Author Archives: Michelle Pyatnychuk

Gene Therapy: Present and Future

If our group has learned anything from these past few months of learning about field of gene therapy is that right now, no one truly has the answers to the ethical, societal and economic implications that this scientific cure could bring.

Taking a step back, we began our research at the start of it all. What is gene therapy? Through thorough research into this, we found that gene therapy is equal parts technology and medicine. Technologically, there are therapies ready to be put on the market to cure diseases such as blindness, Alzheimer’s, Parkinson’s and more but there is more to it than just technology, remember this is medicine. In the tech world, speed is everything. It is all about winning the race against smartphones, smart TV’s, AI, anything. In medicine, there is a time press that comes from the DOH in emergencies but most of the time, speedy remedies to disease put up red flags for those being treated later on. What gene therapy has done is take medicine from a personal relationship, to a big business, one that is on track to be bigger than the pharmaceutical sector. This big business is seen in one of our insights into the cost of gene therapy. The overall controversy of how one can afford to buy a gene therapy is due to the high costs that of the gene therapies that are on the market today such as the first FDA approved gene therapy Kymirah among others.

Such being the case, Dr. Mark McClellan, former FDA commissioner and current leader of the Duke-Margolis Center for Health Policy, concluded that the healthcare system is far behind the medical industry and the advances it has made so, being that patients, doctors and researchers, and insurance companies have mutually exclusive interests, it is impossible for everyone to win. The entire gene therapy field is now looking to monetize on their ability to cure what used to be deemed incurable diseases and this is where the ethics of it all come into play.

Journalist and historian, David Perry wrote, “the pro-information approach demands that everyone involved in genetic counseling have access to the best data and presents it in a value-neutral way.” In giving everyone equal access to gene therapy, this must include attainable price points, no discrimination and equal opportunity for everyone to take advantage of. The FDA is just no longer enough to control the medical and genetic communities. The ethical future of gene therapy is now beyond the typical, “this is wrong” and “this is right” because we have never had to deal with the controversial issues that we are dealing with now as a result of gene therapy. From “designer babies” to biological weapons, these are both things that health professionals and even legislators have yet to encounter scenarios such as these but because of this, getting out ahead of the possible negative implications of these two examples, is more important now than ever.

We are in a place where CRISPR can help farmers combat climate change by genetically altering their crops, incentivize football stadiums to start “DNA Days” and, lead to the insurance and pharmaceutical industries to be left in the hands of tech giants like Amazon, Apple and Google. As such, there needs to be some sort of precedent set so that none of these three scenarios gets any worse than they already have. With genetic testing companies like 23andMe and Ancestry becoming more familiar in the public eye, they are being sold to customers who have no idea that they are basically giving our their privacy rights to their own genetic information for results that are little to completely, inaccurate.

This is why education within this industry is more important now than ever. Without proper understanding, the Frightful Five, the FDA, the federal government could all have influence over every single person residing the in the United States because of those who feel it is important to send a swab of their DNA to a lab in order to see just what percentage English they really are. What people sending in their cheek swabs needs to know is that scientists are still working, to this day, trying to uncover the complex information of the millions of genes that reside in the human body. We simply do not know enough to give sufficient information to people who are just looking to see if they are carrying a genetic mutation. These 23andMe, Ancestry, and Helix tests cannot significantly provide lifesaving information and as a result, should be used with a grain of salt, or even less than that. It seems unlikely for anyone to give up the password to their email but when shipping their DNA to an unknown lab somewhere in the United States, people do not seem to show the same concern for their privacy. Thinking about the millions of people having their data breached across the country, by more companies than one, how can we be sure that this genetic information will not be next? Now even thinking ahead, the potentially 6 million more students studying harder, retaining more information, and ultimately becoming better learners because of gene therapy treatments will be ahead of the present generations. Taking it a step further, if this trend is multiplied across generations, eliminating prohibitive, cognitive disorders in students, it could reshape the entire education system with many subsequent disruptions.

