Gene Therapy Treatment for Alopecia Areata
There is strong evidence to suggest that alopecia areata could be genetically inherited, through genetic markers, and then triggered by an adverse reaction in the immune system. The genetic markers, which warn of a slightly increased chance of developing alopecia areata, may also ultimately result in its cure. A new field of medicine known as gene therapy shows signs of one day not only relieving symptoms, but entirely curing most autoimmune diseases at their source by re-programming the immune system itself by manipulating and altering a person’s genes.
Gene therapy was first successfully used on September 14 1990 to treat a four-year old girl who suffered from a life threatening genetic disorder that did not allow her body to produce a crucial enzyme, and as a result her body could not produce adenosine deaminase (ADA) which made her immune system extremely weak. Physicians took some white blood cells from the girl and inserted genes that could produce ADA into the white blood cells. The cells where then transferred back into the girl, restoring her immune system. Since this time gene therapy has branched out to treat other diseases.
Until now the only treatments available for sufferers of a multitude of autoimmune diseases have been concerned with reducing or eliminating symptoms, rather than the root cause. This is because very little has been understood about the triggers and intelligence at play in the immune systems selection of targets. Fortunately, scientists are now on the cusp of a new understanding of the immune system and how to influence it through gene therapy.
How gene therapy works
Gene therapy is a new experimental technique in medicine. It aims to use genes in the prevention and treatment of a multitude of diseases. Although the various treatments collectively known as gene therapy are very new, they have had significant success in treating many life threatening diseases. One of the most common techniques involved in gene therapy involves the inserting of a “normal” gene into the genome in an effort to replace or swap a malfunctioning gene. Gene therapy also involves the deactivation of a problem causing gene or the activation of a gene that should otherwise be on. More difficult techniques in gene therapy involve directly repairing a dysfunctional gene, returning the gene to its normal state.
Germ line gene therapy - In this form of gene therapy, germ cells – like sperm or eggs, are altered to include the new re-written gene or genes of the individual who currently would carry the genetic disposition of developing a disease. This is carried out before conception, but promises to theoretically eliminate hereditary disease, autoimmune disease and genetic disorders. It won’t help cure current sufferers of alopecia areata but does promise to remove the disease from human memory if this form of treatment is accepted.
Side effects
The side effects of gene therapy are not yet clearly understood, since the treatment is still in its infancy. Long term side effects are especially not known, since there is insufficient data available from people who have received gene therapy treatments many years ago. At present researches have an idea as to the short term side effects. The immune system could react aversely when gene therapy is first administered. Some patients have reported fever, vomiting, headache and nausea following the days after the first application of the treatment. Depending on where treatment was applied, local side effects could be observed as well. For example if treatment is given to the liver, possible side effects can affect the liver.
Given our understanding of genetics and the immune system researchers have hypothesized some other side effects. Such plausible side effects include the damage of healthy cells leading to other diseases. There is also fear that inserting genes into reproductive cells could cause damage to eggs or sperm. Thus these damaged cells could be passed down genetically to future offspring.
Alopecia areata and genetic connections
Alopecia areata is a disease that is difficult to study due to its sudden and random nature in development. It is even more difficult to understand why this disease occurs. What researchers can study however, are the numbers and statistics associated with alopecia areata. One such piece of data is the strong statistical evidence to suggest that alopecia areata can be hereditary in nature. Numerous studies have shown that an individual with alopecia areata has had other people in their family with it as well. Upwards of 1 in 4 people who have/had alopecia areata at some point in their life can identify a close family member who has had it as well. Scientists believe that genetics, along with other factors, can play a triggering role in the activation of alopecia areata. But the common denominator in most cases of alopecia areata is genetic disposition of it.
How gene therapy can one day cure alopecia areata
Currently gene therapy is still a new area of medicine that can pose considerable risk to the patient receiving treatment through it. Because of this, only life threatening diseases with no cure are treated through gene therapy. Due to the non-life threatening nature of alopecia areata, treatments of using gene therapy to treat the disorder are rare. The use of gene therapy in the safe treatment of alopecia areata is still many years away. But in theory gene therapy, if developed properly and applied safely, can provide for a major breakthrough in the treatment of alopecia areata.
What is more important about gene therapy is its possible ability to filter out genes that can activate alopecia areata from human genetic memory. As a result, any modifications and effects will only benefit the individual and not be passed on to future generations. This has been used with great success in the treatment of immunodeficiency, and may be a cure for current alopecia areata sufferers. If researchers can prove that there is 100% pre-disposition of alopecia areata in the genetics of those who have it, gene therapy could be the final answer and cure to alopecia areata.