By David Furlonger
Cystic fibrosis, traditionally regarded as a "white" disease in SA, is proving to be no respecter of colour. Instead of the few dozen patients formerly believed to suffer from the inherited chronic disease, doctors now believe numbers could run into thousands - most of them undiagnosed.
Indeed, some researchers believe thousands of black South Africans may have died from cystic fibrosis complications without the symptoms being recognised.
"Children who died after being diagnosed with malnutrition, lung infections or diarrhoea may well have been displaying the symptoms of cystic fibrosis," says Professor Michele Ramsay, head of the molecular genetics laboratory in the department of human genetics at the SA national health laboratory service.
Cystic fibrosis is caused by a defective gene that causes the body to produce a thick, sticky mucus that clogs the lungs and pancreas. Common results are life-threatening lung infections and an inability to break down and absorb food. Typically, patients suffer from persistent coughing, shortness of breath, poor growth and weight gain despite a healthy appetite, and difficulty in performing bowel movements.
Prof Michele Ramsay - Symptoms unrecognised
Life expectancy among cystic fibrosis sufferers has improved in recent years but it is still a terminal illness. In the 1950s, most children diagnosed with the disease died before they reached primary-school age. As recently as the 1980s, they were lucky to reach their late teens. Today, thanks to improved research and treatment, many cystic fibrosis patients can expect to live into their 30s and 40s.
However, doctors say there is still no cure for cystic fibrosis. International gene therapy research has raised hopes that a cure will be found but Ramsay says success is still some way off. A number of drugs have enjoyed some success but they are generally suitable only for patients with specific mutations of the disease. Some specialists say the best hope for long-term survival is a double-lung transplant. The procedure has enjoyed some success in SA but is very expensive.
Cystic fibrosis is passed on to sufferers from parents, both of whom must carry the defective gene. Carriers are generally unaware of their status. According to specialist cystic fibrosis doctors Professor Mervyn Mer and Cathy Baird, about 4% of white South Africans are carriers, 2% of coloureds and 1% of blacks. Only when two carriers procreate is full-blown cystic fibrosis passed on. There is a one in four chance of their children being infected.
In an article in the SA Respiratory Journal, Mer and Baird write that the presence of cystic fibrosis is far greater than generally recognised. "It translates into an incidence of cystic fibrosis in every 2 800 white children, one in every 10 000 coloured babies and at least one in every 32 000 black babies born in SA." Another study, however, suggests one in 4 624 black babies could suffer from cystic fibrosis.
Even when infected, sufferers do not automatically display standard symptoms. In some patients, the disease becomes evident soon after birth; with others it takes some years. Unnoticed, complications may include diabetes, chronic sinusitis, osteoporosis, abnormal liver function and even male infertility, says Mer.
The problem in SA is that cystic fibrosis is a little-known disease. In countries like the UK, Australia and the US, awareness and corporate sponsorship are huge, raising millions of dollars for research every year. For some multinationals, cystic fibrosis is their flagship charity. International performers like Celine Dion lend their name to awareness of the disease. Fraser, the two-year-old son of British prime minister Gordon Brown, suffers from cystic fibrosis.
In SA, by contrast, "we are severely challenged in critical mass regarding expertise and finances", says Ramsay. "There are so many things to study here in SA that it's difficult to pursue every research direction."
One reason is undoubtedly that it is seen as a white disease. "That idea has been debunked by scientists but the impression persists among many in the public," says Ramsay. "It's a disease that occurs in every shade of our rainbow nation."
Baird says her interaction with doctors and clinics confirms the prevalence of cystic fibrosis in black communities is higher than previously suspected. Mer says: "There are hundreds, possibly thousands, of black people out there unaware they could have cystic fibrosis."
The sooner the disease is identified, the better the sufferer's quality of life may be. "The individual's health and life expectancy is strongly correlated with whether a diagnosis is made, when it is made and the clinical management and treatment following diagnosis," says Ramsay. "For this reason it is vital that an accurate diagnosis is made as early as possible and that appropriate treatment is initiated and sustained."
Excessively salty skin is a tell-tale sign of cystic fibrosis. Traditional folklore in parts of northern Europe tells of mothers licking newborn babies' foreheads to gauge their chances of survival, says Baird - suggesting forms of the disease have been around for centuries. Ramsay says there are even biblical references to the practice.
Baird says the "gold standard" in detecting cystic fibrosis is a simple sweat test, which is inexpensive and non-invasive. The problem, says Ramsay, is that most rural clinics and their staff lack expertise and facilities.
"Developing countries are challenged on several fronts with regard to diagnostic testing," she says. "Populations tend to be spread sparsely over large geographic distances and the health and laboratory infrastructures tend to be poor. The sweat test is often not available. When it is, it is inadequately performed by staff who lack the appropriate education and skills. This affects both sweat collection and the quality of the biochemical testing. To perform DNA analysis requires laboratories with sophisticated equipment and scientists with the knowledge and skill to perform and interpret the tests."
New drugs, improved treatment and intensive physiotherapy all help combat the effects of cystic fibrosis. But for SA sufferers, as in the rest of the world, living with the disease means regular hospital stays, gradually reduced lung function and the awareness that life is likely to be shorter than for most people. In SA, it is also a lonely disease. Lack of public awareness means sufferers get little understanding beyond their immediate families.
Nevertheless, there are many examples of sufferers living lives to the full. Around the world, cystic fibrosis sufferers have excelled in all kinds of endeavours, including business, the arts and even sport. In SA, Paul Dolman, a cystic fibrosis patient with 40% lung capacity, is a regular Comrades Marathon finisher and motivational speaker. Others find different ways to beat the odds, such as climbing Mount Kilimanjaro.
Mer says: "There are some extraordinary people out there living with cystic fibrosis. They recognise that, in the long term, they are unlikely to beat the disease, but at the same time, they are not willing to let it beat them."
