Skip links

A drug development model for the diseases the world forgot

Eschewing profit, over the past 20 years researchers at the Drugs for Neglected Diseases initiative have developed a dozen drugs accessible to the world’s most forgotten patients.

A Visceral Leishmaniasis patient recovering, in Kacheliba, Kenya, 2017. VL is fatal if left untreated. For decades, patients suffered under a toxic drug regime; more recently, scientists at the DNDI have developed a simple, safe 14-day combination treatment. Photo courtesy: DNDI.

By Monique Wasunna

August 24, 2023

Twenty years ago, I travelled to Gedaref State in eastern Sudan. What I witnessed there deeply moved me and strengthened my resolve to contribute to medical innovation and improve treatments for people affected by a deadly and neglected disease called visceral leishmaniasis (VL).

In remote areas I visited, families with members affected by VL found it extremely difficult to reach Kassab Hospital, the nearest facility where diagnosis and treatment were offered. This fatal parasitic disease is prevalent in eastern Africa and causes weight loss, spleen and liver enlargement, anaemia, and intense fatigue – and kills 95% of affected people if left untreated. With 50,000 to 90,000 new cases reported worldwide annually, eastern Africa remains the world’s most affected region.

Due to the inadequate number of healthcare workers at Kassab Hospital, even those who managed to get there suffered financial losses from having to take weeks off work to stay with their sick loved ones. Compounding the issue, the primary treatment for the disease at the time, sodium stibogluconate (SSG), was a highly toxic drug that required 30 days of injections.

I am happy to say that today, the situation has improved significantly. After several years of medical research in Sudan and other endemic countries, the treatment duration for VL has been reduced to 17 days. Furthermore, the results from our recent study demonstrated that a 14-day combination treatment was equally effective, eliminating one painful daily injection and the potential toxicity associated with SSG. The World Health Organization (WHO) is currently reviewing this data.

This incredible success was achieved through non-profit drug development.

Neglected tropical diseases, such as visceral leishmaniasis, primarily affect people in the world’s poorest regions. Unfortunately, commercially driven medical research tends to overlook these populations because they are believed to lack the financial means to constitute a lucrative market for the traditional pharmaceutical industry. Drug development today is primarily skewed to areas with the greatest commercial return rather than those with the greatest public health needs.

But the alternative model that my organization, the Drugs for Neglected Diseases Initiative (DNDi), and our partners have pioneered since 2003 focuses on areas of the greatest need rather than the greatest profit. This means that we prioritize the development of treatments for diseases that have a high impact on public health, even if they are not directly profitable for the companies that develop them.

As I witnessed the suffering of patients with leishmaniasis in Kassab and later in other countries, it became clear that there was a need to work together. Collaborating with stakeholders from Kenya, Sudan, Ethiopia, and Uganda, we formed the Leishmaniasis East Africa Platform (LEAP).

The objective of LEAP was to facilitate clinical testing and registration of new treatments for VL in eastern Africa and ensure sustainable access to these treatments. In Sudan, LEAP’s impact has been significant; it led to successful clinical trials to test new and better medicines with partners at research facilities in Kassab. Similar efforts have been carried out in Ethiopia, Kenya, and Uganda.

Thanks to these efforts, DNDi gave hope to thousands of patients by delivering two shorter, safer treatments for VL in eastern Africa in 2022, including a drug combination for people living with HIV and VL.

This success story is not an isolated case. Since its creation 20 years ago, my organization has successfully developed 12 new treatments for six deadly neglected diseases, showing concretely that a not-for-profit model of drug development can work to improve the lives of millions of patients.

With researchers from all over the world working in partnership to find new treatments, this model encourages collaboration rather than competition. If a new molecule shows promise, we determine if it can be developed and produced at low cost. We put it through clinical trials to demonstrate its safety and effectiveness, have it registered in endemic countries, and ultimately work with partners to make sure it’s accessible to those who need it.

Partners in the Global South, where many neglected diseases are endemic, play a significant role in shaping R&D priorities by providing input in the most pressing diseases – the areas where research is needed – and the interventions that are most likely to be effective.

Teaming up with partners ensures that the research process is more affordable and that the treatments we develop are accessible to everyone, regardless of their socioeconomic status.

A textbook example is our work in the Democratic Republic of Congo, Guinea, and Malawi to find better treatments for sleeping sickness, a deadly disease. Twenty years ago, the only treatment widely available for stage two of this disease was an arsenic derivative so toxic that it killed one in 20 patients. In 2018, DNDi and its partners delivered fexinidazole, the first simple, safe, oral treatment for the most common form of the disease. We are now finalising the development of acoziborole, an all-new, single-dose oral drug that has the potential to eliminate sleeping sickness as a public health threat – for good.

Thanks to our partners, the dedication and hard work of clinicians, doctors, and nurses, and the active involvement of communities, we have successfully conducted complex clinical trials to the highest standards in remote regions that are sometimes shaken by conflict – all that at a fraction of the cost of traditional pharmaceutical research.

Our not-for-profit drug development model encourages open science, including – whether positive or negative. We have also used our experience to advocate for policies and political commitments to ensure everyone can benefit from scientific progress.

This model has built the capacity of African scientists like me to spearhead the critical mission of finding innovative treatments for neglected diseases that disproportionately affect our region. And we are not the only ones: other non-profit medical research organizations are also delivering better treatments for the most neglected populations – such as people affected by tuberculosis – and demonstrating that alternative models of drug development work.

Twenty years after my initial visit to Gedaref State, the situation has improved significantly. Currently, DNDi is supporting two VL treatment centres in Doka and Um el Kheir, where patients affected by the disease can now access improved treatments.

Yet, there is still much work to be done: we need to develop 100% oral medicines to make treatment even simpler for patients. But based on our previous successes in non-profit medical research, I’m confident that we can deliver them.

For the original press release, please click here.