The remarkable history of cell and gene therapy

A look into the most notable trend shaping personalized medicine in 2021

Recent advances in personalized medicine have paved the path for newer cell and gene therapies and a wave of next-generation technologies. Even though cell and gene therapies have been regarded as the future of medicine, very few people understand them and even less people know the history behind how such therapeutics have come into prominence.

To share the history of how researchers have pioneered treatments to reduce defects in the human body, IntuitiveX collaborated with Dr. Chris Xu, CEO of ThermoGenesis, to explore the advent of gene therapy and cell therapy as effective approaches.

Understanding Cells and Genes

In the field of molecular biology, life is explored and studied at the level of atoms and molecules. Central to molecular biology is the discovery that a more basic understanding of any organism could be obtained by studying the cells of which that organism is made. This foundational knowledge lead to the following principles that still dominate molecular biology today:
  1. Key to the way any cell operates are proteins, they are the building blocks cell structures are made; proteins make muscles, bones, and blood, which in turn, support most of our body’s functions such as digestion, making energy, and growing.
  2. Proteins are constructed within cells based on deoxyribonucleic acids (DNA) present in the nuclei of cells.
In short, cells are the basic building block of all living things; the human body is composed of trillions of them. Within these cells, thousands of genes provide the instructions and information for the production of proteins that play a role in determining what an organism is like, its appearance, how it survives, and how it behaves in its environment. These genes are made up of DNA, which contains all of the information necessary to build and maintain an organism.
As a result, genes in your body’s cells play an integral part in your health — a defective gene or genes can make you sick. Recognizing this, scientists have been working for decades on ways to modify genes or replace faulty genes with healthy ones to treat, cure or prevent a disease or medical condition.
Source: U.S. Food and Drug Administration
Read Dr. Chris Xu, CEO of ThermoGenesis thoughts below:
Gene and cell therapies are overlapping fields of research and treatments. While both aim to treat and potentially cure diseases, they have slightly differing approaches and historical backgrounds. Due to growing interest surrounding this field, the general public still has much to learn and understand about each of these potentially life-saving therapies.
Below, we provide a general overview and brief historical context for each type of therapy.

The History of 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 from the patient receiving the treatment) or allogeneic (using cells from a donor for the treatment). While this field of treatment has recently begun to expand, some forms of cell therapy like the cancer-treating hematopoietic stem cell transplantation (HSCT) have been in practice for decades.
While many people have heard of bone marrow transplants, few realize that this procedure is a stem cell therapy. While stem cells can be derived from many sources, such as umbilical cord blood and mobilized peripheral blood, bone marrow derived stem cell therapy is the most commonly used today and has been for more than 50 years.
The first transfusion of human bone marrow was given to a patient with aplastic anemia in 1939. After World War II researchers diligently worked to restore bone marrow function in aplasia patients caused by exposure to radiation produced by the atomic bomb. After a decade of work they were able to show, in a mouse model, that aplasia could be overcome by bone marrow treatment.
The first allogeneic HSCT, which led the way to current protocols, was pioneered by E. Donnall Thomas and his team at the Fred Hutchinson Cancer Research Center and reported in the New England Journal of Medicine in 1957. In this study six patients were treated with radiation and chemotherapy and then received intravenous infusion of bone marrow rich stem cells from a normal donor to reestablish the damaged or defective cells. Since then the field has evolved and expanded worldwide. While almost half of HSCT are allogeneic, the majority of HSCT are autologous, the patient’s own stem cells are used for treatment, which carries less risk to the patient.
In 1988, scientists discovered that they could derive stem cells from human embryos and grow the cells in a laboratory. These newly derived stem cells, referred to as embryonic stem cells (hESCs), were found to be pluripotent, meaning they can give rise to virtually any other type of cell in the body. This versatility allows hESCs cells to potentially regenerate or repair diseased tissue and organs. Two decades after they were discovered, treatments based on hESCs have been slow in coming because of controversy over their source and concerns that they could turn into tumors once implanted. Only recently, testing has begun as a treatment for two major diseases: heart failure and type 1 diabetes.

A Cell Therapy Industry Breakthrough

In 2006, researchers made a groundbreaking discovery by identifying conditions that would allow some cells to be “reprogrammed” genetically. This new type of stem cell became known as induced pluripotent stem cells (iPSCs). Since this discovery, the field has expanded tremendously in the past two decades. Stem cell therapies have expanded in use and have been used to treat diseases such as type 1 diabetes, Parkinson’s and even spinal cord injuries.
There has also been a growing focus on using other immune cells to treat cancer. Therapies such as CAR T-cell are 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 cells are then reengineered to target and kill certain cancerous cells. Several CAR T-cell therapies have been FDA approved, with the first approval being given in 2017 for Yescarta and Kymriah, to be used for the treatment of B-cell leukemia in children and young adults.

The History of 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 the mutated gene, or introducing a new gene to help the patient’s body fight a disease.
While the use of gene therapy to treat humans is fairly new, the science behind it has been used in science for decades. Farmers and geneticists have collaborated for years on crop improvement using cross pollination, genetic engineering and microinjection techniques to create stronger, more resilient crops.

Industry Advancements

The first human patient to be treated with gene therapy was a four-year old girl suffering from severe combined immunodeficiency in 1990. She received treatment for a congenital disease called adenosine deaminase (ADA). Since then, gene therapies have been used to treat diseases such as cancer, cystic fibrosis and hemophilia. In 2017, the FDA gave its first approval of a gene therapy called Luxturna, which is used to treat patients with established genetic vision loss that may result in blindness. Gene therapies are still being studied and developed, with over 1,000 clinical trials currently underway.

A Pioneer in Medtech and Life Sciences Innovation

IntuitiveX is a life sciences catalyst and innovation partner that specializes in intellectual property and growing life sciences companies no matter what stage they are in across medical devices, biotech, pharma, and digital health. With a team of IP experts, physicians, scientists, technologists, engineers, designers, and investors, we bring a combined 100+ years of experience supporting companies from ideation to commercialization. As IP & commercialization experts, we discover and unlock the potential of breakthrough ideas by applying our extensive network and resources to make them possible.

A Pioneer in the Cell and Gene Therapy Market

ThermoGenesis Holdings 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, 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.