What is gene therapy and what are its types?
Gene therapy is a process, usually in the medical field, which focuses on treating diseases by delivering essential nucleic acids into a patient’s body cell or sex cells. Genes influence the production of proteins, enzymes, and basically help the construction and functioning of the whole body.
If these genes turn out to be defective, they hinder normal processes in the body. Gene therapy is replaced by healthy genes to allow proper bodily functions. The first attempt at this was performed by Martin Cline in 1980 and the technology involving this process has been growing ever since.
Gene therapy can be divided into two major divisions: somatic gene therapy and germline gene therapy. In germline gene therapy, the sex cells (either egg cells or sperm cells) are modified by introducing functioning genetic material in their genetic makeup. These changes will be passed down the family line causing a permanent change for all the future generations whereas in somatic gene therapy a therapeutic set of genes are introduced to any cell of the body except the germline cells.
The modifications will cause changes in the patient only and will not be passed down the family line. Somatic gene therapy aims to help only the patient and does not permanently eradicate the disease for their family line.
How is gene therapy done?
The technique of introducing a new set of genetic material to cure an existing problem in the body cells or somatic cells requires a set of highly precise and lengthy steps. The introduction of genetic material can be done in or out of the body. This introduction requires a vector to deliver the essential genetic material. Usually, the vectors commonly used to introduce genetic material are modified viruses, fat-like molecules called liposomes, or circular DNAs from bacterias and other microorganisms.
These gene-carrying vectors can be directly injected into specific areas intravenously to allow cells to take up the material or can be done in a laboratory and then introduced into the human body to allow the gene to produce functioning proteins or enzymes.
The target tissues in this process are usually bone marrow cells, lung cells, muscle cells, or liver cells but can be carried out of any part of the body depending on the problem. The method of delivery of these functional genes is still advancing with new discoveries every day. The whole gene therapy is advancing with the availability of new nanotechnology devices and other extremely expensive devices and manpower.
What is the cost of gene therapy?
People with knowledge about what gene therapy is knowing that this procedure is extremely expensive and lengthy. This therapy aiming to restore normal activities of the cell by introducing functional genes to replace the defective genes the person is born with. There are a few genetic therapies approved by the FDA as of now.
They are two CAR T cell therapies which use altered T cell to help people suffering from a certain type of blood cancers, a treatment for inherited blindness and treatment for spinal muscular atrophy for children under the age of twelve. The number of approved treatments is on the rise with new discoveries happening every day.
In a few years, many diseases will be cured by using gene therapy but all of the therapies are very expensive. The four FDA approved gene therapies range from three hundred thousand dollars per treatment trial to millions per treatment trial. The further waiting to be approved gene therapies may have a higher price mark. A full course treatment of certain diseases like hemophilia is expected to cross $20 million, which is an enormous amount of money and an unaffordable amount for most people.
Should somatic gene therapy be available to all?
The world has come to a strange place in medical history. We have technology that promises to cure waste genetic disorders that almost cripples humans. Innovations like gene therapy will change medical practice methods and uplift the life of individuals but this technology has a very high price mark, one that the majority can not pay for. This is one of the ethical issues that arise while talking about gene therapy and whether it should be made available to all.
Every development in the field of science is commenced with the aim to better the world, tackling the existing problems. The only reason why gene therapy exists is to cure genetic disorders manipulating the genes and thus uplift the health and living part of people and therefore this technology should be made available to all regardless of wealth.
Making somatic gene therapy available for all makes the treatment of the disease a one-time investment. The cost of the treatment will be high, but the investment will be a one-time investment that ensures the complete eradication of the problem. This somatic gene therapy treatment tackles the problem itself rather than subside the issues or tackle the issues.
In addition to ensuring the solution to the problem in the human body for his or her lifetime, it is also a cost effective method. Most of the medical practices regarding genetic disorders treat only the symptoms of the disorder. This causes a lifetime expense on medication and risks further complications in the future.
The complications arising from the disorder increases the amount of expense and affects the quality of life of the affected person and the people around him. Therefore, although the price tag of gene therapy treatment is extremely high, if the amount of money spent on health care and quality of life is compared without gene therapy, gene therapy saves money in the long run.
The cost of lives saved and improved is always greater than the cost of production and manpower. When the quality of life is improved for a person, its ripple effect influences its society and also the nation. Healthy working individuals means the country has to spend less on health care methods and will gain more benefits from them. More healthy working individuals ensure the improved economy of the country and its development. Providing genetic therapy or subsiding its costs is fruitful for the nation in the long run too.
The benefits of gene therapy are widespread and promising. If the gene therapy works for the patient, it drastically changes the life of the affected person and the people around him or her but these treatments do not always work. There is no evidence to show that every person that gets the treatment will get rid of the genetic disorder.
This uncertainty causes doubt as to whether to provide somatic gene therapy available to people or not. The argument to center this is that the percentage of people that do not benefit from somatic gene therapy is a very small fraction of people compared to the people whose life changes after this treatment. The positive side of this treatment therefore outweighs the uncertainty it follows.
Gene therapy’s timeline of development is slowing down due to lack of its practice. It can only develop further if this is practiced on people and learn from it. Further development of this technology will cause the discovery of newer possibilities and thus lower the cost and remove the uncertainty altogether.
It has the potential to revolutionize health care mechanisms and drastically improve the quality of life of people. Therefore in conclusion, regardless of wealth, somatic gene therapy should be made available to people who wish to do it.