Engineer better medicines

Engineers are developing new systems to use genetic information, sense small changes in the body, assess new drugs, and deliver vaccines.

Doctors have long known that people differ in susceptibility to disease and response to medicines. But, with little guidance for understanding and guidance for understanding and adjusting to individual differences, treatments developed have generally been standardized for the many, rather than the few.


How will genetic science change how medicines are made? 

Human DNA contains more than 20,000 genes, all of which are stored in our cells' nuclei. A gene is a strand of chemical code, a sort of blueprint for proteins and other substances necessary for life. Cells make those molecules according to the genetic blueprints.

Each person’s overall blueprint is basically the same, made up of about 3 billion “letters” of code, each letter corresponding to a chemical submit of the DNA molecule. But subtle variants in about 1 percent of our DNA often the result of just a single chemical letter being different give humans their individual identities.

Beyond physical appearance, genes give rise to distinct chemistries in various realms of the body and brain. Such differences sometimes predispose people to particular diseases, and some dramatically affect the way a person will respond to medical treatments.

Already, some aspects of the personalized medicine approach are in place for some diseases. Variants of a gene linked to breast cancer, for instance, can foretell a woman’s likely susceptibility to developing or surviving the disease, a helpful guide for taking preventive measures. In certain cases of breast cancer, the production of a particular protein signals a more aggressive form of the disease that might be more effectively controlled with the drug Herceptin.

Still, multiple challenges remain in the quest for a widespread effective system of personalized medicine. They will be addressed by the collaborative efforts of researchers from many disciplines, from geneticists to clinical specialists to engineers.

What prevents you from creating personalized medicines now?

One engineering challenge is developing better systems to rapidly assess a patient’s genetic profile; another is collecting and managing massive amounts of data on individual patients; and yet another is the need to create inexpensive and rapid diagnostic devices such as gene chips and sensors able to detect minute amounts of chemicals in the blood.

In addition, improved systems are necessary to find effective and safe drugs that can exploit the new knowledge of differences in individuals. The current “gold standard” for testing a drug’s worth and safety is the randomized controlled clinical trial -- a study that randomly assigns people to a new drug or to nothing at all, a placebo, to assess how the drug performs. But that approach essentially decides a drug’s usefulness based on average results for the group of patients as a whole, not for the  individual.  

In a new field called “synthetic biology,” novel biomaterials are being engineered to replace or aid in the repair of damaged body tissues. Some are scaffolds that contain biological signals that attract stem cells and guide their growth into specific tissue types. Mastery of synthetic tissue engineering could make it possible to regenerate tissues and organs.

What are the benefits of personalized medicine?

Ultimately, the personalization of medicine should have enormous benefits. It ought to make disease (and even the risk of disease) evident much earlier, when it can be treated more successfully or prevented altogether. It could reduce medical costs by identifying cases where expensive treatments are unnecessary or futile. It will reduce trial-and-error treatments and ensure that optimum doses of medicine are applied sooner. Most optimistically, personalized medicine could provide the path for curing cancer, by showing why some people contract cancer and others do not, or how some cancer patients survive when others do not.

Of course, a transition to personalized medicine is not without its social and ethical problems. Even if the technical challenges can be met, there are issues of privacy when unveiling a person’s unique biological profile, and there will likely still be masses of people throughout the world unable to access its benefits deep into the century. 

What is engineering's role in creating personalized medicine?

The engineering challenges for enabling drug discovery mirror those needed to enable personalized medicine: development of more effective tools and techniques for rapid analysis and diagnosis so that a variety of drugs can be quickly screened and proper treatments can be promptly applied. Current drugs are often prescribed incorrectly or unnecessarily, promoting the development of resistance without real medical benefit.

Traditional vaccines have demonstrated the ability to prevent diseases, and even eradicate some such as smallpox. It may be possible to design vaccines to treat diseases as well. Personalized vaccines might be envisioned for either use. But, more effective and reliable manufacturing methods are needed for vaccines, especially when responding to a need for mass immunization in the face of a 
pandemic.