From Alzheimer’s to Zebrafish: Eclectic Science and Regulatory Stories 22
There have been many drug and medical device discoveries in the past 10 years or so and
recounting them would be most difficult. One or two truly stand out.
The first is employing a patient’s genetic profile to judge whether a specific drug
will be effective in treating disease. Genetic testing is now used to identify breast cancer
patients who might benefit from therapy with Herceptin (trastuzumab), a monoclonal
antibody. The drug targets cancer cells that overexpress a protein called HER-2, which is
found on the surface of cancer cells. In tumor cells, errors in DNA replication may result
in multiple copies of a gene on a single chromosome. This alteration is known as gene
amplification and leads to an overexpression of the HER-2 protein, resulting in increased
cell division and a higher rate of cell growth. Herceptin is used only to treat cancers that
overexpress the HER-2 protein.1
The second seminal discovery is new ways to administer drugs without swallowing
them or using hypodermic needles. A whole host of methods are now in use, or will be
in the near future. Needle-less devices like the SonoPrep are a good example. The device
applies ultrasonic energy to the skin to disorganize the lipid bi-layer of the stratum cor-
neum, and creates reversible micro channels (temporary pores) in the skin through which
fluids and analytes can be extracted and large molecules can be delivered.2 Other delivery
methods under development include micro-needle patches activated by micro-pumps,
and microchips embedded with medication inserted under the skin where they slowly
and reliably release the drug into the bloodstream.
Many futuristic drugs and devices will be spawned as a result of decoding the human
genome. Although the decoding process is fairly recent, the term “pharmacogenomics”
has been used since the late 1950s. First was the understanding that genetic differences
cause people to metabolize drugs differently, which led to creating drugs in the 1990s
designed for a person’s individual genetic makeup. The 21st century will no doubt be wit-
ness to a number of new discoveries in this field as scientists continue to solve the riddles
of human genomics. Precise genetic testing will be pivotal in employing new monoclonal
antibodies, other new drug therapies and optimal delivery methods. Science is learning
more about the human body on a smaller and smaller scale.
The next wave of drug discovery and delivery methods likely will be what is now called
“nanotechnology,” which guarantees that this century will be replete with scientific terms
beginning with “nano.” It already has begun. Nanoscience has been deemed the technol-
ogy breakthrough of tomorrow and several research groups are developing programmable
implants that could revolutionize the way drugs are administered. Nanoparticles could be
used to deliver chemotherapeutic agents just where they are needed, to avoid the side effects
that so often result from potent medicines. Artificial nanoscale building blocks may one day
be used to help repair tissues such as skin, cartilage and bone.3 According to one expert in the
field, nanomedicine will have extraordinary and far-reaching implications for the medical
profession, for the definition of disease and for the diagnosis and treatment of medical condi-
tions, including aging.4 We now know that shaving ordinary materials down to nanoparticles
causes them to behave in extraordinary ways. However, before embarking on some of the
aspects of nanotechnology, it would be prudent to include a few definitions.
Definitions
The prefix nano means one-billionth. (Nano is thought to derive from the Greek noun for
dwarf.) One nanometer (abbreviated as 1 nm) is 1/1,000,000,000 of a meter, roughly the
length of 10 hydrogen atoms. To get a sense of the nano scale, a human hair measures
50,000 nanometers across, a bacterial cell a few hundred nanometers. The smallest objects
visible with the unaided human eye are 10,000 nanometers across.5
• “Nanoscience” is the study of the fundamental principles of molecules and struc-
tures with at least one dimension roughly between 1 and 100 nanometers. These
structures are appropriately termed nanostructures.
There have been many drug and medical device discoveries in the past 10 years or so and
recounting them would be most difficult. One or two truly stand out.
The first is employing a patient’s genetic profile to judge whether a specific drug
will be effective in treating disease. Genetic testing is now used to identify breast cancer
patients who might benefit from therapy with Herceptin (trastuzumab), a monoclonal
antibody. The drug targets cancer cells that overexpress a protein called HER-2, which is
found on the surface of cancer cells. In tumor cells, errors in DNA replication may result
in multiple copies of a gene on a single chromosome. This alteration is known as gene
amplification and leads to an overexpression of the HER-2 protein, resulting in increased
cell division and a higher rate of cell growth. Herceptin is used only to treat cancers that
overexpress the HER-2 protein.1
The second seminal discovery is new ways to administer drugs without swallowing
them or using hypodermic needles. A whole host of methods are now in use, or will be
in the near future. Needle-less devices like the SonoPrep are a good example. The device
applies ultrasonic energy to the skin to disorganize the lipid bi-layer of the stratum cor-
neum, and creates reversible micro channels (temporary pores) in the skin through which
fluids and analytes can be extracted and large molecules can be delivered.2 Other delivery
methods under development include micro-needle patches activated by micro-pumps,
and microchips embedded with medication inserted under the skin where they slowly
and reliably release the drug into the bloodstream.
Many futuristic drugs and devices will be spawned as a result of decoding the human
genome. Although the decoding process is fairly recent, the term “pharmacogenomics”
has been used since the late 1950s. First was the understanding that genetic differences
cause people to metabolize drugs differently, which led to creating drugs in the 1990s
designed for a person’s individual genetic makeup. The 21st century will no doubt be wit-
ness to a number of new discoveries in this field as scientists continue to solve the riddles
of human genomics. Precise genetic testing will be pivotal in employing new monoclonal
antibodies, other new drug therapies and optimal delivery methods. Science is learning
more about the human body on a smaller and smaller scale.
The next wave of drug discovery and delivery methods likely will be what is now called
“nanotechnology,” which guarantees that this century will be replete with scientific terms
beginning with “nano.” It already has begun. Nanoscience has been deemed the technol-
ogy breakthrough of tomorrow and several research groups are developing programmable
implants that could revolutionize the way drugs are administered. Nanoparticles could be
used to deliver chemotherapeutic agents just where they are needed, to avoid the side effects
that so often result from potent medicines. Artificial nanoscale building blocks may one day
be used to help repair tissues such as skin, cartilage and bone.3 According to one expert in the
field, nanomedicine will have extraordinary and far-reaching implications for the medical
profession, for the definition of disease and for the diagnosis and treatment of medical condi-
tions, including aging.4 We now know that shaving ordinary materials down to nanoparticles
causes them to behave in extraordinary ways. However, before embarking on some of the
aspects of nanotechnology, it would be prudent to include a few definitions.
Definitions
The prefix nano means one-billionth. (Nano is thought to derive from the Greek noun for
dwarf.) One nanometer (abbreviated as 1 nm) is 1/1,000,000,000 of a meter, roughly the
length of 10 hydrogen atoms. To get a sense of the nano scale, a human hair measures
50,000 nanometers across, a bacterial cell a few hundred nanometers. The smallest objects
visible with the unaided human eye are 10,000 nanometers across.5
• “Nanoscience” is the study of the fundamental principles of molecules and struc-
tures with at least one dimension roughly between 1 and 100 nanometers. These
structures are appropriately termed nanostructures.