New breakthrough in cancer treatment: new gene drugs are expected to be approved by the FDA
Cancer has always been one of the diseases that humans hope to learn. In recent years, with the continuous development of science and technology, many scientists believe that genes may be an effective breakthrough in cancer treatment, and gradually carry out in-depth research on gene anti-cancer.
Loxo Oncology, a pharmaceutical company, is working on a broad-spectrum anti-tumor drug that requires comprehensive genetic testing to understand the molecular typing of tumors in order to develop a treatment plan. However, comprehensive genetic testing is expensive and cannot be covered by medical insurance, requiring patients to pay for themselves.
The goal of a small company called Loxo Oncology is to cure every tumor patient, including rare mutations in genetic material. The key to treatment is to identify genetic mutations that occur in the patient.
The core drug larotrectinib, which the company is developing, is a broad-spectrum antitumor drug intended for use in tumor patients with mutations in all tropomyosin receptor kinase (TRK), not just tumors at a specific anatomical location. The drug will be put on the market as early as next year, which is a welcome news for some cancer patients.
In 1982, scientists first discovered that the TRK gene is the chief culprit of colon cancer, and its mutations can lead to developmental defects and certain types of neuronal cell death. In 2013 and 2014, scientists discovered the presence of TRK mutations in breast, lung and skin cancers. It is now believed that TRK gene mutations are widely distributed in a variety of tumors, affecting people of all ages, and have nothing to do with the genetic background of the tumor.
However, the frequency of mutations in the TRK gene is very low, and the limitations of the comprehensive application of genetic testing in clinical applications, the launch of larotrectinib will face: how many patients will be willing to spend high testing costs to understand the genetic characteristics of their tumors, and then Taking drugs to treat tumor problems.
In the spring of 2015, the medical center with Loxo Company screened 55 cancer patients with mutations in the TRK gene by means of genetic testing. These patients, including adults and children, have as many as 17 types of tumors.
Loxo used these patients as subjects to conduct the first round of clinical trials of larotrectinib. The round of tests took two years and tracked and tested 15 indicators. The results of the trial showed that 38 patients had tumor shrinkage after treatment with larotrectinib, and the effective rate was 76%.
At present, there are still 30 patients who continue to take larotrectinib, their tumors have completely lost their invasive ability, and no other types of tumors have occurred in the 12 months after receiving treatment.
Recently, at the American Clinical Oncology Annual Meeting in Chicago, Loxo announced their inspiring clinical trial results. Although the number of participants is small, larotrectinib has won widespread attention.
The first drug approved by the US Food and Drug Administration (FDA) for specific gene features is Merck's PD-1 inhibitor Keytruda, which is used to treat solid tumors in adults and children. This is also the first time the FDA has approved the use of biomarkers to treat tumors, regardless of where the tumor originated in the body.
For example, Keytruda can be used for the treatment of Lynch syndrome. Lynch syndrome is a genetic disease that increases the risk of developing other tumors (especially colorectal cancer), mainly caused by mutations in genes repaired by DNA mismatches. Genetic testing of families with a history of Lynch syndrome and treatment with Keytruda often results in significant therapeutic effects.
Loxo's larotrectinib may be the next FDA-approved drug for a specific gene-mutant tumor. Loxo plans to get FDA approval at the end of this year to put larotrectinib into clinical treatment.
Although it is highly likely that FDA approval will be obtained, the launch of larotrectinib is subject to cost and scope of application. Before clinical use of larotrectinib, doctors first need to know the genetic characteristics of the patient's tumor.
Dan Rhodes, CEO of Strata Oncology, a tumor genome sequencing company, said that gene sequencing is still a bottleneck in the promotion of oncology drugs targeting gene mutations. Strata Oncology will provide genetic testing for participants to help develop a more appropriate treatment plan.
But biotechnologies such as Foundation Medicine, Caris Life Sciences, and Cancer Genetics provide genetic testing that is expensive, costing $5,000 or more.
According to the Cambridge-Massachusets Genetic Testing Foundation, only 12% of patients with advanced metastatic cancer are willing to undergo comprehensive genetic testing to screen for possible genetic mutations. The probability of mutation in the TRK gene is very low, and the ratio is approximately 1:1500 to 1:5000.
Therefore, Robert Doebele, an oncologist at Colorado State University and a researcher on mutations in the TRK gene, fears that many doctors will not recommend a comprehensive genetic test for patients with low mutation rates and high prices. Moreover, considering the rarity of the TRK gene mutation, it is unclear whether the medical insurance can reimburse the cost of this part of the genetic test.
Bilenker said that once larotrectinib is approved, the company's future business strategy is to increase publicity and increase the visibility of TRK mutations in the public. At the same time, the company also needs to develop a more comprehensive genetic testing program, screening a variety of gene mutations including TRK, guiding oncologists how to respond to positive test results, so that more doctors are convinced of the therapeutic effects of larotrectinib.
Bilenker hopes that the clinically significant therapeutic effect of larotrectinib will prompt medical insurance to reimburse the cost of genetic testing, so that there will be a good precedent for the development of targeted gene mutations in the future.
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