Layla Richards: Designer Cells Impact

Introduction: 

One year old Layla Richards suffering from an aggressive form of leukemia is the first to be treated with designer immune cells that were genetically engineered to wipe out her cancer. Given only months to live after the failure of conventional treatments, a miracle has occured. (A) Layla was treated in London by specialists at Great Ormond Street Hospital (aka: GOSH).  They were unsure whether the treatment would work, curing the disease, or delaying its progression but knew that only time would tell. However, there are still many concerns about this type of treatment for all children. Nevertheless, this is a huge leap for new gene engineering technology. (A) When Layla was born, within 3 months she developed fast heartbeat, did not drink her milk, and cried more than expected. Blood tests revealed she had acute lymphoblastic leukemia (aka: ALL) being the most aggressive form that doctors have ever seen.(A) A doctor who cared for Layla, Sujith Samarasinghe, stated that only ~25% of children survive such aggressive forms of leukemia. Next day, Layla was given chemotherapy and had a bone marrow transplant to replace her damaged blood cells. Despite all the conventional treatments, the disease returned.(A)

Research: 

Researchers from UCL showed that modified cells had an anti-cancer effect on mice with leukemia. They gave one vial of the cells to baby Layla through the approval and consent from emergency ethics committee and her parents. Once approved, she recieved 1mL infusion of genetically engineered immune cells taking the infusion a total of 10 minutes. The vial of cells came from donated T cells (which were kept frozen) or more commonly known as WBCs (have vital role in human immunity). However, before the infusion, the cells were given an added gene so that the cells target the leukemia cells. As suspected, the doctors predicted that if the infusion did in fact work, Layla would develop a rash (an indication that the cells were having an effect).  (A) Results: Two months passed, and Layla was still clear of leukemia which allowed doctors to give her a second bone marrow transplant to replace her entire blood and immune system. The treatment had wiped both out so this was necessary and vital part of the whole process. Thirty days later and she was healthy to go home. Doctors and researchers are now inspired to modify the therapy so that they can treat other blood disorders and the various types of cancers that exist. Clinical trials of the cells could be a huge step forward in treating both leukemi and other cancers. (A) 

Concluding Remarks: 

“Re-engineering a patient’s immune cells to target cancer has shown real promise in a small number of patients with leukaemia. This trial has adapted this treatment so that it’s easier to make, and now we need to see if this new approach is effective. Finding a way to make this work in other types of cancer is the next big challenge.(A)”

Links:

(A)http://www.theguardian.com/science/2015/nov/05/baby-girl-is-first-in-the-world-to-be-treated-with-designer-immune-cells

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