Showing posts with label Medical News. Show all posts
Showing posts with label Medical News. Show all posts
Saturday, May 9, 2009
FDA Approves Pancreatic Enzyme Replacement Product for Marketing in United States
Friday, May 08, 2009 - Creon designed to help those with cystic fibrosis, others with exocrine pancreatic insufficiency
Washington, D.C. - infoZine - The U.S. Food and Drug Administration announced that it has approved Creon (pancrelipase), a pancreatic enzyme replacement product designed to help patients with cystic fibrosis and others with exocrine pancreatic insufficiency (EPI) digest and absorb nutrients from foods. Creon is the first FDA-approved delayed-release pancreatic enzyme replacement product to be marketed in the United States as a result of the agency’s unapproved drugs initiative.Creon, which contains a mixture of digestive enzymes extracted from the pancreas of pigs, helps patients lacking the enzymes needed to digest fats, proteins and sugars from food. Creon is approved for use in pediatric and adult patients.The FDA had required the manufacturer of Creon to submit, and the agency has approved, a Risk Evaluation and Mitigation Strategy (REMS), which includes a Medication Guide, to advise patients on risks associated with high doses of Creon, and the theroretical risk of transmission of viral disease from pigs to patients. A rare bowel disorder, called fibrosing colonopathy, can result from a patient’s high-dose use of Creon. While this condition is serious and may require surgery, a patients’ chances of having this condition may be reduced through their adherence to dosing instructions in the labeling. The risks of a rare bowel disorder and viral transmission described in the Medication Guide are considered to be risks related to all porcine (pig)-derived pancreatic enzyme products, including Creon.Instructions for dosing based on weight and age should be followed carefully. Creon may be sprinkled on food. Because Creon is a delayed-release drug, patients should never crush or chew the capsule as this would cause an early release of the enzymes and a reduction in enzyme activity.“The approval of Creon will allow patients to have access to an approved pancreatic enzyme replacement product that meets FDA standards for effectiveness, safety, and manufacturing consistency,” said Donna Griebel, M.D., director, Division of Gastroenterology Products within FDA’s Center for Drug Evaluation and Research (CDER).The FDA’s Office of Compliance and Office of New Drugs within CDER worked with Creon’s manufacturer, Solvay Pharmaceuticals, through the agency’s unapproved drugs initiative to help the company come into compliance with federal laws by obtaining FDA approval. The agency continues to encourage the manufacturers of all other unapproved pancreatic enzyme products (PEPs) to work with the agency to obtain market approval. All PEPs must obtain FDA approval by April 28, 2010, or be removed from the marketplace.“Like other medically necessary drugs, the FDA is exercising its enforcement discretion while it works with the manufacturer toward gaining approval of these drugs,” said Deborah Autor, director, Office of Compliance, CDER. “FDA approval is critical, and the manufacturers of these products have a responsibility to the American public to ensure that patients have drugs that meet our standards of safety, effectiveness, quality, and labeling.”People who are allergic to pork and pork products, suffer from gout or kidney disease, have difficulty swallowing, are pregnant or who plan to become pregnant, or are breastfeeding, should discuss the use of Creon with their health care professional. Common side effects of Creon include flatulence (gassiness), abdominal pain, headache, and dizziness.Creon and other pancreatic enzyme products are made from pancreatic organs of pigs used for food. There is a theoretical risk of contracting a viral infection from pig-derived medicines, but no human illness has been reported.Health care professionals and consumers may report serious adverse events (side effects) or product quality problems with the use of this product to the FDA's MedWatch Adverse Event Reporting program either online, by regular mail, fax or phone. Related linkwww.fda.gov/MedWatch/report.htm
Washington, D.C. - infoZine - The U.S. Food and Drug Administration announced that it has approved Creon (pancrelipase), a pancreatic enzyme replacement product designed to help patients with cystic fibrosis and others with exocrine pancreatic insufficiency (EPI) digest and absorb nutrients from foods. Creon is the first FDA-approved delayed-release pancreatic enzyme replacement product to be marketed in the United States as a result of the agency’s unapproved drugs initiative.Creon, which contains a mixture of digestive enzymes extracted from the pancreas of pigs, helps patients lacking the enzymes needed to digest fats, proteins and sugars from food. Creon is approved for use in pediatric and adult patients.The FDA had required the manufacturer of Creon to submit, and the agency has approved, a Risk Evaluation and Mitigation Strategy (REMS), which includes a Medication Guide, to advise patients on risks associated with high doses of Creon, and the theroretical risk of transmission of viral disease from pigs to patients. A rare bowel disorder, called fibrosing colonopathy, can result from a patient’s high-dose use of Creon. While this condition is serious and may require surgery, a patients’ chances of having this condition may be reduced through their adherence to dosing instructions in the labeling. The risks of a rare bowel disorder and viral transmission described in the Medication Guide are considered to be risks related to all porcine (pig)-derived pancreatic enzyme products, including Creon.Instructions for dosing based on weight and age should be followed carefully. Creon may be sprinkled on food. Because Creon is a delayed-release drug, patients should never crush or chew the capsule as this would cause an early release of the enzymes and a reduction in enzyme activity.“The approval of Creon will allow patients to have access to an approved pancreatic enzyme replacement product that meets FDA standards for effectiveness, safety, and manufacturing consistency,” said Donna Griebel, M.D., director, Division of Gastroenterology Products within FDA’s Center for Drug Evaluation and Research (CDER).The FDA’s Office of Compliance and Office of New Drugs within CDER worked with Creon’s manufacturer, Solvay Pharmaceuticals, through the agency’s unapproved drugs initiative to help the company come into compliance with federal laws by obtaining FDA approval. The agency continues to encourage the manufacturers of all other unapproved pancreatic enzyme products (PEPs) to work with the agency to obtain market approval. All PEPs must obtain FDA approval by April 28, 2010, or be removed from the marketplace.“Like other medically necessary drugs, the FDA is exercising its enforcement discretion while it works with the manufacturer toward gaining approval of these drugs,” said Deborah Autor, director, Office of Compliance, CDER. “FDA approval is critical, and the manufacturers of these products have a responsibility to the American public to ensure that patients have drugs that meet our standards of safety, effectiveness, quality, and labeling.”People who are allergic to pork and pork products, suffer from gout or kidney disease, have difficulty swallowing, are pregnant or who plan to become pregnant, or are breastfeeding, should discuss the use of Creon with their health care professional. Common side effects of Creon include flatulence (gassiness), abdominal pain, headache, and dizziness.Creon and other pancreatic enzyme products are made from pancreatic organs of pigs used for food. There is a theoretical risk of contracting a viral infection from pig-derived medicines, but no human illness has been reported.Health care professionals and consumers may report serious adverse events (side effects) or product quality problems with the use of this product to the FDA's MedWatch Adverse Event Reporting program either online, by regular mail, fax or phone. Related linkwww.fda.gov/MedWatch/report.htm
Sunday, November 30, 2008
Cystic fibrosis: FAQs on the disease of '65 roses'
Cystic fibrosis: FAQs on the disease of '65 roses'
Last Updated: Thursday, November 27, 2008 |CBC News
Roses are a symbol for cystic fibrosis. Cystic fibrosis, or CF, is sometimes called 65 roses. The name came about after a boy overheard his mother talking about the condition on the phone and thought she said "65 roses." A single rose is often used as a symbol for the disease.
A child with cystic fibrosis may cough frequently and need to have a parent clap his or her back every morning to make it easier to breathe.
Cystic fibrosis is an inherited disease of the mucus and sweat glands. Unlike in healthy lungs, where mucus protects the airways, people with CF have mucus that becomes thick and sticky. When the substance builds up in the lungs, bacterial infections may occur.
Mucous-producing cells in the digestive system — including the pancreas, liver and intestines — and respiratory system are the major problem areas for people with CF.
What causes CF?
Jake Szatmary was sick and malnourished for the first three months of his life, before cystic fibrosis was diagnosed. (CBC)People with CF inherit two copies of an abnormal gene, one from each parent. Those with one copy are carriers who don't have the disease or show any symptoms but may pass it on to their children. About one in 25 Canadians carries a defective version of the gene responsible for CF, according to the Canadian Cystic Fibrosis Foundation.
Canadian researchers discovered the gene responsible for CF, called cystic fibrosis transmembrane conductance regulator, or CFTR, in 1989. More than 1,000 mutations of the gene have been associated with the disease.
Who is affected?
An estimated one in every 3,600 children born in Canada has CF. The severity of the disease varies, and both sexes are affected with a similar frequency.
The risk of being a carrier varies by ethnicity, according to HealthyOntario.com:
Ethnicity Carrier risk
Caucasian 1 in 25
Ashkenazi Jew 1 in 25
Hispanic 1 in 46
Black 1 in 65
The disease mainly affects Caucasians, which includes Europeans, Indians and people from the Middle East, said Nadine Imbleau Redman, a spokeswoman for the Canadian Cystic Fibrosis Foundation.
How is it diagnosed?
Doctors may suspect CF in babies who cough a lot and get many lung infections. A simple and painless collection of sweat is done, since people with CF have extremely salty sweat. Chest X-rays, sinus X-rays, lung function tests, analysis of sputum and stool and genetic tests on blood may also be done to help in the diagnosis. Prenatal genetic tests show if a fetus is likely to have CF.
What are the common symptoms?
Frequent coughing with phlegm.
Frequent infections such as bronchitis and pneumonia.
Salty-tasting skin.
Dehydration.
Excessive appetite with weight loss.
Bulky bowel movements.
Poor growth.
Infertility, mostly in men.
How is it treated?
The goal is to keep the lungs clear of mucus to avoid infection. Physical therapy, such as clapping on the chest and back, breathing exercises, wearing a positive pressure expiratory mask and drinking a lot of fluids helps to loosen mucus.
Pancreatic enzymes are taken with meals to aid digestion since mucus in the pancreas makes it difficult to digest food and absorb nutrients. A vitamin and mineral supplement may be needed for the same reason. Drugs that thin the mucus, reduce inflammation in the airways, and antibiotics, taken in pill, intravenous or inhaled form, may be prescribed, according to the U.S. National Heart Lung and Blood Institute.
