Researchers from Indiana University and Johns Hopkins found that the drug safely slowed the deterioration of some daily activities of ALS patients including speaking, walking and dressing.

“The research demonstrates that talampanel appears able to slow the progression of disabling ALS symptoms,” said Dr Jeffrey Rothstein, senior scientist on the study.

“The effect isn’t overwhelming at the dosage of medicine used in this early, very small trial,” he adds. “Still, having promising human data is reason enough to keep it in the drug pipeline where we can really find out where it stands for patients.”

The Phase II testing of talampanel was only designed to discover its competency and safety on a small scale. Larger trials will need to be conducted before research moves forward.

An international trial is currently under way and should be completed by the end of 2010.

Read the full details at Science Daily

Knopp has completed the randomized, placebo-controlled portion of its Phase 2 studies of KNS-760704 in 102 ALS patients and expects to initiate Phase 3 studies in the U.S. and Europe in 2010. Subjects who completed the Phase 2 program were offered enrollment in an ongoing, 48-week open-label safety extension in which all participants are receiving the highest dose tested of KNS-760704.

The Fast Track Program was created to facilitate the development and expedite the review of new drugs with the potential to meet unmet needs in serious or life-threatening conditions. ALS is a universally fatal disease of progressive paralysis with limited treatment options.

“Knopp Neurosciences is very pleased to receive Fast Track designation and the recognition that KNS-760704 holds the potential to address unmet needs in ALS,” said Michael Bozik, M.D., president and CEO of Knopp. “We are pursuing every available opportunity to accelerate the development of KNS-760704, and will take full advantage of the opportunities under Fast Track to work with the FDA in designing a program to demonstrate the drug’s safety and efficacy.”

About KNS 760704

KNS 760704 is a low molecular weight benzothiazole shown to improve mitochondrial function and to confer significant cellular protection in neurons under stress. The chirally pure form of the synthetic benzothiazole (6R)-2-amino-4,5,6,7-tetrahydro-6-(propylamino)benzothiazole, KNS-760704 is highly orally bioavailable, water soluble, renally excreted, and only moderately protein bound. In Phase 1 studies, the compound was shown to be safe and well tolerated in healthy human subjects. KNS 760704 has received orphan drug designation from the U.S. Food and Drug Administration and the European Medicines Agency for the treatment of patients with ALS.

About ALS

Amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease and Charcot’s sclerosis, is a rapid, universally fatal neurodegenerative disorder characterized by progressive muscle weakness and wasting. ALS affects adults in the prime of life and creates a substantial burden for caregivers. U.S. prevalence is approximately 20,000 and the global incidence is approximately two per 100,000. Worldwide prevalence estimates of ALS range from 2 to 9 people per 100,000. Only one drug has been approved for the treatment of ALS. Life expectancy after symptom onset is usually three to five years.

About Knopp Neurosciences Inc.

Knopp Neurosciences is a drug discovery and development company focused on delivering breakthrough treatments for neurological disorders through innovation, experience, and partnership. The company’s lead product candidate is KNS 760704, an orally bioavailable small molecule in development for the treatment of ALS. Knopp’s leadership includes experienced neuroscience drug development and discovery executives formerly associated with major pharmaceutical companies. Knopp’s financing has been led by Saturn Capital Inc. of Boston as placement agent and Saturn Partners II as lead funder.

The molecules, known as immature superoxide dismutase 1 or SOD1, can’t interact normally with metals that provide stability and instead become destructive knots that are seen in one inherited form of amyotrophic lateral sclerosis, the formal name of Lou Gehrig’s disease, the University of Texas Health Science Center at San Antonio reports.

The center’s research, published in the journal Biochemistry, may shed new light on what triggers ALS, says lead author Duane Winkler.

His observations were made after examining new 3-dimensional images of the abnormal molecules in the center’s X-ray crystallography lab.

More than 100 mutations are known to impair the function of SOD1 molecules.

The Texas study focused on mutations that prevent the molecule from interacting normally with a second molecule called CCS, short for copper chaperone for SODI.

Winkler says CCS is like a waiter serving up orders of copper and zinc.

(Apr. 21, 2009) — Amyotrophic lateral sclerosis (ALS) is a fatal disease that damages key neurons in the brain and spinal cord. The disease causes progressive paralysis of voluntary muscles and often death within five years of symptoms. Although ALS (Lou Gehrig’s disease) was discovered over a century ago, neither the cause nor a cure have been found, but several mechanisms seem to play a role in its development, including oxidative stress.

Coffee, Caffeine and ALS

Researchers agree that ALS is a multifactorial disease that involves a complex interplay between a genetic predisposition and environmental factors. One environmental factor is diet. With oxidative stress (which damages the cells) a common concern in ALS pathology, it is worth examining what role antioxidants (which confer benefits to the cells) might play.

