Cell therapy

Eli Lilly in April continued a buyout streak involving the makers of “in vivo” cell therapies when it agreed to pay as much as $7 billion to acquire privately held biotechnology company Kelonia Therapeutics.

Lilly paid $3.25 billion up front for Kelonia and could eventually hand the startup’s shareholders another $3.75 billion if certain unspecified clinical, regulatory and sales milestones are met, it said at the time.

For Lilly, the acquisition furthers a push into what’s become a fast-advancing field of drug research. The cell therapies currently on the market involve “ex vivo” procedures, in which patient cells are extracted, genetically modified in a lab and then reinfused. Newer “in vivo” approaches are designed to alter immune cells without this complex and burdensome process, a workaround that could significantly broaden cell therapy’s reach.

In vivo cell therapy research remains early, with many programs currently only in preclinical or the first stage of human testing. Nonetheless, the concept’s potential has captured the attention of many large drugmakers. At least five have acquired biotech startups working on the technology over the last year or so. Lilly has bought two developers since February: Orna Therapeutics and now Kelonia.

Worth up to $2.4 billion, the Orna deal gave Lilly a preclinical program that’s shown promise in autoimmune disease, as well as a technology for manipulating cells “in vivo.” The Kelonia buyout hands Lilly another way of making these therapies; specially engineered viral particles that can get into cells and teach them how to make a new cancer-fighting receptor.

Kelonia’s most significant attempt at this approach is “KLN-1010,” a treatment in early-stage testing for multiple myeloma. The therapy is designed to help the body generate immune cells that target BCMA, a protein universally found on malignant cells.

According to Lilly, early data presented at a medical meeting last year provided “initial clinical validation” and “promising tolerability.” If successful in further testing, KLN-1010 would represent a “transformative advance” against the disease by “eliminating the complexities” of marketed cell therapies, Lilly added.

Available cell therapies “have meaningfully improved outcomes for patients with various cancers, but significant manufacturing, safety, and access barriers mean that only a fraction of eligible patients actually receive them,” said Jacob Van Naarden, head of Lilly’s oncology division as well as corporate business development, in the statement. “Kelonia's in vivo platform has the potential to change that by delivering rapid, durable responses in a far simpler, off-the-shelf format.”

While the upfront payment “appears full, we believe it is justified given the strength of the clinical data and the competitive dynamics in the space,” wrote RBC Capital Markets analyst Trung Huynh in a note to clients. AbbVie and Bristol Myers Squibb paid $2.1 billion and $1.5 billion, respectively, for companies that didn't have "comparable efficacy data" when they were acquired, he added.

The buyout "positions [Lilly] at the forefront of a potentially transformative technology with broad platform applicability beyond multiple myeloma," Huynh wrote.

An experimental cancer cell therapy developed by Legend Biotech helped reduce or eliminate signs of disease in people with lymphoma enrolled in a clinical trial, sparking hopes the “in vivo” medicine might compete with personalized, marketed treatments like Novartis’ Kymriah.

At the higher of the two dose levels tested so far, all six people treated with the therapy, LB2501, responded. Five had no evidence of lymphoma lesions, according to trial details released ahead of a medical meeting in June.

Legend is one of many companies seeking to develop therapies that fight cancer by reprogramming immune cells inside the body. If successful, these therapies would sidestep an extensive, “ex vivo” process that involves extracting cells and modifying them in a lab.

Multiple Wall Street analysts praised the data, with RBC Capital Markets’ Leonid Timashev noting that a successful “in vivo” product could record blockbuster sales while “ex vivo” counterparts face “logistical bottlenecks” that limit their commercial potential.

Legend is already a prominent cell therapy player, having successfully developed the personalized multiple myeloma treatment Carvykti that it now sells with Johnson & Johnson. It’s now turning to “in vivo” work, an area that’s seen an explosion in venture investments as well as a string of recent drugmaker acquisitions.

With LB2501, Legend uses a modified virus to deliver into immune cells instructions to find and attack cells expressing the proteins CD19 and CD20. Those proteins are found on the surface of malignant B cells and are known targets in lymphoma. Importantly, the treatment doesn’t require a chemotherapy step to prepare the body for treatment, as other cell therapies do. 

