10 to Watch: Hot Biotech Prospects

Technological innovation promises to redefine health care as we know it.

The walls that have traditionally separated scientific disciplines are falling, and the interplay of computer technology, nanotechnology and other sciences with biology will transform the way that doctors monitor, diagnose and treat disease. The question is, to what extent will new technologies be embraced for their ability to cut health care costs by moving us away from today’s model of treating disease toward tomorrow’s model of keeping people well?

Here are some major technologies that promise to alter the biotechnology landscape. They have the potential to change not only the way we diagnose and treat disease, but also how we produce energy and manufacture goods, among other possibilities:

Whole Genome Scanning: The rapidly falling cost of whole genome scanning is expected to advance the promise of personalized medicine, make possible a new understanding of the genetic variation of individuals, and unlock the meaning of vast regions of DNA that now are not well understood. Harvard University genetics researcher George Church, founder of the Personal Genome Project, predicts manufacturers by the end of 2009 will cross the $1,000 threshold, a level at which broad use of the technology would be possible.

Stem Cells: The ability of stem cells to grow into virtually any of the body’s specialized cells is giving drug developers new ways to test drugs in the lab. But their real promise lies in giving rise to an era of regenerative medicine where life-altering injuries and deadly diseases are not just treated, but cured. Large pharmaceutical and biotechnology companies are showing an interest in stem cells through investments in this area, and policy changes in Washington are expected to accelerate progress.

Gene Therapy, RNAi and more: Typically, drugs work by binding with proteins that are the underlying cause of a specific disease. But a number of therapies in development seek to work by acting directly on genes that are responsible for producing the deleterious protein in the first place. There are several technologies for accomplishing this, whether it is the introduction of a gene through a viral vector, RNAi, antisense or the use of so-called zinc finger proteins. These approaches hold the promise of curing not only hereditary diseases, but cancer and infectious diseases that exploit genetic flaws as well.

MicroRNAs: A set of short strands of RNA known as microRNAs have emerged as a new means of diagnosing and treating disease. MicroRNAs work by preventing the translation of messenger RNA into proteins or by initiating the breakdown of messenger RNA. The absence or presence of specific microRNAs in various cells has been shown to be associated with specific human diseases, including cancer, viral infection, metabolic disorders and inflammatory disease. What makes these small strands of RNA particularly compelling to drug companies is that they have the ability to up-regulate or down-regulate not only a single gene, but networks of genes as well. This makes them well suited for being enlisted in the fight against diseases such as cancer and inflammatory disease where multiple genes in a network are at play. They can also serve as important diagnostic tools.

Nanotechnology: Nanotechnology involves working with materials at an atomic or molecular level with particles that are as small as one-billionth of a meter, roughly 75,000 times smaller than the width of a human hair. At such sizes, scientists can change the natural properties of materials, alter their conductive, magnetic, optical or other qualities to meet the needs of a specific application. New nanomaterials promise to impact health care in a variety of ways, including the development of sensors that can monitor changes within a cell, rapid diagnostics, new means of targeted drug delivery and new therapeutics.

Implantable sensors: Intelligent and implantable devices are promising a new era of medicine as they monitor the health of an individual and, in some cases, alert or even respond to changing conditions within the body. The possibilities range from implantable glucose monitors that release insulin as needed, to a so-called “field hospital on a chip” that continuously monitors a soldier’s sweat, tears or blood for biomarkers that signal common battlefield injuries such as trauma, shock, brain injury or fatigue.

Vaccine Technology: Instead of using a weakened or killed virus, DNA vaccines use a piece of genetic code for a pathogen. The technology promises to lead to cheaper and safer vaccines. Unlike conventional production methods using eggs to grow vaccines, DNA vaccines can be made significantly quicker and production is scalable. This could be critical in the event of a pandemic or bioterrorist attack since capacity could easily be added.

Synthetic Biology: Synthetic biology involves the construction of DNA to build organisms that can perform specific tasks. Though synthetic biology has garnered great attention for its potential in developing new biofuels that can break the world’s addiction to petroleum and provide potentially cleaner and renewable sources of fuel, it stands to change everything from the way drugs are made to the way industrial goods are manufactured. The promise of synthetic biology lies in the ability to create organisms that can more efficiently produce a desired end product than traditional production methods and to do so without the need for great inputs of energy or the output of toxic byproducts.

Systems Biology: Traditionally, biology has sought to understand the functioning of the human body by reducing it to small, individual components such as genes and molecules. Systems biology seeks to understand the interaction between networks of genes and their interactions with proteins. This data-intensive approach is made possible through advances in computing, the falling cost of genetic analysis, and other technological advances. This will play a critical role in bringing about personalized medicine.

Computer-based drug development: High-power computing and a rich body of knowledge are helping to cut the cost of drug development and accelerate the process. This is occurring both through the identification of drug candidates and the use of in-silico testing through simulations, which can help predict if compounds will likely be safe and effective or not.

Financial and regulatory pressures are not working in favor of new technologies. Though part of the solution to reining in the rapid growth in spending on health care lies in developing and implementing new technologies, investors need to see a reasonable road to returns. They also want to have confidence that regulators will not needlessly slow their development because of political rather than scientific debates.