‘It’s the next rising platform’: 2 promising technologies backed by billion-dollar drugmakers could transform the human body into a disease-destroying machine

Samantha Lee/Business Insider

Technology is giving scientists better tools for developing innovative new medicines.
More than 30 drug companies are betting on an experimental approach that leverages the trash cans naturally found in the body to obliterate disease.
Another promising new technology could turn the human body into a medicine-making factory, eliminating the need for injections at the doctor’s office.
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Donald Kirsch, a longtime drug developer, knows where you think medicines come from.

At parties, he hears it all the time. People have a vague notion of a highly-scientific, technical process in which pills are methodologically engineered, just like a smartphone.

The reality is far different: the profession is called drug hunting for a reason. Kirsch, who wrote the book on how new drugs are discovered, specializes in the very earliest parts of the process, when a medicine is just a whisper of an idea, or a science-guided theory about how to target disease.

Drug hunters then screen an enormous number of chemicals or proteins for one that matches the profile, before working to enhance it and then test it out.

"It’s mainly luck and serendipity," said Kirsch, who has spent more than 35 years making medicines, including at Bristol-Myers Squibb and Wyeth, before its takeover by US drug giant Pfizer. Today he teaches an online evening course about drug discovery for Harvard Extension School.

"With farming, you plant seeds and know that, unless there’s a catastrophe, plants are going to come up," he said. "With hunting, you go into the chemical woods and hope the chemical is lurking out there. But you don’t know for sure."

Technology has led to sweeping change in how drugs are developed, paving the way for new treatments of highly complex diseases. Now, it has the potential to transform the tools of drug hunting even further, putting decades-old scientific pipe dreams within reach.

Scientists are pioneering two promising new approaches to treating diseases

Armed with advances like better genetic knowledge and testing, scientists are pioneering approaches to treating disease that take advantage of the body’s natural capabilities in bold new ways.

Two especially promising ones are targeted protein degradation, which leverages the body to destroy disease, and gene therapies that could transform the human body into its own drug-manufacturing factory.

Both are in the earliest stages of development, and there aren’t any drugs on the market that leverage either technology. But if experiments succeed, they could upend the traditional limits on what medicine can do.

Using the human body to treat disease is an old idea, one that these new technologies are tapping into in cutting-edge new ways. Another field coming of age today that exemplifies that is immuno-oncology, an innovative way of treating cancer by revving up the immune system.

Immuno-oncology has had remarkable results for some cancer patients, spurring large investments from biopharmaceutical companies in the area.

Yutong Yuan/Business Insider

As the cancer treatments have become more widely used, though, doctors have also been seeing patients suffering from unusual side effects, like organ inflammation and diabetes. The very thing fighting the cancer also unleashes the immune system in harmful ways.

New science that delves deeper and deeper into the human body has to grapple with the limits of our understanding of how the body works, says Jill O’Donnell-Tormey, CEO of the nonprofit Cancer Research Institute.

"You work with a powerful system and there’s kind of a price to be paid for that, I think," O’Donnell-Tormey told Business Insider.

Targeted protein degradation: The new technology that could change how we treat everything from cancer to Alzheimer’s

For drug companies ambitious enough to tackle new treatments for the memory-destroying disease Alzheimer’s, the result has been failure after failure.

Exactly what drives the disease has so far eluded scientists. And it’s been difficult to design treatments targeted at one of the prime suspects thought to play a role in the disease, "tangles" of proteins in the brain called tau.

"Nobody’s been able to drug tau," said John Tallarico, site head of the chemical biology and therapeutics department at Novartis Institutes for BioMedical Research.

A new scientific technology that Novartis and at least 30 other companies are betting on could change that, opening up "unlimited" possibilities in Alzheimer’s disease, cancer, respiratory disease, transplantation and autoimmune diseases, Tallarico says.

Most drugs you take work like a light switch. The drugs either flick the switch off, slowing down what’s driving a disease, or on, instigating a more helpful bodily process. For instance, certain common prescriptions that lower your blood pressure are the first type, because they reduce a mechanism that drives blood pressure up.

