Innovative Cancer Treatments: CRISPR and the Future of Therapy
Written on
Chapter 1: The Promise of CRISPR in Cancer Treatment
Recent advancements in genetic technology have opened new avenues for treating cancer. A recent study involving eleven patients whose cancers were resistant to traditional therapies or had returned after treatment showed promising results using genetically modified donor cells infused with CRISPR technology.
The outcomes were encouraging, as eight participants experienced a decrease in tumor size, with two achieving remission. However, the trial was not without its tragic moments; one participant, a 72-year-old man, passed away, which was attributed to the edited cells.
The biotech company CRISPR Therapeutics revealed these findings last week, aiming to create a scalable cancer therapy utilizing living cells. The initial steps of their process include extracting specialized immune cells known as T cells from healthy donors. In a laboratory setting, scientists employ CRISPR to perform three simultaneous edits on these cells, enhancing their ability to target tumors while evading the recipient's immune response. Following this, the modified cells are infused into the patient.
This innovative approach of using genetically enhanced immune cells in cancer treatment has gained traction in recent years, with scientists believing that CRISPR could significantly boost the efficacy of these immune cells.
In this study, researchers focused on patients with advanced B-cell lymphoma, a cancer originating in white blood cells. The participants were administered four distinct doses of the therapy; notably, the individual who succumbed to the treatment received the highest dosage. Although the preliminary results are hopeful for the other eight patients, it remains uncertain whether this approach will provide a lasting cure.
Dr. Eric Kmiec, director of the Gene Editing Institute at Christiana Care Health System in Delaware, expressed both intrigue and concern regarding the method, highlighting that altering human cells could provoke an immune response due to the cells being perceived as foreign.
Three trial participants encountered cytokine release syndrome, a potentially life-threatening immune response that mimics flu-like symptoms, but they managed to recover.
The goal of CRISPR Therapeutics is to develop a readily available cancer treatment using living cells that can be mass-produced, providing timely solutions for patients in need.
Chapter 2: Past Efforts and Future Directions
CRISPR's application in cancer treatment isn't unprecedented. In 2016, researchers in China initiated trials using CRISPR for various cancer types, followed by a 2019 study at the University of Pennsylvania that involved editing patients' own T cells. While this small-scale study confirmed the safety of reintroducing CRISPR-edited cells, it did not evaluate the therapy's curative potential.
These efforts aimed to refine CAR-T cell therapy, a groundbreaking treatment that modifies a patient's T cells through conventional genetic engineering. So far, three CAR-T therapies have been approved by the FDA, with a 30% to 40% cure rate among patients. However, not all patients benefit from this treatment; some may lack sufficient T cells due to prior chemotherapy, making them ineligible for CAR-T.
CRISPR Therapeutics is addressing these limitations by creating "off-the-shelf" therapies sourced from donor T cells. This innovation could facilitate mass production, allowing faster access to treatment for cancer patients.
In a presentation on the company's findings, Dr. Samarth Kulkarni, CEO of CRISPR Therapeutics, emphasized that these therapies could represent a revolutionary new class of medicine in the battle against cancer.
Despite their potential, such therapies are currently reserved for patients who have relapsed or have not responded to prior treatments, primarily due to serious side effects, including cytokine release syndrome.
Additionally, the complexity and high costs associated with creating these therapies present significant barriers. One FDA-approved CAR-T therapy can cost upwards of $475,000, while two others are priced around $373,000. By developing an off-the-shelf version using donor cells, CRISPR Therapeutics aims to broaden access to potentially life-saving treatments, but the company must first demonstrate safety and efficacy in larger patient populations.
The first video titled "CAR T-Cell Therapy: How Does It Work?" delves into the mechanics of CAR T-cell therapy and its application in treating various cancers.
The second video "Understanding Genomics and Genetic Testing in Cancer Immunotherapy" explores the role of genomics and genetic testing in optimizing cancer immunotherapy approaches.