Two cutting-edge treatments for blood cancers are poised to get approved by the end of the year.
The highly personalized treatment is called CAR T-cell therapy. It’s a type of cancer immunotherapy, or a therapy that harness the body’s immune system to take on cancer cells.
Short for chimeric antigen receptor T-cell therapy, CAR-T treatment takes a person’s own cells, removes them from the body, re-engineers them, and then puts the cells back in the body where they can attack cancer cells. There are a handful of companies developing the therapies, but two in particular are vying to have theirs become the first to ever get approved.
Drug giant Novartis’ first CAR-T bet is CTL019, a treatment for acute lymphoblastic lymphoblastic leukemia. In December, Novartis presented clinical data that showed that the treatment eliminated acute lymphoblastic leukemia, an aggressive blood cancer, in 82% of the patients treated.
The company expected to get a decision from the FDA by October, Bloomberg reports, and the drug will go before the FDA in July for an advisory committee meeting, which could give us a hint on whether or not the therapy will be approved.
The following month, Kite Pharma, the other company in the race, is expected to get an answer from the FDA. That’s for Kite’s CAR-T treatment for aggressive B-cell non-Hodgkin lymphoma (more general than DLBCL). In data Kite released in February, the company found that out of the 101 patients, 36% had a complete response to the treatment after six months.
It’s a type of cancer that Novartis wants to get approval for in the future. On Wednesday, Novartis released data from its Phase 2 trial of CTL019 in patients with diffuse large B-cell lymphoma (DLBCL for short), an aggressive form of lymphoma that’s one of the two types Kite’s data looks at. The trial found that of the 51 patients with DLBCL, 23 had either a complete response (meaning the cancer had disappeared completely) or a partial response (meaning their tumor displayed signs that it was shrinking).
In a release, the trial’s lead investigator said the results were “impressive,” considering the patients had gone through a number of other cancer treatments before taking part in the trial.
But while the results might be promising, there are a few other challenges facing these new treatments:
- The one-time treatments won’t come cheap. While companies might price the drug based on how well it works, the price tag could still be more than $300,000. Manufacturing the drugs is no small feat, considering the personalized treatment requires taking out a person’s cells, reprogramming them, then inserting them again. The company that’s able to do that quickly and safely could have the advantage in the competitive CAR T space. These kinds of treatments have been deadly in the past. In May, Kite Pharma revealed that one person had died in a clinical trial for its late-stage CAR-T therapy from cerebral edema, a condition in which excessive fluid causes the brain to swell. And in July 2016, another CAR-T company, Juno Therapeutics, said four people in its clinical trials had died, all from cerebral edema.