The data reported at the European Hematology Association meeting in June came from a first-in-human trial conducted in China that’s testing LB2501 in people whose disease had progressed after at least two lines of treatment. An abstract posted ahead of that meeting involves results in 12 patients with large B cell lymphoma, follicular lymphoma or mantle cell lymphoma who received one of two tested doses.

No responses were observed at the lower dose, even though there was evidence of activity in five of the six recipients. At the higher dose, though, remissions — defined as a reduction or elimination of lymphoma lesions — were registered in all six recipients. Five had “complete” responses, Legend said.

Eight of the enrollees had an immune response called cytokine release syndrome that is often seen with cell therapies, but only one needed an intervention beyond basic symptomatic treatments for fever, pain or nausea. No patients had an immune response that affected the brain or nerves, another side effect associated with ex vivo cell therapies.

In his note, Timashev wrote further data, such as a six-month follow-up, will be necessary to see how Legend’s therapy stacks up against others, such as an experimental treatment from Lyell Immunopharma. But the news is nonetheless “exciting both for their clinical value of the product, and strategically, because it puts forth a credible second act for [Legend] beyond Carvykti.”

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By PHC Corporation of North America

Cell & gene therapy stands at the forefront of modern medicine, promising transformative treatments for previously intractable diseases. But, as the field continues to advance, a growing body of evidence suggests that we may need to fundamentally rethink one of the most basic aspects of cell culture:  O2 concentration.

While the field has made remarkable strides in optimizing numerous culture parameters, the continued use of non-physiological O2 levels could be stifling our efforts to conduct more insightful research and develop better, more effective therapies.

Normoxia — the norm for cells?

Conventionally, mammalian cells have been cultured at atmospheric or ‘normoxic’ O2 levels — that is, at O2 levels ranging from 20–21%. However, such conditions represent a significant departure from the true environment that cells experience in vivo. In fact, in most living tissues, cells experience O2 concentrations far below atmospheric levels, typically between 2% and 11%, depending on the tissue type. Stem cell niches, for instance, are generally accepted to reside in environments ranging from 3–6% O2 concentration. Some tissues — such as those in the thymus, those in areas of the kidney, and pathological tissues — contain O2 gradients where intercellular O2 levels can even dip below 1%.

A damaging discrepancy

As well as being stark, these differences in O2 levels are hugely consequential; since physiological O2 levels play crucial roles in cellular development, function, and behavior, deviating from them can lead to unpredictable and unwanted cell culture outcomes.

Research has demonstrated, for instance, that cells cultured under normoxic conditions often exhibit altered metabolic profiles, modified gene expression patterns, and different proliferation rates compared to their counterparts grown under physiological O2 levels. Long-term culture of stem cells at atmospheric O2 levels, for example, gives rise to chromosomal abnormalities as well as genomic and epigenomic aberrations, which could make reproducible cell therapy production challenging, and could even compromise the safety of the final cell therapy product (for example, by leading to abnormal tissue formation after implantation).

On the other hand, culturing cells under physiological, hypoxic conditions can offer therapeutic benefits. Indeed, hypoxic cell culture has been shown to improve growth, genetic stability, and therapeutic potential of mesenchymal stem cell (MSC)-based therapies.

Culturing therapeutic cells in more physiologically relevant O2 concentrations can give better early insights into their potential therapeutic efficacy, too. For example, research has shown that, in hypoxic conditions replicating those observed in the solid tumor microenvironment, CAR T-cells display impaired expansion, differentiation, and cytokine production, which could negatively impact their ability to eradicate solid tumors. Such studies could offer valuable insights into where and how such therapies can be optimized to produce the next generation of more effective cancer-targeting medicines.

Taking control of O2 — ushering in the future of cell therapy research

With the importance of O2 concentration becoming increasingly clear in a range of applications — including cell therapy research — researchers are understandably looking to incorporate O2 control into their cell culturing experiments. Now, thanks to advances in CO2 incubator design and function, this is possible.

