What is Cell and Gene Therapy for Cancer?
Gene therapy is a technique that uses genes to treat or prevent disease such as cancer by inserting a gene into a patient’s cells instead of using drugs or surgery. Researchers are testing several approaches to gene therapy, including: replacing a mutated or abnormal gene that causes disease with a healthy copy of the gene; inactivating, or “knocking out,” a mutated gene that is functioning improperly; and introducing a new gene into the body to help fight a disease.

Cell Therapy is the infusion or transplantation of whole cells into a patient for treatment of an inherited or acquired disease like cancer.

Primary Forms of Cell and Gene Therapies for Cancer Treatment

  • Healthy gene medicines that are injected into the body to train the immune system to destroy cancers, repair the damage, and reject new cancers.
  • Individualized drugs delivered directly to the tumor to facilitate cancer cell death.
  • A vaccine that strangulates cancer cells by cutting off their blood supply.
  • An inoculation that ultimately prevents cancer.

The long-term goal of cancer cell and gene therapy is to develop treatments that attack only cancer cells, eliminating adverse effects on the body. Furthermore, these therapies have potential for treating other diseases such as cardiovascular, disorders, cystic fibrosis, hemophilia, sickle-cell anemia, muscular dystrophy, diabetes, and Parkinson’s. All research in this area, therefore, makes a difference.

About Molecular Medicine

Molecular medicine uses the body’s own cells and genes as both the source and medicine for diseases of all types – the basis for all cell and gene therapies.

Molecular medicine began with the identification of DNA in the early 1900’s. Progress was slow until the mapping of the human genome in the new millennium and the rapid technological advances that made it possible to isolate and target specific cells and genes.

This field of study explains the fundamental genetic errors that cause diseases like cancer and helps establish a blueprint for good health.

Molecular medicine and advanced technology make it possible to target cancers directly without damage to other parts of the body.

Molecular medicine is also referred to as genetic medicine, gene therapy, targeted therapeutics, genetic epidemiology or individualized medicine.

“I am extremely grateful to ACGT for funding this next phase of our work,” says Dr. Kasahara. “At a time when federal funding has become scarce, it’s wonderful and inspiring to see private foundations like ACGT step up to support the potential of innovative science. Together, we will continue our fight to find better ways to help cancer patients.” Read more about ACGT Fellow Noriyuki Kasahara, MD, PhD and his groundbreaking work in brain cancer