Our motivation and approach
Despite the significant financial investment we have made, we are still struggling to fully understand and adequately treat the majority of complex diseases. If we look at the brain, neurological diseases account for 11% of the global burden of disease and, as a result, $900 billion a year is spent trying to treat them. Drugs that are used to treat the injured or diseased brain also take about 35% longer to be developed for use in humans compared to drugs for any other type of disease.
Gathering information about a disease is difficult because the disease microenvironment is dynamic, heterogeneous, and highly variable from person to person. Delivering drugs to this environment is also challenging, because the body and the disease are very effective at keeping drugs out or adapting to minimize the effect of the drug. Many drugs often are also directed at only one aspect of the disease or are so complex and poorly understood that the interactions within the body can’t be controlled. In addition, when drugs are administered into the body, very little (usually less than 1%) actually gets to the disease site. To compensate for this, we have to give much more of the drug, or a cocktail of drugs, to have any effect. These can both increase side effects and harm normal healthy tissue.
Nanotechnology, which consists of small (~1-100nm) but highly tailorable platforms, can be used as both an information gathering tool and a therapeutic or diagnostic approach. If we ask meaningful questions, i.e. which aspects of a disease directly impact our ability to effectively deliver a drug to treat that disease, we can then address these questions with nanotechnology. By using nanoparticles to probe the brain, we learn and quantify how accessible the brain is to a therapy in the context of each disease, and how readily a therapy can move to the diseased cells once in the brain. Based on the limitations the nanoparticle experiences, we can then re-engineer the particle (I’ve termed this disease-directed engineering) to overcome these limitations to better deliver the therapy to the specific disease cells, leaving normal healthy cells alone. This leads to more effective drugs. Because there is more specific delivery to the diseased sites, and not to normal healthy tissue, a smaller amount of drug can be used, and there are less side effects.
Importantly, our work is highly interdisciplinary, requiring equal understanding of chemical engineering, drug delivery, biophysics, and neurobiology principles. To do front-line work in an impactful area, we need individuals who are committed to the team and lab family, who are creative, motivated, passionate, and compassionate. We believe being inclusive and supportive only allows us to do better, higher quality work.