Newsletter | October 15, 2019

10.15.19 -- Strategies For CAR T-Cell Therapies And Rare Disease Treatments

A Brief Introduction To CAR T-Cell Therapy

With the approvals of tisagenlecleucel (KYMRIAH) and axicabtagene ciloleucel (YESCARTA) last year, chimeric antigen receptor (CAR) T-cell therapies have changed the treatment paradigm for patients with certain hematologic malignancies. This article breaks down the development of CAR T-cell therapy as well as its advantages and challenges.

A Primer On Cancer Immunotherapy

Widespread use of immune checkpoint therapy to treat cancers is hampered by unpredictable response rates and immune-related adverse events. To address these challenges combination therapies are being studied as a strategy for improving response and overcoming resistance. This article provides an introduction to cancer immunotherapy, exploring its immunological basis and the fundamental principles guiding development of new treatments.

Principles Of CAR T-Cell Therapy And The Pathway To Regulatory Approval

Advances in immuno-oncology have led to the advent of CAR T-cell therapy, which combines a patient’s own T cells with engineered T-cell receptors known as “CARs”. The CAR enables the final product to produce chemicals in the hopes that the “enhanced” product or cells will bind to the cancer cells and kill them. Learn about the principles of CAR T-cell therapy and the ways these technologies can reach patients.

Complete Response: Lymphoma Trial A Success For Five Patients

A trans-Atlantic study to evaluate an antibody for treatment of B-cell non-Hodgkins lymphoma overcame patient recruiting challenges and has already succeeded beyond expectations. Five patients were declared disease-free for one year and counting while still three years from completing patient follow-up.

Registry And Natural History Studies: Vital, Contrasting Roles In Rare Disease Clinical Research

Both registry studies and natural history studies play important roles in rare disease research. Understanding the differences between the two types of studies and how they can be used to inform clinical development can help sponsors plan for success.