Gene Therapy: The Future of Medicine
Only a few decades ago, the idea of altering one’s genes would have been considered science fiction, but today it is an important and promising reality. Gene therapy, the altering of genes in the body to treat or stop illness, is being explored for the treatment of many diseases that currently have no known cures such as cystic fibrosis, heart disease, diabetes, hemophilia, severe combined immune deficiency, AIDS, and various cancers.
Gene Therapy Overview & History: In 1990, the first gene therapy was conducted on a four-year-old girl with ADA deficiency. 13 years later, in 2003, the China Food and Drug Administration (CFDA) approved the first gene therapy. Today, hundreds of applications are sent to the FDA in the US each year, with 800 on file as of January 2019. The FDA has approved only a small number of applications so far; most gene therapy is still only available in clinical trials.
While it is complex in practice, the process of gene therapy is quite straightforward: blood or bone marrow is extracted from the body, exposed to genetic material, and then injected back into the body. Mutated genes are either replaced, fixed, or turned “on and off”. A gene editing tool known as CRISPR has been particularly helpful in the development of gene therapies, as explained by the video below:
Pharma’s Investment: In recent years, pharmaceutical companies have become increasingly invested in gene therapy and its possibilities. Pfizer has publicly stated its aim to create an “industry-leading gene therapy platform”, and many other firms have similar aspirations. In order to reach their goals, companies like Pfizer are collaborating with experts to develop and test successful therapies. In 2014, Pfizer joined Spark Therapeutics in the development of a gene therapy to treat hemophilia B, which has received breakthrough therapy designation from the FDA.
Other companies are acquiring firms who are successfully developing gene therapies. In 2018, Novartis entered into an USD 8.7B agreement to purchase the gene therapy company AveXis, Inc., who has developed a therapy for muscular dystrophy.
Risks, Barriers, & Pricing: Many of the risks associated with gene therapy are due to the use of viruses as the vector to carry the injected genetic material. They include unwanted immune system reactions, the possibility of the wrong cells being targeted, infection induced by the virus, and the growth of tumors. In addition to risks, there are several barriers to use, including finding a reliable way to get the desired genetic material into cells and the presence of side effects.
In addition to risks and barriers, these treatments are also very expensive. The high price tags can be somewhat balanced by their positive impacts. Not only do gene therapies save money in the long-run as they reduce the need for costly year-long treatments, but they are also mostly developed for rare diseases where only a small number of individuals need coverage.
The Future: It is inevitable that gene therapy will result in many major breakthroughs in medicine over the years to come. With a label such as “the future alternative to drugs or surgery”, there is pressure on companies to perfect it by reducing side effects and increasing success rates. This must be done before it can become a reliable treatment for many diseases. It is predicted that by 2025, the FDA in the US will approve 10-20 gene therapy products per year. What may soon be possible with the use of gene therapy is nothing but incredible and exciting. While discussing these possibilities in a meeting with c&en, Ken Mills, CEO of gene-therapy company Regenexbio, simply stated that “this year is going to be huge”.
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