Rapid Advancements in Immunotherapy

A technology called CAR-T immunotherapy, shown to be effective in some cancers, uses a new artificial gene in the patient’s immune cells. The new cells are infused to target the patient’s cancer. Clinical trials proved successful as a gene therapy using CAR-T was approved this past year under Breakthrough Therapy status for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia.

 

Recently, researchers began to edit DNA with CRISPR, sometimes referred to as a molecular pair of scissors, to get to the root of the disease or cancer by fixing genetic errors. Advancements in China using CRISPR edited T-cells to battle cancer in terminal patients, where researchers have engineered the T-cells in ex vivo treatments to attack the patient’s cancer cells, is garnering international interest but also skepticism over long term implications and the ethics around this experimental process; especially since several of the patients in China have either died or have had side effects profound enough for them to leave the trial (like a high fever as their body became overwhelmed with the immune response).²

 

What does this mean for clinical trials in the US?

 

Several CRISPR researchers had hoped to start trials in the US and received approval from the NIH and FDA, but expectations are that clinical trials will probably start in late 2018 or 2019 for about a dozen or so submissions.³  

 

A Phase 1 trial planned to target three different types of cancer: multiple myeloma, sarcoma and melanoma. On this particular trial, before receiving their engineered T-cells, the patients will receive chemotherapy to eliminate their natural immune cells, then receive a single infusion of T-cells, and will be monitored for effectiveness, adverse effects and toxicities over the next five years.⁴

 

As Life Science companies look to clinical trials in gene editing to help patients with specific diseases, there will be a significant amount of time being spent on understanding and balancing the risk with the benefits. Helping both the healthcare providers and the patients understand the process and justify the risks will be synonymous to achieving success.