The Rise of CAR-T Therapy in Leukemia Treatment: A Promising Future

Since the approval of the first CAR-T (Chimeric Antigen Receptor T-cell) therapy in 2017, the market for CAR-T products has been expanding rapidly. While CAR-T therapies have demonstrated remarkable efficacy, their high cost has limited access for a majority of patients. However, in the past two years, several domestically developed CAR-T products have been approved and launched, raising hopes for a future where CAR-T therapy becomes more affordable.

The First Domestic CAR-T Product for Leukemia Treatment:
Recently, according to information from the National Medical Products Administration (NMPA), the CAR-T product developed by Genetron Health, called Najiorense Injection (formerly known as Hejilunsei), has been approved for marketing. It is indicated for the treatment of adult relapsed or refractory B-cell acute lymphoblastic leukemia (r/rB-ALL). Currently, Najiorense Injection has also received Investigational New Drug (IND) approval from the U.S. Food and Drug Administration (FDA) for the treatment of adult B-ALL.

Leukemia Statistics:
The incidence of leukemia in China is approximately 6.21 per 100,000 individuals per year, while in the United States, it is around 12.9 per 100,000 individuals per year. Acute lymphoblastic leukemia (ALL) accounts for about 15% of all leukemia cases, with B-cell ALL (B-ALL) representing approximately 75% of ALL cases.

High Relapse Rate in Adult B-ALL:
In adult B-ALL, the relapse rate after initial treatment is high, with approximately 60% to 70% of patients experiencing relapse or becoming refractory (r/rB-ALL). The prognosis for adult r/rB-ALL patients is extremely poor under traditional treatment methods, such as chemotherapy, with a median survival of only 2 to 6 months. Over the past 30 years, there has been no significant improvement in survival rates. Due to the lack of effective treatment options, there is a significant unmet clinical need for the treatment of r/rB-ALL in China and globally.

Emerging Treatment Options and CAR-T Therapy:
In recent years, some new treatment options have emerged, including Inotuzumab ozogamicin and Blinatumomab, which have significantly improved progression-free survival (PFS) and overall survival (OS) in r/rB-ALL. Early-line use of these therapies has shown greater benefits. However, long-term remission rates are still inadequate without allogeneic hematopoietic stem cell transplantation, and there remains an unmet need for the treatment of r/rALL, especially in patients who experience their first relapse.

Unique Features of CAR-T Therapy:
Compared to traditional drugs and emerging therapies, CAR-T cells possess unique characteristics. The inherent specificity of CARs is unmatched by small-molecule drugs currently available. CAR-T cells rely on the cytotoxicity of T cells and utilize this highly efficient endogenous pathway. As a type of living cell therapy, CAR-T cells have the additional advantage that even a single CAR-T cell can undergo exponential expansion, leading to the killing of hundreds or even thousands of target cells. After the clearance of target cells (when the antigen is no longer present), CAR-T cells can persist and patrol the body for more than a decade, thereby preventing disease relapse.

Najiorense Injection: A Breakthrough in CAR-T Therapy:
According to Genetron Health, Najiorense Injection is a CD19-targeted CAR-T cell therapy product with a unique CD19scFv (HI19a) structure and internationally leading manufacturing processes. In registration clinical studies for adult r/rB-ALL, the success rate of production reached 100%.

Najiorense Injection is not only the first domestically developed CAR-T product targeting CD19 but also the first CAR-T cell therapy product for leukemia treatment in China. It offers deep and durable remission for patients and is expected to transform the treatment landscape for adult r/rB-ALL patients in China.

The approval and launch of domestically developed CAR-T therapies, such as Najiorense Injection, signify a significant step forward in the treatment of leukemia, particularly in adult r/rB-ALL patients. With its unique features and the potential for improved affordability, CAR-T therapy holds great promise for the future of cancer treatment. As more innovative therapies emerge and manufacturing processes advance, the vision of making CAR-T therapy more accessible to a larger patient population may become a reality.