When you think about it, our bodies are just a big bunch of cells. Trillions of them all working together to keep us alive. Some of these cells contain your genes, which are the special ingredient that determines our unique qualities. Things like our physical and character traits, for example, are both gene related.
Did you know that when it comes to our genetic makeup, all humans are 99.9% the same? It’s pretty amazing, only a tenth of a percent prevents us from being literally clones of each other. We’re all working with the same basic template, cut from the same cloth.
Without exception, we are each way more alike than we are different.
With so many complex systems running throughout our bodies, there’s a chance that some of the cells can contain missing or defective genes. This can lead to a wide range of symptoms and severely impact a person’s health.
There are many people born with genetic abnormalities present in their cells, while others can develop genetic diseases later in life. According to government statistics, approximately 3% of babies born have some kind of birth defect, although not all of these birth defects are gene related.
There are far too many genetic diseases to include a full list, but here are some you may have heard of:
Your genes are hereditary, which means they get passed down from your parents. Don’t blame them though - they got those bad genes from their parents too. Every child is at risk for any genetic diseases in his family history. It’s not guaranteed you’ll have the same health issues as your parents due to the other factors that impact your health like diet, exercise, and extracurricular lifestyle choices, but the same defective genes can be passed down for generations. Coping with Chronic Conditions
Many genetic diseases like sickle cell anemia, cystic fibrosis, and many others have been thought to be incurable. At best the symptoms are treated indirectly through a lifetime of expensive pharmaceutical drugs.
The only choice for many patients is to cope with their chronic condition, continually spending money just to make the pain more tolerable. None of the prescription drugs actually do anything to cure the disease. The defective genes live deep in the cells, so treatment requires a different approach than traditional medicine or surgery has been able to provide. What is Gene Therapy?
Gene therapy is the science of transplanting healthy genes into the cells that contain missing or defective genes. It took many years of research and clinical testing for doctors to develop this revolutionary technique.
What is Gene Therapy used for?
Gene therapy is used to eradicate genetic diseases, treat viral infections, and correct genetic problems. It’s a huge breakthrough for people living with cancer, cystic fibrosis, hemophilia, and other genetic disorders. Since gene therapy is still relatively new, it’s currently only being tested for diseases with no cure. There’s still endless potential for how gene therapy could be used in the future.
Gene therapy works by treating diseases directly at the genetic level. The process involves a special method where doctors can tap into the cells that contain unhealthy genes, and replace them with healthy genes. If it’s successful, the procedure can potentially provide a one-time treatment that lasts a lifetime. Why are Viruses Used in Gene Therapy?
Although they have a bad reputation, in gene therapy treatments viruses can also be used for positive results. The viruses are modified so that they will not cause disease or harm to the patient. The process is done by attaching the new gene to a virus, which then acts as a vector delivery system to the cell with the defective gene. Once the virus reaches the cell, it infects the cell with the new gene. What is the Goal of Gene Therapy?
The goal of gene therapy is to help humans to stay healthy using science. No one is perfect, and even the healthiest people get sick. Unfortunately there’s no diet or exercise plan that can make you live forever. It’s just the nature of life: all bodies eventually deteriorate. As far as when this happens, a lot of it has to do with how lucky you are when it comes to getting genes.
If only there were some sort of gene genie who could swap out the bad genes with better genes so we could live longer. Now imagine that this was actually possible, scientifically speaking.
Gene therapy isn’t magic, but it’s practically the next best thing given its unprecedented efficacy at treating what has previously been untreatable. Has Gene Therapy Been Approved in the United States?
Before you can actually get gene therapy, it’s important to note that there are very few treatments which have been approved in the United States. The government currently limits most gene therapy to clinical trials, and only for certain diseases which have no known cure.
Therefore, it’s not as easy as it sounds to get an actual gene therapy treatment.
In addition, the few treatments which have been approved by the FDA come with an extremely high price tag. A single treatment could cost you well over a million dollars, which means that only the most wealthy members of society could ever afford it.
The question remains as to when gene therapy may become more accessible to those who need it. For now, we can only hope things go well in the clinical trials.
Since the first clinical trials took place in the 1990s, interest in gene therapy has grown significantly. There are now more than 400 gene therapy trials currently taking place in the U.S., and there’s another 800 studies that are enrolling patients for future trials.
It’s inevitable that some of these trials will be successful. By the year 2024, at least 60 gene therapies are expected to be on the market.
Many of these treatments have life saving potential. Even with limited data, there’s been major breakthroughs. Gene therapy has proven to be effective in treating many chronic conditions including (but not limited to): sickle-cell anemia, cystic fibrosis, hemophilia, severe combined immune deficiency, leukemia, and blindness caused by retinitis pigmentosa.
Even with so many clinical trials taking place, it’s been a long road to get the FDA to approve gene therapy treatments for the general public. After many years of clinical testing, in 2017 the FDA finally approved the first three gene therapy products for the open market. Two of the therapies were for treating cancer, and the other was to treat a form of congenital blindness.
In May of 2019, the FDA approved a gene therapy for children less than two years of age who have the most severe form of spinal muscular atrophy. This life changing treatment is a game changer for families, however the price of the procedure is over 2 million dollars. This is the highest price ever to be set by a drug company, and raises some questions in regards to the affordability of lifesaving gene therapy treatments.
In case you’re wondering, it’s the pharmaceutical company Novartis in charge of pricing if you want to ask them any questions.
