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Experimental Treatment for Spinal Paralysis

Eight months ago, a car accident left 18-year-old Melissa Holley of Ridgway, Colorado, paralyzed, possibly for life. But a new experimental treatment is providing her some hope that she will walk again. Melissa Holley joins us from her home and Dr. Valentin Fulga, vice president of Proneuron Biotechnologies, the company in Israel that is conducting the clinical trial that Melissa is participating in, joins us from Israel.

It's been 7 months since 18-year-old Melissa Holley received an experimental treatment for paralysis in Israel as part of an FDA-approved clinical trial. The results are promising for the young woman, who was paralyzed from the middle of her back down to her toes as a result of a car accident last year in June. Two of her vertebrae were crushed and her spinal cord was severely injured when her car flipped over on a wet road. She was driving alone to work. Part of the car landed on top of her. The diagnosis was bleak. Doctors didn't say that she wouldn't walk again, but they concluded that the paralysis was probably irreversible.

Her father decided that that diagnosis would not be the final one. He spent 2 days and nights searching the Internet and found out about an experimental therapy for paralysis that was being done in Israel. But it had only been performed on rats. Melissa Holley became the first human to receive this treatment, which is called autologous activated macrophage therapy. The treatment involves using the patient's own white blood cells--drawn from skin and bone marrow--to regenerate the severed nerves in the spinal cord.

On Thanksgiving Day, Holley started getting some feeling in a small part of one of her upper thighs. Since then, she has experienced more feelings in her legs and she can wiggle her toes. Doctors don't know how much improvement in her condition there will be as a result of this therapy. But what they have seen so far looks promising. The scientists involved in the study would like to be able to duplicate the success they have had with rats in humans. Fulga says that rats have been able to walk after receiving the treatment--not normally, but they were able to move their hind legs.

Interview with Melissa Holley

Melissa Holley wants to make more people aware of this clinical trial. She talks about the results that she has experienced from the therapy more enthusiastically than does Fulga. He says the results are encouraging, but he has to be very cautious not to overstate the results.

Holley says that her accident was on June 25th. "I was on my way to work and I had to be there at 4 PM," says Holley. "It had been raining earlier and my wheel just caught on the side of the road." She says the car flipped over and a wheel landed on her T6 and T7 spinal vertebrae. "They found that I had a complete injury," she says. "I was paralyzed from the middle of my back on down. It became more evident that I was going to either start dealing with what I had which was nothing, or look for some hope with this procedure. We learned it was a credible procedure, not just some guy in the middle of nowhere."

Holley says that it was scary to be the first human to have this therapy. Especially when she got to Israel and they explained exactly what they were going to do. "They had only tried it on rats," she says.

Holley describes how she gradually began to regain some feeling after receiving the therapy. "About 2 weeks after the surgery, one of the nurses came to give me a shot in my right thigh, and when she rubbed it down with the alcohol, there was a little feeling--about a half-dollar size--where I could feel the swab," she says. "From there on, week by week, I would get blurbs of feeling on my thighs first, more so up on my leg and then it worked downward. They [the feelings] started connecting in the larger areas, and then I started developing pain sensation in the right leg. If I pinch my right leg it hurts. If I pinch my eft leg, I feel the tug of the skin, but not the pain.

"Thanksgiving night was the first time I acknowledged any type of regain in muscle movement. I was just sitting in my room and I noticed that I was having a spasm, when your nerves shoot off randomly." She says she noticed that she could control a muscle in part of her thigh after the spasm subsided. At first she thought it was the spasm doing it. "After the spasm stopped, I put my hand on the inside of my thigh and tried to contract my muscle and I did. First, it was just my right side and then as the night went on I had my left inner thigh and my quads and I could wiggle my toes," says Holley.

Holley says that she is starting to get some muscle movement back and the feelings that she is experiencing are great. Holley says that she can move her foot and toes up. "I have regained some of my abs and at night, I do some crunches--which are weak crunches, but they are crunches," she jokes.

She says that she thinks of her father as "a life saver." "There was that aspect of time that something had to be done, and it had to be quick. The fact that he found something is like a miracle," she says.

She credits Fulga for translating the therapy that was used for rats into use for humans. "Any advancements or changes in my condition are reported to him," she says. "When I found out that I had the new movement I called him."

Holley says it's unfortunate that this therapy is not being offered to patients with older injuries. She hopes that as more patients are found to participate in studies like this one, the possibility for helping people with older injuries will increase.

Melissa Holley is very fortunate that her father quickly found out about this clinical trial on the Internet, because in this trial the patient has to be treated within 14 days of injury.

