How China's Legend Biotech and Llama Nanobodies Changed the Course of Multiple Myeloma
核心洞察
CAR-T therapy Carvykti, originating from China's Legend Biotech, has produced durable long-term remissions in heavily pretreated multiple myeloma (搜索) patients, with 33% remaining disease-free at five years.
The therapy uses llama-derived nanobodies targeting BCMA (搜索), offering superior efficacy over traditional antibody-fragment CAR-T approaches, with 100% response rates in early trials versus 80% for competitors.
China's streamlined clinical trial ecosystem enabled Legend to move from first patient dosing in 2016 to stunning data presentation by 2017, outpacing American counterparts.
In 2016, a team of Chinese scientists working in what their company describes as a room the size of a freight elevator began enrolling patients in a clinical trial that would ultimately rewrite the prognosis for one of the most painful blood cancers. The therapy they were testing, now known as Carvykti, has since produced something never before seen in multiple myeloma (搜索): durable, long-term remissions in patients whose disease had become refractory to several other treatments, raising the possibility of a cure.
Multiple myeloma (搜索) originates in the bone marrow, where a single abnormal plasma cell begins proliferating uncontrollably, crowding out healthy blood-forming cells and destroying bone from within. The disease disrupts the equilibrium between osteoclasts and osteoblasts, accelerating bone dismantling while silencing bone rebuilding. The spine is especially vulnerable: vertebrae bear the body's weight and harbor the marrow in which myeloma thrives, producing a persistent ache unrelieved by rest.
The treatment regimen has historically been particularly brutal. Under the newest standard approach, patients endure months of induction therapy built around four drugs: daratumumab, bortezomib, lenalidomide, and dexamethasone. For those fit enough, this is followed by a stem cell transplant involving high-dose chemotherapy that destroys the bone marrow entirely, weeks of isolation with no functioning immune system, and months of recovery. Even after this ordeal, most patients relapse, entering cycles of treatment, remission, and relapse with progressively fewer options.
The science behind CAR-T in myeloma
CAR-T therapies make use of a patient's own immune system. T cells are extracted from blood, shipped to a manufacturing facility, and genetically re-engineered to carry a chimeric antigen receptor (CAR) designed to recognize and bind to a specific protein on cancer cells. The modified cells are cultured into the hundreds of millions, frozen, shipped back, and infused into the patient after a short course of chemotherapy to clear space in the immune system. Because they are living cells, they can persist, proliferate, and continue hunting for months or years.
Multiple myeloma (搜索) is perfectly suited to this approach. Unlike solid tumors that form dense, poorly vascularized masses, myeloma cells circulate through the bone marrow and bloodstream, making them accessible to a therapy that travels through the blood. The second reason lies in B cell maturation antigen, or BCMA (搜索), a protein first identified in 1992 by a French research group led by Yves Laabi. BCMA is expressed at high levels on malignant plasma cells in multiple myeloma and is largely absent from most other tissues, providing the selectivity that targeted cancer therapy demands.
Two therapies, two trajectories
Today, two CAR-T therapies targeting BCMA (搜索) are on the US market: Abecma, approved in 2021, and Carvykti, approved in 2022. Abecma emerged from the National Cancer Institute lab of James Kochenderfer, whose preclinical data in 2013 first showed BCMA-targeted CAR-T cells were effective against myeloma cells. The technology was licensed to Bluebird Bio and developed with Bristol Myers Squibb (搜索).
Carvykti took a different path. Legend Biotech, after focusing solely on BCMA (搜索)-targeting CAR-T cells in 2015, began clinical trials in 2016. Central to their approach was a significant departure from convention: instead of using human antibody fragments, which almost always bind to at least two targets and risk off-target side effects, Legend turned to llamas. Camelid animals produce a unique class of antibodies known as nanobodies, whose compact size and remarkable stability allow CAR-T cells to target tumors more efficiently and stay active longer.
The results were striking. In the American first-in-human trial, 80 percent of patients responded to treatment. In the Chinese trial, every single patient responded, and 74 percent saw their cancer completely wiped out compared to 56 percent in the American equivalent.
Within months of presenting these data at a major conference in 2017, Janssen Pharmaceuticals (搜索), a subsidiary of Johnson & Johnson, negotiated a global licensing and codevelopment agreement with Legend Biotech. The deal, announced in December 2017, gave Legend $350 million upfront plus half of any revenue generated in the US.
Clinical evidence and expanding indications
Later-stage trials confirmed Carvykti's superiority. In the CARTITUDE-1 trial, the 12-month progression-free survival rate was 76 percent, compared to 55 percent for Abecma in the KarMMa trial. More strikingly, while Abecma's progression-free survival curve fell continuously, Carvykti's began to plateau. Extended follow-up at five years confirmed that 33 percent of Carvykti patients remained disease-free — patients for whom, on average, four prior lines of therapy had already failed.
The CARTITUDE-1 results led to Carvykti's approval in 2022 for patients who have failed four other lines of treatment. In 2024, the FDA approved Carvykti for patients who have relapsed after just one prior treatment, based on the CARTITUDE-4 trial, where the therapy showed even greater benefits. The best hypothesis for this improvement relates to T-cell fitness: CAR-T therapies are made from the patient's own T cells, but those cells wear down over years of fighting cancer and enduring chemotherapy. Used earlier, the harvested T cells are in far better shape.
Carvykti is currently being evaluated as a first-line treatment for newly diagnosed myeloma patients. If positive, this would represent a historic shift, with a one-time infusion becoming the standard over the arduous sequence of induction regimen, bone marrow transplant, and lenalidomide maintenance.
China's rise in drug discovery
The Carvykti story was an early signal of a transformation now making headlines: the United States is beginning to lose its dominance in drug discovery to China. Kochenderfer's lab at the National Cancer Institute first validated BCMA (搜索) as a target and ran the first-in-human trial, but the therapy that ultimately reached patients and is rewriting the prognosis for multiple myeloma (搜索) did not emerge from that lineage.
By 2016, the same year Legend began its pivotal clinical trial, China had already overtaken the United States in the number of cell-therapy clinical trials. The gap has been growing since. Roughly half of all major drug licensing deals struck so far in 2026 involve drugs originating from China — a share that was nearly zero just a decade ago.
China's biotechnology transformation is the product of deliberate industrial policy. The Made in China 2025 initiative identified biotechnology and advanced medical technologies as strategic national priorities. The Thousand Talents Plan drew overseas Chinese scientists back from Western institutions. BeiGene, Innovent, and Junshi, now three of China's leading oncology biotechs, were all founded or are now led by researchers who trained in the United States before returning home.
Perhaps the most consequential advantage lies in China's clinical trial ecosystem. Chinese hospitals make extensive use of investigator-initiated trials that can be up and running within roughly six months of a patient consultation. In the United States, the same process can take eighteen months or more, bogged down by regulatory preparation including a lengthy Investigational New Drug application. This speed enables tight feedback between clinic and lab, allowing rapid iteration that cannot be replicated in cell culture or animal models alone.
A proposal in the President's 2027 FDA budget would streamline early-stage trials, recognizing that regulatory friction on early experimentation is a competitive liability. But a budget proposal is not a policy and will remain aspirational unless Congress acts. For now, American and European pharmaceutical companies retain the upper hand in late-stage clinical development, but the pipeline feeding those trials is increasingly Chinese. The question is how long Western companies can sustain that advantage when the discoveries making late-stage development possible are increasingly being made elsewhere.