Ride for Life has a long standing commitment to supporting ALS research. Through out Ride for Life’s existence, nearly 4 million dollars to date, have been raised, research represents 50% of our program spending.

Over the last 12 years Ride for life has donated to 19 different research teams, totaling over 1.5 million
dollars! Some of the top recipients of funding are; ALSA Gehrig Challenge, ALS Therapy Development Institute, Dr. Rothstein of Johns Hopkins, MDA Suffolk, Dr. Brown of ALSTI Harvard and Columbia University. These grants would not have been possible with out the support of Ride for Life donors. Your support has had a direct role in this new discovery. You literally had made a difference!

All of the research is incredibly important in reaching the goal of finding a cure for ALS, it is imperative that Ride for Life can continue on its mission in raising funds for research. With out the generous donations and support of donors for Ride for Life, these research grants and these scientific breakthroughs would not have been possible. A giant stride has been made towards finding a cure; please continue to join us in the fight. Become a donor today and help in the most important way possible, funding research. Please visit our website, www.rideforlife.com, to donate.

Scientists have long struggled to identify the underlying disease process of ALS (also known as Lou Gehrig's disease) and weren't even sure that a common disease process was associated with all forms of ALS.

In this new study, Northwestern University researchers said they found that the basis of ALS is a malfunctioning protein recycling system in the neurons of the brain and spinal cord. Efficient recycling of the protein building blocks in the neurons are critical for optimal functioning of the neurons. They become severely damaged when they can't repair or maintain themselves.

This problem occurs in all three types of ALS: hereditary, sporadic and ALS that targets the brain, the researchers said.

The discovery, published Aug. 21 in the journal Nature, shows that all forms of ALS share an underlying cause and offers a common target for drug therapy, according to the researchers.

"This opens up a whole new field for finding an effective treatment for ALS," study senior author Dr. Teepu Siddique, of the Davee Department of Neurology and Clinical Neurosciences at Northwestern's Feinberg School of Medicine, said in a university news release. "We can now test for drugs that would regulate this protein pathway or optimize it, so it functions as it should in a normal state."

This finding about the breakdown of protein recycling in ALS may also prove useful in the study of other neurodegenerative diseases, specifically Alzheimer's and other dementias, the Northwestern researchers said.

ALS afflicts an estimated 350,000 people around the world. About 50 percent of patients die within three years of the first symptoms. They progressively lose muscle strength until they're paralyzed and can't move, speak, swallow and breathe, the researchers said.

More information

The U.S. National Institute of Neurological Disorders and Stroke has more about ALS.

ALS ride kicks off in Riverhead

May 9, 2011 by MITCHELL FREEDMAN / mitchell.freedman@newsday.com

More than a hundred students walked behind Christopher Pendergast, 62, in his motorized wheelchair Monday morning to Riverhead Town Hall, where Pendergast will begin his 12-day trip to Manhattan to raise funds for Lou Gehrig's disease.

Pendergast expects a dozen other patients with amyotrophic lateral sclerosis, a degenerative condition Pendergast was diagnosed with in 1993, to join him for part of the ride to Manhattan, which he calls the "River to River Ride."
The march to town hall Monday morning was led by the Riverhead High School honor society, though most of the students wore green Mercy High School sweatshirts. They were 7th and 8th graders, and many worked to raise $2,000 this year which will go to create a scholarship in Pendergast's name.

"Chris is a Mercy alumni ... he's a 1996 graduate," said Debbie Kneidl, the school's director of institutional advancement, who was walking with the group.

Pendergast and other ALS patients created Ride For Life in 1998, which has raised nearly $2 million for ALS research since it began.

There is no cure for ALS, and 90 percent of the patients who contract it die within five years. The disease is named after former New York Yankee Lou Gehrig. The baseball legend had the disease, which causes patients to lose control of their voluntary muscles. About 30,000 people in the United States suffer from ALS, according to the not-for-profit Ride For Life corporation.

Scholarship named for Ride for Life founder

Followed by more than 200 supporters, Christopher Pendergast began the 14th annual Ride for Life on Monday, a 12-day journey from eastern Long Island to Manhattan.

Pendergast, a Miller Place man who was diagnosed with amyotrophic lateral sclerosis, or Lou Gehrig’s disease, in 1993, founded Ride for Life in 1998 to raise awareness about the disease and funds toward finding a cure.
The trip from eastern Long Island to Manhattan is a familiar one for Pendergast, but this year’s send-off came with a surprise.

