First Published Jan 7, 2011

Lucia Notterpek Ph.D, leads Florida researchers who are focusing on damaged nerve coating as key to finding a cure for trigeminal neuralgia

Lucia Notterpek and Douglas Anderson at Facial Pain Research Foundation

Board Meeting January 7, 2011

One known culprit behind the piercing, repetitive pain of trigeminal neuralgia is damage to myelin, the waxy coating that insulates nerve fibers against electrical signals that are transmitted from cell to cell. Loss of myelin on the trigeminal nerve causes a short circuit that results in facial pain.To define how myelin defects cause havoc in the nervous system, University of Florida Neuroscience Professor Lucia Notterpek, Ph.D., is leading studies of myelin biology and disease. She is addressing basic questions about healthy myelin, which is vital to the normal rapid movement of electrical impulses through the nerve pathways, and about various nerve disorders, including hereditary demyelinating neuropathies, which result from myelin defects or deficiency. She seeks information that will aid the development of ways to repair or rebuild myelin in order to cure trigeminal neuralgia and other demyelinating diseases.In one project, she is carrying out experiments in mice aimed at defining the role of myelin-producing genes in nerve disease, and in another more clinically oriented study, she is evaluating the effectiveness of various pharmaceutical compounds designed to protect nerves. New and better compounds are urgently needed for trigeminal neuralgia, since all medications currently prescribed for this disorder have side effects ranging from bothersome to intolerable.

“We’re finding genetically altered mice are a very good model for investigating myelin damage,” said Notterpek, who also chairs the Department of Neuroscience at UF’s College of Medicine. “We have a known genetic defect—specifically a deficiency of the peripheral myelin protein 22 gene that is linked to myelin damage. We can induce temporary nerve compression that mimics the condition in classic trigeminal neuralgia, and we can measure sensory response in the animals. Later, after sacrificing the mice, we can analyze the status of the nerves at the molecular level.” Co-investigator, Andrew Ahn, M.D., Ph.D., an assistant professor of neurology, neuroscience and anesthesiology specializing in the treatment of headache and facial pain, has expertise in assessing pain behaviors in mice. By measuring behavioral responses to mildly uncomfortable stimuli such as heat, researchers can detect abnormal pain behaviors in mice in comparison to responses by healthy mice. Ahn said the sensory responses observed and measured in mice cannot be directly equated to human pain, but a series of behavioral approaches aimed at analyzing the animals’ sensitivity to various physical stimuli can be used to approximate various aspects of pain in humans.

Currently, Notterpek seeks to figure out how the PMP22 gene normally works to help produce myelin, and what happens in disease conditions to cause this gene to get stuck in nerve cells rather than traveling to the myelin sheath where it is needed. She refers to this problem as “mistrafficking or abnormal expression of genes, like a traffic jam in part of the nervous system.”

She said daily, repeated observations of mice with known myelin defects reveal a puzzling observation: under the same testing conditions using the same type of stimuli, some of the mice show hypersensitive responses that indicate discomfort, while others show delayed sensory response. In a testing procedure now well-practiced in the UF lab, mice are placed in a clear box while a researcher shines a warm light on their feet, creating a situation similar to walking barefoot on hot sand. The hypersensitive mice move their feet within two to three seconds, while other mice don’t seem to be bothered by the warmness of the light for longer times. The responses of the animals are monitored and recorded by a computer for subsequent analysis. Notterpek said correlating specific gene deficiencies or gene abnormalities to myelin damage and to indications of pain requires long, tedious research that can involve behavioral assessments in hundreds of mice.

In previous studies that resulted in two published papers, she discovered that mice generally have to reach a certain age before they show signs of myelin deterioration—similar to the way TN most often affects people over age 50. Now she and Ahn are investigating how myelin damage in different parts of the nervous system may result in different levels of discomfort. They note, for example, that longer nerves such as those in the hind legs are more susceptible to damage than the shorter nerves.

Notterpek also is testing a hypothesis that the peripheral nerves in mice with PMP22 gene deficiency are more sensitive to injury, and require little provocation to initiate myelin injury. She seeks to determine the least amount of nerve damage needed to trigger loss of myelin in normal mice, and in mice that are deficient in the PMP22 gene. At the close of each study, the mice are sacrificed, and the nerve is removed and examined microscopically to assess the status of the myelin. The researchers also measure the levels of molecules involved in both the myelin sheath pathway and the pain pathway.  

