Gene expression, proteomics, stem cells — such terminology may not fluster the scientists and medical doctors who work in pharma. But their support networks of marketers, lawyers, and investors may understand the underlying science only vaguely or not at all.
To remedy this gap, BioTech Primer, a company focused on educating non-science professionals about the basics of biotechnology and pharmaceuticals, has taught basic science concepts to over 35,000 people since it was founded in 2001.
Emily Burke, director of instruction at BioTech Primer, is teaching a two-day seminar in “BioBasics: Biotech for the Non-Scientist,” Thursday and Friday, April 19 and 20, 9 a.m. to 4:30 p.m., for BioNJ, at Robert Wood Johnson Hamilton, 3100 Quakerbridge Road. Cost: $945. For more information, contact Kerri Muir at 410-377-4429, ext. 22 or Muir@BiotechPrimerInc.com. To register, go to www.BiotechPrimerInc.com and follow the links to the BioBasics course.
To tempt the intrepid nonscientists in Central Jersey, Burke talks about a few of the concepts that she will cover in greater detail and with illustrations at the seminar:
Biologics versus small molecule drugs. Not so long ago traditional pharma devoted itself only to producing small molecule drugs, like aspirin and ibuprofen, that are synthesized by chemists in a lab. But today even big pharma is moving into biologics, which tend to be actual proteins that are “manufactured” by living cells based on the “recipe” found in a gene.
The first biologic to be mass produced commercially was insulin in 1982. Scientists at Genentech, founded in 1976, isolated the human gene for the protein insulin and figured out how to put that gene into a bacteria cell. The cell then “reads” the recipe for the insulin protein provided by that gene and makes human insulin, says Burke.
Finding the conditions to scale the production of these biologics can be time consuming, but they have properties that make all the work worthwhile. Biologics are highly specific as to the target of their activity, and as a result have a second benefit — they tend to have few side effects.
This absence of side effects is one of reasons why many companies are starting to pursue biologics, especially for chemotherapy drugs. “Monoclonal antibody drugs can be a highly specific way to target specific tumor cells and avoid the systematic side effects that traditional chemotherapy drugs have,” says Burke. Two examples are Herceptin for certain types of breast cancer and Rituxan for non-Hodgkin’s lymphoma.
Personalized medicine. Sometimes called stratified medicine, this new approach is based on some of the current research that has come out of the human genome project. This project, a 10-year undertaking of labs around the world working together, took DNA samples from anonymous individuals and sequenced them to come up with a representative human DNA sequence.
“Now what is on the horizon is determining each individual’s genome,” says Burke. By deciphering the information on these genomes, says Burke, “we are learning to associate specific mutations with specific types of cancer, for example, and then to develop highly specialized therapies that will target a specific genetic form of a disease.”
Practically, the accumulation of many individual genomes will require that both the cost be lower and the time decreased. As multiple companies are working on this next-generation sequencing, says Burke, they are coming up with new technologies intended to get the cost of sequencing the human genome down to less than $1,000 and having it take only a couple of days. “There are some companies that are very close to that,” she adds.
Once this data is available, scientists need to figure out what to do with it. They will not actually be looking for completely different therapies for each individual, but rather will identify different subtypes of cancer associated with the same mutation.
Burke offers an example: “Herceptin targets women that have breast cancer because of a specific mutation — the one that this drug is highly effective against.” Currently scientists are identifying more of these mutations and associating them with specific diseases, with the hope of finding a drug that is effective for each class of mutations.
Antibodies: Antibodies are used as research tools, diagnostics, and therapeutics, and it is in part their versatility that makes them so valuable, says Burke. Antibodies are proteins that the human immune system makes naturally; they are made by B-cells, a type of immune cell that recognizes foreign proteins in our bodies.
The antibody attaches itself to a particular protein, called an antigen, on the surface of a virus or a bacteria and thereby deactivates it. “The cool thing about the immune system is that you are born with the ability to produce any possible antibody against any possible antigen,” says Burke.
