Stem Cell Transplantation Achieves Non-Toxic Preconditioning, Expected to Benefit More Patients with Hereditary Diseases

                                     Stem Cell Transplantation Achieves Non-Toxic Preconditioning, Expected to Benefit More Patients with Hereditary Diseases

Source

Science and Technology Daily (Reporter Zhang Mengran), originally published in Nature Medicine

Abstract

The new antibody therapy developed by the Stanford University School of Medicine in the United States has made a breakthrough in Phase I clinical trials. It can prepare for stem cell transplantation without relying on traditional toxic busulfan chemotherapy or radiotherapy. The therapy, which used Fanconi anemia patients as test subjects, adopted the CD117-targeted antibody "Blinatumomab" and successfully completed transplants for 3 child patients, all of whom are in stable health. This scheme is expected to benefit more patients with hereditary diseases and bring a paradigm innovation to related treatments.

Content

The Stanford University School of Medicine in the United States announced on July 22 that its newly developed antibody therapy has made a breakthrough in Phase I clinical trials. This therapy can prepare for patients' stem cell transplantation without relying on traditional toxic busulfan chemotherapy or radiotherapy. Although the research team used Fanconi anemia patients as test subjects, it is expected that this scheme will benefit more groups of patients with hereditary diseases. The research results were published in the latest issue of Nature Medicine, and its safety and effectiveness may bring a paradigm innovation to the treatment of hereditary diseases.

Fanconi anemia is a hereditary disease. Patients have defects in hematopoietic function, which makes the risk of standard stem cell transplantation extremely high. The research team innovatively adopted a treatment scheme targeting CD117 antibody and successfully completed transplants for 3 child patients. CD117 is a key protein on the surface of hematopoietic stem cells. By injecting the new antibody "Blinatumomab", it can accurately clear the patients' own stem cells and avoid genetic toxic damage. At present, all 3 children have passed the two-year follow-up period and are in stable health.

Researchers said that traditional pre-transplant conditioning often requires radiotherapy and busulfan chemotherapy, which is extremely risky for children who are already physically fragile. If Fanconi anemia patients fail to receive transplantation in time, they will face the risk of fatal bleeding or infection due to hematopoietic failure. This trial provides a new strategy, that is, to clear the bone marrow environment in a milder way without changing the disease mechanism, creating ideal conditions for the implantation of healthy stem cells, which brings great hope for saving vulnerable patients.

In the future, this antibody therapy is expected to be extended to more indications, allowing more patients to benefit from this low-toxicity and high-efficiency transplantation scheme.