From the St. Louis Post-Dispatch, October 5, 2004 by Tina Hesman

Foundation seeks to jump-start progress toward cures for ALS and other disorders.

Chris Hobler has a plan to find cures for neurological diseases fast. The singer and songwriter has a personal stake in the matter. Hobler has amyotrophic lateral sclerosis, often called Lou Gehrig''s disease or ALS.

There is no cure for the fatal disease, but Hobler intends to mine success from research on other brain disorders to produce treatments for ALS. Along the way, scientists also may discover cures for Alzheimer''s disease, Parkinson''s disease, multiple sclerosis and other disorders of the nervous system. Hobler''s foundation, ALS Hope -- The Chris Hobler/James Maritz Foundation, is teaming with Washington University to create a new basic science research center dedicated to finding the causes of debilitating brain diseases and their cures.

The foundation, which is changing its name and mission, will give the university $1 million to set up the Hope Center for Neurological Disorders, kicking off a multimillion-dollar fund-raising effort. The center will provide resources, grants and intellectual stimulation to researchers working on diseases that affect the brain and spinal cord.

The shift to funding research in multiple diseases is reflected in the organization''s decision to change its name to "Hope Happens," said Hobler, 39. The foundation plans to announce its name change and the establishment of the Hope Center this afternoon at a reception at the Center for Advanced Medicine at Barnes-Jewish Hospital.

It''s a brave move, said Dr. Mark P. Goldberg, director of the Hope Center.

"It''s easy for someone who has MS or ALS to say, ''I want to put all my resources into my disease,''" Goldberg said. "It takes a much bigger vision to realize that sharing resources among many diseases can produce results."

Ideas generated by research on one disease could spark discoveries in others, said Dr. David M. Holtzman, head of the neurology department at Washington University. Although the diseases have different causes -- genetic defects, injury, immune-system attacks or the buildup of harmful proteins -- nerves follow many of the same paths to death or recovery, Holtzman said. With collaboration, researchers investigating different brain diseases are likely to find ways to stop nerve death and promote healing faster than they would be able to working alone, he said.

Speed is of the essence for Hobler and other people with neurological diseases, but the wheels of scientific progress can turn frustratingly slowly.

"When I was diagnosed with ALS in June 2001, I had a different reaction than most," Hobler said in an e-mail. "I had an advantage. I grew up with ALS. My grandfather had ALS for 14 years. I understood what my diagnosis meant. I also understood that nothing had changed in the last 30 years. No cure, no treatment, no hope."

The lack of progress angered him.

"I knew the reason had more to do with money and publishing than science," he said. "I think my life is worth saving. The fact that ALS was viewed as unprofitable by the pharmaceutical industry, and researchers don''t share results because of proprietary publishing and tenure, did not sit well. My voice was gone, my career as a singer-songwriter was over. I wanted to fix the system that had made no progress in 30 years. My immediate reaction was to call my family and say I wanted to start my own lab."

Instead, Hobler founded ALS Hope to raise money to support research that could lead to a cure. The foundation funded research at academic institutions and companies. But breakthroughs in the laboratory often take decades to reach patients. That''s a lifetime, sometimes several lifetimes, for a person with a degenerative nerve disease.

"Because my life hangs in the balance, I find many of the systemic restraints that slow the research process down inexcusable," Hobler said.

The Hope Center will require its members to share data, ideas and equipment. The center does not have one physical location, but four core components -- an animal research core, a molecular biology core, an imaging facility, and an outreach and information core -- will bring scientists together.

The facilities are designed to give scientists research tools they might not otherwise have access to, Goldberg said. The animal research facility will provide scientists with rats and mice that are genetically engineered to get neurological diseases, Goldberg said. The imaging facility contains powerful laser-powered microscopes that can peer into the brains of living creatures. The molecular biology facility will help create viruses for gene therapy. And the information core will organize seminars, including a national summit on neurological diseases.