Eating a healthy diet, avoiding tobacco smoke, exercising frequently, and frequent hand washing are also important.
How is daily life affected?
In 2006, for the first time, more than half of all Canadians with CF were 18 years or older, the Canadian Cystic Fibrosis Foundation said. Half of Canadians with CF are now expected to live into their late 30s or longer.
When lung disease limits daily life, then lung transplants may be an option.
In 2004, a 21-year-old woman with CF received a rare lung transplant from two living donors, her mother and aunt. Such transplants are complex since both donors and the recipient are at risk.
"The CF had clogged up my lungs," Jessie McQuitty of Edmonton said at the time. "I was unable to breathe, I was unable to work, unable to school, everything."
In 2008, Canadian researchers discovered a genetic variant that can increase the severity of lung deterioration in children with CF.
These patients "experience a vicious cycle of infection and inflammation that destroys lung tissue, inhibits lung function, and erodes quality of life," said Dr. Julian Zielenski, an associate scientist in the genetics and genome biology program at the Hospital for Sick Children and a lead author of the study.
People who have the variant produce less of a protein that defends against bacterial infections. The researchers hope to one day develop a genetic test that can predict the severity of a patient's lung disease.
Wednesday, November 12, 2008
New Treatment For Patients With Cystic Fibrosis
New Treatment For Patients With Cystic Fibrosis
Kellye Lynn BALTIMORE (WJZ) ― There's a change in treatment for patients with cystic fibrosis.
Healthwatch reporter Kellye Lynn says many children with CF are not getting enough of an important vitamin.
That vitamin is Vitamin D, which is essential for bone health.
Last summer, Justin Homassel, 10, leaped off his porch backwards and broke his arm.
"I jumped and caught myself with my arm and I broke it," he said.
Broken bones are more common among children like Justin who have cystic fibrosis, a genetic disorder that causes sticky mucous to build up in the body and affects about 30,000 Americans.
"The pancreas doesn't make enzymes to digest food appropriately, especially fat so they don't absorb fat, and therefore fat soluble vitamins, the way they should," said Dr. Peter Mogayzel, Johns Hopkins Children's Center.
The result is low levels of Vitamin D, which can increase the risk of rickets and osteoporosis.
A new study out of Johns Hopkins Children's Center shows current recommendations for treating Vitamin D deficiency don't get far enough.
In the study, giving the standard 50,000 international units of Vitamin D for eight weeks was effective in only 33% of patients. It's a surprising finding and prompted Hopkins doctors to send a letter to each of their CF patients, urging an increase of Vitamin D.
The new recommendation is an extra 1,000 international units of Vitamin D a day.
"I think it's important for patients to take more Vitamin D and we believe the CF Foundation has to reevaluate this to have more appropriate recommendations for therapy," said Dr. Mogayzel.
Justin is now following that advice. His mom hopes doing so will keep looking as good as the rest of his body.
CF patients can also increase Vitamin D levels by spending 15 minutes a day in the sun without sunscreen. Doctors also recommend weight-bearing exercise.
(© MMVIII, CBS Broadcasting Inc. All Rights Reserved.)
Kellye Lynn BALTIMORE (WJZ) ― There's a change in treatment for patients with cystic fibrosis.
Healthwatch reporter Kellye Lynn says many children with CF are not getting enough of an important vitamin.
That vitamin is Vitamin D, which is essential for bone health.
Last summer, Justin Homassel, 10, leaped off his porch backwards and broke his arm.
"I jumped and caught myself with my arm and I broke it," he said.
Broken bones are more common among children like Justin who have cystic fibrosis, a genetic disorder that causes sticky mucous to build up in the body and affects about 30,000 Americans.
"The pancreas doesn't make enzymes to digest food appropriately, especially fat so they don't absorb fat, and therefore fat soluble vitamins, the way they should," said Dr. Peter Mogayzel, Johns Hopkins Children's Center.
The result is low levels of Vitamin D, which can increase the risk of rickets and osteoporosis.
A new study out of Johns Hopkins Children's Center shows current recommendations for treating Vitamin D deficiency don't get far enough.
In the study, giving the standard 50,000 international units of Vitamin D for eight weeks was effective in only 33% of patients. It's a surprising finding and prompted Hopkins doctors to send a letter to each of their CF patients, urging an increase of Vitamin D.
The new recommendation is an extra 1,000 international units of Vitamin D a day.
"I think it's important for patients to take more Vitamin D and we believe the CF Foundation has to reevaluate this to have more appropriate recommendations for therapy," said Dr. Mogayzel.
Justin is now following that advice. His mom hopes doing so will keep looking as good as the rest of his body.
CF patients can also increase Vitamin D levels by spending 15 minutes a day in the sun without sunscreen. Doctors also recommend weight-bearing exercise.
(© MMVIII, CBS Broadcasting Inc. All Rights Reserved.)
Saturday, November 1, 2008
Low vitamin D common in kids with cystic fibrosis
By Megan Rauscher– Fri Oct 31, 9:17 am ETNEW YORK (Reuters Health) – Vitamin D deficiency is prevalent in children with cystic fibrosis (CF) and current treatment recommendations for correcting the deficiency are "inadequate," conclude clinicians from The Johns Hopkins Medical Institutions in Baltimore, Maryland.
They reviewed the charts of 262 children with CF patients treated at Johns Hopkins between 2003 and 2006 and found that vitamin D deficiency has declined but is still high; the prevalence was 86 percent in 2003 and 46 percent in 2006.
Many vitamin D deficient children remained deficient, despite getting "restorative" doses of vitamin D equal to or higher than the recommendations set by the Cystic Fibrosis Foundation, Dr. Deanna Green and colleagues report in the Journal of Pediatrics.
CF is a genetic disorder marked by the body's inability to transport chloride in and out of cells, causing mild to life-threatening complications, including recurrent and severe lung infections and delayed growth. Growing children with CF are especially vulnerable to vitamin D deficiency because a hallmark of the disorder is poor absorption of nutrients and malnutrition.
The recommended treatment dose for vitamin D deficiency is 50,000 international units (IU) of ergocalciferol (a form of vitamin D) once per week for 8 weeks for children 5 years of age or older and 12,000 IU once per week for 8 weeks for children younger than age 5 years.
But the Hopkins team that 50,000 IU per week of ergocalciferol for 8 weeks was effective in only 33 percent of the children with vitamin D deficiency, while increasing the dose to twice a week was effective in only 26 percent of children. Delivering the same dose 3 times a week corrected the deficiency in just 43 percent of children.
As a result of the findings, Johns Hopkins has amended its treatment protocol and now treats both adult and pediatric CF patients who have vitamin D deficiency with 50,000 IU ergocalciferol everyday for 4 weeks, according to a university-issued statement.
"These findings are a big wake-up call not only because they show that many children with CF are lacking vitamin D, but also because the deficiency persists even in those children who are treated with weekly doses twice or 3 times as high as the current recommendations," Green said.
"Clearly there is an urgent need to find more effective ways to restore healthy vitamin D levels," she concluded.
SOURCE: The Journal of Pediatrics, October 2008.
They reviewed the charts of 262 children with CF patients treated at Johns Hopkins between 2003 and 2006 and found that vitamin D deficiency has declined but is still high; the prevalence was 86 percent in 2003 and 46 percent in 2006.
Many vitamin D deficient children remained deficient, despite getting "restorative" doses of vitamin D equal to or higher than the recommendations set by the Cystic Fibrosis Foundation, Dr. Deanna Green and colleagues report in the Journal of Pediatrics.
CF is a genetic disorder marked by the body's inability to transport chloride in and out of cells, causing mild to life-threatening complications, including recurrent and severe lung infections and delayed growth. Growing children with CF are especially vulnerable to vitamin D deficiency because a hallmark of the disorder is poor absorption of nutrients and malnutrition.
