Developing New Agents for Treatment of Childhood Cancer: Challenges and Opportunities for Preclinical Testing
Developing new therapeutics for the treatment of childhood cancer has challenges not usually associated with adult malignancies. Firstly, childhood cancer is rare, with approximately 12,500 new diagnoses annually in the U.S. in children 18 years or younger. With current multimodality treatments, the...
| Main Authors: | , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
MDPI AG
2021-04-01
|
| Series: | Journal of Clinical Medicine |
| Subjects: | |
| Online Access: | https://www.mdpi.com/2077-0383/10/7/1504 |
| _version_ | 1797538795652907008 |
|---|---|
| author | Samson Ghilu Raushan T. Kurmasheva Peter J. Houghton |
| author_facet | Samson Ghilu Raushan T. Kurmasheva Peter J. Houghton |
| author_sort | Samson Ghilu |
| collection | DOAJ |
| description | Developing new therapeutics for the treatment of childhood cancer has challenges not usually associated with adult malignancies. Firstly, childhood cancer is rare, with approximately 12,500 new diagnoses annually in the U.S. in children 18 years or younger. With current multimodality treatments, the 5-year event-free survival exceeds 80%, and 70% of patients achieve long-term “cure”, hence the overall number of patients eligible for experimental drugs is small. Childhood cancer comprises many disease entities, the most frequent being acute lymphoblastic leukemias (25% of cancers) and brain tumors (21%), and each of these comprises multiple molecular subtypes. Hence, the numbers of diagnoses even for the more frequently occurring cancers of childhood are small, and undertaking clinical trials remains a significant challenge. Consequently, development of preclinical models that accurately represent each molecular entity can be valuable in identifying those agents or combinations that warrant clinical evaluation. Further, new regulations under the Research to Accelerate Cures and Equity for Children Act (RACE For Children Act) will change the way in which drugs are developed. Here, we will consider some of the limitations of preclinical models and consider approaches that may improve their ability to translate therapy to clinical trial more accurately. |
| first_indexed | 2024-03-10T12:36:29Z |
| format | Article |
| id | doaj.art-2922349933be40a1b8ddd086ddcd23db |
| institution | Directory Open Access Journal |
| issn | 2077-0383 |
| language | English |
| last_indexed | 2024-03-10T12:36:29Z |
| publishDate | 2021-04-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Journal of Clinical Medicine |
| spelling | doaj.art-2922349933be40a1b8ddd086ddcd23db2023-11-21T14:12:41ZengMDPI AGJournal of Clinical Medicine2077-03832021-04-01107150410.3390/jcm10071504Developing New Agents for Treatment of Childhood Cancer: Challenges and Opportunities for Preclinical TestingSamson Ghilu0Raushan T. Kurmasheva1Peter J. Houghton2Greehey Children’s Cancer Research Institute, 8403, San Antonio, TX 78229, USAGreehey Children’s Cancer Research Institute, 8403, San Antonio, TX 78229, USAGreehey Children’s Cancer Research Institute, 8403, San Antonio, TX 78229, USADeveloping new therapeutics for the treatment of childhood cancer has challenges not usually associated with adult malignancies. Firstly, childhood cancer is rare, with approximately 12,500 new diagnoses annually in the U.S. in children 18 years or younger. With current multimodality treatments, the 5-year event-free survival exceeds 80%, and 70% of patients achieve long-term “cure”, hence the overall number of patients eligible for experimental drugs is small. Childhood cancer comprises many disease entities, the most frequent being acute lymphoblastic leukemias (25% of cancers) and brain tumors (21%), and each of these comprises multiple molecular subtypes. Hence, the numbers of diagnoses even for the more frequently occurring cancers of childhood are small, and undertaking clinical trials remains a significant challenge. Consequently, development of preclinical models that accurately represent each molecular entity can be valuable in identifying those agents or combinations that warrant clinical evaluation. Further, new regulations under the Research to Accelerate Cures and Equity for Children Act (RACE For Children Act) will change the way in which drugs are developed. Here, we will consider some of the limitations of preclinical models and consider approaches that may improve their ability to translate therapy to clinical trial more accurately.https://www.mdpi.com/2077-0383/10/7/1504Patient-derived xenograftspediatric cancerdrug developmentsingle mouse design |
| spellingShingle | Samson Ghilu Raushan T. Kurmasheva Peter J. Houghton Developing New Agents for Treatment of Childhood Cancer: Challenges and Opportunities for Preclinical Testing Journal of Clinical Medicine Patient-derived xenografts pediatric cancer drug development single mouse design |
| title | Developing New Agents for Treatment of Childhood Cancer: Challenges and Opportunities for Preclinical Testing |
| title_full | Developing New Agents for Treatment of Childhood Cancer: Challenges and Opportunities for Preclinical Testing |
| title_fullStr | Developing New Agents for Treatment of Childhood Cancer: Challenges and Opportunities for Preclinical Testing |
| title_full_unstemmed | Developing New Agents for Treatment of Childhood Cancer: Challenges and Opportunities for Preclinical Testing |
| title_short | Developing New Agents for Treatment of Childhood Cancer: Challenges and Opportunities for Preclinical Testing |
| title_sort | developing new agents for treatment of childhood cancer challenges and opportunities for preclinical testing |
| topic | Patient-derived xenografts pediatric cancer drug development single mouse design |
| url | https://www.mdpi.com/2077-0383/10/7/1504 |
| work_keys_str_mv | AT samsonghilu developingnewagentsfortreatmentofchildhoodcancerchallengesandopportunitiesforpreclinicaltesting AT raushantkurmasheva developingnewagentsfortreatmentofchildhoodcancerchallengesandopportunitiesforpreclinicaltesting AT peterjhoughton developingnewagentsfortreatmentofchildhoodcancerchallengesandopportunitiesforpreclinicaltesting |