Chapter 15: The Collaborative Ependymoma Research Network (CERN); Funding Research

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Chapter 15: The Collaborative Ependymoma Research Network (CERN); Funding Research

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Dr. Yung describes his efforts to support research into the rare cancer, ependymoma and talks about the challenges of funding innovative research in a time of fiscal conservatism. He begins by giving an overview of the four types of brain cancers then recounts how the Collaborative Ependymoma Research Network (CERN) and now serves as model for using private funds to fund research. Next Dr. Yung offers his perspective on the conservatism of government funding of research versus private systems that can take risks. Innovative clinical trials require collaboration of government, the drug industry, and private foundations. Dr. Yung then describes a plan now being implemented to bring these forces together to look at the molecular characteristics of different types of glioblastoma to determine which drugs might target them, sharing the financial risk of running the trials. He notes that this approach has some features in common with MD Anderson's Moon Shots Program.

Identifier

YungWKA_04_20140707_C15

Publication Date

7-7-2014

Publisher

The Making Cancer History® Voices Oral History Collection, The University of Texas MD Anderson Cancer Center

City

Houston, Texas

Topics Covered

The Interview Subject's Story - Professional Service beyond MD Anderson; Overview; Definitions, Explanations, Translations; The Researcher; The Clinician; Understanding Cancer, the History of Science, Cancer Research; Business of Research; On Research and Researchers; Philanthropy, Fundraising, Donations, Volunteers; Industry Partnerships

Creative Commons License

Creative Commons Attribution-Noncommercial-No Derivative Works 3.0 License
This work is licensed under a Creative Commons Attribution-Noncommercial-No Derivative Works 3.0 License.

Disciplines

History of Science, Technology, and Medicine | Oncology | Oral History

Transcript

Tacey Ann Rosolowski, PhD:

Very interesting. Hmmm… What about the uh …?

Wai-Kwan Alfred Yung, MD:

Ependymoma? You know, when it comes to primary tumor in the brain, the most common is astrocytoma. The second common is oligodendroglioma. Now in the brain if you re --- if you remember biology of the brain, there are four main types of cells in the brain. One is nerve cell, the neuron, nerve cell and then the nerve cell is actually supported by three groups of supporting cells. One is astrocyte. Astrocyte provides nutrients and spatial support to the --- to the nerve cell. Oligodendrocyte --- Oligodendrocyte is the cell that makes the lining of the nerve fiber to help conduction faster. And the third one is ependymocell. Ependymocell --- In the brain there is a reservoir called the ventricle and an ependymo --- ependymocell line the reservoir and make fluid and move --- propel the fluid. All three types of cells can become cancer --- become tumors. Ast --- When the astrocyte become tumor, it is astrocytoma. When the oligodendrocyte get transformed into tumor, they are called oligodendroglioma. When the ependymocell get transformed into tumor, we call it ependymo --- ependymocell --- sorry ependymoma. Ependymoma. So you have three --- But ependymoma is the least common in adults. Now in children --- in children --- the most common tumor in children is the nerve cell tumor, medulloblastoma, because it’s sort of a, you know, --- there is --- the --- the --- the neuron is still dividing at an early age and that program had gone haywire so formed neural tumor called medulloblastoma. So most of the medulloblastoma is developed --- is developed early and gets discovered early. That’s why it’s a children’s disease as opposed to adult disease. They pick up those tumors in children. Besides medulloblastoma, astrocytomas are very common, then ependymomas also are common in children more --- more than adults but it still is a very small disease. And now I recruited Dr. Mark Gilbert in 2004, you know, to --- to join us from Pittsburgh. Was it Pittsburgh? Yeah, Pittsburgh. And I forgot when maybe 2005 or 2006 or somewhere around there, maybe later in 2006 or 2007, he had a patient who had an ependymoma and the patient’s brother had a foundation and the --- the patient’s brother asked Dr. Gilbert “does this --- how much disease --- how much research is going on for this disease --- this small disease?” and Dr. Gilbert said “not much.” Well then he said, “Well how can we create more research?” The Dr. Gilbert said, “Well we could do that if we have you a --- large sums of research funds.” And the brother said, “Do it. Tell me how much.” Dr. Gilbert said, “It probably takes about 20 million dollars.” He said no problem. So they set up a --- a foundation or --- they set up a foundation and called it the --- The CERN. CERN is Collaborative of Ependymoma Research Network and that --- it is funded by this one family and probably a little more but --- but it is known as The CERN Foundation. This foundation single-handedly fund ependymoma research in adults and children. It’s a joint --- It is a joint project with Dr. Gilbert and Dr. Gilbertson from --- from St. Jude Hospital.

