A University of Pennsylvania led study team revealed data from this first study involving cancer patients who received cells genetically modified with CRISPR. Although still preliminary, the results appear promising reports NPR.
Clinical Investigators Comment
This initial CRISPR cancer study was designed to determine safety and feasibility and was not intended to assess whether the treatment actually worked and reduced or eliminated the cancer. As Principal Investigator, Dr. Edward Stadtmauer noted, “This treatment is not ready for prime time.” However, he continued “…it is definitely very promising.” In fact, other prominent researchers agree such as the well-known CRISPR investigator Jennifer Doudna with University of California, Berkeley who reported, “I’m just so excited about this.” She continued, “It’s an important step on the path toward using CRISPR-Cas genome editing in patients and shows the potential of this technology to be a safe and effective therapy.”
Finally, Dr. Michael Sadelain, a researcher at Memorial Sloan Kettering Cancer Center in New York noted he is glad to see CRISPR-based therapies move into the clinic in the way it has and that the key moving forward will be to follow the patients for a longer time period to understand if the approach is safe.
CRISPR-based Trials Updates
CRISPR studies in China have occurred for years but there is little transparency in regard to results. In the U.S., CRISPR Therapeutics AG is conducting another cancer study evaluating the safety and efficacy of CTX110 in patients with relapsed or refractory B-cell malignancies. In yet another U.S. study, physicians are testing CRISPR for the blood disorder sickle cell disease and beta thalassemia reports NPR. Moreover, researchers are planning on employing CRISPR to edit human cells to treat a genetic form of blindness.
The Cancer Study
The researchers will present the CRISPR study-based cancer study findings next month at the American Society of Hematology meeting in Orlando, FL. The study involved just three patients including two with multiple myeloma and one with sarcoma. This study procedure has involved the infusions of about 100 million of their own immune system cells that had been taken away from their bodies and were thereafter genetically modified in a lab.
Combining CRISPR and Immunotherapy
This study combines the gene editing technique CRISPR with immunotherapy to amount to what Dr. Stadtmauer reports “has really been a revolution in the immunotherapy for cancer.” He continued, “We’ve really learned how to harness the immune system to treat cancer—to crease these miraculous cells that are infused into patients as living drugs to treat cancer.” A challenge is that immunotherapy has worked in some cancers (leukemia and lymphoma patients for example) while the genetically engineered drugs don’t work well in others. Hence, the turn to CRISRP to refine and target a more precise and effective therapy.
The Next Steps
The next steps is to treat additional patients in a clinical trial sponsored by University of Pennsylvania in collaborate with Tmunity and the Parker Institute for cancer Immunotherapy. The Phase I trial will seek 18 participants who have sarcoma, melanoma or myeloma. The study is scheduled to conclude by 2033. It is a first in-human trial proposed to test HL-A*0201 restructed NY-ESO-1 redirected T cells with edited endogenous T cell receptor and PD-1. This “cautious approach” has bene applauded by other researchers such as bioethicist Laurie Zoloth from the University of Chicago reports the NPR.
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