Frequently Asked Questions About Cell and Gene Therapies

Cell and gene therapy are relatively new forms of medical treatment, having only been developed about 30 years ago. As such, there is a lot that the general public has yet to understand about these practices. Here, we answer the most commonly asked questions about cell and gene therapies.

What is Cell Therapy?

Cell therapy is the process of replacing damaged or dysfunctional cells with new, healthy ones by transferring live cells into a patient. These can be autologous  (also known as self-to-self, using cells directly from the patient receiving the treatment) or allogeneic (using cells from a donor).

What is Gene Therapy?

Gene therapy is a process that modifies the expression of a gene or alters the biological process of living cells for therapeutic use. This process can take the form of replacing a disease-causing gene with a new, healthy one, inactivating a mutated gene, or introducing a new gene to help the patient’s body fight a disease.

What is The Difference Between Cell and Gene Therapy?

Cell therapy and gene therapy are overlapping fields of treatment. While the end goal of both cell and gene therapies is the same – to treat and potentially cure diseases the approach is somewhat different. Cell therapy is the transplantation of cells in order to replace or repair damaged tissues and/or cells in the body. The cells are extracted from the patient and enriched. The concentrated cells may then be injected into the patient directly. But sometimes it is important to modify the cells before they are given to the patient. This may involve transferring or altering the genetic material within the cells and is known as Gene Therapy.  

What Kinds of Diseases Do Cell and Gene Therapy Treat?

Diseases for which cell and gene therapies are a good option are those for which there is no effective treatment, those that have not responded to traditional therapies, and for instances in which patients are seeking an alternative to more invasive procedures, like surgery.

Gene therapy has been used to correct defective genes for patients with diseases such as cancer, diabetes, cystic fibrosis, heart disease and hemophilia. Recently, researchers at St Jude Children’s Research Hospital in Memphis Tennessee began using genetically modified stem cells to treat infants with severe immunodeficiency. Cell therapy is used to successfully treat diseases such as type 1 diabetes, Parkinson’s disease, amyotrophic lateral sclerosis, Alzheimer’s disease, stroke, various cancers, and spinal cord injuries.

What is Regenerative Medicine?

Regenerative medicine is a term that refers to a group of biomedical approaches to clinical therapies that may involve the use of stem cells. It is the process of replacing or “regenerating” human cells, tissues or organs to restore, or establish, normal function. The human body is an incredible machine, equipped with the ability to restore and heal itself. Regenerative medicine, then, is a process that is meant to enhance the body’s natural healing process and bridge the gap between nature and science by stimulating the body’s own repair mechanisms and restoring damaged tissues.

Why Are Stem Cells Important in Gene and Cell Therapy?

Stem cells are the cells from which all other specialized functions are generated. In other words, they are the body’s raw materials which, under the right conditions, divide to form daughter cells. These daughter cells then either become new cells or cells with a specific function, such as bone cells and blood cells. There is no other cell in the body that has the ability to generate new types of cells.

Stem cells are important to cell and gene therapies because, with their ability to self-renew and produce specialized daughter cells, they can replace a patient’s diseased cells resulting in effective treatments.

Do Cell and Gene Therapies Have FDA Approval?

While much of cell and gene therapies are still in clinical trials, there are a several treatments that have received FDA approval:

The first gene therapy treatment, Luxturna, was approved in 2017. Luxturna is used to treat patients with established genetic vision loss that may result in blindness.

Zolgensma, a gene therapy treatment for spinal muscular atrophy (SMA), was approved for use in children under two years old in May of 2019.

There are several CAR T-cell therapy treatments that have been FDA-approved. Yescarta and Kymriah have both been approved for the treatment of aggressive B-cell lymphomas in adults as of 2017.  Kymriah has also been approved to treat B-cell leukemia in children and young adults. Most recently, in July of 2020, the gene therapy Tecarta was approved for the treatment of mantle cell lymphoma in adults.

What is CAR T-Cell Therapy?

CAR T-cell therapy is a type of adoptive cell therapy (ACT), which collects and utilizes a patient’s own immune cells to help fight their cancer. As the name suggests, CAR T-cell therapy is dependent upon a patient’s T-cells, which play a critical role in managing the immune response and killing cells affected by harmful pathogens. These pathogens have unique antigens and T-cells are genetically equipped with receptors designed to identify and eliminate them. CAR T-cell therapy harnesses the antigen-fighting power of T-cells and re-training them to target and kill cancerous cells. 

When Did Cell and Gene Therapies Start?

The concepts of cell and gene therapy were introduced in the late 1970s. After years of research, and the development of the science and technology to actually carry out the processing and transfer of cells in a patient’s body, the first trial on humans was performed in 1990. Since then the treatment of various cancers, most specifically cancers of the blood immune system (Leukemias) has expanded quickly while many others such as neurological and auto-immune disorders, cardiovascular diseases and diabetes, are actively in clinical trials. FDA approvals and wide-use of cell and gene therapies in the United States are only dated as far back as 2017.

What is The Success Rate of Stem Cell Therapy?

The success rate of stem cell therapy varies depending on the disease being treated and studied. For example, stem cell therapy for osteoarthritis of the knee has an efficacy rate of 80%, while CAR T-cell therapy has been shown to have an overall patient response rate of 83% in the treatment of lymphoma.

While there is no treatment that is 100% effective for any disease, cell and gene therapies are innovative and effective options that have offered patients around the world a second chance at life.

What is ThermoGenesis?

ThermoGenesis Holdings Inc. (formerly Cesca Therapeutics Inc.), is a pioneer and market leader in the development and commercialization of automated cell processing technologies for the cell and gene therapy fields. We market a full suite of solutions for automated clinical biobanking, COVID-19 testing, point-of-care applications and large-scale cell processing and manufacturing with a special emphasis on the emerging CAR-T immunotherapy market. We are committed to making the world a healthier place by creating innovative solutions for those in need.

For more information on the CAR-TXpress multi-system platform, please contact our Sales team.