EN 
FR | Discovery | Pre-Clinical | Phase 1 |
|---|
Pipeline Progression
CAR-HSC
Pre-B Acute Lymphoblastic Leukemia
CAR-HSC
Rhabdomyosarcoma
Our immunotherapy development program in childhood cancers
Precursor-B Acute Lymphoblastic Leukemia
(Pre-B ALL)
Pre-B ALL is the most common malignancy in childhood with an overall cure rate of approximately 85%. However, relapsed ALL have an event-free survival of only 47%. CAR-T cells were shown efficient in ALL, but as much as 30 to 60% of patients relapse mainly because of the lack of CAR-T cells persistence or antigen loss (CD19).
Also, CRS occurs in 75% to 100% of children treated by CAR-T cells, with between 16% to 46% of patient needing hospitalisation. There is, therefore, a dire need to improve CAR-therapy for children with ALL.
Our immunotherapy development program in childhood cancers
Precursor-B Acute Lymphoblastic Leukemia
(Pre-B ALL)
Pre-B ALL is the most common malignancy in childhood with an overall cure rate of approximately 85%. However, relapsed ALL have an event-free survival of only 47%. CAR-T cells were shown efficient in ALL, but as much as 30 to 60% of patients relapse mainly because of the lack of CAR-T cells persistence or antigen loss (CD19).
Also, CRS occurs in 75% to 100% of children treated by CAR-T cells, with between 16% to 46% of patient needing hospitalisation. There is, therefore, a dire need to improve CAR-therapy for children with ALL.
Rhabdomyosarcoma (RMS)
Although rare (4.3 per 106 <20 years old/year), RMS is the most common soft-tissue sarcoma in childhood. Although progress in treatment has led to a remission rate of ~ 60% of pediatric cases, high-risk RMS patients still have a poor prognosis, and survivors have lifetime consequences from the treatments.
There has been no significant progress in the treatment of this disease in the last 5 decades. Hence, efficient immunotherapy for RMS needs to be developed, and stem-cell based CAR therapy represents a great hope.
Rhabdomyosarcoma (RMS)
Although rare (4.3 per 106 <20 years old/year), RMS is the most common soft-tissue sarcoma in childhood. Although progress in treatment has led to a remission rate of ~ 60% of pediatric cases, high-risk RMS patients still have a poor prognosis, and survivors have lifetime consequences from the treatments.
There has been no significant progress in the treatment of this disease in the last 5 decades. Hence, efficient immunotherapy for RMS needs to be developed, and stem-cell based CAR therapy represents a great hope.
1 The American Childhood Cancer Organization 2022, accessed 5 April 2022, <https://www.acco.org/>
2CureSearch for Children’s Cancer 2022, accessed 12 April 2022, <https://curesearch.org/>