With a Frightful Five company such as Amazon at the brink of breaking into the healthcare industry, it could mean positive things for gene therapy whether it investment in research, funding of drugs, therapies and clinical trials, these tech companies, although you do not want to trust with your health data, can contribute a lot to the gene therapy community. But, there needs to be a point at which the line is crossed and sure, donating genetic information for research is ethical and legal but when it is collected through your Google search history or updates on your Apple health app, there needs to be a call for change. In the end, with the Frightful Five having taken initiative within the healthcare industry to fund gene therapy programs and rebuild the healthcare system of the nation, they can push bounds in the battle for updated health services unlike anyone else can and this gap between the Frightful Five and everyone else is what needs to be addressed.

And to end on a global note, the future of gene therapy is the United States barely has a clear path ahead of it so thinking about how the rest of the world compares is a whole other thing on its own. Isolated countries such as North Korea, Russia and China make it difficult to know how exactly the countries have progressed in their biotechnological warfare, including gene therapy, and what the affects can be on the rest of the world when a country, although small compared to the US, can utilize science to become a super-powered population. It is to avoid scenarios such as the one posed by North Korea that the FDA was put in place. In order to ensure that the medicinal and agricultural sectors of the nation were being regulated and monitored, there needs to be an organization that oversees research that secure public health and safety. But, just having one country, out of hundreds with the capability to genetically alter genes and embryos with this agency in place, is not enough to guarantee that gene therapy will only be used for good.

There are a lot of things to ponder over here, but the best place to start is by educating yourself on the future that could be completely disrupted by this biotechnological innovation and then looking for ways to protect your genetic and health information, so if that means resisting buying genetic testing kits, not updating your Apple health data, or any of the other numerous ways to protect yourself, that at least is a good place to start.

The Ethics of Gene Editing

With the ability of scientists to genetically modify and edit one’s DNA, they have not only unlocked the possibility of curing diseases they are even diagnosed but have also opened a gateway to the unknown that offers little to no protection, security or nonthreatening state of mind. As said by Andrew Joseph, “People have access to more information about their own genes — or, in this case, about the genes of their potential offspring — than ever before. But having that information doesn’t necessarily mean it can be used to inform real-life decisions.” Gene therapy is still in its beginning stages and the reliance that people have on it being able to help them make decisions about their well being and the well being of their offspring should be taken with a grain of salt. These genetics tests or treatments are not always completely accurate and due to this, should not be significant factors when thinking about the ethical implications of a mother and father deciding what to do with their two week old fetus that is tested, and comes back positive, for a gene that could harm them in the future.

Bioethicist Alta Charo on the other hand does not see that the implications of gene therapy will affect the ethics of parents choosing “designer babies” or of the fears by governments that it will induce a new population of people against one another. As such, her comment that “genetics doesn’t tell us everything we need to know…they have tremendous influence … but we don’t have to assume that by having genetic information we will abuse the choices it facilitates,” implies that the worst case scenarios that may occur with gene therapy, may not even happen. China’s leading CRISPR researcher Dr. Lai Liangxue sees gene therapy in a similar light as the atomic bomb, noting, “I say that depends on who use it, right? Like, like, atomic bomb. That’s kind of — if you use it to make electricity, it’s good. If you use it in a bomb, it’s bad.” So, the fear that an entire new race of species of human can be created within a few decades can be seen as unfounded. There are just too many concerning factors but as can be seen from scientists whose research is the embodiment of their entire careers, they are too inclined to see the negative implications that their work can bring and instead seek to call these fears as irrational and unnecessary. These scientists are exhuming their confidence in the human race by noting how most are both rational and good but the underlying theme is, especially using Dr. Liangxue’s example of the atomic bomb, is that the human race is not all born innately good and since if there is a possibility that gene therapy can pave the way for dangerous experiments, someone will and can use it to their advantage.

