Low Cost Biotechnologies can be Inconvenienced but not Halted by Regulators

The coming era of gene therapies will be considerably more distributed and bottom-up than the advent of stem cell therapies. This will be a dynamic industry in which many small groups compete to set up distribution of mail order kits and clinics to provide widespread access to therapies. Regulators will attempt to suppress all of this, and will largely fail, as money talks and many regions will choose to host the businesses that offer gene therapies. This will come to pass because gene therapy technologies are many times cheaper, more easily managed, and capable of centralization and mass production than stem cell technologies. You might look at how medical tourism for stem cells progressed over the past twenty years, and expect the gene therapy industry to grow many times faster once the spark is lit. It will also be far more accessible to members of the public in its earlier stages: cost of the product drives the character of an industry.

There are several very promising targets for the first gene therapies, the best of which, in my opinion, are follistatin and myostatin, which control muscle growth, and are well studied. There are even a few natural human myostatin mutants, to accompany the many well-muscled myostatin mutants in other mammalian species, both natural and engineered. A number of other genes will be targeted in the first years of the industry, such as those that can dramatically lower blood cholesterol, and which also either have thriving human mutants or are already targeted by drug-based therapies. The only thing holding back an explosion of activity is the fact that current methodologies, even those based on CRISPR, are not yet up to scratch. They don't reliably introduce the therapy into a large enough number of cells in adults, and particularly into stem cells in order to make it truly lasting. When that changes, we'll all be in for an interesting ride.

Two companies say they'll continue offering DNA-altering materials to the public. The companies, The Odin and Ascendance Biomedical, both recently posted videos online of people self-administering DNA molecules their labs had produced. Following wide distribution of the videos, the FDA last week issued a harshly worded statement cautioning consumers against DIY gene-therapy kits and calling their sale illegal. A growing number of cases of DIY gene therapy are putting the health regulator in a difficult situation as individuals argue that no law stops them from self-administering the substances. In fact, there is a long history of scientists carrying out experiments on themselves, including some Nobel Prize winners. Last month, Josiah Zayner, CEO of The Odin, which sells DIY biology kits and supplies through its website, posted a video in which he injected himself with the gene-editing tool CRISPR during a biohacker conference.

The problem facing regulators is that interest in biohacking is spreading, and it's increasingly easy for anyone to obtain DNA over the internet. It's also easy to get hold of the recipes necessary to carry out gene editing using CRISPR, a potent new technique for modifying DNA. In October, Zayner's website began selling $20 copies of a DNA molecule containing the necessary genetic information to deactivate the human gene for a certain protein, myostatin, using CRISPR. Human DNA can be purchased through a number of other companies that cater to research labs. The difference is The Odin markets its DNA to amateur biologists. The materials sold by The Odin also can't be directly used to alter a person's genes. Instead, they contain DNA that would have to be produced in larger amounts, purified, and then delivered to the body using methods well beyond the skills of most consumers.

At least one other company appears to have begun offering finished gene-therapy preparations directly to patients for their own use. In October, an HIV patient was filmed injecting himself with a gene therapy designed to generate antibodies that he believed would help his body destroy cells infected with the virus. The material he used was supplied by Ascendance Biomedical, an until recently unknown startup company that promotes "decentralized" testing of new drugs. The company is also developing a herpes vaccine, as well as a follistatin gene therapy to boost muscle mass and reduce fat. Aaron Traywick, the CEO of Ascendance, says Ascendance plans to make both of those therapies available for self-administration by early next year.

Link: https://www.technologyreview.com/s/609568/biohackers-disregard-fda-warning-on-diy-gene-therapy/


I think scientists at the Fred Hutch have already developed interesting nanoparticles to deliver gene therapies:


"And because Stephan's nanoparticles are dried, they don't need special shipping or storage conditions. The simplicity of transporting and using the nanoparticles means that they could bring short-term gene therapy to areas of the world lacking high-tech cell-processing facilities - exactly where most HIV patients are, Kiem said. If someday their cure could be shipped in unrefrigerated vials, "that's much more attractive, much more doable.""

The question is, could a small group cheaply produce these nanoparticles and sell them via mail order?

Posted by: Jim at December 4th, 2017 7:21 AM

Yeah, you would not catch me doing this. I want to know therapies are safe and effective and have passed appropriate trials before I use them.

Posted by: Steve Hill at December 4th, 2017 8:03 AM

I'm pretty happy to see these kind of things popping up. Truth, I've been wanting to look at CRISPR and start playing around myself. Not ON myself per se, however with plants I think I could learn a lot and have a great deal of fun. Always been a bit of a gearhead. We've seen a few results so far in horticulture/agriculture and when I think of gene engineering and plants, I always think of the real life Tomaco.


If people want to self experiment with CRISPR, its only going to add to our knowledge base.

Now we may have some people call for bans and there will be some screaming groups out there trying to kill this, but to be honest, they will lose. CRISPR is so easy and cheap, there's going to be a lot of competition to bring products to the market faster.

Posted by: Mark Borbely at December 4th, 2017 9:37 AM

Very exciting, but dangerous. And intriguing. I hope I can be excused if I let a few hundred others get in line ahead of me. But when the time is right, I'll pay with bitcoin :)

That was a good insight, people avoiding regulators in gene therapies. We've all put up with their obstructionism for decades, but now the internet and decentralized finance is dodging round all the old gatekeepers. I won't mourn their passing, but at the same time everyone's going to need to be extra careful about the medical choices they make. Some of these clinics will dispense miracles, while some will turn out to be worse than useless.

Posted by: Paul at December 4th, 2017 9:48 AM

@steve, a prudent approach if you are 40. A 60 year old may have not the time to wait and see such official recognition

Posted by: JohnD at December 4th, 2017 10:28 AM

@JohnD obviously and I can afford to hold fire.

Posted by: Steve Hill at December 4th, 2017 10:37 AM

These people are pioneers for attempting this work.

But they are fools to rub FDA nose in it.

In doing so they paint a big target on their back.

Posted by: Qing Song at December 4th, 2017 12:44 PM

@Steve - this wider access to gene therapies won't just shake up longevity treatment. What if you were unfortunate enough to be a poor sod living in a poor developing country, or the US without insurance, and you got diagnosed with Leukemia and couldn't afford an expensive approved CAR T therapy? Would you be tempted to buy some of this for a couple of grand off an overseas clinic?


"Nanoparticles turn immune cells into leukemia-fighting powerhouses while they're still inside the body

In the current study, Stephan and his team developed biodegradable nanoparticles that carry CAR-encoding genes and are tagged with molecules that make them stick like burrs to T cells. Once a T cell engulfs the particles, they hitch a ride along the cell's internal traffic system to the nucleus. In this preliminary study, Stephan and his team designed the new CAR genes to integrate into chromosomes housed in the nucleus, making it possible for T cells to begin decoding the new genes and producing CARs within 24 to 48 hours.

Once the team determined that their CAR-carrying nanoparticles reprogrammed a noticeable percent of T cells, they tested their efficacy. Using a preclinical model of leukemia, Stephan and his colleagues compared their nanoparticle-programming strategy against chemotherapy followed by an infusion of T cells programmed in the lab to express CARs, which mimics current CAR T-cell therapy.

The nanoparticle-programmed CAR T cells held their own against the infused CAR T cells. Treatment with nanoparticles or infused CAR T cells improved survival 58 days on average, up from a median survival of about two weeks. "

Posted by: Jim at December 4th, 2017 9:25 PM

That's amazing Jim, I can see this really taking off.

Posted by: Mark at December 5th, 2017 3:03 AM
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