A Food and Drug Administration (FDA) panel opened a new era in medicine on Wednesday, unanimously recommending that the agency approve the first treatment that genetically alters a patient’s own cells to fight cancer, transforming them into what scientists call “a living drug” that powerfully bolsters the immune system to shut down the disease.
If the FDA accepts the recommendation, which is likely, the treatment will be the first gene therapy to reach the market. Others are expected: Researchers and drug companies have been engaged in intense competition for decades to reach this milestone.
Novartis is now poised to be the first. Its treatment is for a type of leukemia, and it is working on similar types of treatments in hundreds of patients for another form of the disease, as well as multiple myeloma and an aggressive brain tumor.
To use the technique, a separate treatment must be created for each patient—their cells removed at an approved medical center, frozen, shipped to a Novartis plant for thawing and processing, frozen again and shipped back to the treatment center.
A single dose of the resulting product has brought long remissions, and possibly cures, to scores of patients in studies who faced death because every other treatment had failed. The panel recommended approving the treatment for B-cell acute lymphoblastic leukemia that has resisted treatment, or relapsed, in children and young adults aged 3 to 25.
One of those patients, Emily Whitehead, now 12 and the first child given the altered cells, was at the meeting of the panel with her parents to advocate approval of the drug that saved her life. In 2012 as a 6 year old, she was treated in a study at the Children’s Hospital of Philadelphia. Severe side effects—raging fever, crashing blood pressure, lung congestion—nearly killed her. But she emerged cancer-free, and has remained so.
“We believe that when this treatment is approved, it will save thousands of children’s lives around the world,” Emily’s father, Tom Whitehead, told the panel. “I hope that someday all of you on the advisory committee can tell your families for generations that you were part of the process that ended the use of toxic treatments, like chemotherapy and radiation, as standard treatment, and turned blood cancers into a treatable disease that, even after relapse, most people survive.”
The main evidence that Novartis presented to the FDA came from a study of 63 patients who received the treatment from April 2015 to August 2016. Fifty-two of them, or 82.5 percent, went into remission—a high rate for such a severe disease. Eleven others died.
“It’s a new world, an exciting therapy,” said Dr. Gwen Nichols, the chief medical officer of the Leukemia and Lymphoma Society, which paid for some of the research that led to the treatment.
The next step, she said, will be to determine “what we can combine it with, and is there a way to use it in the future to treat patients with less disease, so that the immune system is in better shape and really able to fight.” She added, “This is the beginning of something big.”
At the meeting, the panel of experts did not question the lifesaving potential of the treatment in hopeless cases. But they raised concerns about potentially life-threatening side effects—short-term worries about acute reactions, like those Emily experienced, and longer-term worries about whether the infused cells could, years later, cause secondary cancers or other problems.
Oncologists have learned how to treat the acute reactions and, so far, no long-term problems have been detected, but not enough time has passed to rule them out.
Patients who receive the treatment will be entered in a registry and tracked for 15 years.
Treatments involving live cells, known as “biologics” are generally far more difficult to manufacture than standard drugs, and the panelists also expressed concerns about whether Novartis would be able to produce consistent treatments and maintain quality control as it scaled up its operation.
Another parent at the meeting, Don McMahon, described his son Connor’s grueling 12 years with severe and relapsing leukemia, which started when he was 3. McMahon displayed painful photographs of Connor, bald and intubated during treatment. And he added chemotherapy had left his son infertile.
A year ago, the family was preparing for a bone-marrow transplant when they learned about the cell treatment, which Connor then underwent at Duke University. He has since returned to playing hockey. Compared with standard treatment, which required dozens of spinal taps and painful bone-marrow tests, the T-cell treatment was far easier to tolerate, McMahon said, and he urged the panel to vote for approval.
The treatment was developed by researchers at the University of Pennsylvania and licensed to Novartis. Use will not be widespread at first, because the disease is not common. It affects only 5,000 people a year, about 60 percent of them children and young adults. Most children are cured with standard treatments, but in 15 percent of the cases—like Emily’s and Connor’s—the disease does not respond, or it relapses.
Analysts predict that these individualized treatments could cost more than $300,000, but a spokesman for Novartis, Julie Masow, declined to specify a price.
Although the figure may seem high, people with cancer often undergo years of expensive treatment and repeat hospital stays that can ultimately cost even more.
Because the treatment is complex and patients need expert care to manage the side effects, Novartis will initially limit its use to 30 or 35 medical centers where staff will be trained and approved to administer it, the company said.