said.

Arguably the greatest factor in the procedure's success was the health of the kidney, which was in good enough shape for a second transplant despite being inside the first recipient for two years, during which the patient underwent a daily regimen of toxic anti-rejection medications.

Yet Veale was confident the procedure he performed could be duplicated by others more frequently.

"Without even trying I was able to do three in 2017," Veale said. "Could you imagine if we had all 230 transplant centers doing this?"

Others did not sound as optimistic about the procedure's potential. "It is not a common practice, but it's really the amount of time from the first transplant that will dictate whether that kidney can be reused or not," said Dr. Lorenzo Gallon, professor of medicine and surgery and director of the Transplant Nephrology Fellowship program at Northwestern University's Feinberg School of Medicine in Chicago.

In 2012, Gallon was part the Northwestern Medicine transplant team that conducted the first successful removal and implantation of a kidney from one patient to another after the organ failed in the first recipient. The findings of the case were published in the New England Journal of Medicine.

In that case, the kidney came from a living donor and three weeks passed between the first and second transplant.

"For a short period of time, I think there is the possibility of reopening the window (to re-transplant the kidney) when someone dies," Gallon said, adding that scar tissue can begin to form around the newly transplanted kidney within four weeks. "Unfortunately, this is how the body reacts to foreign organs."


Whether or not re-transplantation happens more frequently, the need to meet the rising demand for kidney donors has experts re-thinking the current organ donation framework to increase efficiency and access for waiting patients.

Currently, more than 95,000 people are waiting for a kidney transplant, according to the not-for-profit United Network for Organ Sharing, which administers the country's organ transplantation network.

The average wait time to receive a kidney in the U.S. is three to five years, but the wait can be up to seven years in some parts of the country. An estimated 4,500 people die annually waiting for a transplant, according to the Living Kidney Donors Network.

Gallon said one possible way to address lengthy wait times is for hospitals to create target lists of patients who will benefit from receiving a kidney transplant sooner compared with other patients. The national waiting list system gives first priority to those who've spent the longest time on the list.

Another solution would be using kidneys that in the past would have been discarded because they came from a patient suffering from certain treatable or manageable diseases even if the transplant recipient had the same disease, like HIV or hepatitis C.

"Obviously that doesn't increase the number (of organs) drastically but it increases it a little bit," Gallon said.

It's a problem that is expected to only get worse in the coming years as the country's elderly population increases. The percentage of adults ages 30 or older who will develop chronic kidney disease is projected to increase from 13.2% currently to 16.7% by 2030, according to a 2015 study published in the American Journal of Kidney Diseases.

Other proposals have called for the government to compensate living kidney donors to encourage more people to donate. A 2016 study published in the American Journal of Transplantation estimated a program that had the government compensate kidney donors would save taxpayers an estimated $403,000 a year per kidney dialysis patient for a total savings of about $2.6 billion annually.

Some of the demand for transplant kidneys in recent years has been offset by the opioid epidemic, which has driven an increase in deceased donor organs as a result of the record number of individuals who have died from drug overdose. Organ transplants that came from overdose death rose from 1.1% of all donors in 2000 to 13.4% in 2017.

The crux of the kidney shortage, according to Dr. John Friedewald, medical director of kidney and pancreas transplantation at Northwestern Memorial Hospital in Chicago, lies in being able to successfully harvest donated kidneys and preserve them long enough to be transplanted.

A 2016 study by the United Network for Organ Sharing found the discard rate for deceased-donor kidneys more than tripled from 5.1% in 1988 to 19.2% by 2009, the most recent data available.

"We have been fairly successful in getting people to sign up to be organ donors," said Friedewald. "One of the challenges we have is getting the organs out and keeping them healthy until we can get them into the next person."

Living with PKD

From UK Now (University of Kentucky)

New UK Medical Leave Policy Provides Coverage for Donors

When UK HealthCare employee Kim Powell learned that her brother had polycystic kidney disease and needed a transplant, she didn't hesitate to become a "living donor" and offer her own.

"His kidney disease was inherited," she said. "Any of us siblings could have gotten it -- he was just the one who ended up with it."

She underwent extensive testing, and as it turns out, Powell's kidney was a perfect match for her brother. In March 2011, they went into surgery at UK HealthCare to undergo the kidney swap.

Following a kidney donation, many donors are told to take off work for 4-6 weeks to recover. In Powell's case, she needed the full six weeks before she felt back to her old self.

"At four weeks, I didn't feel like I was ready to go back," she said. "I was very tired for those first six weeks; nauseated. And I was also in some pain just from the surgery."

Having worked at the University of Kentucky for more than two decades prior to undergoing surgery, Powell had accrued enough sick time to cover her long leave. However, she recognizes how fortunate she was to be in that position – without her bank of sick and vacation time, she may not have been able to help her brother.

"I couldn't have taken off any time without pay," she said. "I would have tried anything I could, but I may not have been able to give to him, which is sad."

Now, a new UK medical leave policy will give generous donors like Powell some peace of mind. Beginning April 1st, UK enacted a new leave policy for full-time employees who wish to give others a second chance at life:
Employees who donate bone marrow will receive five days paid medical leave.
Employees who donate a solid organ (kidney, partial liver, or partial lung) will receive 30 days paid medical leave.

