This USC Innovator Explains How Biotech Can Enhance Humans

Today, the phrase "have and have nots" could refer to those with or without access to digital communications. As more of our daily lives—employment, paying bills, healthcare, and banking—move to the web, life becomes harder for those without internet access.

But in the next decade, the phrase might mean "modded or unmodded"—as in, "have you been modified?" With biotech advancements such as brain-implanted neural chips, stem cell research, and gene editing, enhanced humans could usher in a new phase of evolution.

Of course, many of us are enjoying a physical or mental upgrade already. Following life-threatening surgery in 2011, I now rely on medicine to replace an organ function, so I've essentially been "hacked"—as have many people I've met, including those who wear bionic limbs.

But how are the "big thinkers" preparing us, and government agencies, for humans with added powers? I spoke with Dr. Amanda Mason, Assistant Director for Strategy and Planning at MESH Academy within USC's Keck School of Medicine, to learn how her research will prep us for Humans 2.0. Here are edited and condensed excerpts from our conversation.

Firstly, Dr. Mason, can you define biotech, and how it has evolved?
One way to sum it up is: "biotech can heal, feed, and fuel the human race." It's not just about medications and new treatments, but also getting better crop yields and more nutritious crops, and biofuels which can replace the carbon-intensive fuels we rely on now. It's pretty broad. In the end, it's biology plus technology—innovations based on the way living cells work [and] harnessing those processes to create entirely new entities.

You were awarded a PhD in developmental and stem cell biology. Can you explain how stem cells were used in your research and to what end?
There are 6 million Americans living with Alzheimer's today, and the number is growing. It's an incredibly important field to be in—lots of researchers [are] trying to solve this problem—and I was one of them. I worked with pluripotent stem cells, which can develop to become any cell type in the body. I worked with these stem cells in the pursuit of finding treatments for a rare form of dementia.

How did you obtain the stem cells, then use them?
I took skin cells from patients with the disease, then "reprogrammed" them to become pluripotent stem cells, then I turned those cells into neurons [brain cells] and studied them. By taking cells from patients with this disease, I studied—at a cellular level—what was going on inside their brains. I identified a particular pathway, or interaction between proteins, in the cells. We know that certain forms of dementia are caused by too low levels of a certain protein, and I found a pathway to change the level of that protein—to turn it up or down.

To clarify, you took stem cells from the patient, examined them, worked out how to dial up the protein level in the cell itself and then replanted them back into the patient?
Yes. It was a new strategy for dealing with this particular form of dementia—a potential way of changing the trajectory of the disease. However, with research, it would have taken years, a decade, maybe more, to go through trials and so on, before we could have brought a product to market.

Which is why you didn't stay in academia.
To be honest, while I was doing my PhD, I fell in love with startup companies during an assignment at Annexon Biosciences. Then I joined the consulting firm Bain and Company. There I learned how pharma, biotech, laboratory supply companies, and others in this space, become successful—what it really takes—getting vital experience through pipeline planning, up to mergers and acquisitions.

Startups have a faster pace than academia, that's true.
[Laughs] Right. Research is fundamental. But at a startup, everything you're doing will have such a big impact on people's lives—in the near term—and there's nothing more exciting than that.

Is your current role at USC a perfect blend of academic rigor and fast startup culture then?
In a way, that's it, exactly. As Assistant Director for Strategy and Planning, I'm working within the new MESH Academy, which stands for "Medicine, Engineering, Science and Humanities" [and is] part of the Keck School of Medicine [at the University of Southern California]. My role is to forge cross-departmental and multi-disciplinary working parties to solve the biggest challenges in health and disease. Connecting engineers, chemists, biologists, mathematicians—to bring a variety of expertise to bear on the issue at hand.

Because silos occur in even the best-run joints.
It's true. We've found researchers working on similar issues, in completely different fields, who have never met and yet are pursuing complementary goals. My job is to bring them all to the table and create something of their joint efforts.

Do you come from a medical family?
Not at all, but my parents are very curious people, and encouraged my academic career—in large part because neither of them had gone to college themselves. Having said that, my dad did decide to go back and study labor relations—just as I started at Harvard—and we graduated in the same week. He's now the President for the Brotherhood of Railroad Signalmen. It was my dad who inspired me towards science. We would build rockets together and my fondest memories were of going to "star parties" at a local observatory where all these telescopes were pointing at celestial bodies.

