Clinical studies serve as a bridge between biopharmaceutical laboratories and the patients who need therapies. Drugs need to be tested in small populations before they are made available to the world at large. In a 2006 interview with
BioProcess International
, editorial advisor Michiel Ultee (vice president of Process Sciences for Laureate Pharma) said, “Until your product is tested in humans — and shown to be safe and to have some efficacy — then you really don’t have much of a claim, and you don’t really have high odds of making it to the marketplace.”
Clinical Research, Clinical Trials
Before human testing begins, companies file paperwork with the appropriate regulatory authorities. In the United States, an Investigational New Drug (IND) application is filed with the FDA through either its drug or biologics center (CDER or CBER). Elsewhere a variety of documents are filed with other agencies. In Europe, for example, a clinical trial application must be filed with the “Competent Authority” of the memb...
Biotechnology truly is global, with some form of the industry in progress on every continent but Antarctica. Countries such as Brazil are even outpacing Europe and North America in the advancement of biofuels technology. But as in the developed world where this industry began, we see other countries looking first and foremost to the medical applications of biotech — because historically that’s where the money has been. Some countries are building their own fully integrated bioindustrial sectors.
Economic development of life-science businesses is based on the development of bioclusters in which new biotech companies can develop. Many such clusters are specializing in one aspect of the industry to work in synergy with other complementary clusters — especially when one local company reaches the point at which its capabilities become a competitive asset. This requires a “critical mass” of activities to create a self-sustaining biocluster model that assures cost savings, quality adaptability, and flexibility t...
By far the most successful applications of biotechnology have been in the medical field. The vaccine industry is undergoing a complete transformation thanks to biotechnology. And cutting-edge research is giving us whole new ideas about disease therapy using nucleic acids and regenerative medicine.
Proteins and Other Therapeutics
Cancer has been a primary target for many MAb “magic bullets” and a major research area for life scientists over the past quarter-century. Oncologists have identified ∼200 cancers that affect human beings, some more dangerous than others, some more treatable. The newest mechanisms of action in oncology research include oxidative stress, epigenetics, cancer metabolism, and enzyme inhibition. Some of these are already being tested in clinical trials.
RNA Rx:
Gene therapy is based on DNA, but RNA therapy may be the next step. Recent discoveries reveal additional possibilities to target human diseases using treatments such as microRNAs and small RNA-induced gene activation (RNAa).
Re...
No segment of the biotechnology industry has received more public scrutiny than agricultural biotech — except maybe its application to food. And none has been subject to more “hype” and high hopes for instant results than biofuels. By contrast, industrial biotechnology seems almost invisible to the public at large. In general, the more immediate the effects on consumers, the more likely they are to pay attention and either laud or loathe the associated technology.
The general public doesn’t know anything about the economics of large-scale farming or agriculture’s decades-long dependence on herbicides and pesticides, and people don’t much care whether a farmer loses a crop when they can get whatever they want from someone else. And even though consumers ultimately benefit from improved industrial processes, they don’t think about them unless they’re watching an episode of “How It’s Made” on the Discovery Channel. Understanding is never guaranteed — and it is the vital key to public acceptance as well as th...
In the 1980s biotechnology began to transform medicine with the introduction of recombinant hormone treatments and “magic-bullet” drugs based on monoclonal antibodies. In the 1990s, protein kinases offered a key to cancer treatment, and gene therapies promised to address many diseases at their most basic, genetic level. The 21st Century has brought us stem cells, RNA interference, biomarkers, and predictive medicine. None of these things has supplanted the others; instead, they merely expand on the basic idea of biopharmaceuticals. The future of healthcare can be glimpsed in modern molecular medicine.
Biomarkers
The US FDA’s critical path initiative has put pressure on companies to find new and valid biomarkers in their pharmaceutical development. A
biomarker
was traditionally considered to be any specific physical trait used to measure or indicate the effects or progress of a disease or condition (wrinkles and hair loss, for example, were biomarkers of aging). But biotechnology has refined the concept ...
As new medicines, vaccines, biomaterials, and biofuels move through development, companies often face some of their toughest hurdles in moving from benchtop to production-scale processes. These are not only technological, but as technology advances it becomes more difficult to find experienced talent to make use of it. Some regional endeavors, such as the National Biomanufacturing Centre in the United Kingdom and the Massachusetts Biomanufacturing Center in the United States are pooling skills and resources to help companies overcome the technical and workforce challenges in developing the biomanufacturing capabilities these new products require.
Emerging companies face tremendous obstacles in transitioning from research to development organizations, when bioprocess scale-up issues often take them by surprise. Few contract manufacturers specialize in helping small, early stage companies make such a transition because most prefer to focus instead on more mature and experienced clients that are, quite frank...
Calivin Coolidge, who served as president of the United States from 1923 to 1929, is often misquoted as saying “The business of America is business.” According to historians, however, what he really said was, “The chief business of the American people is business.” And he went on in the same speech to say, “Of course, the accumulation of wealth cannot be justified as the chief end of existence.” So the real Coolidge (as opposed to the caricature implied by the misquote) would have been impressed by the biotechnology industry, which was spawned in his home state of Massachusetts. Its business may be to make money, but that is certainly not the chief end of its existence. There are easier ways to make money, after all. But the people involved in biotech want to improve the quality of life on planet Earth — whether through health care, new energy, agriculture, or biomaterials and industrial biotechnology.
The new US president, Barack Obama, was elected on a platform of change during a time when not only the ...
Biotechnology is not simply about science or technology, process engineering or cell lines, corporations or regulations. It’s ultimately about the people involved in all these things — and about the people they hope to serve with their biotech aspirations. It’s about scientists and technicians, businessmen and medical professionals, patients and consumers, farmers and governments, all their lives touched in one way or another by the biotechnology industry — some admittedly more than others. Policy decisions made by legislators affect how companies can do their business. Ethical considerations affect what they do and how they communicate to the public about it. And ultimately, all these things affect how many people become interested in making biotechnology their career.
Bioethics
Two subjects that currently loom large in the bioethics arena, as far as most biotech companies are concerned, are genomics and clinical research. In Chapter One, we mentioned the regulatory aspects of clinical research conducted...
