Breakthrough CAR T-Cell Therapy Offers Hope for Cancer Patients

A promising cancer treatment developed in Wellington could significantly change the lives of patients facing terminal diagnoses. This innovative therapy, known as CAR T-cell therapy, is showing potential for long-term remission in patients with limited time left, and it may be available through New Zealand’s public healthcare system by 2027.

BioOra Limited, a biotech firm partly owned by the Malaghan Institute of Medical Research, is preparing to manufacture this therapy rapidly. Once the current trial concludes, the company aims to provide the treatment for both public and private healthcare sectors. This development is crucial for patients who have been forced to seek expensive treatments abroad, particularly in countries like China.

The high-tech manufacturing facility being constructed in Christchurch will utilize an automated system to produce CAR T-cell therapy at scale. This process is designed to minimize contamination risks and ensure consistent quality. However, John Robson, managing director of BioOra, expressed concern that delays in the public system could jeopardize patient lives and hinder the company’s progress.

Robson stated, “We’re kind of on this knife edge of, will they go fast enough for us? Quarter 1 of 2027 is a distinct possibility for public access, but it has to dovetail with a funding stream.” If public support is not forthcoming, BioOra is prepared to offer the treatment privately, with discussions already underway with interested health providers.

The CAR T-cell therapy itself involves extracting a patient’s immune cells, genetically modifying them to target cancer cells, multiplying them, and infusing them back into the patient’s bloodstream. This treatment has already become a standard for specific blood cancers in countries like Australia, yet it remains unavailable in New Zealand’s healthcare systems.

The Malaghan Institute is spearheading efforts to change this. Their designed CAR T therapy has shown promising results in a phase 1 clinical trial, where more than half of the lymphoma patients—who had exhausted other treatment options—achieved remission within months. Notably, the trial reported significantly lower rates of severe side effects compared to leading commercial CAR T therapies available overseas, making it likely that this treatment could be administered on an outpatient basis, thereby reducing overall costs.

As BioOra prepares for phase 2 of the trial, they expect to enroll their final patient with large B-cell lymphoma around October 2023. Final results will be crucial for obtaining approval from Medsafe, New Zealand’s medical regulatory authority.

BioOra emerged from the Malaghan Institute to transition CAR T-cell therapy from trials to a commercially viable product. Currently, the company produces CAR T cells for clinical trials in Wellington but is raising funds to establish a new facility in Christchurch. Robson noted that they have secured the necessary lease and designs for a Good Manufacturing Practice (GMP) facility, which aims to be certified and operational by the end of 2026. This facility is expected to create approximately 45 to 50 jobs.

The initial manufacturing goal is to produce cells for up to 160 patients in the first operational year, with plans to increase that number to over 300 in the second year. Robson emphasized the need to align the scaling of production with the demand for therapy, particularly as the company seeks additional funding from private investors.

The financial implications of introducing CAR T-cell therapy into the public system are significant. While the upfront costs are estimated to be lower than the $1 million charged in Australia and the United States, BioOra is committed to providing a cost-effective alternative that does not compromise safety. Robson remarked, “The price becomes secondary to, ‘Will I be in ICU, and is my life at risk?’”

BioOra’s business model includes returning a substantial portion of profits to the Malaghan Institute for further research and development of CAR T therapies and other immunotherapies. Future treatments could target various conditions, including childhood acute lymphoblastic leukaemia and solid tumor cancers.

Robson believes that establishing a domestic capability for immunotherapy manufacturing could revolutionize treatment access in New Zealand. “The sovereign capability to manufacture immunotherapy is a game changer,” he stated. “These therapies will challenge disease by disease over the next five to 10 years.”

Additionally, BioOra is exploring options to tap into the Australian market. While CAR T-cell therapy is already integrated into Australian healthcare, the transportation of patient cells to laboratories in the United States or Europe adds delays that can jeopardize patient outcomes.

The Malaghan Institute has launched a campaign to raise funds for the phase 2 trial, with costs expected to exceed $17 million. This funding is separate from that required by BioOra, which raised $15 million in a previous capital round, with plans for another round to secure an additional $30 to $45 million.

Robson stressed that timely access to CAR T-cell therapy could yield significant savings for New Zealand’s healthcare system. “If you have a 20 or 30-year-old treated on a curative pathway, their contribution to society is immense,” he explained.

The New Zealand Cancer Control Agency, Te Aho o Te Kahu, is currently leading discussions on the regulatory and operational implications of introducing CAR T-cell therapy. Acting Chief Executive Nicola Hill mentioned that new legislation, the Medical Products Bill, is expected to facilitate flexible approval pathways for treatments like CAR T-cell therapy, although implementation will not occur until late 2030.

The race to deliver this life-changing treatment to patients is critical, and the coming months will determine whether BioOra can navigate the regulatory landscape to bring CAR T-cell therapy to those in need.