Kymriah Treatment Pulls Young Leukemia Patient from the Darkness

The end of the teenage years is typically a time when people attend college or think about starting a career and, luckily, many do not have to navigate a complex health situation. This wasn’t the case for Lucas D. Novick, who was diagnosed with B-cell acute lymphoblastic leukemia (ALL) in 2009 at age 19.

Now 28 years old, Novick has been in remission for nearly 3 years thanks to participation in a clinical trial of tisagenlecleucel (Kymriah).

Novick told Specialty Pharmacy Times that despite prior treatments with chemotherapies and bone marrow transplants, he experienced 2 relapses in 2013 and 2015. With chemotherapy, he experienced serious adverse events that included nausea, weight loss, bone pain, depression, and avascular necrosis, which lead to hip surgeries. Vincristine chemotherapy also resulted in long-term neuropathy, for which he is still receiving treatment though the severity has lessened.

“At first, it was very manageable and pain was relatively infrequent, but it grew progressively worse, requiring 2 total hip replacements in 2010 before I could walk without a cane,” Novick told SPT.

A bone marrow transplant Novick underwent in 2013 brought similar, but more severe adverse events. As he neared a release from the hospital, Novick suffered a setback when he developed venous occlusion disease and experienced near total liver and renal failure.

A procedure to address this additional health scare should have only required a limited hospitalization, but instead resulted in a 2-month stay that sapped his strength. Even climbing up the stairs after the transplant was extremely difficult and required setting modest goals, Novick told SPT.

Novick described his second relapse as the darkest time of his life.

“I had known something wasn’t right for some time before I actually learned my leukemia had returned,” Novick said. “Maybe beginning around late fall of 2014, I would lay down to sleep and my insides would just hurt.”

At this time, he believed these symptoms were related to recovery from the bone marrow transplant or medication-related adverse events. Once Novick developed headaches and distorted vision that left him dizzy and unable to drive, it was clear that the disease had returned, but this time in the form of cancerous bone lesions in his skull and in his sphenoid sinuses, which are uncommon for ALL.

“There were also solid tumors present around my pancreas and throughout my abdomen, which was believed to be a cause of some of the pain I had been experiencing in the weeks and months prior,” he said.

Novick said his care team was not confident he could achieve remission again or even survive another bone marrow transplant, thus bringing about quality of life discussions. He had to decide whether undergoing chemotherapy was worth the adverse events.

With a lack of success from treatment with traditional ALL therapies, Novick turned to Google and ClinicalTrials.gov to explore his options and stumbled upon a clinical trial for tisagenlecleucel, a chimeric antigen receptor T cell therapy.

Due to limited options, Novick elected to receive chemotherapy to make him more comfortable and address some of the symptoms of ALL while he awaited enrollment in the clinical trial. The idea of undergoing another transplant or chemotherapy along with the related adverse events were a tough pill to swallow.

“We think so much about the cancer and lose sight of the fact that it’s not just the cancer being treated, but the patient. And that patient is a human being,” Novick said.

Novick said that enrolling in the clinical trial raised his excitement due to the buzz surrounding the innovative gene therapy. The new treatment also provided something much more valuable to a patient battling uncertainty and fear while fighting a life-threatening disease.

“It was giving me hope at one of the most hopeless times during the entirety of my treatment,” he said. “I felt that even if it did not put me in remission, the data gathered from my participation would be part of something that could go on to help other people.”

Despite the black box warning of cytokine release syndrome or neurologic symptoms, Novick said that the severity of the adverse events he experienced with tisagenlecleucel were not even close to being as debilitating as those he suffered from previous treatments; however, this may not be the case for all patients.

Just 3 months after treatment with tisagenlecleucel, Novick was back in school full time and living on his own. He also began feeling better within weeks of starting treatment, which was significantly quicker than at any point in time post-transplant.

It has been nearly 3 years since Novick achieved remission. In that time, he has completed undergraduate school and is now aiming to enroll in law school.

Tisagenlecleucel was launched at a cost of $475,000 for a single infusion. Despite the high cost, Novick said that most people would pay any price to ensure a loved one could benefit from the treatment and that the emergence of other gene therapies may reduce the costs.

“I have seen some sources describe [tisagenlecleucel] as the most expensive treatment ever developed in the history of medicine, but it’s a huge step in developing [medications] that treat the disease as much as they treat the patient,” Novick said. “But the concern over cost is warranted and I’m happy there are people out there having that conversation. I just hope that all sides of the debate don’t lose their sense of compassion and empathy as they make their arguments.”

Novick added that many patients who experience multiple relapses may choose to discontinue treatment due to the immense emotional and physical toll it takes on them and their family members. Without the clinical trials, he may have been forced to make that tough choice at a very young age.

“Kymriah and others like it make me more hopeful that there may be a not too distant future where there are more viable options, so people won’t have to make decisions like that,” Novick said. “I don’t think people should have to make such drastic compromises between a lowered disease burden and quality of life. There aren’t very many treatments that I am aware of that make that possible. Yet Kymriah is one of them, and that’s very exciting.”