Stem cell research is still growing. A gaggle of ongoing studies and research projects are delving into the complexities of human stem cells but at the moment there are only a handful of clinical uses with regulatory approval.
You’re an organic being composed of genetic material. Your thick, flowing hair and your red cheeks are like family heirlooms. Unfortunately, some of your genetic package may be flawed. Debilitating diseases can be passed on through generations.
Many researchers believe that stem cell therapy can be used to treat genetic diseases.
“Right now, there are no cures for mitochondrial diseases,” Juan Carlos Izpisua Belmonte, a professor in Salk’s Gene Expression Laboratory, explained. “Very recently, we’ve developed ways to prevent these diseases, so it was natural to next ask how we could treat them.”
Your mitochondria come from your mother. They’re present in every cell in your body. Mitochondrial diseases are triggered by mutations in the genes and can cause crippling disabilities.
The ability to treat such diseases would transform mankind.
Adult vs Embryonic Stem Cells
Embryonic stem cells come from embryos at the beginning stages of their development. Adult stem cells are found in the body post-development. Both might be useful in treating genetic diseases.
Common Genetic Diseases
Mankind is afflicted with a host of genetic diseases. Common examples include
- Thalassemia: This condition affects the amount of hemoglobin that the body can produce. Parents with thalassemia have a 25 percent chance of passing their condition onto their children.
- Down Syndrome: Modern prenatal testing can determine if a child will be born with Down syndrome. It occurs when the cell’s 21st chromosome is copied too many times. Older mothers are more likely to have a baby with the condition.
- Sickle Cell Anemia: Sickle Cell Disease is a serious condition that’s passed along by the Sickle Cell trait. The disease causes red blood cells to have a warped shape.
- Cystic Fibrosis: This is a chronic condition that interferes with the patient’s digestive, respiratory, and digestive systems. The median age of survival is in the mid-30’s.
These are the types of conditions that stem cell therapy may one day be able to treat. Each one is incurable.
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What Can We Do Today?
Most stem cell therapies are highly experimental. Very few stem cell treatments have been approved for human patients.
A strong body of evidence shows that blood stem cells are a useful therapy for blood disorders and health conditions affecting your immune system. The treatment is widely used across the globe. Every year, more than 26,000 patients in Europe are treated with blood stem cells.
Stem cells are also used to stimulate skin graft growth in patients suffering from serious burns on their bodies. Certain bone and corneal diseases and injuries can also be treated by stem cell grafts. In these cases, stem cell therapy is usually reserved for patients with life-threatening injuries.
These are the sole stem cell therapies that have been vigorously tested as well as deemed safe and effective.
Why Are Stem Cell Effective?
Genetic diseases arise from flaws or mistakes in the cell. Patients can use donated tissues but the supply is too small to meet the demand. Stem cells, according to a 2014 study conducted by Pharmacy & Therapeutics, “seemingly provide a renewable source of replacement cells and tissues for transplantation and the potential to treat a myriad of conditions.”
That’s because stem cells have special traits. When each one divides, the new cell can become another unspecialized stem cell or it can turn into one of the particular types of cells needed to create the body.
Stem cells can be coaxed into becoming specific organ or tissue cells. The hope is that the healthy stem cells can be used to replace damaged tissues in the body. Embryonic stem cells are useful because they’re pluripotent, which means that they can potentially develop into any one of the body’s hundreds of cell types.
Adult stem cells are beneficial because the patient can use their own cells. Doing so greatly reduces the risk of rejection post-transplant.
Understanding Induced Pluripotent Stem Cells
Embryonic stem cells are pluripotent. Adult stem cells, however, do not seem to have the same ability.
Induced pluripotent stem cells (iPSCs) are regular adult stem cells that have been altered to resemble their embryonic condition. The new cells are very similar to natural embryonic stem cells. They’re a renewable source of cells that can differentiate into specific cell types.
Much of the research into stem cells has been hindered by ethical objections surrounding the use of human embryos. iPSCs remove these objections. Patients can have access to genetically identical pluripotent stem cells.
If the research proves successful, iPSCs could be used to help patients suffering from genetic diseases.
Is There a Chance of Immune Rejection?
The promise of stem cell therapy for patients with genetic disorders lies in the possibility of using the patient’s own DNA to create new, unblemished organs and tissues. Newly transplanted stem cells can be rejected by the body if the immune system recognizes them as foreign invaders.
Stem cells born from embryos will be genetically distinct from the patient. Doctors often prescribe immunosuppressant drugs to reduce the chance that the immune system will react.
Adult stem cells, on the other hand, match the patient’s body perfectly. The chance of rejection is believed to be greatly reduced.
Are There Any Challenges?
Medical researchers are intensely interested in stem cells but there are few options that are ready for current patients. It’s believed that the field has tremendous potential but so far, very little of that potential has been realized.
The human body is an intricate machine. Healing genetic diseases is an extraordinarily complicated endeavor. Patients often require multi-pronged treatments. In addition, the exact combination of therapy and medication required to treat patient A might look very different than the combination required to help Patient B.
Most of the recent research into stem cells and genetic disorders has centered on “Mendelian” disorders, or those triggered by a single gene. However, the vast majority of conditions are far more complicated.
What’s The Future?
While no one can predict the future, stem cell therapy does show a lot of promise in treating genetic disorders. For instance, in 2017 researchers published a study showing that a young boy who lost most of his skin due to a rare genetic disease made a significant recovery after being treated by stem cell grafts.
The condition, epidermolysis bullosa (EB), is often fatal. The subject’s older brother died from complications related to the disease.
“It is very unusual that we would see a publication with a single case study anymore, but this one is a little different,” Jakub Tolar, a bone marrow transplant physician at the Masonic Cancer Center, University of Minnesota in Minneapolis, said. “This is one of these [studies] that can determine where the future of the field is going to go.”
There are even more experimental studies currently underway. The regulations surrounding the use of embryonic stem cells changed during the last presidency. It’s an exciting moment in the history of stem cell research.