A CAR-T Success Story in China: A Beacon of Hope for Acute Myeloid Leukemia(AML) Patients

A CAR-T Success Story in China: A Beacon of Hope for Acute Myeloid Leukemia(AML) Patients

patient story

#CAR-T #AcuteMyeloidLeukemia #AML #ChinaHealthcare #patientstory #CD33

In the field of cancer treatment, China’s medical advancements have brought new therapeutic opportunities for patients, especially with CAR-T cell therapy. Although CAR-T has achieved significant success in treating lymphoid tumors, its application in myeloid malignancies, particularly acute myeloid leukemia (AML), has been challenging. However, a recent successful case of CD33 CAR-T therapy in China has ignited hope for patients with refractory relapsed AML.

#### Patient Story

The patient is a 25-year-old Chinese male who initially sought medical attention due to dizziness and fatigue. He was eventually diagnosed with high-risk AML associated with the DEK::CAN fusion gene, which has a very poor prognosis. Despite some initial effectiveness of chemotherapy and undergoing allogeneic hematopoietic stem cell transplantation (Allo-HSCT), the disease relapsed six months later.

After multiple attempts with targeted therapies and donor cell infusions, the results remained unsatisfactory. Just when traditional treatments seemed ineffective and hope was dwindling, the patient sought out my Advanced Medicine in China team.

#### Breakthrough Treatment: China’s CD33 CAR-T

The patient received a consultation and treatment from the medical experts of my Advanced Medicine in China team. After admission and initial preparations, he underwent CD33 CAR-T therapy, an innovative treatment specifically targeting myeloid tumors. After three days of chemotherapy conditioning, the CAR-T cells were infused on day 11. Although there was a mild fever during this period, the patient experienced no significant discomfort. Subsequently, the bone marrow examination revealed astonishing news: leukemia cells had completely disappeared, and the DEK::CAN fusion gene was nearly undetectable, indicating that the disease was in a state of complete remission.

Despite experiencing a lung infection during treatment, the patient’s condition improved after antibiotic therapy. He did not suffer from severe side effects such as persistent cytopenia, and his blood parameters returned to normal. He was ultimately discharged smoothly.

#### Sustained Remission and a Bright Future

Two months after receiving CD33 CAR-T cell therapy, the patient remains in a state of complete molecular remission, with normal blood counts and a significant improvement in quality of life. This case demonstrates that CAR-T therapy not only successfully controlled refractory relapsed AML but also significantly enhanced the patient’s chances of survival and quality of life.

#### A New Chapter for CAR-T Treatment of AML in China

This case highlights China’s innovative capabilities in the field of CAR-T therapy, particularly in the treatment of myeloid malignancies. For AML patients like this young man who have faced multiple treatment failures, China’s CAR-T therapy undoubtedly brings new hope.

Although global CAR-T therapies for myeloid malignancies are still in their infancy, this groundbreaking advancement in China indicates that CAR-T therapy is poised to become a crucial method for treating refractory relapsed AML in the future. As research deepens and technologies mature, this therapy may save more patients in dire circumstances.

China’s leading position in CAR-T treatment is bringing new hope to patients worldwide. For all AML patients, this story proves that the path to a brighter future has been paved, and for many, it begins with China’s CAR-T therapy.

To assess whether the condition is suitable for CAR-T therapy, you can submit pathology reports, treatment history, and discharge summaries to the Medical Department of <Advanced Medicine in China> for preliminary evaluation!

WhatsApp: Https://wa.me/+8613717959070

Email: doctor.huang@globecancer.com

#CancerTreatment #InnovativeTherapy #PatientSuccess #MedicalAdvancements #HopeInHealthcare #Immunotherapy

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5 months ago CAR-T

Sustained 7-Month CR, Liver Extramedullary Disease Disappearance: Equecabtagene Autoleucel Brings High-Quality Survival Hope to Ultra-High-Risk RRMM Patients with Extramedullary Disease

### Sustained 7-Month CR, Liver Extramedullary Disease Disappearance: Equecabtagene Autoleucel Brings High-Quality Survival Hope to Ultra-High-Risk RRMM Patients with Extramedullary Disease

RRMM Patients

In December 2019, a man in his 50s was diagnosed with multiple myeloma (MM) at a hospital in Shenzhen, China. The patient had high-risk cytogenetic abnormalities (17p deletion) and underwent various treatments over more than three years, including proteasome inhibitors (PIs), immunomodulatory drugs (IMiDs), and CD38 monoclonal antibodies. Despite these treatments, the patient experienced a second relapse. This relapse was characterized by a P53 gene mutation and liver extramedullary invasion, classifying it as an ultra-high-risk situation. Although the patient initially refused an autologous stem cell transplant (ASCT), he was eventually persuaded to collect sufficient stem cells. However, due to concerns about the side effects of high-dose chemotherapy, he opted for CAR-T therapy.