In noting the comments made by Dr. Liangxue and Dr. Charo, the question then is, are these fears actually irrational and unnecessary? Because if they are, why does the DOD and Pentagon want influence over genetic treatments? Not for nothing, when a nation’s military and security sector want to track and monitor genetic treatments, the unfounded fears that scientists debate against are certainly something significant enough to deem relevant, to say the least. When the Pentagon sees genetic treatments as ways to cause injury to thousands by “agents of war,” there needs to be a wake up call within the healthcare community. The FDA is just no longer enough to prevent genetic therapy from reaching those who do evil and even with its regulation, there is no saying just how much control they will have over medical and genetic treatments. Although it can be argued that in the coming age of CRISPR and other gene therapies, the need for everyone to have the same and equal opportunity to get information will be significant in preventing the ethical and moral issues that can arise when it is not given out.  Best stated by journalist and historian, David Perry, “the pro-information approach demands that everyone involved in genetic counseling have access to the best data and presents it in a value-neutral way.”

The issues that can arise from the lack of motivation on behalf of the gene therapy community to speak on the negative effects of gene therapy because of their involvement in their own research and development of their treatments will be greater than just sidestepping the issue until more government agencies get involved.

The Frightful Five and Gene Therapy

The latest developments within the gene therapy, medical community involves one of the Frightful Five. With Amazon recently purchasing 12 state pharmacy wholesale licenses, it is time to take a look at how the Frightful Five can make an impact on the healthcare, and specifically gene therapy sector, of the United States.

With a Frightful Five company such as Amazon at the brink of breaking into the healthcare industry, it could mean positive things for gene therapy. Already, pharmaceutical companies are looking to work with geneticists in researching cures to basically incurable diseases and with this interest from these companies, it can be inferred that the potential pharmaceutical companies see in gene therapy, is seen through the eyes of the Frightful Five as well. The contributions that these technological companies can make whether it be investment in research, as Google has done, or funding of drugs, therapies and clinical trials, these tech companies, although you do not want to trust with your health data, can contribute a lot to the gene therapy community.

Already we have seen how companies like Microsoft and Apple have taken steps to enter the health sector of the economy through their respective applications, NeXT, and Apple Health and there are no signs of them stopping there. With the Frightful Five taken initiative within the healthcare industry to fund gene therapy programs and rebuild the healthcare system of the nation, they can push bounds in the battle for updated health services. Society is no longer working in the typical, you get sick, you go to the doctor, model anymore. We are at the brink of preventing and curing disease to ensure it will never come back again, or to future generations and this is a business model that only a technological company can understand. Insurance and pharmaceutical companies are not prepared for the biotechnology being created by the likes of Apple and Google but these companies can ensure that they are. Soon both insurance and pharmaceutical companies alike will understand just how significant it is that they account for the digital technologies that will dominate the health market, or the digital technologies that will be made obsolete by gene therapy. The only way to prepare for such scenarios is by understanding the technologies and their capabilities, and in order to understand them, there needs to be a giant like Apple, Google, Amazon and Microsoft who can take these innovations to the mainstream.

In today’s society where new gene therapies are being approved by the FDA on what seems like a biweekly basis, it is important that this continues if not at a quicker rate but at a better quality rate. With the help of Apple and Google, the FDA already sees the potential in allowing these companies to use their health products to monitor people’s heart rates and more. Applying these physical technologies, given the hope that the data collected will be strictly monitored and controlled by the FDA in the utmost ethical manner, can prove crucial to bringing gene therapy to the mainstream. To be able to aide physicians and scientists in their diagnosis’ and research will only help generate a further interest into gene therapy and a better understanding of this sector of health overall.

There is, and should be, an outright fear of the Frightful Five collecting your health data. It is scary for a technological company to have this much control over your private and public life but in the end, the Frightful Five is so named because they have this control over everything and everyone. Since they do, the best way to get new innovations, progressions and more out there for people to actually see and use in their own lives, is to have the Frightful Five show it off. With Amazon’s purchasing of pharmacy licenses, they can change the business model of pharmacy to become more gene therapy based rather than drug based. With Apple and Google’s health devices, they can change the business model of the medical service industry from impersonal to personal. It does not feel comfortable to say that having these companies around is the best solution for us to bring reform to the gene and healthcare industries but for right now, these guys are all that we have to make sure this reform comes, and not in the future, but now.

