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Thursday, June 10, 2010

The Debate Goes Thermonuclear

by Jennifer Phillips, Ph.D.

Editor’s note: This is a planned public debate between Mark Dunning and Jennifer Phillips, the two primary contributors to this blog. The ideas expressed in the posts during this debate will be purposely provocative and unfinished to invite a response from the other party. We hope you find the discussion valuable.

So the good news here is that Mark and I agree on many of these debate points. This is great because it gives us a stepping off place from which to brainstorm about our common goal of improving the flow of information between researchers, clinicians, and families. The bad news (well, bad for Mark, anyway) is that before I can deal with that good news I have to don my sturdiest firefighting attire and tackle the noxious and incendiary content emanating from portions of Mark’s last post.

Figure 1: “Dear Dr. Phillips” preparing to rush into the blazing thicket of WRONG!

Stop, Drop & Flip-flop

In the intro to his last debate post, Mark concedes the point that research scientists do, in fact, talk to each other—or does he? After opening with comments that seem contra his original position, he claims to never have held that position in the first place, stating “I merely said that researchers give the impression that they don’t talk to each other”. Well thanks to the marvels of the internet , we have a handy record of what Mark actually did say, back on April 29th. To wit:

“Researchers are loathe to discuss any research that is currently underway because until the results are not only in but have been peer reviewed, they can not be trusted to be fact…Many researchers take this secrecy to the point that they won’t even discuss what they or others are working on.”

Hmmmmm…Well, perhaps he meant to say that “Researchers merely give the impression that they are paranoid control freaks”. Either way, I think we can consider this “impression” falsified, at least in the generalized manner in which it was originally applied, and move forward.

Shouting “Fire!” in a crowded Symposium

In mulling over how to reply to Mark’s June 3rd post, relating his experiences at the Usher Syndrome Symposium, I reconceived our dialogue in in the form of a 911 transcript. The ‘Caller’ script recapitulates portions of Mark’s last contribution to this debate:

Dispatcher: “911, what is your emergency?”

Caller: Help, Operator! “Few families actually saw what was going on or heard any of the presentations” and “The information was also brutally technical and scientific”!!

Dispatcher: Stay calm, sir; given that this was a professional conference targeting clinicians and scientists rather than families, unrelenting technobabble was completely appropriate—necessary, even!—to convey the important details of these cutting edge scientific studies.

Caller: But…but, “The Researchers Are Too Distant From Their Real Purpose!” “I got the sense that a child with Usher was a rarity in their lives when, in truth, they probably should all have a picture of a patient with Usher taped above their door.”

Dispatcher: Sir, please, you seem to be having difficulty separating your personal investment in identifying a cure for Usher syndrome from other more professional motivations for pursuing research in this area. Let’s say you can persuade every researcher currently working on Usher studies to prominently display a picture of an Usher patient. It probably wouldn’t be difficult, as you concede that at least some of the researchers you have interacted have acknowledged the important perspective that patient interactions can offer. Then what? Will the presence of these pictures inspire formerly indolent or indifferent researchers to greater works? The insinuation here is that researchers who don’t have regular, personal contact with Usher patients (or, indeed, this could easily expand to any basic research field with potential clinical impact) aren’t sufficiently motivated to fully apply themselves to their professional endeavors. That’s deeply insulting, and, more importantly, spectacularly wrong. To suggest that we’re just not that into it because we don’t have a personal stake in the outcome is an absurd mischaracterization. It is undeniable that a personal connection to Usher syndrome would be motivating to someone contemplating a career in research and/or medicine. However, it is far from the only motivator, and absent any further evidence, I reject the assertion that this personal stake is a prerequisite for professional commitment.

Caller: Well, yeah, but “In the end, the families are all that matter. Even if you were an empty hearted researcher you would have to recognize that without Usher families, you don’t have a job. No one cares about developing an Usher syndrome animal model if no one has Usher syndrome!”

Dispatcher: Sir, human biology is incredibly complex. And within that broad category, Usher syndrome is just one of many phenomenally complex diseases. As thinking feeling human beings, we hope fervently that our research will to contribute to future treatment options. As professional scientists we are required to focus on the really nitty-gritty complexities of the system—and we like it! Most of us didn’t become Usher researchers because we know someone with Usher and wanted to help them. Most of us got here because of a deep and abiding interest in understanding complex molecular/cellular sensory processes. Usher syndrome research provides us with abundant complexities to which we can apply our collective decades of scientific training and problem-solving skill sets. Furthermore, just like content of the meeting that you found so dense and unpalatable, it is necessary for scientists to consider the fine and intricate details of Usher pathology at the cellular and molecular level. That we may not also consider the existence of patients and families awaiting a cure from the fruits of our labors during every phase of the experimental process is NOT a liability. In fact, I should think you’d prefer us to have our full attention on the nitty-gritty. Let us attend to the task of figuring out how things work with obsessive, laser-like intensity. Once we gather those data, we can proceed to the logistic steps of incorporating them into a treatment option, but we cannot skip or hasten the first step without running the risk of laying a shaky foundation for all that is to come.

