Frequently Asked Questions (FAQ)

General

What is translational research?

What is Regenerative Health Biotechnology?

Can human tissues regenerate?

What careers are available to me in biotechnology?

Science and Medicine

What are stem cells and why are they important?

What is cell therapy?

What is gene therapy?

What conditions can be treated or cured by gene and cell therapies?

Why is there resistance to stem cell research?

Where can I go to learn more?

Biopharmaceutical Production

What is a biopharmaceutical product?

How do biopharmaceuticals get from the laboratory to the patient?

What is cGMP?

What is a cleanroom?

What is Quality Control?

What is Quality Assurance?

Where can I go to learn more?

Answers

What is translational research?

Translational research is the process by which discoveries made in the laboratory are transitioned into practical applications. Scientists who study diseases at a cellular or molecular level provide information to clinical scientists, process scientists, analytical scientists, and manufacturers, who translate this basic information into drugs that are potential treatments or cures. Discoveries from these clinical studies prompt new studies at the cellular/molecular level, which drive further clinical studies, and this cycle continues until the new drug reaches a level of safety and efficacy for clinical distribution. For more information, please see the NIH Roadmap: http://nihroadmap.nih.gov/clinicalresearch/overview-translational.asp

What is Regenerative Health Biotechnology?

Scientists and doctors are tapping into the regenerative ability of the human body to cure debilitating diseases, repair or replace injured or diseased organs, bones, and nerves, and even grow new teeth. That sounds great - but what is regenerative biotechnology?
In Florida, we commonly find skinks looking for insects or sunning on our houses, decks, and porches. As kids, we learned that if the family cat or a little brother pulled the tail from one of these lizards, the tail would grow back - it would regenerate. A newt can regenerate a severed hind leg, and if its lower jaw is cut off, it will grow back, complete with teeth. The flat worm Planaria, when cut in half, can grow a new head from one half and a new tail from the other. Further up the evolutionary chain, however, this amazing regenerative ability has been lost. Or has it?

Can human tissues "regenerate"?

Not like skinks, newts, or flatworms. Humans can't regenerate teeth, limbs, or organs, but we can grow new bone and skeletal muscle. Certain tissues grow continuously, like hair, skin, and nails. The liver comes closest to a skink-like regenerative capacity. In response to injury, liver cells that were previously not in a reproductive state will begin to divide rapidly while continuing to perform their metabolic function. In this way, they can replace lost tissue in a matter of days.

What careers are available to me in biotechnology?

A biotechnology focus can lead to many careers. In addition to cell and molecular biology, biochemistry, genetics, and other scientific fields, increasing knowledge of the human genome and diseases will impact not only the biotechnology industry, but ancillary industries that interface directly with the biotech industry. These ancillary industries include the fields of computer science, mathematics, counseling, sociology, ethics, religion, law, education, instrumentation, architecture, mechanical engineering, law, and journalism. Most sectors of the workforce can find rewarding, well-paying jobs in the biotechnology industry, whether they are high school graduates or have an advanced degree.

What are stem cells and why are they important?

The term "stem cell" is used to describe precursor cells that can become different tissue types. The categories of stem cells mentioned most commonly are embryonic stem cells and adult stem cells. A very early embryo is made up of embryonic stem cells that are pluripotent; that is, they can differentiate into all different cell types - cells that make up the brain, skin, bones, connective tissue, internal organs, eyes, fingernails, and everything else that comprises a fully developed human. Cells isolated from these embryos can be propogated indefinitely in culture, and can remain undifferentiated while retaining the ability to form any kind of cell. According to the National Institutes of Health, the potential for embryonic stem cells in medicine is "staggering." Researchers have already used these cells to grow heart muscle cells that can beat in unison in a culture dish, blood vessel cells, bone, cartilage, neurons, and skeletal muscle. The great promise of embryonic stem cells is tempered by ethical concerns surrounding the procurement and use of these cells in medical research.
Mature body tissues contain adult stem cells, which can also differentiate, but only into a few kinds of cells, thus are multipotent. For instance, blood stem cells, called hematopoietic cells, can differentiate into erythrocytes, platelets, lymphocytes and myeloid cells, but not into neurons. Mesenchymal stem cells, which maintain bone, muscle, and other tissues, can differentiate into bone, cartilage, muscle, fat, and a few other cell types. According to what we know presently, adult stem cells do not have the biomedical potential of embryonic stem cells. However, some adult stem cell therapies are promising; for example hematopioetic stem cells can be transplanted successfully. Mesenchymal cells are currently undergoing FDA-approved clinical trials for use in bone and cartilage replacement. Small samples of your own tissues might be used to generate tissues for future implantation, avoiding the dangers of immunological rejection or bacterial or viral contamination from a donor. Additionally, research on adult stem cell therapies generally does not engender the same ethical questions as does embryonic cell research.

What is Cell Therapy?

Essentially, cell therapy is a method whereby diseased cells are replaced by healthy cells. The new cells become integrated within the patient's body and treat or cure the condition caused by the diseased cells. A bone marrow transplant is one example of a cell therapy that has been used for many years to treat leukemia and other cancers. Promising new cell therapy studies are ongoing to seek treatments and cures for Parkinson's Disease, Alzheimer's Disease, diabetes, multiple sclerosis, certain heart conditions, and even spinal cord injuries.

Why is there resistance to using embryonic stem cells in research?

Embryonic stem cells come from a very early embryo, called a blastocyst, which is about five days old and 100 cells. Blastocysts are grown and harvested in the laboratory. When the stem cells are harvested, the blastocyst is destroyed. Biologically, a blastocyst is not yet an individual, yet there is concern among some ethicists and religious figures about the ethical implications of experimenting on what some people consider to be a human being with fundamental human rights.