In parallel studies supported by a grant from the National Institutes of Health, the UF team is testing specific nerve-protective compounds—one well established drug and others newly developed. Through a new pilot study, they are beginning to investigate additional commercially available compounds and dietary supplements that may affect the pathways believed to be important to normal myelin formation. They are just starting to test the effects of these compounds on cells that make myelin.

Notterpek’s myelin research is funded in small part by the TNA-Facial Pain Association, which has its national headquarters in Gainesville. She and Ahn currently carry out the TNA-FPA-supported research with one lab technician and the help of non-salaried students on scholarships. They hope to publish the first major manuscript on their findings in 2011.

Although Notterpek has only been at UF for 11 years, her studies of myelin damage and myelin restoration began almost 20 years ago while she served as a research assistant at the Gladstone Institutes of the University of California San Francisco. After entering the doctoral program in neuroscience at UCLA, she completed her thesis on a topic related to multiple sclerosis. She earned her Ph.D. in 1994, followed by a five-year postdoctoral fellowship in neurobiology at Stanford University. Coincidentally about this time, neuroscientists first identified the PMP22 gene and several other genes that play a role in eroding or chipping away myelin from nerves.

While at Stanford, she became involved in a project aimed at determining more precisely how the PMP22 gene may be implicated in myelin injury. When she left Stanford in 1999, she took this project to the University of Florida where she has been working on nerve damage ever since. Her large research program is aimed at translating discoveries in the mice to applications in patients with trigeminal neuralgia and with hereditary demyelinating neuropathies. In both conditions, damage to myelin disrupts the normal transmission of nerve impulses.

Eminent Scholar Douglas K. Anderson, Ph.D., who formerly chaired UF’s Department of Neuroscience and who supports plans for Notterpek’s research, suggests several approaches to myelin repair might be tested in the myelin-deficient mice. One approach would involve patching the demyelinated region of the trigeminal nerve with myelin-producing cells derived from adult or embryonic stem cells, or with the patient’s own Schwann cells, provided these cells do not carry a genetic defect that produces unstable myelin. (Myelin is made up of Schwann cells.)

Anderson also proposes, “Gene therapy might be used to deliver genes that produce endogenous factors known to control pain and/or to knock out genes found to be involved in causing pain. With this approach, it might be possible to manage the pain of TN without actually repairing, or even identifying, the defect producing the pain.”

Anderson is a trustee and scientific advisor for The Facial Pain Research Foundation, who is helping to establish its international consortium of scientists working to cure nerve-generated facial pain. He is internationally known in scientific circles for his development of the first effective medication for acute spinal cord injury and for his pioneering studies of embryonic tissue transplants to halt complicated wounds associated with paralyzing spinal cord injury in people.

Hi,
I came across this website friday 29th June 2012. I have been researching for around two years now in regards to what could be the cause of my sons sudden pain in his cheek and forehead. My son is called riley, around the age of two riley started with funny episodes where he would get facial pain in his cheek and head. This made him sick on several occasions, he would also sleep for sometime after the episodes. 

Riley has been under his paediatrician since they started who thought possible seizures was the cause. Riley has had EEG, ECH metabolic blood tests and urine samples. All were reported as satisfactory. Riley's last appointment was on Monday 11th June 2012 which his paediatrician left us with no answers and asked us just to keep my son off his wii and see how he goes. He was also discharged, so we was still left in the dark. On Friday 29th of June riley woke crying in pain around 11pm we gave him paracetamol. He cried for around 30-40 minutes, in pain holding his cheek.

Asked me "mum why does this keep happening to me" his voice desperate for answers. Riley was 5 in January, this has gone on for some time now and we are still left with no answers. Having to explain to my 5 year old son that I don't know is just not the answer. I sat frantically trying to research Riley's symptoms on the Friday night. I came across this site where I found a story called Emily's story. Emily's story sounds exactly the same as Riley's.

Riley's and Emily's symptoms are very much the same. I have now written to my sons paediatrician with details so hopefully they can look into TN, I would appreciate any information or advice you can give me. I'm desperate for some answers as I can't stand seeing my son like this any longer.

Thank you for taking your time to read my email.

THANK YOU PAT AND AMY TOMASULO

AND CHICAGO

LAUGH YOUR FACE OFF Has RAISED OVER $1,200,000!!!

