From The Toldeo Blade, Ohio, BY ALAN BAVLEY, KANSAS CITY STAR
Early detection is key so it’s easier for doctors to intervene
KANSAS CITY — Two-year-old Marley Martinac has a serious chronic kidney disease, but she’s going to have a better shot at a healthy life than kids born just a decade earlier.
Thanks largely to an ongoing national study with leadership at Children’s Mercy Hospital in Kansas City, doctors now have a clearer picture of the best ways to stop or slow the progression of kidney disease in children like Marley.
The goal is to keep the children growing and thriving for as long as possible, preventing, or at least postponing, the need for kidney dialysis or a transplant.
“Thank God, we’re not at that point at all,” said Marley’s mother, Katie Martinac of Bates City, Mo. “She’s pretty spunky. She hasn’t lost that.”
About 16 percent of the U.S. population has chronic kidney disease, a gradual loss of kidney function caused by high blood pressure, diabetes and a variety of other conditions. How many children have these conditions isn’t known, but 2,500 children nationwide are on dialysis and more than 5,000 have received kidney transplants.
“We’re aiming to head things off in childhood,” said Children’s Mercy kidney specialist Bradley Warady, “to prevent children from requiring dialysis or a kidney transplant, or maybe delay it to give children more chance to grow and for their minds to develop.”
Warady, along with researchers at Children’s Hospital of Philadelphia, is coordinating the Chronic Kidney Disease in Children (CKiD) Cohort, a study that since 2003 has been following close to 900 children with mild to moderate kidney disease recruited from medical centers across the United States and Canada. Their research, funded by the National Institutes of Health, is now the largest long-term study in North America of any chronic childhood disease.
Research by the CKiD group, published recently in the American Journal of Kidney Diseases, shows that the severity of three common conditions in children with kidney disease — high blood pressure, anemia and protein loss through urine — predict how quickly their disease will worsen. For example, when urine has high protein levels, kidneys deteriorate twice as fast.
Because these conditions are all treatable, Warady said, the study offers doctors guidance for improving their patients’ care.
“We want to detect it when it’s mild and easier to intervene,” he said. “We have the tools to treat these things. But the treatment hasn’t been done yet in a consistent way. This emphasizes the importance of these factors. It raises awareness of how significant they are.”
Doctors have not had much data on childhood kidney disease, said Joseph Vassalotti, chief medical officer of the National Kidney Foundation.
This research “really advances our understanding of the natural history of chronic kidney disease in children.”
These diseases include birth defects, such as abnormally developed kidneys that don’t work well or blockages in the kidney’s plumbing; genetic conditions such as polycystic kidney disease, in which fluid-filled cysts destroy kidney tissue; and a variety of diseases that attack the hundreds of thousands of tiny clusters of blood vessels that alter the blood.
“There’s a lot to childhood kidney disease,” Warady said. “It’s a lot more complicated than ‘I just don’t pee.’”
Marley has a condition called nephrotic syndrome that causes her kidney damage. Doctors at Children’s Mercy aren’t sure what is responsible, but they’ve been able to keep the condition under control. [Read more]
Thanks largely to an ongoing national study with leadership at Children’s Mercy Hospital in Kansas City, doctors now have a clearer picture of the best ways to stop or slow the progression of kidney disease in children like Marley.
The goal is to keep the children growing and thriving for as long as possible, preventing, or at least postponing, the need for kidney dialysis or a transplant.
“Thank God, we’re not at that point at all,” said Marley’s mother, Katie Martinac of Bates City, Mo. “She’s pretty spunky. She hasn’t lost that.”
About 16 percent of the U.S. population has chronic kidney disease, a gradual loss of kidney function caused by high blood pressure, diabetes and a variety of other conditions. How many children have these conditions isn’t known, but 2,500 children nationwide are on dialysis and more than 5,000 have received kidney transplants.
“We’re aiming to head things off in childhood,” said Children’s Mercy kidney specialist Bradley Warady, “to prevent children from requiring dialysis or a kidney transplant, or maybe delay it to give children more chance to grow and for their minds to develop.”