The recommended treatment dose for vitamin D deficiency is 50,000 international units (IU) of ergocalciferol (a form of vitamin D) once per week for 8 weeks for children 5 years of age or older and 12,000 IU once per week for 8 weeks for children younger than age 5 years.
But the Hopkins team that 50,000 IU per week of ergocalciferol for 8 weeks was effective in only 33 percent of the children with vitamin D deficiency, while increasing the dose to twice a week was effective in only 26 percent of children. Delivering the same dose 3 times a week corrected the deficiency in just 43 percent of children.
As a result of the findings, Johns Hopkins has amended its treatment protocol and now treats both adult and pediatric CF patients who have vitamin D deficiency with 50,000 IU ergocalciferol everyday for 4 weeks, according to a university-issued statement.
"These findings are a big wake-up call not only because they show that many children with CF are lacking vitamin D, but also because the deficiency persists even in those children who are treated with weekly doses twice or 3 times as high as the current recommendations," Green said.
"Clearly there is an urgent need to find more effective ways to restore healthy vitamin D levels," she concluded.
SOURCE: The Journal of Pediatrics, October 2008.
Wednesday, October 29, 2008
New Test Promises Quicker, More Accurate Evaluation For Cystic Fibrosis Patients
New Test Promises Quicker, More Accurate Evaluation For Cystic Fibrosis Patients
ScienceDaily (Oct. 28, 2008) — Researchers at National Jewish Health have identified a simple gene-based blood test that more accurately and quickly measures cystic fibrosis patients’ response to therapy than current tests. The test, a measure of inflammatory gene expression, could improve patient care and help clear a backlog of promising medications now hung up in clinical trials.
The researchers are publishing the results of a small “proof-of-principal” trial in the November 1, 2008, issue of American Journal of Respiratory and Critical Care Medicine.
“The currently accepted test, a measure of a patients’ ability to exhale air, has several limitations that make it ineffective for some patients and not sensitive enough for clinical trials of many new medications,” said Dr. Milene Saavedra, lead author of the study and Assistant Professor of Medicine at National Jewish Health. “By measuring the activity of genes associated with the immune/inflammatory response, we can get a more accurate picture of the biological processes occurring inside the lungs.”
Cystic fibrosis is the most common lethal inherited disease in the western world, with about 30,000 patients in the US . Most patients die of respiratory failure generally in their 30s or 40s. Lung damage is caused primarily by chronic bacterial infections and the resulting severe airway inflammation. There is a critical need for new effective anti-microbial and anti-inflammatory medications to slow and/or prevent lung damage in young patients. Several promising therapies have gone through early stages of clinical testing but their progress is being hampered by the lack of a sensitive measure of therapeutic response to medications.
Currently, response to medication is measured by how much air a person can rapidly exhale: forced expiratory volume in one second or FEV1. FEV1 cannot be performed effectively for all patients, especially young and very sick patients. Generally patients’ FEV1 does improve when inflammation is reduced, but not all patients’ FEV1 improves significantly, and changes can take weeks to months to show up. It can be prohibitively expensive to conduct phase 2 or phase 3 trials of medications long enough to detect the changes in FEV1.
National Jewish researchers thought white blood cells circulating in the blood might be a good source of biomarkers for a more sensitive and accurate test. White blood cells are the predominant cell type at sites of tissue destruction in CF patients’ lungs. As they circulate through the lungs in the blood they encounter the inflammatory environment and alter their gene expression as a result. By measuring mRNA, scientists can identify which genes are being expressed and how strongly.
The researchers evaluated 18 CF patients who were suffering severe exacerbations of their disease. They withdrew blood before and after two weeks of intravenous antibiotic therapy. The severe exacerbation and antibiotic therapy served as a condensed model of illness and response to therapy; in these cases antibiotic therapy is usually successful, and patients’ clinical symptoms and FEV1 both improve rapidly.
Using microarray gene analysis of the blood samples, the researchers identified 10 genes that differed significantly in their expression before and after therapy. Using real-time polymerase chain reaction and additional statistical tools, the researchers identified three of those genes that most accurately correlated with a positive therapeutic response: CD36, CD64 and ADAM9. CD36 and CD64 are genes associated with cells’ absorption of foreign organisms and cellular debris. ADAM9 is associated with tissue destruction that allows inflammatory cells to move through tissue. This process is also believed to contribute to permanent tissue destruction.
“The expression of these genes correlated with FEV1, other inflammatory markers, and various clinical factors,” said co-author Dr. Jerry Nick , Associate Professor of Medicine at National Jewish. “When combined with FEV1, they offered a more accurate and sensitive measure of response to therapy than either alone. We believe they could be extremely useful in clinical care of patients and trials of new CF therapies.”
The researchers are now conducting a trial of 60 CF patients to provide stronger statistical evidence for the power of CD36, CD64 and ADAM9 to diagnose a positive response to therapy by CF patients.
Adapted from materials provided by National Jewish Medical and Research Center.
ScienceDaily (Oct. 28, 2008) — Researchers at National Jewish Health have identified a simple gene-based blood test that more accurately and quickly measures cystic fibrosis patients’ response to therapy than current tests. The test, a measure of inflammatory gene expression, could improve patient care and help clear a backlog of promising medications now hung up in clinical trials.
The researchers are publishing the results of a small “proof-of-principal” trial in the November 1, 2008, issue of American Journal of Respiratory and Critical Care Medicine.
“The currently accepted test, a measure of a patients’ ability to exhale air, has several limitations that make it ineffective for some patients and not sensitive enough for clinical trials of many new medications,” said Dr. Milene Saavedra, lead author of the study and Assistant Professor of Medicine at National Jewish Health. “By measuring the activity of genes associated with the immune/inflammatory response, we can get a more accurate picture of the biological processes occurring inside the lungs.”
Cystic fibrosis is the most common lethal inherited disease in the western world, with about 30,000 patients in the US . Most patients die of respiratory failure generally in their 30s or 40s. Lung damage is caused primarily by chronic bacterial infections and the resulting severe airway inflammation. There is a critical need for new effective anti-microbial and anti-inflammatory medications to slow and/or prevent lung damage in young patients. Several promising therapies have gone through early stages of clinical testing but their progress is being hampered by the lack of a sensitive measure of therapeutic response to medications.
Currently, response to medication is measured by how much air a person can rapidly exhale: forced expiratory volume in one second or FEV1. FEV1 cannot be performed effectively for all patients, especially young and very sick patients. Generally patients’ FEV1 does improve when inflammation is reduced, but not all patients’ FEV1 improves significantly, and changes can take weeks to months to show up. It can be prohibitively expensive to conduct phase 2 or phase 3 trials of medications long enough to detect the changes in FEV1.
National Jewish researchers thought white blood cells circulating in the blood might be a good source of biomarkers for a more sensitive and accurate test. White blood cells are the predominant cell type at sites of tissue destruction in CF patients’ lungs. As they circulate through the lungs in the blood they encounter the inflammatory environment and alter their gene expression as a result. By measuring mRNA, scientists can identify which genes are being expressed and how strongly.
The researchers evaluated 18 CF patients who were suffering severe exacerbations of their disease. They withdrew blood before and after two weeks of intravenous antibiotic therapy. The severe exacerbation and antibiotic therapy served as a condensed model of illness and response to therapy; in these cases antibiotic therapy is usually successful, and patients’ clinical symptoms and FEV1 both improve rapidly.
Using microarray gene analysis of the blood samples, the researchers identified 10 genes that differed significantly in their expression before and after therapy. Using real-time polymerase chain reaction and additional statistical tools, the researchers identified three of those genes that most accurately correlated with a positive therapeutic response: CD36, CD64 and ADAM9. CD36 and CD64 are genes associated with cells’ absorption of foreign organisms and cellular debris. ADAM9 is associated with tissue destruction that allows inflammatory cells to move through tissue. This process is also believed to contribute to permanent tissue destruction.
