Michael Pasternak, Ph.D., is a Founding Trustee of the Facial Pain Research Foundation. Dr. Pasternak spoke with Mervyn Rothstein, who was an editor and writer at The New York Times for 30 years. Mr. Rothstein now writes the “Stage Directions” column for Playbill.com. His first symptoms of trigeminal neuralgia occurred in 2005. 

MR: Michael, much has happened since our last interview, more than three years ago. What’s the status of the search for a cure for Trigeminal Neuralgia and related neuropathic pain? How many scientists are seeking a cure, in how many cities and countries? What are the ways your medical researchers are seeking answers, and how are they doing so? Please talk about the nine ongoing research projects to find the cure.

Our research is making great progress. Our first-ever research studies are the biggest leap toward finding the causes and lasting cures for Trigeminal Neuralgia in the last 1,000 years. But it’s complicated. Very complicated. Because in the old days, nine years ago, when we got started – January 9, 2011, when we had our first meeting, we hadn’t even started the research, the trustees came together and we made the goal to find the cure – it was all based on the belief that compression on the trigeminal nerve was the only reason for all Trigeminal Neuralgia.

But as we began to develop projects, and do the research, we discovered that there are other reasons as well. We know that trigeminal pain can also be caused by the myelin that sheathes the nerves being unhealthy and damaged. We know that there can be mutated genes responsible, genes that are not functioning properly, that are not protecting the nervous system. We also know that the nerves themselves can be damaged, in one way or another, by surgery or for other reasons. So we had to undertake research in all those areas.

For that reason, we have 18 lead investigators working in the United States, Canada, Israel and the United Kingdom. Numerous locations. And in each location, we also have technical assistants, post-doctoral researchers, many, many people working on our nine different projects to find the cures.

For example, to deal with the damaged nerves, we reached out to Allan Basbaum at the University of California, San Francisco, who had done work on using neuro-stem cells to fix damaged nerves in other areas of the body. And we asked him to apply his work to the trigeminal nerve. That research is ongoing, and is making progress.

To cite another example, for our work on causal, damaged or mutated genes and DNA, we needed 1,000 DNA samples from people with Trigeminal Neuralgia from nine collection centers in the U.S., Canada and the U.K.  And we needed to make sure that the people providing the samples really had TN and not some other related disease. And we succeeded.

No other rare-disease research has ever collected that number of samples in a process that assured excellent phenotyping – screening for the disease. And now we want to look at their DNA, we want to know where the mutant genes are, what the sufferers’ genes have in common.  when you ask what’s the status, the answer has to be very complicated. But the status is that in each of our projects, we have made progress, and we continue to make progress. We now know, for example, where in the brain the pain is occurring. We know the pathway of the pain from the trigeminal nerve to the brain stem to the place in the brain where the pain is experienced. And now that we know this, we’re working on how to block the pain pathway – block the pain from getting to the pain centers.

So the answers, and the cures, are going to be different for different people. Some will need to have their myelin repaired. For example, in our research for the last eight years at the University of Florida, we know that in tested animals there are ways to repair the myelin that can put a stop to the pain. And now, as in most of our projects, we are beginning to talk about human studies.

MR: When the Foundation was started, you set a goal of finding a cure by the end of 2020. That’s now. How do things stand?

That’s a good question. We will soon formally announce that this year, we will be initiating our first two human treatment studies. In scientific timelines, that’s pretty amazing. Because you must remember that over the last 50 years, the treatments for Trigeminal Neuralgia have hardly changed. The same anti-seizure drugs, the same pain relievers, the same surgeries – very little has changed in that regard.  And rates of cure using the old treatments have been really disappointing – in over 50 percent of microvascular decompression surgeries, the pain returns in 10 to 12 years, according to the National Institutes of Health. And the NIH says that the other, less invasive, surgeries have to be repeated in one to three years. They also recognized that in many pain patients the surgeries and the medications aren’t working.

This year we will have our first two human studies. And we feel terrific about that. (The people for the studies will be chosen by the scientific researchers, not by the Foundation.) And in each of our other projects, we can see on the horizon, and are preparing for it, that we will be able to move toward the trials.  The Foundation has raised the necessary funds to pay for this research and the goals are becoming closer every day to being achievable.

Now some of these curative treatments may work for some and not for others, because there are those different causes. That is why we have to create different cures – to repair the myelin, to repair the nerves, to block the ability of the nervous system to send those pain signals to the brain. But we feel great about our progress during these nine years, and we’ll keep going until everyone has the opportunity to stop the pain.

MR: The Foundation is an all-volunteer organization. There are no paid employees. The volunteers handle communications, fund-raising, and all other functions. I volunteer my time because I know first-hand, as someone who has experienced and suffered from the excruciating pain of Trigeminal Neuralgia, how important it is to find a cure. How did you get the thousands of other volunteers to sign up?

I think we did an incredible job of speaking to the public. Remember when we got started, because of this disease, the suffering and the treatment, there were billions of dollars being lost annually in productivity and medical costs. And more important, and more humanly unaffordable, was how dearly the unfortunate people suffering from this disease paid in quality of life – their health and longevity and stability and dignity and independence, their careers and family roles, their relationships and social ties, their physical abilities, their shattered sense of hope, of meaning or purpose. Many of these people were totally disabled and many were reaching out and saying they’d rather be dead.