Warady, along with researchers at Children’s Hospital of Philadelphia, is coordinating the Chronic Kidney Disease in Children (CKiD) Cohort, a study that since 2003 has been following close to 900 children with mild to moderate kidney disease recruited from medical centers across the United States and Canada. Their research, funded by the National Institutes of Health, is now the largest long-term study in North America of any chronic childhood disease.
Research by the CKiD group, published recently in the American Journal of Kidney Diseases, shows that the severity of three common conditions in children with kidney disease — high blood pressure, anemia and protein loss through urine — predict how quickly their disease will worsen. For example, when urine has high protein levels, kidneys deteriorate twice as fast.
Because these conditions are all treatable, Warady said, the study offers doctors guidance for improving their patients’ care.
“We want to detect it when it’s mild and easier to intervene,” he said. “We have the tools to treat these things. But the treatment hasn’t been done yet in a consistent way. This emphasizes the importance of these factors. It raises awareness of how significant they are.”
Doctors have not had much data on childhood kidney disease, said Joseph Vassalotti, chief medical officer of the National Kidney Foundation.
This research “really advances our understanding of the natural history of chronic kidney disease in children.”
These diseases include birth defects, such as abnormally developed kidneys that don’t work well or blockages in the kidney’s plumbing; genetic conditions such as polycystic kidney disease, in which fluid-filled cysts destroy kidney tissue; and a variety of diseases that attack the hundreds of thousands of tiny clusters of blood vessels that alter the blood.
“There’s a lot to childhood kidney disease,” Warady said. “It’s a lot more complicated than ‘I just don’t pee.’”
Marley has a condition called nephrotic syndrome that causes her kidney damage. Doctors at Children’s Mercy aren’t sure what is responsible, but they’ve been able to keep the condition under control. [Read more]
From Charleston Daily Mail, West Virginia, by Charlotte Ferrell Smith
Anna Faye Ray, 77, was 66 when she was given another chance at life thanks to the unselfish gift of two kidneys from a family who lost a 2-year-old child.
“I’ll do everything I can to take care of my body so a part of their child will continue to live,” Ray said.
Ray, a resident of Edgewood Summit, works part-time as a ministerial assistant and is a major advocate for organ donation as she frequents health fairs and serves as a speaker at numerous venues.
However, there was a time that poor health took such a toll that she barely had the energy to walk across a room.
In 1971 she was diagnosed with polycystic kidney disease, a potentially fatal condition she inherited from her father. She tried medication to slow down the disease, but it continued to progress.
By 2003 she was placed on dialysis. At age 65, she was put on a transplant list and told it would likely take up to five years to find a match.
After 12 months, a match was found and she received two kidneys from a 2-year-old who had died.
Her surgery was performed 9 p.m. Feb. 15, 2004 at Charleston Area Medical Center’s General Division. When her pastor came to pray with her before the operation, she told him she was not afraid because she would be fine whether she continued life on earth or in heaven.
“I told him ‘if anything happens, I am going to the best home I ever had.’” she said.
She asked hospital staff if someone else could have those healthy kidneys if she did not survive. She was told it was necessary to be registered as an organ donor before that could happen. She was surprised to learn that, despite health issues, many other parts of her body could be used as well. She immediately became an organ donor.
While she continues to take necessary medications to care for her new kidneys, they have remained healthy and continued to grow.
Her life was saved thanks to a family she may never know because the entire process is confidential. However, she thinks of that family often and strives to do what she can to help others by working hard as an advocate for organ donation. Prior to the surgery, she was very shy but has forced herself to become an extrovert in order to be a good speaker. [Read more]
Drinking Gallon of Iced Tea Daily May Have Caused Arkansas Man's Kidney Failure, Doctors Say
“I’ll do everything I can to take care of my body so a part of their child will continue to live,” Ray said.
Ray, a resident of Edgewood Summit, works part-time as a ministerial assistant and is a major advocate for organ donation as she frequents health fairs and serves as a speaker at numerous venues.
