MedWorm Tags: fibrosis

For Non-Profits: More Investments And More Greed

Pharmalot — Mon, 25 Jul 2011 14:59:40 +0100

Early next year, a drug for cystic fibrosis is expected to be reviewed by the FDA and it is a moment the Cystic Fibrosis Foundation has been waiting years to occur. The non-profit has invested $75 million and approval of the drug - which was developed by Vertex Pharmaceuticals with support from the foundation - would provide a new treatment for patients. And CFF will get a new revenue stream. Why? As Nature writes: CFF has a stake in the intellectual property underlying the drug and is entitled to royalties. So-called venture philanthropy is increasing among foundations, which are managing research projects, making funding dependent on projects reaching predetermined milestones and potentially reaping a return. They are also trying to keep control in the event obstacles emerge. Not surpris...

Nathan Charles

Life in the Fast Lane — Thu, 19 May 2011 01:47:55 +0100

Patients are often a source of inspiration and hope. One such stand out individual is Nathan Charles. (Source: Life in the Fast Lane)

Acute Myelogenous Leukemia (AML)

Inside Surgery — Mon, 25 Apr 2011 06:00:43 +0100

Pathophysiology group of disorders (at least nine different variants) in which a hematopoietic stem cell becomes neoplastic or alternately an individual lineage of stem cells (e.g., erythrocytes, monocytes, granulocytes, or megakaryocytes) becomes neoplastic Signs and Symptoms 1) usually presents with signs of anemia – pallor, fatigue, weakness 2) splenomegaly 3) hepatomegaly 4) hemorrhage in GI tract and CNS if platelets are < 20,000/dL 5) dyspnea owing to infiltration of lung by leukocytes 6) secondary infections 7) gingival hyperplasia Characteristic Test Findings Bone marrow – 1) by definition, > 30% of nucleated cells are blasts Laboratory – 2) anemia 3) thrombocytopenia 4) neutrophilia (but total leukocyte count may be increased or decreased) Histology/Gross Patholo...

Former Biotech CEO Sentenced For A Press Release

Pharmalot — Thu, 14 Apr 2011 19:22:06 +0100

W. Scott Harkonen, who once headed InterMune, was sentenced for wire fraud in connection with disseminating false and misleading statements about clinical trial results for its Actimmune drug. There is no jail time, though. He was sentenced to three years’ probation, six months of home confinement, a $20,000 fine and 200 hours of community service. He was convicted in a September 2009 trial. Harkonen was ceo from 1998 until 2003 and, during that time, he was accused of making up details about how effective the drug was in combatting a fatal lung disease, idiopathic pulmonary fibrosis, or IPF. In effect, he had been charged with orchestrating an off-label marketing scheme for Actimmune (back story). A press release Harkonen wrote touting the benefits of Actimmune to treat IPF in August 20...

Pharmalot… Pharmalittle… Good Morning

Pharmalot — Wed, 30 Mar 2011 11:41:48 +0100

Good morning, everyone. How are you today? A busy agenda awaits us here on the Pharmalot corporate campus, where we have a pile of meetings and deadlines to attack. We trust you can relate. To cope, yes, we are downing a cup or two of stimulation. As always, we invite you to join us. And to get you started, here are some tidbits from the world at large. Hope your day goes well and do stay in touch… Sonova Execs Resign After Insider Trading Probe (Bloomberg News) Gilead And Yale Former Cancer Research Deal (Reuters) Valeant Offers To Buy Cephalon For $5.7 Billion (Bloomberg News) Glaxo’s Lovaza Partner Pronova Settles Patent Row With Apotex (Reuters) UK’s NICE Rejects Bristol-Myers’ Orencia Drug (Dow Jones) Vertex CF Drug Shows Promising Results (Mass High Tech) Merc...