As well as offering careful control of CO2 levels through advanced dual wavelength infrared CO2 sensors, these modern incubator systems can, when desired, also precisely regulate O2 concentration through the introduction of nitrogen and with the help of advanced zirconia sensors — all without the need for additional costly and space-consuming lab equipment.

With the ability to precisely control O2 concentration, researchers can better replicate physiological conditions in their cell culture, which could help drive more insightful, reproducible research, and potentially drive better and safer cell therapies for improved healthcare outcomes.

The most effective drug for Parkinson’s disease hasn’t changed in 50 years. But fresh research published in one of the country’s top scientific journals is helping build the case for a more cutting-edge approach that uses stem cells to restore important brain functions.

The second most common neurodegenerative illness, Parkinson’s is caused by the loss of certain nerve cells. These cells produce a chemical messenger, dopamine, that plays a crucial role regulating movement. Since the 1970s, a drug called levodopa, which the body converts into dopamine, has been the mainstay treatment for combating the tremors, slowness, stiffness and balance issues that come with Parkinson’s.

Levodopa doesn’t stop the disease, however, so scientists have spent decades trying to find a more permanent fix. The latest development in that search comes from two clinical trials testing stem cell-derived therapies transplanted into the brains of Parkinson’s patients.

The first of those trials, conducted at the Kyoto University Hospital in Japan, evaluated seven people who received what are essentially blank slate cells that can turn into dopamine-producing neurons. These cells were created using a technology pioneered at Kyoto University, where roughly 20 years ago, scientists discovered how to reset some adult human cells to act like stem cells.

The study’s main focus was safety. Results, published in April in the journal Nature, show no serious adverse events were reported, though researchers did identify 73 mild to moderate events. Importantly, there was no evidence the introduced cells started growing in an unchecked, tumor-like fashion — a major concern with stem cell implants.

The study also explored whether this therapy had any effect on patients’ disease. Two years post-treatment, the six participants who were evaluable had gotten better on a scale clinicians use to measure the severity of Parkinson’s motor symptoms. Researchers looked at what’s known as “on” time, when medications are adequately controlling these symptoms, as well as the opposite “off” time, and found scores improved an average of 36% and 20%, respectively.

Another test indicated that dopamine-producing cell activity had increased substantially. This was determined through a technique in which researchers strap a radioactive element to a molecule of levodopa and then use a PET scan to follow its path through the nervous system, somewhat like how a submarine tracks objects on a sonar display.

The second trial took place at sites throughout the U.S. and Canada, and employed a different kind of stem cell that comes from human embryos. It enrolled a total of 12 participants and followed them for a year post-transplant.

Similar to the Japan study, there were no deaths, serious adverse events or tumor-like tissue growth related to the introduced cells. Trial runners did report two serious events overall. One participant was hospitalized with COVID-19, while another had a seizure that was attributed to the surgical procedure.

The trial also saw improvements in “off” scores on that disease severity scale and positive results from the radioactive levodopa test.

The cell therapy tested, called bemdaneprocel, is being developed by Bayer subsidiary BlueRock Therapeutics, which back in August 2023 announced some of the trial data. The company expects to start a Phase 3 experiment before the end of June.

Hideyuki Okano, a stem-cell scientist at Keio University in Tokyo, argues more research is needed to confirm these types of cell therapies are effective against Parkinson’s.

Still, in an accompanying Nature editorial, he called the new trial results “encouraging” because they suggest treating Parkinson’s patients with donor cell transplants is likely safe. That both trials “proved to be safe, and hinted at possible efficacy, is an important step towards the establishment of this cell therapy for Parkinson’s disease in wider society,” he wrote.

Cell therapy for Parkinson’s isn’t a new idea. In 1989, a team led by neuroscientist Olle Lindvall performed the first cell transplantations for Parkinson’s patients. While the landmark study didn’t show a major therapeutic benefit, a few interesting signals encouraged the research community to keep exploring.

One ethical concern from that trial was that it used a relatively large amount of tissue derived from aborted fetuses. In the decades since, Parkinson’s and cell biology experts have made significant progress identifying other, less controversial sources of stem cells that can be scaled more easily.