However, there was a time that poor health took such a toll that she barely had the energy to walk across a room.
In 1971 she was diagnosed with polycystic kidney disease, a potentially fatal condition she inherited from her father. She tried medication to slow down the disease, but it continued to progress.
By 2003 she was placed on dialysis. At age 65, she was put on a transplant list and told it would likely take up to five years to find a match.
After 12 months, a match was found and she received two kidneys from a 2-year-old who had died.
Her surgery was performed 9 p.m. Feb. 15, 2004 at Charleston Area Medical Center’s General Division. When her pastor came to pray with her before the operation, she told him she was not afraid because she would be fine whether she continued life on earth or in heaven.
“I told him ‘if anything happens, I am going to the best home I ever had.’” she said.
She asked hospital staff if someone else could have those healthy kidneys if she did not survive. She was told it was necessary to be registered as an organ donor before that could happen. She was surprised to learn that, despite health issues, many other parts of her body could be used as well. She immediately became an organ donor.
While she continues to take necessary medications to care for her new kidneys, they have remained healthy and continued to grow.
Her life was saved thanks to a family she may never know because the entire process is confidential. However, she thinks of that family often and strives to do what she can to help others by working hard as an advocate for organ donation. Prior to the surgery, she was very shy but has forced herself to become an extrovert in order to be a good speaker. [Read more]
From ABC News, Good Morning America
Doctors believe a man's excessive iced tea drinking -- about a gallon daily -- is what caused his previously unexplained kidney failure last year that's been keeping him on dialysis ever since.
The unidentified 56-year-year-old man was admitted to John L. McClellan Memorial Veterans Hospital in Little Rock last May with weakness, fatigue, body aches and an elevated serum creatinine level, which meant his kidneys weren't functioning properly, according to the doctors' letter published in the New England Journal of Medicine Thursday.
The patient's urine sample was "remarkable" for its abundance of calcium oxalate crystals, Dr. Fyed Syed and Dr. Alejandra Mena-Gutierrez told ABC News today. Both doctors treated the patient and co-authored the letter along with Dr. Umbar Ghaffar, who was not immediately available for comment.
Calcium oxalate crystals are molecules that can put someone at high risk to develop kidney stones, Syed and Mena-Gutierrez said.
High levels of oxalate in the body is usually due to a genetic disorder, complications from gastric surgery or consumption of antifreeze, the doctors said, but the patient's history outlined in the letter didn't match any of those explanations.
An explanation was finally found after the patient admitted to drinking 16 eight-ounce glasses of iced tea daily, Syed and Mena-Gutierrez said, adding that black tea, "a rich source of oxalate," accounts for 84 percent of tea consumed in the U.S. [Read more]
Assistant principal in search of kidney donor
MORROW— More than 800 students, parents and faculty of Morrow Middle are expected to walk Friday in support of Assistant Principal Derrica Davis, who has a story to tell about her search for a kidney donor.
Davis, 36, suffers from a rare kidney disease called polycystic kidney disease.
She will be the featured guest at the Morrow Middle School Kidney Walk is a daylong event that will take place on the school’s athletic fields.
She said she was diagnosed at 23. She has been on a waiting list for the past 14 months as the disease has progressed.
“I’m an only child, and I’m the child of an only child, so getting a donor is a challenge,” said Davis, the mother of a 6-year-old boy. “Only 1 percent of the population would be a match for me.”
She said she manages these days with 8 percent of her kidney functions.
“I don’t believe in holding on to hardships in life,” said Davis, who receives notifications with scriptures on her phone several times a day.
She said she tries to stay positive, even when she feels pain and fatigue from her condition.
“There is little help for my disease,” she said. “There is not a lot of research for people with my disease, so giving to organizations such as the National Kidney Foundation is important.”
Davis said residents with the O-blood type, who are interested in donating their kidney, may call the Piedmont Transplant Clinic at 404-605-4605.
“Even if they can’t help me, it’s quite possible they can help somebody else,” she said.