Administrators at Bishop McGann-Mercy Diocesan High School in Riverhead, where the ride kicked off and Pendergast is an alumni, announced a scholarship in Pendergast’s name.

The Christopher Pendergast Scholarship will be given to a current Mercy student who shows a commitment to helping others and will be used to offset the cost of tuition for the following school year, said Deborah Kneidl, director of institutional advancement at the school.

She said it will be for about $500, but the school has not chosen an exact amount.
Christine Pendergast, Chris’ wife, said the scholarship is the only one that has been established in her husband’s name.

“That is a legacy that will live on beyond Chris’ lifetime,” she said. “That makes Chris very proud.”
Touched by talks Pendergast has given at the school, students at the Mercy junior and senior high schools raised $2,000 for the cause this year and about 150 of them followed Pendergast, who rides in a motorized wheelchair, around Riverhead for the first leg of this year’s ride.

Pendergast, who turned 62 last week, said the students raised more money than any other single entity this year.
He has a lot to feel lucky about, including the support of so many, and the fact that he is still alive. He said the average survival rate for someone diagnosed with ALS is two to three years.
Of his 14th ride, he said: “I’m sad that it is still necessary because ALS remains incurable, but I’m glad to be alive to do it.”

To follow Pendergast’s ride, check the Ride for Life website for a list of scheduled stops and receptions.
Photo: Christopher Pendergast prepares to start his 14th annual ride for life from Riverhead to Manhattan with students from Bishop McGann-Mercy Diocesan High School in Riverhead. (May 9, 2011)

Dr. Steven Strongwater, CEO of Stony Brook University Hospital, informed Christopher Pendergast today that the Hospital's ALS clinic will be named after him.

Announcement in Newsday

Ride For Life founder and president, Dr. Christopher Pendergast, made a $65,000 grant to help the clinic cover nursing and related patient service costs. “This is the only comprehensive ALS Clinic on the entire island,” Pendergast pointed out. “The ALS community must support the facility, the great staff, and the excellent care that Stony Brook provides through Clinic. I wish it was more. So much is needed.”

The annual Ride For Life occurs each May during ALS Awareness Month. Patients travel by power wheelchair from the east end to Manhattan over multiple days. Since the Clinic’s inception in 2002, the patient advocacy group has granted over $300,000 to assist Stony Brook’s unique ALS center.

Coming May 15, the Ride For Life will be traveling in the Stony Brook area including a stop at the Veterans Home and the Pinwheel Memorial ALS display in South P Lot. Walkers are encouraged to join the group for the 10 mile event. For more information, visit www.RideForLife.com or call the Ride office on campus at (631) 444-1292.

As a result of the 2009 Ride for Life funded a total of $135,000 dollars for research. After careful thought, discussion and collaboration the funds were distributed among five research groups.
• ALS Association (through Greater New York Chapter) received $35,000 for their aggressive support of research. ALSA supports researchers both in the U.S. and globally.
• ALS Therapeutic Development Foundation, Cambridge, Massachusetts received $30,000 for its work, dedication and determination to find a cure.
• Johns Hopkins, The Robert Packard Center received $30,000. The center under the direction of Dr. J. Rothstein continues to organize and support notable scientific research throughout the U. S. and world.
• The Eleanor and Lou Gehrig ALS/MDA Center at the Neurological Institute, Columbia University received $30,000 under the direction of Dr. H. Mitsumoto for two clinical trials. One clinical trial will measure glutamate (toxic) levels and glutathione (anti-oxidant) through the use of MRS technology. The other clinical trial will examine the underlying factors that potentially influence decisions to use tracheostomy – long term mechanical ventilator.
• MDA/ALS Division received $5,000 for its support of global research scientists in ALS.

Research into the cause and cure is being carried on throughout the world but it remains an orphan disease and many questions still need to be answered. Deciding who should be funded is difficult since the scientists are dedicated in their efforts. To assist researchers in finding a cure takes an incredible amount of time, energy and money but it is the research that will find the cure.

Ride For Life is deeply grateful for all the generous donations received to fund ALS research as we continue the hope to find a cure and end this dreaded and incurable disease.

Norma Steck-Hess, RN, Ride For Life Board Member
PALS Research Chair

WEST MILTON, OH — Artists use their work to communicate their vision of the world, but Gary Wickman's paintings are about the last way he has to communicate at all.