A milestone of that work came in 2020, when the technique invented at Kyoto University was used to turn a Parkinson’s patient’s own skin cells into the early stages of dopamine-producing neurons, which were then implanted into his brain.

According to Okano, as of December, there were 115 clinical trials testing 83 products derived from “pluripotent” stem cells. And among those targeting central nervous system disorders, the ones for Parkinson’s disease are at a more advanced stage of development.

Further behind Bayer and BlueRock, Aspen Neuroscience, a San Diego-based biotechnology company, has a Parkinson’s therapy derived from patients’ own cells currently in early-stage human testing.

An experimental therapy from Allogene helped eliminate signs of cancer better than standard treatment in a Phase 3 trial in first-line large B-cell lymphoma, results suggesting the biotechnology company may have found a role to use donor-derived cell therapy against the deadly blood cancer.  

After 45 days of treatment, seven of the 12 patients given Allogene’s therapy in the study were negative for “minimal residual disease,” meaning that diagnostic tests could no longer detect signs of cancer. By comparison, only 2 of 12 placebo recipients hit that mark, a roughly 42-percentage-point difference that clears an important bar published literature has suggested is crucial for delaying a relapse. 

The results came from an early “futility” analysis Allogene disclosed in April. Allogene is enrolling 220 people in the study and expects to report in 2027 results showing whether treatment staved off cancer’s return. 

Allogene spun out of Pfizer’s cell therapy work in 2018 with plans to prove that donor-derived, or “allogeneic” cell therapies could prove a more convenient alternative to their personalized CAR-T counterparts. But like many of its peers, Allogene suffered several setbacks along the way. It’s lost most of its market value since going public. 

In 2024, though, Allogene came up with a new way to show allogeneic treatments might have an important role to play in lymphoma. Rather than position cema-cel in settings where CAR-T therapies like Breyanzi and Yescarta are available, it set its sights earlier, testing the treatment in people who are at risk of relapse after receiving a widely used drug regimen known as R-CHOP.

Allogene’s study results were an important early step towards validating the company’s choice. The therapy significantly exceeded the 25- to 30-point difference, compared to placebo, on minimal residual disease that investors and analysts had been looking for. The treatment also wasn’t associated with severe occurrences of the kind of immune or neurological side effects often associated with personalized cell therapies. 

Half of the patients in the cema-cel arm experienced neurological side effects such as headache and dizziness that were judged to be “low grade.” Two had mild infections, such as a urinary tract infection or COVID-19, according to Allogene.

“These interim data suggest that an off-the-shelf CAR-T may be able to intervene during that important window before clinical relapse to eliminate residual disease and make earlier intervention feasible in routine clinical practice,” Zachary Roberts, Allogene’s chief medical officer, said in a statement, calling the data “encouraging.”

Personalized, or autologous CAR-T treatments, are largely given at specialized centers. Allogene aims to show that its donor-derived treatment could be more accessible to those who receive care at community cancers. A third of study participants were infused at these centers, which “bodes well for broad adoption,” wrote William Blair analyst Sami Corwin in a client note.

Roger Song, an analyst at Jefferies, added in a separate note that cema-cel could become a $3 billion asset for Allogene “that is currently underappreciated.” Song has previously predicted that cema-cel could bring in peak global revenues of $1.1 billion per year.

Kyverna Therapeutics is seeking clearance of what could be the first marketed cell therapy for an autoimmune condition after the treatment succeeded in a pivotal trial in a progressive condition known as stiff person syndrome.

According to Kyverna, four months after treatment with its therapy miv-cel, Phase 2 study participants had a median 46% improvement from the trial’s start on a type of timed walking test. More than four-fifths had at least a 20% improvement, a threshold that’s believed to be “clinically meaningful,” Kyverna said in December. The therapy met its key secondary study goals as well.

Kyverna said there were no “high-grade” cases of the immune or neurological side effects associated with treatments like miv-cel. There were Grade 3 or 4 instances of neutropenia, or low counts of a kind of white blood cell, but Kyverna said they were “manageable.” The company began filing an application for U.S. approval in May. 

Kyverna has long been a front-runner in the race to transform the types of cell therapies originally developed for cancer into autoimmune disease treatments.