By Kris Gaganidze

The Fourth Annual LAUGH YOUR FACE OFF was held May 8, 2018 in Chicago’s beautiful neighborhood of Lincoln Park at the Park West.  This marks a new date and a new bigger venue!  All of this helped to make this year’s LAUGH YOUR FACE OFF a huge success. With temperatures in the high 70’s and a beautiful sunshine day to come out and help support The Facial Pain Research Foundation (FPRF) in their fight to find a cure for Trigeminal Neuralgia (TN).

To date LAUGH YOUR FACE OFF has raised over 1.2 million dollars for research for a cure for the rare nerve disorder called Trigeminal Neuralgia.  The   Foundation is currently funding six different research programs to find a cure for Trigeminal Neuralgia.  TN is known to be one of the most painful conditions known to humans.  It is often called the suicide disease because there is no known cause and no cure.   

My husband, Kyr and I had the great pleasure of meeting Michael Pasternak six years ago.  He is one of the people who started the Foundation looking for a cure.  We got involved with Pat and Amy Tomasulo four years ago when we were asked to help with LYFO.  What an honor to get to work with and know these amazing people.  They are the brains and Founders of the brilliant LAUGH YOUR FACE OFF! This year’s LAUGH YOUR FACE OFF took place at the Park West Chicago.  The venue was twice the size that we’ve had in the past.  So over twice the number of people (700) attended Saturday’s event.  The evening started with cocktails, appetizers, silent auction and raffle!

My husband and I had the privilege of checking in VIP guests, sponsors and TN patients.  What a joy to thank our sponsors personally and give hugs to TN patients as they entered the event.There was a separate raffle set up just for the TN patients.  I think they appreciated the thoughtfulness of a special raffle just for them!  And a huge shout out to all of the returning and new sponsors!  We appreciate your generosity and support! 

The evening started with the very funny Pat Tomasulo warming up the audience with his great standup comedy.  After Pat got the crowd laughing we never stopped.  He was followed by five, yes five great standup comics.  Thank you to Rocky Laporte, Brian Smith, Jeannie Doogan, Drew Frees and Mike Stanley.

Next, the beautiful Amy Tomasulo read the raffle winners, which was exciting for all of the winners of those amazing packages!  

Followed by two special videos of TN patients, which gave us deep insight and understanding into the daily toll that TN takes on a person’s quality of life.  It’s amazing to see the impact on the audience that those videos made.  It was emotional, inspiring and heartbreaking.  These two brave TN warriors brought tears to many people. 

Then the big give which raised twenty five thousand nine hundred dollars in a matter of minutes!  I continue to be astounded by how generous people are.  

LAUGH YOUR FACE OFF an event where I was surrounded by friends, family, doctors, scientists, TN patients and many supporters.  Everyone was there for a reason, to find a cure for Trigeminal Neuralgia.  Every person I met that evening truly cares about the patients and supporting the research for a cure.  LAUGH YOUR FACE OFF is a huge success because of all the people who show up to show that they care.  Thank you for a beautiful night that gives each and every TN patient hope for a better tomorrow.

Why Is Research

Important

By Kenneth Casey M.D., FACS       

Clinical Associate Professor of Surgery (Neurosurgery) Michigan State University SOM

Clinical Associate Professor of PM&R Wayne State University SOM

Surgical Director Intensive Care Oakwood Southshore

Have you ever gotten up in the morning and been very excited about the prospects for the day?   You’ve have a new friend to meet or you’re going on a trip to an area you’ve never been or you’re going to revisit and reconnect with an old friend.  All these are exciting because something new is going to happen, something different, and you’re going to come away with new feelings and different perspectives.  Research is exactly like that.  The origin of the word from the old French says exactly that, that you’re going to look very intensively for something( Old Fr. recherche). 

Researchers get involved because they get excited about something that’s not known or something that’s not completely explained and they dedicate literally years of their lives, days of their weeks, and hours of their days trying to find these bits of information.  What’s even more fun for a researcher though, is finding other pieces of information, little sidebars as it were, that were not known before.  These sidebars are important ,not as a detour, but as an increasing amount of knowledge that’s adding to the general fund of things we know and things we can work with in our lives.  People talk about the fact that research is important in medicine because it makes it possible for some cures or some improved treatments and that’s certainly the case with the Facial Pain Research Foundation. It means much more than that because if we can find how something works, whether it be something in our own body or something outside our body in the atmosphere or something outside ourselves beyond the planet, we’re going to be that much richer for the experience.  We also know that research allows us to understand several sides of a given question. 