UC San Francisco, where you earned your PhD, has some fantastic geek gear. Did you use robots in your PhD lab research days?
Oh, yes. I was based in the Gladstone Institutes with Professor Steven Finkbeiner and we had robotic microscopes which we made ourselves. We'd set up an experiment and the robotic microscope would run all night, 24/7, assessing what can be seen happening over time. The robotic arm would take out the dish of cells from the incubator [and] take pictures to record what has transpired. So I didn't have to come in at 4 a.m. and check on its progress, then put it back in the incubator.

When I interviewed your USC colleague, Dr. Yolanda Gil (the incoming President of AAAI), she said that AI is a great lab partner.
That's so true. It enabled us to record and see minute changes over time in our experiments. The robotic microscope had the capability to examine 96 or 384 wells on each dish of cells, with different types of cells or growing conditions in each one, and, through its monitoring, we could track cells dying and compare diseased cells to healthy cells. Throughout this process, the robotic microscope was generating terabytes of data. I felt incredibly lucky to work with such powerful instruments at UCSF.

What's your vision of biotech's future?
I see a wide use of CRISPR, which is a type of gene editing, to correct disease—especially in the blood, which is easier to do [than other tissue types]. Then, as the technology matures, we will see editing in other tissues, and a movement away from the existing focus on cancer treatments.

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Focused on adult humans, correct, due to legal reasons?
Yes, because, in the US, editing embryos is illegal right now. The FDA regulates this area and, under current law, it can't consider any trials using gene editing on human embryos which would then be implanted—i.e. have the chance to be born.

Because, right now, we don't know if the resulting human/hybrid would be "enhanced" or something out of a really bad movie.
Right. Having said that, the laws are different in China and in other nations. But I think we may see one day the use of editing human embryos to eliminate disease. Right now, during the IVF process, patients are able to pick the fertilized embryos which don't have certain mutations that happen to be carried by one or both parents. Parents can choose the non-mutant embryos and thus avoid certain devastating and life-threatening illnesses.

It's a tricky and emotive area.
Yes, it is.

Can you explain the significance of the Human Genome Project, and its potential role in enhancing human evolution?
Good question. The importance of the HGP was in giving us an ability to read all the A,C,T,G base pairs contained in human DNA—for the first time ever. Essentially, giving us all the instructions to build a human. Once we can import that knowledge to biotech, once we fully understand the instructions in our very cells, we can create great breakthroughs.

Do you then see a threshold emerging of those "with mods" and those without? Essentially cyborgs versus those who, well, are the "basic bio model"?
I take a more measured view of this.

As opposed to my sci-fi fantasy using the word cyborg?
[Laughs] Perhaps. But let me say this—it's important for us to think about the "mods" we already have available. After you contacted me for this interview, I sat down and made a list, and was surprised how many I came up with, across many medical categories, including: protection from pathogens (vaccinations). That's an interesting "mod" which we take for granted, protecting us from diseases which killed previous generations. Then there are the losses of function, replaced by "mods"—a new knee joint, for example, or a synthetic lens in the eye; even a retinal implant or an artificial heart—there's actually a crazy amount of modifications available today.

But there will be many more. Going back to your own PhD research using stem cells to dial up/down proteins as a possible treatment for dementia.
I guess my interpretation is quite different from a black and white "have versus have not" scenario. With real consideration on the subject, I've realized the pace of these discoveries have been robust over these past few decades, and I hope for a continual gradual process of innovation. Through our work in biotech, I hope these breakthroughs keep being deployed to improve and save lives.

Fair point. Can you tell us what's next on your biotech agenda?
We're planning for our interdisciplinary stem cell symposium, hosted by the MESH Academy and USC Stem Cell, on Nov. 27. I'll be giving a speech on the resources offered by my team, MESH, to entrepreneurs and innovators working on stem cells and we have invited experts from across different fields that intersect with stem cells—including biology, chemistry, engineering, medicine, and business—to share their latest breakthroughs in biotech research and forthcoming treatments.

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