Antioxidants (the vitamins and nutrients that protect the cells from damage) are found in commonly consumed beverages and foods. Coffee in particular has received attention as a potent dietary antioxidant. It is worth noting that coffee has significantly more antioxidant capacity than cocoa and green, black or herbal teas. However, coffee contains several components, the largest of which are caffeine and chlorogenic acid, a dietary polyphenol that is beneficial to the immune system.

Previous studies have shown positive effects with coffee, caffeine, or chlorogenic acid supplementation in improving oxidative stress and its associated cell death mechanisms.

A New Study

A new study investigates the role of dietary intervention focused on an antioxidant popular in diets worldwide–coffee. The researchers examined the effect of coffee, caffeine and chlorogenic acid supplementation on markers of oxidative stress, antioxidant enzyme protein content and cell death in male and female mice models of ALS.

The study, entitled Caffeine Reduces Motor Performance and Antioxidant Enzyme Capacity in the Brain of Female G93A Mice, An Animal Model of Amyotrophic Lateral Sclerosis (ALS) was conducted by Rajini Seevaratnam1 supervised by Mazen J. Hamadeh1,2 , and co-authored by Sandeep Raha2 and Mark A. Tarnopolsky2 (1School of Kinesiology and Health Science, York University, Toronto, ON, Canada; 2Department of Pediatrics and Medicine, McMaster University Hamilton, ON, Canada). The researchers will present their findings at the 122nd Annual Meeting of the American Physiological Society, which is part of the Experimental Biology 2009 scientific conference. The meeting will be held April 18-22, 2009 in New Orleans.

Study Design

* Fifty-one G93A mice were randomly divided into eight groups: control (6 males, 8 females), coffee (5 males, 7 females), caffeine (5 males, 8 females), chlrogenic acid (5 males, 7 females). The control groups were fed a standard rodent diet and were not given any additional supplements. The intervention groups were provided with coffee, caffeine, and chlorogenic acid extracts, respectively, in amounts found in 5-10 cups of coffee per day, controlled for body weight.
* Clinical measures: Food intake, body weight, body condition, ability to move, clinical score, and motor performance were all assessed for the effect of diet and time prior to animal sacrifice.
* Molecular measures: Markers of oxidative stress (4-HNE; 3-NY), antioxidant enzyme protein content (MnSOD; CAT; GPx1; GR; GPx1 to GR ratio), and cell death (Bax; Bcl-2) were analyzed using the brains of these mice at age 108 days.
* Statistical analysis was conducted for males and females separately.

At the end of the study, the researchers found that:

In males:

* Coffee: increased food intake by 21%, decreased markers of oxidative stress by 39-65%, increased markers of antioxidant enzyme protein content by 46-139%, and decreased markers of cell death by 34-36%.
* Caffeine: increased food intake by 22%, decreased markers of oxidative stress by 45-81%, increased markers of antioxidant enzyme protein content by 21-99%, and decreased markers of cell death by 17-22%.
* Chlorogenic acid: increased food intake by 12%, decreased markers of oxidative stress by 25-35%, increased markers of antioxidant enzyme proteins by 23-44%, and decreased cell death by 41-44%.

In females:

Coffee: increased food intake by 30%, decreased markers of oxidative stress by 64%, but did not increase markers of antioxidant enzymes or decrease markers of cell death.

Caffeine: increased food intake by 28%, decreased motor performance by 20%, decreased markers of oxidative stress by 58%, decreased markers of antioxidant enzyme protein content by 11-48%, and increased cell death by 23-74%.

Chlorogenic acid: increased markers of oxidative stress by 178%, had equivocal effects on markers of antioxidant enzyme protein content, and decreased cell death 33-39%.

Conclusion

According to Ms. Seevaratnam, “If we were to extrapolate these results to human patients with ALS, then coffee appears to be beneficial for men, both reducing oxidative stress and cell death, and increasing antioxidants.  But for women, caffeine appears to be harmful. Women with the disorder may want to restrict caffeine consumption, or switch to decaffeinated products which contain the antioxidants, but with little caffeine.”
Adapted from materials provided by American Physiological Society.

“This is the world’s first search engine where patients can share disease-linked genetic information and use it to find other patients like them,” says James Heywood, co-founder and chairman of PatientsLikeMe. “Combined with our outcome, treatment, and symptom information, this represents a patient-centered model for realizing the goals of personalized medicine today.”

A major step toward incorporating genetics into the PatientsLikeMe platform for all diseases, the Genetics Search Engine is designed to allow patients to share additional data on genetic causes of ALS and help the research community learn more about the disease.