At UK HealthCare, the ability to receive organ or tissue donations is crucial to many of the complex medical treatments offered.

“With an academic medical center focused on specialty medicine, including transplants, we recognize the generosity and sacrifice of bone marrow and organ donors," said Kim Wilson, vice president and chief human resources officer at UK. "The University offers this new paid leave to allow employees time to recover and to recognize their generous spirit.”

In Kentucky, more than 1,000 people are on the organ transplant waiting list, and the vast majority of those patients – approximately 800 – are in need of a kidney. Last year, the UK Transplant Center performed 98 kidney transplants, with roughly 28 percent of those transplants coming from a living donor.

While the organ donor registry may be able to provide a suitable kidney from a deceased donor, patients often have to wait for years, surviving through weekly dialysis until a suitable donor becomes available. Receiving a kidney from a living donor both removes patients from the transplant waiting list and increases the chances of a better overall outcome from the surgery.

"The message this sends to our UK community is one of hope – we want donors to be recognized and supported for their selfless gift," said Dr. Thomas Waid, medical director of the Kidney & Pancreas Transplant Program at UK. "This policy does just that. Even more, we want other institutions to take note. We have a real opportunity to impact the lives of Kentuckians and those around the region, and the more people that donate, the more lives we can save.”

At the UK Markey Cancer Center, physicians perform upwards of 110 bone marrow transplants each year. For patients dealing with deadly blood cancers, receiving donated bone marrow may be their only option for survival. Although recovery times vary, a bone marrow donor can expect to take anywhere from 1-7 days until they feel normal.

Be the Match is a national registry that matches anonymous donors with patients in need of a bone marrow transplant. After joining, UK HealthCare employee Donna Wall received two calls from the registry for a possible opportunity to donate, but both fell through. But about five years ago, she was called upon for a third time to donate her marrow to a young child in Europe.

For the extraction, Wall was placed under general anesthesia while a physician used a needle to withdraw the liquid marrow from her pelvic bone. The most common side effects of this procedure include back or hip pain, fatigue, and muscle pain.

Wall notes that she felt sore for several weeks after, similar to a bruise, but says she was ready to go back to work within a few days.

"I donated on a Thursday and took Thursday and Friday off work, but I was back to work Monday," she said. "It really wasn't that bad."

Wall, who has been with UK for 35 years, says she was inspired to join Be the Match after spending time working in both UK's tissue typing/IMP and stem cell processing labs. Like Powell, she was also able to cover her time off, but hopes that the new policy will inspire others to give.

"I think it's awesome!" she said. "Maybe more people who didn't have the time to take off would be more interested in doing it. The chance to help someone is a good thing."

“Implementation of this medical leave policy is a fantastic step in the right direction, as it will help UK donors to fulfill their goal, which is to help and possibly provide the only chance for cure to our patients and to patients around the globe," said Dr. Gerhard Hildebrandt, division chief for Hematology and Blood and Marrow Transplantation. "It will break down barriers, increase access to donors and grow our donor pool. And leading by example, UK hopefully will be followed by many others on this path."

Currently, 37 U.S. states have a statute regarding living donor leave for either state employees or all public or private employees, although Kentucky is not included in that number. With this new policy, UK becomes the largest university -- and one of the largest employers -- in the state to offer this extra benefit.

For Powell, whose brother is alive and well thanks to her gift, the new policy is a "wonderful idea" that she hopes will encourage others to choose living donation to help someone in need.

"I would donate again tomorrow if I could," she said. "It's a life-changing thing for someone else. You're helping someone that really needs it."

PKD Foundation

From Nasdaq Global News Wire

XORTX Announces PKD Foundation Collaboration

XORTX Therapeutics Inc. ("XORTX" or the “Company”) (CSE:XRX) is pleased to announce that the PKD Foundation has recognized XORTX as a leader in advancing the development of treatments for progressive kidney disease, including polycystic kidney disease (“PKD”) and rare diseases such as autosomal dominant polycystic kidney disease (“ADPKD”).

At the present time, despite considerable effort, very few approved therapeutic options exist to treat progressive kidney disease. There is reason for hope, however. Based on recent clinical study evidence, the presence of uric acid above the normal range can act to negatively affect the health and therapeutic outcomes of patients with PKD. The PKD Foundation is supportive of new treatments to lower and maintain uric acid levels in patients. Towards this goal, the PKD Foundation will make available its scientific expertise to support and collaborate with XORTX to discover these new treatments.

Dr. David Baron, Chief Scientific Officer, PKD Foundation stated, “We believe that the combination of the PKD Foundation's and XORTX's scientific expertise may enable an entirely new class of highly active and potentially important treatments for patients for whom current biopharmaceutical approaches have had limited success.”

