Roger B. Cohen, MD, is a professor of medicine at the Hospital of the University of Pennsylvania (Penn) and associate director of clinical research at Penn's Abramson Cancer Center. Along with his colleagues at Penn, he is investigating a novel CAR T-cell product as a treatment for adults with medullary thyroid cancer (MTC).

MTC is a rare disease, accounting for 1% to 2% of thyroid cancer cases. The prognosis for these patients is poor, and there are few treatment options for those with relapsed/refractory disease.

CAR T-cell thearapies have had limited success in solid cancers, but Donald Siegel, MD, PhD, at Penn, has discovered a unique protein, GFRα4, that is expressed only on MTC cells. Dr Cohen explains how this protein provides a target for GFRα4 CAR T-cells in patients with MTC.

Videos

Dr. Cohen on Potential With CAR T-Cell Therapy in Medullary Thyroid Cancer

Donald Siegel, MD, PhD, Dr. Cohen's scientific collaborator, discovered GFRα4, a protein uniqely expressed on the parafollicular cells that become malignant in MTC. In the clinical trial, Dr. Cohen will assess whether a one-time infusion of autologus GFRα4 CAR T cells can kill tumor cells without causing the toxicities common to immunotherapy.

GFRα4 May Represent a Unique Therapeutic Target in Medullary Thyroid Cancer

Investigators leading clinical trials must go the extra mile to recruit diverse patient populations and the challenge is even greater for those exploring rare cancers. Here, Dr. Cohen explains the steps Penn is taking to recruit the broadest variety of patients.

Dr. Cohen on Diversifying the Patient Population for a Clinical Trial in Medullary Thyroid Cancer

Maria E. Cabanillas, MD, an oncologic endocrinologist, tenured professor and the faculty director of clinical research in the Department of Endocrine Neoplasia at The University of Texas MD Anderson Cancer Center, discusses how to deliver the optimal surgical approach to patients with medullary thyroid cancer (MTC).

As a clinician and clinical researcher, she treats patients with both early and advanced thyroid cancers, including medullary, differentiated, and anaplastic thyroid cancers. MTC accounts for 1% to 2% of all thyroid cancers. For patients with metastatic disease, the 5-year overall survival is less than 40%.

At present, thyroidectomy is the only curative option for patients with MTC. Unfortunately, she says that many patients do not get the "right" surgery. She said that surgeons want to see good imaging, including a CT scan of the neck with contrast, before proceding with surgery.

Maria E. Cabanillas, MD, an oncologic endocrinologist, tenured professor and the faculty director of clinical research in the department of Endocrine Neoplasia at The University of Texas MD Anderson Cancer Center, discusses how to deliver the optimal surgical approach to patients with medullary thyroid cancer.

Dr Cabanillas on Delivering the "Right" Surgery to Patients With MTC

Maria E. Cabanillas, MD, oncologic endocrinologist, tenured professor and the faculty director of clinical research in the department of Endocrine Neoplasia at The University of Texas MD Anderson Cancer Center, discusses current systemic treatments in medullary thyroid cancer (MTC), along with the potential for peptide receptor radionuclide therapy (PRRT).

As a clinician and clinical researcher, she treats both patients with early and advanced thyroid cancers, including medullary, differentiated, and anaplastic thyroid cancers. MTC accounts for 1% to 2% of all thyroid cancers. For patients with metastatic disease, the 5-year overall survival rate is less than 40%.

Although surgery is the only curative treatment currently available for MTC, Cabanillas said there are a number of FDA-approved systemic therapies including vandetanib (Caprelsa) and cabozantinib (Cabometyx). She is anticipating data on PRRT, a precise form of radiation under investigation for MTC and currently used to target neuroendocrine tumor cells with hormone receptors.

Maria E. Cabanillas, MD, oncologic endocrinologist, tenured professor and the faculty director of clinical research in the department of Endocrine Neoplasia at The University of Texas MD Anderson Cancer Center, discusses current systemic treatments in medullary thyroid cancer, along with the potential for peptide receptor radionuclide therapy.

Medullary Thyroid Cancer | Videos

Dr. Cohen on Potential With CAR T-Cell Therapy in Medullary Thyroid Cancer

GFRα4 May Represent a Unique Therapeutic Target in Medullary Thyroid Cancer

Dr. Cohen on Diversifying the Patient Population for a Clinical Trial in Medullary Thyroid Cancer

Dr Cabanillas on Delivering the "Right" Surgery to Patients With MTC

Dr Cabanillas on Current Systemic Treatments in MTC and the Potential for PRRT