“With 64 patients sharing genetic information in the community, we have already begun to see variations in the progression rate by specific mutations,” says Catherine Brownstein, Ph.D., geneticist and researcher at PatientsLikeMe. “Transformative questions about how the disease varies by cause might be able to be answered far quicker than before.”

Margrit Betke, a College of Arts & Sciences associate professor of computer science at Boston University, believes the networked world isn’t nearly as inclusive as it ought to be.

“The community of people with severe disabilities is not really well served by computer science,” Betke says. Many people impaired by diseases like multiple sclerosis or ALS can’t type Google searches. They can’t play video games, and they can’t click on a friend’s e-mail.”

Click to find out how Margrit is helping folks with ALS interact with ther computers…

The campaign shines the spotlight on men and women from all walks of life who despite having the progressive, neurodegenerative disease - which on average has a survival rate of two to five years from the time of diagnosis - think of and help others in similar circumstances before themselves. Caregivers of people with ALS also are profiled in the campaign.

A former editor, golf course superintendent, soldier and local caterer, and the list goes on.

People who chose quite different career paths, yet all with something in common: Each is fighting ALS and has not given up on life and is helping others.

“Each of the 30,000 people in this country fighting ALS is a hero and has an inspirational story to tell,” said Jane H. Gilbert, president and CEO of The Association. “We are honoring those battling Lou Gehrig’s Disease who courageously embody the spirit of living life to the fullest and are making a difference in their community.”

The campaign also is reinvigorating those on the frontlines of helping patients and their loved ones.

“The national campaign ‘ALS Across America’ shares stories of courage and strength and unites ALS communities,” said Marilyn Simon-Gersuk, vice president of community services for The Association. “These people inspire and encourage us to be relentless in our search for a cure and in our commitment to help people with ALS and families.”

Prior to his diagnosis in 1997, Michael Jack, 42, rode his bicycle to work every day and participated in various physical activities including rollerblading. A resident of Alexandria, Va., Jack was the editor at the American Institute of Architects and an account executive for the public relations firm Porter Novelli. His new passion is helping The Association’s DC/MA/VA Chapter help others.

“Michael has always been someone looking to help others,” said Dan Iglhaut, 44, his caregiver. “As a volunteer ‘buddy’ and team leader with the Whitman Walker Clinic, an AIDS service organization, Michael continued to work with his client well after his ALS diagnosis and was honored as volunteer of the year. Michael has embraced The ALS Association not just for his needs but as an outlet to support his fellow patients with information, friendship and laughter.”

Steve Franks, 50, may have lost the ability to perform many of his tasks at his former job at Mainlands Golf Course in Pinellas Park, Fla., however, that has not stopped him from spearheading plans to transport 150 mannequins across the state in the “Piece by Piece Awareness Campaign.” Each mannequin represents a person who ALS has affected and whose body has literally been stolen “piece by piece.”

“The mission Steve is embarking on this year will be his biggest attempt to focus the public’s attention on this devastating disease,” said Kamden Kuhn, public relations coordinator for The Association’s Florida Chapter.

A former soldier and Army Security Agency communications worker, Bill Hamley, 65, gives inspiring speeches to such groups as the Physical Therapists Club at Quinnipiac College. The club members formed a Walk to Defeat ALS™ team.

“Bill is important to the chapter because of who he is and how he relates to others,” said Andy Byrne, patient services assistant for The Association’s Connecticut Chapter. “At every opportunity he helps to spread awareness both of the disease and of The ALS Association. Bill is a gentle man with a great deal of quiet strength. He has a deep spirituality that is reflected in everything he does.”

A local caterer turned ALS-educator and advocate, Jenny Hoff, 50, has made a strong impact on the community by tirelessly working on the telephone and her computer to assist others living with ALS and is a mentor to young people who she encourages to excel in their homework.

“Jenny is a tremendous advocate for The ALS Association who has contributed so much of her time locally and throughout the region to help educate and raise ALS awareness,” said Jennifer Hanson, development associate for The Association’s Evergreen Chapter. “She has embraced her ALS diagnosis with an incredible positive attitude and has become a tremendous advocate in our region to help teach others about this disease.”

Throughout ALS Awareness Month, The Association and its more than 100 affiliates will reach out to communities across the country to educate the public about Lou Gehrig’s Disease and urge people to join The Association in the fight to make ALS a disease of the past. National ALS Awareness Month activities include proclamations issued by cities and states.

The Association’s National ALS Advocacy Day and Public Policy Conference, which has grown to be the single largest gathering of the ALS community, and is part of ALS Awareness Month, will be held this year on May 10-12 in Washington, D.C.

