Chapter Nine:

Future of Biotechnology in Healthcare

Biotechnology can offer patients more and better healthcare choices. New, innovative diagnostics and therapies are changing how some human diseases are prevented and others are treated. This monumental healthcare shift is in its early stages, with novel medicines, diagnostics and technologies in development that hold great potential to improve patients lives.

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Personalized Medicine Personalized medicine is the concept that patients should be treated with therapies and medicines based specifically on each patients unique genetic makeup, for optimal results . Currently, the practice of medicine is based on standards of care that are determined by averaging responses across large groups of people . Personalized medicine is a new paradigm that proposes to manage a patients disease based on the individual patients specific characteristics, including age, gender, height, weight, diet, genetics and environment . Genetic testing is beginning to allow the development of genomic personalized medicinemedical care based on a patients genotype or gene expression profile .
BIoFaCT Molecular diagnostic tests analyze DNA, RNA or protein molecules to identify a disease, determine its course, evaluate responses to therapy or predict individual predisposition to a disease .

Advances in DNA technology are the keys to both pharmacogenomics and personalized medicine . These advances allow for testing and identifying an individuals unique genetic makeup and then comparing those differences with the population at large . Knowledge of the human genome, variations of the genome among individuals and variations of the encoded proteins produced enables researchers to develop medicines that address the individual needs of each patient . Pharmacogenomics and personalized medicine promise to improve clinical trials for new drugs, advance screening technology for diseases and result in moreeffective individualized healthcare and advances in preventive medicine . Genetic Testing The biotechnology industry has brought about vast improvements in testing and diagnosis for genetic diseases . The discovery of singlenucleotide polymorphisms (SNPs)singlenucleotide changes in the DNA sequence was one of the major breakthroughs in genetic testing . SNPs (pronounced snips) represent one of the most common forms of genetic variation among individuals . When a SNP occurs in a gene sequence that encodes for a specific protein, it may change that protein and cause a disease or increase a patients susceptibility to a disease . Utilizing technology to detect SNPs allows for more-accurate diagnosis of genetic diseases and therefore facilitates treatment decisions . Genetic testing provides patients with both an understanding of possible risks for certain diseases and possible opportunities for prevention .
BIoFaCT Approximately 10 million SNPs have been identified in the human genome .

Pharmacogenomics A major movement in healthcare is pharmacogenomics. Pharmacogenomics takes advantage of the fact that individuals have unique genomes representing their genetic makeup . Each genome is likely to react differently to a particular drug and dose amount . The challenge is to identify which drug and which dose will work most optimally for each person or for groups of individuals who share similar genetics . By understanding a patients genetic makeup, a physician can better prescribe a drug and dose level that will optimally work to combat a particular disease .

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Future of Biotechnology in Healthcare

Gene Therapy Gene therapy is an emerging area of applied genetics that utilizes recombinant DNA techniques . In this case, the recombinant DNA molecules themselves are used for therapy . Gene therapy involves inserting genes, created by recombinant DNA technology, into the cells and tissues of patients to treat their diseases . Scientists are studying gene therapies for a number of inherited human diseases involving defective genes . The idea is to replace them with new, functional genes .

Cell therapies also could be developed in which undifferentiated stem cells may be implanted along with growth factors to guide their differentiation in the patients body . The aim is to replace the damaged cells with healthy, disease-free cellshence the term regenerative medicine for this approach . The hope is that stem cells, directed to differentiate into specific cell types, could be a renewable source of replacement cells and tissues used to treat a wide range of diseases . Nanotechnology

Since the first clinical trial was initiated in 1990, gene therapy research has expanded greatly, with an increasing number of human trials . The field, still in experimental stages, focuses its efforts on patients with severe and life-threatening diseases who usually have few treatment options or who have failed all available therapies . Stem Cells Stem cells are unspecialized cells that can renew themselves indefinitely to produce more stem cells . They can mature and develop specialized functions or differentiate under specific growth conditions . Stem cells eventually differentiate to form all of the different types of cells that make up the body . The broad potential of an undifferentiated stem cell to make a variety of other cells is the focus of stem cell research . Stem cell therapy, which is still in experimental stages, involves growing stem cells in the lab and guiding them toward a desired cell type by adding different growth factors . The differentiated cells are then surgically implanted . The theory is that stem cells may then integrate into the diseased tissue, replace diseased cells and reverse the effects of the disease .

Nanotechnology deals with the manipulation of molecules and structures on a nanometer (one-billionth of a meter) or atomic scale . Applying nanotechnology for the improvement of human health is called nanomedicine. Biotechnology nanomedicine harnesses living organisms and/or their components on a very small scale . One example of nanomedicine is the experimental use of nanoshells to selectively target and destroy cancer cells at the cellular level . Nanoshells are nanoscopic metallic lenses that are selectively delivered to specific organs or tumors through the bloodstream . Nanoshells have the ability to capture infrared light shown through the skin of a cancer patient and convert it to heat, which kills only the targeted cancer cells . Nanoparticles called buckyballsuniquely shaped and constructed carbon molecules are also showing potential for drug delivery to target molecules or cells . They may make it possible to deliver drugs that do not dissolve in water . Also, because of their small size, they allow more of the drug to be delivered per volume . Scientists are working on nanoparticles to unclog blocked arteries .

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New Drug Delivery Systems Biomedical researchers are studying new ways of delivering drugs within the body that could improve effectiveness . One example is the development of microscopic particles called microspheres that have tiny holes just large enough to carry and deliver drugs to their targets . They are made out of materials that resemble naturally occurring fats in cell membranes and are delivered as a mist sprayed into the nose or mouth . Microsphere therapies are currently available for lung cancer and respiratory illnesses . Current research is investigating the use of microspheres to deliver anticancer drugs to active tumors and for use with anesthetics in pain management .

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Future of Biotechnology in Healthcare