Dr. Allen Davidoff, XORTX’s CEO added, “We are thrilled to receive this important endorsement of our programs from the PKD Foundation. Their support and collaboration will help advance XORTX’s programs to define the beneficial effects of our therapies, in ADPKD patients but potentially in other forms of polycystic kidney disease as well. There are substantial benefits to working with the leading polycystic kidney disease foundation in the world and collaborating on the development of treatments that will redefine how physicians treat this disease in the future.“

About PKD Foundation

The PKD Foundation began over 35 years ago to find treatments and a cure for polycystic kidney disease (PKD) and to improve the lives of those it affects. The Foundation does this through the promotion of research, education, advocacy, support and awareness on a national level, along with direct services to local communities across the U.S. The PKD Foundation is the largest private funder of PKD research, having invested more than $42 million in basic and clinical research, nephrology fellowships and scientific meetings with a simple goal: to discover and deliver treatments and a cure for PKD.

About XORTX Therapeutics Inc.

XORTX Therapeutics Inc. is a BioPharmaceutical company focused on developing innovative therapies to treat progressive kidney disease. XORTX has lead programs to develop treatments for progressive kidney disease due to diabetes, diabetic nephropathy and polycystic kidney disease. Secondary programs focus on developing therapies for health consequences that accompany pre-diabetes, diabetes and cardiovascular disease. Additional information on XORTX Therapeutics is available at www.xortx.com.

PKD Symptoms, PKD Foundation: From Care to Cure, ADPKD Lower Death Risk on Dialysis, Medicare Cuts to Affect Dialysis Access

PKD

From My Southern Health, by Linda Zettler
Symptoms of polycystic kidney disease

These are the warning signs for an inherited condition that can lead to kidney failure.

A sharp spike in blood pressure was James George’s first symptom before a diagnosis of polycystic kidney disease, but it wasn’t his first warning.

“My mom had discussed it with us and explained the disease,” said the now 67-year-old father and grandfather. His mother had the disease and so did her sister. All four of James’ siblings have been diagnosed.

What is polycystic kidney disease?

Polycystic kidney disease is an inherited disorder in which cysts – clusters of fluid-filled sacs – form in the kidneys, interfering with kidney function and sometimes causing issues for other organs, including the liver.

A somewhat common genetic disorder, polycystic kidney disease affects about 500,000 people in the United States. Children whose parents who have the disease have a 50 percent chance of developing it.

The cysts caused by polycystic kidney disease are non-cancerous but can grow very large and hamper the kidneys’ natural ability to filter waste from the blood. About half of polycystic kidney disease patients experience kidney failure by the time they turn 60, leading to dialysis treatments, in which the blood is filtered, and kidney transplants.

In James’ case, he needed a liver transplant first because of the damage the disease did to his liver. As his kidney function decreased, he began taking dialysis, often going with this mother, then had a kidney transplant about 10 years after the first surgery.

“People who need kidney transplants have some form of disease that causes kidney failure,” said Rachel Forbes, M.D., James’ Vanderbilt kidney transplant surgeon. “James’ was polycystic kidney disease. It’s usually inherited in families, and causes cysts in the kidneys and liver. The cysts basically take over the normal function and the kidneys are unable to perform as they usually would. These cysts often get very large, sometimes thirty pounds, because it is filled with so many fluid cysts.”

Signs of polycystic kidney disease

Knowing you family history and discussing it with your healthcare provider are both important for detection of polycystic kidney disease.

Symptoms appear differently in patients, but the most common symptom is high blood pressure. 

Other symptoms include:
Pain in the back and side
Blood in the urine
Frequent kidney infections

A polycystic kidney disease diagnosis may require patients to change the way they eat to control blood pressure. According to the National Institute of Diabetes and Digestive and Kidney Diseases, following a healthy eating plan can help. Healthcare providers often recommend a diet for people with polycystic kidney disease called the DASH diet, which focuses on fruits, vegetables, whole grains and foods lower in sodium.

Kidney Dialysis

From Renal and Urology News

ADPKD Linked to Lower Mortality in Peritoneal Dialysis Patients

Patients with autosomal dominant polycystic kidney disease (ADPKD) who are on peritoneal dialysis (PD) have a lower death risk than non-ADPKD patients on PD, findings from a new meta-analysis presented at the National Kidney Foundation's 2018 Spring Clinical Meetings suggest.

The meta-analysis, which was presented by first author Boonphiphop Boonpheng, MD, of East Tennessee University in Johnson City, included 12 observational studies with a total of 14,673 patients on PD (931 with and 13,742 without ADPKD). Compared with the non-ADPKD group, the patients with ADPKD had a significant 32% decreased odds in a pooled analysis. The investigators found no significant associations between ADPKD status and the risks of technique failure or peritonitis.

Separately, in a 12-year study presented at the 2018 Annual Dialysis Conference in Orlando, Florida, researchers found lower mortality rates among patients with vs without ADPKD regardless of whether their initial ESRD treatment modality was PD (17.4% vs 24.7%) or intermittent hemodialysis (26.8% vs 39%).

In patients with ADPKD-associated ESRD, enlarged polycystic kidneys within the peritoneal cavity raise concerns about increased risks of abdominal hernia and dialysate leak related to increased abdominal pressure, senior author Wisit Cheungpasitporn, MD, of the University of Mississippi Medical Center in Jackson, explained. “However, in our meta-analysis, we demonstrated that the risk of technique failure was not significantly different between ADPKD patients and non-ADPKD patients on PD,” he told Renal & Urology News. “Thus, notably, the concerns about increased risks of abdominal hernia and dialysate leak did not translate to higher technique failure of PD requiring the need to transfer to hemodialysis.”

