Multiple myeloma is brutal. We may finally have a cure, but American regulatory inertia means that it was discovered abroad.
Multiple myeloma is among the most painful of all cancers. The disease originates in the bone marrow, where a single abnormal plasma cell, one of the blood cells that normally fights against infection, begins to proliferate uncontrollably. Throughout its development, myeloma destroys the bone from within.
Healthy bone is maintained by an exchange between a type of cell called osteoclasts, which dismantle old bone, and another type called osteoblasts, which rebuild it. Myeloma disrupts this equilibrium, by accelerating the action of osteoclasts and silencing that of osteoblasts. Overall, this leads to more bone being destroyed and less being rebuilt. The spine is especially exposed, as its vertebrae harbor the marrow in which myeloma thrives. As they are eroded from within, the result is a persistent, chronic pain. On top of this, as the disease advances, weakened vertebrae may collapse, causing painful fractures.
The treatment of advanced multiple myeloma was transformed in the mid-2010s, by the arrival of a new class of genuinely transformative drugs: immunotherapies. Carvytki, which was first approved by the FDA in 2022 for patients who had returning disease after four or more lines of therapy, belongs to this relatively novel class of drugs. It marks a turning point in the treatment of multiple myeloma for two reasons. First, unlike the conventional approach, in which patients endure continuous cycles of treatment, remission, and relapse for the rest of their lives, it is administered as a single, one-time infusion. Second, it is producing something quite unprecedented in this disease: durable, long-term remissions in patients which had been refractory to several other treatments, raising the possibility of a cure.
But Carvykti matters beyond multiple myeloma. In retrospect, its development story, which began in 2016, was an early signal of a transformation that is only now, a decade later, making headlines: the United States is beginning to lose its dominance in drug discovery to China.
Although much of the science underpinning Carvykti was American, the actual therapy that has changed the field came from a Chinese biotechnology company. One important difference that has allowed China to pull ahead in biotech is its ability to test scientific hypotheses in clinical trials. If the US does not address the clinical trial bottlenecks that have put it at a disadvantage compared to China, more breakthroughs like Carvykti will be developed elsewhere, with potential negative consequences for the entirety of the American biopharmaceutical ecosystem.
The brutality of myeloma treatment
The treatment regimen for multiple myeloma is harsh and begins with an induction regime composed of four drugs: daratumumab, an antibody that directs immune cells to attack myeloma cells; bortezomib, a proteasome inhibitor injected under the skin; lenalidomide, an immunomodulatory drug taken as a pill; and dexamethasone, a steroid.
For patients that can withstand it, this induction regimen is followed by a stem cell transplant. This begins with high-dose chemotherapy that destroys the bone marrow entirely, followed by weeks of isolation with no functioning immune system. Recovery can take months after discharge. This stage is accompanied by repeated infections, extreme fatigue and mucositis, a painful inflammation of the digestive tract lining from mouth to gut. Patients are also profoundly vulnerable to even ordinarily minor pathogens that would pose no risk in a healthy person.
What is worse is that even after this brutal treatment most patients relapse, meaning their cancer returns. For these patients, what follows is further cycles of treatment. Doctors start patients on new combinations of drugs, including proteasome inhibitors and more chemotherapy. While these combinations can lead to remission, the myeloma almost always relapses again, leaving patients with progressively fewer options. Each treatment cycle means a lower possibility of survival.
My other car is a T cell
CAR-T therapies make clever use of a patient’s own immune system. They are made by extracting T cells, a type of immune cell that can attack tumours, from a patient's blood. Then, they are genetically re-engineered to carry a new receptor on their surface, also known as a chimeric antigen receptor, which is designed to recognize and bind to a specific protein on cancer cells.
Multiple myeloma is perfectly suited for CAR-T therapies for two main reasons. First, because it is a blood cancer. Solid tumors, a class that most cancers belong to, usually form dense, poorly vascularized masses that T cells struggle to penetrate. This greatly limits the therapeutic potential of these therapies. By contrast, blood cancers like multiple myeloma do not suffer from this problem. Myeloma cells circulate through the bone marrow and bloodstream, meaning that they are easily accessible to therapeutic CAR-Ts.