Breakthroughs in gene therapy have allowed doctors to treat diseases which have been previously incurable. But what if you can’t afford the price for the treatment? Should your health insurance cover a procedure that costs over 2 million dollars? What if you don’t have insurance?
What if you need the treatment in order to stay healthy, or alive?
In most cases, insurance companies would cover gene therapy testing if it’s medically necessary. But because it’s such a new thing, there are some insurance policies which would not cover gene therapy treatments. The current insurance system is not designed to sustain such high up front costs, so it remains to be seen how things will unfold in the future. Insurance executives are certainly motivated to make it work for patients, as this recent article shows.
Setting an affordable price on gene therapy treatments is an important part of making it more accessible. There may need to be alternative payment options, such as a prolonged series of payments similar to a mortgage. The government could also assist for those who need financial help, it could be similar to how the subsidies work for ACA health insurance plans.
In any event, people besides from the extremely wealthy will need access to these important lifesaving procedures. As more clinical trials prove positive, it’s likely that gene therapy will become more of a standard and prices will come down as a result.
It’s counter intuitive, but having an expensive gene therapy treatment could actually save a lot of money in the long run. The American Consumer Institute did a study showing the savings for the health system made possible through gene therapies - even with the high upfront costs.
Gene therapy treatments can replace expensive long-term treatments, as well as reduce the need for hospitalization for the patient. Not to mention, the treatment could greatly improve a patient’s quality of life and make them feel healthy again. It could also enable a person to keep working and earning an income for many more years than without the treatment.
Taking all these factors into consideration, how much should a gene therapy treatment cost? It depends, how much is it worth? It’s complicated, how much are you worth? How much is anyone worth?
With infinite potential to improve so many aspects of life, the medical industry is committed to making gene therapy more mainstream for the future. Whether we’re ready for it or not, we’ve already entered a new phase of healthcare. Doctors and scientists are working all the time to improve the efficiency for how people receive medical treatment, and the healthcare industry is changing as fast as technology will allow.
With telehealth services you can talk to a doctor online whenever you have a question. You don’t even have to leave the house. You can get medical advice from a professional 24 hours a day - it’s very futuristic.
For the times you do need to physical travel to see a doctor, it’s common for medical facilities to use automated robots in place of human workers. Administrative costs has decreased, efficiency has increased, and as a result it’s improved the patient experience. Robots can be programmed to perform many routine procedures with expert precision - and take less time than their human peers.
In March 2019 genome engineers set a new record: the most amount of modifications to a single human cell, an astonding 13,000 edits. This achievement was made possible with technology known as a CRISPR base editor, a revolutionary new scientific breakthrough.
CRISPR is a microbiological term which stands for “Clustered Regularly-Interspaced Short Palindromic Repeats”. Scientists have derived a method that enables genome editing through special CRISPR-Cas technology. This new breakthrough has big potential for the medical field, and for general society as well.
Editing genes on a large scale essentially means we can modify people’s genes before they are even born. Parents could pay for their baby to be born with certain genes that give them a certain hair or eye color. Gene editing could also make someone immune to viruses, or physically stronger. If you’ve seen the movie Gattaca, it does a good job of portraying a future where gene editing is standard, and people with weaker genes are treated as inferior.
If we’re already using robots and gene therapy in 2019, do you ever wonder how the medical industry will look 50 years from now? One thing is certain, it’s going to be different.
Think about how much it’s changed since the 1950s. Doctors used to smoke in the office and endorse brands of cigarettes in newspapers. They certainly put a stop to that once the cancer research came out.
Aside from casinos and certain outdoor areas, cigarettes are no longer welcome in public. There’s an endless supply of information available on how diet and exercise affect our health. No matter how you measure it, society has come a long way.
As a result, there’s a growing number of health conscious Americans aiming to combat aging through balanced eating and an active lifestyle. Doctors are committed to finding a cure for the cancer that cigarettes caused, and they're getting closer through new innovations in gene therapy. This has huge implications for the anti aging industry.
It’s not too hard to imagine a future where ads for gene therapy run in place of prescription drugs or botox procedures.
“Ask your doctor about how gene therapy. You may be getting older, but there’s still time to feel young again. Guaranteed results or your money back. Call now for a free consultation.”
As more gene therapy treatments become available, consumers who need them will be able to find an affordable option. As demand increases, there will be new insurance options to cover the cost, as well as extended payment plans for patients who need the treatment.
In addition, over time the price for the gene therapy treatments will go down. It’s only inevitable as the science continues to improve. Don’t forget that the first cell phone came out in 1983. It was thick as a brick and priced at $4000. Very few people could afford it at that price.
Now everyone has a cell phone, even kids. In addition, even the cheapest cell phone is exponentially more powerful than any 1980s version. The devices have gotten a lot better with technological advancements in production, ultimately scaling to a point where cell phones are affordable to almost everyone. Strong consumer demand means a robust market in which a broad range of different companies will compete for your business.
Gene therapy is opening new doors of discovery in the medical field. There’s still time before it becomes the medical standard, but we are getting closer. Even with limited data, it’s clear that gene therapy is becoming the way of the future. It has incredible potential to replace drugs and surgery altogether. The treatments can make us live longer, age slower, be stronger, faster…even smarter. Not to mention all the other uses we’ve yet to discover.
As more people realize the potential, it’s only a matter of time before ideas are turned into reality. The cost of gene therapy is going to come way down - and the possibilities are endless. In the meantime, we still need to do things the old fashioned way and stay healthy through diet and exercise.
Disclaimer: This does not constitute as medical advice. Consult your doctor for all medical issues.italic text
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