5 emerging therapies presented at ASCO 2025
May 28, 2025
Medically Reviewed | Last reviewed by on May 28, 2025
Every year, the American Society of Clinical Oncology Annual Meeting provides a platform for sharing exciting new cancer therapies. In 2025, MD Anderson clinicians and researchers will present more than 120 studies. Five stand out for their potential to change the way cancers are treated.
They include therapies for thyroid cancer, colorectal cancer and a rare type of leukemia. The studies represent a variety of first-in-class approaches for treating cancer, from a lipid nanoparticle-encapsulated mRNA to a new oral small molecule inhibitor.
1. Thyroid cancer combination therapy
Patients with a type of anaplastic thyroid cancer in which BRAF V600E is mutated tend to have a poor prognosis because they are often diagnosed when the disease is already advanced and can¡¯t be removed with surgery.
In this Phase II trial, , professor of Head and Neck Surgery, patients with Stage IV BRAF V600E-mutated anaplastic thyroid cancer were given neoadjuvant pembrolizumab in combination with dabrafenib and trametinib ¡ª a combination called DTP. Afterward, they had surgery to remove any remaining cancer, and on average, this was far more successful than historic averages in patients who hadn¡¯t been treated first with DTP. Two-thirds of patients had no residual anaplastic thyroid cancer, and these patients had an overall two-year survival rate of 69%.
These results are a strong indication that DTP treatment before surgery enables a higher rate of successful surgical resection.
2. New BRAF-mutated metastatic colorectal cancer first-line treatment
BRAF V600E¨Cmutated metastatic colorectal cancer is an aggressive subtype with a poor prognosis. A Phase III clinical trial, led at MD Anderson by co-principal investigator , professor of Gastrointestinal Medical Oncology and associate vice president of Translational Integration, is testing whether encorafenib plus cetuximab, with or without chemotherapy, will be a better first-line treatment.
Earlier results showed a 60.9% overall response rate with the three-drug combination, compared with 40% for the current standard-of-care treatment. These findings led to accelerated Food and Drug Administration (FDA) approval of this combination therapy for BRAF V600E¨Cmutated metastatic colorectal cancer, including as first-line therapy. take that one step further and show significantly longer progression-free and overall survival for patients treated with the three-drug combination, compared with those who received the current standard of care.
3. First-in-class mRNA-encoded, bispecific antibody
, professor of Investigational Cancer Therapeutics, will of a new compound called BNT142, a lipid nanoparticle-encapsulated mRNA. Once BNT142 has been given, cells in the patient¡¯s liver translate it into encoding the anti-CLDN6/CD3 bispecific antibody RiboMab02.1.
CLDN6 is a protein on the surface of cells that is silenced in normal adult tissues but is activated in testicular, ovarian, non-small cell lung and other cancers. Therefore, anti-CLDN6 compounds could be effective in patients with this mutation on their cancer cells.
This study examines weekly BNT142 treatments to determine the best dose among seven different dose levels. Early results indicate that the therapy has a manageable safety profile and promising anti-tumor activity at the higher doses. This study represents the first clinical proof-of-concept for an mRNA-encoded bispecific antibody.
4. Oral KIF18A inhibitor
Another first-in-human study is a Phase I/II trial of VLS-1488, an oral KIF18A inhibitor. KIF18A is a kinesin protein that is important for the successful division of cancer cells with chromosomal instability but is not required for normal cells. Preclinical research had shown that the compound causes dose-dependent inhibition of tumor growth, and this study¡¯s goal was to assess its safety and tolerability in humans.
, assistant professor of Investigational Cancer Therapeutics, , which indicate that the drug is generally safe and tolerable. Early results also show anti-tumor activity in patients who have already undergone several rounds of other types of treatment unsuccessfully. The study team will now evaluate the best dose of VLS-1488 in the next phase of the study.
5. Treatment for a rare leukemia
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare, aggressive type of leukemia that tends to primarily involve the skin, bone marrow and lymph nodes. CD123 (IL-3R¦Á) is a protein that abounds on the surface of all BPDCN blasts, which makes it an ideal target for novel immunochemotherapy. A new treatment called pivekimab sunirine (PVEK) is a first-in-class antibody-drug conjugate that includes three different parts: a high-affinity CD123 antibody, a cleavable linker and an indolinobenzodiazepine pseudodimer payload. Together, they should inhibit the growth of cells with CD123 (IL-3R¦Á).
The , which , professor of Leukemia, will present, shows that PVEK is generally safe and tolerable and demonstrates promising efficacy, with high and durable composite complete remission responses. Based on these results, the researchers are applying for approval from the FDA for PVEK to be a new treatment option for adult patients with BPDCN.
MD Anderson at ASCO
MD Anderson investigators are involved in a wide variety of basic, clinical, translational and population science research that will be presented at ASCO. These studies highlight the strength of our clinical trials program toward developing the next generation of standard of care treatments for our patients and cancer patients around the world. It also displays our strong commitment to research-driven patient care. This year, MD Anderson fellows received 10 ASCO Young Investigator Awards, indicating that the next generation of clinician scientists is also driving research innovations. I¡¯m looking forward to sharing and collaborating with our colleagues from around the world in Chicago at the ASCO Annual Meeting.
, is a physician-scientist and head of the Division of Cancer Medicine at MD Anderson.
Learn about research careers at MD Anderson.
These studies highlight the strength of our clinical trials program toward developing the next generation of standard of care treatments for our patients and cancer patients around the world.
Christopher Flowers, M.D.
Physician & Researcher