Gene Therapy – SunAct Cancer Institute

Therapies

What is Gene Therapy?

It is a technique that involves altering the genetic material of cells to treat or prevent disease. It aims to introduce a normal, functional gene to compensate for the defective, disease-causing gene.

How does Gene Therapy work?

It is a technique that involves altering the genetic material of cells to treat or prevent disease. It aims to introduce a normal, functional gene to compensate for the defective, disease-causing gene.

The different aspects of gene therapy are :-

It employs various approaches: replacing a mutated gene with a healthy copy, inactivating a mutated gene, and introducing a new gene.
Unlike traditional drugs, gene therapy targets the root genetic causes inside cells.

Target Cancers

Gene Therapy is primarily used to treat:

Hematologic cancers: Such as leukemia, lymphoma, and multiple myeloma.
Non-cancerous blood disorders: Aplastic anemia, sickle cell anemia, and thalassemia.
Genetic disorders: Severe combined immunodeficiency (SCID) and certain metabolic disorders.
Solid cancers:- Brain tumors, gynaecological malignancies and other solid malignancies

Gene Therapy Focused on Cancer

Suicide gene therapy
Angiogenesis targeting therapy
Immunization gene therapy
Targeting cancer associated fibroblasts
Targeting tumor cells derives exosomes
Genome editing
miRNA targeted therapy
Transcription factor decoys
Oncogene silencing
Tumor suppressor gene replacement

How effective is this treatment?

In cancer treatment, gene therapy has demonstrated potential in enhancing the effectiveness of immune therapies, targeting tumors more precisely, and improving patient survival rates.

Source: Gene Therapy for Cancer Treatment: Past, Present and Future - PMC

Who is eligible for Gene therapy?

Gene therapy is typically recommended for patients with advanced cancers who have not responded to conventional treatments like chemotherapy or immunotherapy. A thorough medical evaluation is required to determine eligibility.

Risks and Side-Effects

Immune response is stimulated, which can reduce the effectiveness of Gene Therapy.
Viruses used as vector for gene transfer may cause toxicity, immune response, and inflammatory reactions int the host.
Disorders caused by effects in multiple gene can’t be treated effectively using gene therapy.
They can carry a limited amount genetic material. Therefore, some genes may be too big to fit into viruses.
Random insertion can disrupt normal gene.
Gene therapy holds immense potential for revolutionizing healthcare by providing novel treatments for genetic disorders, personalized medicine, and targeted cancer therapies. However, it also comes with inherent risks and ethical challenges that must be carefully considered and managed.

Next steps if Gene therapy doesn’t work

Alternative options may include:

Other immunotherapies
Targeted therapies
Participation in additional clinical trials
Supportive care to manage symptoms and improve quality of life.

Insurance Coverage

Insurance coverage varies by provider and plan. Patients are advised to consult with their healthcare provider and insurance company to understand coverage options and potential costs.

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Therapies

What is Gene Therapy?

How does Gene Therapy work?

Target Cancers

Gene Therapy Focused on Cancer

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