New method is expected to make hematopoietic stem cell transplantation safer
New method is expected to make hematopoietic stem cell transplantation safer June 08, 2016 Source: Bio Valley Currently, bone marrow transplantation is the only effective treatment for these blood diseases. But if new transplanted hematopoietic stem cells are to work, then defective hematopoietic stem cells are first "expelled" or killed. To do this, patients must be required to endure chemotherapy and radiotherapy - the role in the body is relatively fierce, often with adverse effects that accompany life. Tetanus Shot,Tetanus Vaccine,Hepatitis B Injection,Hep B Vaccine FOSHAN PHARMA CO., LTD. , https://www.fospharma.com
In a new study, researchers from Harvard University, Massachusetts General Hospital (MGH), Boston Children's Hospital, and Dana Farber Cancer Institute developed antibodies using antibodies specifically targeting hematopoietic stem cells in mice. A non-toxic method of transplantation. They hope that this approach will make hematopoietic stem cell transplantation less toxic to patients. The relevant research results were published online June 6, 2016 in the journal Nature Biotechnology , entitled "Non-genotoxic conditioning for hematopoietic stem cell transplantation using a hematopoietic-cell-specific internalizing immunotoxin".
This new treatment removes more than 98% of hematopoietic stem cells from mice, making it as effective as chemotherapy and radiation.
Dr. David Scadden, author of the paper, Harvard Medical Professor and HSCI Deputy Director, said, "We are not using non-targeted drugs that produce a lot of collateral damage. Instead, we can take advantage of the immune system, especially antibodies. Targeting."
As part of the immune system, antibodies naturally seek and destroy foreign substances in the body. The first author of the paper and Rahul Palchaudhuri, a postdoctoral researcher at Scadden Labs, equipped the CD45 targeting antibody with a load that only destroys existing blood cells (saponin). This saporin kills blood cells by acting differently than current standard therapies.
Palchaudhuri said, “Antibodies can specifically act on their targets. We can target them to CD45, a cellular marker that is expressed only in the blood system. This way we can avoid toxicity to non-blood tissue. ."
Unlike chemotherapy and radiation therapy - indiscriminate damage to cells and tissues (whether healthy or diseased), CD45-targeted antibodies do not affect thymus and bone marrow - in T cells and innate immunity A place where cell formation plays a vital role - causing harm. Mice treated with this antibody are resistant to infections that are lethal to mice receiving radiation therapy. Currently, post-transplant infections occur frequently and can be severe, resulting in a significant number of patient deaths.
After receiving standard treatment (chemotherapy or radiation therapy), approximately one in ten patients cannot survive after receiving a transplant. These patients may develop growth retardation, mental retardation, infertility, and DNA damage; currently, patients can only attempt therapeutic transplantation by increasing the risk of developing cancer later.
Because of this, Scadden explains that family and doctors often avoid choosing a transplant regimen, especially when it comes to child care, and this limits the extent to which breakthroughs in gene therapy and gene editing can be applied.
Mice receiving this antibody treatment received a bone marrow transplant for 10 days in the window period, and individual mice that did not receive bone marrow transplantation were able to fully recover without side effects. Furthermore, mice treated with sickle cell anemia were successfully transplanted by this antibody method, and their anemia can be cured. If this applies to humans, then a few months of hospitalization may be replaced by an outpatient procedure, and the transplant will not be fatal even if it fails.
“If this method is also effective in the human body, then it will really change the interaction between the doctor and the patient,†Scadden said, especially those who suffer from these underlying genetic diseases, and new genetic editing and Those patients whose gene therapy technology is being developed."
Now, researchers are trying to identify antibodies that are effective in the human body, and have formed a company to clinically transform the study and determine which animal models are best suited for use in preclinical studies.