Drugs for Neglected Diseases initiative

Dr. Monique Wasunna is the Director of the Drugs for Neglected Diseases initiative Africa Regional Office. She is a physician and infectious disease and tropical medicine specialist. She is the Founding Chairperson of the Leishmaniasis East Africa Platform, which promotes clinical research and capacity strengthening for this deadly neglected disease. Prior to joining DNDi, Dr. Wasunna worked at the Kenya Medical Research Institute where she rose from the position of an Assistant Research Officer to that of the Acting Director and Chief Executive Officer. She holds a Bachelor of Medicine and Surgery degree from the University of Nairobi, an MSc and a PhD in medicine from the London School of Hygiene and Tropical Medicine, and a diploma in Tropical Medicine and Hygiene from the Royal College of Physicians of London. She is a former member of the International Bioethics Committee of UNESCO. She is well published in peer reviewed journals.  

DNDi is committed to developing effective drugs for diseases affecting millions of people in low- and middle-income countries overlooked by the traditional pharmaceutical industry because there is no profit to be made. For leishmaniasis, a fatal parasitic disease prevalent in my part of the world (Eastern Africa), Asia, South America, and the Mediterranean, we have developed improved treatment recommended by the World Health Organization. We’ve built clinical trial capacity in regions that had none, with the Leishmaniasis East Africa Platform, which I co-founded. We’ve renovated clinics and trained staff, in South-South collaboration, Kenya and Sudan investigators helping Uganda and Ethiopia develop capacity to conduct clinical trials to the highest international standards, supported by DNDi. This has helped with our COVID-19 trials – the sites are ready. How do we elevate humanity? By promoting the idea that scientific advances must benefit the most neglected communities. This elevates us all.

Only 1% of the 1393 new drugs approved between 1975 and 1999 were for neglected diseases, and the ratio has improved little since. That’s why DNDi was founded in 2003 by Doctors Without Borders, the Kenya Medical Research Institute, and others – because there were few effective treatments for many diseases exclusively or predominantly affecting the poor. Millions of people with life-threatening diseases still face treatment options that are sub-optimal, even toxic, and I have seen far too many patients suffer or die because we don’t have the right treatments for their diseases. For visceral leishmaniasis, my lifelong passion, 600 million people are at risk, with 20,000 to 30,000 people dying from it annually. Treatment options are either toxic, costly, logistically impractical and/or are seeing an increase in resistance in hard hit areas. Each year sees 50,000 to 90,000 new cases globally, with 50% of all cases children in the most affected countries. For people living with HIV, the risk of contracting leishmaniasis is 2,000 times higher and treatment has to be carefully adapted to treat them. We’re developing new chemical entities that can become simple oral treatments – today people are hospitalized for 17 days and receive painful injections.

Thanks to DNDi’s research, people with leishmaniasis are no longer hospitalized for 30 days for treatment. We conducted clinical trials, with partners, using different combinations of existing drugs in Sudan, Ethiopia, Uganda, and Kenya, and made treatment shorter, safer, and more effective. Now we’re developing a new generation of treatments for this devastating parasitic disease. We’ve discovered entirely new chemical entities, replenished the pipeline, and run clinical trials for improved treatment. We’ve completed more than ten trials and are on the cusp of developing an oral treatment, which would be revolutionary. We’re also helping make sure resistance doesn’t develop by using two drugs, not one, crucial because when you have so few drugs you have to be careful that resistance does not develop. Once we start trials for the oral treatment we will need to retrain staff at the different clinical sites because it will be a whole new approach, to make sure we conduct the vigorous, intensive, meticulous trials we always conduct. For DNDi as a whole, in our 16 years of existence, we have developed eight treatments for five deadly diseases - malaria, sleeping sickness, pediatric HIV, Chagas disease, and leishmaniasis – saving millions of lives.

