VIDEO: NIH’s Cancer Chief Sees Advances as Historical

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By Jeannie Baumann

Immunotherapy sounded like science fiction the first time someone described it to Ned Sharpless long before the White House tapped him to lead the nation’s cancer research programs.

“It really did not sound like a real thing. But now we’re curing people with that approach,” Sharpless said from a building on the National Institutes of Health’s campus in Bethesda, Md., that overlooks the world’s largest research hospital.

Immunotherapy essentially trains the immune system to fight off cancer, and has been shown to be highly effective in some cases. When Novartis’s Kymriah ( tisagenlecleucel) cleared the Food and Drug Administration last summer, it marked the first-ever FDA approval of a treatment using CAR-T, a type of immunotherapy that specifically uses a patient’s own T-cells to attack cancer cells. The approval of another CAR-T therapy, Kite Pharma, Inc.'s Yescarta ( axicabtagene ciloleucel), followed a few months later. Other therapies are currently undergoing testing, such as a Bristol-Myers Squibb’s i nvestigational immunotherapy treatment for prostate cancer.

“It’s an exciting new area of therapy, and we’re just really at the beginning of understanding how it’s going to work,” Sharpless said.

The pioneer of immunotherapy is Steven A. Rosenberg, chief of surgery at the National Cancer Institute, part of the NIH. But not everyone in the cancer community was as excited about this field as they are now, Sharpless recalled. “Many people thought Steve was absolutely insane—for about 20 years.” Thankfully, Rosenberg persevered, he said. “This was a long time coming, and we don’t know yet how far it’s going to go.”

The success of immunotherapy is a testament to why Sharpless believes the NCI needs to maintain its bottom-up, investigator-initiated approach to science, and that it’s imperative to fund both basic and clinical research. By limiting bureaucracy and creating an atmosphere that lets research flourish, he said, those so-called crazy ideas can really pay off.

Norman E. “Ned” Sharpless became the NCI director Oct. 17, overseeing a $5.3 billion annual budget, which is the largest of the NIH’s 27 institutes and centers. His position at the agency is the only one besides the NIH director whom the president must nominate. Sharpless spoke Jan. 24 to Bloomberg Law about some of his priorities and why he thinks some of those “eye-popping” prices for cutting-edge therapies will come down.

Exciting Times

A deeper understanding in the basic biology of cancer, its genomic underpinnings, and the discovery that cancer is a group of thousands of diseases instead of a single disease make it an exciting time to lead the NCI, Sharpless said. He likened current cancer researchers to Robert Koch and Louis Pasteur, whose discoveries in how bacteria worked in the 1800s changed the course of treatment for infectious diseases like tuberclosis, anthrax, and cholera.

“We’re going to remember cancer researchers today the way we talk about Pasteur and Koch and antibiotics,” Sharpless said. “It’s just that kind of time going on for cancer research.”

Financial Toxicity

As excited as he is about the potential to help cancer patients, Sharpless acknowledged some treatments come with a hefty price tag, such as Kymriah’s $475,000 estimated cost. Even without these cutting-edge treatments, the total cost of care for cancer patients has jumped to about $50,000 a year. Ensuring patient access to treatment is a very important issue for the NCI. “This leads to this issue of financial toxicity,” he said. “We have data that this is a significant barrier to care.”

The price of CAR-T and other novel therapies will drop over the years, as patents expire and as the technology and manufacturing improve, Sharpless said. When statins first came out in the 1980s, there were concerns these cholesterol-lowering drugs would “break our ability to pay for care,” he recalled. “But now those drugs are available on the $4 list at Wal-Mart.”

Data Sharing

Sharpless is still on what he described as a listening and learning tour before he fleshes out specific NCI priorities and policies, but he’s already expressed an interest in data sharing. It can help every facet of cancer research, he said, from an investigator running a clinical trial deciding what order to administer drugs, or what dosing levels to prescribe to health disparities researchers who want to figure out why some populations don’t benefit as well from certain treatments.

Because cancer isn’t one disease but spans 10,000 or more different types, it’s impossible to get enough data from a single clinical trial, he said. “The way to get at these problems is by aggregating large numbers of patient-linked data and then seeing what happens. And I’m not talking about 1,000 patients. I’m talking about a million patients.”

Data sharing isn’t cheap and there are still privacy and security issues that must be addressed, he said. But failing to share data also comes at a cost in terms of understanding the effectiveness of these treatments. “Our ability to predict better what’s going to happen will be greatly improved by real data aggregation.”

There’s also a need to train more people with big data skills and find ways to keep big data scientists interested in working on cancer biology.

“If you say you want to have a big data revolution, you’ve got to have people who do big data,” he said. “People who are good at big data have so much commercial value in Google and Amazon that not enough people are staying in the biological sciences.”

The development of the future biomedical research workforce overall is a focus area for Sharpless, which is also a high priority for the agency as a whole.

Clinical Trial Changes

Sharpless also expects to change models for conducting cancer clinical trials. “It’s unimaginable to me that we won’t be spending a lot of time focusing on how we prosecute clinical trials in the United States,” he said.

A traditional trial model builds on the idea that all patients are the same and would split 1,600 patients into two groups of 800 to test two different drugs, in a costly, slow process that yields incremental results. “Now we realize that’s 1,600 patients who are all different in terms of the molecular biology of the cancers,” he said. “I don’t think you can defend that kind of clinical trial structure any longer.” But oncologists can now conduct smaller, more targeted clinical trials based on the genetic underpinnings of a patient’s cancer.

To contact the reporter on this story: Jeannie Baumann in Washington at

To contact the editor responsible for this story: Randy Kubetin at

For More Information

A video of Bloomberg Law's interview with Sharpless is available at information on Sharpless is at

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