KEY POINTS

• CNBC asked biotech experts to weigh in on the biggest breakthroughs of the past decade.
• They include the first Ebola vaccine and a curative treatment for Hepatitis C.
• But much of the work of the past decade is foundational, and could be setting us up for many more advancements in the next ten years.

When it came to biomedical breakthroughs, this past decade represented a lot more hype than substance. We were promised a new era of “precision medicine,” where every patient would receive highly personalized treatments to target specifics fault in their genes. But in recent months, reports surfaced that these experimental therapies were failing most of the time.

Vas Bailey, a biotech investor with Artis Ventures, acknowledges that it might have seemed like a disappointing decade, barring a few important breakthroughs in the fields of gene editing and therapeutics. But Bailey thinks that there’s a lot more to the story.

In recent years, he notes, scientists have focused on the “tools and building blocks” that will drive the breakthroughs in next decade.

“With HIV, we made progresses in life expectancy but didn’t cure it; with gene-editing, we achieved one approval that could lay the foundation for others; with AI we learned how to develop it, but haven’t yet applied it; and with the microbiome, we have learned more about the relationships (between organisms) but we still haven’t designed next generation drugs.”

So with the help of a team of biotech experts, here’s our list of the most important advancements in biomedicine, many of are likely to propel great advancements going forward.

The roll-out of cell therapies

CAR-T sounds like the stuff of science fiction: Take blood from a cancer patient, re-engineer the blood cells to target and fight cancer cells, and re-infuse the cells in the human body.

But it’s real, with the U.S. Food and Drug Administration granting approval in 2017 to the first two drugs that use this approach: Novartis’s Kymriah for acute lymphoblastic leukemia (ALL) and Gilead Sciences’ Yescarta for certain types of non-Hodgkins lymphoma.

But this therapeutic approach has faced roadblocks, including price -- the drugs cost hundreds of thousands of dollars -- and the logistical complexity of making a treatment out of the patient’s own T cells. It can take up to a month to manufacture the cells while the patient’s health might be getting progressively worse from the cancer. But this decade also saw progress in companies making off-the-shelf T cells, which are made from healthy donor cells and used for multiple patients.

The next ten years could see advances in using these donor cells -- in particular, reducing the likelihood that patients’ immune systems will attack them -- by applying technologies and tools like CRISPR/Cas9.

And that brings us to...

The discovery of CRISPR/Cas9

CRISPR has been hailed as one of the most important breakthroughs of all time. It’s essentially a pair of molecular scissors, or a technique to make precise edits in DNA. It was discovered by scientists while exploring the immune system of bacteria.

Scientists have subsequently learned that there are risks to making these modifications. There are also ethical considerations, which were brought to the fore this decade when a scientist in China called He Jiankui reported that he had used the technology to create the first human babies with CRISPR-edited genes.

But this decade ended on a positive note. NPR reported that the first patient in the United States with a genetic disease -- a blood disorder called sickle cell anemia -- was treated with the CRISPR technique. The director of the National Institutes of Health Dr. Francis Collins, told NPR it could give patients a “chance for a new life.”

A cure for Hepatitis C

After decades of research into better treatments for Hepatitis C, a debilitating liver disease that could affect as many as 4 million Americans, the U.S. Food and Drug Administration approved a new treatment in 2017 that can reverse the disease in as little as 8 weeks.

The drug from AbbVie followed approvals for similar drugs from fellow drug makers Gilead and Merck, providing a welcome alternative to the prior regimen of shots and pills that didn’t always work and came with many side effects. Gilead, which saw its first drug for Hepatitis C approved in 2013, provided a cure in 12 weeks for about 90 percent of patients.

The drugs are extremely expensive, and led to a big spike in revenues for Gilead, but new entrants will provide more competition, potentially leading to lower prices.

HIV Prevention drugs

This decade, scientists did not cure HIV, the virus that causes AIDS, but they did make some major breakthroughs.

In 2012, the Food and Drug Administration approved Truvada, the first drug to reduce the risk of HIV infection in uninfected individuals who might be at high risk. The drug, which can be taken daily, is used for pre-exposure prophylaxis (PrEP) to lower chances of becoming infected with HIV if exposed.

Unlike some of the other breakthroughs on this list, Truvada has become more pervasive and widespread throughout the decade as costs have come down. The Trump Administration recently unveiled a plan to make these drugs free for people who don’t have insurance coverage, and virtual medicine companies like Nurx and Plushcare have sprung up to prescribe PrEP online without a lab visit.

The Ebola vaccine

In November of 2019, European regulators finally approved an immunization against Ebola. That decision means that Merck can market its vaccine and distribute it beyond Africa. It has also been approved by regulators in the U.S.

Several vaccines are in development to prevent the outbreak of the fever, which causes such symptoms as diarrhea and bleeding, but Merck’s is the only one that was tested during a real outbreak from 2014 to 2016. Ebola has killed more than 2,000 people in the Congo since the middle of this year alone.

Merck has said that it expects to start manufacturing licensed doses of the vaccine in the third quarter of 2020, and that it is working closely with the U.S. government, public health organizations and other groups.

The Ebola vaccine

These are just five of the breakthroughs from the past decade, which also saw developments in new “biologic” drugs, which contain or are produced from living organisms, advancements in prosthetics, and in a heartwarming twist, a new medicine for a single patient.

But as Bailey reminds, the best might still be to come. In the past ten years, scientists also made great strides to map out all the cells in the human body and to understand contributors to disease encoded in DNA. Teams of researchers also sought to create reference databases for the collections of microbes living in our bodies, and computer scientists teamed up with biologists to extract meaning from huge volumes of medical information, including X-ray imaging and pathology slides.

With all this in mind, Bailey thinks we could see in the next ten years regulatory approvals for the first blood test to screen for cancer at the earliest stages, new therapies that take advantage of our understanding of RNA (the intermediary between DNA and the proteins it instructs the body to make), drugs based on our ever-growing knowledge of the human microbiome, a cure for HIV, and novel approaches to ending multi-drug resistance.

What we’ve built in recent years are “enabling systems,” said Bailey, “and we will benefit from it in the next decade.”

This article was originally published in CNBC on Dec 28, 2019. Read it here: https://www.cnbc.com/2019/12/27/biomedical-breakthroughs-of-the-2010s-crispr-hep-c-treatment-prep.html