The second reason relates to the presence of a protein called BCMA. BCMA was first identified in 1992 by a French research group led by Yves Laabi, a part of a set of genes preferentially expressed in mature B cells. For most of the decade following its discovery, BCMA sat in relative scientific obscurity: an interesting receptor without an obvious clinical application. But researchers gradually began to understand that BCMA was expressed at high levels on the malignant plasma cells in multiple myeloma and that it was largely absent from most other tissues in the body. This meant it could be harnessed to selectively target CAR-Ts to the right target cells.
In 2013, preclinical data from the lab of James Kochenfelder at the National Cancer Institute showed that BCMA-targeted CAR-T cells were effective against myeloma cells in the laboratory. But what followed illustrates the enormous and often underappreciated gap between a promising laboratory finding and a treatment that reaches patients.
The importance of being llama
Today, there are two CAR-T cell therapies for multiple myeloma in the US market: Abecma, approved in 2021, and Carvykti, approved in 2022. Both target BCMA on the surface of myeloma cells. Yet they arrived by strikingly different routes: Abecma from American research institutions and corporate laboratories in the early 2010s; Carvykti from an early-stage biotechnology company and a clinical trial first conducted in China in 2016. Only later was it licensed to a Western company and eventually brought to global approval.
Of the two, Carvykti is the clear winner. Its story speaks to the outsized role that China's 2015 regulatory reforms have played in accelerating the country's path from laboratory to clinic, and to producing medicines that work. Being first to market or doing the fundamental science counts for far less than being the nimblest in getting to the clinic. Carvykti also carries another important lesson for the industry: never underestimate the llama.
The technology developed in Kochenfelder lab was licensed to the biotech start-up Bluebird Bio, which developed it in partnership with the large biopharmaceutical company Bristol Myers Squibb into what would eventually become Abecma, the first CAR-T therapy approved for multiple myeloma. Over 80 percent of patients saw their cancers shrink in the National Cancer Institute’s first-in-human trial, published in 2016. This validated the premise of BCMA targeting and set the field moving.
Meanwhile, a parallel story was unfolding on the other side of the world. In 2014, a team of Chinese scientists began investigating cell therapies for cancer, initially working in what the company describes as a room the size of a freight elevator. After focusing their research solely on BCMA-targeting CAR-T cells in 2015, Legend Biotech began conducting its first clinical trials in 2016.
Central to their approach was a significant departure from convention. Traditional CAR-T constructs relied on using existing antibody fragments, derived from humans, to seek out and bind to their target protein; in this case, BCMA. Legend took a different path, turning to an unlikely source: the llama. Human antibody fragments almost always bind to at least two targets, meaning that if doctors give a patient too much of the drug, they risk causing side effects associated with the second protein the drug binds to.
Camelid animals, including llamas and alpacas, produce a unique class of antibodies known as nanobodies, which can be engineered to do the work of their much larger human counterparts. Their compact size and remarkable stability allow CAR-T cells armed with them to target tumors more efficiently. CAR-T cells using nanobodies also seem to stay active longer and kill tumors more effectively.
The Chinese researchers enrolled their first patient in a clinical trial in 2016, the same year the American team published their initial results. But they moved fast. By 2017, just a year later, they were presenting stunning data at one of the biggest conferences in the field. The decision to learn from the llamas seemed to have massively paid off.
Xi loves you (yeah, yeah, yeah)
It can take years for enough people in a cancer trial to die to clarify that one treatment is prolonging survival over another, so researchers rely on proxies instead. In the American trial, 80 percent of patients responded to treatment, meaning their tumors shrank to some measurable degree, a result already considered impressive given how much these patients had already been treated, to no avail. The Chinese trial did better: every single patient responded. What’s more, 74 percent of patients in the Chinese trial saw their cancer completely wiped out, compared to 56 percent in the American equivalent.
Such results did not go unnoticed. Within months of the 2017 presentation, Janssen Pharmaceuticals, the pharmaceutical subsidiary of the large American company Johnson & Johnson, was in negotiations with Legend Biotech. In December 2017, the two companies announced a global licensing and codevelopment agreement: Legend got $350 million upfront plus half of any revenue generated in the US, while also contributing to half of the development costs.
After years of clinical development, later-stage trial results confirmed what earlier data had suggested: Carvykti was the superior treatment. In later stage trials, oncologists and regulators switch from looking at how many patients respond at all to looking at progression-free survival, or the length of time a patient lives without their disease worsening or claiming their life. The gold standard is overall survival, but it is also the hardest to measure, requiring trials long enough to capture the full arc of a patient's outcome. For that reason, regulators often grant approval on the basis of progression-free survival data, with overall survival figures following later as evidence accumulates.
