What is CML?
The so-called chronic myeloid leukemia (CML) is a common name for chronic myeloid leukemia, accounting for 15% of adult leukemia in China. The patients in China are younger than those in western countries, mostly aged 45-50. The basic manifestations of CML in the early stage are dizziness, fatigue, abdominal distension and weight loss. The most obvious change is pale and bloodless. If you do B-ultrasound, you can see that the spleen is getting bigger. The disease develops very slowly at first, so it is called chronic leukemia. In the first five years, the patient didn't have too many problems, but after five years, it will suddenly accelerate and the patient will die soon.
The cause of CML was discovered by cancer research experts long ago. It was because chromosomes 9 and 22 were broken and then white blood cells were repaired that Zhang Guan Dai Li, because it was first discovered in Philadelphia, USA, called this strange new chromosome in medicine.
Unfortunately, a fusion gene, bcr gene +abl gene, was produced in this error repair, and the fusion protein (Bcr-Abl) produced by this fusion gene began to push white blood cells to a state of chronic uncontrolled proliferation again and again.
Illustration: Professor Norwell who discovered the mutant chromosome of CML.
Medicine for treating chronic myeloid leukemia
After finding the cause, it took the researchers 465,438+0 years off and on, and finally found a way to inhibit the function of this new gene, which is the sensational imatinib (Gleevec).
200 1, Gleevec was approved for listing by FDA. In 2009, Professor Brian J. Druker and others who participated in Gleevec's research and development won the Lasker Medical Research Award, which is known as the Nobel Prize in Medical Prediction. Why? Because of Gleevec, the deadly CML has been transformed into a chronic disease that can make patients live longer, a chronic disease similar to diabetes and hypertension.
Illustration: Professor Brian J. Drucker won the Lasker Prize.
Before Gleevec was born, traditional chemotherapy drugs could only make less than 30% patients live to five years. However, Gleevec increased the rate from 30% to 89%, and after five years, 98% of patients still achieved complete remission in hematology. This incredible percentage made Gleevec quickly enter the list of essential drugs of the World Health Organization. You know, the so-called essential drugs mean "the most effective, the safest and the most needed". Only drugs that meet these three harsh conditions can enter the World Health Organization's list of essential drugs.
However, Gleevec also has the disadvantage that patients can't stop taking drugs. In addition, after patients take it for too long, there are always some unlucky patients who will have anti-cancer problems, which will be miserable. Therefore, the progress of medicine cannot be stopped. Fortunately, the second generation of drugs has come out, which can be used directly or after the occurrence of gleevec resistance. Moreover, the good news is that after several years of continuous use of the first and second generation drugs, even the treatment remission (TFR) cannot be achieved, that is, the curative effect can still be maintained after stopping the drug. This will greatly reduce the pressure of patients or the government on drug costs.