Revolutionary CAR T-Cell Therapy Offers Hope for Cancer Patients

A groundbreaking cancer treatment developed in Wellington has the potential to transform the lives of patients facing terminal diagnoses. The innovative CAR T-cell therapy, which has already demonstrated remarkable success in clinical trials, could be available to the public healthcare system in New Zealand as early as 2027, pending regulatory approval and government support.

BioOra Limited, a biotech company partly owned by the Malaghan Institute of Medical Research, is preparing to manufacture CAR T-cell therapy for both public and private healthcare systems. This follows a successful phase 1 trial, where over half of the patients with lymphoma who had exhausted other treatment options achieved remission. Currently, patients seeking this therapy in other countries, such as China, are facing substantial costs.

The company, with aspirations to become “the Rocket Lab of immunotherapy,” is constructing a state-of-the-art manufacturing facility in Christchurch. This facility will employ an automated system to produce CAR T-cell therapy efficiently and with reduced contamination risks. However, John Robson, managing director of BioOra, has expressed concerns about the speed at which the public healthcare system may respond. He stated, “We’re kind of on this knife edge of, will they go fast enough for us?”

Challenges Ahead for Public Access

Robson highlighted that while the first quarter of 2027 is a realistic target for public access, it hinges on securing necessary funding. If the public system does not act swiftly, BioOra may provide the therapy privately, with discussions already underway with interested providers.

CAR T-cell therapy involves modifying a patient’s immune cells to target and eliminate cancer cells. While it has become a standard treatment for certain blood cancers in countries like Australia, it remains unavailable in New Zealand. The Malaghan Institute aims to change this by developing a CAR T therapy that has shown promising results in clinical trials with fewer serious side effects compared to existing therapies.

Phase 2 of the trial is expected to enroll its final patient with large B-cell lymphoma around October 2023, with results critical for regulatory approval from Medsafe.

BioOra, which was established in 2021 with backing from the investment firm Bridgewest Ventures, is currently producing CAR T for trials from Wellington. The company is actively raising tens of millions of dollars from private investors to establish its new facility, slated to be operational by late 2026. The facility will create approximately 45-50 jobs in Christchurch and aims to manufacture treatment for up to 160 patients in its first year.

The Path to Regulatory Approval

The timeline for public access to the therapy is closely linked to the ongoing clinical trial results, which will be submitted to Medsafe incrementally throughout the year. The agency is expected to take three to six months to review the data and grant approval or raise objections. While Robson could not disclose specific costs, he assured that the price would be significantly lower than the $1 million charged for comparable treatments in Australia and the United States.

BioOra plans to allocate a significant portion of its profits back to the Malaghan Institute for further research and development of CAR T therapies and other immunotherapies. Future treatments could address a range of conditions, including childhood acute lymphoblastic leukaemia and various solid tumour cancers.

Robson emphasized the importance of establishing a sovereign capability to manufacture immunotherapy, stating, “The sovereign capability to manufacture immunotherapy is a game changer.” This development could lead to more affordable treatments and potentially attract international patients seeking care in New Zealand.

BioOra’s strategic focus includes exploring access to the Australian market, where CAR T-cell therapy is already integrated into public and private healthcare systems. Current practices require transporting patient cells to laboratories in the United States or Europe, which delays treatment for patients who cannot afford to wait.

The Malaghan Institute has initiated a fundraising campaign to cover the costs of the phase 2 trial, estimated to exceed $17 million. This financing is separate from BioOra’s funding, which has included a recent capital raising round of $15 million. The next round aims to secure an additional $30-45 million.

Robson underscored the potential health and economic benefits of securing public access to this therapy. He noted that while the upfront costs are substantial, the long-term savings from reduced hospitalizations and improved patient outcomes could be significant.

As health authorities, including Te Aho o Te Kahu and the Cancer Control Agency, work together to streamline the regulatory process, new legislation known as the Medical Products Bill is set to introduce more flexible approval pathways for treatments like CAR T-cell therapy this year. Nonetheless, full implementation is not expected until late 2030.

The future of CAR T-cell therapy in New Zealand hangs in the balance, and the decisions made in the coming months could determine whether this life-saving treatment becomes widely accessible to those in need.