In the highly competitive world of science, researchers often hold results close to the vest to prevent others from stealing ideas. But secrecy can impede rapid scientific progress, Hobler said.

Geneticist Alison Goate plans to initiate a search for the genes that put people at risk for getting ALS. She sees an advantage to sharing samples and data.

"If you share your data at an early stage when other people can replicate them, you find out quicker whether you''re going down the right path or a blind alley," Goate said.

And the investment makes economic sense, Hobler said. Pooling resources will allow researchers to act quickly on discoveries and stretch funds, he said.

Private money allows researchers to try things that more restrictive government grants wouldn''t allow, Holtzman said. The Hope Center will give grants to researchers for preliminary research and established projects. Some of the grants may go to researchers outside Washington University, he said.

The university announced Monday that it would move clinical trials for neurological diseases to a central facility, called the neuroclinical research unit. A $1.6 million grant from the National Institutes of Health and matching funds from Washington University will pay for the renovation of a 14,000-square-foot facility in the McMillan and Irene Walter Johnson buildings at Barnes-Jewish Hospital, said Dr. Joel Perlmutter, director of the neuroclinical research unit.

The facility, which will open in 12 to 18 months, will include office space for researchers, examination rooms, testing areas, interview rooms for social workers, and a kitchen and bathroom area where patients can practice daily living skills.

"This is people-oriented research," Perlmutter said.

The researchers don''t expect to find cures for neurological diseases immediately, Goldberg said. He doesn''t want to oversell the center or offer false hope, he said.

"But I know for sure that we''re going to discover things we weren''t expecting to discover."

Diseases researchers will tackle

Researchers at the Hope Center for Neurological Disorders will study brain diseases that grow progressively worse. Some of the diseases they will focus on include the following:

Amyotrophic lateral sclerosis (ALS), also called "Lou Gehrig''s disease," a fatal neurodegenerative disease in which the nerves of the spinal cord and brain die. Patients become paralyzed and cannot speak, but their minds are unaffected by the disease.

Whom it affects: About 30,000 people in the United States have ALS, with more than 5,600 new cases diagnosed each year. The disease strikes men more than women. People with ALS are commonly diagnosed at an average age of 55, but some people get the disease in their mid-30s.

What to expect: Half of people with ALS die within three years of diagnosis.

Alzheimer''s disease, a type of dementia that affects memory and thought. Certain proteins build up in the brain and are thought to kill neurons.

Whom it affects: Alzheimer''s disease is most common in older people. About 4 million people in the United States have the disease, including 3 percent of people ages 65 to 74 and half of people 85 and older.

What to expect: People commonly live with Alzheimer''s disease for eight to 10 years, but may live for 20 years.

Parkinson''s disease, a movement disorder caused by the death of neurons in a part of the brain called the substantia nigra. The cells usually produce the brain chemical dopamine. The chemical is a neurotransmitter, or brain messenger, that helps with coordination.

Whom it affects: About 1.5 million Americans have Parkinson''s disease, with about 60,000 new cases diagnosed each year. About 15 percent of cases are in people younger than 50, but the disease usually develops after age 65.

What to expect: The disease progresses at different rates. People with Parkinson''s disease can live a normal lifespan with proper treatment.

Multiple sclerosis (MS), an autoimmune disorder. MS is caused when the immune system attacks and damages a sheath of fat, called myelin, which insulates nerves and helps them conduct electricity.

Whom it affects: About 400,000 people in the United States have MS. Every year more than 10,000 people are newly diagnosed. The disease strikes women two times to three times more often than men. Most people are diagnosed between the ages of 20 and 50.

What to expect: The disease has four different patterns of progression. Multiple sclerosis is not considered fatal, and most people with the disease do not become severely disabled.

Sources: ALS Hope; the Alzheimer''s Disease Education and Referral Center; National Parkinson Foundation; The National Multiple Sclerosis Society