For nearly three years, Canadians with multiple myeloma have been anxiously waiting for a promising form of immunotherapy to become publicly funded – and some worry their time may be running out. Carvykti, a type of CAR T-cell immunotherapy approved by Health Canada, has been hailed by researchers and patient advocates as a significant advancement in the treatment of multiple myeloma, an incurable blood cancer that affects plasma cells in the bone marrow. The treatment works by collecting a patient’s own T-cells, genetically modifying them to target cancer cells and then infusing them back into the body. Unlike many myeloma treatments that require ongoing therapy, Carvykti is designed as a one-time treatment. While Health Canada has authorized the therapy and Canada’s drug review agency has recommended it for reimbursement under certain conditions, public funding has yet to be approved. Negotiations between Johnson & Johnson Innovative Medicine and the pan-Canadian Pharmaceutical Alliance (pCPA), which negotiates drug prices on behalf of provincial, territorial and federal governments, remain ongoing. For patients like Lorelei Dalrymple of Okotoks, the uncertainty is difficult to watch. “I’ve lost seven people in 18 months,” said Dalrymple, who has been living with multiple myeloma since 2009. “I’ve lost a lot more than that in the 17 years since I’ve been diagnosed, but to see this remain under negotiations is incredibly frustrating. This drug could have been available for them; it should have been available for them.” Living with an incurable cancer Multiple myeloma is the second most common blood cancer in Canada. Although treatments have improved significantly in recent decades, the disease remains incurable and patients typically cycle through periods of remission and relapse. Dalrymple’s path to diagnosis was anything but straightforward. She first began experiencing symptoms in 2005 when she lost feeling in her feet and legs. Doctors initially struggled to determine the cause, and she spent several years being treated as a patient with multiple sclerosis before receiving a diagnosis of myeloma in 2009. “I didn’t present in the usual way,” she said. “I didn’t have any bone involvement. I didn’t have any infections. My bloodwork was all normal.” Since then, she has undergone years of treatment, radiation and surgeries related to the disease. “I’ve been on some form of treatment now for almost 17 years,” she said. “I’ve had numerous rounds of radiation, I’ve had fractures, I’ve had breaks, I’ve had a metal rod put in my femur to prevent a break.” The disease continues to affect her daily life. “I’m in some level of pain every day, so the years that are supposed to be my golden years are a little bit more challenging.” Dalrymple says Carvykti represents something different from the treatments she has relied on for nearly two decades. “This could be a real game changer for some people – especially if you’ve been through so many different treatments – and it could save health care costs in the long run,” she said. “We need to look at this holistically. We need to say, ‘Okay, if I can give someone this really expensive treatment, then maybe they can stay out of the health system much longer.’” A treatment offering new hope Health Canada first approved Carvykti for certain heavily pre-treated multiple myeloma patients in 2023, and later expanded its authorization to include some patients who have received fewer previous treatments. Clinical trial results have shown the therapy can significantly reduce the risk of disease progression or death compared with standard treatments. More recent data suggests some patients remain alive and disease-free years after receiving a single infusion. Martine Elias, CEO of Myeloma Canada, says the treatment has generated considerable excitement within the myeloma community. “Thirty-three per cent were still alive five years after their CAR-T treatment,” Elias said of patients in long-term studies who had advanced disease and had already received numerous treatments. “To have a therapy that can keep you alive in 33 per cent of the cases is game changing.” Elias says the potential benefits could also be even greater if patients receive the treatment earlier in their disease progression. “The earlier you treat, the better the outcomes are,” she said. Elias described Carvykti as a major step forward because patients can potentially go years without additional treatment after receiving it. “You take the CAR-T, and then you don’t have any other treatment for your myeloma until you relapse – and hopefully you don’t relapse,” she said. Cost remains a major hurdle The challenge, however, is cost. According to Elias, the publicly listed Canadian price for Carvykti exceeds $630,000 for the therapy itself. Additional expenses include hospital stays, specialized laboratory work, physician care and monitoring. Patients currently have limited options to access the treatment. Some participate in clinical trials, while others seek treatment outside Canada at significant personal expense. “I’ve got one patient who actually went to the United States to pay for it out of his own pocket at an exorbitant amount,” Elias said. For many patients, that simply is not possible. “It is very expensive,” said Calgary resident Bev Dunn, who was diagnosed with multiple myeloma in 2018. Dunn, who is also the president of the Southern Alberta Myeloma Patient Society, has undergone a stem-cell transplant and multiple rounds of treatment. After relapsing twice, she is currently responding well to another therapy. Still, Dunn says she worries about those who have exhausted most of their options. “The people I think about in my dealings with the support group that I’m involved with, are people who are on their last line of treatment now and have no further options,” she said. “Their option would be to travel outside the country to get Carvykti. That drug would save their lives, and we know that It can save lives.” Negotiations continue The pCPA confirmed negotiations with Janssen Inc., the manufacturer of Carvykti, remain active. “The negotiation with Janssen Inc. is ongoing,” said Mauro Chies, CEO of the pCPA, in a statement. “Our negotiation team has been dedicated to this file and has remained engaged throughout the process. As negotiations are two-way streets, we unfortunately don’t have a timeline to share with you.” Should a deal eventually be reached, individual provinces and territories would still need to make their own decisions regarding public coverage. The Alberta government said it recognizes the importance of additional treatment options for patients. “We know how important having treatment options like Carvykti are for Albertans facing multiple myeloma,” said Jaye Lang, press secretary for Primary and Preventative Health Services, in a statement. The province noted that no province or territory currently provides public funding for the therapy, and jurisdictions across Canada are awaiting the outcome of pCPA negotiations. “Until then, patients continue to have access to publicly funded treatment options, and their oncology teams are best positioned to guide care decisions for each patient’s needs.” Patients urge action While negotiations continue behind closed doors, patients and advocates say they hope a resolution can be reached soon. “It shouldn’t take that long to get to an agreement,” Elias said. “The longer it takes, the more concerning and very frustrating it is for patients.” Dalrymple says while she is fortunate that her current treatment continues to work, she worries about people whose disease is progressing now. “I’m really hoping it’s there for me at my next relapse,” she said. “But right now I really want it there to save the people that can’t afford to wait.” “There are lots that just simply can’t afford to wait.”