Leishmaniasis affects the poorest of the poor, primarily in remote rural areas. The patients I see in Eastern Africa are mostly nomads and pastoralists (Kenya, Sudan, Uganda) who follow water sources with their cattle and set up camp in different places, making organizing clinical trials particularly challenging. 70% of patients overall are under 15 years old. In Ethiopia, people affected by leishmaniasis are mainly migrant workers. Patient needs are at the center of what we do in DNDi. Priority setting involving communities is key in helping us understand their needs. Some of the village chiefs have been treated by us for leishmaniasis. They have become a voice for the communities that are voiceless and marginalized. Community engagement before, during, and after the clinical trials is impactful. The community sees in our eyes that we’re honest. Some communities feel they’re in a different country – they see us well-dressed, they’re extremely poor, suffering from this, and other diseases for a long time. They have seen us renovate clinics, treat and cure their families and community members, so they know we’re there to do good. But we want to do better and innovate a simple pill to treat this disease. 

People suffering from neglected diseases have been left behind for decades – by the pharmaceutical industry, governments, even their own communities, where stigma can lead to ostracization. When it comes to leishmaniasis, there have been many epidemics in the past in which thousands of people have died, yet because they are poor, there has been no interest in developing a cure on the part of the pharmaceutical industry. Without treatment infected patients die. People with a cutaneous (skin) form of the disease are shunned by communities and that’s a form of the disease we’re also focusing on.

DNDi was created in 2003 by Doctors Without Borders/Médecins Sans Frontières (MSF), the Kenya Medical Research Institute (KEMRI), and several other public research institutes to fill a gap in pharmaceutical R&D – treatments for patients with neglected diseases. The idea began within MSF, in the form of a Neglected Diseases Working Group, made up of doctors frustrated at the lack of medical tools at their disposal for many of the diseases they were encountering in the course of their work. I was working for the Kenya Medical Research Institute at the time, with a focus on leishmaniasis, and I was equally frustrated by the lack of treatment options for my patients. When I was approached by the MSF working group to help with the creation of a not-for-profit research and development institution that would put patients at the heart of medical innovation, I quickly came on board. I was the KEMRI liaison to DNDi, and instrumental in KEMRI’s decision to become a founding partner. I set up DNDi’s first meeting in Africa with more than 70 organizations from Africa in attendance, co-launched the Leishmaniasis East Africa Platform, and made sure that DNDi prioritized clinical trials for leishmaniasis in its portfolio. 

I was born in Uganda to a Ugandan father and a Rwandese mother. While in high school, my younger sister became seriously ill with measles and doctors said she was going to die. As I watched my terrified mother take care of her, I saw the fear and pain in her eyes, I promised myself to work hard and become a doctor. My father had a decent job but all around us there was so much poverty. Children walking around in rags and sleeping on empty stomachs. High infant mortality and under five survival was poor. Malaria, tuberculosis, soil-transmitted helminths, trachoma, and other infectious diseases were on the rise. I received a government scholarship to study medicine at the University of Nairobi Medical School. My sole aim was to get knowledge and skills that would equip me to help my mother and our people. In my first job, I admitted a child who was very sick with visceral leishmaniasis and needed specialist treatment. Unfortunately, he died in my arms as I was taking him for specialist review. This moment has never left me. I dedicated my life’s work to leishmaniasis and neglected diseases affecting poor, voiceless, marginalized communities in Africa.

As a member of a humble family of 12 siblings from Uganda, I was one of only 10 students sponsored by the Ugandan government to study medicine at the University of Nairobi in 1975. After concluding an  internship at the Kenyatta National Hospital, I was employed by the Kenya Medical Research Institute (KEMRI) and received grants from the Special Programme for Research and Training in Tropical Diseases of the World Health Organisation (WHO/TDR) to pursue a master’s degree and then a PhD at the London School of Hygiene and Tropical Medicine. As a member and then Director of the Centre for Clinical Research at KEMRI, I became actively involved in various infectious diseases clinical trials, such as for HIV/AIDS, tuberculosis, and malaria, and especially visceral leishmaniasis. Since 2003, initially as the head of the Drugs for Neglected Diseases initiative (DNDi) Africa Liaison Office within KEMRI and then as Director of the Africa Regional Office in Nairobi, I have not only been leading the implementation of DNDi’s leishmaniasis strategy in the region but have also been actively involved in research that has enabled the development of much needed improved treatments for the disease. I was the founding Chair of the Leishmaniasis East Africa Platform, created to bring together specialists and practitioners from across the region to promote clinical research and capacity strengthening for this neglected and deadly disease. I also lead a team of 40 that is conducting policy, communications, networking, and managing clinical trials into mycetoma, pediatric HIV, and leishmaniasis.