Reductions in Drinking for Hep C Patients

Twelve Step Facilitation.com — Thu, 30 Sep 2010 07:04:14 +0100

DISCUSSION: Brief treatment addressing heavy drinking delivered by hepatitis clinicians with psychiatric-specialist follow-up was associated with abstinence or a significant reduction in alcohol consumption in over 50% of patients. Eric Dieperink, M.D., Samuel B. Ho, M.D., Sara Heit, M.S., R.N., C.N.S., Janet M. Durfee, R.N., M.S.N., APRN, Paul Thuras, Ph.D., and Mark L. Willenbring, M.D. Psychosomatics 51:149-156, March-April 2010 See also Hepatitis C – Does sexual transmission occur? Counselor Magazine’s Addiction Professional Reference Guide Disturbing Denial Improving Treatment Compliance Wernicke-Korsakoff Syndrome Random ArticlesRecovering Alcoholics Effective in Helping OthersFamilies, mental health & alcohol abuseAlcohol Across the LifespanBrief-TSF DescriptionPrinci...

Primary Biliary Cirrhosis

Inside Surgery — Mon, 06 Sep 2010 20:20:23 +0100

Pathophysiology 1) progressive inflammation and destruction of the intrahepatic bile ducts 2) causes development of cirrhosis 3) likely has an autoimmune component Signs and Symptoms 1) fatigue 2) jaundice 3) pruritus 4) hepatomegaly 5) splenomegaly 6) xanthelasma 7) diagnosis is often made on asymptomatic laboratory detection Characteristic Test Findings Laboratory – 1) increased alkaline phosphatase (3 times normal value) 2) mildly elevated AST and ALT 3) antimitochondrial antibody Histology/Gross Pathology Ludwig’s classification describes histology – portal and periportal scarring and fibrosis and cirrhosis Associated Conditions 1) osteoporosis 2) hypercholesterolemia 3) thyroid disease (15%) 4) scleroderma (15%) 5) joint pain 6) dry eyes/mouth (75%) 7) gallstones (30...

The InterMune Shocker: What The Wags Are Saying

Pharmalot — Wed, 05 May 2010 14:45:52 +0100

Last night, a high-flying biotech, InterMune, was brought back down to earth when the FDA issued a surprise rejection of its drug to treat idiopathic pulmonary fibrosis, which Wall Street was betting would generate $1 billion a year or more in annual sales. In anticipation of approval, the stock had increased roughly five times, reaching almost $50 a share - until now. The stock is around $10. About 100,000 folks in the US suffer from this fatal lung ailment, but InterMune is unlikely to win approval any time soon, because the FDA saked for a new clinical trial of the medicine, called Esbriet or pirfenidone, to prove it delays progression. As TheStreet points out, InterMune should have raised more money after an FDA panel voted 9 to 3 to recommend approval last March. Now it has just about...

When A Patient Outgrows A Childhood Disease

Better Health — Tue, 04 May 2010 14:00:00 +0100

At a [recent] session on caring for adult survivors of pediatric diseases, Bradley J. Benson, FACP, and Niraj Sharma, FACP, had some interesting statistics to share. For example, more than 90% of children with a chronic or disabling health condition are expected to live more than 20 years, meaning they’ll eventually need an internist’s care, and every year more than 500,000 children with special healthcare needs turn 18. As Dr. Sharma noted, “We’re not talking about a handful of folks.” (more…) *This blog post was originally published at ACP Internist* (Source: Better Health)

Silicosis

Inside Surgery — Mon, 12 Apr 2010 19:59:09 +0100

Pathophysiology 1) pneumoconiosis owing to chronic inhalation of crystalline silicon dioxide 2) can result in pulmonary fibrosis from chronic injury to lung tissue Signs and Symptoms 1) nonproductive cough 2) in advanced disease with pulmonary fibrosis, dyspnea and tachypnea occur Characteristic Test Findings Radiology – 1) chest radiograph shows “eggshell” calcifications in hilar lymph nodes and honeycomb lung PFTs – 2) both restrictive and obstructive impairment occurs Histology/Gross Pathology 1) macrophages phagocytose silica particles 2) results in nodule formation in lung parenchyma (areas of fibrosis entrapping silica particles) 3) especially affects upper lung lobes Associated Conditions 1) increased incidence of tuberculosis 2) Caplan’s syndrome (fibr...

