Advances in Anaplastic Thyroid Cancer Research and Treatment

The ATC Multidisciplinary Petrick Research Program

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The University of Texas MD Anderson Cancer Center sees approximately 5,000 thyroid cancer patients yearly, amongst the highest number in the world, with a significant advanced thyroid cancer population. 

Anaplastic thyroid cancer (ATC) represents 1% of thyroid cancers, but causes up to 50% of thyroid cancer-related deaths.

Prior to very recent significant treatment advances, historic median survival has been about 3 months. ATC progresses very rapidly, often leading to life-threatening complications within weeks, including loss of speech, swallowing and breathing.

In 2014, MD Anderson established the Facilitating Anaplastic Thyroid Cancer Specialized Treatment (FAST) program, now treating several hundred ATC patients per year, making it the largest ATC center globally. The FAST program provides the clinical backbone for multidisciplinary collaboration across departments and has demonstrated great capacity for clinical trials. 

A landmark trial with dabrafenib (BRAF inhibitor) and trametinib (MEK inhibitor), led by MD Anderson, resulted in FDA approval for the first targeted agents for BRAFV600E-mutated ATC in 2018.

With significant advances in treatment over the last two decades, median patient survival has quadrupled, increasing from 7 months to approximately 28 months, and is continuing to improve.

However, challenges remain due to ATC’s heterogeneous genomic profile. While 30-40% of ATCs harbor the BRAFV600E mutation, resistance to BRAF/MEK inhibitors limits success in many patients. For the 60% without this mutation, treatment options remain limited to chemoradiation with poor response rates and a 3-5 month survival. These challenges underscore the need for strengthening translational research to better understand ATC pathogenesis, resistance mechanisms and to explore new therapeutic opportunities.

The FAST team treats more ATC patients than any other cancer center in the world.

The FAST team treats more ATC patients than any other cancer center in the world.

Thanks to generous donors like you, over the last several years, we have made a tremendous impact for patients with anaplastic thyroid cancer.

Thanks to generous donors like you, over the last several years, we have made a tremendous impact for patients with anaplastic thyroid cancer.

Since 2020, the FAST program at
MD Anderson has benefited from the ongoing generous contributions from the Petrick family and friends like you.

These contributions have established the ATC Multidisciplinary Petrick Research Program, which supports key research initiatives aimed at advancing the understanding of ATC, addressing critical gaps in research and ultimately improving patient quality of life and survival.

With your generous support, we are confident that this effort will continue to drive future breakthroughs, thereby saving and prolonging the lives of patients with this aggressive disease.

We are conducting groundbreaking clinical trials through which we hope to to improve outcomes for patients with ATC.

Pembrolizumab, Dabrafenib, and Trametinib Before Surgery for the Treatment of BRAF-Mutated Anaplastic Thyroid Cancer

Principal Investigator: Mark Zafereo, M.D.

IMRT Followed by Pembrolizumab in the Adjuvant Setting in Anaplastic Cancer of the Thyroid (IMPAACT): Phase II Trial Adjuvant Pembrolizumab After IMRT in ATC

Principal Investigator: Maria E. Cabanillas, M.D.

Lenvatinib and Pembrolizumab for the Treatment of Stage IVB Locally Advanced and Unresectable or Stage IVC Metastatic Anaplastic Thyroid Cancer

Principal Investigator: Maria E. Cabanillas, M.D.

Atezolizumab with Chemotherapy in Treating Patients with Anaplastic or Poorly Differentiated Thyroid Cancer

Principal Investigator: Maria E. Cabanillas, M.D.

In 2024, we built upon our ongoing research and
proposed new groundbreaking studies.

We believe this work has the potential to significantly improve the survival and quality of life for anaplastic thyroid cancer patients.

Through the following studies, and with your generous support, we aim to  fulfill this potential.

