The first treatments are aimed at Acute Myeloid Leukaemia (AML), a blood cancer that carries a very poor prognosis, and Small Cell Lung Cancer (SCLC), an extremely aggressive subtype of lung cancer that is related to smoking. The 5-year survival rate for AML-patients is less than 30%, the 5-year survival rate for patients with SCLC is barely 5%. Over the last decades, advancement in therapy has had little effect in these cancers.
AML is a blood cell cancer that arises when descendants of blood stem cells, which should normally mature into lymphocytes or myeloid cells, stop maturing and continue to proliferate. The immature cells fill up the bone marrow and prevent other cells from maturing.
The diagnosis and determination of the subtype of AML are usually done by bone marrow aspiration and examination of peripheral blood. AML is initially treated with chemotherapy. If untreated, the leukaemia causes death within a few weeks or months.
The effectiveness of chemotherapy may be limited because the leukaemia cells can become resistant to it over time. The majority of patients will relapse, even after an initial successful treatment. Relapses carry a poor prognosis; most patients no longer respond to treatment and die from anaemia, infection, or multiorgan failure.
The frequency of AML increases dramatically with age. The prognosis for patients over 65 years of age is poor because patients in this group often have low tolerance for aggressive chemotherapy, are most resistant to chemotherapy and most likely to relapse.
AML is a relatively rare disease. GLOBOCAN, a cancer database, estimates that the worldwide incidence of AML for 2012 is 350,000 cases (4.7 cases per 100,000 people). In the US, there are an estimate 20,050 new cases of AML in 2019 and 11,400 deaths.
Lung cancer is the leading cause of cancer mortality around the world accounting for 1 million deaths annually. There are two major subtypes: non small-cell lung cancer (NSCLC) and small-cell lung cancer (SCLC). SCLC is strongly correlated to a history of smoking and makes up for 13-15% of all lung cancers. In contrast to NSCLC, where significant advances have recently been achieved with immune therapy, the SCLC landscape has remained static for the last thirty years. Many agents have been tested in recent years, but none has demonstrated convincing clinical benefit.
SCLC has a peak incidence at 68-80 years of age and the 5-year survival rate is around 5%, therefore, any significant progress in overall response rate and survival in the SCLC population would constitute real progress in this disease .
The SCLC incidence is slightly higher than the AML incidence. In 2019, the US accounted for 18.25% of new cases of SCLC in the 8 major markets (5 EU countries, Japan, China, US) adding 32,910 new patients.