Learn more about Davis’s cause on Facebook by searching “akidneyfor.Derrica.” Also, find out what the school plans to do for its National Kidney Walk event May 30.
Nine years ago Jeremy Williams’ mother was in need of a kidney after she was diagnosed with polycystic kidney disease (PKD). She didn’t ask her son to donate a kidney, but Williams couldn’t sit back and watch her suffer, he said.
He went in for testing to see if he would be a good match to donate a kidney to his mother when, at age 30, he learned something he expected to hear—but not nearly this soon. He too had PKD.
“They did the scans and just by the questions that I asked the girl in the room that she wouldn’t answer, I could tell there was a problem,” Williams said, now 39. “I wasn’t really that upset at the time. I guess I more or less expected it.”
PKD is an inherited disorder that causes clusters of cysts to develop primarily within a person’s kidneys, which can lead to kidney failure. The chance of inheriting PKD is 50 percent, a statistic that rings true in Williams’ family.
His mother had three brothers, and out of the four of them, two had PKD. His uncle passed away from it in his early 40s. Williams’ grandfather also had it and died from it in his 40s.
“I guess I expected all the time growing up that if I was going to get it, I would have serious problems by the time I was in my 40s. I didn’t really expect that I would hear about it when I was 30,” he said.
After Williams’ initial diagnosis, he had his condition monitored twice a year. His kidney function was holding steady around 50 percent. But in December 2013, Williams found out his kidneys only were functioning at 17 percent, and within another year, his kidney function dropped to 9 percent. Williams said, best case scenario, it’s at 9 percent today, though he said it’s likely worse now. It can’t be tested anymore since Williams now undergoes daily peritoneal kidney dialysis.
“It’s so unexpected for me to be as bad as I was,” he said. “I had no reason to believe that I was in any way going to go this far down the hill. The doctor said she was surprised, that she hadn’t ever seen someone’s [kidney function] drop that fast.”
The unexpectedness came as a shock to the Williams family, as they were planning their family. Williams and his wife, Jenni, had their first child, Ashton, in 2003. That was several years before Williams knew he had PKD. He said he and his wife tried for years to have a child before resorting to in vitro fertilization.
“Through the in vitro process, we got her pregnant, and she had him,” he said.
Soon, Williams’ wife wanted more children, but Williams had found out he had PKD. Williams resisted for a while, but he said he caved at the point when he realized his disease wasn’t really affecting him other than high blood pressure at that time.
Through in vitro, Williams’ wife got pregnant again, this time with twins, who are now 2. It was soon after the children were born that Williams’ kidney function plummeted. [Read more]
On Wednesday, officials with Erlanger will raise a “Donate Life” flag in the hospital courtyard to recognize and honor 35 Erlanger patients who gave the ultimate gift of life as organ donors in 2014.
Officials said, "Thanks to the organ donations of these 35 individuals, 123 lives were saved last year."
At 11 a.m., Erlanger will join thousands of hospitals and organizations across the nation by flying the flag in recognition of Donor Awareness Month, and in honor of the lives touched by organ, eye and tissue donation. Representatives from the Erlanger Health System, Tennessee Donor Services, Donate Life, Erlanger’s transplant program and other medical professionals will join Paula Palmer, a kidney transplant recipient, and Paula Boring, mother of an organ donor. [Read more]
Kidney Health
From Santa Monica Mirror, CA, by ALIA TUQAN, M.D
Filtering The Facts On Kidney Health
March is “National Kidney Month,” highlighting the role of our kidneys in maintaining overall health and the importance of keeping them healthy. This month’s column covers kidney health and disease and what can be done to protect these vital organs.
Our kidneys filter our body’s waste to produce urine. They help balance electrolytes, the charged particles needed for cell functioning. They also are involved in maintaining blood pressure and producing red-blood cells.
As we grow older, our kidneys slow down. Their ability to filter waste diminishes. That’s why older people are more susceptible to the effects of alcohol and medications.
