“Leukemia was once universally regarded around the world as an ‘incurable disease,’ but with advances in technology and treatment strategies, it has become treatable and controllable,” said Zhang Xiaohui, chief physician at Peking University People’s Hospital, at a press conference held by China’s National Health Commission on August 21.
Leukemia is not a single disease, but a broad group of malignant tumors originating in the hematopoietic system. Depending on the speed of disease progression, the type of abnormal cells involved, and genetic and molecular characteristics, leukemia can be divided into multiple types, with substantial differences in treatment and prognosis. Among them, chronic myeloid leukemia (CML) and acute promyelocytic leukemia (APL) are two of the most representative diseases in terms of treatment advances over the past several decades, with Chinese doctors playing an important role in advancing treatments for both diseases.
Zhang Xiaohui particularly noted that China’s medical community developed the “Shanghai regimen” for APL treatment and the “Beijing Protocol” for hematopoietic stem cell transplantation, addressing two major challenges in leukemia treatment. China has also made significant advances in the treatment of CML: with the continued development of targeted drugs, improved drug accessibility, and expanded health insurance coverage, CML is increasingly becoming a chronic disease that can be managed over the long term in China.
CML: From a Deadly Disease to a Chronic Condition
CML is characterized in most patients by the presence of an abnormal BCR::ABL1 fusion gene. A fusion gene can be understood as two segments of genes that should not normally be connected becoming “miswired,” creating an abnormal switch that continuously sends signals for cell proliferation and causes leukemia cells to keep increasing.
One drug used to treat CML is Gleevec, whose generic name is imatinib. Imatinib works by precisely inhibiting this abnormal signaling pathway. It is a landmark drug in the treatment of CML and one of the most important milestones in the development of modern targeted cancer therapy. Unlike conventional chemotherapy, which broadly targets rapidly dividing cells, targeted drugs act on specific molecular abnormalities that cancer cells depend on, allowing them to target cancer cells more precisely.
In the past, long-term treatment with the imported originator version of imatinib in China was prohibitively expensive, placing a heavy financial burden on many families. Subsequently, with the introduction of domestically produced generic drugs, their inclusion in the national health insurance system, and the advancement of centralized pharmaceutical procurement, costs fell significantly. In 2018, domestically produced imatinib passed China’s consistency evaluation for the quality and efficacy of generic drugs. It was subsequently included in the “4+7” volume-based procurement program, a centralized purchasing scheme in which bulk purchasing was used to negotiate lower drug prices, with the selected price falling to about 624 yuan per box (approximately US$87). Based on the commonly used treatment dosage, monthly medication costs fell substantially, while patients’ own financial burden was further reduced by health insurance reimbursement.
For many patients, regular treatment with TKIs (tyrosine kinase inhibitors), which target the abnormal proteins driving leukemia cell growth, together with periodic hematological and molecular monitoring, can keep the disease under long-term control, allowing them to continue working and living normally.
Even if drug resistance develops, there are now more treatment options available. For example, some patients may develop the T315I mutation, rendering commonly used targeted drugs ineffective. In 2021, China’s independently developed third-generation BCR::ABL1 inhibitor olverembatinib was approved, providing a new treatment option for these patients.
CML treatment has therefore moved from the past question of “Is there a drug?” to today’s question of “Which drug is right for the patient’s specific molecular abnormality?”
Two Key Breakthroughs: The Shanghai Regimen and the Beijing Protocol
In the field of leukemia treatment, Chinese doctors have made two particularly notable contributions: the “Shanghai regimen” and the “Beijing Protocol.” The former addressed the question of “How should this type of leukemia be treated?” while the latter addressed the question of “What can be done when a patient needs a transplant but cannot find a suitable donor?”
The “Shanghai regimen” primarily targets APL. In the past, this type of leukemia was extremely dangerous. Patients were prone to severe coagulation disorders, which could lead to both thrombosis and major bleeding, and some patients could die within a short period from complications such as cerebral hemorrhage.
In the 1970s, Professor Zhang Tingdong and others began exploring arsenic-based treatment for APL. In the 1980s, the team led by academician Wang Zhenyi discovered that all-trans retinoic acid (ATRA) could induce abnormal leukemia cells to differentiate and mature. Subsequently, teams led by Chen Zhu and Chen Saijuan further developed treatment strategies combining retinoic acid with arsenic trioxide, gradually establishing what later became widely known as the “Shanghai regimen.”
