Good news! If these great results are confirmed by more trials, this could be a breakthrough! The cost for such treatments could be significantly lowered!
"Three people with severe autoimmune diseases are in remission after being treated with bioengineered and CRISPR-modified immune cells called chimeric antigen receptor (CAR) T cells. They are the first to be treated with engineered immune cells from donors. The advance could represent the first step towards mass production of CAR-T therapies for conditions such as lupus and multiple sclerosis. The ongoing success and safety of the therapy need to be demonstrated in more people before it can be considered for wider use, but it “could prove paradigm shifting” ..."
"... The three individuals from China are the first people with autoimmune disorders to be treated with engineered immune cells created from donor cells, rather than ones collected from their own bodies. This advance is the first step towards mass production of such therapies. ...
If successful, they would allow pharmaceutical companies to scale up manufacturing, potentially slashing costs and production times. Instead of making one treatment for one person, therapies for more than a hundred people could be made from one donor’s cells ..."
If successful, they would allow pharmaceutical companies to scale up manufacturing, potentially slashing costs and production times. Instead of making one treatment for one person, therapies for more than a hundred people could be made from one donor’s cells ..."
From the highlights and abstract:
"Highlights
• TyU19 is a genetically engineered, healthy donor-derived CD19-targeting CAR-T product
• TyU19 caused B cell depletion in three patients with refractory autoimmune diseases
• TyU19 alleviated severe skeletal muscle damage in a patient with refractory IMNM
• TyU19 reversed extensive fibrotic damage to critical organs in two patients with dcSSc
Summary
Allogeneic chimeric antigen receptor (CAR)-T cells hold great promise for expanding the accessibility of CAR-T therapy, whereas the risks of allograft rejection have hampered its application. Here, we genetically engineered healthy-donor-derived, CD19-targeting CAR-T cells using CRISPR-Cas9 to address the issue of immune rejection and treated one patient with refractory immune-mediated necrotizing myopathy and two patients with diffuse cutaneous systemic sclerosis with these cells. This study was registered at ClinicalTrials.gov (NCT05859997). The infused cells persisted for over 3 months, achieving complete B cell depletion within 2 weeks of treatment. During the 6-month follow-up, we observed deep remission without cytokine release syndrome or other serious adverse events in all three patients, primarily shown by the significant improvement in the clinical response index scores for the two diseases, respectively, and supported by the observations of reversal of inflammation and fibrosis. Our results demonstrate the high safety and promising immune modulatory effect of the off-the-shelf CAR-T cells in treating severe refractory autoimmune diseases."
World-first therapy using donor cells sends autoimmune diseases into remission "The treatment’s success in three people raises hopes for mass production of cutting-edge CAR T therapies."
Allogeneic CD19-targeted CAR-T therapy in patients with severe myositis and systemic sclerosis (no public access)
Graphical abstract
Engineered immune cells, called CAR T cells (yellow), have revolutionized treatment for some tumours (pink) and show promise for treating autoimmune conditions.
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