Dr Cheungpasitporn noted that the follow-up time for all studies included in this meta-analysis was less than 5 years, which is relatively short. Consequently, future studies are required if survival benefits of PD over hemodialysis among ESRD patients with ADPKD may decrease over time.

From PKD Foundation

Video: From Care to Cure

From Modern Healthcare, By Virgil Dickson

Medicare cut to ambulance pay threatens dialysis patients' access

Patient transportation providers are worried that a looming cut to ambulance reimbursement could cause some dialysis patients to lose access to care, as the new rate reimburses companies below their cost to provide rides.

The CMS has started sending notices to Medicare administrative contractors telling them to reduce reimbursement for regular, non-emergency ambulance transportation for dialysis appointments by 13% starting Oct. 1. Congress called for the cut in its February continuing resolution, known as the Bipartisan Budget Act of 2018.

The agency notes that this cut will be on top of a 10% reduction that took place in 2013, so reimbursement for such rides will be 23% less than they were five years ago.

Joyce Noles, who runs emergency medical services for the West Tennessee Healthcare system based in Jacksonville, said she expects the base reimbursement rate for such services to fall to $165 per transport compared to the $264 it was receiving on average before the 23% cut.

"This is going to be a large impact on many services and the cost will have to be passed on somehow," Noles said.

Once the cut kicks in, she expects to lose money for dialysis rides under the new reimbursement plan. On average, West Tennessee Healthcare's emergency transportation services unit will lose at least $65 per ride, Noles said.

West Tennessee Healthcare may switch to using stretcher vans for rides due to the reimbursement cut, which would mean that only non-medical personnel would be in the vehicle, leaving patients without immediate help if they experience a medical episode in transit.

Josh Watts, CEO of MedTrust, an ambulance provider in South Carolina, said his company has all but stopped taking on dialysis patients over the last year as revenue dropped for such rides.

While patients in his territory had other transport options, he expects healthcare access will suffer in rural areas. If ambulance providers in those regions also stop taking on dialysis patients due to the cut, there aren't other options for those patients, he said.

"In these areas, this will absolutely devastate the access to care transportation for at-risk patients," Watts said.

Transportation for dialysis patients can mean the difference between life and death for some, as many aren't able to drive or don't own cars, according to Alice Andors, a spokeswoman for the American Kidney Fund, a charity dedicated to helping kidney patients pay their insurance costs.

"Having a good transportation system is vital for dialysis patients, whose lives depend on getting to treatment three times a week," Andors said.

Ambulance providers across Southern and Mid-Atlantic states are especially furious about the cut, as they are now operating under a demonstration that requires Medicare beneficiaries to obtain prior authorization for regular, non-emergency transportation in order for the rides to be covered. The experiment spans eight states and the District of Columbia.

"It's a double hit if you're in" one of these states, said Dean Bollendorf, vice president of Healthfleet Ambulance, based in Fort Washington, Pa. "We're being treated differently from other providers in different states as they are not subject to both the cut and prior authorization."

Prior to implementing the model, spending on repetitive, scheduled non-emergent ambulance transports in the three initial states averaged $18.9 million per month, according to a CMS spokesman. By the start of 2017, spending decreased to an average of $6 million per month.

While ambulance providers supported the prior authorization demonstration because they believed it would weed out bad actors, they say they feel punished by the latest reimbursement cut.

"We've already paid our dues," Bollendorf said. "We've worked with the government to address the fraud problem."

Fraud experts agree that incidents of ambulance companies' improperly billing Medicare have dropped in recent years, but problems persist. Notable cases of improper billing for dialysis rides have been the subject of legal action in California, Ohio and Georgia over the last two years.

"There are bad players in the market who are in the business acting only with criminal intent," said Anthony Minge, a partner with Fitch & Associates who specializes in medical billing. "In my opinion, the government should shift more of the focus to rooting out these elements."

Nearly all Medicare fraud cases, including those committed by ambulance providers, are filed under seal under the False Claims Act and take years to investigate and prosecute. During that period, no one but the investigating authorities and the judge who has the case are aware of the fraud, according to Jim Barger, an attorney with the law firm Frohsin & Barger who specializes in False Claims Act lawsuits.

"Accordingly, there is no way to access the number of frauds that are currently under investigation. Our office, for one, has continued to receive reports of Medicare ambulance fraud related to non-emergent dialysis transport," Barger said.

Both the prior authorization demonstration and the cuts to reimbursement are the result of historical abuse by some ambulance providers of the non-emergency benefit.

A 2010 report from HHS' Office of Inspector General indicated that 20% of the agency's spending on non-emergency ambulance trips were improper because ambulance companies overbilled Medicare or transported people who didn't need or qualify for the service.

In 2012, Medicare Part B paid $5.8 billion for ambulance transports, almost double the amount it paid in 2003, according to the OIG.

CVS to Offer Home Dialysis, Kidney Age, Bionic Kidney Development, Help for Battling Transplant Surgery

Kidney News

From MedicalXpress, 
Nuffield Department of Primary Care Health Sciences, University of Oxford

'Kidney age', not kidney disease

There should be a rethink in how doctors talk to some patients with reduced kidney health, replacing the term 'chronic kidney disease' (CKD) with different bands of kidney age, according to a group of experts writing in the Canadian Medical Association Journal.

