Landmark Advances in Cancer Research Highlighted by NCI Director
Key Insights
A clinical trial of dostarlimab showed a 100% complete response rate in patients with mismatch repair deficient, locally advanced rectal cancer (search), lasting over two years.
Immunotherapy made significant strides, including FDA approvals for small cell lung cancer (search) treatments like tarlatamab and durvalumab, and advancements in childhood leukemia with blinatumomab.
Research into cancer cachexia (search) showed promise with ponsegromab reversing weight loss, while studies on ecDNAs revealed their role in treatment resistance and metastasis.
As 2024 concludes, Dr. W. Kimryn Rathmell, Director of the National Cancer Institute (NCI), reflected on the year's significant strides in cancer research, marking her one-year anniversary in the role. The year was characterized by advancements in treating stubborn cancers and unraveling the complexities of cancer biology.
Remarkable Results with Dostarlimab in Rectal Cancer
One of the most notable findings came from a clinical trial of the immunotherapy drug dostarlimab (Jemperli) in patients with locally advanced rectal cancer (search) exhibiting mismatch repair deficiency. The results, presented in June, revealed that all 42 patients treated with dostarlimab for six months achieved a complete response. These responses persisted for over two years in most participants, showcasing the potential of targeted immunotherapy based on tumor markers.
Expanding Impact of Immunotherapy
Immunotherapy continued to demonstrate its broad applicability across various cancer types. In kidney cancer (search), pembrolizumab (Keytruda) became the first adjuvant therapy to improve overall survival in adults. The FDA also approved tarlatamab (Imdelltra) and durvalumab (Imfinzi) for small cell lung cancer (search), a disease historically resistant to treatment. Blinatumomab (Blincyto), a bispecific T-cell engager, showed promise in treating acute lymphoblastic leukemia (search) (ALL) in infants and children, potentially increasing cure rates.
CAR T-cell therapy also offered hope for children with aggressive brain cancers like diffuse midline gliomas (search), with some patients showing responses lasting two years or more. Additionally, the FDA approved the first tumor infiltrating lymphocyte (TIL) therapy for advanced melanoma (search) and the first T-cell receptor (TCR) therapy for advanced synovial sarcoma (search).
Advances in Cachexia Treatment and Understanding ecDNAs
Significant progress was made in treating cancer cachexia (search), a wasting syndrome contributing to approximately 30% of cancer deaths. A clinical trial of ponsegromab demonstrated the reversal of cachexia-induced weight loss in advanced cancer patients. This development underscores the importance of basic science in identifying key molecular players in cachexia.
Studies on extrachromosomal DNAs (ecDNAs) revealed their involvement in treatment resistance and metastasis. These findings suggest that targeting tumors reliant on ecDNAs may offer a novel therapeutic approach.
Telehealth and Early Detection
Telehealth emerged as a valuable tool in cancer care, improving symptom management and palliative care access for both adult and pediatric patients. Studies demonstrated the effectiveness of telehealth in managing symptoms and treatment side effects in children with cancer and ensuring palliative care.
Progress in early cancer detection included promising results from blood tests for circulating tumor DNA, particularly for pancreatic cancer (search). A study in Scotland reported no cases of cervical cancer (search) among women fully vaccinated against HPV (search) as teenagers, reinforcing the potential of HPV vaccination in cancer prevention.