### Personalized Treatment: From ASCT to CAR-T Therapy

Under the care of Professor Chen’s team at the hospital, the patient was evaluated for CAR-T therapy. Despite the risks, he underwent the treatment and showed significant improvement. As of March 2024, he has maintained a complete response (CR) for seven months, with PET/CT scans indicating the disappearance of liver extramedullary disease.

#### Patient Background

– **Age**: 54

– **Initial Symptoms**: Back pain after exercise

– **Diagnosis**: Multiple Myeloma (IgA κ type, DS I A stage, ISS I stage, R-ISS II stage, mSMART high-risk)

### Treatment Journey

– **2019**: Diagnosed with MM; high-risk cytogenetic abnormality (17p deletion)

– **2020-2022**: Treated with PIs, IMiDs, and CD38 monoclonal antibodies

– **Early 2023**: Second relapse with P53 mutation and liver extramedullary disease

– **July 2023**: Stem cell collection for ASCT

– **August 2023**: Initiated CAR-T cell (Equecabtagene Autoleucel) therapy

– **September 2023**: Discharged with strict complete response (sCR)

– **March 2024**: PET/CT scan confirms sustained complete response (CR) with no liver lesions

### Breakthrough Results and Future Prospects

CAR-T therapy has emerged as a promising treatment for relapsed and refractory multiple myeloma (RRMM), especially for patients resistant to conventional therapies. This case highlights the importance of personalized treatment plans, considering disease factors, previous treatment outcomes, and patient preferences. Equecabtagene Autoleucel (IKEA-Lunsay) CAR-T therapy has given this high-risk patient a new lease on life and holds the potential to offer hope to many more MM patients in China.

By showcasing such success stories, we aim to raise awareness about the advancements in CAR-T therapy and its significant impact on improving the quality of life for MM patients. This breakthrough not only marks a significant achievement for the hospital but also signals a new era of hope for countless MM patients.

🎉🎉To assess whether the condition is suitable for CAR-T or clinic therapy, you can submit Advanced Medicine in China  for preliminary evaluation!

WhatsApp: +8613717959070

Email: doctor.huang@globecancer.com

#MedicalBreakthrough #CARTTherapy #MultipleMyeloma #CancerTreatment #PatientSuccess #HealthcareInnovation #CancerResearch #MMTreatment #LifeSavingTherapies #MedicalAdvancements #HealthcareHeroes #InnovativeMedicine #CancerCare #HopeForMM #CancerAwareness

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6 months ago Solid tumor

Tumors Vanish After One Year of Treatment! The First Advanced Gastric Cancer Patient to Receive the New Cancer Vaccine Achieves Lasting Remission

**Tumors Vanish After One Year of Treatment! The First Advanced #GastricCancer Patient to Receive the New #Cancer Vaccine Achieves Lasting Remission**

Cancer Vaccine

Mrs. Liu, who was battling advanced gastric cancer, never imagined that one year ago, she was on the brink of death due to chemotherapy, with her disease progressing rapidly and cancer spreading throughout her body… Later, she won the “lottery” of her life. She received one of the world’s most advanced immunotherapies—cancer vaccines—in Fujian. One year later, all her cancer lesions have completely disappeared! Her successful case was even published in a prominent international journal, a Nature sub-journal, causing a huge sensation.

**First Advanced Gastric Cancer Patient to Receive the New Cancer Vaccine Achieves Lasting Remission! Tumors Vanish After One Year of Treatment**

In July 2017, 66-year-old Mrs. Liu was unfortunately diagnosed with stage IV gastric cancer. After surgery, she underwent FOLFOX chemotherapy to eliminate residual cancer cells, and everything seemed to be going well. Unfortunately, just eight months later, Mrs. Liu’s cancer recurred, and multiple lesions were found not only in her stomach but also in her peritoneum and lymph nodes, indicating that the cancer had rapidly spread throughout her body. Mrs. Liu had to undergo chemotherapy again. However, this time, her body could no longer withstand the side effects. After two cycles of Nab-paclitaxel, she was on the brink of death due to severe side effects and had to stop chemotherapy.