From the Universities of Oxford, Bristol and Johannesburg, the researchers argue that for some people a reduced level of kidney function is not necessarily a disease, but a normal and asymptomatic sign of ageing, given the clear link between decreasing kidney health and increasing age.

Since 2002, the different stages of CKD have been used by doctors to label reduced glomerular filtration rate (GFR) – the rate at which blood passes through tiny capillaries in the kidney each minute, but this leads to confusion and worry for some patients.

"Qualitative studies show that communicating a diagnosis of 'CKD' to patients can be uncomfortable and unsatisfactory for all concerned, and primary care physicians often face an up-hill battle to retrieve the situation with reassurance," said lead author Richard Stevens, an Associate Professor at Oxford University's Nuffield Department of Primary Care Health Sciences.

"Some doctors prefer not to mention the condition to patients so as to avoid any unnecessary worry, given that a reduced but stable kidney function in elderly patients requires regular monitoring rather than immediate intervention."

CKD stage 5 (where the filtration rate falls below 15 ml/min/1.73m2) is classed as disease at any age, since the kidneys no longer function and dialysis is required.

However, after analysing previously published data from the National Health and Nutrition Examination Survey (2015/16), the researchers argue that CKD up to stage 4 (where GFR declines from 90 to 15 mL/min/1.73m2), does not meet the criteria for disease. Instead they link this with increasing age, from 51 to 95 years old respectively, since this gradual decline in kidney function is not abnormal in older age groups.

When a person's kidney age is higher than their actual age, the researchers suggest doctors communicate this with discussion of further monitoring and in the context of potential health implications, such as the risk of cardiovascular disease or end-stage renal disease.

Co-author Professor Richard Hobbs, an inner-city Birmingham GP and Head of Oxford University's Nuffield Department of Primary Care Health Sciences says that applying the same concepts used by doctors to describe heart or vascular age would likely have greater resonance with patients.

"Relabelling CKD stages up to 4 as categories of normal kidney ageing would clear up a common misconception by patients that they require dialysis and transplant due to the use of terms 'chronic' and 'disease', when their condition is just a normal sign of ageing. For example, a doctor would tell their patient that their kidney age is 68 to 77 years, instead of diagnosing CKD stage 3A."

The researchers stress that kidney disease remains a significant health concern for many and the term kidney age should only apply to age-related kidney decline. Conditions affecting the kidney such as nephrotic syndrome and polycystic kidneys fall outside the scope of the definition of kidney ageing.

Before a widespread adoption of the kidney ageing terminology by health professionals, the researchers call for further discussion with patient groups and broader studies of kidney decline across different populations and ethnic groups to better understand the link between kidney health and ageing.

The research is funded by the National Institute for Health Research (NIHR) School for Primary Care Research.

From Chain Store Age, BY DAVID SALAZAR

CVS Health delves deeper into monitoring, providing care for chronic diseases

CVS Health wants to help improve patient outcomes and control costs for patients with chronic kidney disease.

The company is launching a new initiative that will focus on early identification of kidney disease and an expanded home dialysis offering designed to help optimize care and contain costs for patients with chronic kidney disease. The program, which includes the introduction of new home hemodialysis technology, positions the company to positively disrupt and reshape the kidney care space, CVH Health said.

“In dialysis today, there is an enormous unmet medical need with high levels of mortality, frequent hospitalizations and poor quality of life for affected patients,” said Alan Lotvin, M.D., executive VP and head of CVS Specialty. “As we explored this area it became clear that our enterprise assets from our experience with complex patient home care through Coram, the breadth of our chronic disease management capabilities with CVS Specialty and Accordant, and our deep payer relationships at CVS Caremark will enable us to create a unique value proposition to help reshape dialysis treatment.”

The company will approach the new program in stages, with the initial efforts focused on early identification and patient education, followed by the development of a comprehensive home dialysis program. As part of the program, CVS Health will begin a clinical trial to demonstrate the safety and efficacy of a new hemodialysis device that it plans to submit to the FDA to get market clearance.

“The device has been designed with features intended to make home hemodialysis simple and safe for patients, in order to facilitate longer, more frequent treatments,” CVS Health stated. “In published clinical research, longer, more frequent hemodialysis treatments lead to better outcomes in appropriate patient populations.”

Roughly 700,000 Americans have end-stage renal disease, and roughly half a million of them are on active dialysis, with 120,000 new cases diagnosed annually. In addition to the cost burden associated with the illness — it costs Medicare nearly $65 billion a year and another $34 billion related to patient care — its outcomes lag. Mortality rates for in-center dialysis patients are 10 times higher than the general Medicare population.

“While in-center dialysis clinics are currently the most common choice for hemodialysis treatment, published clinical research has shown improved cardiac health, metabolic control, and survival for patients who are treated with longer, more frequent dialysis treatments,” said CVS Specialty chief medical officer Bruce Culleton. “This treatment paradigm is best delivered in the convenience of a patient’s home. CVS Health is uniquely positioned to build a solution that will enable us to identify and intervene earlier with patients to optimize the management of chronic kidney disease, while at the same time making home dialysis therapies a real option for more patients.”

