Rhabdomyosarcoma accounts for about 3%-4% of childhood cancers, and there are about 350 new cases in the United States every year. The disease mainly occurs in teenagers, and half of the patients are under the age of 10. Some people call rhabdomyosarcoma skeletal muscle, as the name suggests. Rhabdomyosarcoma is a malignant tumor caused by incomplete differentiation of tissues containing rhabdomyosarcoma antigen.
At present, the main treatment of rhabdomyosarcoma is surgical resection, but because rhabdomyosarcoma is difficult to be completely removed and easy to metastasize, chemotherapy or radiotherapy is still needed in most cases after operation. At present, one of the main chemotherapy drugs for rhabdomyosarcoma is vincristine, which is the same as vinorelbine wrapped by TLC 178. After being encapsulated by liposome, its toxicity is greatly reduced, and animal experiments show that TLC 178 has a considerable effect on rhabdomyosarcoma, which is an opportunity for TLC 178 to obtain the identification of rare diseases in pediatrics.
The physiological characteristics of children and adults are different, which leads to great differences in pharmacokinetics and pharmacodynamics of children's drugs. However, in the absence of drugs specially developed for children, doctors can only give children less adult drugs according to their age and weight. This unscientific medication method increases the hidden worries and risks of children's medication. Therefore, many countries have promulgated some laws and regulations to encourage the research and development and production of children-specific drugs. "Identification of drugs for rare pediatric diseases" is a legal preference given by the US Food and Drug Administration (FDA), aiming at encouraging the research and development of drugs for rare diseases and dosage forms suitable for children. When the drug certificate is submitted for review, the drug with this logo can apply for the "Pediatric Priority Review Certificate for Rare Diseases", which can shorten the drug review period of more than 10 months to 6 months. Because this kind of priority certificate is not limited to the company's own use, it is often heard that this kind of certificate will be resold at a high price in the market, and the price has soared from $67.5 million in July 200014 to $350 million in August 200015.
TLC 178 is currently conducting phase I/II human clinical trials in Taiwan Province province and the United States. If all patients who were given the highest tolerated dose in this trial showed no obvious safety problems, the dose could be used in the hub trial.
Title: TLC 178, Taiwan Province Microsystems.