When she and her family were in despair, the doctors brought them good news. A nationally renowned cancer center was conducting a clinical trial of a cancer vaccine. Remarkably, this new type of cancer vaccine was a hot topic of research worldwide and had achieved significant breakthroughs.

The new cancer vaccine is a personalized, neoantigen-loaded monocyte-derived dendritic cell vaccine, known as Neo-MoDC. This vaccine fully mobilizes the immune system: dendritic cells (DCs) recognize cancer antigens, present antigens to activate T cells, promote T cell enrichment, and mobilize T cells to eliminate cancer cells, ultimately launching a series of precise and powerful attacks on cancer cells throughout the body.

Mrs. Liu decided to participate in the clinical trial of this new therapy.

This new therapy is actually much more complex than described above. Doctors first performed whole-exome sequencing on her tumor tissue samples, identifying 1,096 somatic non-synonymous mutations. They then determined her HLA (human leukocyte antigen) types as HLA-A02:07, HLA-A24:02, HLA-B40:01, HLA-B46:01, HLA-C01:02, and HLA-C03:04. Based on the binding affinity of these predicted mutant peptides with the patient’s HLA I class alleles, eight soluble mutant epitopes with the highest affinity were identified to prepare a personalized neoantigen dendritic cell cancer vaccine specifically targeting her cancer cells.

Mrs. Liu successfully received two months of subcutaneous Neo-MoDC vaccine injections to activate the T cell anti-tumor immune response. Researchers confirmed that the Neo-MoDC vaccine successfully induced a neoantigen-specific T cell response. For better therapeutic results, from day 65, Mrs. Liu received nivolumab (PD-1) treatment every 14 days.

An unexpected surprise occurred.

Five days later, Mrs. Liu’s CA-125 level rapidly dropped from 596 to 64 U/ml;

Two weeks later, malignant ascites disappeared, and clinical imaging showed a 30% reduction in the volume of metastatic supraclavicular lymph nodes;

Two months later, all metastatic lymph nodes reduced to less than 1 cm, and evaluations showed complete remission (CR) in the lymph nodes, with a 20% reduction in the longest diameter of ovarian metastatic lesions.

On the 231st day after the initial vaccination, imaging showed complete regression of all lesions;

On the 389th day of combined treatment, a CT scan showed that the ovarian metastatic lesions had also completely disappeared;

Subsequent CT scans confirmed that by the time of the paper’s publication (October 2021), complete regression had lasted for over 25 months.

This result indicates that the neoantigen vaccine Neo-MoDC and PD-1 in combination therapy may enhance each other’s efficacy, achieving better treatment outcomes, and providing a new immunotherapy combination for metastatic gastric cancer and other tumor types.

Mrs. Liu said that after the failure of chemotherapy, she was in despair, counting down the days of her life. She couldn’t imagine that she would have the opportunity to receive such cutting-edge therapy in China, let alone believe that she would become the fortunate one in this milestone research, successfully defeating cancer and returning to normal life!

In June 2022, this study was published in a prominent international journal, a Nature sub-journal, marking the first known case of complete and lasting tumor regression in gastric cancer through neoantigen-based DC vaccine and PD-1 therapy, which is of milestone significance!

**Cancer Vaccines Will Ultimately Become a Sword Against Cancer!**

Throughout the long history of humanity, various vaccines have been developed to combat devastating diseases, leading to the eradication of deadly diseases like smallpox and poliomyelitis. Similarly, the medical community is actively developing various vaccines to fight cancer, hoping to awaken our immune system to recognize and kill cancer cells upon injection, and to generate immune memory responses that protect us continuously, attacking any emerging cancer cells before they form tumors. This vision will eventually become a reality!

Immunotherapy is hailed as the hope for conquering cancer. Whether it’s the currently approved PD-1 immune checkpoint inhibitor therapy for various cancers, adoptive cell immunotherapy represented by CAR-T, or numerous cancer vaccines under development, they represent just the tip of the iceberg in immunology. Researchers are uncovering more information from this iceberg, learning how to control immune responses, and using these therapeutic approaches in clinical practice to benefit patients, giving us increasing hope for survival. The hope for conquering cancer may lie within these advances. We look forward to more breakthroughs in immunotherapy, bringing more miracles to cancer patients.