People with ALS and families from across the country travel to the Nation’s Capitol to tell their stories in meetings with nearly every member of Congress, advance The Association’s public policy priorities, educate Congress about the true nature of this disease, and let them know why more must be done in the fight for a treatment and cure.

The ALS Association is the only non-profit organization fighting Lou Gehrig’s Disease on every front. By leading the way in global research, providing assistance for people with ALS through a nationwide network of chapters, coordinating multidisciplinary care through certified clinical care centers, and fostering government partnerships, The Association builds hope and enhances quality of life while aggressively searching for new treatments and a cure.

For more information about The ALS Association, visit our Web site at www.alsa.org

The Department of Veterans Affairs has determined that there is a connection between military service and amyotrophic lateral sclerosis, which is known as ALS and Lou Gehrig’s disease.

Joseph Armstrong, the state director of the Order of the Silver Rose, provided the Sanford News with a press release detailing the department’s announcement. Armstrong lives in Sanford and works with fellow veterans and supporters to bring awareness to veterans’ issues ranging from Agent Orange exposure to prisoners of war and those missing in action.

Veterans with ALS may receive badly needed support for themselves and their families after the Department of Veterans Affairs (VA) announced that ALS will become a presumptively compensable illness for all veterans with 90 days or more of continuously active service in the military.

“Veterans are developing ALS in rates higher than the general population, and it was appropriate to take action,” Secretary of Veterans Affairs Dr. James B. Peake said.

Peake based his decision primarily on a November 2006 report by the National Academy of Sciences’ Institute of Medicine (IOM) on the association between active-duty service and ALS.

“We are extremely grateful to Secretary Peake, Congressman Henry Brown and Senator Lindsey Graham for standing on the side of veterans with ALS across the country,” said Gary Leo, the president and CEO of The ALS Association. “Thanks to their leadership, veterans with ALS will receive the benefits and care they need, when they need them. Thanks to their efforts, no veteran with ALS will ever be left behind.”

The report, titled “Amyotrophic Lateral Sclerosis in Veterans: Review of the Scientific Literature,” analyzed numerous previous studies on the issue and concluded that “there is limited and suggestive evidence of an association between military service and later development of ALS.”

“ALS is a disease that progresses rapidly, once it is diagnosed,” Peake explained. “There simply isn’t time to develop the evidence needed to support compensation claims before many veterans become seriously ill. My decision will make those claims much easier to process, and for them and their families to receive the compensation they have earned through their service to our nation.”

According to the ALS Association, ALS is a “progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord.” The disease leads to complete paralysis in its victims — but almost always leaves their minds intact.

Sanford resident Gary Sullivan, the co-founder and president of the ALS-Maine Collaborative, was diagnosed with ALS in December of 2004. He expressed gratitude to Maine Senators Olympia Snowe and Susan Collins for their support of the bill Congress passed last September that provides immediate benefits to all Veteran’s diagnosed with ALS.

Sullivan also reacted to the VA’s determination that military service and ALS are linked.

“As a person with ALS, I am greatly saddened that those who have served our country with honor and courage now are at a greater risk of having Lou Gehrig’s disease because of that service,” Sullivan said. “The ALS-Maine Collaborative is proud to have taken a lead in the state in collaborating with the Veterans Affairs Department and the Togus VA Medical Center and other ALS-related organizations in addressing the medical needs and quality-of-life issues of all people with ALS in the state, including our veterans.”

A collaborative research effort spanning nearly a decade between Harvard researchers at Massachusetts General Hospital (MGH) and King’s College London (KCL) has identified a novel gene for inherited amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig’s disease).

This is the fourth gene associated with familial forms of the devastating neurological disorder. Two papers report mutations in FUS/TLS, a gene known to play a role in DNA repair and the regulation of gene expression.  The mutations affect the behavior of the FUS/TLS protein within cells and lead to deposits of abnormal protein within motor neurons.

Click for the full story at Harvard Science…

Thank you for your support as I raced for Barry Winovich and the Bright Side of the Road Foundation in their fight against ALS!

On Nov. 23, 2008, I’ll tested myself in a punishing 140.6 mile course across the desert in the Ironman Arizona competition in Tempe, Arizona.

During my race, there were several valleys where I was ready to throw in the towel. However ,there was no way I was not rolling across that finish line. I had Barry, as well  the Blazeman Warriors, carrying me the last 10 miles of the run!

Here were the final results for each part of the course:

SWIM: 1:21:48
BIKE: 5:51:28
RUN: 4:57:26
OVERALL: 12:22:09

I’ve posted a detailed report on the entire race. Click to check it out!

A big heartfelt thank you for everyone who sponsored my efforts in the Ironman Arizona as well as to my entire training and race day support crew!

~Kevin Sypolt