Lou Gehrig's Disease - Motor Neuron Disease - Amyotrophic Lateral Sclerosis
Thought it had been cured by now? Still no known cause. Still no cure. Still quickly fatal. Still outrageous.

Tuesday, December 30, 2008

WSJ - FDA Is Killing _____ Patients

Following was an opinion piece in this morning's (12/30/2008) Wall Street Journal. It perhaps might elicit some very pertinent thoughts from PALS.

Letters and comments to the Editors of the Wall Street Journal may be directed to


DECEMBER 30, 2008
The FDA Is Killing Crohn's Patients
Science didn't require that I get a placebo.


Right now there are millions of individuals whose lives are
directly dependent on the rate at which new drugs come to market. I'm one of
them. I'm fighting for my life.

To date, half of my intestine has been removed to manage Crohn's disease. Last year, at
age 23, I enrolled in a clinical trial for a treatment that could save my life:
an adult stem-cell therapy that helps damaged intestinal tissue regenerate from
the relentless inflammation and scarring caused by Crohn's.

The sponsor, Osiris Therapeutics, reported that Crohn's patients in the therapy's Phase II
trial all experienced clinical improvement after receiving the cells. A Phase
III trial for the treatment is now nearing completion, but Food and Drug
Administration (FDA) approval could be years away, despite its FDA "fast track"

In accordance with antiquated FDA policies, the Phase III trial is randomized with three groups of patients, and double-blinded, which means neither the doctors nor patients are told what treatment is being administered. One group received full-strength stem cells, another received half-strength, and a third got a placebo (the proverbial "sugar pill"). It appears I got the placebo.

Foregoing all other treatments, I received the four scheduled infusions, and yet my
disease progressed with a vengeance. In a matter of weeks, I became dangerously
malnourished. I've since been readmitted to the hospital countless times, as my
doctors continue to plead with Osiris for information. But Osiris has refused,
citing adherence to FDA protocol.

I am now a lab rat. I have no right to know what happened to me in the study, nor do I
have a right to try the promising treatment as my health deteriorates. It
doesn't have to be this way.

Under the Fifth Amendment's guarantee that "No person shall be deprived of life, liberty or property without due process of law," a critically ill patient should have access to a potentially lifesaving drug that has been deemed safe for human consumption, if the patient agrees to
bear the risks involved. But earlier this year, the Supreme Court refused to
hear a case on the issue, denying countless patients their right to pursue life.

Thankfully, some members of Congress have stepped in to ensure our
rights as patients. In May, Sen. Sam Brownback (R., Kan.) and Rep. Diane Watson
(D., Calif.) introduced the Access, Compassion, Care and Ethics for Seriously
Ill Patients Act. If passed, this bipartisan legislation will begin to restore
the rights of millions of patients by widening access to promising
investigational drugs.

Human clinical research is an intricate scientific and moral process, but it does not justify taking immoral advantage of patients. Tragically, FDA and Osiris think it does.

Typical approval protocols almost always guarantee patients taking
the placebo access to the actual drug -- at the very least -- after the study
has ended. But in what appears to me a deliberate act of cruelty, Osiris hung
its patients out to dry without any recourse, refusing to confirm which patient
got what. The FDA has endorsed Osiris's decision by enabling it to proceed with
the study.

Withholding a potential cure is just as bad -- if not worse -- than the potential death sentence of a serious illness. If patients like myself have the audacity to put their lives on the line for the betterment of science and those in their predicament, their decision should not only be embraced, it should be rewarded.

Furthermore, trials without ethical recourse can lead to inadequate and
incomplete data, compromising the integrity of the study. If trial patients are
treated like lab rats, they won't feel obliged to cooperate unconditionally and
report accurate data -- something the FDA and the drug industry rely on heavily,
but have failed to consider.

Everyone agrees it is a fundamental right for patients to dictate their course of treatment with FDA-approved drugs. So why do the rules evaporate at the most critical moment, when the only life-preserving options are highly promising investigational drugs?

Mr. Sofer is a student at the University of California, Berkeley.

Write to

Thursday, December 25, 2008

We Can Only Hope That President-Elect Obama Read The Morning Paper in Hawaii Today