Big Data in Genetics

Privacy is just not something to be expected in this day and age of technology and apart from the voluntary use of social media sites and applications that blatantly infringe on this right, people set themselves up for even more invasion when they request their genetic information. Ancestry and family lineage sites for the last few years has been picking up due to shows like “Who Do You Think You Are” and basically any tear-jerking story about long lost siblings who found one another through searching their lineage that you read on your Facebook page every morning. Lately, however, these sites, and not even getting to genetic testing sites like 23andMe, have been asking for more than just a last name and a few answers to personal questions. They have been asking for their customer’s DNA, their genetic data and the repercussions of allowing for a corporation to have the ultimate template and profile of you are greater than you even know. 

It seems harmless enough, just take a swab of your cheek and ship the sample to a company who within two weeks will mail you back a sheet that has your genetic lineage and maybe even a genetic profile to warn you of some genetic disorder you may have. Speaking on a positive note, what could this company have to gain from helping people learn more about their past and their own selves besides doing it from the perspective of a service to others. Well, in fact they have a lot to gain. Since so many people refrain from actually reading the privacy policy before shipping their genetic data through the mail, they do not even realize that they are signing away their right to sole ownership of the data that is collected. Ancestry.com now has the rights to use the data that has been sent their way for their own purposes and if these purposes are to sell the information to the highest bidder, worst case scenario, they have the broad right to do so. Although legally companies such as ancestry.com and Helix do not have the license to sell away your genetic information to third parties, they do reserve the right, without your deliberate permission, or with it following your purchase of the genetic testing kit, to sell it to pharmaceutical and research companies. Now, this already seems like the only boundary these genetic testing sites are crossing is an ethical one where the whole hurt one for the greater good comes into play but when you find out how one of Ancestry’s partnerships is with Calico Labs, which is founded and operated by Google, Inc., you might sing a different tune. With a technology giant such as Google becoming so interested in the health sector of the nation’s economy, certain eyebrows should, and must be, raised before consenting to shipping off DNA that could potentially wind up in the hands to the very people who endlessly track and monitor its users. It is already a breach of rights when a genetic-information-collection company can claim rights to someone’s genetic information but when it can ultimately be shared with a corporation that is not always looking out for the welfare of its users, there needs to be a stop to it.

Before you decide to get your DNA tested for signs of genetic anomalies, keep one thing in mind, scientists are still working, to this day, trying to uncover the complex information of the millions of genes that reside in the human body. We simply do not know enough to give sufficient information to people who are just looking to see if they are carrying a genetic mutation. These tests cannot significantly provide life saving information and as a result, should be used with a grain of salt, or even less than that. Knowing this, as well as the fact that the genetic industry as a whole lacks substantial privacy laws and regulations, there should be more wariness when it comes to giving up your genetic data so easily. It seems unlikely for anyone to give up the password to their email but when shipping their DNA to an unknown lab somewhere in the United States, people do not seem to show the same concern for their privacy. Is this precaution for not sharing DNA samples a little extreme right now, considering how the healthcare and tech industry still cannot utilize genetic information for anything more than research? Perhaps. But, with millions of people having their data breached across the country, by more companies than one, how can we be sure that this genetic information will not be next? People need to expect more from their legislators when it comes to protecting their privacy so whether it be securing the pass-codes on their smartphones or protecting the use of their genetic data, people should care because even though it is not here yet, it is well on its way and preparation for it is vital.

The Future of Agriculture Depends on CRISPR

CRISPR does not only the capability of curing disease and altering the genes of human DNA, but it also has the potential of altering the genetic data of crops. GMOs have been a staple of the American farming industry for the better half of the last century because it produces efficiency within the economy. GMOs cut the time needed for crops to grow and for farmers and agricultural industries to turn a profit. Although it is beneficial to the economy, and trying to feed the nation as a whole, there are obvious repercussions for taking the easy way out.

These are the increased resistance to pesticides, the appearance of new allergies and other organism effects that are a result of consuming genetically modified foods. GMOs are spread primarily into crops through chemicals and other toxins that stimulate growth, help the crop retain its color and prevent the crop from being pest infested. What CRISPR can offer is the answer the organic farming community has been looking for: how can one ensure no food shortages, healthy options and increased human health? Although still a form of genetic modification, CRISPR does not come with the toxins that chemicals bring to people and the environment. The toxins that are emitted onto crops and harming the environment are creating more problems than solutions for the world. Instead, what genetically modifying tools can help create is altering the crop’s actual DNA and take away the harmful toxins that are in the environment and eaten by people altogether.