“The expression of these genes correlated with FEV1, other inflammatory markers, and various clinical factors,” said co-author Dr. Jerry Nick , Associate Professor of Medicine at National Jewish. “When combined with FEV1, they offered a more accurate and sensitive measure of response to therapy than either alone. We believe they could be extremely useful in clinical care of patients and trials of new CF therapies.”
The researchers are now conducting a trial of 60 CF patients to provide stronger statistical evidence for the power of CD36, CD64 and ADAM9 to diagnose a positive response to therapy by CF patients.
Adapted from materials provided by National Jewish Medical and Research Center.
Carrier Screening For CF In US Genetic Testing Laboratories:
Carrier Screening For CF In US Genetic Testing Laboratories: A Survey Of Laboratory Directors
Main Category: Cystic Fibrosis
Also Included In: Genetics
Article Date: 28 Oct 2008 - 3:00 PDT
Market pressures may have more influence on clinical practice than laboratory guidelines: That's one conclusion of a Center survey, which found that guidelines for cystic fibrosis (CF) carrier screening have only been partially adopted. David Kaufman, Sara Katsanis, Gail Javitt, Juli Murphy, and Kathy Hudson report in the October issue of Clinical Genetics that seven percent of responding genetic testing laboratories adopted two major screening guidelines issued by the American College of Medical Genetics and the American College of Obstetricians and Gynecologists, and that 13 percent have adopted neither recommendation. These findings indicate that factors other than clinical guidelines may influence laboratories' CF screening practices.
CF is an incurable, progressively disabling, and fatal disease of the sweat and mucus glands, which develops in people who have inherited two mutated copies of the CFTR gene. One in 31 Americans is a carrier of a mutated CFTR gene, making CF one of the most common major genetic disorders. In 1997, a panel convened by the National Institutes of Health concluded that genetic testing for CF should be offered to couples currently planning a pregnancy as well as couples seeking prenatal testing. Because more than 1,300 disease-causing mutations have been identified in CFTR, and different laboratories can test for different mutations, in 2001, the American College of Medical Genetics and the American College of Obstetricians and Gynocologists issued sets of CF testing guidelines (revised in 2004) to improve and standardize population-based CF carrier screening. The study conducted by GPPC focused on two primary components of these guidelines. First was the recommendation that prospective parents be tested for the a CF mutation known as the 5T allele only as a "reflex" after another CF mutation, called R117H, has been identified first. This is because although the 5T allele is relatively common, it can cause CF only when found in combination with R117H. Because the 5T allele is found in between five and nine percent of the general population, screening all pregnant couples for the 5T allele instead of testing only those with an R117H mutation can identify and alarm a large number of individuals who are not at risk of having a child with CF, and provoke unnecessary invasive prenatal testing. The second major recommendation made to laboratories was that they should only screen a panel of 23 of the most relevant, well-studied mutations, which reliably identifies the majority of couples at risk of having a child with CF but remains cost-effective.
The Center survey sought to examine how guidelines for genetic testing for CF have influenced the practice of laboratories that perform the tests - a "necessary first step," write the authors, "in understanding the effectiveness of developing such guidelines, the realities and barriers that may limit uptake, and whether adoption of guidelines (or the lack thereof) has any serious consequences for genetic screening programs." If research shows that the adoption of genetic testing guidelines improves the health outcomes of people getting the test, then finding ways to overcome barriers to their adoption will be critical.
Of 190 responding genetic testing laboratories (a 55 percent response rate), 45% offered CF screening to potential parents for the presence of a mutated CFTR gene. The survey assessed these laboratories' adoption of two primary components of the CF carrier screening guidelines: the reflexive reporting of 5T test results, and the recommended 23-mutation screening panel. While four of five CF screening laboratories followed the 5T guidelines, 90% of laboratories tested more than 23 mutations (some tested up to 150 mutations). Just seven percent of laboratories adopted both guidelines, and 13 percent of respondents adopted neither the 5T guidelines nor the 23-mutation panel.
These results suggest that laboratories' screening practices are affected by factors other than clinical guidelines - possibly including market pressures to purchase CF testing kits that test for more than 23 mutations, despite the lack of clarity about the utility of such testing in the general population. Laboratories' choice of which CF testing kits to use also may be limited by the testing platforms used in a particular lab. The authors note that their findings carry implications beyond CF, and may impact the development of recommendations for other types of genetic testing: "As new guidelines for other population-based genetic tests emerge, it will be useful and important to understand the extent of their influence on practice."
Written by Sara Brinda
Kaufman, D.J., S.H. Katsanis, G.H. Javitt, J.A. Murphy, J.A. Scott, and K.L. Hudson. Carrier screening for cystic fibrosis in US genetic testing laboratories: a survey of laboratory directors. Clinical Genetics 74: 367-373.
The Genetics and Public Policy Center at Johns Hopkins University
The tremendous success of the Human Genome Project has laid the foundation for a true revolution in public health, promising improved diagnosis, more effective medicines, and individually tailored health care. The Genetics and Public Policy Center was created in 2002 at Johns Hopkins University by Pew Charitable Trusts to help policymakers, the press, and the public understand and respond to the challenges and opportunities of genetic medicine and its potential to transform global public health.
http://www.dnapolicy.org
Main Category: Cystic Fibrosis
Also Included In: Genetics
Article Date: 28 Oct 2008 - 3:00 PDT
Market pressures may have more influence on clinical practice than laboratory guidelines: That's one conclusion of a Center survey, which found that guidelines for cystic fibrosis (CF) carrier screening have only been partially adopted. David Kaufman, Sara Katsanis, Gail Javitt, Juli Murphy, and Kathy Hudson report in the October issue of Clinical Genetics that seven percent of responding genetic testing laboratories adopted two major screening guidelines issued by the American College of Medical Genetics and the American College of Obstetricians and Gynecologists, and that 13 percent have adopted neither recommendation. These findings indicate that factors other than clinical guidelines may influence laboratories' CF screening practices.
CF is an incurable, progressively disabling, and fatal disease of the sweat and mucus glands, which develops in people who have inherited two mutated copies of the CFTR gene. One in 31 Americans is a carrier of a mutated CFTR gene, making CF one of the most common major genetic disorders. In 1997, a panel convened by the National Institutes of Health concluded that genetic testing for CF should be offered to couples currently planning a pregnancy as well as couples seeking prenatal testing. Because more than 1,300 disease-causing mutations have been identified in CFTR, and different laboratories can test for different mutations, in 2001, the American College of Medical Genetics and the American College of Obstetricians and Gynocologists issued sets of CF testing guidelines (revised in 2004) to improve and standardize population-based CF carrier screening. The study conducted by GPPC focused on two primary components of these guidelines. First was the recommendation that prospective parents be tested for the a CF mutation known as the 5T allele only as a "reflex" after another CF mutation, called R117H, has been identified first. This is because although the 5T allele is relatively common, it can cause CF only when found in combination with R117H. Because the 5T allele is found in between five and nine percent of the general population, screening all pregnant couples for the 5T allele instead of testing only those with an R117H mutation can identify and alarm a large number of individuals who are not at risk of having a child with CF, and provoke unnecessary invasive prenatal testing. The second major recommendation made to laboratories was that they should only screen a panel of 23 of the most relevant, well-studied mutations, which reliably identifies the majority of couples at risk of having a child with CF but remains cost-effective.
The Center survey sought to examine how guidelines for genetic testing for CF have influenced the practice of laboratories that perform the tests - a "necessary first step," write the authors, "in understanding the effectiveness of developing such guidelines, the realities and barriers that may limit uptake, and whether adoption of guidelines (or the lack thereof) has any serious consequences for genetic screening programs." If research shows that the adoption of genetic testing guidelines improves the health outcomes of people getting the test, then finding ways to overcome barriers to their adoption will be critical.