Various diseases can decrease kidney function as well. Uncontrolled high blood pressure, also known as hypertension, and diabetes can damage kidneys. Less common diseases, such as lupus, an autoimmune disease, and polycystic kidney disease, a genetic disorder, can affect kidneys too.
In addition, certain medications can affect kidneys. Non-steroidal anti-inflammatory drugs (NSAIDs) – a class of pain medications, including over-the-counter ibuprofen and naproxen – can damage kidneys if taken for prolonged periods of time.
People who take diuretics – certain types of medications commonly used to treat high blood pressure and leg swelling – need periodic blood work to make sure electrolytes and kidney function remain stable on these drugs.
People with kidney disease can be more sensitive to drugs and susceptible to drug side effects because their kidneys no longer filter the body’s waste as efficiently as they once did. In addition, they are at risk of high blood pressure, low red-blood-cell counts, also known as anemia, and bone diseases.
What can you do to maintain kidney health and prevent kidney problems? These recommendations can help reduce your risk:
• Get regular checkups with your doctor, who may order blood tests and review medications.
• Get medical treatment for high blood pressure or diabetes.
• Take over-the-counter and prescriptions drugs as prescribed.
• Review herbal supplements with your doctor to ensure they are safe.
• Eat a healthy diet.
• Exercise regularly, but discuss any new activities with your doctor beforehand.
Follow these simple steps for better kidney health – and a healthier you!
Dr. Alia Tuqan is a board-certified geriatrician with the highly regarded UCLA Geriatrics Program in Santa Monica and Westwood. For more information, call 310.319.4371 or visitwww.uclahealth.org.
From MiBiz, Written by Mark Sanchez
Metabolic Solutions Development Co. plans to seek $25 million to $35 million in additional capital later this year to support the continued development of its new drug targets.
Founded in 2006 by former Upjohn Co. researchers Jerry Colca and Ralph Kletzien, the Kalamazoo-based Metabolic Solutions has already raised about $70 million from investors in the last seven to eight years to develop compounds to treat diseases connected to metabolic dysfunctions. In particular, the company has targeted compounds that could treat kidney, liver, Parkinson’s and Alzheimer’s diseases, as well as Type 2 diabetes.
The additional capital Metabolic Solutions will seek to raise toward the end of 2015 will “accelerate pipeline development and position (the company) for access to public markets in 2016,” CEO Stephen Benoit said during a presentation this month at the BioCentury Future Leaders event during the Biotech Industry Conference in New York City.
“We’re in a pretty good position to achieve a pretty significant inflection in value here over the next 24 months,” Benoit said. “The real benefit here in being where we are relative to what we need to accomplish over the next 24 months is quite a step up in enterprise value for investors.”
Current investors will “continue to participate going forward,” Benoit said.
Davis, 36, suffers from a rare kidney disease called polycystic kidney disease.
She will be the featured guest at the Morrow Middle School Kidney Walk is a daylong event that will take place on the school’s athletic fields.
She said she was diagnosed at 23. She has been on a waiting list for the past 14 months as the disease has progressed.
“I’m an only child, and I’m the child of an only child, so getting a donor is a challenge,” said Davis, the mother of a 6-year-old boy. “Only 1 percent of the population would be a match for me.”
She said she manages these days with 8 percent of her kidney functions.
“I don’t believe in holding on to hardships in life,” said Davis, who receives notifications with scriptures on her phone several times a day.
She said she tries to stay positive, even when she feels pain and fatigue from her condition.
“There is little help for my disease,” she said. “There is not a lot of research for people with my disease, so giving to organizations such as the National Kidney Foundation is important.”
Davis said residents with the O-blood type, who are interested in donating their kidney, may call the Piedmont Transplant Clinic at 404-605-4605.
“Even if they can’t help me, it’s quite possible they can help somebody else,” she said.
Learn more about Davis’s cause on Facebook by searching “akidneyfor.Derrica.” Also, find out what the school plans to do for its National Kidney Walk event May 30.
Community to hold fund raiser to benefit Williams family April 11
Nine years ago Jeremy Williams’ mother was in need of a kidney after she was diagnosed with polycystic kidney disease (PKD). She didn’t ask her son to donate a kidney, but Williams couldn’t sit back and watch her suffer, he said.
