Since the first gene transfer in 1989, gene therapy has been gathering pace at such a rate it may replace more traditional methods. Previously, small molecular pharmacology was the only treatment for genetic conditions, that may change as progressive methods of gene therapy have developed and have the potential to be a long-term solution. Nonetheless, the consideration for both sides must be looked at in more detail.
What Is Gene Therapy?
Gene therapy is a treatment used to help patients by altering their genes to cure or reduce the effects of their genetic condition. It has been defined by the European Medicines Agency (EMA) as using a recombinant nucleic acid with the purpose of adding, deleting, or repairing a genetic sequence, and the preventative or diagnostic effect of this is linked exactly to the nucleic acid sequence or as a result of the expression of the sequence.
The initial concept of gene therapy was first projected around fifty years ago with limited success. However, scientists have learned from their failures and now believe that gene therapy may be able to help cure problems that were once thought incurable, such as neuromuscular diseases, immunodeficiency, and blindness.
One area that has seen huge leaps in progress is the use of the CRISPR (clustered regularly interspaced short palindromic repeats)/Cas9 system in gene therapy, which has become more widespread in recent years. This mechanism has long been used in some bacterial immune systems to fight off attacks from viruses by using Cas9 proteins, part of the virus’ genome is inserted into the bacteria’s genome, meaning that the bacteria has DNA that can be transcribed into RNA that acts as a template for Cas9 to target DNA of the same sequence.
However, CRISPR can also be used for genetic modification in organisms, in this case, Cas9 proteins associate with the template RNA that is desired by scientists and cut DNA so that the RNA can bind. How the organism repairs this cut will determine if the gene loses its function or, through homology-directed repair, if the gene will repair into the correct form of the gene.
Gene Therapy vs. Small Molecule Pharmacology
Small molecular pharmacology is the theory of using drugs to treat diseases and infections on a molecular level; looking at how the shape of a compound can have a different effect to that of a similar isomer or seeing how specific compounds or atoms can act as receptors to trigger a reaction in the body.
Each condition requires individual comparison. Take, for example, Huntington’s disease. Huntington’s disease (HD) is a genetic condition that affects neurological function resulting in the patient suffering from involuntary movements and a decrease in cognitive ability. There is no current cure for HD, and only side effects can be treated, such as antidepressants, high-calorie diets, and installing grab rails. However, scientists believe they have identified the gene responsible (HTT) and with early intervention believe that HD could be stopped with gene therapy (Glorioso et al., 2015). So, in this case, gene therapy may be far more beneficial for a disease that affects 4-8 per 100,000 people across Europe.
In cystic fibrosis, the cystic fibrosis transmembrane regulator (CFTR) gene does not release chloride ions, which leaves an incorrect salt balance, and thick mucus builds up in lung cells and blocks the pancreas, stopping digestive enzymes from reaching the intestine. There is no current cure for this, but medication can be used to treat symptoms. The potential for future treatment does look more promising as gene therapy has already tested various ways of inserting the CFTR gene into the cell of the lungs. Drugs may also be developed through scientists developing an understanding of bacteria genomes to discover potential medications that may prevent lung infections that are fatal for patients with cystic fibrosis.
In most cases, using drugs to treat a condition is easier to produce, more accessible, and more cost-effective in the short term. Nonetheless, if a treatment has the ability to cure a genetic condition, it could be cheaper as a one-off treatment. It could mean the patient would have a less complicated life instead of taking medication repeatedly or in cases where no drugs are available to target the condition directly. Despite this, it is only financially viable to produce such treatments where the genetic condition affects enough people. Should a very rare condition affects few people globally, there is a very little financial incentive to develop a gene therapy solution if drugs can offer reduced side effects.
Future Advances
Gene therapy has made huge advances so far from its inception with the first gene transfer experiment in 1989 but has the potential to continue to expand to the point of developing treatments for genetic and infectious diseases. It has been suggested that genetic modification through CRISPR and other methods could lead to ‘choosing’ genes and poses major ethical questions. Despite this, the opportunities implied mean a hopeful future for people suffering from genetic conditions.
Small molecular pharmacology is still extremely important and will always have a place as a treatment for infections and symptoms. There are many advantages over gene therapy: the ethical issue of ‘choosing’ how someone is edited is removed, as well as being more widespread in development, and has more previous research to fall back on.
Conclusion
In my opinion, I feel that gene therapy will become more common than small molecular pharmacology for the treatment of genetic conditions because of the long-term fix it poses to the patient. However, I do believe that it must be regulated to avoid becoming commercialized and used as a way of genetically choosing the perfect genes. Additionally, I believe that the use of drugs will still be popular for conditions that are not genetic like viral infections as they are cheap, easy to access, and somewhat trusted by the public due to the length of time they have been around.
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