For years the world was flat and everyone believed you could sail off the end.  Researchers and explorers showed that based on their calculations from the time of Galileo and da Vinci to the times of Columbus and other explorers that that’s not true. It is a circle of land, water, and people. The world exists as a collected community.  Research can provide truth and put paid to certain mistruths or errors in knowledge that may hold us back and may stop us from going in the right directions. It’s a way to support day to day life and to support fundamental truth. 

Research offers us all an opportunity to pursue more each day when  we get up and when a researcher puts together a hypothesis and begins the steps to take that hypothesis piece by piece and develop it into a theory and the theory translates into practice, every single one of us will benefit. 

Research is important because we go back and search again through what’s happened and look again at what has already been written down and done and with a very critical eye and an unparalleled intensity, we ask what more can we do, what more can we accomplish, what more can we learn. 

Mervyn Rothstein

Interviews

Douglas K. Anderson, Ph.D.

In 2018 Douglas K. Anderson was the recipient of the US Congressional Certificate of Recognition and The University of Florida McKnight Brain Institute established the Douglas K. Anderson Facial Pain Research Program to fund future research to find cures for TN and neuropathic pain.

Douglas K. Anderson, Ph.D., is eminent scholar professor and chair emeritus of the Department of Neuroscience at the University of Florida, College of Medicine, former director of the McKnight Brain Institute at the University of Florida. He is director of research and a trustee of the Facial Pain Research Foundation. He was interviewed by Mervyn Rothstein, who was a writer and editor at The New York Times for nearly 30 years and now writes the Stage Directions column – a series of interviews with theater directors – for Playbill.com. His first symptoms of trigeminal neuralgia occurred in 2005

Why did you want to be a scientist, and how did you become one?

I had a cousin who was a physician, and he kept after me to go into medicine, saying we would go into practice together. So between my junior and senior year at the University of Texas I got a summer internship at MD Anderson Hospital in Houston, which is a world-class cancer hospital. I was there for about three months, and about a month into it I realized that if I had to make a living as a doctor I would starve to death, because it was difficult for me to work with sick people. I was dealing with pediatric cancer cases and I would go home and cry. But I had spent about half my time working in the research laboratory, and I loved the lab. So I went back to school and changed my major from zoology pre-med to straight zoology. I didn’t tell my parents until Christmas – which was an interesting Christmas. And I went back to Anderson Hospital after I graduated and worked with a Ph.D. instead of an M.D. I got my masters at the University of Houston, and my Ph.D. at Michigan State in 1972.

You worked for 33 years for the Veterans Administration. What kind of work did you do?

I was there from 1973 until I retired in 2006. The V.A. was very good about allowing us to have appointments at medical schools, so I had a dual appointment. I was working on spinal cord injury both with my academic appointments and at the V.A. I was for a year the Department of Veterans Affairs Deputy Director of Medical Research.  

Why after more than 30 years of researching spinal cord injury did you decide to turn your efforts to finding a cure for trigeminal neuralgia?

The main reason was that spinal cord injury was so complex, because the spinal cord is so complex. We were working on locomotor function – movement – which is an incredibly difficult thing to do. Spinal cord injury is a constellation of symptoms – bladder function, sexual function, pain, temperature regulation, so many things. So I decided that instead of trying to hit a home run, I would go after a bunt single. One of the major complaints most spinal cord patients have is pain, and all scientists generally in a situation like that look for the simplest model. I’m not saying that trigeminal neuralgia is a simple model, but relative to spinal cord injury it is. One nerve, a big nerve, and the pain is in that nerve. I became interested in mechanisms and the patho-physiology of pain.

How is the Facial Pain Research Foundation unique from other research organizations?

We try to keep the bureaucracy at a minimum. We try to keep, while diversified, the number of projects we fund to maybe four, five, six at the most. We have a purely volunteer organization. Nobody gets paid anything. Our scientists don’t get paid. Our Trustees don’t get paid. Our support staff doesn’t get paid. I don’t get paid. Ninety-five percent of the money that comes in goes directly to the researchers for the research. I try to look for the best people in their field in the world to work for us. And to work with us.

How were you able to attract top researchers in the world to such a small foundation?

Instead of having them submit a grant, we say, this is what you’ve been doing, give me something we can hang a hat on, then go and do what you think will be exciting. So instead of their having to deal with a bureaucracy and submitting a grant every six months or a year, we, on a small scale, give them the money quickly, give them their leeway and see what they can do.  I call this “money with soul.” This is money given by people directly. This is not some faceless National Institutes of Health grant given with your taxpayer dollars. 

