Rare diseases could have a cure if enough money, time, and research are allocated to them. This is a complicated and difficult mission, but in some cases, it’s not impossible. A lot of research has shown that some clinics are using gene therapy to treat certain types of diseases. This type of genetic research is an important innovation in the health world and can help to find the cure for diseases which until now were incurable. One example is the case of the Combined Immunodeficiency Syndrome. In the early 2000’s, this was one of the first diseases treated with genetics. Until then, bone marrow and umbilical cord transplants were the only treatments.
One example is the case of the Combined Immunodeficiency Syndrome, commonly known as the “Bubble Boy Syndrome” which is a rare genetic disorder characterized by the disturbed development of functional cells which causes patients to be affected by severe infections early in life. In the early 2000’s, this was one of the first diseases treated with genetics. Until then, bone marrow and umbilical cord transplants were the only treatments.
A lot of research has shown that some clinics are using gene therapy to treat certain types of diseases. This type of genetic research is an important innovation in the health world and can help to find the cure for diseases which until now were incurable. One example is the case of the Combined Immunodeficiency Syndrome, commonly known as the “Bubble Boy Syndrome” which is a rare genetic disorder characterized by the disturbed development of functional cells which causes patients to be affected by severe infections early in life. In the early 2000’s, this was one of the first diseases treated with genetics. Until then, bone marrow and umbilical cord transplants were the only treatments.
In May of 2016 a treatment called Strimvelis, produced by GlaxoSmithKline was approved to treat this rare disease that only affects around 1 in 100,000 births.
Many companies working on treatments, are switching to researching solutions for more common diseases, like blindness.
The problem with gene therapy treatment is the high cost (over 500,000€). Even though, this is the half of the cost of its predecessor, Glybera, which was about 900,000€, as reported by the MIT Technology Review. Strimvelis, is a complex treatment because the bone marrow cells of the patient are removed and then modified outside the body. After that, the modified cells are transferred back to the patient intravenously. This treatment is currently only available in Milan, Italy.
As an alternative, many patients are applying to other clinical trials which are offered in hospitals in cities like London, where have had good success with local treatments according to Susan Welsh, director of a U.K.-based charity.
In the United States and in Canada there is also the option of joining gene therapy clinical trials, run by a start-up staffed by former GlaxoSmithKline executives. These trials are taking place at the University of California in Los Angeles.
Over the next two years, the goal is to effectively transport the cells so as they can be modified and sent to the patients without the need for travel.
After analysing the results, we can see that genetic therapy, though effective, is an extremely expensive treatment. This is due to the low number of affected people. That’s why, many companies working on treatments, are switching to researching solutions for more common diseases, like blindness. North American laboratory Spark Therapeutics is planning to apply for approval to start working on a treatment for blindness. While this is bad news for patients with rare diseases, with new treatments and cures for common diseases maybe the progression in the field of rare diseases treatment will benefit as well. We hope that all diseases in the future will have a treatment thanks to gene therapy.
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