Custom Drug Treatments for Children

More from this show

Researchers from Phoenix Children’s Hospital, TGen, and the University of Arizona College of Medicine are working on developing individualized drugs for children with cancer and other diseases, with the drugs based on their genetics. Dr. Robert Arceci is the Division Chief of Oncology at Phoenix Children’s Hospital, and he will talk about the research.

Ted Simons: Local researchers are working on using genetic information to develop individualized drugs for children with cancer and other diseases. Dr. Robert Arceci is the division chief of oncology at Phoenix children's hospital. He joins us now with more on this, very good to have you here.

Dr. Robert Arceci: Thank you.

Ted Simons: This is, this is -- it's always -- you get filled with hope when you talk about this, and what we're talking about, it sounds like what genetic-based treatments for kids.

Dr. Robert Arceci: Exactly right, what we're trying to do is to change the way that we treat patients, in the past, we have for instance, taken 100 patients with a certain type of leukemia, and given them the same three drugs, and half respond, and that is their disease response, and half of them don't respond and, and they all end up in the hospital for a month and, and literally, about 2% or 3% of them will die from the treatments. And it seems barbaric at so many levels, and yet, that has led us to, to many cures, but still, many patients are not being cured, so we're trying to do now is to capture the secrets in the genome, which is in our genes, and to really try to make the cancer much more susceptible by identifying the Achilles heels.

Ted Simons: Can you find that in there or is it still -- in other words, can you do a sniper action or is it a bit of an elephant gun?

Dr. Robert Arceci: It's getting far more precise, and we like to call it precision medicine because we can take, you know, about $2.3 billion base that is comprise our genomes, and actually, sequence all of that. It's like, taking a tale for two cities and cutting it up into individual letters and throwing it in the waste basket and having someone say can you reassemble that? That's what we are trying to do but with the help of smart people and a lot of computer power, we can do that, and actually, identify single changes.

Ted Simons: I was going to ask, what can the genetic makeup of a patient tell you about that patient?

Dr. Robert Arceci: So, it's an amazing amount of information, of course, and this is where you get into some of the ethical issues, so, if we do a genetic sequencing and try to identify key drivers of leukemia, and that we can target, and, and we also need to compare that to the normal DNA, and in that normal DNA, we find sometimes things that, that are, are, for instance, a susceptible to other cancers, or maybe a susceptibility to a disease that could affect other people in the family. So, there are real implications of doing this work.

Ted Simons: What do you do when you find those susceptibilities?

Dr. Robert Arceci: There is a protocol that, that we, and many, and many investigators and the Government and, and ethical bodies have put together, and an approach to, to up front getting consent, telling people what, what to expect, and we, we tell them that, that we will inform them of things that, that we, we can do something about, or that, that are going to be relevant.

Ted Simons: Ted Simons: It sounds like these are clinical trials on kids, for kids, as opposed to the adult component in there, and, and am I wrong? And, and is that, is that different than the way that things have been done?

Dr. Robert Arceci: We have had a problem, in pediatrics and, and in general, but in pediatric oncology, and that is getting new drugs to be tested and to be used in children. There have been several Government acts that have encouraged companies to do that, and they have been partially successful, so, so what we're doing here is to, is to really take children with the worst cancers that we have and, and, and to, to, to try to find those new drugs, and we've been working with the pharmaceutical industry, as well as the Government to try to bring those new drugs earlier to the children.

Ted Simons: And I would imagine because, because you are children focused, that would speed the development of the drugs?

Dr. Robert Arceci: It would, because often, children will respond to, to those, those drugs, much more quickly, much more effectively than adults because of the complexity of the tumors.

Ted Simons: I have heard and, and read on this, that r&d for pediatric diseases has fallen off in recent years. First of all, is that accurate? And secondly, if it is, why?

Dr. Robert Arceci: Well, it's a complicated scenario. We, we do believe that, that children somewhere, have often gotten the short end of, of funding and support, although people do continue, including the Government and, and philanthropists continue to help. But in many ways children don't vote and, and their parents do. But, the children don't vote, and it's a very difficult thing to, to, to bring extra money to the table for the 2% or 3% of patients with cancer, and even though if we cure it every child with cancer, it would have an effect as if we cured every patient with breast cancer.

Ted Simons: And you certainly would say the development, as well, because you have got someone who is young and you can see how far along things might go. And I think that that would be valuable.

Dr. Robert Arceci: And we save a three-year-old's life. It might be the next precedent.

Ted Simons: There you go. And so, how far along are these drugs and what's going on? -- so, we are taking drugs some pharmaceutical industries have developed, and we're also going back and repurposing older drugs to find out if they could work like the newer drugs, which sometimes we cannot get for children, but those other drugs were out there for a long time. So, if we can get those and create a, a way to, to make them for children, that's another way that we can do this.

Dr. Robert Arceci: We had a story last week, regarding with men with advanced prostate cancer, that some of these generic - that no one is testing anymore, they are bringing these things back and showing more promise than the newer drugs.

Dr. Robert Arceci: Sometimes we get lucky on the screening we are doing on pediatric cancers, they are identifying some of these older drugs, so, we have to, of course, manufacture them, and get fda approval to use them in people, but nevertheless, they are sitting there.

Ted Simons: So what is next? What do we look at from the study and the research? What can we expect to see in the headline shortly?

Dr. Robert Arceci: I hope that what we will do is to be able to increase the, the ability to predict responses of cancers to various drugs. Right now, our, -- we do about five to 10% of patients will respond to a drug, and we don't know which 5% or 10%. We would love to utilize the approach to, to bring -- if I could tell a family, or a drug company, that you have an 85%, 90% chance of responding, that would be fantastic.

Ted Simons: It would, and we all wish you and your research the best of luck.
Thank you very much for being here.

Dr. Robert Arceci: Thanks, Ted, appreciate it.

Dr. Robert Arceci:Division Chief of Oncology, Phoenix Children's Hospital;

Illustration of columns of a capitol building with text reading: Arizona PBS AZ Votes 2024

Arizona PBS presents candidate debates

An armed forces bugler playing the trumpet in front of the United States Capitol building.
aired May 26

National Memorial Day Concert 2024

Graphic for the AZPBS kids LEARN! Writing Contest with a child sitting in a chair writing on a table and text reading: The Ultimate Field Trip
May 26

Submit your entry for the 2024 Writing Contest

Rachel Khong
May 29

Join us for PBS Books Readers Club!

Subscribe to Arizona PBS Newsletters

STAY in touch

Subscribe to Arizona PBS Newsletters: