Could CAR-T therapy be engineered directly inside the human body? New findings reported in Nature suggest that this approach may be possible—and could eventually transform how certain autoimmune diseases are treated.CAR-T cell therapy has traditionally involved removing a patient's T cells, genetically modifying them in a laboratory to recognize specific disease-causing cells, and then infusing the engineered cells back into the patient. While this approach has produced remarkable results in some blood cancers, it can be complex, expensive and time-consuming.
Now, researchers are exploring a different strategy: making the therapeutic CAR-T cells inside the patient's body.In a small clinical trial involving 16 people with multiple sclerosis and other autoimmune conditions, researchers used a modified lentivirus to deliver genetic instructions directly to immune cells. These instructions enabled the participants' T cells to produce chimeric antigen receptors (CARs), allowing them to target B cells associated with the production of harmful autoantibodies.Following treatment, researchers observed an increase in CAR-T cells alongside reductions in B cells and autoantibodies. Participants with multiple sclerosis also experienced improvements in motor and cognitive function and reductions in fatigue, while participants with other autoimmune conditions showed improvements in muscle strength and inflammation.The findings are encouraging, but they are not yet proof that the treatment is effective or provides a permanent reset of the immune system. The trial was small, and participants were followed for approximately six months. Larger studies and longer follow-up will be necessary to establish the treatment's safety, effectiveness and durability.If successful, in-vivo CAR-T therapy could represent an important evolution in cell and gene therapy. Instead of manufacturing therapeutic cells outside the body, biotechnology could potentially provide the genetic instructions needed for the patient's own immune system to produce them.For researchers and the wider biotechnology sector, this development demonstrates how advances in gene delivery, immunology and molecular biotechnology are opening new possibilities for treating complex diseases.
At MyAfroDNA, we are committed to advancing biotechnology and supporting scientific research through molecular testing, DNA and RNA services, sequencing, species identification, paternity testing, molecular diagnostics and access to African biospecimens.Read the original report in Nature for more on this emerging approach to CAR-T therapy.