Novartis is focused on discovering and developing new medicines to treat disease and improve human health. We set our research priorities at the intersection of unmet medical need and strong science. Only later do we consider commercial opportunity. This focus leads us to actively target diseases where we can make a meaningful difference. Our “science without borders” approach to research recognizes and embraces the needs of increasingly global and diverse patient populations, including those with rare or neglected diseases.
Our R&D strategy is to focus on therapeutic areas that represent our core strengths – including oncology, cardiovascular, neuroscience, eye care and biosimilars – and to explore other areas that we believe are ripe for innovation, such as immuno-oncology, aging and regenerative medicine. We are also focused on developing new therapies to treat and prevent infectious diseases that disproportionately impact the developing world.
One challenge to public health – this one common to both developed and developing countries – is the growing resistance of bacteria to antibiotics. Without effective drugs, even the most basic infections could become life-threatening. The most serious threat is from multidrug-resistant gram-negative bacteria, which are showing resistance not just to older antibiotics derived from penicillin, but also to carbapenems – a potent antibiotic class that is typically used when everything else has failed. Gram-negative bacteria have two membranes at the cellular level, and antibiotics must make it past both, a difficult challenge. The Novartis infectious disease research team is working to develop new antibiotics to treat these forms of bacteria. In January 2016, Novartis joined forces with more than 80 leading international pharmaceutical and biotechnology companies to call for a global, united front with governments against the emergence of drug-resistant infections.
We remain committed to finding new medicines to treat neglected, infectious diseases that can be devastating in developing countries. Our infectious disease team is exploring new treatments for viral infections, including respiratory viruses (such as influenza and respiratory syncytial virus, or RSV) and viruses that threaten patients with HIV/AIDS. The Novartis Institute for Tropical Diseasesin Singapore is dedicated to finding new medicines for dengue fever, human African trypanosomiasis, malaria, and others neglected diseases.
Novartis has a longstanding commitment to malaria and currently has two treatments, KAE609 (cipargamin) and KAF156, in Phase II clinical development. Both are active against Plasmodium vivax and Plasmodium falciparum, the parasites responsible for most malaria-related deaths. In July 2016, a higher dose of our artemisinin-based combination therapy for the treatment of malaria received World Health Organization prequalification. With this higher dose, a malaria patient can take just six tablets – versus the previous 24 – to complete a full course of treatment.
We also undertake adaptive development by, for example, modifying existing medicines to better meet the needs of underserved and vulnerable patient groups like children and the elderly, and those living in high-heat and tropical climates. These may include formulations that are age-appropriate, heat stable or in different dosage strengths to help increase treatment adherence. Some examples include the higher dose of our artemisinin-based combination therapy for the treatment of malaria, as well as our development of the first artemisinin-combination therapy specifically to meet the needs of children.
Collaborating to eliminate rheumatic heart disease (RHD) in Zambia
RHD has been eliminated in most developed nations, but sub-Saharan Africa studies show that approximately 2-3% of school-age children have signs of this often fatal disease.1 Zambian healthcare providers from the Lusaka University Teaching Hospital, Novartis Institutes for BioMedical Research, physicians from Massachusetts General Hospital (MGH) and the Pan-African Society of Cardiology (PASCAR) are collaborating to promote RHD prevention by treating children with streptococcal infections and silent RHD. The collaboration has screened over 3,000 Zambian children in 50 Lusaka schools using new technology: hand-held portable echocardiography machines. Children diagnosed with RHD are offered treatment with monthly penicillin injections to prevent recurrent strep throat and associated cardiac damage, according to national guidelines in Zambia. In addition, multiple trainings of healthcare staff and school teachers have taken place, while an open-source mobile health platform, called the eRegistry, has been developed and launched to help local doctors track patient care. At the invitation of the health authorities in Zambia, the collaboration plans to extend the rollout of the RHD training and treatment effort from the initial Lusaka scheme to provinces across Zambia, with the ultimate goal of eliminating RHD in the country.
Partnering with the first drug discovery center on the African continent
Supported by The Novartis Research Foundation, Novartis has entered a research partnership with H3-D, the first fully integrated drug discovery and development center on the African continent, affiliated with the University of Cape Town in South Africa. The goal of the center is to educate the next generation of African scientists and to work on diseases that affect people on the African continent. The partnership between Novartis and H3-D involves research collaborations in TB and malaria, support with the set-up of a clinical study site at H3-D, scientific exchange programs and infrastructure support.