Duchenne Muscular Dystrophy
Floor Speech
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Mr. BARRASSO. Mr. President, I rise today as a Senator and, as the Presiding Officer is, a doctor. I want to talk about a disease called Duchenne muscular dystrophy. Earlier today Senator Rubio was on the floor talking about the disease, and I know earlier today Senator Wicker was on the floor talking about the disease. It is a topic that is, as an orthopedic surgeon, very personal to me.
I was introduced to Duchenne more than 30 years ago and, as an orthopedic surgery resident, worked at a muscle disease clinic with young people with muscle disease. One of those muscle diseases is called Duchenne. It is a disease that affects young boys. I met patients and I met their families in the fight against this disease. The experience has left a lasting lifelong impression on me, and it is something I continue to work with today.
I think the reasons we have gone into medicine are to help people and to make a contribution. One of the reasons I chose orthopedic surgery was that I really enjoyed seeing the relief--the care that I gave could help people, causing relief of their symptoms, relief of their pain, relief of problems they were living with from day to day. It is extremely rewarding to be able to work with a patient and tell that patient the surgery you performed was successful, and they are going to get better. They are going to get back to normal.
As a doctor, I was able to see patients go on to graduate from college, get married, have children of their own. When I was overseas visiting our troops, I met a young man, a commander--a pretty big guy-- and he told me I had taken care of his broken leg. I looked at him and didn't really recognize him. I said: When was that? And he said: I was only 8 at the time.
We take care of patients and, as we do, we see people through their lives, and it is encouraging to see them go on and strive and get stronger and bigger and more productive. But for patients with Duchenne muscular dystrophy, that kind of treatment doesn't exist. It doesn't exist today with all the breakthroughs and research.
When I saw patients in the muscle disease clinic who suffered from this condition, I knew the day that I saw them was going to be their best day from there going forward. Many of them had brothers. It is a disease that affects young men. It is a disease that may be coming in their family to children who had not yet been born. In some families there were several brothers in the line who had the disease. As one was diagnosed, then another younger brother was diagnosed a couple of years later with the same disease because this does tend to run in families.
As a doctor, one wants to see somebody get better and stronger every day. Parents want to see their own child going from crawling to walking to running, getting stronger and bigger every day, but patients and families who live with this disease every day know too well the unrelenting force of Duchenne muscular dystrophy. What it does is cause degeneration of muscles and weakness.
The vast majority of people with this disease are boys, and they are usually diagnosed between the ages of 3 and 5. Typically, parents start to notice their son isn't meeting all of the developmental milestones they might expect. He might be a late walker, or he may appear less coordinated than other children his age. Most parents aren't worried; they are just cautious. They may mention it to the pediatrician, and the doctor may run a test or two. Once the diagnosis of Duchenne muscular dystrophy is made, patients pretty quickly and parents, specifically, very quickly find out that their son doesn't just have a developmental delay; they learn their son is typically going to lose the ability to walk by the time he is a teenager, graduate to a wheelchair, which then can make that young man prone to conditions like scoliosis, a curvature of the spine often requiring surgery to correct it. As the muscles continue to deteriorate--as they always do with Duchenne--that young man will lose lung function, which puts him at a higher risk of infection, pneumonia. Eventually, he will have to use a machine to breathe, to clear his lungs. The muscle deterioration doesn't just occur to the skeletal muscles--the muscles of the arms and legs--but also can occur to the heart, which is itself a muscle.
When a young man with Duchenne muscular dystrophy catches a cold, it can be life threatening. Even when the patients get the best medical care--and so many of them do get the best medical care--they usually lose their fight against Duchenne muscular dystrophy by the time they are in their 20s. That is the devastating reality of this disease, and we cannot allow it to continue.
Because of my experience with these patients, I have been working for years to actually help raise money for awareness for muscle disease and treatment for the disease. I served as a local host in Wyoming for the Muscular Dystrophy Association's annual Labor Day telethon.