The latest technology takes an entirely different approach. Called "targeted protein degradation," the drugs use a garbage disposal system that naturally exists in our cells to get rid of disease-causing proteins entirely. You can think of it like tearing down the light switch and the wall entirely and going straight to the source.

That’s "game-changing" for scientists at Novartis and elsewhere, according to Tallarico, because the tech redefines what a drug needs to be able to do, in the process dramatically expanding the universe of potential medications.

It also infuses new energy into common chemically-made drugs, which the pharmaceutical industry has started shifting away from in favor of more complex drugs called biologics. Yutong Yuan/Business Insider

Targeted protein degraders still have to be tested out in patients. One biotech, New Haven, Connecticut-based Arvinas, just started that process, but as a whole the technology is still years away from being sold in pharmacies, said Craig Crews, the Yale University professor who pioneered it.

Because targeted protein degraders are large, with molecules roughly twice the size of most chemically-made drugs, there has long been skepticism that they will translate to pill form. Arvinas has been able to do it, though, according to Crews, who is the biotech’s founder and chief scientific advisor.

The tech is burgeoning today, with companies small and large jumping in.

"I couldn’t be more excited," Novartis executive Jay Bradner told Business Insider earlier this year. "It’s the next rising platform."

A next-generation gene therapy that could turn your body into a medicine-making factory

In 2015, when the biotech MeiraGTx was founded, decades of scientific work in the field of gene therapy was poised to take off.

Gene therapy is a powerful technology with the ability to fix problems at the root, genetic level, by altering your DNA. Inherited conditions are an obvious target, as gene therapy could potentially treat those diseases or even cure them.

Yutong Yuan/Business Insider

MeiraGTx’s founders were interested in those uses; the biotech’s programs today are in diseases of the eye, salivary gland and brain.

But "we wanted to have a broader perspective on how you could potentially use gene therapy" too, CEO Alexandria Forbes says.

That vision is a high-tech, futuristic one, in which the human body can essentially become a medicine-making factory, enabled by gene therapy. But it’ll require more research, and is still years from fruition.

For the millions of people who take complex, pricey drugs that must be injected, rather than taken as a pill, this could make life more convenient, and maybe less expensive. Imagine: instead of going to the doctor once a month for a drug that treats vision loss, you’d instead take a simple pill that kicked off the production of the drug in your own body.

"Is that possible? It might be possible, and our technology is working towards that," Forbes told Business Insider.

MeiraGTx calls this gene regulation, because it would require being able to start up and then stop the process in a way that doesn’t currently happen in gene therapy. The $370 billion drug giant Johnson & Johnson has also signaled its interest, with a research partnership on the tech announced last fall, the terms of which were not disclosed.

Gene regulation "has always been a goal" for scientists, at least in part because it’s reversible, unlike other forms of genetic engineering, said Stanley Qi, an assistant professor in Stanford’s department of bioengineering.

The field has long faced logistical and technical challenges, but Qi predicted "enormous progress" for it in the next three to five years.

Meira’s approach homes in on another key player in genetic information, the RNA that changes DNA into usable instructions for the body. The biotech created its own gene controllers by using pieces of RNA with a specific shape, taking advantage of the way RNA shapes naturally turn genes on and off in the world of bacteria.

The switching process has worked in animals, according to the biotech, although it hasn’t yet been tested in humans.

If it works, all kinds of new treatments could be possible. Drugs for diseases of the brain encounter a barrier that limits their effectiveness, Meira’s Forbes noted, but this treatment could be put directly in the organ, for instance.

Another application might be in treating obesity, she said. Gut peptides help the body take in food and feel full, and thus have become a natural focus for obesity researchers. But the peptides also don’t stay in the body for very long, making it hard to turn them into medications. Gene regulation could render that and other issues with dosing medications moot.

But in a field that’s notoriously prone to failure, longtime drug developer Kirsch says we need to stay humble.

"I think we always have gotten better and better," he says.

"If you ask me where we’re going to get better, I’ll tell you when we get there… I couldn’t have predicted a lot of this stuff."

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