MSF sponsors and runs own TB clinical trial

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Four years ago, Médecins Sans Frontières (MSF) made the decision to sponsor and run its own tuberculosis clinical trial because ‘too few pharmaceutical companies, organisations or universities were doing enough about it’, writes Dr Bern-Thomas Nyang’wa, a TB specialist at MSF, in The Guardian. The aim was to find a new treatment regimen for drug-resistant tuberculosis (TB) that was radically better than what was currently available.

Nyang’wa writes: “As an organisation that specialises in delivering short-term emergency healthcare, this was a bold and new direction to take. But it was a decision that came from our frustration, anger and impatience on behalf of the more than 20,000 people with TB that we treat every year. We felt compelled to search for improved treatments ourselves because too few pharmaceutical companies, organisations or universities were doing enough about it.

“Every day 4,900 people die from TB, a infectious disease that affects the lungs and causes fever, coughing, and makes it difficult to breathe. It is one of the top 10 causes of death worldwide and now kills more people than HIV.

“Yet the disease does not receive anywhere near the attention it deserves. Care, treatment and diagnostics remain woefully underfunded. Despite the fact that the number of new people being diagnosed with TB every year is decreasing, the overall number of people living with TB is at an all-time high as we fail to cure people already living with the disease.

“An estimated two in every five people who fall sick with the disease are left undiagnosed and untreated. Medicines to treat TB have barely improved in 50 years. The inadequate diagnostics and medicines mean that the complexity and severity of the disease is getting worse. TB needs to be treated with a range of different antibiotics but the same drugs have now been used for decades. The result is that an alarming number of strains of TB have become resistant to these antibiotics. Growing numbers of patients are being told that their six-month course of treatment hasn’t worked and they still have TB.

“People diagnosed with drug-resistant TB face a gruelling two years of treatment, during which time they must swallow more than 10,000 pills and have painful daily injections. The side effects are often incapacitating; they include constant nausea, joint pains, irreversible deafness and even psychosis. Many patients must spend prolonged periods in hospital, unable to earn a living and cut off from friends and family as well as all semblance of a normal life.

“At the end of the two years, only half of these people will be told their treatment has been successful.

“One key reason for the lack of investment in TB is that most people with the disease live in low- and middle-income countries, so there is little financial incentive for pharmaceutical companies to develop or research new drugs. It is also because drug companies make financial gains by patenting drugs. This patent restricts how the drug can be used. As a number of drugs are required to kill TB, it is difficult to trial new drugs together if they’ve been developed by different companies who have all put their own patent on their own drugs. As a result, the only two new drugs developed in the past 50 years remain out of reach of most patients. There is not sufficient evidence or research to recommend them for widespread use nor is there any evidence on how to use them as part of a novel regimen.

“As an organisation of doctors, nurses and other medical staff we have been left feeling frustrated as thousands of our patients continue to suffer from these long, toxic and failing treatments. Finally we had had enough, so we decided to do something ourselves to find better treatment for our patients.

“We wanted to run a trial that would put patients’ at its heart, with a focus on those people most in need of improved treatment, and where the outcome would have a real impact on people’s lives. We decided to trial a combination of drugs that only need to be taken for six months, with no daily painful injections, fewer pills, that hopefully has more bearable side effects and is potentially much more effective at curing all drug-resistant forms of TB.

“After years of hard work, TB PRACTECAL started at the end of January 2017 in Uzbekistan. It is a full phase III clinical trial with four sites across Uzbekistan, Belarus and South Africa. The 630 participants will either take a combination of two new anti-TB drugs (bedaquiline and pretomanid) with three other existing drugs (linezolid, clofazimine and moxifloxacin), or be placed in the control group.

“When the first patient took the first pill of the trial this felt like an exciting and important milestone. But the reality is we still have a long way to go.

“A first set of trial results are expected within two years and final results within four. If successful, this combination of treatment could be recommended for widespread use by the World Health Organisation and rolled out in countries across the world.

“But even if this trial is successful, while we hope it could have a real impact on the lives of some people with drug-resistant TB, it will be just a small step forward in the efforts to tackle this global epidemic.

“For a start, we will need an ongoing ‘pipeline’ of new drugs to replace the older ones as they become less effective. MSF has also initiated the 3P Project which aims to create a new, more collaborative approach to funding and developing TB drugs, as the current model is clearly not working.

“Of course, MSF cannot solve the TB crisis alone. We also need better diagnostic tools that are also accessible to those who need them most. We need improved ways of developing individual drugs and quicker ways of combining them into regimens. We need combinations that are suitable for children, and all treatment needs to be affordable. With thousands of people dying from TB each day, there needs to be a global response to this global crisis.”

The Guardian report

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