Is 2018 a Breakthrough Year for Biotech?

15 January 2018

Healthcare technology investment grew by 15.5 percent in 2017, with biotech and cancer treatments reeling in the lion’s share of dollars, according to a recent report by AngelMD. Does that mean gene therapy and other cutting-edge medical advancements are soon to be on the horizon?

The AngelMD report breaks healthcare into five categories: biotechnology, healthcare insurance, hospitals and physicians, pharmaceuticals and “other care.” It focused on biotechnology, pharmaceutical and other healthcare startups. Biotechnology was the most invested-in sector, with $13.8 billion in investments. That figure represents investment that more than doubled from 2016’s $6.3 billion in investments. Meanwhile, pharmaceutical investments fell to $2.6 billion in 2017, compared with $7.1 billion in 2016, the report showed. Overall investment in healthcare technology is $25 billion.

Medical biotechnology is under the microscope, so to speak. Medical biotech uses living cells and cell materials to research and produce pharmaceutical as well as diagnostic products that help treat and prevent human diseases. Commonly, medical biotech is used in manufacturing enzymes, antibiotics and vaccines. In addition to pharmacology, applications of medical biotech include gene therapy, stem cells and tissue engineering. It has long been an area of great hope and promise.

It’s not that pharmacological uses are new, far from it. For example, insulin was first produced in Escherichia coli through recombinant DNA technology 40 years ago, in 1978. In 1982, the first insulin using rDNA technology, Humulin R and N, were marketed. Previously, insulin for human use was produced from animal pancreas -- a complex process that could not always meet demand. At the time, Dr. Henry Miller, the medical officer in charge of Humulin at the Food and Drug Administration, told the New York Times the development was a major step forward in the scientific and commercial viability of recombinant DNA techniques, saying, ''We have now come of age.”

Roughly 10 years later, biotech heralded the advent of gene therapy, where genetic material is used to treat or prevent disease. In 1990, geneticist William French Anderson injected cells with altered genes into a four-year-old girl with severe immunodeficiency disorder. The target was to conquer deadly diseases such as AIDS, cancer and heart disease. But it’s taken researchers another 25 years to fulfill part of that promise.

Just two months ago, in November 2017, the first gene therapy was approved for sale in the United States. Kymriah, a gene therapy produced by Novartis for a form of childhood leukemia, was approved by the FDA. The drug has the ability to reprogram a cancer patient’s own cells, thereby fighting off and attacking the cancerous ones.

It’s an historic moment, similar to that of the creation of the laboratory-produced insulin. And, just as in 1982, cost is a concern. Novartis’ Kymriah is extraordinarily expensive at $475,000, compared with an average cost of $114,000 for chemotherapy using Erwinia for acute lymphoblastic leukemia. Likewise, when Humulin came on the market, it was twice the price of animal-produced insulin -- the average daily patient cost was between 50 and 55 cents a day for the treatment, a significant increase from between 26 and 30 cents.

In the years ahead, scientists will need to determine how to attack ailments that are not the result of a single genetic mutation but several. A gene-editing technique called CRISPR, might help scientists achieve some of those gene therapy goals as it can be applied to thousands of conditions caused by specific inherited mutations.



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