The Trial

This study is in phase I of clinical trials. According to Dr. Dan Lammertse, the medical director aCraig Hospital in Denver that is tracking Melissa Holley's progress, the results of this feasibility study looks promising. "The scientific basis makes sense," says Lammertse. "It appears to have very low potential for adverse outcomes and complications, so it has a lot of inherent advantages. But the test is to provide this treatment to a number of people and to use the best scientific means to figure out if it has helped them." Lammertse says that if you take 100 people with a spinal cord injury, some will get better and some won't. "If you have an experiment like this," and you have a small number of people who are getting somewhat better, "it's hard to put it into perspective in the context of the natural course of recovery. We don't have a precise way of predicting recovery for an individual."

Lammertse says that the people in the study have so far experienced a better-than-typical recovery, but at this point, it's hard to know where they will wind up in terms of how much they will be able to function.

How It Works

Macrophages are white blood cells that play a role in healing wounds and regenerating tissue. "When we are talking about spinal cord injuries, we are looking at nerves in the central nervous system and periphery nerves," says Fulga. "The difference between these two types of nerves is that nerves in the central nervous system don't re-grow by themselves and nerves in the periphery do." Fulga says the therapy that Melissa Holley received involves taking some of the macrophages from the periphery and putting them into the central nervous system so they can educate them to work. Fulga says that they help the nerve cells to regrow. "The macrophages in the central nervous system are scarce and lazy," he says.

Fulga says that it's important to understand that macrophages do not turn into nerve cells, they just help the nerves to regrow. "They bring about an environment that is conducive for regeneration," he says. So, in a nutshell, we take blood from the patient, we isolate the macrophages from a bit of the patient's skin, and bring them to Proneuron [the biotech company he works for]. We put them together and we culture them for approximately 1 day," says Fulga. "We take the macrophages and put them with the wounded skin, so we educate them. We then take the macrophages, which are now more mature and hopefully more effective, and we put them into a small syringe. The neurosurgeon injects them into the spinal cord and that's all."

Doctors braced Holley's spinal cord with two steel rods before injecting 4 million macrophages, less than a thimbleful, into the injured area. She and her father spent 3 months in Israel.

Interview with Dr. Valentin Fulga

Holley had what we call a complete spinal cord injury. She had been diagnosed by her neurosurgeon in Grand Junction, Colorado, and her father approached us. She had no sensation and motor function below the leve of injury. It was a little higher than the waist, from the middle of the back down, no feeling sensation or movement.

The term "autologous" pertains to the person himself, meaning we don't take tissue or blood from one person and give it to another. We take the patient's own cells and we use them. Macrophages are white blood cells and they exist everywhere in the body. We have them in the lungs, in the liver, of course in the blood, and also in the central nervous system and also in the peripheral nervous system. The central nervous system is the brain, the spinal cord, and the optic nerves. The peripheral is all the rest of the nerves in the body.

Melissa Holley was a perfect candidate for this study. She met all of the criteria. Candidates for the study have to be acute patients, within 14 days from the injury. We don't know if a longer period will be suitable. The reason for these 2 weeks as a window of opportunity is based on the work that we have had in animal studies so far. We know that we can treat animals after 14 days from the injury, so that's what we have tried in humans. Every day many people call me and email me asking for treatment. Unfortunately, I cannot give them any treatment. First of all, I am not the neurosurgeon, but that's not the issue. It's very difficult to find people within the 14 days of injury who know about the study and are in good-enough condition to travel.

Secondly, the level of injury is important. People who are too high or too low are not eligible because at this stage we cannot take patients that have respiratory problems, and we cannot treat patients that are injured above the C5 vertebrae, the fifth vertebrae of the neck.

I don't think there is anyone in the world that really knows what is going to be for Melissa. It's a novel therapy, and she is the first one to receive it. We know what we got from rats. At least until now, we see a resemblance in the recovery that we saw in rats, in terms of . . . they were able to walk.

Holley has gotten back some motor function and with some physical therapy she may be able to improve more, but we don't know. On the average, the animals recover after 12 weeks. Think of the rat whose spinal cord is less than half a centimeter in diameter and think of the human who has a spinal cord on average 1 centimeter or 1.2 centimeters in diameter. The amount of tissue that has to regrow is significantly larger for a human. In general, what is suggested in the literature is that it will take three to four times longer in a human in terms of a spinal cord injury. We can extrapolate that what happens in a rat in 3 months, may happen in a human after something like 9 to 12 months, but we don't know for sure.

We are encouraged by the results of the three patients in the study. At this stage, we have submitted the clinical study to the FDA, and we asked for some changes in the manufacturing stage. We have to wait for the FDA decision to enroll more patiens ecause we want to comply with all of the regulatory requirements. They are looking for about eight participants in the first phase of this study. The Israeli Ministry of Health is also monitoring this study.

She is a hero. She and her father are heroes. I thought to myself, "Would I be able to do this?" To go to another country and have an experimental treatment? I don't know. It's amazing.

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