Gene Therapy – the need
We are in the midst of a genetic revolution.
During the last 20 years there have been unprecedented advances in our understanding of the molecular basis of human disease, and this in turn has led to increased diagnostic power. Indeed we are on the brink of being able to quickly and cheaply sequence the entire genetic blueprint of every child born, with a view to early diagnosis and management of genetic disease. Unfortunately, however, the development of new treatments for genetic diseases is lagging well behind this new-found diagnostic power, such that doctors are frequently left telling parents “we know what the problem is, but there’s nothing we can do about it.”
One extremely exciting possibility being actively pursued by researchers at the Children’s Medical Research Institute is gene therapy. This approach involves being able to repair or correct the underlying genetic defect using genes as medicine. A single treatment has the potential to deliver life-long cure. Due to considerable foresight and support by both the Children’s Medical Research Institute and The Children’s Hospital at Westmead, we have become globally recognised leaders in the field. We are focused on translating this growth in knowledge and diagnostic power through to novel treatments for infants and children.
This journey from the laboratory bench to the patient bedside is long, hard and resource intensive. Specialised clean-room facilities are required, along with highly trained staff, systems to ensure quality control and regulatory compliance, and finally gene delivery formulations specifically tailored and manufactured for the treatment of individual diseases.
Despite these formidable challenges we are making excellent progress. The necessary facilities are in place, and early trials are underway, but the future promises much more. The entire gene therapy field is on the brink of widespread therapeutic success across some of the most troubling and difficult to treat diseases affecting children. Against this background, and with the strong foundations already laid, we are in an excellent position to grasp the exciting possibilities at hand, bringing world-leading medicine to Australian children at the earliest possibility. But to do this we need additional funding support.
One of the fundamental requirements listed above for any gene therapy clinical trial is the manufacture of the gene delivery formulation necessary to perform genetic repair of patient cells. While we currently have a limited capacity to manufacture these gene transfer formulations we remain heavily dependent on supply from overseas facilities at great cost. For example, we have recently costed the supply (from the US) of a gene delivery formulation to treat infants with urea cycle defects (genetic liver disease) at $1,000,000 AUD. Multiple different trials using multiple different formulations would be correspondingly more expensive. Our thinking therefore is to further develop our local gene delivery formulation manufacturing capacity. This would dramatically reduce the fixed and recurrent costs involved, allowing us to drive a much greater number of novel therapies to the clinic.
We currently estimate that we require approximately $1,500,000 to establish this manufacturing capacity. Our vision would be to move to a revenue neutral model by leveraging additional funding from state and federal government agencies, and by supplying research grade formulations at cost to researchers within the Westmead Research Hub and across Australia.