Beth Sufian Fights for Those Living with Cystic Fibrosis

Disruptive Women in Health Care — Wed, 07 Apr 2010 12:17:26 +0100

Beth Sufian is one of the oldest survivors of Cystic Fibrosis. As an attorney, the Houstonian has fought for the medical rights of thousands of patients — even from her own hospital bed — and travels the country teaching parents how to advocate for their children. She took a few minutes to talk with Disruptive Women’s Wendy Grossman. Q: You’ve dedicated your career to fighting for people living with CF. A: Yes. Q: Can you tell me a little bit about your work? A: Working with CF is about half of what I do — the other half is serious medical conditions. Q: Like what? A: I run a hotline for people with CF from all over the country to call and get information about health insurance and benefits and rights and employment. We’ve been in existence since 1998, and w...

Reductions in Drinking for Hep C Patients

Twelve Step Facilitation.com — Fri, 26 Mar 2010 09:34:46 +0100

DISCUSSION: Brief treatment addressing heavy drinking delivered by hepatitis clinicians with psychiatric-specialist follow-up was associated with abstinence or a significant reduction in alcohol consumption in over 50% of patients. Eric Dieperink, M.D., Samuel B. Ho, M.D., Sara Heit, M.S., R.N., C.N.S., Janet M. Durfee, R.N., M.S.N., APRN, Paul Thuras, Ph.D., and Mark L. Willenbring, M.D. Psychosomatics 51:149-156, March-April 2010 See also Hepatitis C – Does sexual transmission occur? Counselor Magazine’s Addiction Professional Reference Guide Disturbing Denial Improving Treatment Compliance Wernicke-Korsakoff Syndrome Related Reading: Share/SaveRandom ArticlesPatient Mental Illness in a Dental School ClinicComorbid anxiety or alcohol disorderAA and recovery from alc...

Cystic fibrosis

Nursing Comments — Wed, 24 Mar 2010 11:02:58 +0100

Cystic fibrosis or CF is an inherited disease involving epithelial cells. Epithelial cells are found lining the skin, sweat glands, and respiratory, gastrointestinal and genitourinary tracts. In people with cystic fibrosis, the epithelial cells do not function properly. These impaired cells cause abnormal regulation of the flow of salts and water. The result is abnormal secretions such as a thick, sticky mucus that clogs the lungs. It is a life-threatening disorder that causes severe lung damage and nutritional deficiencies. The affected gene, which is inherited from a child’s parents, is a recessive gene. With recessive genes, children need to inherit two copies of the gene, one from each parent, in order to have the disease. If children inherit on...

Archives of Dermatology 2010 (Vol. 146 No. 2)

Fade Library — Mon, 22 Mar 2010 10:42:09 +0100

Contents page Fade Fave: Cystic Fibrosis Presenting With Dermatitis Fade Skinny: Given the frequent delay in diagnosis, as well as the increased morbidity and mortality associated with protein-energy malnutrition in these patients, it is important to consider cystic fibrosis as a possible diagnosis in any infant presenting with a rash and other signs of malnutrition. The relative contribution of specific nutritional deficiencies and the degree to which they influence and interact with each other in producing the dermatitis remain unclear, although they may all affect a common underlying metabolic pathway. (NHS Athens is required to access this article online) Filed under: Athens Password, Current Awareness, E-Journals, Journals Tagged: Athens Password, CF, Current Awareness, Cystic Fibro...

General Electric, Renal Risks And A Libel Lawsuit

Pharmalot — Tue, 22 Dec 2009 12:46:17 +0100

Last month, European medical regulators recommended that anybody who needed an MRI scan should be given a check to ensure their kidneys are healthy if they were to be given General Electric’s Omniscan or two similar contrast agents. And the FDA is conducting its own review. But Henrik Thomsen isn’t talking about it, even though he heads the department of diagnostic sciences at the University of Copenhagen and is part of a small group of clinicians credited with alerting patients and regulators to the potential risks of Omniscan for renal patients. Why? A talk he gave last year prompted GE Healthcare to sue him for libel, The Times of London writes. GE has already racked up costs of more than $600,000 pursuing Thomsen, who has authored or co-authored nearly 400 papers and will ...