On both measures, Carvykti pulled clearly ahead. In the CARTITUDE-1 trial, its 12-month progression-free survival rate was 76 percent. In the KarMMa trial for Abecma, by contrast, this figure was 55 percent. But what happened afterwards is perhaps even more striking: in the Abecma progression free survival curve, the line falls continuously. By contrast, in Carvykti, the line starts to plateau. Extended follow-up at five years confirmed that 33 percent of Carvykti patients remained disease-free. The significance of this result cannot be overstated. These were patients for whom, on average, four prior lines of therapy had already failed and whose immune systems were heavily challenged.
The CARTITUDE-1 trial results led to Carvytki’s approval in 2022 for patients with myeloma who have failed four other lines of treatment. In 2024 the FDA approved Carvytki in patients who have relapsed after just one prior treatment, on the basis of results from the CARTITUDE-4 trial. Here, Carvykti shows even greater benefits over standard care. The best hypothesis as to why has to do with the fitness level of the patients’ T cells. CAR-T therapies are made from the patient's own T cells, but those cells wear down over years of fighting cancer and enduring multiple rounds of chemotherapy. Used earlier, after just one prior treatment, the harvested T cells are in far better shape, and the therapy built from them is more effective at fighting disease.
Carvykti is currently being evaluated in clinical trials as a first-line treatment, which means that it would be given to newly diagnosed myeloma patients before any other therapy has been tried. If the trial results are positive, this would be a historic game-changer in how patients are treated, with a one-time injection becoming the standard over the arduous procedure of induction regimen, bone marrow transplant and lenalidomide maintenance.
Made in China
Just last week, in late May 2026, the New York Times warned that the US was losing ground to China when it came to the discovery of novel drugs: roughly half of the dollar value of biopharmaceutical licensing deals now goes to Chinese biotechnology companies. What is striking is that this share was nearly zero just a decade ago.
The Carvykti case should have been an early warning. Already by 2016, the same year Legend began the clinical trial that would first reveal Carvykti’s potential, China had overtaken the United States in the number of cell-therapy clinical trials. The gap has been growing since, mirroring the equally dramatic change in licensing value.
China’s ongoing biotechnology transformation is the product of deliberate industrial policy. The Made in China 2025 initiative explicitly identified biotechnology and advanced medical technologies as strategic national priorities, and a series of targeted policies followed. Such policies include the Thousand Talents Plan, designed to draw overseas Chinese scientists back from Western institutions. BeiGene, Innovent, and Junshi, which are now three of China's leading oncology biotechs, were all founded or are now led by researchers who had trained in the United States before returning home.
Yet perhaps the most consequential advantage China has built lies in its clinical trial ecosystem. Chinese hospitals make extensive use of a type of trial known as investigator-initiated trials (IITs). These are early-stage studies that allow oncologists and other doctors to quickly assess whether a drug shows genuine promise and iterate based on the findings. In China, such a trial can start within roughly six months after a promising drug is designed. In the United States, the same process can take eighteen months or more, in large part due to regulatory requirements that the FDA itself has recently admitted to being excessive for this stage.
The most valuable thing early-stage trials enable is iteration. They allow tight feedback between the clinic and the lab. There are countless ways to engineer a better CAR-T cell, and many cannot be evaluated in the laboratory alone. No cell culture or animal model fully replicates the complexity of a human tumor, and AI is unlikely to close that gap anytime soon. We simply lack the training data to capture what tumors are actually like in vivo: their geometry, vascularization and biomechanical properties.
The policy world has, belatedly, taken notice of the structural advantage that China’s regulatory environment offers to its biotechnology industry. A proposal in the President's 2027 FDA budget would streamline early-stage trials. This a welcome recognition 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 largely retain the upper hand in the later stages of clinical development, as shown by the fact that Legend ultimately licensed Carvytki to a large American biopharmaceutical company to get it approved. But the pipeline that feeds those late-stage trials is increasingly Chinese. Such early-stage dominance turned into vertical integration of the entire chain in solar panels, batteries and electric vehicles, and LCD panels. The question is how long Western companies can sustain their advantage at the later stages, when the discoveries that make those stages possible are increasingly being made elsewhere.
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