Experimental CAR T-Cell Therapy for Relapsed/Refractory ALL Patients Receives FDA Designations

Targeted Oncology recently published an article describing a group of T cell malignancies associated with blood disorders (hematologic cancers).

Currently, a high rate of patient deaths and relapses are occurring in children and adults with relapsed/refractory T cell acute lymphoblastic leukemia (T-ALL). There are currently no approved targeted immunotherapies.

Targeted Therapies

Targeted antibodies are a type of immunotherapy that can interrupt the activity of cancer cells and direct the immune system to eliminate cancer cells.

CAR T is Waiting in the Wings

Immunotherapy is our own body harnessing the power of our immune system in the fight against cancer. A new and exciting form of immunotherapy is CAR (chimeric antigen receptor) T cell therapy. The CAR T process involves removing immune cells from the patient, engineering them to identify cancer, and reinfusing the engineered cells back into the patient in greater numbers.

These cells are then “living drugs” that express a synthetic receptor that binds a tumor antigen.

However, the development of CAR-T has been limited due to CAR-T fratricide (self-killing). This is compounded by an inability to harvest a sufficient number of autologous T cells.

The FDA’s Designations for UCART7

In response, on July 19, 2022, the biotech company Wugen Inc. issued a press release announcing that the FDA granted Rare Pediatric and Fast Track Designations for its WU-CART-007 (UCART7). The cell therapy is Wugen’s answer to relapsed and refractory T-ALL and lymphoblastic lymphoma (LBL).

Wugen’s statement contained a description of UCART7 and its targeting of CD7+T cell malignancies. As a result of CRISPR/Cas9 editing, UCART7 does not have CD7 or TRAC expression. UCART7 showed its efficacy when targeting human T-ALL cell lines without xenogeneic GvHD induction, a lethal factor that occurs after certain stem cell transplants.

At present UCART7 is under scrutiny for efficacy and safety in relapsed and refractory T-All/LBL patients in clinical trial NCT04984356. Trial participants are currently undergoing the phase of the trial involving dose escalation..

The trial is a global, first-in-human, open-label phase ½ study with the goal of evaluating the safety, dose recommendations, and WU-CART-007 anti-tumor activity.

Dr. Dan Kemp, CEO of Wugen expressed the company’s appreciation for both FDA designations. He stated that the FDA honors re-affirm that patients with R/R T-ALL/LBL are in dire need of new treatment options.

What are your thoughts about becoming a rare disease advocate?

Share your stories, thoughts, and hopes with the Patient Worthy Community

Rose Duesterwald                      August 6, 2022

Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.

Share this post

Share on facebook
Share on twitter
Share on linkedin
Share on pinterest
Share on print
Share on email