Cancer Cures and DIY Gene Therapy

February 17, 2016 | ProgressTH  News services around the world have recently reported on what Gizmodo calls a "revolutionary cancer therapy" that "shows promise in terminally ill patients."

It involves the re-engineering of a patient's T-cells so that the cells can then recognize and destroy cancer cells. The Gizmodo report claims: 
...more than 90 percent of terminally ill leukemia patients had their symptoms disappear completely. 
Terminally ill patients are those that are certain to die without intervention. A 90% rate of not only a response to the therapy, but full recovery is nothing short of extraordinary. 

The technique used is called "gene therapy," and gene therapy can be used in a wide variety of ways to treat many more conditions beyond leukemia. 

Clinical trials on both humans and animal models have shown promise for restoring sight, hearing, heart tissue, and even reversing debilitating genetic conditions and diabetes. 

And while gene therapy and its effectiveness in curing leukemia in terminally ill patients is making headlines this week, it has been under development for years and has in fact been proven in clinical trials as early as 2012. 

One of the first and most spectacular stories was that of Emily Whitehead. From a website created by her family as part of a charity organization set up in gratitude to Emily's second chance at life, it tells that story: 

Emily was diagnosed with acute lymphoblastic leukemia (ALL) at age five in May 2010 and relapsed twice. After the second relapse, the Whiteheads were told they were out of options to treat her cancer. Not willing to give up, the Whiteheads pursued a radical new treatment called T-cell therapy at Children’s Hospital of Philadelphia (CHOP).
Emily was the first child enrolled in the phase I clinical trial (known as CART-19 or CTL019) in April 2012. Her T-cells (a type of white blood cell) were collected from her body, and then, in a process using a disabled form of the human immunodeficiency virus (HIV), the T-cells were genetically reprogrammed to recognize and attack cancer cells. When the modified T-cells were put back into Emily she became very sick and spent several weeks in the intensive care unit on a ventilator. At one point her doctor said she had only a 1 in 1000 chance of surviving the night. Not only did Emily survive that night, but a few weeks later her family was given the miracle they had prayed for: the T-cell therapy worked. Doctors couldn’t detect a single cancer cell in her body. Today, Emily is nearing three years cancer free.
Four years later, it seems doctors and scientists are still making progress. However, gene therapy is still not an option for many children diagnosed as "terminal." Why?

It would be US President Joseph Biden at the very center where Emily Whitehead's gene therapy was developed, who would note that "cancer politics" stood in the way of faster progress. The Guardian would recount in their article, Joe Biden decries 'cancer politics' in kick-off of 'moonshot' to cure disease, that:
His meetings revealed a community rife with competition, territorialism and “stove-piping” of information that’s left researchers and their discoveries cloistered in their own corners, aides and others who met with Biden said. His campaign this year will work to encourage more data-sharing about patient data and treatment outcomes.
While there is undoubtedly truth to Vice President Biden's statement, those familiar with how these aspects of the biotechnology and pharmaceutical industry have developed and how they have been perpetuated, may realize that even if he wanted to, the Vice President would be fighting an uphill battle. Any genuine effort by government to break down these barriers and accelerate progress are surely welcome, but with literally life and death hanging in the balance, it is better if many others take responsibility in not only attempting to reform the existing system, but perhaps create another, parallel system altogether. 

DIY Gene Therapy? 
The use of DIY conjures in many's minds arts and crafts. More recently with the rise of global-spanning networks of makerspaces, it has come to mean electronics, rapid prototyping, robotics, and a whole host of other never-before-possible localization of manufacturing, research, and development. 

It also has come to mean something in terms of biotechnology, where professionals and amateurs alike have come together either at the edge of established universities and industry, or just beyond their fringes to pursue their own projects, research, and interests. 

For now, DIY biology remains in its early stages, but somewhere between DIYbio and big biotech corporations, lies an expanding landscape of start-ups that carry at their heart a wide variety of ethos. Some anti-establishment, some aspiring to find their place among it.

Where biotech startup Bioviva lies, only they themselves could say, but their CEO Liz Parrish's announcement that she tried her company's anti-aging gene therapy experiment on herself certainly breaks from conventions. How it relates to DIYbio and the promise of accelerating research, breakthroughs, and a speedy means of getting gene therapy to those who need it the most is very relevant. 

MIT Technology Review would elaborate on the experimental therapies Parrish had administered to herself. In their article A Tale of Do-It-Yourself Gene Therapy, they also mention: 
[Harvard Medical School genomics expert George] Church says he didn’t agree with dodging regulators and added that BioViva appears to be “a one-person show.” But he says he found Parrish’s claims plausible. A student in his lab, he says, could prepare a genetic treatment suitable for experiments in animals in a matter of days.
A well-equipped lab could indeed produce a genetic treatment for experiments in animals in the matter of days, and as technology and techniques become more easily accessible and as larger opensource communities expand it will get easier still.

See More: Interview with Liz Parrish of Bioviva

Parrish herself sees an industry and a regime of regulations posing a barrier for progress, and decided to take the risk herself to climb over them. Only time will tell if the gene therapy Parrish took works as expected, but the possibility of an industry and institutional outsider tackling gene therapy themselves has now been turned into a reality. Many others will inevitably follow her example, either out of curiosity or out of desperation especially when diagnosed with a terminal disease a gene therapy has been developed to cure, but a therapy they cannot get through traditional healthcare systems.

DIY Gene Therapy + Curing Cancer 

Bioviva and Parrish's work build on many of the ideas behind another innovator's bid to shake up human healthcare, Andrew Hessel and his Pink Army Cooperative. According to its website, the Pink Army Cooperative seeks to: make safe and effective cancer medicines available as fast as possible at the lowest price possible. We are accomplishing this mission by working with world-leading genetic experts to create computer-designed synthetic oncolytic viruses specific for individual cancers. This novel approach can produce laboratory-tested medicines in just a few days at very low cost while potentially avoiding the slow and expensive phased clinical trials required to bring a mass-market drug to market. The cooperative business structure ensures that the research and development done can be openly shared with the scientific, medical, and patient communities.
For those dying from cancer, facing what is essentially a death sentence, there isn't much to lose by taking something that is experimental. People already do this all over the world and in a variety of ways. With gene therapy, there is no doubt that if done properly, it can work. The only doubts that linger are when it will be accessible to the public and how much it will cost.

Large companies already admit that gene therapy poses serious obstacles to their current business practices. Cheap to produce and effective usually with just one injection, entire portfolios of antiquated treatments and pharmaceuticals will be wiped out. 

People may condemn these companies for either not pursuing what is essentially the cure for many of the most devastating diseases humanity faces because they are not profitable, or for taking them up, but charging absurd prices for them based on "value" rather than on actual cost. But if the Vice President's condemnation doesn't make them flinch, likely neither will ours.

Instead, it is clear that if we want access to gene therapy, we will have to get it ourselves. Following the examples of Parrish and Hessel who have invested time in understanding the technology and assembled teams of professionals dedicated to solving the problem rather than profiting from it, DIYbio and DIY gene therapy can go from a promising vision of tomorrow's healthcare to the cures of the incurable today. 

Check out your local DIYbio lab, get involved at local university labs, or just simply begin talking about gene therapy with others who might not yet know about this. Individually there is not much we can do, but together, there is nothing we can't do.