Two US patients with cancer treated with controversial CRISPR technology

The genes of two human patients have been modified in one of the many CRISPR clinical trials currently underway in the United States.

The University of Pennsylvania uses revolutionary – and sometimes controversial – technology to treat patients with myeloma.

A university spokesman confirmed that two patients had been treated in an email addressed to NPR.

The University of Pennsylvania (U Penn) trial comes just months after Chinese scientists seized the wonder and anger of the international community when they announced the birth of twin girls whose gene had been modified in the uterus.

But U Penn's study (along with some 25 other people in the United States and abroad) only deals with mutations in adult patients, dismissing ethical concerns about the modification of the hereditary human genome born from the Chinese experience.

The invention of CRISPR marked a turning point for the medical community.

Instead of trying to solve problems resulting in errors in the genome, the gene editing tool could allow scientists to repair the DNA itself.

There are 26 CRISPR clinical trials registered with the FDA.

Several of them aim to use the gene editing tool to remove sections of DNA that cause cancer cells to grow uncontrollably and replace them with healthy genes.

U Penn uses CRISPR to treat several types of cancer, including melanoma, sarcoma and multiple myeloma, in patients for whom other treatments have failed.

So far, only two of the 18 people enrolled in the clinical pathway have received advanced treatment.

One person has myeloma, while the other is fighting sarcoma.

They had both been treated with more established therapies, but had since relapsed, reported NPR.

The U Penn test is a kind of merger of two of the most telling scientific developments of the last decade: CRISPR and Immunotherapy.

After extracting the immune cells from the test participants, scientists use CRISPR to modify these cells.

Instead of using a virus to strengthen the immune system, CRISPR modifies the DNA of these cells to make them natural killers of cancer.

Then the cells are reinjected into the patients.

It is too early to tell whether the treatment is effective or not, and the study should not be completed before 2033.

At the same time, US and Broadworld researchers are entering the early stages of clinical trials to use CRISPR to treat genetic disorders of the blood, including sickle cell disease, a form of blindness and other cancers.