We had a very sad situation. Great strides had been made in curing other diseases or creating better treatments for them, but with Trigeminal Neuralgia nothing new had been achieved for more than 50 years. There were the same treatments for TN that had existed for a half century. So when we reached out with our communications to tell people that we wanted to create cures, and that we wouldn’t stop until we found the answers, we were readily and strongly embraced.

I think a part of it was that we expressed the commitment to be an all-volunteer organization – that nobody was going to make money off of this. That almost all of the money we collected – close to 98 percent – would go to the research projects.

And I believe that this was very meaningful to the scientists as well. Many of their institutions dropped overhead costs for us, knowing that we were a nonprofit organization paying for this research. So we saved money in that way too. And I believe that it was the right time and the right place for a group of us who had been working hard to assist people with this terrible pain, and the public appreciated what we were doing, and they joined us.

In addition, I think that 90 percent of our volunteers are sufferers or family and friends of sufferers. They are people who have first-hand knowledge of the terrible toll of this disease, and they are contributing, they are developing fund-raising events and other activities. They are an important part of this Foundation.  Without our volunteers we would not be able to fund the research to find the cures.

MR: Is it still true that 95 to 98 percent of the donations go directly to the research projects. And how is this possible?

It’s absolutely still true. We have no employees. I think if you add up the amounts of money that we save as a result, it’s far beyond the money that we spend on the research.  Imagine hiring someone like you to do these articles for the Foundation, for the web newspaper. You’re not cheap, Merv. You’ve made a pretty good salary doing what you do. Just multiply that to all the little things that are done by all the volunteers – like a former college professor and a school principal who spend hours every week sending out thank you letters that are required by the I.R.S. for the donations. Imagine the volunteers who are meeting with the scientists on a regular basis, paying for their own travel costs to Canada or the U.K. or the East or West Coasts. When you start adding up the amounts of money that we are saving because of volunteers, it’s simply amazing what we’ve created.

MR: In the Foundation’s nine years of existence, how much money has it received in donations? How much more do you think you will need?

Cash coming in may have been $7 million, but the real number is closer to $45 million. Because we also have a program where we approach other foundations that give the money directly to our scientists – they prefer sending the money to the universities, for example. In many instances, people are giving in kind or matching money. Even the stamps we use for our envelopes are donated. It’s been very successful.

And another example is our Facebook page, which has been wonderful for getting donations. They can be $1 or $5 or $10 or $100 or $1,000, and they are anonymous. And much appreciated.

How much more will we need? That’s a great question. Much more, especially once you get to the human trials. For our first trial, much of it will be paid for by the National Institutes of Health, which is the first time something like this has occurred. But for the second trial, we’ll need probably another $3 million.

As an example, take Neurona, the company that was formed by Allan Basbaum out of U.C.S.F. for the neuro-stem research. Neurona raised close to $50 million to be able to take those neuro-stem cells and develop them, and they have 34 full-time scientists working on this project. Neurona has three directions they’re going – one is research on epilepsy, one is back pain and the third is Trigeminal Neuralgia and related neuropathic pain. They need those cells tested on animals by the very best testing service. We’re going to pay, at one of our centers, $370,000 to test those stem cells – which we believe are going to stop the pain and fix those nerves – on the animals.

When that is completed, Neurona is committed to putting at least millions into the Food and Drug Administration and clinical-trial approval process. We won’t have to pay for that. Neurona’s goal is to take the cures to the market.  This one example of how our FPRF scientific consortium can work.  In this case, the University of Florida, Neurona and us. This is  the first time there are three distinct organizations pulling together to get a research job done.  Neurona developed the cells, UF tested the cells and the Facial Pain Research Foundation covered the testing costs.  And how that happened was simply a matter of bringing those scientists together at our International Consortium of Scientists, which we hold at least every other year. And the the scientists work together on their projects. How that happened was simply a matter of the FPRF functioning in a leadership role to say how can we get this done. And it just made sense to do it this way.  And it’s not how things normally happen in scientific research.

MR: Both you and I have suffered from Trigeminal Neuralgia, and we both have been lucky. You, and other sufferers, have had a surgical procedure that has eliminated the excruciating pain. Others, like me, take medication that has been effective in keeping the pain at bay most of the time. But, as you’ve noted, the surgery and the medication don’t work for everyone, and often don’t last forever, and for the many others less fortunate, severe pain has been part of their daily existence. In truth, Trigeminal Neuralgia can be a fatal disease. It has historically been known as the suicide disease, and both you and I know of people who have so sadly decided they can no longer live with the horrible pain.  What can you say to those who still suffer? Why isn’t the research moving faster? And what can be done to accelerate the pace? Will there be a cure?

The first thing I would say to them is that I’m sorry we didn’t start earlier. We relied too long on the medical community to create better answers and better treatments. And I can say to them to have hope. To believe that a cure can really be found. If you look at our scientists, you’ll see this is something meaningful. They are outstanding in their fields. Take a look at our work and you can see that progress is being made and that you need to have hope and that there are great people working on this problem. And we’re going to get the job done. We’re committed to doing it and we believe it can be done.

It’s going to take more money. It’s going to take a great deal of money. But no scientist has said no to us, and we have been able to raise funds all along, not dependent on federal money, because we can’t wait for federal money. That system works for some, but it’s very bureaucratic. And we can’t wait for the government to pay for this research. We need it tomorrow because people are suffering.  We invite everyone to join us on this incredible journey to find the cures.