Doctors at London’s Great Ormond Street Hospital used an “off-the-shelf” technique to deliver specially engineered T cells (the key to your immune system) to the babies via an IV. The T cells were designed to fight leukemia, and were delivered directly to the young patients’ veins when they were needed. The London experiment was notable not for this technique, however, but for the extent to which the cells were genetically modified. As the MIT Technology Review points out, the cells saw a total of four genetic changes, one of which allowed the donor T cells to work within another person, and another which allowed them to target cancer cells.
The 11 and 16-month-old infants had both previously undergone different treatments which did not successfully cure them of the disease. But this latest experiment appears to have done its job. Both children are currently in remission.
That said, some researchers have noted that there may be confounding variables at play. Both patients also received standard chemotherapy, so it’s unclear if the cell treatment alone is the reason they are currently cancer-free. “There is a hint of efficacy but no proof,” says Stephan Grupp, director of cancer immunotherapy at the Children’s Hospital of Philadelphia. “It would be great if it works, but that just hasn’t been shown yet.”
Great would be a serious understatement. If off-the-shelf cell therapy with these specially modified cells work, it would not only be effective, but efficient and relatively cheap as well. The other technique currently used to deliver engineered T cells to a patient involves collecting a patient’s blood cells, engineering their own T cells, and then returning the blood back to the patient. This, is a hugely expensive process (around $75,000) that must be customized to each individual patient.
But with this alternative off-the-shelf approach, “The patient could be treated immediately, as opposed to taking cells from a patient and manufacturing them,” says Julianne Smith, vice president of CAR-T development for Cellectis, which specializes in supplying universal cells. Smith continued, “We estimate the cost to manufacture a dose would be about $4,000.”
It’s still too early to determine whether or not this is truly a viable treatment option, but it looks like progress no matter how you slice it.
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