Every year, I was amazed at the dedication and the generosity of people around the country who would call in pledges to pledge centers at the 200 so-called ``love networks'' in Casper, WY. People would call in. We would always raise over $100,000. People were very committed to finding a cure for muscle disease and to sending young people with the disease to summer camp, where they found a level of freedom and friendship that they did not often find throughout the rest of the year. It was a great time for the young people with the disease. It gave their parents a rest as well.
I think many of us in this body remember Jerry Lewis hosting the Jerry Lewis Labor Day Telethon, as it was called, for more than 40 years. He would always end the telethon by signing a song. The song was ``You'll Never Walk Alone.'' So I come to the floor today to make sure that these patients and these families know that today they are not alone. Congress is listening. We heard from Senator Rubio earlier today and we heard from Senator Wicker. Those families and those patients know how critically important it is, and we know how critically important it is that we find a cure for this rare disease known as Duchenne muscular dystrophy.
In 2012, Congress passed the Food and Drug Administration Safety and Innovation Act. One of the key parts of this law gives the FDA more flexibility to approve treatments that have the potential to help people with rare diseases. It also allows the FDA to do followup studies to confirm the clinical benefits of the treatment.
Well, we want to give people real hope. It is not good to give people false hope. We are interested in giving patients and giving families a fighting chance. I believe the FDA needs to use the tools that Congress has given them so patients can come across and get access to potentially lifesaving drugs. So a couple of weeks ago I signed a letter that was written by Senators Wicker and Klobuchar--a bipartisan letter. It called on the FDA to take full advantage of this accelerated approval authority.
So we also asked the FDA to ensure that the prospective of patients is fully considered in this review process, when it comes down to the regulations. More than 20 Senators signed this letter because we know how important this issue is to patients as well as to their families.
Last Friday the Wall Street Journal ran an editorial entitled: ``The FDA vs. Austin Leclaire.'' This article talked about a young man named Austin Leclaire, 17, who has Duchenne muscular dystrophy, and so does his younger brother Max. As we talked, I mentioned that this runs in families. Sometimes, there is the diagnosis of a son in a family in which there is a younger son who has not yet been diagnosed but likely will have the disease.
Well, back in 2011, Max was able to get an experimental drug to treat his disease. Now, Austin was not eligible to get the same drug. Remember, Austin is the older brother. So today Max is 14 and he is still able to walk. He can still play sports, and he can still dress himself.
For most of us who have had healthy children, these are the things that people take for granted. So for a family where one of their sons has Duchenne, this kind of small victory can seem like a miracle. I can't even imagine how hard it must be when a mother has two or three children--two or three sons--with this disease, and especially when one of her children can get access to an experimental drug and the other cannot.
The family looks at it. One son is being helped, and the other is not being helped. They can see the difference in their sons. So how would any of us here in the Senate react if we were in that same situation? How much heartbreak should one family have to bear? Those are the challenges for families who live with muscle disease every day.
Well, the FDA, I believe, needs to work with patients like Austin and Max. We all know that this agency needs to make sure that treatments are safe and effective. That is not a question. We also know that people at the FDA are caring and careful professionals. The practice of medicine relies on hard science and on following data to understand and to treat illnesses.
As a doctor, I know that the practice of medicine requires an equal measure of compassion. I think the FDA needs to take into account the unique needs of this patient population. We talk about double-blind studies, where you give one patient the real treatment and one patient something else, a sugar pill, something else that is not really the real treatment, the real medication.
To really evaluate the impact of these medications, sometimes it involves doing muscle biopsies and putting people though painful tests. I think it is hard for a family living with a child with muscle disease to say: Well, we are going to participate in the experiment. We don't know. It is a 50-50 chance if our child is even going to get the real thing. But we still put them through all of these tests that can be painful, as they take muscle biopsies.
I think it is unrealistic to ask a family to make that decision. I think we need to make sure that the FDA--and the FDA needs to make sure, in their compassion--doesn't lose sight of these kids. These young people really don't have a moment to lose in terms of potential treatments. I think the FDA needs to hear the calls of patients and to give these young people, living with a devastating disease, a chance to beat Duchenne muscular dystrophy.
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