When we started the Leishmaniasis East Africa Platform (LEAP) we were a group of academics, researchers, people from a background of “publish or perish.” There was tension as to who should be the first author on papers published. Initially there was something of a clamor, and we had to sit down and discuss it. As the founding chair, it was my responsibility to steer the debate in the right direction. I was able to bring us all to the reason we do this work in the first place – for our patients, not for publication. I reminded people we were blessed to be where we are. I had many talks with different members of the team, privately and in groups, and eventually people saw the light and it became easier to decide who should lead on which projects. Instead of disintegrating because of in-fighting over who gets which papers, we focused on the need, becoming more consultative, turning it all around to put the people who are suffering at the heart of everything we did. We overcame diverse cultures, religions, and backgrounds to deliver innovation for our patients.

My father believed in his daughters; we were equal to boys in his eyes. He ensured we all had opportunities. In Africa, it was difficult for a woman to get a leadership role. You had to work a hundred times as hard as men to prove yourself. I believe I am a role model because I have been a woman leader in science and the Acting Director of Kenya’s leading research institute, showing women and girls they can be leaders too. I was one of the 36 independent experts on the International Bioethics Committee of UNESCO for eight good years, just one of two African women then in the world contributing to this international group looking at life sciences and its applications. We held many public debates and wrote many books together, and I am proud to have helped the scientific world with my expertise. I am proud of the trust that was put in me to be able to steer the science in an ethical way. This is an experience that will last a lifetime – one that I hope will be followed by many more African women in the future.

For leishmaniasis, it is innovative to run clinical trials to international standards in the remotest of African settings. Through this work, we came up with a treatment that has been recommended by WHO as the treatment of choice for first-line therapy. For DNDi as a whole, our R&D model is innovative because we are partnership-based, bringing together partners to work toward a common goal, in a field where competition, not collaboration, is the norm. We make sure that any drugs we develop are accessible to patients – affordable, approved by the relevant country regulatory authorities, and are easy for health care workers to administer and for patients to take. We’ve created clinical research platforms – like the Leishmaniasis East Africa Platform – for several diseases, to bring together experts and practitioners in a collaborative network to share advances and advocate for improvements, conduct trainings, and renovate facilities. We make sure the fruits of our studies are open-source – and have initiated projects with pharmaceutical laboratories where compound libraries are shared, speeding up the process of discovering potential new treatments. This is all unique in the field of medical R&D, disruptive simply by its not-for-profit and collaborative nature, and illustrating that drugs do not need to cost astronomical amounts if the profit factor is taken out of the equation and collaboration toward a common good is the main motivator. We’re currently co-launching clinical trials in 15 African countries into treatments for COVID-19, in the hope of reducing hospitalization rates seen elsewhere.

My work has an impact on humanity by saving lives. Neglected diseases affect mostly vulnerable and marginalized people – in remote and unstable settings who do not represent a lucrative ‘market’ for the pharmaceutical industry, and who are therefore overlooked by pharmaceutical research and development (R&D). They have a profound impact in terms of disease burden, quality of life, loss of productivity, and aggravation of poverty, as well as the cost of long-term care for those whose illnesses leave them disabled for life. DNDi works at all ends of the drug R&D process, from discovery to delivery, from bench to bedside. DNDi’s primary activities are to: 1) deliver a total of 25 new treatments by 2025; 2) utilize and strengthen existing research capacity in disease-endemic countries; and 3) raise awareness about the urgent need to develop new drugs for neglected diseases and advocate for increased public responsibility. Some immediate outcomes include: the delivery of improved treatments using existing drugs that provide rapid, tangible benefits for patients; upgrading of clinical trial sites either by creating data centers, providing equipment (beds, medical supplies, microscopes), or by improving the necessary infrastructure; and training researchers and building the skills of the local community. DNDi ultimately aims to reduce overall morbidity and mortality in the most affected communities via breakthrough treatments, sustainably building the capacity of local communities, and ultimately delinking the cost of investments in R&D from the price of the end product.