What is Gene Therapy?

Genes are the basic physical and functional units of heredity, and encode instructions on how to build the proteins that make up the majority of cellular structures and perform most life functions. When these genetic instructions contain an error, however small, defective proteins are built, leading to genetic disease.
Gene therapy can correct diseases by introducing or modifying genetic material as a means for therapeutic intervention. There are two modes of gene therapy: ex vivo, where cells are transduced outside the body, and in vivo, where cells are transduced within the body
With gene therapy, a normal gene is transferred into the defective cell or tissue using a vector to augment or supplement the defective gene. Common vectors for gene therapy are viruses that have been genetically altered. Scientists remove the genes that encode the viral proteins, and insert therapeutic genes. The viral vector then "infects" the patient's target cells and unloads the therapeutic gene into the target cells, giving those cells the ability to produce their own functional proteins.

Are there ethical issues associated with gene therapy?

Yes, gene therapies can potentially be used for non-medical applications, like eye color, skin color, and athletic ability. So the question of who decides what genes are desirable and which are not becomes an issue.
Gene and cell therapies have tremendous potential to improve the quality and length of life. In developing these therapies, scientists must adhere to the highest ethical standards, follow the rule of law, and use sound scientific practices and principles.

Where can I go for more information on gene and cell therapies?

Stem Cell Research Foundation: http://www.stemcellresearchfoundation.org/
Human Genome Project:          http://www.ornl.gov/sci/techresources/Human_Genome/project/info.shtml
National Institutes of Health (NIH): http://www.nih.gov/
Wikipedia: http://en.wikipedia.org/wiki/Main_Page
Access Excellence Resource Center:          http://www.accessexcellence.org/RC/CC/
American Society of Gene Therapy: http://www.asgt.org/
NIH Office of Biotechnology Activities: http://www4.od.nih.gov/oba/

What is a biopharmaceutical product?

A biopharmaceutical product is a medicinal drug in which the main ingredient is a biological product, such as cells, proteins, antibodies, and the genes that are vectored to target cells to correct defective genes.

How do biopharmaceuticals get from the laboratory to the patient?

Through basic research, translational research, highly regulated human testing, and manufacturing that meets rigorous FDA standards.
Studies on how cells grow, divide, differentiate, communicate, and mutate are the foundation of discoveries that lead to biopharmaceutical drug development. Combining these studies with similar basic research on pathogenic organisms, our natural immune defenses, and many other important aspects of disease and immunity, provides the impetus for ideas on what kinds of therapies might work for different diseases. Once an idea is generated, the drug candidate is produced in the laboratory and tested for safety and efficacy. If the drug candidate proves to be safe and effective in the laboratory, it may advance to clinical trials using human volunteers. The drug development process can take many years and is expensive. The FDA will allow a drug to be produced for commercial distribution only when it meets stringent safety and efficacy requirements. Even after a drug is approved, the FDA continues to monitor the therapy's performance and associated risks.
The production of biopharmaceuticals for human use is strictly controlled, as mandated by the U.S. Food and Drug Administration. Rigorous procedures are established for the production and testing of every drug, and include specifications for facilities and equipment, personnel, and raw materials. The manufacturing process must be controlled, aseptic, reliable, and consistent. Quality systems, including Quality Control (QC) and Quality Assurance (QA) must be in place. These procedures and systems must ensure that patients receive a safe, pure, potent, and stable product.

What is cGMP?

The manufacturing processes that are utilized must be in compliance with current Good Manufacturing Practices (cGMP), which are a set of regulations enforced by the U.S. Food and Drug Administration to ensure that the procedures and controls used for producing and testing biopharmaceuticals are appropriate. The cGMPs are designed to ensure that the resulting drugs are safe, properly identified, pure, and are of the correct strength and the highest possible quality.

What is a Cleanroom?

The physical environment in which biopharmaceutical products are produced is tightly controlled. Much of the process takes place in a "cleanroom," where humidity, temperature, and airborne particulates are precisely controlled. The level of control is defined by the International Organization for Standardization (ISO), which sets standards for operational methods, equipment, and facilities, as well as standards for testing procedures to ensure that contamination of equipment and facilities is minimized. ISO classes range from ISO 1, which restricts airborne particulate matter to only 10 particles of 0.1 Ám size and two 0.2 Ám particles, to ISO class 9, which allows for 35,200,000 0.5 Ám particles, 8,320,000 1 Ám particles, and 293,000 5 Ám particles. To put these numbers in perspective, over 20 million 5 Ám particles can fit on a postage stamp. Cleanrooms have been designed to maintain low levels of particulates, and when operational, personnel are gowned when working, and the facility is cleaned frequently to reduce particulates.

What is Quality Assurance?

Quality Assurance (QA) is responsible for regulatory oversight. Specifically, QA audits and inspects the manufacturing process and all materials used in these processes. QA also monitors compliance with written procedures (i.e., standard operating procedures [SOPs] and production records). The ultimate decision on the releease of raw materials for use in production or the release of manufactured products to clients for use in clinical trials is QA's most important function.

What is Quality Control?

Quality Control (QC) is responsible for testing raw materials and all manufactured products. Through environmental monitoring (EM), QC also verifies the integrity and quality (cleanliness) of the production rooms, equipment, and personnel

Where can I go for more information?

U.S. Food and Drug Administration: http://www.fda.gov/
National Institutes of Health (NIH): http://www.nih.gov
ISO—International Organization for Standardization:          http://www.iso.org/iso/en/ISOOnline.frontpage

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