You were chairman of a prestigious neuroscience department and director of the McKnight Brain Institute.  Why is the Facial Pain Foundation’s research so important to you? Is it in part because a solution to neuropathic facial pain would also help provide scientific answers for multiple sclerosis, epilepsy, back pain, and other neuropathic pain?

Yes. I think it has a chance of making a difference. Mother Nature in my opinion is very conservative. The neurotransmitters in the gut are very similar to those in the brain, except you have different receptors and they work in a slightly different way. I look at this, and I think other people do too, as hopefully being sort of a Rosetta stone for pain, for neuropathic pain. Fundamental mechanisms are involved, and I think we can have a broader effect than just trigeminal neuralgia, also maybe diabetic pain and other pains. I think this research has a chance to be bigger than what you might expect it be.

How difficult is it to describe the science and the research to nonscientists?

For me, it is not too difficult. I’ve dealt closely in spinal cord injury and now in trigeminal neuralgia with the patient population we’re serving. I’ve always felt it was important to go to them and try to explain what we’re doing. I just hope that when I simplify it for lay people I’m not overdoing it so that I’m giving them incomplete or false information. 

At the beginning of 2011, the foundation set a goal of finding cures within ten years of establishing its research program.  At a minimum, what do you hope to accomplish before the end of 2020?

I’m hoping we will have a handful of genes or one gene we can hand off to others to test, and do animal studies and see if we can chart models of neuropathic pain by using the gene or the constellation of genes working together. To see if we have pain because they’re not working together, or we have pain because they’re overworking. Also, at the very minimum we hope we will have done human tracking with MRIs to chart the pain pathways that are involved, and locate the pain signal from the nerve to the brain, to where the brain can process it. And once you have that, then you have targets to do your intervention, do your injections. When you see which pathways are involved, you can define the neurotransmitters, so you can attack it that way. We are also hoping that the gene people will have solid data on how complex the gene interactions are – which is the alpha dog gene, or if there is an alpha dog gene, and how the genes interact.  

You wrote the following Facial Pain Research Foundation mission statement:

    “The Facial Pain Research Foundation is to establish a well funded translational research continuum that is dedicated to identifying the mechanisms underlying neuropathic facial pain and to developing groundbreaking therapeutic strategies that will permanently stop the pain of TN and related neuropathic pain syndromes.”

In your opinion, how are the foundation, its scientists, trustees and volunteers doing in relationship to its purposes and goals?

I think we’re doing fantastic, and I’m not someone prone to hyperbole. I’m very careful. I never would have dreamed in 2011 when we had zero dollars that we would now have collected a little more than $4 million. That $4 million has had a multiplier effect and raised even more funds for the research. I find that to be phenomenal. I think we are doing and our scientists are doing a wonderful job and making strong progress. I’m gratified and surprised that we have done as much as we have done in the period we have been in existence. 

Mervyn Rothstein Interviews

Todd E. Golde, M.D., Ph.D.

Todd E. Golde, M.D., Ph.D., is executive director of the Evelyn F. and William L. McKnight Brain Institute of the University of Florida. He oversees neuroscience and neuromedicine research programs across the University of Florida campus. A professor of neuroscience and neurology, Dr. Golde joined the University of Florida faculty in 2009 and became founding director of the university’s Center for Translational Research in Neurodegenerative Disease, which he led until taking charge of the McKnight Brain Institute in December 2016. He is also director of the NIH funded 1Florida Alzheimer’s disease Research Center group of institutions.

An internationally known expert in the scientific understanding of Alzheimer’s disease, Dr. Golde has published more than 240 papers that have been cited more than 30,000 times, (https://scholar.google.com/citations?user=X_9xacsAAAAJ&hl=en), and has expanded his leading-edge research to include other neurodegenerative diseases, cancer and even malaria. He is lead investigator for the Facial Pain Research Foundation’s Gene Therapy Research Project, at the University of Florida, to find the cures for TN. 

 Dr. Golde spoke with Mervyn Rothstein, who was a writer and editor at The New York Times for nearly 30 years and now writes the Stage Directions column – a series of interviews with theater directors – for Playbill.com. His first symptoms of trigeminal neuralgia occurred in 2005.

How and why did you become Involved In the “World” Of Scientific Research?