Wickman was diagnosed with ALS (amyotrophic lateral sclerosis, or Lou Gehrig's Disease), an incurable progressive neurodegenerative disease, three years ago. He is approaching its final stage.

"We were watching a PBS auction that included an oil-painting set. He turned and said, 'I think I could still do that,' " said Jill Wickman, Gary's wife.

In the past year, painting up to five hours at a stretch, Gary has produced dozens of small canvases.

"Everything is all about outdoors and nature. Maybe it goes back to where he grew up, on the Upper Peninsula," Jill said of Gary's Michigan upbringing.

All came from pictures he had seen or that were found on the Internet by his wife. Though he can barely sign his paintings, each is a riot of detail and color. Trained as a mechanical engineer, Air Force Maj. Wickman was preparing for his next posting — back to Wright-Patterson Air Force Base. Within days came the diagnosis. In the space of two months, Gary retired from the service after 20 years, the couple sold their house in Maryland and moved into what was once Jill's grandmother's house in West Milton.

"We just piled everything into the house, bought two Harleys and headed West," she said. Gary smiled as his wife recalled their summer of '03 farewell tour.

A year-plus later, the Harley was parked in favor of a wheelchair. Now the wheelchair has been replaced by an overstuffed recliner and a ventilator.

Don Shough has spent 13 years as a home health assistant, most recently with Gary and Jill. He has assisted a number of ALS patients.

"It's hard for a man to let somebody help you. It's especially hard when you can't do anything for yourself," Don said. Though Gary has lost most of his mobility and his voice, Don sees things in Gary the disease cannot touch.

"He's ready for the inevitable. He's a good man with a good sense of humor."

Gary communicates on canvas and through hand signs — and humor. He's not above teasing his wife with a twinkling eye and a sly glance.

Propped up against the TV in front of his recliner is a plaque from an Air Force buddy.

"A friend," it reads, "will come and bail you out of jail, but a true friend will be sitting next to you saying, 'Damn, that was fun!' "

FALCONER, NY — For Keven Hulings, the number four is special.

The same number once appeared on the jersey of baseball’s immortal Lou Gehrig — Hulings’ hero.

A victim of Amyotrophic Lateral Sclerosis, the New York Yankees hitter died in 1941 after a two-year battle with ALS — known since as Lou Gehrig’s Disease.

ALS, a disease of the nervous system, attacks muscle-controlling nerves. Eventually, all muscles — with the exception of involuntary muscles such as those in eyelids — are destroyed, and paralysis follows.

Today, Hulings is chalking up a 15th year of his own war against ALS.

The disease has wasted his muscles, wiped out his voice and immobilized his body. Yet, his mind races at the speed of sound, and unmistakable glints of humor light up his eyes.

When his voice succumbed to the disease, Hulings turned to technology.

Now, he once again ‘‘talks’’ and argues issues.

Hulings likes nothing better than debates, especially when he proves his challengers wrong. Offering no quarter to the lethal enemy stalking him, he lives with all the purpose and passion of that South Side boy who never missed an after-school baseball game, or a chance to cheer on his beloved Yankees.

Hulings also faces another challenge. About three years ago, he was diagnosed with diabetes, and daily insulin treatments were added to his regimen.

In April, Hulings competed in the two-day segment of the ninth annual Ride For Life fund-raiser for ALS research and patient services.

The wheelchair event in New York City is sponsored by RFL, Columbia University and the Yankees.

Along the RFL route, Hulings saw Gehrig’s birthplace, a home on 102nd Avenue, and visited Yankees Stadium.

‘‘It was a great time,’’ Hulings said, ‘‘except, the Yankees were beaten by the Toronto Blue Jays.’’

Hulings collected $1,500 in pledges for the fund-raiser, all of which he solicited through letters to businesses and individuals. With his cousin, Howard Hulings, and nurses, Jennifer Davidson and Patty Zdunski, supplying ‘‘push energy,’’ Hulings’ wheelchair was among the first to cross the Brooklyn Bridge and the finish line.

Several days ago, a television crew crowded into Hulings’ apartment, and Buffalo’s Rich Kellman interviewed his host. The film will be featured as part of the 2006 Buffalo Multiple Distrophy telethon, an annual Labor Day weekend event.

None of this would be possible, without the cadre of nurses who care for him around the clock, said Hulings.

