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“I was in a wheelchair, taking 5 liters of oxygen per hour, barely able to breathe continuously, I was dying.” Nichol Miller was the first cancer patient in the world to receive larotrectinib, when she was at a terminal stage of cancer , her life was in jeopardy, but larottinib turned the tide and almost cleared the tumor from her body. Larotrectinib is a foreign “anticancer drug” and a broad-spectrum anticancer drug that is not limited to cancer. It was approved by the US FDA in 2018 and has been approved by more than 40 countries. In April of this year, China also approved larottinib for the domestic market, for the treatment of adult and pediatric patients with solid tumors carrying NTRK fusion gene, regardless of the cancer region, which means Cancer patients have greater hope for survival.

1. The first patient in the world to receive larotrectinib from the advanced stage of cancer to the disappearance of the tumor

< p>In the fall of 2014, Nichol Miller was diagnosed with the highest grade soft tissue sarcoma in the leg, followed by systemic chemotherapy and surgery, but it still failed to stop the spread of the cancer. At that time, Miller also had a tumor in her lungs and could not breathe on her own. She had to take oxygen every day to continue her life. At the same time, she had to endure very severe pain, and despair emerged in her heart.

Luckily, Miller’s doctors didn’t give up on her, and the attending physician performed a comprehensive genome sequencing of her and discovered a rare mutation in her tumor — the NTRK gene fusion.

At that time, there was a clinical trial for this mutation, that is, larotrectinib treatment, but everything was unknown at the beginning of the trial. Miller, who got the news, decided to give it a go, because there was nothing worse than the current situation, and in March 2015, she started treatment.

Unexpectedly, within three days of taking the drug, Miller was able to sit down and speak completely, and a month later she could breathe completely. It’s normal, at six weeks, she can ride a horse. Everything was improving, and Miller’s final CT showed that the dense tumors in both lungs had disappeared. In other words, Miller gradually recovered after treatment with larotrectinib.

What is the larotrectinib therapy that works so well? I believe that many people have never heard of this. Next, Xiao Ai will take you to learn more about larottinib therapy.

Second, Larotrectinib: not limited to cancer, only recognizes gene mutations, effective against a variety of cancers

Larotrectinib is a highly The specific TRK inhibitor can bind to the protein product encoded by the NTRK fusion gene with high selectivity, block the activation and transmission of downstream signaling pathways, so as to inhibit the continuous proliferation of cancer cells with these mutations. , play an anticancer effect.

The currently recognized advantages of larotrectinib therapy are two: 1. Not limited to cancer types, only gene mutations are recognized

TRK gene fusions are many One of the “bane” of tumor pathogenesis, tumor is the fusion of NTRK and another unrelated gene to form abnormal TRK protein, also known as TRK fusion protein.

These proteins have conformational activation and will continue to over-activate the downstream of the cell transduction pathway in vivo. No matter where the tumor occurs, these TRK proteins can be fused into it to promote the spread and growth of the tumor. . Unlike other driver genes, TRK fusion genes are widely distributed in various tumor types, and the patients with the disease are not limited to a certain tumor.

Larotrectinib can provide precise treatment for tumor patients carrying TRK fusion protein, benefiting more cancer patients.

2. Fast, efficient and durable responses

ASCO June 6, 2021 At the congress, an analysis of four different studies confirmed that larotrectinib has a strong, long-term clinical profile for all cancers of all ages.

Across all NTRK fusion cancer patients, regardless of tumor type and age, the expanded pooled data of 206 evaluable admitted, pediatric cancer patients showed an objective response rate of 75% %. And the average duration of remission reached more than 4 years (49.3 months). In addition, the data does not cover just one cancer, but 21 different cancer types.

Can new drugs with such good efficacy really cure all cancers? Should cancer patients no longer have to worry about no cure? 3. Can the anticancer drug larottinib cure all cancers? Zhang Xiaodong, chief physician of Peking University Cancer Hospital, said that larotrectinib is only suitable for patients with NTRK gene fusion mutations. The population in China is relatively small, and some cancer types account for less than 1%, and some cancer types are 1-2 %between. For patients in our country, very little can be used.

In order to use larotrectinib, accurate genetic testing is required, and it cannot be used blindly. During the detection process, the patient needs to take a part of the tumor tissue for RNA sequencing, by detecting whether it is NTRL fusion, if it is fusion, the treatment efficiency is very high. But if it is not integrated, it is basically ineffective after the drug.

And larotrectinib also has certain side effects. During the treatment of larotrectinib, some patients will experience leukopenia, decreased neutrophil count, vomiting, anemia and weight gain and other side effects; some patients also experienced side effects such as nausea, diarrhea, constipation, dyspnea. Also, larotrectinib therapy is not recommended for pregnant and breastfeeding women, as it may cause harm to a developing fetus or newborn.

To put it simply, larotrectinib is not suitable for everyone. It can be effective under the premise of being suitable, but it is also “worthless” and has no therapeutic effect when it is not suitable. Right mindset. There is also a question that everyone is most concerned about. Is the price of such a good anticancer drug prohibitive?

Fourth, the most concerned question: Is the price affordable to ordinary people? Currently, the domestic price of larotrectinib is unknown, but we can refer to the foreign price. The purchase price of 100mg oral capsule is US$36,213.91 per month and US$434,566.92 per year (about 2.8 million RMB) /strong>. For most patients, this price is “astronomical”. The so-called “anti-cancer magic drug” cannot be used for all cancer patients. It is necessary to have a correct attitude and do not blindly follow. Under the premise that suits you, it is the “last word” to choose your own treatment method. Reference:

[1] Luo Xiangchong, Liu Jingjing, Li Gaofeng. Research progress of larotrectinib in the treatment of NTRK gene fusion-positive cancer patients [J]. China Oncology Clinic, 2019, 46(11): 575-580.

[2]Anti-cancer “new drug” is not a “magic drug”, and broad-spectrum anticancer drugs should not be superstitious. China Net.2018.12.5

[3]” Larotrectinib, a targeted drug for “all types of tumors”, was approved in China, and the domestic tumor precision treatment is now at the forefront. Hope — larotrectinib.