From The Salem News, MA, By Jean DePlacido Correspondent

Jean DePlacido column: Adams sets up website to help those battling transplant surgery

Linda Adams knows how difficult it can be to go through transplant surgery — and the former Beverly resident wants to help others.

She set up the Live and Battle web site (liveandbattle.com) and, after years of dialysis three times a week for three-and-a-half hour sessions, was fortunate to have a former co-worker donate a non-matching kidney for her transplant at Georgetown University Hospital in 2009.

A Beverly High graduate, Adams has been through a lot: sickness, surgeries, depression. But through it all, she has remained determined to help others.

Setting up her charity — live the dream, battle the nightmare — was the first step.

"I was going through a hard time with the dialysis treatment while waiting for a kidney, and I always thought I had to make the best of a really bad situation. So I talked to other people every time I went to the clinic," said Adams, who is married to Tommy Hayes, a former Beverly High quarterback (Class of 1984). His late father Bill was a well-known basketball coach and high school football referee.

"I realized I was making people a little happier, and want to continue to do all I can to help make the situation a little better."

Adams works out regularly and eats healthy foods. Doctors encourage kidney transplant recipients to keep up physical activity. She had polycystic kidney disease, something her mother suffered from for years and causes kidney failure.

Following the donation of a life saving kidney by former co-worker John Willinger, Adams suffered acute rejection, which landed her back in the hospital. She was later diagnosed with T cell lymphoma and, five years ago, with melanoma.

While the list of health problems would discourage anyone, Adams kept thinking positive thoughts, and her goal to help others never wavered.

Former Peabody High football star Mike Ryan urged Adams to keep lifting weights and reach out to others going through similar health difficulties. When Adams and Hayes lived in Las Vegas two years ago before moving to Jupiter, Fla., they traveled to Venice, Calif. to visit Ryan, who is a famous trainer for the stars, and work out at his facility.

"Mike is a great friend of ours and I worked out with him," said Adams. "He encouraged me to do all the things I need to in order to be as healthy as I possibly can.

"Through my live and battle charity, I hope to raise money to help others. For one thing, those dialysis chairs are the most uncomfortable things. I would like to see something better used. I want to raise money to travel to hospitals to talk to people waiting to undergo transplants, and another goal is getting hotels in big cities to offer discounts to families with loved ones in the hospitals nearby. I know of one family that had to spend three months in a hotel, which is so expensive. Those are some of the things I want to help with through this charity."

Artificial Kidney

From WSMV, Channel 4, NBC Affiliate Nashville, TN, Reported by Alan Frio

Vanderbilt doctor working to develop a bionic kidney

There are two options for a person with kidney failure. A kidney transplant or dialysis, a machine that filters the blood of toxins, normally cleaned by healthy kidneys.

Thanks to research conducted at Vanderbilt University Medical Center and five other universities around the country, a third option may be available that can mean people on dialysis can lead a normal life as if they didn't have kidney failure at all.

Right now without dialysis, a person with kidney disease can live for only a few weeks, perhaps months.

A tiny device that can fit in the palm of your hand may be a game changer. It’s called the bionic kidney.

Vanderbilt nephrologist Dr. William Fissell is part of a nationwide team of doctors and biologist who have been working on the device for 17 years.

"It's fair to call it a bionic kidney because it's a hybrid of technology and living cells," said Fissell.

It's been a long journey for Fissell. He hopes to begin clinical trials on the bionic kidney later this year and to get the device to kidney patients as soon as safety permits.

"Know that we are doing it as fast as we can, and know that the rest of the country has mobilized in a way you may not expect," said Fissell.

If the bionic kidney makes that huge jump from clinical trials to where it becomes available to all kidney patients, the implications for someone in renal failure is huge.

"What I want to do is enough, so that sick people can become well, not burdened by the disease, so that patients aren't stuck to a machine plugged into a wall 15 hours a week, so they don't have to have a special diet, so that patients don't have to starve themselves to avoid intoxication with waste products," said Fissell.

PKD Changes Life Direction, Customize Kidney Dialysis, PKD Genomics: Pre-Natal Diagnosis

Living with PKD

From the Stoke Sentinel, United Kingdom, ByJenny Amphlett

Kidney transplant donor launched new business with help from Google 

Business hasn’t always been a piece of cake for Danielle Hyman. While her afternoon tea delivery service now treats guests to an exquisite experience to celebrate weddings, retirements and more, getting it off the ground took some real, digital dedication.

For more than two decades, 50-year-old Danielle from Moseley worked as the manager of her local doctors surgery, where her husband was the GP.

When he fell ill with polycystic kidney disease, Danielle’s work outlook changed.

“There were no compatible transplant donors to help my husband, so we went through the Paired Kidney Exchange,” says Danielle. “I donated my kidney to someone else, and in turn my husband received a kidney.

“It was a turning point for us. I’m quite creative so I knew this was the time to make a change.

“I love baking, and everyone said that I should consider making my afternoon teas for other people. So I gave it a go.

“I wanted to do hotel-style afternoon tea, very minimalist and smart, served on white bone china with crisp, white napkins. So I made a very basic website and some business cards which I left around.