‘‘I can be difficult. At times frustrations get to me ... sometimes, my leg or arm itches, and I can’t get a message out to them to take care of it fast enough,’’ he said. ‘‘But, we have bonded. We are family. We argue, we celebrate and, at times we commiserate.’’

Two of the care-givers who have been with Hulings the longest, are Jennifer and Jean Saylor.

The group, said Jean, ‘‘is a clan of individualists that only God could have put together ... We’ve become an integral part of Keven’s life. What’s important to him has become just as important to us.’’

Jennifer agreed. In addition to tending to her patient, administering medications, relieving itches and other annoyances, and monitoring the yards of tubing that crawl over and around his body, the nurse assists him in many other ways.

For example, when Hulings was not up to taking part in this year’s Billy Mead 5K run at Dunkirk, Jennifer entered the event as a representative of the Hulings ALS camp.

Patty said Hulings is ‘‘amazing ... very forceful.’’

Her involvement as his care-giver, she said, has been a life-changing experience. Other members of the group are Pamela Wesley, and Bruce Berry, who reinforces Hulings’ male perspectives.

Hulings was stricken with ALS in 1991. The former Cummins, Inc. employee was 35. He walked the last time in 1992, and his arms gave out two years later. A ventilator has taken over his breathing.

Does he ever despair? Does he ever ask, why me?

Pressing his chin against a device that guides a mouse from letter to letter, Hulings rapidly types responses, word by word, on the screen of a machine, called a DynaVox. Once messages are complete, they are then released by a computer ‘‘voice.’’

Hulings says it’s all a matter of a commitment to life. Not just to live, but to accomplish something.

‘‘ALS can’t take my mind, and I won’t let it take my spirit,’’ he said. ‘‘As long as I have those two things, I figure I have a life. ‘‘I’ve fought hard to live, and it’s well worth it even though I get frustrated at times — like when I want to hug my niece or nephew and I can’t. I hate it when I’m stymied by this damn disease.

‘‘I used to chase women — unfortunately, I didn’t run fast enough. I used to drink too much, and I played hard,’’ he said, with that same glint in his eyes. ‘‘Now my life has meaning. I have purpose, and I get to be Uncle Lou Gehrig’s right-hand man with a mission: to help people who’ve been hit by ALS or other diseases that wipe out their mobility.’’

Katherine Hulings says her son has ‘‘the tenacity of steel ... Keven never gives in. Once he makes up his mind, no one’s going to budge him, no matter what.

‘‘In 1997,’’ she recalls, ‘‘doctors told Keven he would die within a year or two if he left the hospital. They said he’d have to accept the fact he’d be hospitalized the rest of his life.’’

For months, Hulings refused to buy that prognosis. He could only exist in a hospital, he argued. He wanted to live. He wanted to go home. Thanks to family and friends an addition was added to his parents’ mobile home on Elmwood Avenue. Finally, with the help of a local physician, the rebellious patient was — as he puts it — ‘‘sprung’’ from WCA Hospital.

Nine years later, he’s still beating the odds.

‘‘He wasn’t supposed to live two years,’’ Mrs. Hulings said, ‘‘yet, here he is, still with us.’’

Leaving the hospital was a defining moment for the ALS patient. Battle lines were drawn. While the disease might take him down, Hulings would not allow it to take him out.

After settling in his apartment, he marshaled family members and friends, old buddies like the Kilmartin brothers — Bill, Kelly, and Sean.

‘‘They’re my brothers,’’ said Hulings, ‘‘even if they are Red Sox fans.’’

Months later, the Friends of HUGO — an organization focused on fund raising — and the HUGO Fund were founded. The fund continues to provide assistance to others with physical handicaps who wish to remain in homes.

In the spring of 1997, the HUGO Fund became part of the Chautauqua Region Community Foundation. Later, the HUGO Loan Closet was established and housed in space donated by Falconer’s J.P. Diamond Co. Available at the Closet are donated items such as walkers, wheelchairs, furnishings and many other items.

Through HUGO, those with physical disabilities also find assistance in cutting red tape and paperwork required to arrange home environments. In addition, a number of people are guided to appropriate agencies for assistance, such as the Veterans Administration, Medicare, etc. The organization also works toward legislative changes that will promote home care.

Each year, funds are made available for one to three college scholarships. Eligible to compete are high school seniors who wish to pursue professions that would enable them to help the physically challenged, and, who attend Falconer, Southwestern, Maple Grove, Jamestown and Frewsburg schools.