I will talk about two projects we work on from our regional office – leishmaniasis and mycetoma. Eastern Africa harbors the highest burden of Visceral Leishmaniaisis (VL) worldwide with 30,000 to 40,000 new cases every year. VL affects mainly poor communities in remote rural areas and arid regions. The majority of patients are children except North Ethiopia, where the disease affects mainly young male adults in work-related settings. Across our clinical trials sites in eastern Africa, our project treats about 3,500 people/year (those participating in the clinical trials and those treated outside the clinical trials), 10,500/3 years and 17,500/5 years depending on the climatic changes and epidemic outbreaks. Educating the community about the diseases and the need to seek treatment involves many people in the endemic areas in Ethiopia, Kenya, Uganda, and Sudan where we work. It is not possible to estimate these numbers. The impact of our activities in the disease endemic countries of eastern Africa is high. We have delivered new treatments and influenced policy change and national guidelines review in Ethiopia, Kenya, Uganda, and Sudan to include the new treatments we delivered. Our project also involves finding treatment for mycetoma patients through doing clinical trials. Global burden of mycetoma is not known, but the disease is endemic and has been reported from countries in Africa, especially in Sudan. We treat about 100 patients/year, 300 patients/3 years and 500patients/5 years. Most patients seek treatment from traditional healers. A lot of health education is required in these remote areas.

We plan to have an oral treatment for leishmaniasis in Eastern Africa in the next five years in a form that is safe and easy to use and that will be a combination of two drugs, so that we can protect patients from the threat of drug resistance. The hope is to deliver a product that can be taken at home, removing the need for prolonged hospital stay, which is extremely difficult and costly in terms of time lost to work and family. The treatment itself is also very painful, either intramuscular or intravenous injections with heavy metal-based drugs, with infusions taking up to two hours. It is just an awful way to treat a disease, and we need to do better by these patients and deliver a simple pill – as we have successfully done for sleeping sickness, a devastating disease that for years was treated with a drug so toxic it killed one in twenty patients, and today we have a simple course of pills, thanks to DNDi and partners. We will also start trials into treatment for cutaneous leishmaniasis, a form of the disease that disfigures, and creates a lot of stigma, especially for women who get it on their faces. Overall, DNDi aims to have developed a total of 25 treatments by 2025. We are halfway there and are currently in the midst of a review of our business plan which will likely make additions to our portfolio, including the scourge of snake bites.

The major obstacle to our work right now is to secure sustainable funding sources in light of the COVID-19 pandemic, which has affected the current global health landscape in several ways. An enormous amount of money has been devoted to COVID-19 research, with a lot of funders pivoting their funding to support efforts to find a vaccine or treatment, thereby affecting the capacity of those same donors to fund neglected tropical diseases. A second major obstacle for our work also relates to funding. It is not a new challenge, but it has been exacerbated due to uncertainties created by COVID-19, and that is the ability to secure long-term funding. Developing drugs takes many years and the challenges of securing sustainable longterm funding have become more acute in this new climate. A third challenge is the long-term engagement of our industrial partners. DNDi partners with a range of players to develop drugs, and the pharmaceutical industry is key as a manufacturing partner. We are seeing a worrying trend among these partners in the bigger pharmaceutical companies to steer away from neglected tropical diseases, even in this partnership-based model. Finally, DNDi is launching COVID-19 clinical trials with primarily African partners, recruiting between 2,000-3,000 patients in at least 20 clinical sites across more than 10 African countries. This has created unexpected and immediate funding needs, along with the measures put in place to ensure the safety of our patients and teams, which requires additional funding for PPE and other materials.

Our response to our first two challenges is to pursue diversification of funding, including among private partners, an area the Elevate Prize opportunities could help with. We are looking for partners who share our values, who believe that patient needs, not profits, should drive R&D research, and that people in the world’s poorest parts of the world should reap the benefits of the best that science has to offer. Partners who can see the long view and understand the complexity of drug development. We have many funders who fit this profile and believe there are many more we have yet to reach. In a strange way, the COVID-19 pandemic is helping to make people more aware of the complexities of developing drugs, and we believe there will be an openness in new partners to explore supporting DNDi. When it comes to the case of industrial partners, we are developing new relationships with regional pharmaceutical producers so that we do not rely on the big companies, and can create capacity to produce drugs that way – particularly in regions affected by the diseases we work on. Finally, the total estimated budget for DNDi’s COVID-19 response is $45 million (2020-2023) period. DNDi has secured $17 million from the German government in total for its COVID-19 response so far and is in discussion with key existing donors for approximately $18 million more. DNDi is seeking a remaining $10 million in funding – with an immediate need of $3 million from the philanthropic community.

