Creating Cell-Based Therapies

Before reaching patients, new treatments must first be tested in the body’s building blocks.

We are all made up of a collection of diverse and specialized cells working together to carry out the processes of life. As a result, much can be learned about human health and disease by studying these microscopic building blocks of the body. Moreover, by creating cell models with traits found in particular illnesses, scientists can elucidate the dysfunctions that underlie diseases and test therapies to correct them.

IRP scientists have made great strides in the study of both healthy and malfunctioning cells. Our investigators have illuminated cellular processes including how cells move to the correct location within the body, how cells control the activity of their genes, and how rearranged pieces of DNA can lead to cancer. These kinds of cell-based studies have led to treatments for diseases from cancer to arthritis to rare primary immune deficiency diseases (PIDDs). In particular, IRP researchers were among the earliest scientists to investigate the idea of training the immune system to battle cancer, leading to the cutting-edge field of immunotherapy and the development of successful treatments for numerous previously untreatable tumors. The IRP has also made momentous contributions to the field of gene therapy, including testing the first-ever gene therapy back in 1990.

Advancements in cell-based therapies, particularly those utilizing induced pluripotent stem cells (iPSCs) that can be turned into any cell type, could soon revolutionize the way clinicians treat HIV, cancer, neurological disorders like amyotrophic lateral sclerosis (ALS), and autoimmune conditions like type 1 diabetes and Crohn’s disease. To aid IRP researchers working in this area, the NIH’s Regenerative Medicine Program (RMP) provides resources to hasten the translation of cell-based discoveries into effective medical interventions and support the many clinical trials of these therapies being performed at the NIH Clinical Center. The RMP has also developed stem cell lines for use in research, created standard procedures for the production and use of the cells, designed training courses, and established partnerships for cell storage. In addition, in 2015 the RMP launched the Stem Cell Translation Laboratory (STCL), a state-of-the-art research center tasked with helping scientists overcome the scientific and technological hurdles of iPSC research.

With these programs supporting their work, IRP researchers are constantly making new discoveries about how our cells function and producing landmark achievements in the treatment of numerous ailments. Our investigators will continue to push forward cell-based therapies by:

• Investigating how healthy and diseased cells carry out basic processes from cell division and cell death to making energy and transporting biological materials
• Using stem cells to produce specialized cells like liver cells and neurons for the purposes of testing new therapies or directly treating illnesses
• Reprogramming immune cells to induce them to attack cancerous cells
• Establishing quality control procedures for the creation and use of stem cell lines
• Developing new, cutting-edge molecules to supplement or replace current research materials

Explore these pages for more information about the past, present, and future of IRP research on cell-based therapies:

• Stem Cell Biology Scientific Focus Area
• Cell Biology Scientific Focus Area
• The NIH Regenerative Medicine Program (RMP)
• The Stem Cell Translation Laboratory (STCL)

Check out all 12 of the domains in which we are Accelerating Science to learn about how IRP scientists are tackling important biomedical challenges.