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What are issues arguments opinions are there on genetically modified MEDICINE? And who has that opinion? e.g religious leaders, lay people, scientists etc... Ive researched a bit myself but I only found one about insulin. Are there anymore?

Submitted by: Leen Alkhiyami


Answer

Expert response from Robert Murray, MD

Professor, Human Nutrition, The Ohio State University

Thursday, 27/12/2018 21:25

Although the use of gene editing technology to treat human diseases is fairly new, it has already started a revolution in medicine’s approach to disease treatment, drugs, vaccines, and prevention. The simplicity of the CRISPR technique – hailed around the world as a major scientific breakthrough – allows us to cleanly replace individual genes. It has resulted in a surge of new research studies as scientists apply the tool to treat highly complex clinical disorders.

 

Gene editing can either create molecules that turn on or turn off an undesirable gene or it can be used to directly replace a dysfunctional gene with a functional version. For example, you may have seen news articles just this month on the FDA’s approval of Vitrakvi (larotrectinib), a drug that treats a dozen very different formerly “untreatable cancers”. Based on a defect in just one gene (the TRK gene) cells would grow out of control, causing cancers. By blocking cell receptors that read TRK, the drug shuts down cancer growth. Insulin production is the most well-known drug mass produced using gene splicing. But many others have been or are now being developed. A few common examples you may recognize: Remicaid, Epo (erythropoietin), Avastin, and Neulasta. Avastin (bevacizumab), produced by gene editing, treats several forms of cancer, including brain, colorectal and cervical. People who suffer from anemia, have the hormone epoetin alfa available now. GMO methods have spurred the development of new, highly specific vaccines, such as those for cholera, malaria, and influenza, with many more under development. The Human Papilloma Virus (HPV) vaccine, which not only prevents an infection but also prevents its associated cervical cancer, was a product of genetic engineering. The ease of using CRISPR technology to produce drugs offers hope for many new inexpensive and widely available medications and vaccines throughout the world, helping to treat such devastating diseases as HIV, Zika, and Ebola, to name a few.

 

Immune dysfunction and uncontrolled inflammation are at the root of many different diseases. GMO technology is being used for priming, boosting, controlling, modulating, and maintaining the immune response. This strategy can not only help boost vaccine effectiveness for prevention, but also can help a variety of drugs work better on existing medical conditions, such as cancer or immune weakness due to chemotherapy or aging. Such highly targeted manipulation of very specific parts of the immune system will minimize unwanted side effects.

 

Direct replacement of a defective gene with a healthy gene is now being used for disease treatment. The first gene therapy treatment approved by the FDA was for a rare inherited condition that causes progressive blindness starting in childhood. Patients from 4 to 44 years of age were treated by injecting normally functioning genes into the eye. More than 90 percent of those participating showed improvement in vision. In another study, scientists recently reported that they had successfully replaced a gene (CEP290) that causes kidney disease in patients suffering with Joubert’s syndrome. Joubert’s syndrome is a brain disorder that causes physical, mental and sometimes visual impairments. Without treatment, one-third of patients will develop kidney failure, forcing on to long-term dialysis or transplantation. The drug should be available within a few years. Even pre-natal gene therapy research was encouraged recently when the National Academy of Sciences indicated that altering the genes of embryos might be allowed under strict criteria if the objective is to prevent serious disease.

 

But this is important to understand: these many new therapies arising from gene-editing technology -- whether it is direct gene-replacement in humans or drugs and vaccines produced using the gene modification tools -- all of these therapies must still undergo the same strict study controls and meet existing regulatory standards to ensure that the treatments are both effective and safe. That will not change in the future.

 

Applying this powerful gene modification technique needs to be performed transparently under stringent guidelines to prevent misuse and unanticipated consequences. Just this week a Chinese scientist claimed that he had used CRISPR to alter embryos before implanting them in a woman, who then gave birth to twin girls. The action appeared to be a violation of China’s scientific regulations and drew immediate condemnation from fellow-scientists, ethicists, the lay public, and Chinese legal representatives. Monitoring the uses of gene editing technology will require a well-informed public. The media and the blogging community will need to step away from the sensationalism that often characterizes public discussion about genetic manipulation and instead provide accurate, scientifically-based perspectives so that the public understands the risks, the benefits, and the process being used.  A good way to keep up with the whole field of genetic modification for biotechnology, agriculture, nutrition, and health is to follow the Genetic Literacy Project at https://GeneticLiteracyProject.org.