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 P-coul, 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, M-decins Sans Fronti-res/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.