CNBC | May 24, 2019
CNBC’s Healthy Returns Summit was held earlier this week in New York City, featuring keynote presentations and panel discussions on the future of the health-care industry. Topics ranged from controversial genetic-editing technique CRISPR to the way AI is changing the practice of drug discovery and medicine and reform of the U.S. health-care system. An audience of health-care leaders from the private and public sector shared their views. Here are some edited excerpts.
A Chinese scientist last year made history by using CRISPR technology to genetically modify two newborns, pushing the ethics of human germline gene editing to the forefront of public debate.
Dr. Samarth Kulkarni, CEO of CRISPR Therapeutics, said what the Chinese scientist did was “a bit unfortunate,” but he said there is no going back. “CRISPR is here to stay,” Kulkarni said. “The technology itself will become a mainstay.”
CRISPR Therapeutics announced in February that it treated a patient for sickle cell disease with gene-edited hematopoietic stem cell therapy. CRISPR Therapeutics does not conduct any germline gene editing.
Former FDA Commissioner Scott Gottlieb said at Healthy Returns that the Chinese scientist’s research was a “horrible experiment and it established a horrible precedent” that risked causing people to “rightfully” turn away from the science.
Cannabis company Tilray is working with the Sandoz generic drug business of Novartis, supplying non-smokable and non-combustible medical cannabis products where it is legally allowed. The deal, announced in December, was the first major partnership between a pharmaceutical company and a cannabis business.
But Novartis CEO Vas Narasimhan said that cannabis is not a priority for the Swiss drugmaker despite the partnership.
“I don’t know what the deal is doing. It is a relatively small distribution deal for us. For us it was more the opportunity to use the Sandoz infrastructure around the world to support Tilray. Cannabidiol is not a focus for the company.”
The Novartis CEO said the science just is not there yet to speak to drug development. “We need good studies to understand the data.”
Novartis just received approval from the FDA for its $2 million gene therapy treatment for spinal muscular atrophy.
Biogen recently joined a long list of companies that failed to find a cure for Alzheimer’s disease after promising early drug trials. Venture capital firm Andreessen Horowitz general partner Jorge Conde said a cure for the disease, which affects 5.8 million Americans, is still far off.
“We don’t even really know the very first layer, so the probability that you’re going to find the right target and hit it is going to be unlikely, unless we can get that first level of understanding the disease,” he said.
“One of the things we look for when we make investments, we talked earlier about binary risk, is when developing a new therapeutic, you have to understand what the disease is doing. You have to understand what you need in it to affect the disease process, and then you have to have the capability to actually make that molecule or that cell or that gene.”
Alzheimer’s is “very, very difficult to solve, and it’s going to require new technology to do it, and I just don’t think we have the technology,” Conde said.
President Donald Trump has said in the past that the drug industry is “getting away with murder,” and there has been a rising tide of public backlash against drug pricing, in particular.
“We are facing those issues, and frankly, some of those issues with the industry have been self-inflicted,” said Johnson & Johnson CEO Alex Gorsky. “We need to do a better job in talking about the impact we have on lives and the pricing of products and advertising.”
Asked what his company’s relationship with the Trump administration was like, Gorsky said, “Like anybody else, we try to stay focused on the facts and issues because, look, we are in a very political world these days, and more than anything else, one of the things we try to do is make sure we are educating people about health care and some of these issues. … We hear a lot of broad, sweeping statements that are not supported by underlying facts.
“Anyone who is in this industry will likely be participating in and working with some type of health-care reform over the next several decades.”
The J&J CEO also stressed that there is a flip side: Fifteen years ago the industry was worried about redundancy in therapies, but today it is a much different conversation.
“We are sitting around in conference rooms frequently now, talking about the opportunity to transform something like multiple myeloma, which could have been a death sentence 5 to 10 years ago, to a cure.”
Gorsky also spoke about breakthroughs with the hallucinogenic ketamine as a treatment for antidepression and its ketamine-derived nasal spray Spravato, approved earlier this year.
“We were very excited we got breakthrough designation with the FDA. It’s the first new medication in over 30 years in this space, but it is still early days,” Gorsky said. “The bar to get over in showing efficacy, you had to show efficacy on top of another antidepressant, and the history of phase 3 trials with antidepressants is loaded with failed trials.”
This week Google made major news in health care when a study conducted using its AI technology as a lung cancer diagnostic tool succeeded. But the advances in AI for medicine should be met with caution, members of a Healthy Returns panel said. Google’s study was limited to patients who had already been treated, so the company could not make claims about new patients. It also would likely require a randomized controlled trial before it could be put into actual practice.
Peter Lee, corporate vice president of Microsoft Healthcare, said there are so many places where machine learning and AI can be applied in the diagnostic space, but the rapid advance of technology cannot be allowed to supplant scientific protocols.
“There is a process that has been honed over a century, going through peer review and scientific validation that is sometimes skipped in hype and buzz around AI,” Lee said.
Lee noted that it is not at all clear that even if we could review the genes of all humans — in the entire history of the planet — we would know what to make of the data.
“When you put the complexity of human biology versus fewer than 110 billion people who have ever existed, even if we had genetic samples for all of them, it is not clear we would have enough data if we just took a machine-learning approach.”
But Lee indicated that the general direction in the health-care industry of digitizing records is a key trend.
“Fifteen years ago less than 10% of health records in the U.S. were in digital form … to over 95% today. It is an incredible pace of change, but incredibly messy today, and there is lots of work to do to address problems. But at least there has been that first step.
Bioprinted organs are coming within a decade
Martine Rothblatt, founder and CEO of United Therapeutics, believes that bioprinting of many human organs, including lungs, hearts and kidneys, is coming by the end of the 2020s.
The technique involves growing a modified tobacco plant, which has genome that is “fairly easy” to modify, Rothblatt said, so the plant produces human collagen, which is the most abundant protein in our bodies. The collagen is separated from the tobacco and used to 3-D print a framework for a human organ, referred to as a scaffold, which is then cellurized with the patient’s own cells. When the organ is transplanted back into a patient, it will seem to the patient as their own and not require use of immunosuppressants.
“This is our earthshot for the 2020s. I know some people feel, ‘Wow, Martine, that sounds like a moonshot,’ but I believe before the end of the 2020s, we will have completely bioprinted organs — one heart and kidney matching a patient’s own DNA, transplanted into the patient — and the patient will be able to leave the hospital with no immunosuppressants at all.”
Democratic candidates for president are pushing a government-run health-care system that will never pass, said President Barack Obama’s former budget chief Peter Orszag.
Orszag compared the “Medicare for all” policy as the liberal equivalent of Republican bids to end Obamacare.
“Single-payer is the Democratic equivalent of repeal and replace. It’ll never be legislated, because the details are too hard,” said Orszag, now CEO of financial advisory at Lazard.
He thinks the public will begin to see a “bogeyman” version of government-run health care emerge after the 2020 presidential election.
Full panel discussions from Healthy Returns are now available to watch on CNBC.com.