Jerry Lewis’ Pulmonary Fibrosis

A Hearty Life — Sun, 06 Sep 2009 11:12:28 +0100

Many of us associate Labor Day with the Jerry Lewis Telethon for Muscular Dystrophy. But this many people are wondering if Lewis will also take the time to speak about his own disease, pulmonary fibrosis (PF). PF is a fatal lung disease, and what’s even scarier is that little is known about the disease. Other celebs like Robert Goulet and Marlon Brando had (and died) of the disease as well. PF scars the lungs and is untreatable and fatal. Most patients did within three to five years of diagnosis. Lewis has been relatively quiet about his disease. If I had to guess I would say he probably won’t speak about his own disease – but rather – focus on Muscular Dystrophy instead. Image: Zuma Press Post from: Blisstree Jerry Lewis’ Pulmonar...

An Antibiotic Against Genetic Diseases?

Genetics and Health — Fri, 21 Aug 2009 12:14:00 +0100

Antibiotics are normally used to treat the wide variety of bacterial infections, however they are ineffective against viruses (like the flu and colds) and fungal infections. And while genetic diseases can be treated with therapy and proper management, the gene will always be defective. AND, antibiotics are not known or normally used to treat genetic defects. Well, this antibiotic may be the exception. Israeli scientists have modified a potent antibiotic that may allow it to treat cystic fibrosis and other genetic diseases caused by “stop mutations”. The anti-bacterial antibiotic gentamicin is one of the highly toxic antibiotics called aminoglycosides that doctors only use when less powerful drugs prove ineffective. but gentamicin is extremely toxic and lethal when taken long te...

Swine Flu Recuperation

Frankie Speaking Frankly — Thu, 09 Jul 2009 21:22:00 +0100

Today I feel better. Yesterday after posting that I was on the mend I then seemed to take a turn for the worse - my temperature started to rise again, I developed pain across one side of my chest, started to sweat a lot and sensed my cough was changing into something maybe worse. So after 10 days of fighting this thing and feeling completely exhausted, I accepted it was about time to start antibiotics since my infection was likely turning bacterial. I started to read about pneumonia and decided I didn't want to take the risk.I then started to worry about my father who seemed worse than myself and not really getting better either. I called my mother and insisted that she call the doctor the next morning whether he agreed or not. My father, in the typical British manner, prefers to struggle ...

Why Jews have High IQ and disease-prone

Genetics and Health — Sun, 03 May 2009 04:27:25 +0100

Ashkenazi Jews are known to have an average IQ between 107-115, putting half of the ethnic group into the genius range. Unfortunately, Ashkenazi Jews are also plague with genetic diseases! One fourth of the population is a carrier of one of several genetic conditions, which include Tay-Sachs Disease, Canavan, Niemann-Pick, Gaucher, Familial Dysautonomia, Bloom Syndrome, Fanconi anemia, Cystic Fibrosis and Mucolipidosis IV. A “carrier” for a gene means that the person carries only one copy of the gene. The gene is not expressed in that person’s trait or phenotype. However, marrying another carrier or someone with two copies of the gene may produce children with the trait. For example, a carrier for blue eyes may have a different eye color but has children with blue eyes. When talk...

2 lungs better than 1 in transplantation

A Hearty Life — Thu, 23 Apr 2009 11:53:54 +0100

“Two lungs are better than one - when it comes to lung transplantations,” thanks to a new study that looked at the long-term survival rates of patients who had lung transplants. Lung transplantation may be an option for people with cystic fibrosis , emphysema, idiopathic pulmonary fibrosis , or sarcoidosis , although there may be other illnesses in the group as well. The study, done by researchers from Johns Hopkins, found that having both lung replaced by healthy lungs because if one lung is left behind, there may be some residual (left over) parts of the disease, which could end up causing problems. The researchers also point out that “when both lungs are replaced, the new lungs, which must breathe together as a pair, are already adapted to each other.” The encour...

