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Gene Therapy

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...

Stem Cell Therapy for Tissue Regeneration

Stem Cell Therapy for Tissue Regeneration

Stem cell therapy is a method used in regenerative medicine to repair and restore function to damaged tissues and organs. Stem cells have the potential to proliferate and differentiate into various tissue types, making them ideal candidates for tissue regeneration. For example, hematopoietic stem cell transplants are commonly used in blood cancer treatment to replenish damaged bone marrow and restore healthy blood cells.
Types of Stem Cells used in Stem Cell Therapy
The two main cell...

Cancer Therapies

Cancer Therapies

Cancer therapies are various modes of treatment, such as surgery, radiation therapy, and chemotherapy that are administered to cancer patients.
However, cancer treatments can pose several challenges, as therapies used to kill cancer cells are generally also toxic to normal cells. Moreover, cancer cells mutate rapidly and can develop resistance to chemical agents or radiation therapy. Besides, all types of cancer cells may not respond to the same therapy. Some cancer cells respond to one...

Targeted Cancer Therapies

Targeted Cancer Therapies

The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against...

Mesenchymal Stem Cells

Mesenchymal Stem Cells

Mesenchymal stem cells (MSCs) are adult stem cells that can differentiate into most connective tissue cell types, except for hematopoietic cells, depending upon the source of MSCs. For example, bone-marrow-derived MSCs (BM-MSCs) can differentiate into osteocytes, hepatocytes, and pancreatic and neuronal cells. MSCs can be isolated from various sources such as bone marrow, placenta, adipose tissue, teeth, and Wharton’s jelly, a gelatinous substance in the umbilical cord. The ease of their...

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Gene and Cell Therapy for Sarcomas: A Review.

Sant P Chawla¹, Skyler S Pang¹, Darshit Jain¹

¹Sarcoma Oncology Center/Cancer Center of Southern California, Santa Monica, CA 90403, USA.

|April 14, 2025

Summary

Gene and cell therapies show promise for treating sarcomas, with a few already approved. Further research and clinical trials are advancing these innovative treatments for better patient outcomes.

Keywords:

CAR-T therapy cell therapy gene therapy natural killer cell therapy sarcoma solid tumors virotherapy

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Tumorsphere Derivation and Treatment from Primary Tumor Cells Isolated from Mouse Rhabdomyosarcomas

Tumorsphere Derivation and Treatment from Primary Tumor Cells Isolated from Mouse Rhabdomyosarcomas

Published on: September 13, 2019

Area of Science:

Oncology
Molecular Biology
Immunotherapy

Background:

Sarcomas present treatment challenges due to heterogeneity, recurrence, and metastasis.
Developing effective therapies for these rare cancers remains a critical need.
Current treatment limitations necessitate exploration of novel therapeutic strategies.

Purpose of the Study:

To comprehensively review gene and cell therapies for sarcomas.
To analyze current literature and ongoing clinical trials in this field.
To identify approved therapies and emerging treatment modalities.

Main Methods:

Conducted a comprehensive literature search across PubMed, Medline, Google Scholar, and clinicaltrials.gov.
Utilized keywords including "gene therapy," "cell therapy," "CAR-T therapy," and "sarcoma."
Reviewed clinical trial data, noting NCT numbers, status, condition, and phase.

Main Results:

Only three gene and cell therapies for sarcomas have received regulatory approval.
Approved therapies include Rexin-G (2007, Philippine FDA), Gendicine (2003, China), and Afami-cel (2024, US) for synovial sarcoma.
Numerous other promising therapies are under investigation and progressing through clinical development.

Conclusions:

Gene and cell therapies offer significant future potential for sarcoma treatment.
Advancements in research are moving these therapies toward late-stage clinical development.
Integrating these novel therapies into standard care could improve patient quality of life and outcomes.