Banking the embers

Ok, I think the smoke and fumes have dissipated enough—for now—to get to the more substantive issues. For me, those issues involve identifying ways in which we ALL--Patient families, Clinicians, and Researchers--can improve and expand our communication with each other

The concerns Mark and I have voiced about withholding or selectively disseminating information, about media coverage, and overall about patient access to the research findings that could benefit them are complex, but addressable. The tricky part of this, of course, will be agreeing on the best ways to address these problems. Discussion of the pros and cons of different solutions will, I think, be the principal topic of the remainder of our open debate. I have some specific ideas about how to proceed; I know Mark does too, and it’s probably not too much of a stretch to predict that our respective opinions on what constitutes the best choices will not perfectly align. But it’s a conversation worth having, and I’m looking forward to the process. As we flesh out some of these ideas for public consumption, I hope that our readers will get involved in the discussion as well, so don’t be shy about commenting!


Dianrez said...

Regarding taping a picture of an Ushers child above the door:
Better idea: tape a picture of the entire student body of any school for the deaf. All those fresh-faced alert young kids, from the smallest to the most gangly teen. Caption it this way:
TEN TO FIFTEEN PERCENT OF THESE CHILDREN HAVE USHERS SYNDROME. It's not far off the mark...some schools, especially in the South, have more. Most do not find out until their teens, if at all.

sabrina said...

I want to answer to the previous comment in this post, and in some ways to Mark’s previous post in the debate. (If you have not read my last comment, I am a researcher working on Usher Syndrome.) I think the point Jennifer was making is that we do not need motivation to “remind ourselves why we are doing research”.

Researchers do not have a “9 to 5” job at the end of which they can go home and think about something else. Researchers give their life to their research. Our schedule is “what ever it takes to have things done”. We spend long days, week-ends, sometimes nights. When we go home, we don’t relax: we are still thinking about the result we got, about the reason why the experiment didn’t work, about what should be done next. We are truly sacrificing a lot of our life, and this even includes time with our children, to bring the research forward.

And we have chosen to do it. Why? Not for the fame or the money (there is none in research). Not because we have someone in our family or in our group of friends with Usher syndrome. We do it because we are dedicated to understanding the process of the disease and ultimately finding how to help people with Usher Syndrome. We do not need a picture of (or to be involved with) a kid or an entire community with Usher syndrome to give up so much of our lives to research and ultimately help people we don’t even know. We just do it.

Anonymous said...

Hi Jennifer,

Thanks for letting us know about the researcher's side of things. I, along with many others, desperately want a cure for blindness, so I suppose we can get rather frantic about it. I wish there was an ambitious and official national goal (complete with a hefty bounty) for getting a meaningful cure for retinitis pigmentosa into the hands of the public by 2020. I really wonder what would expedite progress. I think the biggest thing that I personally feel is the lack of cause and effect. If I raise a certain amount of money, how much does that progress research towards curing autosomal recessive retinitis pigmentosa? (I have Usher Syndrome Type II, I believe). I also eagerly follow along on the latest research, but it's frustrating when there's no indication of what the next step is for a particular research venue. For example, I am very interested in the potential of aminoadipate to trigger dormant Muller glial cells into turning into progenitor cells and then into useful retinal cells that become photoreceptors and so on. Yet the last I've heard about it is in 2008, that there was some success using aminoadipate in restoring vision to mouse models of RP and macular degeneration. Since then, however, I haven't learned anything new about that particular research, even when I looked at the researchers' website at Schepen's Eye Institute at Harvard. I would really like to know how the research is progressing (that it's even still being worked on) and what the next step is and WHEN it's expected to take place. I like rough time-frames fully realizing that new discoveries can either speed or slow down research. I would also like to know what challenges have come up that might be slowing it down (financial or biological). Would you think it'd be okay to write to specific researchers like this and ask them questions? I don't want to bother someone but I really would like to know. Do you know anything about this particular research's progress by the way? I must be rambling by now, but I hope it conveys my sense of disconnect. I need something to reasonably hope for, and at the very least, continual progress reports help. I also want to know what I and others can do to really help things along. If there's a particular avenue of research that I think is promising, I'd like to be involved in making it happen much sooner than later.

Thanks again for all the information you are providing us and I look forward to your response and future blog entries.

Jen Phillips said...

@ Diane: It is becoming clearer to us all the time that Usher is more prevalent at the population level than previously believed. We definitely need to work using this information to change the standard of care for hearing impaired children from birth.

@Sabrina: Thanks for your support!

@Anonymous: great questions, and thanks for reading. I saw quite a few talks from Schepens folk at the ARVO meeting last month. Everything presentation I heard on their emerging therapies was stem-cell based (iPSCs, etc.). The Müller cell research is definitely continuing in many labs around the world. We know these cells can become stem-cell like in order to give rise to replacement neurons in response to injury. The trick is identifying what regulates this behavior (at the cell/molecular/genetic level) and then figuring out how to harness it for therapeutic use. Unfortunately, this is the kind of thing that it's really hard to put a timeline on, at least until it gets close to preclinical trial readiness. I will probably do a post about Müller cells in the near future, so watch this space, as they say.

In general, the best way to stay abreast of the latest research efforts and various timelines to clinical trials is to stay in touch with FFB, the Usher Syndrome Coalition, and this blog. Both the former sites really stay current with the progress and post updates regularly. FFB is also working on a way to give patients access to info on clinical trials. I will continue to blog about relevant peer-reviewed research here. We really do want to work on giving patients better access to the full range of current research, but I know it's hard to wait for it all to come together. Thanks again for your comment