Biotechnology: 21st century medicine

The 21st century might be a safe time to get sick. As you read this, scientists are searching for ways to take genetic material from one individual and fuse it with the genetic material of another in order to repair genetic deformities. Researchers are attempting to map all three billion base pairs of the human genome. And biologists are discovering ways to tailor drugs to fight diseases that occur in specific individuals.

All of these breakthroughs have one thing in common: They all come out of the biotechnology industry, one of the fastest growing industries in the United States.

An industry is born

The Biotech Industry Organization, an industry trade group, traces the birth of the modern biotech industry back to 1973, when Herbert Boyer and Stanley Cohen completed the first successful attempt to recombine DNA. This process, which is one of the basic tools of biotechnology, works by using enzymes to cut and remove individual genetic information from one organism and transplant - or recombine - it with that of another. In 1976, Boyer co-founded Genentech, which later became the first company to market a recombinant DNA drug. The drug, human insulin, was licensed to Eli Lilly and Co.

With the exception of the Internet, few industries have matched the unprecedented growth of the biotech industry. Within the last year alone, the industry went from $16.1 billion in revenue in 1997 to $18.6 billion in revenue in 1998. Approvals for new indications for biotech drugs and vaccines have also risen steadily since the early days of the industry, going from two in 1982 to 21 in 1998. These numbers might not seem impressive to those in the pharmaceutical industry, but consider that approximately one-third of the 1,283 biotech companies in the United States employ fewer than 50 employees, and more than two-thirds employ fewer than 135 people.

But it would be inaccurate to say that the growth of the biotech industry has been consistent over the last 10 years. Like any new industry – especially one in health care – expectations can be inordinately high, a fact that makes setbacks even more disappointing. "People 10 years ago may have overestimated the ability to get answers quickly," said Jim Shire, managing editor of Bioworld, an online and print bio-tech industry newspaper.

"The industry now has matured to the level that it realizes there's probably going to be answers out there but they're not going to occur overnight."

Charlie Craig, director of publications for the Biotech Industry Organization in Washington D.C., said that although setbacks can be disappointing, they are often no more noteworthy than those experienced in the pharmaceutical industry. "The expectations of gene therapy were high because of what gene therapy was trying to tackle," said Craig, "It takes a long time to develop these drugs. Gene therapy is not progressing any slower than any other drug development."

The smaller size of most biotech companies compounds investor disappointment when failures do occur. "It's the economics of the full [pharmaceutical] industry, but centered on one or two things," said Scott Clarke, chief operating officer at Biospaces.com, a Web site devoted to news and information on the biotech industry. "Where big pharma might have hundreds of things that they're going forward with at various stages you're talking about most biotech start-ups having one or two - maybe one lead and a few backups in a therapeutic class."

Because of the small size of most biotech companies, many of them are aligning themselves with pharmaceutical companies. "Everyday we're writing about collaborations between pharmaceutical companies and biotech companies," said Shire. "They need each other and they will continue to need each other. Biotech companies help fill the pipelines of pharmaceutical companies. Pharmaceutical companies provide the funding, regulatory expertise and marketing that many biotech companies need."

Added Clarke, "What biotech is doing is getting leads and going forward, but the best group that's actually able to take [a product] and get it through the regulatory process and take it into the whole sales and marketing side is definitely big pharma."

Even larger biotech companies frequently partner with the pharmaceutical industry. Over the years, Genentech has entered into partnerships with Schwarz Pharma, Roche and Pharmacia & Upjohn, to name just a few.

And when biotech companies are not partnering with pharmaceutical companies, economic factors often necessitate that smaller biotech companies partner with the larger ones, a trend Shire believes will only continue in the future. "The smaller companies don't partner that often with one another, although they do sometimes," he said. "What's more common might be mergers and acquisitions, but the small companies most often partner with larger companies."

Right now, the National Institutes of Health and several private companies, like Rockville, MD-based Celera Genomics and Human Genome Sciences Inc., also of Rockville, are attempting to map out each of the three billion base pairs in the human genome. Once this is done, the information provided will open doors for research into new biotech products, and rapidly accelerate the growth of the biotech industry.

Researchers anticipate that, once the human genome is mapped, they will be able to isolate the genes that cause diseases and specifically target them for genetic therapy. Some drugs may even be able to target specific individuals – an area of research called pharmacogenomics (see sidebar, page 19) – so that they can receive personalized pharmaceutical therapy.

With the possibility that the human genome will be mapped within the next five years (see Pharmaceutical Representative, December 1999), the future of the biotech industry looks strong. "Over the next couple of years, we're going to know all the genes in the genome, which is pretty amazing when you consider that all the drugs discovered to date have probably been based on the knowledge of one gene," said Clarke. Added Graig, "It will help in discovering disease genes, it will help in discovering the molecular causes of disease and it will give [scientists] more molecular targets for drugs, so they know exactly what they're aiming at." PR