He went in for testing to see if he would be a good match to donate a kidney to his mother when, at age 30, he learned something he expected to hear—but not nearly this soon. He too had PKD.
“They did the scans and just by the questions that I asked the girl in the room that she wouldn’t answer, I could tell there was a problem,” Williams said, now 39. “I wasn’t really that upset at the time. I guess I more or less expected it.”
PKD is an inherited disorder that causes clusters of cysts to develop primarily within a person’s kidneys, which can lead to kidney failure. The chance of inheriting PKD is 50 percent, a statistic that rings true in Williams’ family.
His mother had three brothers, and out of the four of them, two had PKD. His uncle passed away from it in his early 40s. Williams’ grandfather also had it and died from it in his 40s.
“I guess I expected all the time growing up that if I was going to get it, I would have serious problems by the time I was in my 40s. I didn’t really expect that I would hear about it when I was 30,” he said.
After Williams’ initial diagnosis, he had his condition monitored twice a year. His kidney function was holding steady around 50 percent. But in December 2013, Williams found out his kidneys only were functioning at 17 percent, and within another year, his kidney function dropped to 9 percent. Williams said, best case scenario, it’s at 9 percent today, though he said it’s likely worse now. It can’t be tested anymore since Williams now undergoes daily peritoneal kidney dialysis.
“It’s so unexpected for me to be as bad as I was,” he said. “I had no reason to believe that I was in any way going to go this far down the hill. The doctor said she was surprised, that she hadn’t ever seen someone’s [kidney function] drop that fast.”
The unexpectedness came as a shock to the Williams family, as they were planning their family. Williams and his wife, Jenni, had their first child, Ashton, in 2003. That was several years before Williams knew he had PKD. He said he and his wife tried for years to have a child before resorting to in vitro fertilization.
“Through the in vitro process, we got her pregnant, and she had him,” he said.
Soon, Williams’ wife wanted more children, but Williams had found out he had PKD. Williams resisted for a while, but he said he caved at the point when he realized his disease wasn’t really affecting him other than high blood pressure at that time.
Through in vitro, Williams’ wife got pregnant again, this time with twins, who are now 2. It was soon after the children were born that Williams’ kidney function plummeted. [Read more]
On Wednesday, officials with Erlanger will raise a “Donate Life” flag in the hospital courtyard to recognize and honor 35 Erlanger patients who gave the ultimate gift of life as organ donors in 2014.
Officials said, "Thanks to the organ donations of these 35 individuals, 123 lives were saved last year."
At 11 a.m., Erlanger will join thousands of hospitals and organizations across the nation by flying the flag in recognition of Donor Awareness Month, and in honor of the lives touched by organ, eye and tissue donation. Representatives from the Erlanger Health System, Tennessee Donor Services, Donate Life, Erlanger’s transplant program and other medical professionals will join Paula Palmer, a kidney transplant recipient, and Paula Boring, mother of an organ donor. [Read more]
Kidney Health
From Santa Monica Mirror, CA, by ALIA TUQAN, M.D
Our kidneys filter our body’s waste to produce urine. They help balance electrolytes, the charged particles needed for cell functioning. They also are involved in maintaining blood pressure and producing red-blood cells.
As we grow older, our kidneys slow down. Their ability to filter waste diminishes. That’s why older people are more susceptible to the effects of alcohol and medications.
Various diseases can decrease kidney function as well. Uncontrolled high blood pressure, also known as hypertension, and diabetes can damage kidneys. Less common diseases, such as lupus, an autoimmune disease, and polycystic kidney disease, a genetic disorder, can affect kidneys too.
In addition, certain medications can affect kidneys. Non-steroidal anti-inflammatory drugs (NSAIDs) – a class of pain medications, including over-the-counter ibuprofen and naproxen – can damage kidneys if taken for prolonged periods of time.
People who take diuretics – certain types of medications commonly used to treat high blood pressure and leg swelling – need periodic blood work to make sure electrolytes and kidney function remain stable on these drugs.