Male fertility gene found!

Genetics and Health — Wed, 08 Apr 2009 14:04:00 +0100

Some men have more success than others in producing children, genetically speaking, that is. Large European family, circa 1950s. New findings have identified a gene that endows some men more reproductive prowess than their neighbor, according to an article by The Scientist. And the secret is a switch in amino acids within one gene – from an amino acid called methionine to valine. Carole Ober of the University of Chicago, presented her findings at the Sackler Colloquium on Evolution in Health and Medicine in Washington, DC and she found that “men who carried two copies of the valine allele were 2.6 times more likely to conceive than men with two copies of the methionine allele at the same locus”. Man, that’s double the advantage! The gene in the spotlight? CFTR, or cystic fibrosis t...

Genetically Altering Pigs to Find Cure for Cystic Fibrosis

Secondhand Smoke — Fri, 26 Sep 2008 04:45:00 +0100

This is an example of how animal research can lead to tremendous alleviation of human suffering. Pigs are being genetically altered to have CF and cloned for use in research. From the story:Cystic fibrosis (CF) is triggered when a person inherits two copies of a faulty gene carried by about one in 25 of the population. The disorder causes widespread damage to internal organs, especially the lungs and gut, by clogging them with thick, sticky mucus.Now a team at the University of Missouri has developed a pig which appears to closely mimic the disease. The striking similarities suggest that the pigs will help improve understanding and may also speed discovery of new treatments.Animal rights activists would say that the pigs lives are as important and valuable as all those people with cystic f...

Link between cystic fibrosis and diabetes

Diabetes Notes — Sun, 17 Aug 2008 18:49:31 +0100

Doctors have found that 80 per cent of Tasmanians with cystic fibrosis over the age of 18 have diabetes. Cystic fibrosis is a disease of the mucous and sweat glands. There is hopes that this discovery will increase life expectancy. “They are now put on to insulin once or twice or four times a day and we are seeing benefits in their lung function, their weight is going back up and they are, we hope, going to do better for longer,” he said. via ABS News Tags: connection between the diseases, cystic fibrosis, Diabetes, linkShare This (Source: Diabetes Notes)

By Secretary or By Professional Report

Gene Sherpas: Personalized Medicine and You — Thu, 14 Aug 2008 15:31:00 +0100

A recent study caught my eye. Done by multiple centers..... from the Division of Laboratory Systems,* Centers for Disease Control and Prevention, Atlanta, Georgia; the Wadsworth Center, New York State Department of Health, Albany, New York; the Albert Einstein College of Medicine, New York, New York; ARUP Laboratories and the University of Utah, Salt Lake City, Utah; the Department of Pediatrics, University of Washington School of Medicine, Seattle, Washington; San Ramon Valley Primary Care, San Ramon, California; the Genetic Services Laboratory,** Sequenom Incorporated, San Diego, California; and the Department of Human Genetics, Mount Sinai School of Medicine, New York, New York.What did they investigate? Simple....how genetic tests were orderded and how results were given. What really g...

Hearty linky love from stem cells to chocolate bars

A Hearty Life — Fri, 25 Apr 2008 18:15:28 +0100

Here are some Friday links for you… Novel Way to Prevent Cardiac Fibrosis Identified- In a study that points to a new strategy for preventing or possibly reversing fibrosis — the scarring that can lead to organ and tissue damage — researchers at the University of California, San Diego School of Medicine have determined that a molecule called Epac (Exchange protein activated by cAMP1), plays a key role in integrating the body’s pro- and anti-fibrotic response. Chocolate Bar Shown To Lower Cholesterol- “Eating two CocoaVia dark chocolate bars a day not only lowered cholesterol, it had the unexpected effect of also lowering systolic blood pressure,” said John Erdman, a U. of I. professor of food science and human nutrition. Heart Derived Stem Cells Develop ...

