New medicines for neglected diseases

Blazing a trail: Neglected disease research at Novartis

Innovation is the essence of corporate citizenship at Novartis.

The Group has a proud legacy of pioneering medicines in diseases ranging from cancer and mental disorders to organ transplantation. During 2009, medicines and vaccines from Novartis were used to treat and protect more than 930 million people around the world.

Thanks to its commercial success, Novartis is able to provide medicines at no profit – or sometimes free – to patients in the developing world. Novartis also offers discounts and support programs to patients without medical insurance or other financial resources in industrialized countries. During 2009, treatments worth USD 1.5 billion were contributed through these access-to-medicine programs, reaching more than 79 million patients in need.

In 2003, Novartis established a new research center dedicated to “neglected” diseases that take a heavy toll in the developing world. The Novartis Institute for Tropical Diseases (NITD) focuses on research and early development of treatments against dengue fever, malaria and drug-resistant tuberculosis (TB). All medicines discovered at NITD will be made available to patients in low-income, endemic countries.

In 2007, Novartis inaugurated a sister research institute for development of vaccines. The Novartis Vaccines Institute for Global Health (NVGH) was the first such initiative with a nonprofit mission established by a major vaccine manufacturer. The initial focus is diarrheal diseases, one of the biggest killers of children across the developing world. All vaccines developed by NVGH will also be provided at an affordable and accessible price to populations of developing countries.

“It will be a real challenge,” said Rolf Zinkernagel, Nobel laureate, senior scientific advisor to both NITD and NVGH and a member of the Novartis Board of Directors since 1999. “These diseases are neglected because making drugs or vaccines against them is difficult. But this is also an opportunity to try new things – combining expert science and the know-how of a company with a proven track record of translating ideas into concrete products.”

NITD and NVGH are applying state-of-the-art tools – and sometimes channeling expertise and technologies from commercial research operations – to diseases where research was either moribund or virtually non-existent.

“For commercial reasons, major pharmaceutical companies traditionally devoted little, if any, research activities to major diseases that occur predominantly in the poor parts of the world,” says Paul Herrling, Head of the Novartis Institutes for Developing World Medical Research, the organization that includes NITD and NVGH. “That is changing, and Novartis has allocated some of our research and development skills and technologies to address neglected diseases.”

NITD is the biggest drug discovery center worldwide focusing on dengue fever, a viral disease transmitted by mosquitoes that has spread relentlessly in tropical and subtropical regions in recent years. Dengue virus infects more than 50 million people each year but about 500 000 cases escalate from a simple fever, lasting from 7 to 15 days, to a potentially life-threatening form called dengue hemorrhagic fever that causes an estimated 20 000 deaths, mainly children.

Though research targeting malaria commenced in 2007 – later than dengue and TB – NITD has assembled a broad portfolio of early-stage projects by forging collaborations with academic researchers around the world.

By contrast, drug-resistant tuberculosis has remained an intractable target. Public health officials underscore the urgent need for new treatments: More than a million people die every year from TB, and the number of drug-resistant strains of the TB bacillus has exploded in recent years. Yet science has failed to keep pace with that mounting burden: “This is a disease where there has been very little basic or applied research for decades,” emphasizes Dr. Herrling.

The aspiration to make NITD a role model in all aspects of drug discovery includes training of students from developing countries in the special skills required to translate basic science into new medicines. Training programs at NITD, and later NVGH, have been welcomed as a step to reverse the “brain drain” of healthcare professionals from Africa to developed countries. “We need more innovative ways to increase the proportion of trained people who stay and work in endemic countries,” says Ogobara Doumbo, Director of the Malaria Research and Training Center of the University of Bamako, Mali.