Originally formed to pursue so-called Treg cell therapy, the company changed direction and licensed a more conventional cell therapy technology that’s yielded what’s now known as miv-cel. Fueled by academic studies suggesting these therapies might rewire the immune systems of people with autoimmune disorders like lupus, it raised hundreds of millions of dollars in private and public funding. But mixed early results and growing competition tempered excitement about these treatments, depressing the share price of Kyverna and many of its peers. 

Kyverna swapped CEOs and, under a new leader, began developing miv-cel for stiff person syndrome, a disorder that causes intensifying muscle stiffness. While rare, the condition has less therapeutic competition than Kyverna’s other targets and, because of its fast progression, offered a quicker path to clinical results.  

Since then, Kyverna has begun clawing back some of its stock value and quickly completed enrollment in a registrational trial. Its goal in the 26-patient study was for miv-cel to help drive at least a 20% change on a timed, 25-foot walking test. Executives had pointed to case reports suggesting miv-cel could produce more powerful effects.

According to Leerink Partners analyst Thomas Smith, the fresh results are a “best-case scenario” for Kyverna, enabling the company to bring to market the first and only therapy for stiff person syndrome as well as, more broadly, the first approved cell therapy for an autoimmune disease.

In addition to clearing the important walking test threshold, miv-cel also helped many patients no longer need a walking device after 16 weeks and, so far, has freed all study participants from the immunotherapies typically used to treat their condition. 

“We believe this highlights miv-cel's potential to provide unprecedented clinical benefit, while significantly reducing or eliminating chronic treatment burden,” Smith wrote. 

Gilead Sciences in February deepened its investment in cell therapy, agreeing to acquire its longtime development partner Arcellx in a $7.8 billion deal centered around an experimental multiple myeloma treatment. 

Gilead already owned about 11.5% of Arcellx’s equity prior to the deal’s announcement. It then paid $115 per share for the rest of Arcellx’s stock. Arcellx stockholders could receive another $5 per share in future payouts, too, should the biotech’s top program, “anito-cel,” win approval and go on to generate $6 billion in cumulative net sales through the end of 2029. 

Gilead is best known for its HIV drugs. But it’s also long been a leader in cancer cell therapy, a way of modifying the body’s immune defenders to hunt and kill tumors. Through its $12 billion acquisition of Kite Pharma nearly a decade ago, the company acquired two blood cancer therapies now known as Yescarta and Tecartus. Those therapies are the centerpiece of a business that now brings in more than $1 billion annually and that Gilead has looked to as a way to diversify into oncology. 

Still, Gilead’s cell therapy business took years to ramp up, part of a broader struggle companies have had profitably selling complex treatments that take weeks to make and are largely administered at major institutions. And of late, sales have declined amid pressure from newer competitors.

Yet Gilead sees anito-cel potentially turning that business around. Arcellx medicine imbues T cells with the ability to fight multiple myeloma, a progressive cancer of the bone marrow. That therapy has already been submitted for U.S. approval based on study results showing a 96% response rate among recipients whose multiple myeloma had relapsed, or hadn’t responded to, at least three prior treatments. A decision is expected by Dec. 23. 

Effective, marketed cell therapies from Johnson & Johnson and Bristol Myers Squibb work similarly, as do a handful of “bispecific” antibody therapies. Gilead, for its part, said that anito-cel has demonstrated “deep and durable responses” with a “predictable and manageable safety profile,” which addresses “key challenges” associated with existing multiple myeloma cell therapies. The companies have been aiming to prove, for instance, that people who receive anito-cel have a lower risk of a cell therapy-related side effect called parkinsonism. 

Antio cel “could become a foundational treatment for multiple myeloma over time, including earlier lines of therapy,” said CEO Daniel O’Day, in a statement.

The therapy has “shown generally comparable efficacy” to Carvykti with the potential for a better safety, wrote RBC Capital Markets analyst Brian Abrahams, in a Monday note to clients. But Gilead and Arcellx are behind competitors in an area “where early entrenchment may be important. The “degree of market acceptance” for a cell therapy in the earliest lines of care is also “unclear,” he added.