Its greatest difference from conventional chemotherapy is that it can both induce abnormal leukemia cells to differentiate and mature and directly trigger their apoptosis, while also targeting key molecular abnormalities. It therefore combines the dual effects of differentiation induction and targeted killing. Today, under standardized diagnostic and treatment conditions, the 5-year disease-free survival rate for patients with APL can exceed 90%. The disease has been transformed from one of the most aggressive forms of acute leukemia into one of the most highly curable types of adult leukemia.
The “Beijing Protocol,” meanwhile, primarily addresses the donor challenge in hematopoietic stem cell transplantation. For some patients with high-risk, relapsed, or refractory leukemia, hematopoietic stem cell transplantation remains an important potentially curative treatment. In the past, however, transplantation generally required a high degree of HLA matching between donor and recipient. HLA can be understood as a set of “identity codes” on human cells, and the probability of finding a fully matched donor among biological siblings is only about one in four.
Over two decades, the team led by Huang Xiaojun at Peking University People’s Hospital tackled this problem and pioneered a haploidentical transplantation system. This approach allows half-matched relatives, such as parents and children, to serve as donors; uses granulocyte colony-stimulating factor to prime donor stem cells; and combines this with an immunosuppressive regimen, enabling the haploidentical graft to engraft successfully without causing severe graft-versus-host disease. This means that almost every patient can find a donor among their immediate family members. The team subsequently continued to refine conditioning regimens, immune modulation, and post-transplant management, gradually developing what later became internationally known in the medical community as the “Beijing Protocol.”
New Frontiers: CAR-T and Cancer Vaccines
If targeted drugs are like precisely cutting off the abnormal signals of cancer cells, and hematopoietic stem cell transplantation is like rebuilding a healthy blood-producing factory, then immunotherapy takes a more intelligent approach: rather than directly attacking the tumor, it activates and arms the patient’s own immune system so that it can recognize and eliminate cancer cells again.
The most representative example is CAR-T cell therapy, which can be understood as “equipping immune cells with a navigation radar.” Doctors extract T cells from a patient’s blood and genetically modify them in the laboratory, equipping the T cells with receptors capable of recognizing specific targets on the surface of cancer cells. After being modified and expanded, these T cells are infused back into the patient, where they can precisely seek out and destroy tumor cells.
China’s CAR-T industry has established a relatively complete development pipeline.
In 2023, Eucure Biopharma’s Fucaso was approved for adults with relapsed or refractory B-cell acute lymphoblastic leukemia, providing a new option for patients who continue to relapse after multiple lines of treatment. In November 2025, Chongqing Precision Biotech’s independently developed Fucaso was approved for marketing for patients aged 3–21 with CD19-positive relapsed or refractory B-cell acute lymphoblastic leukemia, becoming the first CAR-T product approved in China specifically for pediatric and adolescent leukemia.
In addition, a number of Chinese companies are also pursuing optimized autologous CAR-T therapies, off-the-shelf CAR-T therapies, and in vivo CAR-T approaches, with the aim of further shortening manufacturing times and reducing costs.
However, CAR-T is not a “one-shot cure.” Some patients may relapse, and cytokine release syndrome may occur, in which massive activation of immune cells causes the release of inflammatory cytokines, triggering reactions such as high fever and hypotension and requiring close monitoring. In addition, the complexity of individualized manufacturing and the high cost are major barriers to broader adoption.
Another more forward-looking avenue of exploration is mRNA cancer vaccines. The core concept is to use mRNA instructions to train the immune system to recognize neoantigens specific to cancer cells. The technology is still in the clinical validation stage and is currently focused primarily on solid tumors, but progress is accelerating. Chinese companies have established a differentiated development pipeline: E-verse Bio’s EVM16 induced neoantigen-specific T-cell responses in 8 of the first 9 participants; Likang Life Sciences’ LK101 has entered a registrational Phase II trial for advanced lung cancer (approved for clinical trials in 2023, making it the first personalized cancer vaccine in China to enter clinical development); and Abogen Biosciences’ ABO2102 has also been approved to enter clinical trials, emerging as an important subsequent player in the field.
Although mRNA vaccines have not yet become a mature treatment for leukemia, their iterative development alongside CAR-T therapy is jointly driving cancer treatment away from “finding one effective drug” and toward “tailoring a treatment strategy for each patient.” Precision cancer treatment is moving from an ideal into reality.
Editor: LQQ
