Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder that affects around 10,000 people globally. The red blood cells in PNH patients are weak and are destroyed more rapidly than normal. This causes the urine to turn red or dark during an episode (or paroxysm) of red cell destruction (or hemolysis).
Usually the urine is darker first thing in the morning (nocturnal) and clears through the day. Each episode of dark urine usually lasts for a few days and episodes may occur very occasionally or very often. During an attack of dark urine many patients have mild abdominal discomfort. However, some patients with PNH never have attacks of dark urine, so the condition can be erratic.
The standard treatment for PNH was blood transfusions every three months to stave off chronic tiredness, jaundice and difficulty swallowing. Alexion Pharmaceuticals has developed a new treatment for PNH called Soliris (eculizumab), an infusion therapy given every couple of weeks that reduces the need for dependence on blood transfusions. Eculizumab is a humanised monoclonal antibody that inhibits terminal complement activation.
The catch?
The treatment costs approx. $400K per year, something that has raised a lot of concern, as you can see from reports here, here and here.
It's hard to justify such a high price for a therapy when the top end cost for drugs that target around 10,000 patients globally is under $300K a year; even some UK PCT's thought it was not cost effective in the absence of NICE review, as reported recently on BBC News.
Showing posts with label competitive intelligence. Show all posts
Showing posts with label competitive intelligence. Show all posts
Monday, November 3, 2008
Friday, July 25, 2008
Forgotten side of the Iraq war: shortage of meds for cancer patients
This interesting (and sad) letter in the New England Journal of Medicine highlights the shortage of chemotherapy treatments for children suffering from leukemia:
"There was a significant inverse relationship between the amount of prescribed chemotherapy that was administered and the risk of relapse."
The study reinforced the notion that the most important factor in improving survival, even in children with leukemia, is adequate treatment.
"There was a significant inverse relationship between the amount of prescribed chemotherapy that was administered and the risk of relapse."
The study reinforced the notion that the most important factor in improving survival, even in children with leukemia, is adequate treatment.
Sunday, July 20, 2008
Cancer: tracking progress for the treatment of CML
This fascinating short video interviews some of the top leukemia doctors around the world and looks at how tracking faulty chromosomes is now routine for people with chronic myeloid leukemia. Experts such as Dr Brian Druker and Prof. John Goldman explain what sophisticated laboratory tests can tell about treatment success:
Video
Video
Monday, May 12, 2008
Knocking out cancer cells in Leukemia
Cancer initiating cells (CICs) or leukemia initiating cells (LICs) are stealth populations impervious to conventional chemotherapy and resistant to targeted cancer therapies. When a leukemia patient relapses following a period of remission, it is the LICs that are the cause of the disease’s re-emergence.
Researchers at the Beth Israel Deaconess Medical Center (BIDMC) have found that a tumour suppressor protein known as PML appears to be the factor that enables LICs to maintain their quiescence, the inert state that protects them from being destroyed by cancer therapies. They suggest that inhibition of PML is a promising target for new agents.
Their findings, which appear in Nature, also demonstrate that PML can be degraded with an arsenic-based agent called arsenic trioxide, which has been used in traditional Chinese medicine. When combined with chemotherapy, the arsenic-based therapy, already proven safe and non-toxic in clinical trials for a rare form of leukemia called Acute Promyelocytic Leukemia (APL) can also potentially treat the more common Chronic Myeloid Leukemia (CML) and reduce risk of relapse.
The concept is a very simple one; 90% of existing cancer treatments are anti-proliferative agents. They target the pool of proliferative cells, leaving behind the dormant LICs. By knocking out the quiescent cells with arsenic trioxide, the risk of relapse is much reduced. This approach might be useful in other treatment approaches for different leukemias.
Researchers at the Beth Israel Deaconess Medical Center (BIDMC) have found that a tumour suppressor protein known as PML appears to be the factor that enables LICs to maintain their quiescence, the inert state that protects them from being destroyed by cancer therapies. They suggest that inhibition of PML is a promising target for new agents.
