For years, researchers have been working to deliver functional gene therapy drugs to the market. Instead of treating symptoms or adding chemicals meant to assist the body in combating illness, gene therapy drugs would essentially overwrite DNA to make it more effective. This could lead to novel treatments for a number of as-yet untreatable illnesses, and revolutionize the way medicine approaches treatments more generally.
Recently, and for the first time, the FDA approved the use of a gene therapy drug in the USA. It’s an important milestone on the road to wider adoption of this branch of medical technology, and something that incoming pharmaceutical pros could see lead to greater adoption as their careers progress.
Read on to learn a little more about this breakthrough.
America’s First Gene Therapy Treatment Is for Children With Leukemia
Leukemia is a cancer of the bone marrow and blood, and while it occurs most often in adults, it is the most common form of cancer suffered by children. The first gene therapy treatment approved in the US, called “Kymriah,” targets a particular variant of this disease, known as “acute lymphoblastic leukemia” most commonly found in young children. This variant of leukemia sees the body produce a large quantity of cancerous white blood cells, which prohibits the creation of enough normal blood cells and impedes proper organ function.
The Kymriah treatment involves extracting a patient’s T-cells, which are white blood cells that work as part of the immune system, and genetically modifying them to target and kill specific leukemia cells. The cells are then reintroduced to the patient in the hopes that they will help cure the cancer.
It’s an interesting approach to cancer medication, one that is sure to be watched closely once it begins to be more widely used outside of preliminary testing. Professionals with pharmaceutical regulatory affairs training will be needed to help conduct this sort of monitoring in the years to come. There could be exciting opportunities to work with this, and other gene therapies, in your own future career.
Students Preparing for Careers in Regulatory Affairs Should Know it Is Not a First Choice Treatment
That Kymriah is at the cutting edge of medicine, and makes use of an individual’s own cells for treating disease, might make it seem like the best or most impressive choice for treating acute lymphoblastic leukemia. Because the treatment is so new, though, it is very expensive to administer. In the US, costs could be close to $500,000 (or $625,000 CAD) and so it will only be used in patients who are not responding well to more traditional forms of treatment. These traditional treatments include options like chemotherapy, radiation, and immunotherapy, typically offered in combination.
There is also a risk of side effects present with the drug, including high fevers, flu-like symptoms, decreased oxygen, and a number of other effects that could lead to serious illness or injury.
Over the long term, the goals with this type of treatment would be to get costs down and levels of safety up—both of which would require a fair amount of research and development to accomplish. After graduating with your regulatory affairs diploma, consider bringing your talents to this type of work, and help ensure safer and more accessible treatments for the world of tomorrow.
Graduates of Pharmaceutical Regulatory Affairs Training May See This Technology Used Elsewhere
The Kymriah treatment works by specifying a particular type of cell that should be targeted for elimination by the immune system. Researchers hope that this exact same treatment method could be applied to other sorts of cancers and diseases in the future, without too much additional research and development needed to get the ball rolling.
As with any medical advance, however, restrictions are in place. Industry regulations ensure that future iterations of the Kymriah treatment, which would target other sorts of illnesses, would need to go through the regular routine of trials before entering the market. Therefore, professionals who understand how to safely usher new medicines through the development process will be necessary on these kinds of projects. They can help ensure that products are thoroughly tested, and still move things along quickly enough to be of assistance to the people who need these medications—an important role, given the great potential of gene therapies.
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