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Scientists at the Seattle Children's Research Institute are pioneering the use of gene-modified B cells – a type of white blood cell in the immune system – to treat a wide range of diseases that can affect children, including hemophilia and other disorders due to protein deficiency, autoimmune diseases and infectious diseases. If successful, their research would open the door to offering this experimental cell therapy as the first of its kind in clinical trials conducted at Seattle Children's in the next five years.
B cells play a central role in the immune system. When the body is confronted with an infection, it turns into plasma cells that release protective antibodies that fight current infections and prevent future ones. Unlike other cells in the immune system that have a relatively short lifespan, plasma B cells can survive and produce antibodies continuously for decades.
Since plasma cells are so effective at making and secreting proteins such as antibodies, they could be the ideal cell type for the production of therapeutic proteins.
Dr. David Rawlings, Dr. Richard James, and colleagues at the Research Institute's Center for Immunity and Immunotherapies are among a small number of US research groups dedicated to researching how clinicians might ever use plasma cells to fight children's diseases. As part of a first scientist, a team of basic scientists led by Rawlings and James genetically reprogrammed human B cells to act as cell factories capable of delivering high and sustained doses of a therapeutic protein introduced by genetic editing.
"Theoretically, the B cells we created could provide long-term treatment for a wide range of diseases for which the body does not have the ability to produce a certain protein, as in the case of the bleeding disorder," he says. 'Hemophilia B', said James. Such a cell therapy could also have many applications for autoimmune diseases, where the introduced protein could be used to disable abnormal immune responses or to disarm infectious diseases by secreting known protective antibodies.
New research confirms the longevity of B cells
The other question that researchers had to answer was whether reprogrammed B cells would persist in the body after their introduction, and that is precisely what James and his team did.
James presented at the annual conference of the American Society for Gene and Cell Therapy, in 2019, a study that was the first to demonstrate that modified human B cells can survive indefinitely in any model. To do this, scientists have developed mouse models with the same molecular characteristics as those required for the survival of cells secreting long-lived antibodies in humans.
According to James, this is a feature of the Seattle Children's gene-modified B cells that could offer an advantage over others.
"While others have developed a relatively short-lived product, the reprogrammed B-cells studied as part of our research have the potential to reside in the bone marrow and produce high levels of therapeutic protein during a lifetime. a much longer period, "he said.
Now that James and his team have models with B cells that last, their future studies will use these models to test new potential treatments.
"This is a significant advance because, until now, we have not yet been able to study long-lived B cells in animal models either. the possible therapies that they can offer, "said James. "This study sheds a light on how to do it."
Adjustment of B cells in therapeutic plants
Seattle Children's will conduct experiments over the next few years to improve modified B cells during their initial studies. This work will specifically focus on how best to manipulate these B cell products so that they can be translated into clinical use.
The scientific team is considering how to exponentially expand the modified B cells to reach the volume needed for infusion in a patient. Other studies will look for ways to produce more therapeutic proteins from each cell, as well as identify approaches to ensure that the modified cells take root and persist when they are removed. they are reinjected into a patient undergoing treatment.
"A new cell therapy using modified B cells has the potential to transform the care provided to children and adults with serious illnesses, so it's exciting to start working on creating a clinically available product at this stage." of our research, "said James. "We hope to open a Phase 1 clinical trial in the next few years."
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