Their findings, which appear in Nature, also demonstrate that PML can be degraded with an arsenic-based agent called arsenic trioxide, which has been used in traditional Chinese medicine. When combined with chemotherapy, the arsenic-based therapy, already proven safe and non-toxic in clinical trials for a rare form of leukemia called Acute Promyelocytic Leukemia (APL) can also potentially treat the more common Chronic Myeloid Leukemia (CML) and reduce risk of relapse.
The concept is a very simple one; 90% of existing cancer treatments are anti-proliferative agents. They target the pool of proliferative cells, leaving behind the dormant LICs. By knocking out the quiescent cells with arsenic trioxide, the risk of relapse is much reduced. This approach might be useful in other treatment approaches for different leukemias.
Wednesday, April 30, 2008
Hematology - heparin contaminant may have been deliberate
A US Congressional hearing was held on Tuesday to discuss the recent heparin contamination issue. The hearing was dubbed, "The Heparin Disaster: Chinese Counterfeits and American Failures".
Baxter, which supplied half of the US market, issued a voluntary recall of the drug in January 2008 and again in February after multiple patients starting experiencing severe side effects like abdominal pain, decreased blood pressure, burning sensation, chest pain, diarrhea, dizziness, loss of consciousness and vomiting. The drug eventually caused more than 80 deaths.
The company denied that it was responsible for contaminated batches of Heparin, an anticoagulant used mostly in hospitals, and that it may have been a deliberate contamination. It's CEO, Robert Parkinson, said that, CEO Parkinson said, "We're alarmed that one of our products was used, in what appears to have been a deliberate scheme, to adulterate a life-saving medication, and that people have suffered as a result".
The contaminant was an altered form of chondroitin sulfate, a drug that mimics the affects of Heparin. The company said during the hearing that the drug was in the batches before it reached Baxter's supplier, Scientific Protein Laboratories. Several other companies in multiple other countries found traces of the contaminant in their supplies of Heparin, as well. Parkinson testified that the company is still trying to understand where in the supply chain the contaminant was introduced, but the company believes it was in the raw material.
Heparin is one of several products coming out of China that have been recalled because of problems with production. Last year, there were recalls concerning toys and toothpaste made in China. The Chinese government has maintained that the contaminated Heparin was not associated with the adverse affects and deaths. This theory was debunked by Kishimoto et al., who provided a scientific rationale for a potential biologic link between the presence of over-sulphated condroitin in suspect lots of heparin and the observed clinical adverse events. They reported their findings in the NEJM earlier this year.
Heparin is a generic that has been around for more than 70 years. It is made from pig intestines. At the hearing, members of Congress discussed the rising cost of hogs as a potential reason for someone substituting the Heparin with the lower-cost chondroitin sulfate. The contaminant is an unnatural substance that was highly processed, pointing to the likelihood that the substance was deliberately added.
The committee appeared to be laying blame with the US FDA inspectors; the FDA only inspects foreign drug makers every 13 years. Baxter relied on inspections and audits of the SLP factory done by its predecessor, Wyeth, and did not conduct audits of the Chinese factory itself.
In short, it was a disaster waiting to happen; no one took responsibility or accountablity for the quality of either the raw materials or the final product, ultimately compromising patient safety.
Baxter, which supplied half of the US market, issued a voluntary recall of the drug in January 2008 and again in February after multiple patients starting experiencing severe side effects like abdominal pain, decreased blood pressure, burning sensation, chest pain, diarrhea, dizziness, loss of consciousness and vomiting. The drug eventually caused more than 80 deaths.
The company denied that it was responsible for contaminated batches of Heparin, an anticoagulant used mostly in hospitals, and that it may have been a deliberate contamination. It's CEO, Robert Parkinson, said that, CEO Parkinson said, "We're alarmed that one of our products was used, in what appears to have been a deliberate scheme, to adulterate a life-saving medication, and that people have suffered as a result".
The contaminant was an altered form of chondroitin sulfate, a drug that mimics the affects of Heparin. The company said during the hearing that the drug was in the batches before it reached Baxter's supplier, Scientific Protein Laboratories. Several other companies in multiple other countries found traces of the contaminant in their supplies of Heparin, as well. Parkinson testified that the company is still trying to understand where in the supply chain the contaminant was introduced, but the company believes it was in the raw material.