CRISPR is not only useful for altering the DNA of crops for farming purposes but for health purposes as well. This gene-editing tool can enable vegetables and fruits to carry even more health benefits and nutrients for those who cannot eat or drink certain foods.

As seen in the article linked above, corn now has the capability of carrying the same health benefits of meat and even more so, is on the verge of becoming the healthiest vegetable. Through doing this, imagine the hundreds of thousands of vegetarians who would no longer be deprived of the nutrients of meat or fish because those very same proteins are in the vegetables that they are more inclined to eat. If anything what CRISPR can accomplish is a win for the picky eaters who choose not to eat a certain food because they do not prefer the taste and now, they do not even have to. On that note, CRISPR has also been used to aid those with allergies such as those with allergies to gluten. Therefore, the allergies that people are suffering from, due to genetically modified chemicals, can now be combatted with gene therapy. As a result, these therapies can prove to be vital in easing the way of life for those with extensive food allergies whilst protecting people from future allergies to different crops because with all things remaining the same, not every human body will respond to a certain crop, genetically modified or not.

What CRISPR can also bring to the agriculture sector is a way to combat the environmental changes that are enveloping the world. With natural disasters and extreme weather becoming so common within the world, genetic engineering provides a solution to farmers and industries in this field. To think that CRISPR can be used to alter the genes of say oranges and allow for them to grow fruitfully in the winter or wheat that is capable of bracing extremely dry, or extremely wet, weather. This will make all types of crops accessible to places all over the world, battling hunger and providing for an a boost in the global economy for easily traded goods that be mass produced worldwide and shipped to various nations as a direct result. CRISPR gives nations the opportunity to expand their fight against world hunger in a way that was once thought to be impossible. Already this fall, apples that do not brown are going to be released into supermarkets and just with this launch alone, it should evident that genetically modifying the DNA of fruits and vegetables is not a thing of the future, but something that is happening and happening now.

The Baltimore Ravens: Bringing Biotechnology to the Mainstream

At-home genetic testing has grown more and more relevant in the last few years with companies like 23andme and Orig3n taking genetic tests from the lab to the local pharmacy. Biotechnological companies such as the two linked above have found a way to tap into the driving force of people: curiosity. Curiosity is what keeps shows like “Who Do You Think You Are” and websites like ancestry.com running because people are innately curious about their past, themselves, and how they ought to be living. With this growth in biotechnology, people now have the capability to check their DNA for signs of disease and even their religion. Coming off of the right intentions, having access to the inner threads of one’s DNA could be not all that bad, but from the opposite side, the realization that mailing a swab of your DNA to a lab in who knows where and being tested by who knows who, can definitely be off putting. Realizing privacy within the fourth industrial revolution has become harder and harder, and now, we are at a point where a person’s age, gender and health information can be shipped from a residential house to a lab without much question, and with the naïve intention of trying to figure out just how European their lineage really is.

One company, already mentioned above, that took this naivety of people and used it to their advantage was Orig3n. Being so convinced that Baltimore Ravens fans would be curious as to the makeup of their DNA, they partnered with the Ravens to hold a local “DNA Day” at the home stadium of the NFL team.

This, of course, did not go over too well with the Maryland Department of Health and the Food and Drug Administration who both pulled the plug on this day before it even began. Looking back on this, Orig3n has decided not give up and is looking to reschedule this day as it is something that they, and Ravens fans supposedly, have been looking forward to doing for months. However, when it comes to asking for a cheek swab of a football fan coming into a public stadium, thinking that something like this does not concern governmental approval, is a little too confident by part of Orig3n.

