MSF tuberculosis clinical trial finds shorter, more effective, and safer treatment

Addressing the ocean of gaps in the treatment of tuberculosis in children and adolescents

India 2021 © Atul Loke/MSF

New results show a much shorter treatment regimen for drug-resistant TB is superior to current options

NEW YORK/LONDON, OCTOBER 20, 2021—A new all-oral, six-month treatment regimen is safer and more effective at treating rifampicin-resistant tuberculosis (RR-TB) than the current options, according to results from TB PRACTECAL, the first-ever multi-country, randomized, controlled clinical trial of this regimen, announced Doctors Without Borders/Médecins Sans Frontières (MSF) today during the 52nd Union World Conference on Lung Health.

These results signal the start of a new chapter for people with drug-resistant (DR-TB) who currently face lengthy and ineffective treatment regimens of up to 20 months that can include painful injections and up to 20 pills a day that can cause severe side effects.

“When we embarked on this journey, patients with DR-TB around the world were facing lengthy, ineffective, and grueling treatment that disrupted their lives,” said Bern-Thomas Nyang’wa, MSF medical director and chief investigator of the trial. “Patients were telling us how hard it was to adhere to treatment, but little progress was being made to find kinder treatments because diseases most prevalent in low- and middle-income countries don’t attract pharmaceutical investment. So we were compelled to pursue new treatment options ourselves. These results will give patients, their families, and health care workers worldwide hope for the future of DR-TB treatment.”

MSF intends to publish the trial results in full in a peer-reviewed journal later this year. The international medical humanitarian organization is also sharing the data with the World Health Organization (WHO) ahead of the WHO DR-TB treatment guideline review with the hope of influencing national DR-TB guidelines and improving the treatments that the approximately 500,000 people who fall sick each year with multidrug-resistant TB (MDR-TB) are given.

Launched in January 2017, TB PRACTECAL tested a six-month regimen of bedaquiline, pretomanid, linezolid, and moxifloxacin (BPaLM), against the locally accepted standard of care. The trial enrolled 552 patients aged 15 years and older, of which 301 were included in these results. The trial took place in seven sites across Belarus, South Africa, and Uzbekistan.

The phase II/III clinical trial found that the new shorter treatment regimen was very effective against RR-TB; 89 percent of patients in the BPaLM group were cured, compared to 52 percent in the standard of care group. Four patients died from TB or treatment side effects in the control group, while there were no deaths among patients on the new regimen. Additionally, trial results showed that the new drugs lead to a significantly lower rate of major side effects, with 80 percent of patients avoiding any major side effects compared to 40 percent in the control group.

“[The shorter treatment] would mean a lot as I think when you are on treatment, some parts of your life feel like they are put on hold,” said Awande Ndlovu, who was enrolled in the trial at the THINK Hillcrest Clinical Trial Unit in South Africa. “Before [the trial] gave me hope, I couldn’t even see the slightest glimpse of recovering from MDR-TB.”

MSF and its TB PRACTECAL partners continue to provide care and check-ups for patients who are finishing their treatment in the trial, with the last patient follow-up scheduled for summer 2022.

MSF hopes these results will serve as a major contribution to the growing body of evidence for global treatment recommendations to be updated to include a short, effective, and safe treatment regimen. Ultimately, MSF believes these results prove that a change in clinical practice is now due. Additionally, MSF plans to work closely with National TB Programs, Ministries of Health, and other key stakeholders to ensure that this treatment is available as soon as possible to patients.

“MSF is committed to providing TB care and advocating for effective and affordable treatments,” said Dr. Christos Christou, MSF international president. “Last year, our teams helped 13,800 people start on TB treatment, including 2,100 with DR-TB. As one of the largest non-governmental providers of TB treatment worldwide, we are excited about what these results will mean for those with MDR-TB. We’d like to extend our huge thanks to the staff and patients who have been so committed to this trial. Thanks to every one of them, we have new evidence for drastically shorter, effective, safer, and kinder treatments that the TB community have long waited for.”

MSF is one of the largest non-governmental providers of TB treatment worldwide. In 2020, MSF started 13,800 people on TB treatment, including 2,100 with DR-TB. The TB PRACTECAL trial complements the endTB clinical trial, also sponsored by MSF, which aims to provide additional high-quality evidence on shorter, all-oral regimens.