DNDi is a partnership-based organization and we cannot succeed without a wide range of public, private, academic, not-for-profit, and philanthropic partners. Globally, DNDi works with more than 180 R&D partners, including our founding partners, the Institut Pasteur (France), the Fundacao Oswaldo Cruz (Brazil), the Kenya Medical Research Institute (Kenya), the Malaysian Ministry of Health (Malaysia), the Indian Council of Medical Research (India), Medecins Sans Frontieres, and the World Health Organization’s Special Programme for Research and Training in Tropical Diseases. We have created the COVID-19 Clinical Research Coalition with 150+ partners to accelerate research in those areas where the virus could wreak havoc on already-fragile health systems and cause the greatest health impact on vulnerable populations – and are co-launching clinical trials with several primarily African partners into treatment, including here in Kenya. In East Africa, we work with 20 partners through the Leishmaniasis East Africa Platform. These are local research institutions, universities, national ministries of health and drug regulatory authorities from Kenya, Sudan, Ethiopia, and Uganda as well as Médecins Sans Frontières and WHO.  For our mycetoma program we work with 7 partners. For our pediatric HIV program, we work with 30 partners, including research institutions, ministries of health, civil society, the pharmeceutical industy, implementing partners, and universities.

DNDi’s approach to innovation stresses collaboration over competition and promotes openness and transparency to facilitate the greatest possible sharing of research knowledge, data, and costs. Our alternative model of R&D is based on partnerships – with scientists, and others in government; academia; the pharmaceutical industry; NGOs and civil society; and philanthropy. In all that we do, we put patients first, as we create medicines that are safe, effective, affordable, and adapted to the needs of the people and places most in need. We construct or upgrade laboratories and clinics, train staff, and facilitate technology and knowledge transfer to manufacturers in neglected disease-endemic countries, encouraging south/south scientific cooperation. 

DNDi conducts state-of-the-art clinical trials in some of the world’s remotest regions to the highest of international standards. At any given time, we are running roughly 20 clinical studies for several diseases in 59 sites in over 20 countries. We have also created the most robust research pipeline ever for some of the world’s most neglected diseases, which includes 20 potential new chemical compounds. Our model is proof that alternative approaches to R&D that place patients’ needs – not profit maximization – at the heart of medical R&D are possible. 

DNDi carries policy analysis and advocacy at global, regional, and national policy fora and joins with partners around the world to ensure the broadest possible sharing of research knowledge and data and guarantee that all new health technologies are affordable, available, and accessible for all.  

To allow for the greatest flexibility in decision making needed for the R&D portfolio management strategy, and to allow greater independence in its operations, DNDi’s priority is to raise unrestricted core funding versus project-specific or earmarked funding. One of the most important ways in which DNDi secures its independence is through diversification of funding to prevent unhealthy influence by or dependence upon any single donor. This is why DNDi’s funding policy, as established by its founding partners in 2003, in addition to diversifying funding sources, also seeks to maintain a balance of public and private support, to minimize as much as possible earmarked donations, and to ensure that no one donor contributes more than 25% of the overall budget. DNDi seeks diversified sources of funding – cash contributions, in-kind contributions, grants, sponsorships, and legacies – from individuals, governments, public institutions, companies, foundations, NGOs, and alternative mechanisms that share a commitment to DNDi’s vision and mission. There is also a strong advocacy component to our financial sustainability. We advocate for neglected diseases and patients to be included in the global health agenda to stimulate funding from both public and private donors for initiatives benefiting neglected diseases and patients.