”Even with delicious finger sandwiches, scones and sweet treats on offer, Danielle didn’t hear anything from her initial attempt.

“I was so disheartened. I knew I could offer a great service, but I needed to look professional online.

“I’d heard about the Google Digital Garage on the radio, and popped in when I was passing while shopping in town. The Google trainers were so friendly and knowledgeable, and they soon gave me confidence that I just didn’t have.

”Danielle went to 10 sessions initially, learning social media strategy, how to form a digital marketing plan and more.

“They do one-to-one sessions too, so I’d take what I’d learnt, put it into practice and then go back with more questions! They were always so understanding and helpful, and I’d take them brownies or cakes as a treat!

“They helped me to tidy up my site and to launch my social media pages. I had this idea that I needed to do everything at once - Facebook, Twitter, Instagram - and I struggled with that.

“They reassured me that wasn’t the case - that I could just focus on one and get it right - and my skills grew from there.

”Now Danielle has a new, growing social media presence and she’s been getting great reviews online. It’s given her the confidence to further grow her business.

“I set myself up on Google My Business and the team at the Garage even taught me to use Google forms to take orders online - it’s a brilliant tool and it looks so much more professional. I have videos and more on my pages now too.

“It doesn’t matter what level you’re at, going to the Google Garage helps build confidence. You’re dealing with professionals and they help you develop by sharing their expertise for free.

“I met so many people there, of all ages and abilities, and the Google trainers used our real life situations in their practical training.

“I’d encourage anyone to go.”

• The Google Digital Garage is open on 134B New St, Birmingham B2 4NS . Expert coaches are there to answer any questions you have about creating your CV or improving your digital skills.

From Medical Tourism Magazine, by Greg Okhifun

How Precision Medicine is Reshaping Healthcare in Europe

Genomic medicine, also termed personalized medicine, precision medicine, and stratified medicine, was introduced into medical science with the success of the Human Genome Project about two decades ago. Since then, it has led to groundbreaking advances in diagnosis and treatment of disease.

Precision medicine employs an individual’s unique genetic profile and DNA sequences to determine their susceptibility to disease, the most suitable and individualized treatment for their disease and focused preventive strategies to adopt. This will, in no small way, reduce the number of unnecessary procedures and exposure to unnecessary and potentially toxic drugs administered to patients.

Genomics has led to the development of cutting-edge drug therapies that simplify the treatment of certain diseases. Genomic medicine is associated with high success rates and efficacy at reduced costs to the patients.

Europe is now taking the lead in pushing investments, innovation, and research in this novel field as genomics has advanced the treatment and diagnosis of a number of diseases in this region including cancer, diabetes, and rare metabolic diseases, as stated in a report in Future Medicine.

Precision medicine not only makes healthcare personalized for patients, it saves them a lot of money. It is fast becoming a booming market in the medical field. According to Markets and Markets, the global market for genomic medicine was worth $13.45 billion in 2016 and it is estimated to reach $23.88 billion in 2022.

While genetic testing and biogenetics have been well-established fields in the past in Europe, it has only recently been tapped into for its benefit in therapeutics and advanced diagnosis. For example, the prenatal diagnosis of certain pediatric conditions was developed by the pioneering Department of Pediatrics at the University of Athens in 1976.

The Department of Medical Genetics at the Choremon Research Laboratory of the University of Athens uses modern techniques in genomics for prenatal diagnosis of a number of genetic disorders including Wilson disease, muscular dystrophies, polycystic kidney disease, and rare disorders such as mitochondrial disorders.

Queen Elizabeth University Hospital in Glasgow is taking steps to become a global leader in genomic medicine. Teaming up with Aridhia, a clinical genetic company in the city, it is developing therapies through genomics for the treatment of cancer, rheumatoid arthritis, COPD, and multiple sclerosis.

A number of breakthrough innovations and discoveries in clinical genetics have been made to provide and individualize healthcare in Europe. For example, Jason Chin and Oliver Rackham, finalists at the European Inventor Award 2012, created a method of producing custom-made proteins using DNA sequencing. These procedures have been successfully employed for development of therapies in insulin treatment and cancer treatment.

The European Union (EU) has begun developing policies that will see precision medicine advance healthcare in Europe. It has, since 2010, invested heavily in genomics with a total of €3.2 billion driven into research and innovation in precision medicine. About a third of this investment has been channeled through the Innovative Medicines Initiative (IMI), the largest public-private partnership globally in the field of biological sciences.

The IMI was developed by concerted efforts of the EU and the European Federation of Pharmaceutical Industries and Associations with the aim of promoting drug research and innovations.

The IMI precision medicine project has produced significant advances in medicine across Europe. In one instance, a project by IMI tagged NEWMEDS (Novel methods leading to new medications in depression and schizophrenia) has revealed the genetic variants in the development of schizophrenia and autism.

Another IMI project revealed, with the help of DNA sequencing, that there are three different subtypes, which previously was unclassified and treated as a single type, ensuring each asthma patient receives individualized treatment based on the asthma subtype they suffer from.

In a novel project, called The Glioma Actively Personalized Vaccine Consortium, researchers, and geneticists from a number of EU countries including Denmark, Germany, The Netherlands, the UK, Switzerland, Spain, and Israel are developing a personalized immunotherapy for the treatment of Glioma.