Tacey Ann Rosolowski, PhD:

So is this --- it sounds like maybe this is very unique, that there’s not a lot of research.

Wai-Kwan Alfred Yung, MD:

This is very unique and at least because the --- there’s not a whole lot of research going there because it’s such a small disease. Now if you think about --- about public funding from the government. So when you have a big disease, a big clinical need, and also big efficacy voice, you get more funding. When you have a small disease, not that many efficacy voice to raise, so you don’t get much funding. So you know that if something happened to a rare disease most of the time you need, you know, private funding. And so this family and Mr. Clay, what is his last name? You could probably get his last name if you go on site --- go online and look up CERN, maybe you can find it. He’s not that keen on announcing his name so everything is talked about CERN but --- but --- but this one piece of private funding has publicized the plight of patients with ependymoma because it stimulates a lot of --- especially more like tumor research and drug discovery research in this disease and it’s --- is a model actually. I mean this is a model that we are following even with bigger disease like glioblastoma and I’m working with the National Brain Tumor Society to mount a national effort, you know, 50 million dollars along the same line that there is not enough public funding to really mount a big effort so you really can mount a --- a strong concentrated effort with private funding. We can seek out the --- the --- the talents, the strong scientists and high-impact program to get them to work together to --- to devote more time and more brain power into this problem and we’ll --- we’ll be able to make inroad faster.

Tacey Ann Rosolowski, PhD:

Is it the case too --- I mean I know when I was talking to Gabriel Hortobagyi he was raising certain issues that came up with private versus public funding for research and saying that even with certain private foundations and certainly with government, you know, there’s a tendency to take a safer road in funding research whereas private funds can often be --- they have more freedom to fund things that --- that are innovative.

Wai-Kwan Alfred Yung, MD:

I think he’s right --- he’s right. The system that we set up with NIH with the peer review system. It is --- It is a more conservative system because the peer review system, you know, utilized the view and take the view that “well tell me what you want to do” and this is what you want to do or why do you want to do it? What is the evidence that this is the right thing to do? So you --- they require a lot of preliminary data and it requires you know a --- a sort of --- a lot of safety valves, you knows, check and balance to say you know I think of this but this has not worked as well. So it takes a more conservative way of thinking. In general because of the peer review system it’s set up like this that the investigators do not really --- cannot take risks that much. You cannot say, “I want to do this even though I don’t have a whole lot of data to support why I want to do this.” You never get money from the public system when you say, “I want to do this just because of hunch. I don’t have any data to support why that --- that it will work. Just a hunch.” The public seminar gives you money to do this. The private systems works. If you --- If you say --- You know if you say that, “Gee, we need to take that risk.” Especially you know if a group of advisors get together to say, “We have seen this. We --- We need to take this risk.” And the private funding has the freedom because they don’t --- they don’t --- well they do have to answer to the constituents --- the ones who raise the funds, but they don’t have to answer to a lot of government regulations that say, “Well did you use the money right?” You know? So I do believe that private funding allows you to take more risks which is what we need, to take risks.

Tacey Ann Rosolowski, PhD:

I also wanted to ask you about the clinical research piece because I mean I was just talking to Robert Bast [Oral History Interview] this morning and --- and I’ve talked with other people who do translational research too, who you know have observed that there tends to be you know more emphasis on the pure basic research approach than a clin --- a more clinically based research. And I’m wondering if you feel that that’s also a trend in funding. You know that I mean it’s the basic research that gets into the high-impact journals that maybe attracts more --- more funding more quickly and that if private funding can be used for more clinical approaches, more translational approaches.

