FDA panel backs gene-altering leukemia therapy
Treatment would use patient’s cells to fight disease.
A Food and Drug Administration 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 leukemia, 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, and it is working on similar types of treatments for another type of leukemia, 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.
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.
“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.”
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.
Use of the treatment 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 — the disease does not respond, or it relapses.
Analysts predict that the individualized treatments could cost more than $300,000, but Novartis declined to specify a price.