DNDi is supported by a range of donors. In 2019, our organization raised funds in the form of charitable grants from various private and public funding sources: 

- UK government (UK Aid) = $21,672,158.12 

- Bill and Melinda Gates Foundation = $8,500,734.60 

- Médecins Sans Frontières = $6,678,600.25 

- Dutch Government DGIS = $3,033,093.60 

- French Government AFD/MEAE = $780,545.29 

- German Government BMBF through KfW = $2,471,409.60 

- Swiss Government SDC = $2,834,923.62 

- GHIT Fund, Japan = $4,401,610.83 

- Wellcome Trust, UK = $2,629,428.16 

- European Union EDCTP = $3,002,732.33 

- Unitaid = $940,957.75 

- Takeda Global CSR Programme = $691,272.36 

- Fundación Mundo Sano (Priority Review Voucher) = $1,651,106.07 

- US Government NIH/NIAID/USAID = $825,259.83 

- Various other donors = $643,342.74 

- Medicor Foundation, Liechtenstein = $100,000 

- Republic and Canton of Geneva = $204,550.71 

- Pharmaniaga, Malaysia = $1,102,209.36 

- Starr International Foundation, Switzerland = $150,508.84 

- IDRC Canada = $22,547.12 

DNDi has also received recognition through numerous awards, including the Rockefeller Foundation’s 2013 Next Century Innovators Award, the 2013 Carlos Slim Health Award for Outstanding Institution, the Bill & Melinda Gates Foundation’s 2014 Innovative Fund Award, the Drug Information Association’s 2016 President’s Award for Outstanding Contribution to Global Health, and the 2017 Geneva Innovation Prize in recognition of DNDi’s innovative and collaborative model. 

In 2020, DNDi seeks to raise an additional $5 million to complete its priority projects. For the next phase of DNDi’s business plan (2021-2028), DNDi seeks to secure $683 million in grants to finance its portfolio of projects. Of that, $86 million has already been secured and we are therefore looking to secure $597 million both from existing and new public and private donors. During the last 15 years DNDi successfully attracted more than $700 million USD in grants to fund its portfolio of activities. DNDi is looking for donors that are interested in accompanying the long process of drug development, while staying focused on the interest of neglected patients.

In 2020, DNDi’s initial budget was $72 million but this had to be revised due to the COVID crisis. Like many of our peers, our organization had to revise its budget at the beginning of the second quarter of the current fiscal year. As our main objective is always the well-being of our patients, we had to integrate the shift in our clinical work to include potential delays in activities, milestones and deliverables, the need for appropriate equipment and our newly created COVID-19 program, all to ensure the continuity of our projects in the long run. DNDi’s revised budget for 2020 is now $65 million. 

I am applying for the Elevate Prize with a certain amount of humility but with a great awareness that I am one of very few African women in leadership positions in the world of medical R&D. I would love to be able to provide inspiration and mentorship to younger women embarking on medical and scientific careers, who can say, Yes, I can do that, too! I would also like to take advantage of the opportunity to raise awareness about our work in Eastern Africa to my fellow Africans for identifying new funding sources, partners, and visibility opportunities for my team and our patients. Personally, I would benefit enormously from the mentorship and training offered – particularly the media and marketing trainings to help raise my social media profile and that of DNDi’s work. I recently published an Op Ed in the Guardian talking about the importance of Africans driving research in Africa, and I would use the opportunity of the Elevate Prize to further this point. And of course, the $300,000 that the Elevate Prize offers will be immediately applied to our lifesaving programs to bring the best science to the most neglected.

I would be interested in mentorship and coaching on fundraising and networking, with the African market in mind. As one of so few African women in leadership roles in the world of science, an elevated social media platform could go a long way to both inspiring young women and bringing the message of DNDi to a wider audience: that people with neglected diseases should receive the best science has to offer, no matter who they are or where they live. And while we are not a personality-driven, but rather issue-drive organization, I do understand that people connect to issues through other people, and that in order to humanize our work, we need to humanize our workers, and I would use such a platform to bring attention to the remarkable work of my colleagues and the resilience and needs of our patients.

As a partnership-based organization, DNDi has more than 180 partners in the field of R&D and thrives on partnerships. I would be happy to discuss what opportunities might exist for collaboration – on the scientific level, advocacy level, and linking us to potential new funding opportunities with entities with an interest in open-source science, bringing the best science to the most neglected, and putting patients before profits. On a personal level, I look forward to networking and mentoring opportunities particularly when it comes to media training and profile-building on social media so that I can be a visible role model for young African women and a voice to end the neglect of patients with leishmaniasis and other neglected diseases affecting my continent and beyond.