Stem cells and regenerative medicine. Embryonic stem cells, which come from embryos at the eight-cell stage of development, have the possibility of developing into any type of cell in the body. To use them, researchers are trying to figure out what differentiation factors must be added to a stem cell for it to develop into various tissue types. In specific, says Burke, “we are trying to get stem cells to differentiate into something like nerve or brain tissue that we’re not good at regenerating ourselves.”
Scientists have had some success using stem cells to treat rats that have been surgically treated to mimic the conditions of Parkinson’s disease. Although the causes of Parkinson’s are unknown, scientists do know that there are people with the disease who have a signaling defect in the neurons that secrete the neurotransmitter dopamine.
After years of research scientists are now able to add the correct factors to stem cells so that they develop into this type of neuron; and when these neurons are transplanted into rats mimicking Parkinson’s disease, the rats show an improvement in function in terms of coordination and other measures.
Given the politics around stem cells, scientists have also figured out in the last couple of years how to develop induced pluripotent stem cells from adult cells. These stem cells also have the ability to differentiate into any other cell in the body. By introducing four particular genes into an adult skin or other type of cell, the cell reverts into having the character of an embryonic stem cell.
The current research on stem cells will probably not be used immediately to produce therapeutics. We need to know, for example, how long these cells will remain differentiated. “We have to demonstrate safety and that they will not revert back to stem cells once they are implanted into humans,” says Burke.
But these cells do have an immediate use — they are useful in creating good research models in the lab. For conditions like Alzheimer’s and Lou Gehrig’s disease, for example, scientists don’t have access to the diseased cells because they do not have access to a patient’s brain.
So instead, scientists can take skin cells from someone with Alzheimer’s, induce the cells to become pluripotent stem cells, and then induce them to become brain cells that will have some of the characteristics of Alzheimer’s brain cells. “It’s a way to study brain cells from an Alzheimer’s patient without actually taking brain cells,” says Burke.
Burke grew up in Omaha, Nebraska, where her father was an electrician. Interested in being a scientist from about age 12, she received a bachelor of science in biological sciences from Carnegie Mellon University.
An avid long distance runner who did track and cross country in college, Burke decided to get a master’s degree in sports medicine, which she did at the University of South Alabama in Mobile. Realizing during the master’s program that she really was looking for something more academic, she stayed on and earned a doctorate in molecular biology.
She did a three-year postdoctoral fellowship at the Scripps Research Institute in La Jolla, California, where she was involved in laboratory research. What she liked best about cutting-edge research, she says, was interacting with some of the smartest people in the world, but she found the pace too slow and realized that a lifetime in the lab was not for her. “I love science, and I love learning about science, but learning about science and doing science are very different things,” she says.
Next she tried scientific writing for Isis Pharmaceuticals in Carlsbad, California, which she enjoyed but missed the people contact.
During this time, Burke also taught biology and biotechnology classes at San Diego Miramar College and did some teaching for BioTech Primer. In the process she realized how much she loved teaching. Realizing that BioTech Primer offered her the best of both worlds — she was teaching but also paid to keep up with the latest scientific developments and to talk to scientists still active in the lab, she became a fulltime employee. “You get to keep learning, but I like a little faster pace than being in the lab,” she says.
Burke believes it is important for nonscientists in biopharma to understand some science for several reasons, and the reasons will vary depending on the non-scientist’s own role.
“Investors, of course, will have a better time asking the right questions if they know the basics,” she explains in an e-mail. “Communications and marketing people will be able to best describe their company’s cutting edge research or unique products.
“Legal and financial officers will be better able to communicate internally with their own scientists, who often will not be skilled at explaining what they do in simple terms, or have the time to provide the background required for adequate understanding. Human resource professionals will be better at screening applicants and understanding resumes. IT people will understand better the applications that they are developing, and so on.”