Cell therapy grabbed quite a few headlines this month – starting, of course, with Celgene’s blockbuster deal to buy Juno Therapeutics and its CAR-T pipeline for about $9 billion.
The market was already bullish on CAR-T, as evidenced by Gilead Science’s $11 billion purchase of Kite Pharma last year. The new deal reinforces the industry’s enthusiasm for this therapeutic approach, which involves re-wiring a patient’s T cells to attack tumors.
Celgene expects Juno’s lead CAR-T candidate, a treatment for patients with aggressive lymphoma, to generate peak sales of $3 billion a year.
But amid the hype, it’s worth remembering that CAR-T technology still has a long way to go. The approach has demonstrated success in some blood cancers (albeit in early phase trials with small numbers of patients), but has not yet been shown to work in solid tumors. The treatments also carry considerable risk of acute, severe side effects and any potential long-term adverse effects are unknown.
Elsewhere in CAR-T news, Gilead announced this month that it will team up with Pfizer on a clinical trial to study a new combination therapy in patients with refractory large B-cell lymphoma. The trial will pair Gilead’s recently approved CAR-T, sold as Yescarta, with an experimental drug from Pfizer called utomilumab.
And while we’re on the subject: The first CAR-T to hit the market in the U.S. – Novartis’ Kymriah – may soon be marketed in new indications. Kymriah was approved last August for pediatric and young adult patients with a form of acute lymphoblastic leukemia. This month, both the European Medicines Agency and the FDA granted priority reviews for Kymriah’s use in adult patients with relapsed or refractory diffuse large B-cell lymphoma who are ineligible for autologous stem cell transplant.
Our final item in this month’s news roundup comes from the Wall Street Journal, which reports that China is racing to test the gene-editing tool CRISPR in desperately ill patients.
The first human CRISPR trial in the U.S. is still working through a rigorous review process that has taken nearly two years so far. In contrast, a hospital review board in China took just a few hours to approve the use of CRISPR in patients with esophageal cancer. The physician leading that study, Dr. Wu Shixiu, told the WSJ that he recognized an irony in China taking the lead on clinical use of a tool that was developed in the U.S.
“China shouldn’t have been the first one to do it,” he said. “But there are fewer restrictions.”
That lax oversight worries some scientists and bioethicists, who fear the rush to begin research studies in China could jeopardize patients. Looser rules could also make it difficult to fully assess the benefits and drawbacks of using CRISPR.
Dr. Carl June, a leading CRISPR researcher who has been working to get approval for a U.S. trial, told the WSJ that regulatory oversight is important – but that the U.S. is in danger of “losing our lead in biomedicine” to China. “It’s hard to know,” he said, “what the ideal is between moving quickly and making sure patients are safe.”