Since 2010 some laws have been adopted by the EU to ensure precision medicine is developed and placed at the center of healthcare in Europe.

Some of these regulations include the Clinical Trail Regulation which promotes the conduct of clinical trials on genomics in the EU, the General Data Protection Regulation which ensures precision medicine and the techniques involved are protected under the law, and the In Vitro Diagnostics and Medical Device Legislation which aims to promote legislation in favor of technological and research advancement in precision medicine.

The European Commission launched an initiative “Personalized Medicine 2020 and beyond – Preparing Europe for leading the global way (PerMed)”. This initiative was birthed with strategies to develop awareness and empowerment among stakeholders, integrate information and ICT solutions, encourage clinical research, and shape healthcare around precision medicine.

This has led the Director General of Research and Innovation of the European Commission to begin discussions with researchers and policymakers from all around Europe. These discussions further led to the creation of an International Consortium for Personalized Medicine, or IC PerMed.

The IC PerMed has created plans and strategies to perform its key responsibilities which include;
Infuse precision medicine into basic healthcare
Provide evidence-based treatment options for citizens of the EU
Establish Europe is a major key player in precision medicine
Promote strong research in precision medicine.

According to a 2015 report by the European Alliance for Precision Medicine, more work still needs to be done in tapping from the well of resources that genomics has in store. The report noted that there is currently no screening guideline for Lung cancer, the continent’s number one cause of cancer deaths, further recommending the need for education of patients and wider screening programmes.

The era of precision medicine holds a lot of promise in paving the way for patients to receive effective care and eliminating unnecessary cost and drug adverse effects. However, there are a lot of challenges for this budding field in Europe.

One of such challenges is the management and control of the enormous amount of patient information genomic medicine would make available, a phenomenon termed as a “genomic tsunami” by the European Society of Human Genetics.

In a bid to properly manage the amount of patient data exposed to researchers, scientists, and doctors, the EU launched the IT Future of Medicine to ensure the privacy of patient’s health data and keep personalized medicine truly personal.

Learn more about precision medicine and the future of genomics here.

Kidney Dialysis

From MedicalXpress

New microsensor enables kidney dialysis customization

Microscope picture of the microfluidic device developed by Manoj Sharma. The horizontal stripe is the microchannel, which measures 0.2 mm across. The six other stripes are optic fibers that capture the fluourescent light and lead it to a spectrometer. The fifteen dots in the middle are micropillars. Credit: Eindhoven University of Technology

Researchers from TU Eindhoven (Netherlands) have developed a microsensor that makes it possible to directly monitor and adjust the composition of kidney dialysis fluid. This is a crucial step towards patient specific dialysis, which will remove a significant proportion of the serious side effects of the use of standard dialysis fluid. On 29 March Manoj Kumar Sharma will be awarded a Ph.D. for his research on this sensor.

Kidney failure makes around two million people worldwide dependent on kidney dialysis to clean their blood. A tube is connected to a blood vessel and the blood is passed along a membrane, with dialysate (dialysis fluid) on the other side. Because the concentration of salts in the blood is higher than in the dialysate, salt passes through the membrane and enters the dialysate. The rate at which this happens depends on the difference in concentration between blood and dialysate. Since the concentration of salts varies widely among different patients, and the concentration in dialysate has the same standard value, the speed is often not ideal. This causes serious side effects, such as heart rhythm disturbance and renal bone disease. It would be better to continuously adjust the concentrations of salts in the dialysate so that they are optimal for the patient. This, however, requires that the concentrations of salts in the dialysate can be monitored live, but there had not been a reliable technique for that to date.

Manoj Kumar Sharma has devised an ingenious solution for this. He developed a micro-system with a centrally positioned microchannel through which dialysate flows. He covered the walls of the microchannel with sensor molecules, which are only fluorescent in the presence of a salt, such as sodium. The more sodium there is in the dialysate, the stronger the fluorescence. To reinforce this effect, he introduced micropillars into the microchannel, resulting in even more surface covered with sensor molecules.

A laser light shines on the microchannel, and activates the fluorescence of the sensor molecules. Sharma captures this fluorescence using glass fibers that he connected to the channel in the micro-system. The light passes through the fibers to a spectrometer for analysis. The laser light, which is of a different wavelength, is first filtered out. Then, based on the measured intensity of the fluorescence, the sodium concentrations can be read out.

It is important to ensure that the sensor molecules are not disturbed by other salts, so that a pure measurement of the concentration of a specific salt is possible. The 'microfluidic sensor system' of approximately 5x2 centimeters built by the Eindhoven researcher is able to measure sodium, the most important salt in the blood, accurately and live. He expects that it will be relatively easy to extend the micro-system with channel sections coated with other 'photo-induced electron transfer' (PET) sensor molecules, which are sensitive to the other essential salts, such as potassium and phosphate.

Sharma thinks that his technique has a very good chance of being used in dialysis machines. The technique is relatively cheap, stable and very accurate. In addition, he expects that the size of his sensor system can be further reduced, to about 1x1 centimeter, facilitating integration into dialysis machines. His technique may also eventually become part of a portable artificial kidney, a solution that will significantly ease the life of kidney patients.