Of 190 responding genetic testing laboratories (a 55 percent response rate), 45% offered CF screening to potential parents for the presence of a mutated CFTR gene. The survey assessed these laboratories' adoption of two primary components of the CF carrier screening guidelines: the reflexive reporting of 5T test results, and the recommended 23-mutation screening panel. While four of five CF screening laboratories followed the 5T guidelines, 90% of laboratories tested more than 23 mutations (some tested up to 150 mutations). Just seven percent of laboratories adopted both guidelines, and 13 percent of respondents adopted neither the 5T guidelines nor the 23-mutation panel.
These results suggest that laboratories' screening practices are affected by factors other than clinical guidelines - possibly including market pressures to purchase CF testing kits that test for more than 23 mutations, despite the lack of clarity about the utility of such testing in the general population. Laboratories' choice of which CF testing kits to use also may be limited by the testing platforms used in a particular lab. The authors note that their findings carry implications beyond CF, and may impact the development of recommendations for other types of genetic testing: "As new guidelines for other population-based genetic tests emerge, it will be useful and important to understand the extent of their influence on practice."
Written by Sara Brinda
Kaufman, D.J., S.H. Katsanis, G.H. Javitt, J.A. Murphy, J.A. Scott, and K.L. Hudson. Carrier screening for cystic fibrosis in US genetic testing laboratories: a survey of laboratory directors. Clinical Genetics 74: 367-373.
The Genetics and Public Policy Center at Johns Hopkins University
The tremendous success of the Human Genome Project has laid the foundation for a true revolution in public health, promising improved diagnosis, more effective medicines, and individually tailored health care. The Genetics and Public Policy Center was created in 2002 at Johns Hopkins University by Pew Charitable Trusts to help policymakers, the press, and the public understand and respond to the challenges and opportunities of genetic medicine and its potential to transform global public health.
http://www.dnapolicy.org
Wednesday, October 15, 2008
Vitamin D Guidelines Too Low for Cystic Fibrosis Kids
Vitamin D Guidelines Too Low for Cystic Fibrosis Kids
MONDAY, Oct. 13 (HealthDay News) -- Existing guidelines for treating vitamin D deficiency in children with cystic fibrosis are too low and put patients at high risk for bone loss and rickets, a new study says.
Researchers from Johns Hopkins Children's Center looked at 262 children with cystic fibrosis (CF). Nearly half the children were vitamin D-deficient, and most of them remained persistently deficient, despite receiving restorative vitamin D doses equal to or higher than the Cystic Fibrosis Foundation-recommended 50,000 IU of ergocalciferol (a form of vitamin D) per week.
The study was published in the October issue of The Journal of Pediatrics.
CF is a genetic disorder in which the body is unable to transport chloride in and out of cells. This causes mild to life-threatening complications such as recurrent and severe lung infections and delayed growth. Because CF results in poor absorption of nutrients and malnutrition, growing children with CF are especially prone to vitamin D deficiency, according to background information in a Hopkins news release.
"These findings are a big wake-up call, not only because they show that many children with CF are lacking vitamin D, but also because the deficiency persists even in those children who are treated with weekly doses twice or three times as high as the current recommendations," study leader and lung specialist Dr. Deanna Green said in the news release.
"Clearly there is an urgent need to find more effective ways to restore healthy vitamin D levels," she added.
Until that happens, doctors should consider increasing vitamin D intake beyond the current recommendations in CF patients who are vitamin D deficient, Green and colleagues suggested. Doctors should also check vitamin D levels at least once a year in all CF patients and more frequently in patients with abnormally low vitamin D levels.
As a result of the findings, Hopkins amended its treatment protocol and now treats adult and child CF patients who have vitamin D deficiency with 50,000 IU of vitamin D daily for four weeks.
MONDAY, Oct. 13 (HealthDay News) -- Existing guidelines for treating vitamin D deficiency in children with cystic fibrosis are too low and put patients at high risk for bone loss and rickets, a new study says.
Researchers from Johns Hopkins Children's Center looked at 262 children with cystic fibrosis (CF). Nearly half the children were vitamin D-deficient, and most of them remained persistently deficient, despite receiving restorative vitamin D doses equal to or higher than the Cystic Fibrosis Foundation-recommended 50,000 IU of ergocalciferol (a form of vitamin D) per week.
The study was published in the October issue of The Journal of Pediatrics.
CF is a genetic disorder in which the body is unable to transport chloride in and out of cells. This causes mild to life-threatening complications such as recurrent and severe lung infections and delayed growth. Because CF results in poor absorption of nutrients and malnutrition, growing children with CF are especially prone to vitamin D deficiency, according to background information in a Hopkins news release.
"These findings are a big wake-up call, not only because they show that many children with CF are lacking vitamin D, but also because the deficiency persists even in those children who are treated with weekly doses twice or three times as high as the current recommendations," study leader and lung specialist Dr. Deanna Green said in the news release.
"Clearly there is an urgent need to find more effective ways to restore healthy vitamin D levels," she added.
Until that happens, doctors should consider increasing vitamin D intake beyond the current recommendations in CF patients who are vitamin D deficient, Green and colleagues suggested. Doctors should also check vitamin D levels at least once a year in all CF patients and more frequently in patients with abnormally low vitamin D levels.
As a result of the findings, Hopkins amended its treatment protocol and now treats adult and child CF patients who have vitamin D deficiency with 50,000 IU of vitamin D daily for four weeks.
Nasal AAT achieved significant reduction in inflammation in the lungs.
Kamada reports positive results in cystic fibrosis study
Nasal AAT achieved significant reduction in inflammation in the lungs.
Gali Weinreb15 Oct 08 15:36
Kamada Ltd. (TASE: KMDA) reports positive results of its Phase II clinical trial of its Alpha-1 Antitrypsin (AAT) protein for the treatment of cystic fibrosis. The company said that AAT achieved significant reduction in inflammation in the lungs, compared with the placebo. Lung inflammation is the main symptom of the disease and the main cause of patients' deterioration.
On the basis of the trial results, Kamada will now be able to begin Phase III clinical trials with the US Food and Drug Administration (FDA). The Phase II trial was conducted with the European Medicines Agency (EMEA).
Nasal AAT achieved significant reduction in inflammation in the lungs.
Gali Weinreb15 Oct 08 15:36
Kamada Ltd. (TASE: KMDA) reports positive results of its Phase II clinical trial of its Alpha-1 Antitrypsin (AAT) protein for the treatment of cystic fibrosis. The company said that AAT achieved significant reduction in inflammation in the lungs, compared with the placebo. Lung inflammation is the main symptom of the disease and the main cause of patients' deterioration.
On the basis of the trial results, Kamada will now be able to begin Phase III clinical trials with the US Food and Drug Administration (FDA). The Phase II trial was conducted with the European Medicines Agency (EMEA).
Tuesday, October 7, 2008
NO CURE for CF
Cystic Fibrosis affects more people than Down syndrome, muscular dystrophy, and sickle cell anemia combined and the worst part is there is no cure it.
Friday, September 26, 2008
Scientists use pigs to try to beat cystic fibrosis
Scientists use pigs to try to beat cystic fibrosis
By Will Dunham Thu Sep 25, 2:45 PM ET
WASHINGTON (Reuters) - U.S. scientists have created pigs that appear to develop cystic fibrosis just as people do, a step they hope will accelerate efforts to tackle the disease.
Writing in the journal Science on Thursday, scientists at the University of Iowa and the University of Missouri said they created genetically engineered piglets with the same mutation that causes cystic fibrosis in people.
Studying how the disease unfolds in these pigs may provide insights into cystic fibrosis that thus far have eluded scientists and could point toward new treatments or maybe even a cure, said Dr. Michael Welsh of the University of Iowa.
People get CF when they inherit two mutated copies of a gene called CFTR, which was pinpointed as the cause in 1989. The disease causes mucus to accumulate and clog some of the organs in the body, especially the lungs and pancreas.
The Cystic Fibrosis Foundation, which helped fund the study, said about 70,000 people worldwide, including 30,000 people in the United States, have CF. People with the disease can expect to live to about age 37.
