Rome/Geneva – Today at the 6th International AIDS Society (IAS) Conference on HIV Pathogenesis, Treatment and Prevention, the Drugs for Neglected Diseases initiative (DNDi) announced the launch of a new drug development programme to address critical unmet treatment needs of children with HIV/AIDS.
Because HIV transmission in young children has largely been eliminated in high-income countries due to effective prevention of mother-to-child transmission (PMTCT) interventions, little market incentive exists for pharmaceutical companies to develop antiretroviral (ARV) drugs adapted for children. The World Health Organisation (WHO) recommends immediate antiretroviral therapy (ART) for all HIV-positive children less than two years old, but the safety and correct dosing of key ARVs have not been established in very young children, and appropriate child-adapted formulations do not exist.
Current paediatric ARV formulations are unpalatable for these children, are impractical for caregivers due to multiple liquid preparations that have to be adjusted according to weight, and have undesirable interactions with tuberculosis (TB) drugs.
“There are millions of children with HIV/AIDS in low- and middle-income countries, but their needs are absent from the HIV research and development agenda, and this is largely because they are poor and voiceless and do not represent a lucrative market,” said Dr. Bernard Pécoul, Executive Director of DNDi. “Working with partners, we hope to help fill this terrible gap and offer improved treatment options for children with HIV/AIDS.”
Last year, Médecins Sans Frontières/Doctors Without Borders (MSF), UNITAID, and other organisations called upon DNDi to apply its expertise to paediatric HIV/AIDS based on its track record in delivering new medicines for neglected diseases. DNDi, a not-for-profit R&D organization, develops new treatments for neglected patients suffering from sleeping sickness, leishmaniasis, Chagas disease, and malaria.
“Our medical teams in the field have for years faced difficulty treating young children with HIV due to the lack of appropriate treatment tools,” says Dr. Unni Karunakara, International President of MSF. “We will do everything possible to accelerate the development of and access to improved formulations for children with HIV/AIDS, and look forward to being able to introduce better, affordable medicines to treat HIV-positive babies and children where we work.”
After an in-depth needs assessment and consultation with experts – including those from endemic countries such as South Africa, Uganda, Cote d’Ivoire, and Thailand, and from public sector research institutions such as the US National Institutes of Health, UK Medical Research Council, and Agence Nationale de Recherche sur le Sida in France – ideal specifications for improved treatments were developed. There is consensus around the need to develop an improved first-line protease inhibitor-based regimen for children under three years of age, irrespective of prior exposure to ARVs, and this will be DNDi’s first priority.
Ideally, this new first-line paediatric HIV therapy needs to be easy to administer and better tolerated by children than current drugs, as well as heat stable, easily dispersible, and dosed once daily or less. It must also carry minimal risk for developing resistance and be suitable for infants and very young children, with minimum requirements for weight adjustments. Finally, any new formulations must be compatible with TB drugs, and, importantly, affordable.
“Paediatric HIV has indeed been a neglected area for innovation in drug development," said Dr. Gottfried Hirnschall, Director of WHO’s HIV/AIDS Department. "Children's access to HIV treatment has been low with only 28% of the children in need of HIV treatment receiving it at the end of 2009. WHO appreciates DNDi's focus on new paediatric HIV treatments, and we look forward to working together to deliver more and better medicines for children in need.”
DNDi’s paediatric HIV programme will be led by the newly appointed Marc Lallemant, MD, formerly head of the Programs for HIV Prevention and Treatment (PHPT), a clinical research consortium of Chiang Mai University, Harvard School of Public Health, and IRD (Institut de Recherche pour le Développement), based in Chiang Mai, Thailand. He has studied and conducted research on HIV in pregnant women and children and on the prevention of mother-to-child transmission of HIV since 1985. Dr. Lallemant led two major clinical trials on PMTCT (PHPT 1 and 2), which served as the basis for WHO recommendations for PMTCT in resource-limited settings. He is also involved in paediatric HIV research with PENTA, the European clinical research network and the NIH-funded International Maternal Pediatric Adolescent AIDS Clinical Trials Group (IMPAACT).
“Children living with HIV/AIDS are a neglected population, and pediatric AIDS can be considered a neglected disease,” said Dr. Lallemant. “While we must make every effort to eliminate new HIV infections among infants through large-scale access to PMTCT and maternal ART, we cannot neglect the millions of children currently and newly infected with the virus who are in dire need of treatment today.”
About Paediatric HIV/AIDS:
According to WHO, there are currently more than 2.5 million children under the age of 15 living with HIV, 2.3 million of whom (92%) are in sub-Saharan Africa. Each day, more than 1,000 children are newly infected with HIV, and 700 die from AIDS-related complications. According to UNAIDS, only 355,000 children with HIV/AIDS have access to antiretroviral therapy (ART), representing just 28% of those in urgent clinical need. Without treatment, one-third of children born with HIV will die before their first birthday, 50% will die before they turn two, and 80% will die before they are five years old.
About the Drugs for Neglected Diseases initiative (DNDi)
DNDi is a not-for-profit research and development organisation working to deliver new treatments for neglected diseases, in particular human African trypanosomiasis, leishmaniasis, Chagas disease, malaria, and, with the recent expansion of its portfolio, specific helminth-related infections and paediatric HIV. DNDi was established in 2003 by Médecins Sans Frontières/Doctors Without Borders (MSF), the Oswaldo Cruz Foundation from Brazil, the Indian Council for Medical Research, the Kenya Medical Research Institute, the Ministry of Health of Malaysia, and the Pasteur Institute of France. The WHO Special Programme for Tropical Disease Research (WHO/TDR) serves as a permanent observer. Since 2003, DNDi has delivered four new treatments for neglected patients: two fixed-dose antimalarials (ASAQ and ASMQ), nifurtimox-eflornithine combination therapy (NECT) for late-stage sleeping sickness, and sodium stibogluconate and paromomycin (SSG&PM) combination therapy for visceral leishmaniasis in Africa.