Despite distinct advances in drug development, there is nevertheless a great unmet need for medications to treat some serious diseases. Treatment options, especially for rare, aggressive types of cancer are still inadequate. Following diagnosis, life expectancy is often only a few months to a few years.
Trabedersen is Antisense Pharma’s first drug whose safety, tolerability and efficacy are being tested in clinical studies. Trabedersen inhibits the formation of TGF-β2 (transforming growth factor-beta 2) – a factor that is overproduced by different aggressive types of tumors and that plays a decisive role in the rapidly advancing course of the diseases.
Trabedersen has been tested in clinical studies involving the following types of cancer:
Malignant brain tumors (high-grade gliomas)
High-grade gliomas are among the most frequently occurring brain tumors and are especially aggressive. They arise from glial cells – supportive cells of the nerve cells – and are therewith classed as so-called primary brain tumors, in contrast to brain metastases, which have their origin in other tissues.
Worldwide, nearly 240,000 people per year are newly diagnosed with malignant tumors of the brain and central nervous system. Depending on which region of the brain is thereby affected, such tumors often drastically impair the individual’s quality of life. Even today, treatment of malignant brain tumors still poses a huge therapeutic challenge: The 5-year survival rate of patients with anaplastic astrocytoma (brain tumor, WHO grade III) is around 30%, and around only 3.4% for patients with glioblastoma (brain tumor, WHO grade IV).
Brain tumor cells are especially capable of migrating to other areas of the brain, so that upon diagnosis, it is likely that, in most cases, individual cells have already migrated to healthy brain tissue. The current, established therapy consists of complete surgical removal of the tumor, radiation therapy and chemotherapy – yet the disease recurs still in over 90% of the cases (recurrence).
Advanced pancreatic cancer (pancreatic carcinoma)
Pancreatic cancer is one of the most aggressive tumor diseases for which there is still no satisfactory treatment option, despite the most recent advances: According to the Robert Koch Institute, the 5-year survival rate for men is only 6.4% and for women is 7.6%.
Each year, nearly 280,000 individuals worldwide are diagnosed with pancreatic cancer – frequently affected are persons older than 50- to 60-years. Also smoking, diabetes, chronic pancreatitis, liver cirrhosis, unhealthy diet and lack of exercise are among the known risk factors. Furthermore, the risk of developing this type of cancer increases three-fold when there is a family history of pancreatic cancer.
There is no early diagnostic test like those available for breast or prostate cancer, which is why, in most cases, this type of cancer is only recognized in an advanced, metastasizing stage. At the time of diagnosis, only 10 to 15% of the tumors are operable, and inoperable tumors respond to chemotherapy only inadequately.
Advanced skin cancer (malignant melanoma)
Melanoma is a malignant tumor that arises from the pigment cells (melanocytes) of the skin. Although melanoma is one of the less frequent types of skin cancer, it accounts for the most skin cancer-related deaths. Worldwide, nearly 200,000 persons per year are diagnosed with skin cancer. The incidence of malignant melanoma differs from country to country. The European data show that in the Mediterranean countries, 3-5 persons per 100,000 inhabitants have this disease, compared with 12-20 persons per 100,000 inhabitants in the Nordic countries.
When early stage melanomas are recognized as enlarging and discoloring moles, they can be well treated by means of surgical removal and, in most cases, can be even cured thereby. Because this type of tumor tends to metastasize and spread early on via the blood and lymph stream, at the time of diagnosis in most cases other organs are affected, e.g. the brain. The chance of a cure is very low in metastasizing stages. As a rule, current therapeutic options offer merely short-term improvement, with no prospect of a cure.
Advanced colon cancer (colorectal carcinoma)
Colon cancer is one of the most frequently occurring cancers, affecting worldwide around 1.2 million people per year (new diagnoses); the mortality rate for newly diagnosed cases is approx. 50%. Important factors contributing to the development of colon cancer include life style and diet as well as a family history of the disease.
Screening examinations are of special significance for this type of tumor: When diagnosed early, colon cancer responds well to treatment and is also curable in most cases. Symptoms such as gastrointestinal complaints, constipation and cramps occur first in an advanced disease stage: Approx. 25% of patients already have metastases upon first diagnosis, and nearly 50% of colon cancer patients develop metastases over the course of the disease.
As a rule, treatment of advanced colorectal carcinoma (stage III/IV) consists of surgery, radiation therapy and chemotherapy. Nevertheless, the 5-year survival rate for stage III-patients is only 55-65%, and for patients with stage IV colon cancer, it is only 5%.