Wai-Kwan Alfred Yung, MD:

Well, I mean I think that there’s --- there’s a lot of NIH funding going to the clinical trials and I don’t think there’s enough private funding unless --- well there are s --- there are several high value foundations that they can --- they can fund you know clinical research in a way --- in a scale that the NIH is funding. Because doing clinical trials is very expensive. It’s really very expensive you know and --- and that’s either funded by the federal government or funded by the industry and private foundation funding unless you’re talking about a big foundation like Coleman Foundation, you know, Ford Foundation those --- and Leukemia Foundation. Those big ones, they can handle multi-million dollar clinical trials. Many small foundations really cannot mount that kind of support for clinical trials. I think clinical trials require --- require a lot of --- if you can do it requires a lot of collaboration between the government and the drug industry and the private foundations for us to do more risky, innovative trials. The government funding tend to do, again, more conservative trials and --- and that’s what the cooperative groups are doing, they --- conservative, you know, trials. Early --- But now even though the government and NIH are trying to fund the phase 1 and phase 2,, but phase 1 and phase 2 are traditionally still in the hands of the drug industry and the drug industry funds this kind of trial in big disease because they need to --- they need to get their money back. For a small disease like brain cancer, brain tumor, thyroid cancer, sarcoma they’re always second fiddles or third fiddles when it comes to industry funding where there’s --- this is where the foundation comes in.

Tacey Ann Rosolowski, PhD:

If you had, you know, your wish granted, what would be one or two clinical trials that you would like to see handled in that way. You know, a risky trial funded by government, the drug industry, and private foundations all unifying their money. What would you like to see done?

Wai-Kwan Alfred Yung, MD:

For brain tumor?

Tacey Ann Rosolowski, PhD:

Yeah.

Wai-Kwan Alfred Yung, MD:

Well we are actually doing some planning for that with a group of people and --- and thinking and planning and hoping that we can get these forces together. I mean what we --- what we have now to --- to --- that we need to take risks as we talked about in the research level we have a better understanding of the biology of a tumor. We know that, you know, glioblastoma can be divided in several subtypes with some clinical markings. So what we need to do then --- that --- is you know taking some of these molecular determinant and look at what drug could be matched with the molecular determinant and we can quickly test it. Getting one of drug A and drug B to treat disease A you know and disease B. Drug A and drug B if it does not work for C and D for disease A. Kind of doing these kind of mock-guided trials in a subgroup level. And --- And we can do this kind of trial in a very rapid changing fashion. A does not work, we go to B, B does not work, we go to C, C does not work, we go to D and there are technology for us to do that kind of design. The question is getting the company to allow us to use their drug which they are more happy --- you know, they would be more happy to give it to lung cancer and breast cancer but not as much to --- to --- to glioblastoma so we need to get the buy-in from the drug company saying, “Yeah we are going to come in to allow you --- to give you the drug.” That’s the collaboration you know and then we have money to --- and we share the risk with the company by going “You don’t need to give us money to do the trial but just give us the drugs early enough or give a small amount of money for drug or foundation we could have more money to support the rest of the pot.” That kind can allow us to really take this kind of risk. There’s not a lot --- There’s not a whole lot of preliminary data to say A should work with group A. There’s not a whole lot of preliminary data to say B should work with A also. But what we do --- we carefully design the trial, monitor the patients carefully and we do a more very small number of patients. We can get the test in the patient itself without relying on testing of animals.

Tacey Ann Rosolowski, PhD:

What’s --- So this is the planning stages? Have you --- Have you had conversations at all with drug companies about this?

Wai-Kwan Alfred Yung, MD:

We’re beginning to through the foundation and through the National Brain Tumor Society.

Tacey Ann Rosolowski, PhD:

Because it sounds almost like I mean it’s reminding me a little bit of some of the --- the mindset of the Moon Shots, you know, that kind of view the short --- short. So I was right?

Wai-Kwan Alfred Yung, MD:

That’s kind of in the mindset of the Moon Shots. You --- You --- I mean in Moon Shot we --- we say you know what --- where’s --- take the lower hanging fruit and materialize the lower hanging fruit first. And --- and here I would cause more risk taking and de-risking each other so they would bring the company early de-risking them with foundation money and ---and also with the government comes in to also de-risk the company because you --- you allow some of the, you know, --- you allow the risks to be taken through the government red tape and also give the company that if we do this we may be able to forego some of the red tape and keep the registration down.

Tacey Ann Rosolowski, PhD:

That’s kind of adapting the Moon Shots approach to a situation which as you said the last time there isn’t any low-hanging fruit for glioblastoma. So you kind of have to adapt the model to the reality of that situation. Yeah. Interesting. Very interesting.

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Chapter 15: The Collaborative Ependymoma Research Network (CERN); Funding Research

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