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Orkambi: Cystic Fibrosis Treatment May Improve And Prolong Patients’ Lives

Cystic Fibrosis Drug

A “groundbreaking” combination treatment for cystic fibrosis could improve and prolong the lives of patients suffering this disease, doctors have announced. Doctors said that the combination of lumacaftor and ivacaftor could improve lung function while simultaneously increasing life expectancy in cystic fibrosis patients, according to a report by the International Business Times.

Patients often die before their 40s as mucus clogs and damages their lungs and leaves them prone to infection. Errors in patients’ DNA inherited from their parents  damage the microscopic machinery that controls salt and water levels in the linings of the lungs.

A major trial headed by a team of researchers from University of Queensland School of Medicine in Australia and the Queens University in Belfast, which analyzed 1,108 patients, showed a combination of drugs could bypass the genetic errors that cause the disease and may increase life expectancy.

The Cystic Fibrosis Trust said it could “improve the lives of many,” according to a BBC News report.

Researchers also noted a 40-percent decrease in “pulmonary exacerbations,” which leads to inflammation and infections, causing death among the patients, the Daily Telegraph reported. However, the study  which was published in the New England Journal of Medicine  noted that this cystic fibrosis treatment will not work for all cystic fibrosis patients because the new treatment is limited to the targeting of a specific gene mutation.

Professor Stuart Elborn, who led the European part of the trial from Queen’s University, was quoted in the aforementioned BBC News report as having said that it’s “very exciting and it really demonstrates that we can correct the basic defects in cystic fibrosis.”

This is very exciting and it really demonstrates that we can correct the basic defects in cystic fibrosis (…) This is likely to become a fundamental treatment for cystic fibrosis (…) Starting in children may prevent the disease process developing if we correct the basic defect early in life (…) Will this improve survival for people with cystic fibrosis? We would anticipate it would have a really good chance of doing that, but we don’t know for sure yet.

The U.S. drug watchdog, the Food and Drug Administration (FDA), is expected to make a decision on whether to approve the drug known as Orkambi, which was developed by the Boston-based Vertex company, come July 5, 2015, according to the Daily Mail. Its advisory committee voted 12 to one in favor of approving the drug. An application for its approval in Europe is being considered by European Medicines Agency, with a decision expected to arrive this upcoming autumn.

Jillian McNulty, 39, a campaigner and fundraiser for cystic fibrosis patients, was one of eight adults accepted for the trial in Ireland. Recalling her experience with the new treatment, McNulty told the Daily Mail that she felt “very sick” and was in the hospital for three months after she started the treatment. Since which time, she claims she hasn’t “looked back,” as her health has continued to “improve daily.”

Since then I haven’t looked back, my health has improved daily (…) My lung functions have remained the same since July 2013, at times my lung function has increased by 8 per cent (…) Orkambi has changed my life – it isn’t a cure, I still need to do my daily routine of physiotherapy and nebulisers and medications but it has stopped the progression

What do you think of this new combination treatment’s reported success in improving the lives of those afflicted with cystic fibrosis?

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