Like some of its peers, Gilead has also invested in “in vivo” treatments that modify cells inside the body and are seen as more convenient alternatives to existing therapies. Gilead claimed that some Arcellx research could be useful there, in addition to helping produce “next-generation” cell therapies and bispecific antibodies.

Chinese biotechnology company Oricell Therapeutics in April banked more than $110 million in a “pre-IPO” venture funding to help it advance a portfolio of cell therapies for tough-to-treat solid tumors. 

Oricell’s lead program is being tested against advanced hepatocellular carcinoma, an aggressive liver cancer that most frequently occurs in people with chronic organ damage. That therapy, Ori-C101, has already completed early testing in humans and demonstrated what the company claims to be a “best-in-class efficacy and safety profile.” 

Most of the cell therapies that have reached market so far are used to treat blood malignancies. Solid tumors have proven trickier foes, however. These tumors often have cells expressing a variety of antigens, making them hard to target with a therapy aimed at only one. They also use a layer of immune-suppressing cells and other molecules as a protective shield against the body’s defenses.  

Oricell is relying on multiple approaches to help, among them a kind of “armored” technology that helps cell therapies fight through this immune-weakening barrier. Its lead program is also aimed at a protein, GPC3, that’s highly expressed on cancerous liver cells and largely not on healthy tissue. In a small study presented at the annual American Society of Clinical Oncology meeting in 2025, the company said six of 10 patients treated with its therapy responded to treatment, and nine achieved disease control. 

Oricell’s pipeline includes six other named drug prospects for other solid tumors and blood cancers. One, “OriCAR-017,” is in a Phase 1/2 trial in China for multiple myeloma. Results are expected later this year. Oricell is also working on “in vivo” CAR-T technology, an increasingly popular approach through which immune cells are engineered within a patient’s body.

“We are committed to delivering transformative therapies that offer real hope to cancer patients worldwide, positioning Oricell as a dominant force in the global immunotherapy arena,” Huanfeng Yang, Oricell’s CEO, said in a statement.

The Food and Drug Administration last year eased limitations it imposed around the complex cancer drugs known as CAR-T therapies, removing several onerous requirements as well as loosening restrictions on which facilities can provide treatment.

The FDA’s action applied to therapies made by Bristol Myers Squibb, Gilead Sciences, Johnson & Johnson and Novartis that are used to treat several types of blood cancer. Specifically, it removes so-called “Risk Evaluation and Mitigation Strategies” from the drugs’ labeling, which are used to help manage serious side effects of treatment.

According to the FDA, these REMS requirements are no longer necessary to ensure safe use of these CAR-T therapies as both physicians and hospitals are now well versed in managing the two syndromes most commonly associated with the drugs.

Since the FDA’s approval of Novartis’ Kymriah for leukemia in 2017, six more CAR-T therapies have reached market. These medicines are made from a patient’s own immune cells, extracted from the body and engineered in a lab to hunt down specific proteins found on the surface of malignant clones.

For some people with leukemia, lymphoma or multiple myeloma, they can prompt powerful responses and even long-lasting remission. But their administration also comes with notable risks, including a hyperactive immune response known as CRS and neurological toxicity dubbed ICANS.

When CAR-T therapies were new, physicians and the hospitals they worked at faced a learning curve handling these treatment-associated conditions, which require specific management and drugs to bring under control.

The REMS imposed by the FDA was designed to help mitigate these risks by requiring hospitals receive special certifications before they could administer treatment, and mandating specialized reporting of side effects to the agency.

“Given the established management guidelines and extensive experience of the medical hematology [and] oncology community in diagnosing and managing the risks of CRS and neurologic toxicities across products in the class of BCMA- and CD19-directed autologous CAR-T cell immunotherapies, FDA has determined that the safe and effective use of CAR-T cell immunotherapies for the indicated population can be assured without a REMS,” the agency said in a statement last June. BCMA and CD19 are the protein targets of the seven approved CAR-T therapies.

Reporting rates for CRS and ICANS have “remained stable,” the FDA added.