GRASSROOT — Gathering of Real-world data in patients with Advanced thyroid cancer on Standard of care and Specialized Interventions — Registry of Oncologic Outcomes with Testing and treatment

This critical project will expand the outreach of our expertise to sites around the world who don’t see a high volume of ATC cases. This will help take our expertise in ATC to those patients who cannot travel to MD Anderson.

Principal Investigator
Priyanka Chandrasekhar Iyer, M.B.B.S.

Headshot of Dr. Tina Cascone. Headshot of Dr. Tina Cascone.

Elucidating and characterizing resistance to BRAF/MEK inhibition in aggressive thyroid cancer

By developing and establishing a BRAF/MEK inhibitor resistant patient-derived xenograft model, we will have a one-of-a-kind pre-clinical tool to study the mechanisms behind drug resistance development, while utilizing this model to test the latest therapies being developed for anaplastic thyroid cancer

Principal Investigator
Anastasios Maniakas, M.D., Ph.D.

Headshot of Dr. Tina Cascone. Headshot of Dr. Tina Cascone.

Animal and cell line model core

We continue to develop the world’s most comprehensive collection of cell lines and patient-derived xenograft models for anaplastic thyroid cancer. These models represent the molecular diversity of the disease and are invaluable for our continued research to improve patient survival.

Principal Investigators
Stephen Y. Lai, Ph.D., Marie-Claude Hofmann, Ph.D.

Headshot of Dr. Tina Cascone. Headshot of Dr. Tina Cascone.

Characterizing tumor evolution in resistant/persistent subclones following BRAF-directed therapy and targeting these populations via a theragnostic approach

We are characterizing the evolution of resistant subclones in ATC during BRAF/MEK inhibition, with a focus on identifying persistent populations that drive therapeutic resistance. Using a theragnostic approach, we aim to target these resistant subpopulations with radionuclide-based imaging and therapies to improve treatment efficacy.

Principal Investigator
Xiao Zhao, Ph.D.

Headshot of Dr. Tina Cascone. Headshot of Dr. Tina Cascone.

Unravelling variation in treatment response using spatial transcriptomics in BRAFV600E-mutated ATC

This study seeks to identify spatial interactions between tumor cells, stromal cells and immune cells to understand how interactions drive response to therapy in ATC.

Principal Investigator
Jennifer Rui Wang, M.D., Ph.D.

Headshot of Dr. Tina Cascone. Headshot of Dr. Tina Cascone.

Zanzalintinib with cemiplimab in treating patients with ATC

This trial will evaluate the effectiveness of zanzalintinib combined with cemiplimab in treating anaplastic thyroid cancer. By utilizing novel immunotherapy and targeted drugs, we aim to improve treatment outcomes compared to standard therapies.

Principal Investigator
Maria E. Cabanillas, M.D.

Headshot of Dr. Tina Cascone. Headshot of Dr. Tina Cascone.

RECENT UPDATES

Funded Grants

The following grants were funded thanks in large part to your generous contributions:

Targeted Therapy Combined with Pembrolizumab in Anaplastic Thyroid Cancer: Two Phase II Clinical Trials

Gateway Cancer Research (2021-2025)

Principal Investigator: Mark Zafereo, MD

Intratumor Heterogeneity in Anaplastic Thyroid Carcinoma: Implications for Treatment Resistance

The Mark Foundation for Cancer Research, ASPIRE Award (2020-Present)

Principal Investigator: Jennifer Wang, MD, PhD

Multidisciplinary Research Program: Improving Treatment Strategies for Anaplastic Thyroid Cancer Through Integration of Novel Therapies and Assessment of Treatment Response

The MD Anderson Cancer Center Multidisciplinary Research Program (MRP) (2018 –2021)

Principal Investigators: Stephen Y. Lai, MD, PhD and Marie-Claude Hofmann, PhD

Select Publications

Maniakas A, Dadu R, Busaidy NL, Wang JR, Ferrarotto R, Lu C, Williams MD, Gunn GB, Hofmann MC, Cote G, Sperling J, Gross ND, Sturgis EM, Goepfert RP, Lai SY, Cabanillas ME, Zafereo M. Evaluation of Overall Survival in Patients with Anaplastic Thyroid Carcinoma, 2000-2019. JAMA Oncol. 2020 Sep 1;6(9):1397-1404.

