Osteosarcoma is a rare cancer affecting the bones. It is also the most common bone cancer in children and young adults but can also occur in older adults. It mostly affects the knee, thigh bone, shin bone or upper arm. Symptoms include pain and swelling around the affected bone. Current treatment options have improved the prognosis of this disease, however, a recurrence of osteosarcoma may appear in other parts of the body after the primary cancer has been treated. The majority of the recurrences occur in the lungs. Symptoms of lung involvement may include cough, breathlessness, pain or discomfort in the chest.
Inhaled Lipid‐complexed Cisplatin or inhaled liposomal cisplatin (ILC) is an anticancer drug being developed for the treatment of osteosarcoma that has reoccurred and spread to the lungs. Cisplatin is already available in the UK as an intravenous injection for treating multiple types of malignant conditions. ILC is administered via inhalation has the potential advantage to deliver the drug directly to the site of action (the lungs) with significantly reduced systemic side effects and toxicities. If licensed, ILC will offer an additional treatment option with the potential to improve effectiveness and safety for patients with pulmonary relapse of osteosarcoma.
Selinexor is the first treatment option that targets XPO1, a protein that is responsible for exporting tumour suppressor proteins from the cell nucleus. It belongs to a new family of therapies called selective inhibition of nuclear export (SINE) compounds that blocks XPO1 leading to controlled death of myeloma cells. Currently there is no standard of care for the fifth line treatment of MM. Selinexor and low-dose dexamethasone are being developed as an oral treatment. If licensed, this combination could be an effective treatment option for a patient group with clear unmet need.