Scientists plan to start human trials soon on CRISPR: NPR

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We have news of a new way to fight against cancer. NPR has learned that doctors are using for the first time a powerful new technique of gene editing in the treatment of cancer. This news is only the latest in the technique called CRISPR. Some news has disrupted much of the world, as when a Chinese scientist announced last year that he had used CRISPR to create genetically modified babies. Now, this new less controversial. NPR health correspondent Rob Stein met with Rachel Martin.

RACHEL MARTIN, BYLINE: OK. Rob, let's start with a big picture. Remind us all what is CRISPR, exactly.

ROB STEIN, BYLINE: CRISPR is an entirely new type of genetic engineering that gives scientists the power to modify DNA and make accurate DNA modifications much easier than ever before. Scientists believe that CRISPR could revolutionize medicine, how we prevent and treat many diseases, repair genetic abnormalities, change genes in cells or turn them into drugs.

MARTIN: What was this Chinese scientist doing?

STEIN: He used CRISPR to edit the genes inside human embryos, which means any changes that he would have brought could be passed on for generations to come. In this case, for medical treatments, changes are only made to the individual patient's DNA. This does not raise all the scenarios of the "brave new world" about the reengineering of the human race.

MARTIN: OK. So explain what is happening now.

STEIN: Yeah. What is happening now is that we have finally reached the point where CRISPR is leaving the laboratory to seek treatment at a clinic around the world. Until now, there have been only a small number of studies in which scientists have tried to use CRISPR to treat diseases and almost all have gone to China to treat cancer. Today, scientists from Europe, the United States and Canada are launching a wave of studies to try to use CRISPR to treat various diseases. This is Fyodor Urnov. He is a scientist at the Altius Institute for Biomedical Sciences in Seattle.

FYODOR URNOV: 2019 is the year when the training wheels stand out and the whole world sees what CRISPR can really do for the world in the most positive sense.

MARTIN: OK. So what does it mean? What diseases are we talking about here?

STEIN: Yeah. Thus, the first are blood disorders, such as sickle cell disease and a similar condition, a genetic disorder called beta-thalassemia. And in fact, the first patient with beta-thalassemia has recently been treated in Germany, and others are about to receive their CRISPR-treated blood cells soon in a second hospital in Germany. Then there will be treated patients in Toronto, London. And the first patients with sickle cell disease could begin to get DNA from their blood cells very quickly in this country.

And there is yet another remarkable study, and it's a study that will try to treat a form of hereditary blindness. And the reason is that this is the first time scientists have tried to use CRISPR to edit the genes of the human body. Other studies involve the removal of patients' cells, their revision in the laboratory, and their reinjection into the patient's body.

MARTIN: Wow. These are all very important conditions and diseases. What about cancer, though?

STEIN: Well, Rachel, a CRISPR study is going on in the United States to treat cancer. This is at the University of Pennsylvania. And I have just learned that two patients have been treated for multiple myeloma and sarcoma. However, several other cancer studies are starting soon as part of CRISPR this year in Texas and New York.

MARTIN: OK. So, all this is exciting. All this seems to be very positive, good news. But is there a dark belly here? I mean, are there any concerns about this?

STEIN: Well, of course. You know, every time you try something completely new and more powerful, people are always a little worried that something is wrong. Everyone remembers the beginnings of gene therapy when some patients, you know, have even died. This is Laurie Zoloth. Bioethicist at the University of Chicago, I discussed this.

LAURIE ZOLOTH: All humans on the planet should hope this technology works. But it could work. It may not be. This is unknown. This is an experience. So you need quality care, and you really have to think ahead of time by doing a thorough ethical review of how you do this type of work.

MARTIN: Okay. So, do we have any idea when we will know if these CRISPR treatments work?

STEIN: Yeah. All these studies are therefore very early. They are very preliminary. And they mainly aim to check if these experimental CRISPR treatments are safe. That said, scientists are looking for clues to find out if they could help patients. So we could have a look at it later this year or maybe early next year.

MARTIN: NPR Health Correspondent, Rob Stein. Thank you very much, Rob.

STEIN: Thanks, Rachel.

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