Ruxandra Teslo 🧬@RuxandraTeslo
China is winning the drug discovery race. There's no better example of this than multiple myeloma.
worksinprogress.co/issue/the-bloo…
It's one of the most painful cancers, destroying bone from within. For decades, patients endured cycles of brutal treatment and relapse. Then came Carvytki: a one-time CAR-T infusion that appears to cure some patients who have failed multiple treatments.
Its development story, beginning in 2016, was an early signal of a shift now making headlines: the US is losing biotech dominance to China. Though the foundational science was largely American, a nimble Chinese company moved faster with a better molecular engineering idea.
Unless the US addresses clinical-trial bottlenecks slowing early in-human data, more breakthroughs will be developed elsewhere, weakening the ecosystem American biopharma depends on.
Some key points from my article for @WorksInProgMag, with my friend Amol Punjabi, of @EvidenceOpen:
1) Multiple myeloma is not only extremely painful in and of itself, but also one of the most brutal cancers to treat. As first-line therapy, patients endure four drugs simultaneously, then a stem cell transplant, followed by continuous maintenance therapy. And most still relapse, with each treatment round carrying worse chances.
2) A drug called Carvykti, approved in 2022, is changing the treatment landscape. Carvytki acts as a single, one-time infusion. It's a CAR-T therapy, part of a new wave of transformative immunotherapies: made from the patient's own immune cells and reprogrammed to hunt cancer. In patients who had already failed 4+ other treatments, 33% were still disease-free after 5 years. The results as earlier line therapy look even more promising.
3) Most of the foundational science was American. Decades of CAR-T research, and in 2013 the NCI showed BCMA-targeted CAR-T cells could kill myeloma in the lab.
4) But the drug that ultimately changed myeloma, Carvytki, originates from China. Carvytki beats Abecma (the American CAR-T for myeloma) by a wide margin: 36 months of progression free survival in heavily pre-treated patients versus Abecma's 9 months.
5) In 2016, Legend Biotech was just beginning clinical trials. This was the same year the American team was publishing their first-in-human results. Legend started later, but moved faster. Clever engineering and China's ability to get drugs into humans quickly gave them the edge. Large American biopharma J&J ended up striking a deal with Legend and developing the therapy.
6) Never underestimate the llama: US-developed Abecma used mouse antibody fragments to target BCMA. Chinese startup Legend used llama nanobodies instead. These are smaller, more stable and bind more cleanly to BCMA. The usage of llama as opposed to mice antibodies is what is believed to lead to Carvytki's superior efficacy.
7) In retrospect, Carvytki should have been an early warning. China is winning the drug discovery race through deliberate policy. Their first-in-human clinical trials can launch in 6 months vs 18+ months in the US, letting them iterate faster between lab and clinic. The @nytimes recently reported that ~50 percent of major drug deals this year involve Chinese-origin drugs, up from nearly zero a decade ago.
8) The US still leads in late-stage development, as shown, but the pipeline feeding it is increasingly Chinese. The worry is that this will mirror what happened in solar, batteries, and EVs, where early-stage dominance eventually became control of the entire chain.
9) A proposal to streamline early stage trial regulatory requirements to keep the US competitive has made it into the President's 2027 budget for the FDA. But Congress has to act to make it a reality.
