Rosen: Thyroid cancer is a pretty rare disease. There are only about 50,000 cases every year in the United States. Usually thyroid cancer is a completely curable disease and if not curable it’s very slow growing so it doesn’t tend to be a big clinical problem. If someone develops thyroid cancer, they have their thyroid taken out. They are treated with radioactive iodine and that’s usually it. The cancer will rarely come back or, at the time it’s discovered, already have spread to other organs. Those are the times when traditional therapy completely fails.

How fast does thyroid cancer spread?

Rosen: We’re trying to put it into context. If you compare thyroid cancer that’s advanced to other types of cancers, it’s incredibly slow growing. People can live with it for 10 to 20 years at a time. Tell that to someone who is 45 years old, that they have a 10-year life span, so yes, people can die of it. There are rare cases, not even one a year, that people will have lots of shortness of breath, or the cancer will rapidly grow and they could die within months or years of being diagnosed with the advanced part of the disease.

What are typical treatments?

Rosen: Right now, more than 90 percent of thyroid cancers are taken care of just by removing the thyroid gland, then doing a scan to see if there are any residual thyroid cells somewhere floating around the system and using radioactive iodine as a single treatment to get rid of it. If the cancer were to come back, or at the moment it was diagnosed it had spread, you could give repeated treatments of the radioactive iodine, but you would eventually reach a maximum where it becomes unsafe. Then, that person would have to either look at additional surgeries, traditional kinds of radiation, or chemotherapy. With traditional chemotherapy, the problem is that toxicity from the chemotherapy is usually high compared to how well it works.

So there are a few different types of thyroid cancer?

Rosen: Yes, there are probably about three or four. It depends how fine you want to draw the line. There are different types of thyroid cancers. The bulk of them are what we call differentiated thyroid cancers. They are very slow growing. The kind we’re talking about are usually treated with surgery and radioactive iodine. There are rare cases in which people can have advance disease that will take their lives. The other thyroid cancers are inherited genetically.

What's the process for receiving radioactive iodine?

Rosen: Typically what happens is you have your thyroid removed and you’ll come back a couple of months later and they’ll do a scan to see if there’s any residual uptake -- or a hot spot of thyroid cancer cells or thyroid tissue that might be left there. Radioactive iodine is literally a pill that comes in a metal container because it does emit radiation and you swallow the pill and you usually have to stay away from children or pregnant women for a couple of days and that’s it.

Then six months or a year later, you’ll come back and you’ll have a scan to see if there are any more hot spots. Those people are monitored, maybe once every couple of years if they should need a repeated dose. It's not recommended to do too many repeated doses of radioactive iodine because, beyond a certain maximum, the levels of radiation make you predisposed to get some types of leukemia, so you could have that consequence of having too much radioactive iodine.

Tell me about this new drug and how it works.

Rosen: About 10 or 12 years ago, people started being able to experiment with drugs that affected the blood supply to a tumor. This idea that as cancers grow, they need to create new blood vessels to feed themselves, bring them oxygen and send out signals to grow and to spread, is several years old. Drugs that interfere with this process were and tested on colon cancer, lung cancer, kidney cancer and certain types of gastrointestinal tumors as well.

It's possible it may even apply in breast cancer. There are a number of drugs that interfere with this process now. Way back when, when we were experimenting with some of these drugs as they were coming into human clinical trials, there was really nothing to do for thyroid cancer patients other than the conventional chemotherapy that doesn’t really work, but with thyroid cancer patients enrolled in these new drug testing trials, serendipitously they are all doing great.

Not to say that everybody is being cured, but they were all doing amazingly well, and so we started to ask ourselves, 'Why thyroid cancer,?' It turns out that either the thyroid cancer is very susceptible to these drugs because the thyroid is a very vascular organ. It’s very rich in blood vessels and therefore would be emendable to therapy that’s manipulating the blood vessel or there’s certain gene that’s turned on in certain kind of thyroid cancer that these drugs also hit.

We don’t really know why exactly they work, but as long as they work, I always say that we’ll figure it out later. The drug we are talking about now, is a drug that works not just by blocking the main angiogenesis pathway or the main pathway of new blood vessel formation, but it blocks several other related receptors that sit on the surface of the cancer cell and again, there are several of these drugs that are approved in other cancers. There’s a drug that’s in clinical development, not approved yet, being studied in a number of different diseases and similar to other drugs that are already approved.