People with kidney disease can be more sensitive to drugs and susceptible to drug side effects because their kidneys no longer filter the body’s waste as efficiently as they once did. In addition, they are at risk of high blood pressure, low red-blood-cell counts, also known as anemia, and bone diseases.
What can you do to maintain kidney health and prevent kidney problems? These recommendations can help reduce your risk:
• Get regular checkups with your doctor, who may order blood tests and review medications.
• Get medical treatment for high blood pressure or diabetes.
• Take over-the-counter and prescriptions drugs as prescribed.
• Review herbal supplements with your doctor to ensure they are safe.
• Eat a healthy diet.
• Exercise regularly, but discuss any new activities with your doctor beforehand.
Follow these simple steps for better kidney health – and a healthier you!
Dr. Alia Tuqan is a board-certified geriatrician with the highly regarded UCLA Geriatrics Program in Santa Monica and Westwood. For more information, call 310.319.4371 or visitwww.uclahealth.org.
PKD Research
Metabolic Solutions Development Co. plans to seek $25 million to $35 million in additional capital later this year to support the continued development of its new drug targets.
Founded in 2006 by former Upjohn Co. researchers Jerry Colca and Ralph Kletzien, the Kalamazoo-based Metabolic Solutions has already raised about $70 million from investors in the last seven to eight years to develop compounds to treat diseases connected to metabolic dysfunctions. In particular, the company has targeted compounds that could treat kidney, liver, Parkinson’s and Alzheimer’s diseases, as well as Type 2 diabetes.
The additional capital Metabolic Solutions will seek to raise toward the end of 2015 will “accelerate pipeline development and position (the company) for access to public markets in 2016,” CEO Stephen Benoit said during a presentation this month at the BioCentury Future Leaders event during the Biotech Industry Conference in New York City.
“We’re in a pretty good position to achieve a pretty significant inflection in value here over the next 24 months,” Benoit said. “The real benefit here in being where we are relative to what we need to accomplish over the next 24 months is quite a step up in enterprise value for investors.”
Current investors will “continue to participate going forward,” Benoit said.
MOVING BEYOND DIABETES
The company has redirected its lead compound, known as MSDC-0602, to be used on nonalcoholic steatohepatitis (NASH), a liver inflammation caused by the buildup of fat, and polycystic kidney disease (PKD), a genetic disorder where cysts develop in the kidneys and impair function. PKD presently has no treatment in the U.S.
An insulin sensitizer, the MSDC-0602 compound was proven to be safe and effective in treating Type 2 diabetic patients in a Phase 2 human clinical trial.
Given the regulatory requirements and an “almost complete inability to raise capital around an asset in Type 2 diabetes,” the company decided to refocus the compound around liver and kidney disease “where the timeline and the cost to market are a lot more reasonable,” Benoit said in the presentation.
Metabolic Solutions plans to move later this year into Phase 2b clinical trials for MSDC-0602 and another compound, called MSDC-0160, which has the potential to treat neurodegenerative diseases such as Parkinson’s and Alzheimer’s, Benoit said.
The Phase 2b study for MSDC-0602 will involve 320 NASH patients over 12 months in at least 60 sites globally and the same number of PKD patients over a year that will include sites in Europe and Japan.
The company has redirected its lead compound, known as MSDC-0602, to be used on nonalcoholic steatohepatitis (NASH), a liver inflammation caused by the buildup of fat, and polycystic kidney disease (PKD), a genetic disorder where cysts develop in the kidneys and impair function. PKD presently has no treatment in the U.S.
An insulin sensitizer, the MSDC-0602 compound was proven to be safe and effective in treating Type 2 diabetic patients in a Phase 2 human clinical trial.
Given the regulatory requirements and an “almost complete inability to raise capital around an asset in Type 2 diabetes,” the company decided to refocus the compound around liver and kidney disease “where the timeline and the cost to market are a lot more reasonable,” Benoit said in the presentation.
Metabolic Solutions plans to move later this year into Phase 2b clinical trials for MSDC-0602 and another compound, called MSDC-0160, which has the potential to treat neurodegenerative diseases such as Parkinson’s and Alzheimer’s, Benoit said.
The Phase 2b study for MSDC-0602 will involve 320 NASH patients over 12 months in at least 60 sites globally and the same number of PKD patients over a year that will include sites in Europe and Japan.
PARTNERSHIPS AHEAD?
Metabolic Solutions should have “some interesting news in the next few months on the partnering front” involving the upcoming clinical studies on NASH and PKD and the capital raise as the company converts from a middle-stage to a later-stage drug development pipeline, Benoit said. [Read more]
Urine Fetuin-A is a biomarker of autosomal dominant polycystic kidney disease progression
Autosomal dominant polycystic kidney disease (ADPKD) is a genetic disorder characterized by numerous fluid-filled cysts that frequently result in end-stage renal disease. While promising treatment options are in advanced clinical development, early diagnosis and follow-up remain a major challenge.
We therefore evaluated the diagnostic value of Fetuin-A as a new biomarker of ADPKD in human urine.
Results: We found that renal Fetuin-A levels are upregulated in both Pkd1 and Bicc1 mouse models of ADPKD. Measurement by ELISA revealed that urinary Fetuin-A levels were significantly higher in 66 ADPKD patients compared to 17 healthy volunteers or 50 control patients with renal diseases of other causes .
Receiver operating characteristics (ROC) analysis of urinary Fetuin-A levels for ADPKD rendered an optimum cut-off value of 12.2 μg/mmol creatinine, corresponding to 94% of sensitivity and 60% of specificity (area under the curve 0.74. Furthermore, urinary Fetuin-A levels in ADPKD patients correlated with the degree of renal insufficiency and showed a significant increase in patients with preserved renal function followed for two years.
Conclusions: Our findings establish urinary Fetuin-A as a sensitive biomarker of the progression of ADPKD.
Further studies are required to examine the pathogenic mechanisms of elevated renal and urinary Fetuin-A in ADPKD.
Author: Nathalie PiazzonFlorian BernetLinda GuihardWouter N LeonhardSéverine UrferDmitri FirsovHassib ChehadeBruno VogtSophia PiergiovanniDorien PetersOlivier BonnyDaniel B Constam
Credits/Source: Journal of Translational Medicine 2015, 13:103
Metabolic Solutions should have “some interesting news in the next few months on the partnering front” involving the upcoming clinical studies on NASH and PKD and the capital raise as the company converts from a middle-stage to a later-stage drug development pipeline, Benoit said. [Read more]
From 7th Space Interactive
We therefore evaluated the diagnostic value of Fetuin-A as a new biomarker of ADPKD in human urine.
Results: We found that renal Fetuin-A levels are upregulated in both Pkd1 and Bicc1 mouse models of ADPKD. Measurement by ELISA revealed that urinary Fetuin-A levels were significantly higher in 66 ADPKD patients compared to 17 healthy volunteers or 50 control patients with renal diseases of other causes .
Receiver operating characteristics (ROC) analysis of urinary Fetuin-A levels for ADPKD rendered an optimum cut-off value of 12.2 μg/mmol creatinine, corresponding to 94% of sensitivity and 60% of specificity (area under the curve 0.74. Furthermore, urinary Fetuin-A levels in ADPKD patients correlated with the degree of renal insufficiency and showed a significant increase in patients with preserved renal function followed for two years.
Conclusions: Our findings establish urinary Fetuin-A as a sensitive biomarker of the progression of ADPKD.
Further studies are required to examine the pathogenic mechanisms of elevated renal and urinary Fetuin-A in ADPKD.
Author: Nathalie PiazzonFlorian BernetLinda GuihardWouter N LeonhardSéverine UrferDmitri FirsovHassib ChehadeBruno VogtSophia PiergiovanniDorien PetersOlivier BonnyDaniel B Constam
Credits/Source: Journal of Translational Medicine 2015, 13:103
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