Fund-raisers, arranged by The Friends of HUGO, keep members more than busy, said Bill Kilmartin.

‘‘Since HUGO was organized, we’ve raised a total of about $50,000 at golf tournaments, alone,’’ he said.

Kilmartin describes Hulings as ‘‘a die-hard Yankees fan, who’s been giving me the business about the Red Sox, since high school days.’’

But, in 2004, when the Sox won their first World Series in 86 years, Kilmartin said, ‘‘Keven was the first person to share the big moment with me.’’

Robert Terreberry, a close friend of Hulings, was the first chairman of The Friends of HUGO, and headed the organization for some time.

‘‘Keven has tremendous fortitude and an uncanny ability to attract caring people to the HUGO cause,’’ Terreberry said. ‘‘In turn, he’s a very caring individual, the first to contact his friends on special occasions. When I came up a winner in a recent Chautauqua Lake Idol competition at Bemus Point, there was an e-mail from Keven, minutes after I returned home.’’

Like Hulings, Terreberry is convinced home environment should be an option for the handicapped.

‘‘It ensures a quality of life that just can’t be provided in an institutional setting. Because of that, most patients at home have more meaningful and longer lives. Keven’s case proves that. Aside from all the human factors, staying home is less costly.’’

Heading the HUGO Fund are: Harriet Willett, chairman, and Hulings founder. Others include: Kelly Kilmartin, treasurer; Robert Terreberry, scholarships; and, Jennifer Davidson, secretary and Loan Closet administrator.

? ? ?

Hugo is a nickname Hulings was given by a friend, back in the days, he said, when ‘‘all the guys’’ worked extra hours all week, so they could catch their favorite football games on weekends. The name also is an acronym for ‘‘Humans Under God’s Oddities,’’ related to people with debilitating health problems.

Hulings said he’s looking forward to busy days ahead. For one thing, he’s deeply involved in the Ride For Life’s efforts to come up with prototype housing for patients with ALS.

‘‘The medical gurus,’’ he said, ‘‘are trying to put all ALS patients in nursing homes, instead of places that would meet our needs. ALS does not affect the mind.

‘‘We have a lot to offer in that respect. What we need is a type of group home that provides private space as well as opportunities to meet and compare notes.

‘‘My dream,’’ he said, ‘‘would be to have a ‘HUGO Home’ in our county.’’

Another priority, Hulings said, is spurring interest in contributions for Project ALS Research.

‘‘It would be just great if every person in the United States would donate $1 to the HUGO Fund for research, and if every person in Chautauqua County would give the same amount, we’d have about $140,000 as a starter.’’

Some time ago, Hulings programmed the DynaVox, and through use of an infra-red connection, and an ‘‘educated chin,’’ he has become a ‘‘computer whiz,’’ said Jennifer.

‘‘The programming took a long time,’’ Hulings said. ‘‘I lost track after 5,000 hours.’’

With that task finished, Hulings plans to become a Web master, and to update the HUGO Web page.

Meanwhile, he cherishes his latest memento — a special commemorative bat presented to him by Yankees owner George Steinbrenner.

‘‘There’s only one other like it,’’ Hulings said, ‘‘and, Steinbrenner has that one.’’

Hulings will never swing or even lift that bat. But, he’s hoping for a winning season this year — in terms of ALS donations to The HUGO Fund.

NEW YORK (Reuters Health) - Compared to years past, people who come down with Lou Gehrig disease nowadays seem to have slower disease progression and to live long. Nonetheless, amyotrophic lateral sclerosis or ALS, as the disease is known formally, is still always fatal, ultimately.

Baylor University researchers, reporting in the Archives of Neurology, have not found any specific factor to account for the improvements in recent years.

Dr. Adam Czaplinski and colleagues in Houston, Texas analyzed survival time and disease progression in two groups: 647 patients diagnosed with ALS between 1984 and 1999, and 394 patients diagnosed between 1999 and 2004.

The average survival after symptom onset was 3.22 years for patients in the first group compared with 4.32 years for patients in the more recent group.

Time to "a clinically evident change in a patient's clinical status and ability to perform activities of daily living" was 10 months in the current era compared with 9 months in ALS patients treated in earlier years.

The researchers observe that the improvement in outcome could not be attributed to any particular ALS-specific treatment, but they say they cannot rule out an effect of concurrent illnesses, "which could have influenced medical treatment and survival."

