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The Children’s Hospital of Philadelphia is now home to a device that helps make mobile CAR-T treatment much less expensive.
-Stephan Kadauke (left) and Steve Grupp of the Children’s Hospital of Philadelphia in the empty room where immunotherapy remedies are produced. (Maiken Scott/WHY)
More than 10 years ago, pediatric oncologist Stephan Grupp made headlines around the world when he helped a young patient on the brink of death with a new experimental genetic treatment treatment. Today, his goal is to make those treatments more available and available. less expensive.
In 2012, Emily Whitehead became the first girl to receive the mobile curative CAR-T treatment at Children’s Hospital of Philadelphia, after her leukemia relapsed twice. Emily was seven years old at the time and her parents had been told she would want to be transferred to hospice care. They then discovered that she might have access to an experimental remedy employing modified T-mobiles, a technique developed under the leadership of Carl June of the University of Pennsylvania.
“We’re genetically modifying those T cells and giving them anti-cancer properties,” Grupp said.
T cells are part of the immune formula that normally helps the body fight infection and disease, as well as cancer. But cancer manages to evade the immune formula. For treatment, T cells are collected from a patient and modified to target an express response. type of cancer, such as B-cell leukemia, and then returns to the patient.
“What happens with CAR-T is those cells go into the patient, and then they see all this cancer and they’re like, ‘Oh, we want to bring staff here,'” Grupp said.
These therapies are a living drug, and each CAR-T cell can kill many cancer cells, which is why often a single treatment is sufficient to bring a patient into long-time remission.
Immediately after receiving the remedy, Emily was given worse things, but then she was given more and more. More than 10 years after her impressive recovery, Emily is still in remission and doing well. The remedy that won was approved by the FDA in 2017 under the so-called Kymriah.
Lowering Gene Therapies’ Steep Costs
Since then, five other CAR T-cell treatments have gained FDA approval and more than 25,000 people have been treated with this type of immunotherapy. But the production procedure is very complex and the value of these tailor-made medicines is high.
“We took a patient’s phones and made them in a two-week process,” Grupp explained. “At the end of the day, we have mobiles where we’ve done a million tests, so we can safely return them to the patient. And the personalized nature means that we do those kinds of tests for both one and both batches, for one and both and for both mobile products that are intended for a patient. These tests are confusing and build increases the load and time.
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Individual doses can cost several hundred thousand dollars, making it difficult to predict a future in which such drugs are used more widely and in other situations. The T cell technique does not yet work for false tumor cancers, such as breast or lung cancer. However, researchers are working on it, and Grupp is mulling over the implications.
“If we need to expand into more common tumors, if we need to expand into countries that can’t have the kind of value that you see in the first world, then addressing the production process is the number one challenge,” he said.
The Children’s Hospital of Philadelphia is moving in that direction. An on-site clean room facility at CHOP now houses a machine that could be a game changer. Stephan Kadauke, Associate Director of the Cell and Gene Therapy Lab, says the CliniMACS Prodigy machine can combine all the raw materials that go into the production of a CAR-T cell and all of it is automatically done by a computer with minimal hands-on time.
“Seven to 10 days later, their CAR-T mobile product is ready to be infused,” Kadauke said.
From what this device achieves, it seems strangely simple, reminiscent of a fancy coffee maker. Kadauke says it’s true.
“The MOT is very similar to the MOT of a coffee machine,” he said. “They gave you those peristaltic pumps, they gave you anything that cools or heats things up, and they gave you a way to push fluid along a plastic tube. In a way, it’s a very undeniable mechanical process.
The device is manufactured by Miltenyi Biotec, of Bergisch-Gladbach, Germany, and other corporations are beginning to produce similar devices. Kadauke and Grupp say they first had to accept and approve this on-site production procedure through the FDA, which involved a lot of paperwork, but now it is helping to make the drug much cheaper.
“In our express environment, where we already have a mobile lab and a lab medicine department, we can manufacture the product at a much lower cost than the advertising product,” Kadauke said.
He says they’ve done load analysis that comes with hard work and testing.
“This doesn’t include facilities and anything else,” he explained. “But we can do this for about $30,000 per patient.” Drug maker Novartis quoted the price for Kymriah at $475,000 in 2017.
And now, Kadauke is building foreign relationships to expand this procedure to more hospitals. For example, it is collaborating with Brazil’s National Cancer Institute to offer lower-cost mobile CAR-T treatments to treat children with leukemia in Rio de Janeiro.
“Price is one of the factors, but the other factor is that we’ve spent a lot of time figuring out how to make it work for us, how to convince the FDA that it’s safe,” he said. Comprehensive written folk operating procedures that incorporate much of our knowledge and experience, and we think it’s a very smart thing for us not to keep it under lock and key ourselves, but to use that wisdom for patient access. .
For Grupp, solving this question of scale and burden is, in fact, the next frontier: the next challenge for such therapies.
“It’s been the hope, right, that you start with whatever confusing thing works and say, ‘Oh, this is so exciting that it’s working,'” Grupp said. “And now let’s look for a way to make it more automated and make it more available. “
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Go on an adventure into unexpected corners of the health and science world each week with award-winning host Maiken Scott.
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