Scientists create animal "models" of a disease in order to perform experiments that would not be possible with people. Mice with the genetic abnormality that causes cystic fibrosis have been developed, but the disease presents itself very differently in these rodents than in people.
"Unfortunately, the mice leave something to be desired," Welsh, who helped lead the study, said in a telephone interview.
"They don't get the pancreatic disease like people with CF get. They don't get the lung disease like people with CF get. They don't get the intestinal disease like people with CF get. There's been many questions that can't be answered," he added.
"The onset of these problems is difficult to track down in humans because sometimes they happen before birth or sometimes they happen silently," added Christopher Penland of the foundation.
This is the latest example of pigs used for human medical needs. For example, cardiac patients can get pig heart valves.
The scientists developed pigs with cystic fibrosis because their lungs have many characteristics of human lungs.
"Right now, if you want to do experiments to find treatments or therapies for the lung disease that is fatal for people with CF, you would have to experiment on kids that have CF," Randy Prather of the University of Missouri added.
The disease in pigs closely mimicked the initial stages of the disease seen in people. The scientists are waiting for them to develop lung disease typical of CF "so we can start experimenting in ways that have never been possible," Prather said in a statement.
By Will Dunham Thu Sep 25, 2:45 PM ET
WASHINGTON (Reuters) - U.S. scientists have created pigs that appear to develop cystic fibrosis just as people do, a step they hope will accelerate efforts to tackle the disease.
Writing in the journal Science on Thursday, scientists at the University of Iowa and the University of Missouri said they created genetically engineered piglets with the same mutation that causes cystic fibrosis in people.
Studying how the disease unfolds in these pigs may provide insights into cystic fibrosis that thus far have eluded scientists and could point toward new treatments or maybe even a cure, said Dr. Michael Welsh of the University of Iowa.
People get CF when they inherit two mutated copies of a gene called CFTR, which was pinpointed as the cause in 1989. The disease causes mucus to accumulate and clog some of the organs in the body, especially the lungs and pancreas.
The Cystic Fibrosis Foundation, which helped fund the study, said about 70,000 people worldwide, including 30,000 people in the United States, have CF. People with the disease can expect to live to about age 37.
Scientists create animal "models" of a disease in order to perform experiments that would not be possible with people. Mice with the genetic abnormality that causes cystic fibrosis have been developed, but the disease presents itself very differently in these rodents than in people.
"Unfortunately, the mice leave something to be desired," Welsh, who helped lead the study, said in a telephone interview.
"They don't get the pancreatic disease like people with CF get. They don't get the lung disease like people with CF get. They don't get the intestinal disease like people with CF get. There's been many questions that can't be answered," he added.
"The onset of these problems is difficult to track down in humans because sometimes they happen before birth or sometimes they happen silently," added Christopher Penland of the foundation.
This is the latest example of pigs used for human medical needs. For example, cardiac patients can get pig heart valves.
The scientists developed pigs with cystic fibrosis because their lungs have many characteristics of human lungs.
"Right now, if you want to do experiments to find treatments or therapies for the lung disease that is fatal for people with CF, you would have to experiment on kids that have CF," Randy Prather of the University of Missouri added.
The disease in pigs closely mimicked the initial stages of the disease seen in people. The scientists are waiting for them to develop lung disease typical of CF "so we can start experimenting in ways that have never been possible," Prather said in a statement.
Tuesday, September 23, 2008
Promising New Approach To Treating Cystic Fibrosis Lung Infection
Researchers at the University of Calgary have found a new method of fighting severe lung infections in people with cystic fibrosis (CF). These findings are published in Proceedings of the National Academy of Science USA, this week. Communities of bacteria grow in the lungs of people with CF. Pseudomonas aeruginosa is a common bacterium found in these communities and is often associated with severe lung infections. Pseudomonas represents a constant and ever present threat to the health of people with CF. Dr. Michael Surette, Professor of Medicine at the University of Calgary, and his team, working with Dr. Harvey Rabin and the Calgary Adult CF Clinic have found that a group of previously overlooked and often undetected bacteria in these communities, the Streptococcus milleri group (SMG), compounds the danger of Pseudomonas aeruginosa. Currently, doctors have treated Pseudomonas with antibiotics, however, the Pseudomonas family of bacteria is increasingly becoming resistant to treatment. Dr. Surette's research shows clinical benefit simply by treating SMG, and thereby disrupting the bacterial community. Doctors at the Calgary Adult CF Clinic (Foothills Hospital) have already tested this new approach successfully, with patients admitted to hospital with severe lung infections. People treated with SMG-targeted therapies quickly returned to a stable state. "This is important new information," said Dr. Michael Surette. "In our small patient group, the laboratory findings have been used to guide treatment, with positive results." The research project, funded by the Canadian Cystic Fibrosis Foundation, has led to a real alternative to combating severe lung infections in persons with CF. Early study results show that it may also be a treatment option for individuals with chronic lung infections unrelated to CF. "These findings underline the importance of supporting CF research," said Cathleen Morrison, Chief Executive Officer of the Canadian Cystic Fibrosis Foundation. "In this case, laboratory research has been translated rapidly into actual treatment, helping people with cystic fibrosis fight back against aggressive infections." ----------------------------Article adapted by Medical News Today from original press release.
Wednesday, September 10, 2008
Changes Ordered for Inhalers
FDA REQUIRES ASTHMA INHALER CHANGES
Albuterol metered dose inhalers (MDIs), also called “short-acting” or “rescue” inhalers, are made by several manufacturers. They have traditionally used chlorofluorocarbons (CFCs) to “propel” the albuterol into the lungs. But now, a new safe and effective alternative propellant, hydroflouroalkane (HFA), has been created to replace CFCs, and will become the required standard for all inhalers starting in 2009. (See official FDA announcement to learn more.)You don’t have to wait until 2009 to make the switch, you can do it today. Many HFA albuterol inhalers are already available. Talk to your doctor today about writing a new prescription for an HFA inhaler and make the switch early so you'll make the 2009 deadline. Between now and December 31, 2008, CFC inhaler production will begin to be phased-out. But as the supply of CFC inhalers decreases, supplies of HFA inhalers will increase to ensure that the total supply meets the total patient demand, up to and beyond the 2009 required transition date.HFA albuterol and levalbuterol medications currently available include:
Proventil-HFA (NDC 00085-1132-01) from Schering-Plough, Ventolin-HFA (NDC# 0173-0682-00) from GlaxoSmithKline lbuterol-HFA (NDC 59310-579-20) from IVAX ,Xopenex-HFA (NDC 63402-510-01) from Sepracor
Albuterol metered dose inhalers (MDIs), also called “short-acting” or “rescue” inhalers, are made by several manufacturers. They have traditionally used chlorofluorocarbons (CFCs) to “propel” the albuterol into the lungs. But now, a new safe and effective alternative propellant, hydroflouroalkane (HFA), has been created to replace CFCs, and will become the required standard for all inhalers starting in 2009. (See official FDA announcement to learn more.)You don’t have to wait until 2009 to make the switch, you can do it today. Many HFA albuterol inhalers are already available. Talk to your doctor today about writing a new prescription for an HFA inhaler and make the switch early so you'll make the 2009 deadline. Between now and December 31, 2008, CFC inhaler production will begin to be phased-out. But as the supply of CFC inhalers decreases, supplies of HFA inhalers will increase to ensure that the total supply meets the total patient demand, up to and beyond the 2009 required transition date.HFA albuterol and levalbuterol medications currently available include:
Proventil-HFA (NDC 00085-1132-01) from Schering-Plough, Ventolin-HFA (NDC# 0173-0682-00) from GlaxoSmithKline lbuterol-HFA (NDC 59310-579-20) from IVAX ,Xopenex-HFA (NDC 63402-510-01) from Sepracor
What is PHOTOPHERIS?