The researchers conclude that that there are other possible interpretations of the findings, including the possibility that there has been "a fundamental change in the natural history of the disease."

Dodd Introduces Measure to Ensure Coverage for Individuals with Terminal Illnesses
August 11, 2006

Washington- Senator Chris Dodd (D-CT) has introduced legislation in the Senate that would amend the Social Security Act (SSA) to ensure that individuals suffering from certain terminal diseases are entitled to receive social security disability benefits. Under current law, an individual who contracts a covered terminal illness, and who has not been part of the workforce for a period of time, may not qualify for social security disability benefits they would otherwise be entitled to.

“This bill is really about ensuring fairness. We should reward people who contribute to the social security system, not punish them,” Dodd said. “Under the current system hardworking Americans are being denied benefits at a time when they need them most,” Dodd said.

The Claire Collier Social Security Disability Insurance Fairness Act would amend the SSA to provide that the eligibility standard for disability insurance benefits not be applicable in the case of a disabled individual suffering from a covered terminal illness. Currently, the SSA mandates that a person earn 20 quarters of Social Security earnings during the 10 years preceding a disability to collect benefits. This discriminates against people who have earned the required number of credits outside of the time period prescribed.

This bill is named after Claire Collier, a Stamford mother of three, who was diagnosed with amyotrophic lateral sclerosis (ALS) in 2003. Sen. Dodd became aware of this issue after meeting with Ms. Collier in Washington

BILL TEXT
S 3839
VERSION: INTRODUCED IN SENATE
Aug. 3, 2006

109th CONGRESS

2d Session

S. 3839

To amend title II of the Social Security Act to provide that the eligibility requirement for disability insurance benefits under which an individual must have 20 quarters of Social Security coverage in the 40 quarters preceding a disability shall not be applicable in the case of a disabled individual suffering from a covered terminal disease.

IN THE SENATE OF THE UNITED STATES

AUGUST 3, 2006

Mr. DODD introduced the following bill; which was read twice and referred to the Committee on Finance

A BILL

To amend title II of the Social Security Act to provide that the eligibility requirement for disability insurance benefits under which an individual must have 20 quarters of Social Security coverage in the 40 quarters preceding a disability shall not be applicable in the case of a disabled individual suffering from a covered terminal disease.

Be it enacted by the Senate and House of Representatives of the United States of America in Congress assembled,

SECTION 1. SHORT TITLE.

This Act may be cited as the “Claire Collier Social Security Disability Insurance Fairness Act”.

SEC. 2. EXCEPTION FROM 20/40 REQUIREMENT FOR DISABILITY INSURANCE BENEFITS FOR INDIVIDUALS SUFFERING FROM A COVERED TERMINAL DISEASE.

(a) Exception From Recent Work Requirement.—

(1) IN GENERAL.—Section 223(c)(1) of the Social Security Act (42 U.S.C. 423(c)(1)) is amended in the flush matter following subparagraph (B)(iii) by inserting “or suffering from a covered terminal disease” after “216(i)(1))”.

(2) CONFORMING AMENDMENT.—Section 216(i)(3) of such Act (42 U.S.C. 416(i)(3)) is amended in the flush matter following subparagraph (B)(iii) by inserting “or suffering from a covered terminal disease” after “paragraph (1))”.

(b) Definition of Covered Terminal Disease.—Not later than 60 days after the date of enactment of this Act, the Commissioner of Social Security shall issue a proposed rule defining the term “covered terminal disease” for purposes of sections 216(i)(3) and 223(c)(1) of the Social Security Act (as amended by subsection (a)) that shall include (but not be limited to) those diseases that are incurable, progressive, and terminal, including neurodegenerative and neurological diseases that are likely to cause death within a 5-year period of onset.

(c) Interim Final and Final Rules.—

(1) INTERIM FINAL RULE.—Not later than 90 days after the date of enactment of this Act, the Commissioner of Social Security shall issue an interim final rule defining the term “covered terminal disease” in accordance with the requirements of subsection (b) and shall provide for a period of public comments on such rule.

(2) FINAL RULE.—Not later than 6 months after the date of enactment of this Act, the Commissioner of Social Security shall issue a final rule defining the term “covered terminal disease” in accordance with the requirements of subsection (b) and consideration of any public comments received during the period required under paragraph (1).

(d) Effective Date.—The amendments made by subsection (a) shall take effect on the date of enactment of this Act and shall apply to any applications for disability insurance benefits under title II of the Social Security Act that are pending or filed on or after that date.

