For the first time in the United States, researchers use genetic editing to treat cancer

The first attempt to use a genetic editing tool (CRISPR) for cancer treatment appears to have been safe in the United States. In total, three patients were treated in this first clinical trial. For the moment, however, it is still too early to conclude on improving the chances of survival of the sick, said the doctors last Wednesday.

The researchers were able to collect T cells from the immune system (from the blood) of patients and genetically modified them to help them recognize and fight cancer, with minimal and controllable side effects.

The treatment developed has the effect of removing three genes that could suppress the ability of cells to attack the disease, and adds a "fourth feature" to help them do the job.

" It's the most complicated cellular and genetic engineering that has been attempted so far,  " said Dr. Edward Stadtmauer, director of the study, at the University of Pennsylvania in Philadelphia. " This is proof that we can safely edit genes for these cells  ."

After two or three months, one patient's cancer continued to deteriorate and another was stable. The third patient has been treated too recently to find out how she will cope. The plan consists of treating another 15 patients and assessing the safety of the treatment.

" It's very early, but it encourages me a lot, " said an independent expert, Dr. Aaron Gerds, a cancer specialist at Cleveland Clinic. Other cell therapies for some blood cancers "have been a resounding success, taking incurable diseases and healing them," and gene editing could help improve them, he said.

Revive the cells to fight the disease

Gene editing is a way to permanently change DNA to tackle the root causes of a disease. CRISPR is a tool for cutting DNA in a specific place. It has long been used in the laboratory and is currently being tested for other diseases.

This treatment is not intended to directly modify the DNA in the body: instead, it seeks to eliminate, modify and give back to the patient very powerful cells to fight cancer. A form of immunotherapy.

In this January 2019 image, CRISPR-published T-cell containing pouches are prepared for administration to a patient at the Abramson Cancer Center in Philadelphia. The first results, released on Wednesday, November 6, show that doctors have been able to take cells from patients' immune systems and genetically modify them to help them recognize and fight cancer. Credits: Penn Medicine via AP

Chinese scientists have reportedly tried CRISPR on cancer patients, but this is the first study of its kind outside of this country. It's so new that it took more than two years to get approval from US government regulators before it could go to trial.

The first results were published by the American Society of Hematology. Details will be given at its annual conference in December. The study is sponsored by the University of Pennsylvania, the Parker Institute for Cancer Immunotherapy in San Francisco and a biotechnology company, Tmunity Therapeutics.

Multiple myeloma and sarcoma

Two of the patients in the study had multiple myeloma of the blood (a cancer of the blood) and the third a sarcoma, a cancer that forms in connective or soft tissues. All had already done standard treatments that unfortunately failed. Recently, more promising options were offered to them, including this essay.

A unique treatment based on genetically modified T cells

Patients' blood was filtered to remove T cells (real soldiers of the immune system). The latter were then genetically modified in the laboratory and then returned to patients by the intravenous route. This is a unique treatment.

Once administered, the cells multiply in the body and act like a real army. " Until now, the cells have survived and multiplied as expected, " Stadtmauer said.

" This is a brand new therapy, " so it's unclear when anticancer effects will be observed. It will therefore be necessary to follow these patients longer and carry out more clinical trials, he concludes.


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