How is it given?

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	Health Beat A certain drug could help fight advanced levels of thyroid cancer.

Rosen: It’s a pill. It’s very easy. You can take it once a day, twice a day, three times a day, it depends on the drug and the schedule. We were finally able to convince somebody to do a dedicated study just in thyroid cancer patients to define the response rate, to define how well it works, to define the side effects and answer the question, 'Are these just a couple of isolated cases or is there really something when you study across a number of different centers?'

What do you think the side effects will be like?

Rosen: There are side effects with any drug. This drug can cause profound fatigue, it can cause some mouth sores or sores in the hands and feet, but there are other people that have no side effects whatsoever. I had a gentleman who couldn’t even get out of bed because he was coughing and coughing up blood, but within a week of taking this drug, he was jogging.

Another gentleman on a related drug, not this drug but another very similar agent, has a neck mass that makes it look like he's all tumor from chin to chest, and it softened up within two weeks of taking the drug. His schedule is to take the drug for two weeks on and one week off, so during the two weeks, the tumor shrinks up, during his week off, it grows back again.

What have you found so far in the study?

Rosen: This is a study that many institutions around the United States are looking at. Right now, we're looking at patients with thyroid cancer, all who have been resistant to radioactive iodine or couldn’t receive it, who all were taking the same dose of the drug. That’s the typical what we call a Phase 2 study.

We're basically looking at how well the drug works. So fa,r it showed that probably about 30 percent had their tumors shrink by more than half, or go away completely, and another 40 percent had their cancer, which had been growing, stop growing. So now you’re talking about nearly 70 percent to 75 percent of patients who’ve received some really profound benefits and our longest patients have been on this study for three to three and half years already.

Was the study only for people with advanced thyroid cancer?

Rosen: These are all cases with advanced or metastatic disease, who feel like they have no other options. Again, it’s one of a number of drugs that work in this class of angiogenesis inhibitors, drugs that work by cutting off the blood supply.

How long are people taking this drug?

Rosen: The drug is still experimental so patients have access to the drug as long as they receive benefit from it, and in a cancer stud,y usually you’re stopping a study within 4 months or 6 months because people with progressive disease have died, but here you have patients that are still on 3 years later, so it’s wonderful problem to have. If the drug gets approved one day, the study participants would either go and get drug commercially or most of the time people in various studies will have the opportunity to continue receiving drug for free for having participated in the clinical trial.

What’s the next step?

Rosen: This drug is being developed in a number of different indications just like all the other drugs in its class. I don’t know what their registration will be, whether they will be seeking approval in thyroid cancer or another drug. One of these drugs, whether it’s this one or another one of them will be approved for thyroid cancer soon.

How can people get access to these types of thyroid cancer drugs?

Rosen: The issue is in a disease that’s as rare as thyroid cancer. Often people don’t put in the resources to develop a drug for the market of thyroid cancer patients. These drugs are going to work for different cancers, so people are going to commercially want to target lung cancer, breast cancer, prostate cancer and more common cancers.

What I would tell a patient with thyroid cancer to do is speak to your doctor or get yourself to a local cancer center that’s doing research because there is nothing magic about this particular drug. It’s one of a class that you may be able to have access to because your insurance will pay for it, because there are patient compassionate use programs or you could be enrolled in a clinical trial with one of these drugs that is available.

Are drugs ever the first step for treatment?

Rosen: In this particular case, with thyroid cancer, you want to go through the surgery and the radioactive iodine because the therapy is easier and we know it works so we are talking about people whose disease has broken through. It’s really a general comment for all cancer patients. If you're diagnosed with cancer you want to start with curatives first, you want to start with what’s known first and then you want to make sure you have access to a major cancer center that’s doing research to say, 'Is there something newer and better that I could do now or just keep my eye open for what I might need in the future?'

What does this mean for people with this disease?

Rosen: I think people with advanced thyroid cancer or any type of advanced cancer, there’s hope. Once again, you have options when someone tells you, 'I’m sorry, go home and die.' Be optimistic that there’s always something around the corner.