[ In the case of NTM, the barrier to effective treatment is in gaining access to the interior of infected macrophages. ARIKAYCE liposomes are effective delivery systems that penetrate these barriers and provide high levels of drug in the lung for a long time.]
2. Wikipedia -
[ When in the lungs, M. tuberculosis is taken up by alveolar macrophages ]
3. World Health Organisation -
[ WHO estimates that up to half a million new cases of multidrug-resistant tuberculosis (MDR-TB) occur worldwide, each year. Current treatment regimens for MDR-TB present many challenges: treatment lasts 20 months or more, requiring daily administration of drugs that are more toxic, less effective, and far more expensive than those used to treat drug-susceptible TB ... fluoroquinolones or the second-line injectable drugs (kanamycin, amikacin, capreomycin) ... are the two most effective classes of second-line drugs.]
4. Insmed -
[ Amikacin sulfate ... is often recommended by physicians as part of the standard treatment regimen for some NTM patients ... Because the drug is delivered for months at a time, resulting in high systemic (blood) levels of the drug, there can be considerable toxicity, including ototoxicity and nephrotoxicity, associated with intravenous treatment. ]
5. Insmed -
[ We believe ARIKAYCE has the potential to offer a safety profile different from that of intravenous delivery of amikacin ... We believe this may reduce the potential for the occurrence of any drug-related systemic toxicity, such as nephrotoxicity, which is especially important with diseases like NTM that require long-term drug administration. ]
6. Insmed -
[ In 2013, we concluded the 9 month the dog inhalation toxicity study. In summary, the final report from the study stated that the lung macrophage response in dogs was similar to that seen in our previous 3 month dosing dog study, and there was no evidence of neoplasia, squamous metaplasia or proliferative changes. ]
[ CHICAGO (Reuters) - New federal guidelines expand the range of patients who might benefit from Johnson & Johnson's Sirturo drug for the treatment of multi-drug-resistant (MDR-TB), the first new type of TB drug to win U.S. regulatory approval in more than 40 years. ]
[ The guidance on how best to use Sirturo comes as rates of MDR-TB continue to climb, said Dr. Sundari Mase of the CDC's TB elimination branch and lead author of the guidelines, published in the CDC's Morbidity and Mortality Weekly Report.
Although TB infection is rare in the United States, it has become a global public health threat as poor oversight of available drugs has spawned strains of the bacterium that resist treatment by all but a handful of drugs.
In its annual TB report issued this week, the World Health Organization said 8.6 million people developed TB in 2012 and 1.3 million died from the disease. It warned that 3 million people with TB are being missed by health systems, and drug-resistant "superbug" strains of the bacterial infection have put progress against TB at risk.
According to the report, 450,000 people fell ill with MDR-TB in 27 countries in 2012, with China, India and Russia worst affected.
"Right now, the United States is the only country in the world implementing this drug and rolling it out," Mase said, adding that China is close behind.
The drug, known generically as bedaquiline, was approved by the U.S. Food and Drug Administration last December for MDR-TB as part of combination therapy in adults over the age of 18 with TB infections of the lung.
The approval was based on the available data, but the drug had not been studied in other populations at high risk for the infection.
The CDC's guidelines offer advice on so-called "off label" uses of the treatment for categories of patients with drug-resistant strains of TB that have not yet been studied, said Mase.
She noted that most drugs used to treat TB are "off label". ]
The smart money knows that share price manipulation will prevent the vast majority of retail investors from seeing the game-changing implications of these three dots -
1. Establishing a significant concentration of amikacin in the bloodstream is one of the most effective therapies for the half a million people infected with MDR-TB each year - half of whom still die from the disease.
2. Mycobacterium tuberculosis is taken up by alveolar macrophages.
3. In the case of NTM, the barrier to effective treatment is in gaining access to the interior of infected macrophages. ARIKAYCE liposomes are effective delivery systems that penetrate these barriers.