Stanford cell therapy technologist Thomas Orozco thaws the treated immune cells from a patient with advanced melanoma. The cells are collected from the patient’s tumor and fortified in a laboratory to better fight the cancer. Photo provided by Stanford Health Care
The promise of cell and gene therapies has intrigued scientists for decades, but progress was slow, with many setbacks. Now the long research journey has finally paid off, with FDA-approved products entering the clinic. “I’m very happy that it’s here now. … I’ve been walking the tightrope and I didn’t fall off,” said a Stanford patient, who asked not to be identified.
Why? Word hasn’t yet gotten out, so sick people aren’t getting referred from their community physicians, said Miklos. Treatment is risky. Or patients may live far away from the nation’s estimated 30 “centers of excellence,” like Stanford and UCSF, and are daunted by the cost of travel and housing. The total cost for gene therapies over the next decade has been estimated to reach an eye-popping $35 billion to $40 billion. The bill for future cell therapies, which could help a bigger pool of patients, will likely be higher.