She underwent a stem cell transplant and a round of chemotherapy to treat her cancer. It was the best thing that could have happened. It was very disappointing when they found out that my non-Hodgkin's had exploded back up, after I went back for a PET Scan in May of 2020. Five years ago, she was first diagnosed. His doctors told him he had exhausted his options. That was hard to deal with. Bartolome is 74 years old and lives in Santa Barbara, Calif. In the last few years, some doctors have begun using the gene-editing technique to try to modify cells of the immune system to treat cancer. Even though she says she's a tarot card reader, she volunteered for the study because she wanted to find out if the approach worked.
I'm like, 'Yes.' I would like to sign you up. She says she will be your guinea pig.
Bartolome was also a former NBA player. I thought it was a science fiction movie. Bartolome thinks that was cool.
After getting the experimental treatment more than a year ago, both remain in remission, raising hopes that the method may offer new hope to some cancer patients. The doctor presented encouraging data on 32 patients at the conference. The stories of Bartolome and Kopp were told byNPR.
She says her experience is amazing.
CRISPR, which allows scientists to make very precise changes in DNA much more easily than ever before, had already shown promise for a number of genetic disorders. Attempts to use CRISPR to treat other diseases have been disappointing.
One of the most exciting developments in cancer treatment in decades is called CAR T-cell therapies. These are called "living drugs" because they're living cells of the immune system, taken from cancer patients and then re infused after being genetically engineered to attack the patient's tumors.
In contrast to drugs, this is a living therapy. For weeks to months and sometimes beyond that, your patient can be injected with a drug that is alive. Better CAR T-cell living drugs that are more potent and effective at treating more common cancers are hoped for by McGuirk and others.
"off-the-shelf" CAR-T is the approach tested on the two men. It's made in huge batches that would be ready for any patient, instead of having to wait weeks or even months for custom CAR T-cells made for each patient. Patients who are too sick to wait could be helped by these off- the-shelf therapies. Patients with aggressive diseases don't have enough time to care for them. Some patients will die before the therapy can be generated in the laboratory if they are too sick to receive it.
Custom-made CAR T-cell treatments can be more expensive than off-the-shelf ones. I'm very happy about this. Dr. Carl June is a CAR T-cell pioneer who is not involved in the studies.
To create these cells, doctors take immune system cells from a healthy donor and reprogram them to do three things: leave alone the healthy cells in a patient's body, hide from the recipient's own immune system, and zero in on them.
McGuirk says that the T cell sucks up against the cancer cell, releases molecule that punch holes in the cancer, and releases small machinery. They run through those holes and they chop the cancer cell's genetic material. The results of McGuirk's research were presented on Monday at the American Society of Hematology meeting. According to McGuirk, the approach shrunk tumors in more than 70% of patients with the same type of cancer. Kopp, who's shown no evidence of malignancy for more than two years, was one of forty percent who had a complete remission of their cancer.
McGuirk says this is the most exciting part of his career. I have always been excited by the work we have been doing. This is the first of it's kind.
Similar results have arisen from Bartolome volunteering for a study.
The University of Texas M.D. Anderson Cancer Center in Houston is leading that research. The studies are being funded by a Boston-based company. Iyer says that they have not had a therapy like this before.
The researcher who was not involved in the research agrees that the prospects are better than they were a decade ago. The field is moving quickly.
Some researchers are not as enthusiastic about CAR T-cell treatments. The generic version of the therapy doesn't seem to last as long and may not be as effective as the original versions which rely on a patient's own cells. The main problem we have here is that one. It's quicker. It is easier to do. It's possible that it's less expensive. James Kochenderfer is a researcher at the National Cancer Institute. No matter what you do, that's a fundamental problem that you can't completely solve.
Kochenderfer and Dr. Sadelain agree that there is room for improvement
The results so far are positive. The rate of responses isn't as good as with a patient's own cells. Further investigation is needed.
More research is needed to figure out how well the off-the-shelf approach works, how long it lasts, and how to make the cells work better.
McGuirk says that the number of patients that would have died is a big advance. We are not happy with that. We need to improve. Some of the weaknesses could be overcome by giving patients more than one injection.
The two men are thrilled. It has been more than two years since he was last seen. I joke that I have been a Homeopathic all my life. "I'm genetically modified." There is a small amount of cells that can change my life. Wow! Medical miracle.
The day the doctors told Bartolome they couldn't find cancer in his body will stay with him forever. More than a year ago, that was the case. It was a big deal. He says that he was bubbling up inside. It was a wonderful day. Since then, I have thanked my lucky stars.
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