Source: Government Printing Office
From CQ Bill Text Service
Providing government documents on demand, in context.
© 2006 Congressional Quarterly Inc. All Rights Reserved.
______________________________

Packard scientist Don Cleveland and his colleagues at the University of California, San Diego, reported details this week of a promising therapy-aimed study in ALS animal models that essentially shuts off action of offending genes.

The article describing the research appears in the August issue of the Journal of Clinical Investigation.

"Our work suggests that this technique can be an effective, regulatable way to treat types of ALS and other neurodegenerative diseases where we know the causative gene and the protein it produces," Cleveland says.

The study, in conjunction with researchers at California's Isis Pharmaceuticals, used therapeutic molecules called antisense oligonucleotides delivered to the brain and spinal cord of disease-bound rats, as well as healthy controls, via a tiny pump implanted beneath the skin.

Carried in the cerebrospinal fluid that bathes the central nervous system, the specially tailored oligonucleotides were designed to block effects of the mutant SOD1 gene that causes roughly 20 percent of inherited ALS.

Diffusing into brain and spinal cord tissues usually harmed by the disease, the antisense oligonucleotides attached to the very molecules that carry out the mutant gene's bidding, making them vulnerable to destructive enzymes. In test cultures of cells, antisense lowered mutant SOD1 protein - the gene's byproduct - to 20 percent of what’s typical.

And in the ALS model rats given antisense oligonucleotides a month before symptoms of disease typically appear, the effect could be seen as a 37 percent increase in the animals' survival. It slowed the rate of the insidious advance of paralysis.

"Antisense had no effect on the illness’s onset in the animals," Cleveland says. They still developed symptoms. And some mutant SOD1 protein still accumulated, even as high as 5 percent of protein in the brain. "But even with that, remarkably, there was dramatic slowing of the disease’s progress."

Much of the study was dedicated simply to proving the principle that antisense oligonucleotides, given this way, can squelch protein production. So Cleveland's team showed that was the case in healthy rats and also in the healthy Rhesus monkeys they tested. Animals apparently suffer no ill effects from being without some, most, or even all normal SOD1 protein, Cleveland says.

One arm of the research also depleted a protein thought responsible for much of disease progression in Alzheimer's disease. So it shows the potential of antisense therapy for a number of neurodegenerative illnesses, including Parkinson's and Huntington's disease.

As for its use in humans, nothing, so far, discourages continued research. "Safety of local and systemically given oligonucleotides is well established," Cleveland adds. More than 500 human subjects in other studies have had exposure to oligonucleotides similar to those used in this one.

"Our results support infusing antisense oligonucleotides as a treatment for familial ALS, especially for patients with the SOD1-A4V mutation which accounts for about 50 percent of SOD1 mutations in this country and which has an unusually rapid disease course after onset," says Cleveland.

If the formal safety studies underway in animals and the planned Phase I trials, to be carried out with Packard Center Clinicians, in human familial ALS patients support it - both, admittedly clear hurdles for a new technique - antisense could have genuine impact on the disease.

Richard Smith, of the Center for Neurologic Study, La Jolla, CA., and Tim Miller, a colleague of Cleveland's at UCSD, were also co-investigators in the study.

Cleveland is a consultant at Isis Pharmaceuticals Inc., which will manufacture the agent used in clinical trials.

Experiments in rats suggest it is possible to slow down amyotrophic lateral sclerosis using an antisense approach to therapy.

Amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig's disease) is a progressive disease of the nervous system which causes paralysis and death within a few years of diagnosis. It affects around 30,000 Americans and there is no effective treatment. Now a team at the University of California, San Diego, report on results of a trial in animals involving antisense oligonucleotides. These are molecules which can turn off a gene for a mutant protein which otherwise kills neurons in ALS.

In this new study, the researchers delivered the antisense molecules to the brain and spinal cords of rats through the cerebrospinal fluid and found the course of ALS to be slowed down. They hope this means that ALS could be managed as a chronic disease in humans. Their plan is to insert a small pump into the patient with ALS using a procedure already approved for the management of pain. Then a small catheter would be implanted into the area around the spinal cord so that the antisense drug could be pumped through. They also believe that this kind of antisense approach could work for other neurodegenerative diseases like Alzheimer's, Parkinson's and Huntington's diseases.

Source
Journal of Clinical Investigation early-advance 27th July 2006 (print, August)