Heparin is one of several products coming out of China that have been recalled because of problems with production. Last year, there were recalls concerning toys and toothpaste made in China. The Chinese government has maintained that the contaminated Heparin was not associated with the adverse affects and deaths. This theory was debunked by Kishimoto et al., who provided a scientific rationale for a potential biologic link between the presence of over-sulphated condroitin in suspect lots of heparin and the observed clinical adverse events. They reported their findings in the NEJM earlier this year.
Heparin is a generic that has been around for more than 70 years. It is made from pig intestines. At the hearing, members of Congress discussed the rising cost of hogs as a potential reason for someone substituting the Heparin with the lower-cost chondroitin sulfate. The contaminant is an unnatural substance that was highly processed, pointing to the likelihood that the substance was deliberately added.
The committee appeared to be laying blame with the US FDA inspectors; the FDA only inspects foreign drug makers every 13 years. Baxter relied on inspections and audits of the SLP factory done by its predecessor, Wyeth, and did not conduct audits of the Chinese factory itself.
In short, it was a disaster waiting to happen; no one took responsibility or accountablity for the quality of either the raw materials or the final product, ultimately compromising patient safety.
Tuesday, April 29, 2008
Oncology news - lowering risk for leukemia
A new analysis of published studies has found that children who attended day care or playgroups had about a 30% lower risk of developing acute lymphoblastic leukemia (ALL) than children who did not.
ALL is the most common type of childhood leukemia, accounting for more than 80% of cases, and typically occurs in infants between the ages of 2 and 5 years. It is one of the most common cancers in children in the industrialized world, affecting about 1 in 2000 children.
One theory about how the disease develops focuses on early infection. Some proponents of this theory believe that if the immune system is not challenged early in life and does not develop normally, then it mounts an inappropriate response to infections encountered later in childhood, the charity explains. This could provoke the development of leukemia in children who are susceptible, for example, because of a genetic mutation.
Children who attend day care and playgroups are likely to be exposed to common infections early in life; such environments are known to increase the spreading of infection. The latest finding supports the theory that early exposure to infection offers some protection against the disease.
The analysis included 14 published studies and involved 6108 children with and 13,704 without leukemia. Parents were asked about day care and playgroup attendance and other forms of social interaction. Twelve of the studies showed that social interaction had a protective effect against leukemia and 2 showed no effect. Overall, the risk for leukemia was lowered by about 30%. This remained the case when the researchers re-analysed the data and considered only children who had attended day care before the age of 2 years. When 5 studies were excluded because of concerns about the methodology that had been used, analysis of the remaining 9 studies found that the risk for leukemia was lowered by 40%.
Source: 2nd Children with Leukaemia Causes and Prevention of Childhood Leukemia Conference. Presented April 29, 2008.
ALL is the most common type of childhood leukemia, accounting for more than 80% of cases, and typically occurs in infants between the ages of 2 and 5 years. It is one of the most common cancers in children in the industrialized world, affecting about 1 in 2000 children.
One theory about how the disease develops focuses on early infection. Some proponents of this theory believe that if the immune system is not challenged early in life and does not develop normally, then it mounts an inappropriate response to infections encountered later in childhood, the charity explains. This could provoke the development of leukemia in children who are susceptible, for example, because of a genetic mutation.
Children who attend day care and playgroups are likely to be exposed to common infections early in life; such environments are known to increase the spreading of infection. The latest finding supports the theory that early exposure to infection offers some protection against the disease.
The analysis included 14 published studies and involved 6108 children with and 13,704 without leukemia. Parents were asked about day care and playgroup attendance and other forms of social interaction. Twelve of the studies showed that social interaction had a protective effect against leukemia and 2 showed no effect. Overall, the risk for leukemia was lowered by about 30%. This remained the case when the researchers re-analysed the data and considered only children who had attended day care before the age of 2 years. When 5 studies were excluded because of concerns about the methodology that had been used, analysis of the remaining 9 studies found that the risk for leukemia was lowered by 40%.
Source: 2nd Children with Leukaemia Causes and Prevention of Childhood Leukemia Conference. Presented April 29, 2008.
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