Susan Burroughs during Photopheris treatment in
Birmingham August 2008
Photopheris is a therapeutic technique in which a patient’s lymphocytes (white blood cells) are
collected by a blood separation device. The lymphocytes are then exposed to ultraviolet light in combination with the drug 8-Methozypsorlen. This drug is a photosensitizing agent which becomes active when it is exposed to ultraviolet light. After this exposure, the lymphocytes
are returned to the patient. These treated cells stimulate the immune system to attack
the cells causing the problem. The process is similar to an auto vaccination.
By: Susan Burrough
Monday, September 8, 2008
Breakthrough In Fight Against Deadly Superbug
Early Detection Method Greatly Increases Chances Of Survival
ScienceDaily — A research team led by University of Sunderland scientists has made a major breakthrough in the fight against a deadly hospital infection which kills tens of thousands of people every year, and it will be available within the next year.
Experts have discovered a technique for the early detection of the superbug pseudomonas aeruginosa which particularly infects patients with cystic fibrosis. 70,000 people worldwide are affected by cystic fibrosis and on average around 50 percent of those will be infected with the superbug – 50 percent of those will die.
Although the research concentrated on the superbug’s relation to cystic fibrosis, pseudomonas aeruginosa also attacks patients with localized and systemic immune defects, such as those suffering with burns, patients with AIDS and cancer.
According to the Centre for Disease Control and Prevention in the USA, Pseudomonas aeruginosa accounts for 10 per cent of all hospital infections.
While the superbug is very difficult to cure as it is highly resistant to antibiotics, early detection makes a huge difference to a patient’s chances of survival. Now for the first time, the University of Sunderland–led team has discovered a technique that can identify the superbug within 24-48 hours of infection, greatly increasing a patient’s chances of survival.
The team is led by Professor Paul Groundwater and Dr Roz Anderson at the University of Sunderland, in collaboration with colleagues Professor John Perry, Freeman Hospital, Newcastle, Professor Arthur James, Northumbria University and Dr Sylvain Orenga, bioMérieux, France
Prof Groundwater says: “This superbug has a massive impact on people who are immunocompromised, for example patients with severe burns, cancer and AIDS.
“It is calculated that 28 per cent of people who have undergone transplant surgery are infected by pseudomonas aeruginosa. We hope our research will make a big difference in the survival rate of many thousands of vulnerable people throughout the world.
“The bacteria infect the fluid on the lungs of cystic fibrosis sufferers. It also infects patients in intensive care units. It is really difficult to treat, and hospital staff need to know very quickly if someone has been infected by it.
“In our new diagnostic method a non-coloured compound reacts with an enzyme present in pseudomonas aeruginosa and produces a very distinctive purple colour which indicates the presence of the bacteria. This technique works on 99 per cent of the strains of this superbug.”
The research has been sponsored by the multinational biotechnology company bioMérieux. The company, based in France, designs, develops, and produces a wide range of diagnosis systems for medicine and industry.
“bioMérieux is very proud to have participated in and supported this research that will help in the fight against healthcare associated infections - a strategic focus for our company,” says Dr. Peter Kaspar, bioMérieux corporate vice-president of research and development. “This discovery will enable bioMérieux to bring additional high-medical value tests to clinicians and positively impact patients’ treatment and their follow-up care.”
Background
Cystic fibrosis is an inherited chronic disease that affects the lungs and digestive system of about 70,000 children and adults worldwide. A defective gene and its protein product cause the body to produce unusually thick, sticky mucus that clogs the lungs and leads to life-threatening lung infections, obstructs the pancreas and stops natural enzymes from helping the body break down and absorb food.
Pseudomonas aeruginosa is a major cause of infection among patients with immune defects. It is tolerant to many detergents, disinfectants and antimicrobial compounds and is difficult to control in hospitals and institutional environments. It causes urinary tract infections, respiratory system infections, dermatitis, soft tissue infections, bacteremia, bone and joint infections, gastrointestinal infections and a variety of systemic infections, particularly in patients with severe burns and in cancer and AIDS patients who are immunosuppressed. Pseudomonas aeruginosa infection is a serious problem in patients hospitalized with cancer, cystic fibrosis, and burns. The case fatality rate in these patients is near 50 percent.
According to the Centre for Disease Control and Prevention in the USA, pseudomonas aeruginosa currently accounts for 10.1 per cent of all hospital infections
ScienceDaily — A research team led by University of Sunderland scientists has made a major breakthrough in the fight against a deadly hospital infection which kills tens of thousands of people every year, and it will be available within the next year.
Experts have discovered a technique for the early detection of the superbug pseudomonas aeruginosa which particularly infects patients with cystic fibrosis. 70,000 people worldwide are affected by cystic fibrosis and on average around 50 percent of those will be infected with the superbug – 50 percent of those will die.
Although the research concentrated on the superbug’s relation to cystic fibrosis, pseudomonas aeruginosa also attacks patients with localized and systemic immune defects, such as those suffering with burns, patients with AIDS and cancer.
According to the Centre for Disease Control and Prevention in the USA, Pseudomonas aeruginosa accounts for 10 per cent of all hospital infections.
While the superbug is very difficult to cure as it is highly resistant to antibiotics, early detection makes a huge difference to a patient’s chances of survival. Now for the first time, the University of Sunderland–led team has discovered a technique that can identify the superbug within 24-48 hours of infection, greatly increasing a patient’s chances of survival.
The team is led by Professor Paul Groundwater and Dr Roz Anderson at the University of Sunderland, in collaboration with colleagues Professor John Perry, Freeman Hospital, Newcastle, Professor Arthur James, Northumbria University and Dr Sylvain Orenga, bioMérieux, France
Prof Groundwater says: “This superbug has a massive impact on people who are immunocompromised, for example patients with severe burns, cancer and AIDS.
“It is calculated that 28 per cent of people who have undergone transplant surgery are infected by pseudomonas aeruginosa. We hope our research will make a big difference in the survival rate of many thousands of vulnerable people throughout the world.
“The bacteria infect the fluid on the lungs of cystic fibrosis sufferers. It also infects patients in intensive care units. It is really difficult to treat, and hospital staff need to know very quickly if someone has been infected by it.
“In our new diagnostic method a non-coloured compound reacts with an enzyme present in pseudomonas aeruginosa and produces a very distinctive purple colour which indicates the presence of the bacteria. This technique works on 99 per cent of the strains of this superbug.”
The research has been sponsored by the multinational biotechnology company bioMérieux. The company, based in France, designs, develops, and produces a wide range of diagnosis systems for medicine and industry.
“bioMérieux is very proud to have participated in and supported this research that will help in the fight against healthcare associated infections - a strategic focus for our company,” says Dr. Peter Kaspar, bioMérieux corporate vice-president of research and development. “This discovery will enable bioMérieux to bring additional high-medical value tests to clinicians and positively impact patients’ treatment and their follow-up care.”
Background
Cystic fibrosis is an inherited chronic disease that affects the lungs and digestive system of about 70,000 children and adults worldwide. A defective gene and its protein product cause the body to produce unusually thick, sticky mucus that clogs the lungs and leads to life-threatening lung infections, obstructs the pancreas and stops natural enzymes from helping the body break down and absorb food.
Pseudomonas aeruginosa is a major cause of infection among patients with immune defects. It is tolerant to many detergents, disinfectants and antimicrobial compounds and is difficult to control in hospitals and institutional environments. It causes urinary tract infections, respiratory system infections, dermatitis, soft tissue infections, bacteremia, bone and joint infections, gastrointestinal infections and a variety of systemic infections, particularly in patients with severe burns and in cancer and AIDS patients who are immunosuppressed. Pseudomonas aeruginosa infection is a serious problem in patients hospitalized with cancer, cystic fibrosis, and burns. The case fatality rate in these patients is near 50 percent.
According to the Centre for Disease Control and Prevention in the USA, pseudomonas aeruginosa currently accounts for 10.1 per cent of all hospital infections
Subscribe to:
Posts (Atom)
Posts
