Gene therapy is an advanced means of treating cancer that promises to address the disease’s root cause: our genes. It involves correcting or replacing faulty genes within cancer cells to prevent further growth or make them more susceptible to removal. This new technology provides hope for targeting cancer in a more precise and effective way.
Unlike other traditional treatments, like chemotherapy or radiation, gene therapy is actually the replacement of faulty genes, the introduction of new ones, or modification of existing ones in a way to suppress the growth of cancer or to strengthen the body’s natural defenses.
This new approach may bring better targeted and personalized cancer therapy. Gene therapy tries to treat the problem at its very source: understanding the genetic mutations causing cancer, thereby reducing the possibility of harming healthy cells. Although still in its developing stage, it has shown promise in treating certain cancers, thus offering new hope to patients where conventional methods might not be enough.
In this blog, we’ll explore what gene therapy is, how it works in cancer treatment, its potential benefits, and the challenges involved in making this therapy widely available.
What is gene therapy and how does it work?
Gene therapy represents a new medical approach at disease treatment or prevention through the direct modification of the genetic material within the cells of a person. This type of treatment is directed toward the root cause of genetic diseases, which lies in their abnormal genes that need correction or compensatory mechanisms to counteract or rectify diseased state development.
How Does Gene Therapy Work?
Gene therapy involves several basic steps:-
Therapeutic Gene: Healthy copies of the defective genes are prepared in the lab. These therapeutic genes, thus prepared, are capable of replacing the faulty gene so that the malfunctioning genes may be inactivated or introduced into the system to assist in combating the disease.
The therapeutic gene is introduced into patient cells via a vector often a genetically modified virus, such as an adenovirus or lentivirus engineered to carry genetic material so that it cannot cause an infectious disease.
- The therapeutic gene is integrated into the genome of the cell after transfection of the target cells or remains as an extra-chromosomal element, which makes the cell produce the desired proteins reversing the disorder.
- Therapeutic Effect: The expression of the introduced gene results in the production of functional proteins or inhibition of deleterious ones, relieving symptoms or curing disease.
This holds very significant potential in treating different diseases, including the genetic disorders like cystic fibrosis, hemophilia, and some types of cancer. Gene therapy is still at an under-developed stage and so continues to be researched with greater safety, efficiency, and modes of delivery.
How Is Gene Therapy Applied?
Gene therapy is an orderly and targeted treatment aimed at working effectively and safely. Here is a rundown on how this treatment method is administered:
Selection of the Vector- The vector usually carries the therapeutic gene in it; a modified virus that may bring the genetic material into the body in a safe manner. Popular vectors are:
Viral Vectors- Adenoviruses, lentiviruses, or retroviruses are genetically engineered to introduce the therapeutic gene without causing the disease.
Non-Viral Vectors- Lipid-based nanoparticles or plasmid DNA can also be used for gene transport.
Delivery Method- The selected vector containing the therapeutic gene is administered into the patient through one of the following methods:
- In Vivo Therapy: The vector is injected directly into the body of the patient, with the aim of targeting certain tissues or organs. In cancer treatment, for instance, the vector may be injected into a tumor.
- Ex Vivo Therapy: Cells are extracted from the patient, genetically modified in the lab, and then reintroduced into the body. It is mainly applied in the treatment of blood disorders and immune-related diseases.
Targeting Specific Cells- Gene therapy is targeted at the affected cells or tissues. For example:
In genetic diseases, the therapy focuses on correcting the defect in the gene in appropriate cells
In cancer, it could be a matter of reprogramming immune cells to identify and destroy the cancerous cells.
Follow-Up and Monitoring- Monitoring should be done after administration in patients to evaluate its effect and look out for side effects. Long-term follow-up should be there to confirm the safety of gene modification overtime and make sure it achieves its intended purpose.
Gene therapy is a highly technical treatment that requires precision and high expertise in its administration and is, therefore, mostly offered in advanced medical facilities. With the ongoing research into these delivery techniques, many hope to treat a variety of genetic and acquired conditions.
Are there potential side effects of gene therapy?
Despite having such tremendous potential in the treatment of various genetic disorders and diseases, gene therapy, just like every other medical treatment, remains a developing field. Side effects are not impossible either; they depend upon the kind of therapy, its method of delivery, and the disease for which it is intended to treat. However, researchers and doctors still try to make gene therapy safer and more efficient.
Following are a few side effects of gene therapy:
Immune Response
The most common side effect of the treatment is actually an immune response to the viral vectors used for introducing the therapeutic gene. Since these are the vectors created using a virus as an element, in certain situations, the immune system would consider these as alien components; the immune response could thereby elicit symptoms like fever or even induce allergic response accompanied with swelling. In rare cases, the immune system may even destroy the introduced gene or the modified cells, thereby reducing the efficiency of the therapy.
Insertional Mutagenesis
This results when the transferred gene disrupts other essential genes in the patient’s DNA. This has resulted in adverse effects such as causing cancer. This occurs infrequently, but it is significant and, as more research continues, this is minimized through the use of safer vectors and delivery mechanisms.
Infection or Inflammation
In some instances, the introduction of viral vectors leads to infection or inflammation at the injection site or in the treated tissues. This is more likely to be seen with the in vivo methods of gene delivery where the gene is administered directly into the body. The patients are observed for signs of infection and medication is given to control any inflammation or infections that are developed as a result of the treatment.
Organ-Specific Reactions
Reactions based on the specific organ targeted. Sometimes the therapy causes reactions to a certain organ or tissue. The person would then have liver toxicity or damage if it targets the liver. These reactions are traced by conducting regular tests on the patient while undergoing the therapy so that it does not have a permanent adverse effect on critical organs.
Short-Term Side Effects
Some patients may present with short-term side effects, which can be fatigue, nausea, or headache, especially soon after the therapy is given. These are usually mild and can be managed with supportive care.
Unpredictable Results
Since gene therapy is still an evolving treatment, its long-term consequences are not known completely. At times, the intended therapeutic effect may not work; it may not even influence the target cells to make those required changes in their genetic content. This unpredictability, though, is one challenge since it deals with the treatment of genetic conditions whose cause might be complex.
Despite these possible side effects, gene therapy has shown promising results on diseases that otherwise could not be treated. Doctors diagnose the condition of each patient and meticulously plan the therapy so as to reduce the risk factors as much as possible while enhancing the potential benefit. Continuous research and clinical trials have made gene therapy safer and further widened its applications.
Contact Dr. Pooja Babbar
Dr. Pooja Babbar is an excellent and caring medical oncologist in Gurgaon who strives for providing the best cancer treatment. She has more than ten years of experience. She has effectively treated tens of thousands of patients based on the latest chemotherapy, immunotherapies, and target therapies. Her personal approach focuses on tailoring the perfect treatment for each patient.
Whether it is the management of complex cancer cases or emotional support throughout treatment, Dr. Babbar’s expertise and commitment make her a very trusted name in oncology. If you or any of your loved ones seek expert cancer care, here is a dependable choice for your treatment and recovery: Dr. Pooja Babbar, Gurgaon.
