A gene with a desirable trait can be moved from one organism to another organism as a means to change it. The traditional way is through selective breeding, which is slow, time consuming, inefficient, and transfers more than one gene, so other unexpected and unwanted traits can cause problems. But genes also can be moved in a laboratory, resulting in what has been called a genetically modified (“transgenic”) organism (GMO). GM technology moves only one gene, eliminating other, unwanted traits being produced. Think of overcoming antibiotic resistance in bacteria or blocking emerging new infections, such as Ebola. The method has been used for over 40 years successfully, cheaply and safely. There are no credible studies showing harm to humans from use of GM technology in crops or pharmaceuticals.
Medical scientists have used transgenic methods to produce insulin and thyroid medications, vaccines against hepatitis, cholera, malaria, cancer therapies, treatments for anemia and stroke. Many new medications were designed using GM techniques, including Remicaid, Epo, Avastin and Neulasta.
This year for the first time, scientists successfully edited the genes of human embryos to repair a genetic mutation. The editing technique, called CRISPR, is so simple and efficient it was named scientific breakthrough of the year in 2015. Not only can gene editing prevent a fetus from developing a serious genetic disease, such as Huntington’s disease, thalassemia, sickle cell disease, or Tay-Sachs, but also it can eliminate the transfer of the abnormal gene to subsequent generations. Strict guidelines have limited the use of this powerful tool to only severe, life-threatening gene mutations. It is not for changing the cosmetics of the baby.
GM technology and the new CRISPR tool promises to make drugs easier, cheaper and safer to bring to market. But access to the newest, most powerful drugs within developing countries is complex. There are four pillars of drug “access:” rational selection of the most essential drugs, negotiating affordable prices, establishing sustainable financing, and developing a reliable supply network within the local health care system. Most of the essential medications are now off patent. Despite this, half the world’s population is too poor to afford them, especially when purchasing them through non-government subsidized sources.
HIV treatment with anti-retroviral therapy has been the exception. Access has been a huge problem worldwide. These drugs are expensive. The drugs control the disease but do not cure it, meaning that individuals must take them for life. There are side effects and risks. CRISPR may change all that. The tool is so simple that its use is not limited to researchers in a few major pharmaceutical companies. The first clinical trials using the patient’s own gene-edited T cells was successful in boosting immune resistance to HIV. A vaccine now seems feasible in the near future. GM is a powerful 21st century scientific tool that will revolutionize health care for all of us, particularly those in developing countries.