It’s the ultimate example of personalized medicine — for some cancer patients who have run out of options, scientists can extract their immune cells and genetically re-engineer them to fight against the tumors.
But there are only two companies in the U.S. approved to make these medicines, known as CAR-T therapies, and it costs both money and time — the therapies list for $373,000 and $475,000 and take two to three weeks to produce for each patient.
So researchers around the globe are toiling away on a so-called off-the-shelf CAR-T, which could be produced en masse, kept on reserve in a hospital, and given to patients with little delay. It could be cheaper and potentially even usable in patients whose immune cells aren’t otherwise healthy enough for current bespoke versions of CAR-T.
“It’s so much easier and cheaper if you could have a single manufacturing step and just send out the cells [to] whoever needs them, whenever they need them,” said Waseem Qasim, a professor of cell and gene therapy at Great Ormond Street Hospital in London.
Already, some patients have gotten an off-the-shelf therapy, though only in early-stage trials or through experimental procedures. It’s not at all clear yet if the therapies are as effective as their bespoke counterparts, and commercially available off-the-shelf therapies are years away, scientists said. And even that’s only if researchers can surmount a fundamental scientific challenge: They need to make sure the CAR-T therapy doesn’t attack the patient herself — and that the patient’s immune system doesn’t disable the therapy.
“Because they’re third-party T cells, they have the potential to react against a patient’s normal tissue,” said Dr. David Porter, who teaches at the University of Pennsylvania and helped develop Kymriah, the CAR-T therapy sold by Novartis for a certain type of lymphoma.