The company has yet to offer a reason for why they believed “DNA Day” was going to be a good idea, why they have decided to pair with a football team to get it started and most importantly, for what they would be using these DNA samples for. Now why Orig3n thought it was going to be okay for hundreds of people to give up their DNA on a cotton swab without being given an explanation for it being done, is definitely sketchy at best. Since Orig3n has not come out with information about their plans for the DNA collection, based on their past tests offered through their website, it can be assumed that within a few months the hundreds of football fans who gave up their DNA will be given information about their skin aging, muscle force, language ability and vitamin D levels. As can be seen by this short list, none of these results would really influence a lifestyle change for anyone that is having these tests conducted and so, comes the question: why bother to do this at all.

The most this particular company can tell is that this person needs more get outside more and since this is not a significant amount of information that can be told through a strand of DNA, how is it that Orig3n has managed to lock a deal with a national football team? We are talking about a vast biotechnological industry full of companies that dominate the market simply because it can warn women that they are carrying the BRCA gene, or warn people for signs of genetic conditions and yet, the Ravens chose Orig3n. Since Orgi3n has yet to unlock the capability of warning someone of a serious disease or genetic condition it seems that they are looking into doing so, but, through Ravens fans that have no idea what they are in for.

Regulating CRISPR: How the US and the Rest of the World Compare

The gene therapies that are currently within clinical and observational studies today in the United States encompass treatments for various diseases and genetic disorders. However, the FDA had only announced at the end of last month that it approved Kymriah as the nation’s first gene therapy to be placed on the market as a treatment for acute lymphoblastic leukemia. This should come as a surprise considering how long scientists and researchers have been working within the genetic field of medicine but have only just been given the green light to put these treatments. It is the restraint of the FDA to approve genetic therapies that begs the questions: what are they afraid of and, is their fear going to put us behind the rest of the world?

The FDA has been known to be strict when it comes to their federal guidelines of accepting clinical trials of gene therapies before releasing them to the drug markets but it is significant that they be so tedious in their accepting of therapies that are the first of their kind. The United States is a country where litigation is man’s best friend and as a result, even corporations who fund gene therapies take steps to ensure that there are no serious repercussions can occur because of their negligence. One of the biggest fears facing the genetics community is the effects of creating or modifying human embryos, done to prevent future generations from getting genetic diseases or disorders from previous generations. An army of perfectly born humans can definitely been seen as something as a threat to the world but there are still a few countries who do not see perfecting humans and their genetics as something to be feared, but something to be researched, tested and even implemented. As can be seen, scientists have already been able to edit dangerous mutations in DNA and genes and in doing so, open up a can of worms that can never be closed again.

Currently, the United States is in its own boat when it comes to its stance on genetically modifying CRISPR embryos and this boat is one where the government does not completely ban the practice as other countries such as those in Europe and South America have. However, the regulation of the research being done and tested in monitored by private, independently based organizations. It is significant in noting that the most lenient countries of the world that do not ban the testing of CRISPR embryos are China, India, Japan and Russia. These four countries have already been known to conduct clinical trials in using CRISPR to genetically modify embryos and other types of cells within the human body, without much publicity. Since there are countries that are in the world right now who do not have to go through the FDA policies and guidelines before being accepted to conduct clinical trials or have the luxury of being monitored by private entities, there is no doubt a fear that these countries can endanger the lives of those around the world.

Especially with isolated countries such as North Korea, it is difficult to know how exactly the country has progressed in its biotechnological warfare, including gene therapy, and what the affects can be on the rest of the world when a country, although small compared to the US, can utilize science to become a super-powered population. It is to avoid scenarios such as the one posed by North Korea that the FDA was put in place. In order to ensure that the medicinal and agricultural sectors of the nation were being regulated and monitored, there needs to be an organization that oversees research that secure public health and safety. But, just having one country, out of hundreds with the capability to genetically alter genes and embryos with this agency in place, is not enough to guarantee that gene therapy will only be used for good.

To know that there are four major countries in the world right now who have little to no oversight can be unsettling to say the least and begins the debate on whether there should be global law or committee to oversee genetic manipulation so that it does not get out of hand. Being that it would be difficult to oversee global research into gene therapy, having at least a discussion about the global point of view, repercussions and growing opinions about this new generation of gene therapy and healthcare is best step forward in protecting the future generations that will be the ones to deal with the aftermath of gene therapy.