To assess whether the condition is suitable for New Cancer Vaccine or clinic therapy, you can submit Advanced Medicine in China for preliminary evaluation!

WhatsApp: 137 1795 9070

Email: doctor.huang@globecancer.com

#CancerVaccine #StomachCancer #Immunotherapy #CancerResearch #MedicalBreakthrough #CancerTreatment #HealthcareInnovation #PatientSuccess #Oncology #MedicalAdvancements

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6 months ago CAR-T

One Year After Approval: How Effective is China’s First BCMA-Targeted CAR-T Therapy in Treating Multiple Myeloma?

### One Year After Approval: How Effective is China’s First BCMA-Targeted CAR-T Therapy in Treating Multiple Myeloma?

Multiple Myeloma

“In the past 20 years, the treatment of multiple myeloma has advanced rapidly, thanks in large part to the swift progress in drug development. Over the past year, China’s first independently developed and the world’s first fully human BCMA-targeted chimeric antigen receptor T-cell (CAR-T) therapy was approved. In our real-world clinical applications, we have treated over 20 patients with an overall effectiveness rate of nearly 100%.”

On June 30, 2024, exactly one year after the approval of China’s first BCMA-targeted CAR-T therapy (Iquilonsen Injection), and also the world’s first fully human BCMA-targeted CAR-T therapy, how effective has the treatment been for patients? Professor Qiu Lugui, Director of the Lymphoma Treatment Center at the Chinese Academy of Medical Sciences Hematology Hospital, was interviewed by the People’s Daily Health Client.

“Multiple myeloma has a slow onset and early stages often show no obvious symptoms, making it easy to misdiagnose. To date, once diagnosed, the vast majority of patients experience one or more relapses, entering a refractory state, which is an incurable disease,” Professor Qiu Lugui told the People’s Daily Health Client.

Professor Qiu explained that current drugs for treating multiple myeloma fall into three categories: immunomodulators, proteasome inhibitors, and CD38 monoclonal antibodies. The indications for these drugs have gradually moved from refractory cases to frontline treatments, transforming multiple myeloma from a deadly disease with a median survival of around three years to a relatively controllable malignant hematological tumor with a median survival of 10 years or more after systematic multi-drug therapy.

CAR-T cell therapy is a cutting-edge technology for treating malignant hematological tumors. China’s independently developed fully human BCMA-targeted CAR-T drug (Iquilonsen Injection) is designed for multiple myeloma patients who have relapsed or whose disease remains uncontrolled despite traditional treatments including proteasome inhibitors and immunomodulators.

One particularly memorable case for Professor Qiu was a 70-year-old patient. “At that time, the patient was extremely weak and had already undergone all available treatments, including two types of immunomodulators, two types of proteasome inhibitors, CD38 monoclonal antibodies, and intensive chemotherapy, with no other effective options left,” recalled Professor Qiu. “However, the patient had a strong desire to live. Seeing his eager eyes, we couldn’t remain indifferent.”

“After confirming with the patient, we decided to proceed with the fully human BCMA-targeted CAR-T Iquilonsen therapy. One month after the treatment, the first evaluation showed complete remission. To date, the patient remains in complete remission,” said Professor Qiu.

“In real-world applications, we have treated over 20 patients with an overall effectiveness rate approaching 100%. However, due to the high cost of the drug and the fact that it is not covered by medical insurance, making the drug accessible remains a challenge,” Professor Qiu told the People’s Daily Health Client. “Currently, there are two methods to address this issue: one is to meet the needs of economically disadvantaged patients through commercial insurance; the other is to meet the needs of patients who meet the criteria for inclusion in CAR-T clinical research.”

“Additionally, in the year since the approval of China’s first CAR-T therapy for treating multiple myeloma, not only domestic patients but also patients from Europe, Asia-Pacific, Africa, and other regions have come to China for CAR-T treatment. Overall treatment driven by dynamic prognostic stratification will be the future path to cure for multiple myeloma patients both in China and globally,” said Professor Qiu.

#MultipleMyeloma #CARTTherapy #CancerTreatment #MedicalBreakthrough #BCMATargeted #ChinaHealthcare #InnovativeMedicine #PatientSuccess #GlobalHealth #Hematology #Immunotherapy