TG: I’ve loved biology from as far back as I can remember. The real catalyst was when I was in my junior year in college. I didn’t know what I was going to do. I was thinking about going back and teaching at a boarding school for a while before I figured out what I was going to do with my life. But I was a biology major at Amherst College and I took a course with a professor named Richard Goldsby, a wonderful teacher, who was basically teaching a graduate level course to undergraduates. We had to write a term paper, a research-hypothesis type paper, the first time I’d had to do that in college, and the response back was that this was one of the best papers he’d read in many years, undergraduate or graduate. Please come talk to me. I think you might like to do research. 

Your laboratory conducts disease oriented research with specific, but not exclusive focus on neurodegenerative diseases such as Alzheimer’s disease and Parkinson’s disease. How did you get involved in developing a research project to find a cure for Trigeminal Neuralgia and related neuropathic pain?How did the development of this research project come about, and what motivated you to take on the leadership role?

TG: I make no claims about being a facial pain or trigeminal neuralgia expert. I’m just thinking opportunistically about how we can make a difference. When I came to the University of Florida, I was the founding director of the Center for Translational Research in Neurodegenerative Disease. The chair of the department of neurology at that time had hired somebody named Dr. Andy Ahn. Andy worked on headache and pain, and the director needed a place for him. I had some space at that time, so Andy was a scientific neighbor, and we would have discussions.

The University of Florida is one of the hotbeds in the world in terms of a gene therapy approach based on adeno-associated viral vectors – we call it AAV for short. A former dean actually discovered this virus. One of our senior faculty members participated in creating the first gene therapy vectors around it. My lab had already been using AAV vectors, but we doubled or tripled down when we came to the University of Florida. AAV has a naturally affinity to get into neurons.

So Andy sort of triggered this, but my bandwidth was stretched at that point – it’s still stretched. I kept on tweaking Andy to go down this road. Andy left academics, and at some point I said I should do this.

We felt it could be something that could be rapidly translatable. Finding a cure for trigeminal neuralgia is a huge unmet medical need, but it’s an orphan disease that doesn’t affect enough people to attract a huge amount of attention.

There’s a chance for harm if we get into humans, but the nice thing about working with trigeminal neuralgia is we can at least start our initial studies in humans in people whose next option is surgical ablation of the trigeminal nerve. That means that there’s an out if we actually happen to make people worse. When we do these kinds of trailblazing experiments or envision them in humans, we need to make sure that if something could go wrong we have a way out.

Now we’ll begin the animal phase of study – testing in animals. And I know how much this research can really affect people’s lives. It may not work, but we have everything in place to test it. So let’s try.  

If Gene Therapy isn’t new, why do you believe that it can work to block pain signaling in the nerve? 

TG: It really comes down to technical details. People have used the wrong vectors before that had their own liabilities. I honestly just don’t think people have thought that much about this approach.

Last year, gene therapy had its first really radical major success outside of the eye, which was with children who had a disease called spinal muscular atrophy (SMA). They were treated systemically with a AAV vector – a different flavor of the same thing. These kids basically get spinal motor neuron degeneration when they’re young, by the time they’re two years old, typically, they die. Fifteen children were treated with huge amounts of virus, and reports last October showed that every one of these kids was alive at two years of age. And those with high-dose vectors, many of them were almost asymptomatic. At that age the expected survival rate would have been eight percent. So there are some children walking and living normal lives after gene therapy who otherwise would be most likely dead.

So I think that was a turning point in the field, to say this, AAV-based gene therapies, really will have its place in modern medicine. And that study in SMA was much more heroic and much more challenging than what we envision. So I think the time’s right. We know we can deliver the gene to the nerve. So now it’s just finding the right gene that will dampen pain signaling in that set of neurons, hopefully without doing harm.

Supposing your theory and project is proven successful. What implications or lessons wil this work provide for other researchers?

TG: I think it would say that perhaps the gloves come off on gene therapies for many disorders, especially things that are targeting nerve cell dysfunction. 

Because of the Hippocratic Oath – physician, do no harm – we need an out, so we wouldn’t want to go and do this on intractable sciatic nerve pain. Because what happens if you make them worse – you can’t cut their sciatic nerve without serious implications.

But I think success could open up the door to many different approaches targeting pain. And given the current opioid epidemic, driven from people at least initially taking these medications to treat chronic pain – (whether that’s right or wrong or medically appropriate is another question) – I think this viral approach enables us to deliver the medicine or treatment precisely to the cells or organ or nerve and hopefully avoid the systemic side effects that would come from taking a pill or an antibody or other approaches.

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