Alongside removal of the REMS, the regulator also reduced its requirement that patients remain nearby the facility they received treatment from one month to two weeks. And its limitation on how long patients must wait before they can drive or operate machinery again after treatment is lowered from two months to two weeks.

Removal of the REMS should lower the logistical hurdles patients face in receiving CAR-T treatment as well as the cost of administration, the Alliance for Regenerative Medicine, a trade group, said in a post to LinkedIn.

“We believe the reduction in CAR-T burden for patients and caregivers, enabled by FDA's new monitoring and driving requirements, will drive expansion of the U.S. cell therapy market,” Daina Graybosch, an analyst at Leerink Partners, wrote in note to clients.

In a statement, Bristol Myers, which sells the CAR-T therapies Abecma and Breyanzi, noted that only two in 10 eligible patients receive the treatments “due to the confluence of complex logistical and geographic barriers affecting patients and providers.”

The changes could help expand use of CAR-T from specialized medical institutions, where it’s most commonly given, to community health centers, Bristol Myers said.

Breyanzi, Kymriah and Gilead’s Yescarta and Tecartus are cleared to treat lymphoma, although Kymriah is more commonly used for leukemia, where it is also approved. Abecma and J&J’s Carvykti are used for multiple myeloma.

The most recently OK’d CAR-T treatment, Autolus Therapeutics’ Aucatzyl for lymphoma, was the first to be cleared without an initial REMS program.

Available cell therapies for cancer aren’t easy treatments to receive. Personalized to each patient, the drugs are complicated and time-consuming to manufacture, and must be delivered and administered on an exacting schedule. And because they come with serious risks, patients need to remain close to the hospital where they were treated for weeks of monitoring.

All of these characteristics make Johnson & Johnson’s choice to roll out direct-to-consumer advertising for Carvykti, a cell therapy it sells for the blood cancer multiple myeloma, somewhat unexpected. The ad, which will air on streaming services like YouTube, Paramount and Roku, is J&J’s first for the drug and a rare instance of DTC marketing for this kind of cell therapy, known as CAR-T.

According to June Lanoue, J&J’s head of U.S. hematology, the company aims to raise awareness about cell therapy, particularly among patients who may not be receiving care from the kind of specialized academic centers where CAR-T is often given.

Carvykti is currently approved in the U.S. to treat multiple myeloma in patients whose disease either relapsed or becomes resistant to treatment after an initial round of therapy. Another CAR-T therapy, called Abecma and sold by Bristol Myers Squibb, is also cleared for this use, but only after two or more prior lines of treatment.

Among these “relapsed, refractory” patients, about half are treated at community oncology centers, Lanoue said. “The actual awareness level of these options is very low,” she added. “In the community, if they’re not able to get it, doctors are not talking about and therefore they don’t have visibility to having a new option.”

J&J’s choice to advertise is a signal of confidence in its ability to manufacture enough Carvykti to meet any rise in demand. Early on in the drug’s launch, production challenges forced the company to limit orders from treating centers. (Bristol Myers ran into similar issues.)

“We feel very confident around production,” said Lanoue. Treatment sites can “order Carvykti without any constraint.”

The launch of the ad came several weeks after the Food and Drug Administration took steps to improve access to CAR-T treatment broadly. The agency eased some side effect reporting requirements and lessened the time it mandates patients stay near their treating facility for monitoring. It also said patients could resume driving or operating machinery sooner after treatment than previously prescribed.

“We think it will give more opportunity to more patients who [live at] a little bit further distance to be able to receive Carvykti,” Lanoue said.

J&J’s 120-second ad introduces a composite patient, “Marvin,” and describes the heavy burden of injections and pills typically used to control multiple myeloma. Carvykti, by contrast, is a one-time infusion that can deliver lasting benefits in some.

Recent follow-up data from an early study of around 100 patients treated with Carvykti showed that, at five years, one-third had not yet experienced disease progression. A story published in June by The New York Times that highlighted these findings led to more patients requesting information about Carvykti, according to Lanoue.

J&J did not share how much it’s spending on this marketing campaign, but Lanoue said it is “significantly less” than what a pharmaceutical company would typically spend on a regular TV advertisement. “This is not a share of voice play; this is ultra targeted,” she added.