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CELL THERAPY 101 

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What is regenerative medicine?

Regenerative medicine is a broad definition for innovative medical therapies that will enable the body to repair, replace, restore and regenerate damaged or diseased cells, tissues and organs. This broad field encompasses a variety of work areas including cell therapy, transplantation, growth factors, tissue and biomaterials engineering. Regenerative medicine promises to extend healthy life spans and improve the quality of life by supporting and activating the body’s natural healing.

What is cell therapy?

The FDA defines cell therapy as, “The prevention, treatment, cure or mitigation of disease or injuries in humans by the administration of autologous, allogeneic or xenogeneic cells that have been manipulated or altered ex vivo.” The goal of cell therapy, overlapping with that of regenerative medicine, is to repair, replace or restore damaged tissues or organs.

What are stem cells and why are they important?

A stem cell is a cell (either adult or embryonic) that is capable of indefinite renewal through cell division and retention of its generic or unspecialized state while at the same time maintaining its potential to give rise to daughter cells of a more specialized type.

What are embryonic stem cells?

Embryonic stem cells are derived from embryos, specifically the blastocyst, a hollow ball of cells that forms approximately five days after conception. Embryonic stem cells are pluripotent, meaning they have the ability to differentiate into any of the 200-plus cell types required by the body and contain the most long-term promise for novel cell therapies and tissue regeneration. Most embryonic stem cells used for research today in US have been donated from excess blastocysts created during in-vitro fertilization.

What are adult stem cells?

Adult (Somatic) stem cells are unspecialized cells that are found in different parts of the body and are capable of self-renewal and give rise to daughter cells that are specialized to form the cell types found in the original body part. Recent scientific studies suggest that adult stem cells may be pluripotent having the ability of a stem cell from one tissue to generate the specialized cell type(s) of another tissue.

Where do stem cells come from?
  • The first human stem cells were embryonic stem cells isolated in 1998 at the University of Wisconsin from excess embryos obtained from in-vitro fertilization clinics.
  • The first human adult stem cells used for transplant in the 1970’s came from bone marrow and were used to treat hematologic malignancies (leukemias, lymphoma…)
  • Subsequently, adult stem cells have been found and isolated from a long variety of adult tissues: from adipose (fat) tissue, bone marrow, peripheral blood and umbilical cord blood. Recently stem cells have been found in skeletal muscle, skin, hair, blood vessels, retina, liver, pancreas brain and teeth
How is cell therapy being used today?

* Bone marrow transplants have been used for the past 40 years to regenerate the blood and immune systems of patients with leukemia, lymphoma, severe aplastic anemia or inherited metabolic diseases. Unfortunately, the major limitation with allogenic (different donor and recipient) bone marrow transplants is the availability of matched donors.

*Umbilical Cord Blood (UCB) stem cell have emerged as an alternative to bone marrow transplants, providing an easily obtainable and readily available source of treatment.

 

  • UCB transplants are mostly limited to pediatric patients due to the low cell stem cell dose.
  • UCB transplants have less stringent requirements for donor matching compared to bone marrow transplants, increasing the likelihood that an appropriate donor can be found for patients.
  • UCB transplants may result in a lower incidence of transplant complications and immune host disease, common in patients receiving a transplant from an unrelated donor.
What are the potential uses of stem cells in the future?

In addition to regenerating the blood and immune systems, scientists and physicians anticipate that stem cells will be extensively used to replace damaged or diseased tissues and organs.

  • Clinical trials are ongoing to repair scarred or dying heart muscle after a heart attack or during congestive heart failure.
  • On-going research in diabetes is focused on understanding how stem cells might be trained to become the type of pancreatic islet cells that secrete needed insulin.
  • Repair of debilitating spinal cord injuries is also a goal of researchers through the regeneration of neurons, myelin and nerve cells.
  • Stem cells may be used to generate liver cells or other tissues that can be used in screening new drug candidates for safety in pharmaceutical drug development. Using human cells and/or tissues may provide a better model for toxicology testing than the traditional animal models in use today.
  • Stem cells may also be used to bring chemotherapeutic agents directly to the targeted cancerous cells. Cancer vaccines, a type of adoptive immunotherapy, are in clinical trials for prostrate, breast, ovarian and colorectal cancers. Combining tumor cells from the patient with dendritic cells can lead to a vaccine that will seek out and destroy the cancerous cells.
What needs to be overcome before cell therapies move into widespread use?

Before cell therapies move from the present basic lab research and basic clinical use into widespread use in for medical treatment, several technical obstacles must be overcome.

  • Identify, isolate and purify different adult stem cell types. Purified and/or expanded stem cells will be required for safe and efficacious treatments.
  • Control the differentiation of stem cells needed to treat the disease such that sufficient quantities of the differentiated cell can be generated for treatment.
  • Demonstrate clinical improvement and normal cell development and function once stem cells have been transplanted into the patient’s body. Stem cells must become integrated with the patient’s own tissues and learn to function as one of the patient’s natural body cells.
  • Better understand and control the mechanism of turning undifferentiated cells into specialized cells. This involves identifying the complex signals needed to turn the genes on and off that initiate and govern the differentiation of cells.

As basic lab research and basic clinical use continue, Scientists worldwide are presently engaged in research activities that may enable repair of damaged heart muscle after a heart attack, replacement of skin for burn victims, restoration of movement after spinal cord injury and regeneration of pancreatic tissue to produce insulin for people with diabetes.

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