The neuroblastoma therapeutics pipeline is expected to witness considerable growth in the future due to high prevalence of tumour in children. According to the American Cancer Society, around 7.0% of total cancer cases occurred in the age group of 0 to 14, observed as neuroblastoma, with most of the cases being reported at an age of 2 to 3 years. There are various immunotherapies and targeted drug therapies that are being developed for treatment of neuroblastoma which are expected to further lead to the growth of its pipeline in the future.
Neuroblastoma is the most common type of cancer found in children, diagnosed mostly at the age of 1 year, characterized by broad range of clinical behavior. Neuroblastoma is derived from primordial neural crest cell which ultimately present into the sympathetic ganglia and adrenal medulla. It has been stated that the chances of survival in an event free from neuroblastoma are less than 50.0%. Neuroblastoma in chest can cause wheezing, chest pain, drooping eyelids and unequal pupils. The disease usually metastasized in bone marrow, skin, liver and bones until it is diagnosed. Abdominal pain, swelling in legs and altered bowel habits are the common symptoms of abdominal neuroblastoma. Fever, shortness of breath, high blood pressure, watery diarrhea and uncontrolled eye movement are the less common symptoms of neuroblastoma. Scientist have also concluded that neuroblastoma can also occurs due to genetic mutation. The elevation of level of urinary catecholamine is the primary evidence of neuroblastoma.
In March 2015, unituxin was approved as first line therapy for pediatric patients suffering with neuroblastoma. Severe pain, fever, low platelet counts, infusion reactions, low blood pressure, low levels of salt in the blood (hyponatremia), elevated liver enzymes, anaemia, vomiting, diarrhoea, low potassium levels in the blood, capillary leak syndrome are the most common side effects of unituxin. Many other drugs have also shown promising targeted therapies to treat neuroblastoma such as, 131I-metaiodobenzylguanidine, a radiopharmaceutical that is taken up by human norepinephrine transporter, leading to expression of 90.0% of neuroblastoma. Various other therapeutics have also shown positive results in treating metastatic neuroblastoma such as chemotherapy, immunotherapy and targeted drug therapy. Other drugs such as topotecan, irinotecan and temozolomide are also used for the treatment for neuroblastoma and isotretinoin has proven to prevent the occurrence of cancer after treatment. One of the primary target for neuroblastoma is anaplastic lymphoma kinase gene, which leads to the development of the cancer. Adriamycin PFS, Clafen, Vincasar PFS are also approved drugs for treatment of neuroblastoma.
Many companies are collaborating for the research and development of neuroblastoma drugs. Comedix Inc. has signed an agreement with Pediatric Oncology Experimental Therapeutics Investigators Consortium for developing a proprietary formulation of taurolidine for rare pediatric tumours. Sutro Biopharma collaborated with Memorial Sloan-Kettering Cancer Centre for production of bispecific antibodies for the treatment of neuroblastoma.
Some of the key players having a pipeline of neuroblastoma therapeutics include AstraZeneca Plc, Celgene Corporation, Progenics Pharmaceuticals, Inc., Novartis AG, Abbvie, Inc., MabVax Therapeutics Holdings, Inc., Pfizer Inc., Syros Pharmaceuticals, Inc., Array BioPharma, and Takeda Pharmaceutical Company Limited.