For many years, drugs were the only way to treat or mitigate the symptoms of a disease. But scientists around the world are working on a new approach that could eventually cure people with incurable diseases and even eradicate some diseases altogether. How do they plan to do this? Researchers are attempting to alter human beings at the most basic level using gene therapy. By introducing specific genes into a patient’s DNA, scientists believe they can drastically change medical care as we know it.
Read on to learn more about gene therapy.
Students in Drug Safety Courses May Know How Gene Therapy Works
Gene therapy is making waves in the medical industry. It works by introducing corrective sections of DNA called genes into a patient’s body, which work to correct or abolish a problem in their body’s functioning. Gene therapy techniques have been around since 1972, but scientists who have dedicated much of their professional lives to gene therapy science have faced many boundaries and setbacks. However, students earning their drug safety and pharmacovigilance certificate may be interested to know that positive results in gene therapy research over the last several years indicate that the future may in fact be bright for gene therapy.
There are two types of gene therapy techniques: augmentation and inhibition therapy. In augmentation therapy, a gene that has the correct function or missing genetic element that a patient is lacking is injected into the patient’s body. In inhibition therapy, a gene is injected into the body that either stops a bad gene that exists within the body from behaving harmfully or interferes with its activity. The type of therapy used depends on the disease being treated. For example, a recent gene therapy trial aims to help individuals whose blood can’t clot by providing the gene that helps with this function. This is an example of an augmentation therapy at work.
Students in Drug Safety Courses Can Understand the Risks Involved in Gene Therapy Trials
Gene therapy’s slow start may be attributed to the immense risk many clinical trial patients face, the extensive research process involved, and expensive production costs.
Gene therapy is extremely challenging for scientists and researchers. Not only do they have to identify the problem and the solution, but they also need to find an effective way to transport the new gene into the body and only have it affect the desired targets. A gene reaching the wrong target could result in health issues for the patient.
Gene therapy is a risky process
Whenever something foreign is put into the body, its natural response is to reject it. Ironically, scientists are constantly working against the body’s natural defenses, which want to block the helpful genes used in gene therapy. This aversion can cause severe reactions in patients and in some cases lead to death. As students in drug safety courses may know, risky clinical trials can have a hard time getting off the ground.
Graduates of Drug Safety Courses May Know there Are Financial Barriers to Gene Therapy
Although there are serious risks involved, some gene therapy clinical trials have had promising results. With that being said, another issue with gene therapy trials is that patients may have excellent results right away, but it’s hard to determine whether these effects will be lasting.
Gene therapy is intended to be a permanent solution, because it alters the body’s DNA at its core. However, it’s impossible to know if five, ten, or even 15 years after the trial, the results will last. And because these gene therapies are incredibly expensive to produce, the price tag for the few that are on the market are mind-boggling. For example, the gene therapy Glybera, which helps patients who build up too much fat in their blood stream, costs about $1.4 million USD for a one-time treatment cycle. Without knowing the long-term effects, those suffering from the disease are wary of spending that much money on a treatment. To date, only one person has purchased it.
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