Wang JR, Montierth M, Xu L, Goswami M, Zhao X, Cote G, Wang W, Iyer P, Dadu R, Busaidy NL, Lai SY, Gross ND, Ferrarotto R, Lu C, Gunn GB, Williams MD, Routbort M, Zafereo ME, Cabanillas ME. Impact of Somatic Mutations on Survival Outcomes in Patients With Anaplastic Thyroid Carcinoma. JCO Precis Oncol. 2022 Aug;6:e2100504.

 Zhao X, Wang JR, Dadu R, Busaidy NL, Xu L, Learned KO, Chasen NN, Vu T, Maniakas A, Eguia AA, Diersing J, Gross ND, Goepfert R, Lai SY, Hofmann MC, Ferrarotto R, Lu C, Gunn GB, Spiotto MT, Subbiah V, Williams MD, Cabanillas ME, Zafereo ME. Surgery After BRAF-Directed Therapy Is Associated with Improved Survival in BRAF(V600E) Mutant Anaplastic Thyroid Cancer: A Single- Center Retrospective Cohort Study. Thyroid. 2023 Apr;33(4):484-491.

 Hamidi S, Iyer PC, Dadu R, Gule-Monroe MK, Maniakas A, Zafereo ME, Wang JR, Busaidy NL, Cabanillas ME. Checkpoint Inhibition in Addition to Dabrafenib/Trametinib for BRAF(V600E)-Mutated Anaplastic Thyroid Carcinoma. Thyroid. 2024 Mar;34(3):336-346.

 Hamidi S, Dadu R, Zafereo ME, Ferrarotto R, Wang JR, Maniakas A, Gunn GB, Lee A, Spiotto MT, Iyer PC, Sousa LG, Akhave NS, Ahmed S, Learned KO, Lu C, Lai SY, Williams M, Hosseini SM, Busaidy NL, Cabanillas ME. Initial Management of BRAF V600E-Variant Anaplastic Thyroid Cancer: The FAST Multidisciplinary Group Consensus Statement. JAMA Oncol. 2024 Sep 1;10(9):1264-1271.

Research Retreat

The ATC Research Retreat, organized by Dr. Cabanillas and Dr. Wang at MD Anderson and sponsored by the ATC Multidisciplinary Petrick Research Program, took place on November 22, 2024, at the Texas Medical Center.

This event brought together distinguished speakers from MD Anderson and other leading institutions, including Memorial Sloan Kettering Cancer Center, Washington University in St. Louis, Cleveland Clinic, Vanderbilt University, Houston Methodist and Universidad Nacional de Córdoba.

The retreat focused on addressing critical treatment gaps in ATC and exploring innovative strategies for potential clinical applications within five years. This event also fostered collaboration among experts to advance care and improve outcome for ATC patients.

Sponsored by the ATC Multidisciplinary Petrick Research Program, the 2024 ATC Research Retreat brought together experts in the filed from around the world.

Sponsored by the ATC Multidisciplinary Petrick Research Program, the 2024 ATC Research Retreat brought together experts in the filed from around the world.

Patient Success Stories

Mr. Jeffrey Foskett

After his first thyroid cancer surgery ended his singing career, a clinical trial gave Jeffrey "bonus" years to use his voice in new ways.

Mr. Max Nickless

When Max was diagnosed, all he wanted was more time. A clinical trial at MD Anderson gave him just that.

Your Support is Driving Innovation.

And we can't do this without you.

Help us achieve our goal of raising $4M by December 2026 to continue funding groundbreaking ATC research.