The company believes the treatment is cheaper than alternative therapies, such as laser-based light-activated therapies, and can be tailored based on the number size and shape of the tumour.
Aptocine is a water-soluble compound that is injected into the patient in an outpatient clinic. The activation device consists of a narrow, flexible coated micro-wire that has an array of light activated diodes (LED) at one end.
This tube is inserted into the tumour, which is found using ultrasound, and when in place the LED emits red light at a discrete frequency and intensity for a fixed time period.
When the light comes into contact with Aptocine the therapy is activated. Aptocine molecules become energised and emit singlet oxygen molecules, which can kill target tissues through apoptosis (cell death).
Furthermore, the treatment causes vascular closure and this starves the remaining cancer cells of oxygen and nutrients. Consequently, the size of the tumour can be reduced in the 60 days following the treatment.
Preclinical and human studies also suggest that the treatment may have an additional anticancer effect. Following the creation of large apoptotic masses in tumours using light-activated drugs tumour-specific clones of CD8+ T-cells have been produced.
These cells can infiltrate distant, untreated tumours and reduce their size.
Treating PN
The Phase I trial is for patients with debilitating, severely disfiguring, life-threatening or progressive plexiform neurofibromas (PN) tumours that are not surgically resectable. LSO intends to enrol 18-24 patients aged three to 21.
PN is a complication arising from neurofibromatosis type 1 (NF-1), an autosomal genetic disorder and represents a significant unmet medical need, according to LSO.
The tumours form along peripheral nerves, become very large and cause pain, disfigurement and functional impairment. Currently complete surgical removal is the only effective treatment but this is rarely possible.