In 1998, when Stephen Heywood was first diagnosed with ALS, his brother Jamie decided he had to do something. Correspondent Charlie Rose reports on their story, which was first reported in The New Yorker.
Jamie Heywood became a kind of guerilla scientist, pushing the limits of research to help his brother.
As a result, what began as Jamie Heywood's singular mission - saving his brother - could also help thousands of others. By that time, though, Stephen Heywood may be dead. "Ninety percent of the people die within five years," says Stephen Heywood of his prospects.
Now in his 30s, Stephen Heywood has watched his body degenerate in stages. First, he lost the use of his right hand; then his left. Stephen Heywood's condition has continued to deteriorate. He recently began using a wheelchair, and his speech has become more and more slurred. His mind will never be affected.
Jamie Heywood refused to accept his brother's disease. "We're just part of each other's lives," he says. "[I] just couldn't imagine him not being here."
The two brothers have been looking out for each other since childhood. After the diagnosis, Jamie Heywood became obsessed with finding a cure for his brother.
"No one was developing anything that was going to arrest the disease," says Jamie Heywood incredulously. "There was no one saying, 'This is something that's going to stop this disease from killing people.'"
Jamie Heywood decided to fill this absence. He quit his job in California and moved, with his wife Melinda, back home to Newton, Mass.
With an engineering degree, but no formal medical training, Jamie Heywood learned everything he could about ALS, which kills 14 people a day on average. He discovered that no one understands what causes ALS. Part of the reason is that ALS is an "orphan disease." There are too few patients to make a treatment profitable, so most pharmaceutical companies are unwilling to fund research.
He has completely dedicated himself to his mission. Says Stephen Heywood about his brother: "He sees my deterioration as a personal affront, as a personal reminder of how slowly he thinks he's going."
Everyone in the Heywood family has become involved. His parents took a second mortgage, so that Jamie Heywood could buy a house and launch the ALS Therapy Development Foundation, a group that tries to move promising ALS research from the lab into human clinical trials.
"There's all these great ideas sitting in these laboratories waiting for someone like us to come in and spend half a million dollars or $1 million and move it from that research lainto a clinical program," says Jamie Heywood.
And he is changing the field of ALS research. Because of his efforts, 30 more researchers in various labs are working on ALS. The foundation funds several labs, including Dr. Steven Gullans' at Harvard Medical School. Using robotic equipment, Gullans' staff randomly test hundreds of approved medications, herbal remedies and even vitamins. They have already found several drugs that reverse the affect of ALS on nerve cells.
Gullans says he is close to finding a drug "cocktail" that would delay the progression of ALS. Jamie Heywood hopes that these treatments may buy time for his brother, so that researchers can find a more complete cure.
Jamie Heywood's most ambitious and controversial project combines ALS research with gene therapy. Essentially, gene therapy involves the replacement of a diseased segment of human DNA with healthy DNA. It is still experimental, and there are very few success stories. But the procedure may be able to cure some diseases.
The gene therapy approach began when Jamie Heywood read a report by Dr. Jeffery Rothstein of Johns Hopkins University. Rothstein theorized that the missing genes in the spinal cords of most ALS patients contribute to the disease. When ALS-infected mice are bred with these genes, they live twice as long before succumbing to ALS.
Jamie Heywood took the research to Dr. Matt During, who runs the gene therapy lab at Jefferson Medical College in Philadelphia. With During's help, he devised a plan for the first clinical trial using gene therapy to treat ALS. It involves a risky procedure: inserting a needle into the spinal cord.
"It's a little bit daunting when you look at all the problems we face," During says. "Thinking about putting needles into the spinal cord is something that makes all of us queasy."
Some worry that this approach is too aggressive. "I'm just concerned that the set point for their initial gene therapy proposal was not biased enough toward safety," says Dr. Robert Brown, an ALS specialist at Harvard Medical School who diagnosed Stephen Heywood's ALS and also advises Jamie Heywood on his gene therapy proposals.
In this case, speed is paramount, says Jamie Heywood. "Every doctor I've spoken to from the beginning has said to me, when we talk about these trials, we have to be careful," he says. "We first do no harm. Well, in this case harm is being done every single day. And if we delay something a year, then we've just done a great deal of harm."
But speed must be weighed against the pitfalls of unproven science. In 1999, 18-year-old Jesse Gelsinger died in a gene therapy trial at the University of Pennsylvania. In March President Clinton instituted new safety guidelines for all gene therapy experiments involving humans.
No one involved in Jamie Gelsinger's trial believes gene therapy alone will cure Stephen Heywood. But it is one of the most exciting projects i ALS research.
And it would not have happened without Jamie Heywood, who has spent hours learning about ALS. As a result, he can talk about it like a pro. "I had assumed because he was so fluent with the language that he was most definitely a scientist," says During of Jamie Heywood.
"Jamie did know his stuff. Jamie knew the lingo. Jamie knew enough biology, knew enough science, knew enough at every level of this project to be able to make an impact. You need both money and energy and the insight in terms of biology and (to) be able to put the people together," During adds.
"He clearly helps in a number of ways," Dr. Brown says. "The greatest help is that he's a kind of intellectual catalyst. He is, in a very appropriate way, a gadfly, who puts on the table new lines of thought, new lines of applied approaches that we perhaps have not given enough consideration to."
Stephen Heywood also says his brother has been a lynchpin. If given the choice today to have the disease or not, he would choose to have it, because his brother's work has done so much to bring a cure even if it doesn't save him, he adds.
"Do I believe it might help me?" Stephen Heywood asks. "It's a tough question. Do I believe my brother will have a significant impact on curing ALS? Absolutely."
Like his brother, Jamie Heywood is realistic. "Something we're doing - and there are a lot of projects now - will extend [Stephen's] life," he says. "Will it save it? I don't know. But something we're doing will make it different. But I'm a realist....Look at (the) odds. You look at the things that go to trial and finally look at the records of previous therapies. Those are bad odds."
Jamie Heywood says the foundation has tried several experimental treatments; so far, none have had a discernable effect on the progress of ALS.
While he waits, Stephen Heywood hasn't stopped living. He credits his brother with helping him continue to think positively. Earlier this year, he married Wendy Stacy, who is expecting their first child, a boy, very soon. He won't develop ALS, since his father's form of the disease is not hereditary.
Says Jamie Heywood: "If nothing works, the thing I gave Stephen was the peace of mind to know that he could do what he needed to do in life. And that I would take care of making sure that if there was something that would make a difference, he would get it."