Hard Up For Research Dollars? Try A Non-Profit

Pharmalot — Mon, 07 Apr 2008 11:12:59 +0100

In addition to raising venture capital and launching stock offerings, Massachusetts biotechs are increasingly turning to another source of funding to support early drug research: nonprofit foundations dedicated to fighting serious diseases, The Boston Globe writes. For instance, the Cystic Fibrosis Foundation, has awarded more than $300 million to for-profit companies over the past decade to help develop cutting-edge therapies, including $192 million in the Boston area. Epix Pharmaceuticals is receiving up to $37 million, in addition to about $12 million it has already received, to help discover new cystic fibrosis drugs. The money is contingent on Epix’s meeting certain goals. Other foundations are following suit. For example: The Juvenile Diabetes Research Foundation said it has gi...

Health Highlights - March 3rd, 2008

Highlight HEALTH — Mon, 03 Mar 2008 17:01:43 +0100

This article was published on Highlight HEALTH. Related articlesHEALTH Highlights - Monday, December 10thHEALTH Highlights - August 21st, 2007HEALTH Highlights - June 1st, 2007HEALTH Highlights - January 14, 2008The Highlight HEALTH Network (Source: Highlight HEALTH)

1,000 Genomes Project Will Help Identify Further Research For Diabetes

Diabetes Notes — Tue, 22 Jan 2008 14:27:55 +0100

The 1,000 Genomes Project, which is launched today by an international consortium of scientists, aims to identify every genetic variant that is carried by at least 1 per cent of the human race, to unlock how these influence health. When the map is complete in two years’ time, it will include the complete genetic codes of more than 1,000 individuals, providing an index to the human genome that will significantly enhance medical research. This medical research will enhance our knowledge of diseases like diabetes and cystic fibrosis. It is said that all humans are more than 99% similar at the genetic level and it is that 1% variant that explains differences in peoples susceptibility to chronic diseases, medications and infections. This is a very exciting project and is truthfully mind blowi...

The Risks of MRI Contrast in Patients with Kidney Disease: Gadolinium and Nephrogenic Systemic Fibrosis (NSF)

Tech Medicine — Mon, 28 May 2007 16:33:41 +0100

Gadolinium is a type of intravenous contrast given during MRIs to help radiologists visualize blood vessels. Recently, the FDA has warned that this type of contrast, in patients with decreased kidney function, has been associated with a new type of potentially fatal skin disease called "nephrogenic systemic fibrosis" (NSF). First, some background. Patients with kidney disease very frequently... (Source: Tech Medicine)

Rescuing mutated genes as potential therapy….

one in ten thousand — Tue, 15 May 2007 17:32:41 +0100

It’s rare that a disease or disorder can be attributed to a single mutation in a gene let alone to a single gene but in certain cases such as in Cystic Fibrosis and Rett syndrome this is exactly what happens. If you were able to find the gene that was responsible for such a disease you might think that you were well on the way towards developing a treatment or cure, but this has proved not to be the case. The problem lies in the fact that the mutations that are often associated with disease usually lead to “silencing” of a vital protein, be it an enzyme or receptor, meaning that the protein is non-functional or is not expressed at all. Medicinal chemistry is only good at modulating (ie. inhibiting or activating) proteins that work normally so there is a fundamental...

Interferon-gamma trial for IPF is discontinued

Pulmonary Roundtable — Mon, 05 Mar 2007 23:35:00 +0100

Well, the press-release was today so it is public info: the phase 3 trial testing IFN-gamma vs. placebo in patients with IPF has been discontinued. The independent data monitoring committee did an interim analysis and found no difference between IFN-gamma and placebo for mortality (14.5% in the Actimmune group as compared to 12.7% in the placebo). The adverse reactions were consistent with prior clinical experience(constitutional symptoms, neutropenia and possibly pneumonia).“The interim results of the INSPIRE trial and our decision to discontinue the trial are disappointing,” said Steve Porter, M.D., Ph.D., Chief Medical Officer at InterMune.I'm not sure what this means for the company, but they are also running the perfenidone trial, but not so sure this will fare any better...To me,...