Artist's strength, living with incurable illness, will be an inspiration foreverSTORY SUMMARY
In a daily countdown, the Star-Bulletin is profiling 10 people who have made a difference in Hawaii during the past year.
These are people who worked in any field - community service, education, politics, law, labor, medicine, science, business, sports, entertainment, the arts - to make a difference. Some fought controversial battles in public.
All that matters is that each, to the one, had a devotion to their cause that made a profound impact on Hawaii.
By Betty Shimabukuro
POSTED: 01:30 a.m. HST, Dec 25, 2008 "A celebration of life" - that is the phrase we use to take the edge of sadness out of a funeral. But when services were held for artist Peggy Chun on Dec. 5, it would have been hard to call it anything but a celebration.
"Mourners" wore butterfly wings, antennae atop their heads; a few dressed as penguins - and although tears were shed, smiles and laughter were in abundance as well.
Chun died on Nov. 19 at age 62 after a six-year battle with amyotrophic lateral sclerosis - ALS, or Lou Gehrig's disease - although "battle" might not be the right word. Even as the disease robbed her of all power of movement and speech, Chun seemed to view each progression as inspiration, not degeneration.
"I'm happier now than I've ever been," she said in 2003, a year into the disease and already traveling by wheelchair. "It's been one hell of an adventure after the diagnosis."
In 2008, although she could communicate only by directing her eyes toward letters arranged around an alphabet frame, Chun supervised the completion of a mosaic of Father Damien, made of thousands of tiny squares of paper that were painted by students at Holy Trinity School.
Although well respected for her art, the indelible mark that Chun leaves is in the example she set, finding purpose and fulfillment while coping with a devastating illness. She rallied hundreds of people around her - a volunteer army called Peg's Legs who shared her care and who were certain that her mind remained active and her spirits high until the end.
"She was a force of nature," said Lynn Cook, who became the family's spokeswoman in the final days of Chun's life. "If she didn't want the gallery to get wet, I swear she could deflect storms."
Chun prepared for each stage of her illness so that she could keep painting. She used her left hand when her right failed, then held her paintbrush in her mouth, then used a computer program that allowed her to paint through eye movements. She treated each adaptation not as a compromise, but as a new skill that added texture to her work. ...

Monday, December 22, 2008

Go, Ravens... Beat ALS

From the Baltimore Sun, December 22--
The team dedicated Saturday night's victory to director of player development O.J. Brigance, who attended his second road game of the season despite his battle with amyotrophic lateral sclerosis. "We dedicated it to O.J. Brigance because of the things that O.J. Brigance talked to our football team about last summer when we started training camp on the very first day," Harbaugh said. "And we told them no matter what your condition, no matter what your circumstance, no matter what hardship you face in life or football, you overcome it. And that's what our football team did."

OK, advocates. How do we leverage the fact that an NFL team is using an ex NFL player with ALS to motivate it through the most exciting part of the football season? How do we get the word out about Brigance and the many other NFL alumni who have or have died from ALS?

The longer the Ravens stay alive in the playoffs, the more opportunities there are for O.J. Brigance to be front and center in their fight... and in his fight for his life.

These Students Rock

We can't do enough to engage young people in the ALS cause. They often have a gift for cutting to the chase and getting publicity and attention. They also are tomorrow's physicians and scientists and political leaders.

Sunday, December 21, 2008

Here's A Beautiful Column on Friendship, Courage, and ALS

...and when you read columns like this, they're perfect for supportive letters to the editor. That can encourage more writing on the subject and certainly keeps ALS in the public eye in one more way.

Thursday, December 18, 2008

ALS Is Murder, She Wrote

The internet rumor mill indicates that Angela Lansbury may be featured in Sunday's PARADE magazine and her involvement with the ALS cause may be mentioned.

Thank You, Lou Gehrig.

After all these years, Lou is still the one who can get this stinking disease with the name that is impossible to pronounce into the public eye.

Too bad there wasn't more media coverage.

Tuesday, December 9, 2008

Petition Being Circulated - National ALS Day, Lou Gehrig Farewell, July 4, 2009

The following petition is being circulated and was passed to . The power of the internet is mighty. For your consideration...

Petition to make July 4th National ALS Day

To: All Major and Minor League Baseball Franchises, US Congress, US House of Representatives On April 30th 1939 The New York Yankees were defeated by The Washington Senators in what would be the last game of Lou Gehrig's legendary baseball career. Two years later Lou Gehrig would lose again, but to a more formidable opponent, Amyotrophic Lateral Sclerosis (ALS). ALS, a progressive and usually fatal degeneration of the nerve cells that cause voluntary muscle movement was first described by French neurologist Jean-Martin Charcot in 1874. It’s been close to 150 years since then and we still don't have an effective treatment or cure for this disease. It is estimated that everyday in the U.S. 15 people are diagnosed with ALS. I know that doesn’t sound like very many people, but consider the fact that this disease has no cure and no treatment. Each year 5,600 people in our country alone are faced with the prospect of gradually making the transition from cane, to walker, to wheelchair. Losing their strength, mobility and independence. Losing their hope that a treatment or cure for this disease will be discovered in their lifetime. There is a gross lack of funding for the research of this disease. Many of you reading this petition are well aware that despite the valiant efforts of several organizations across the country, WE ARE LACKING THE FUNDS TO EFFECTIVELY RESEARCH AND DEVELOP TREATMENTS FOR THIS DISEASE. On July 4th 1939 Lou Gehrig stood on the field at Yankee Stadium in front of a packed house and said goodbye to the game that he loved so much. The spotlight has never shone as brightly on ALS as it did with the passing of Lou Gehrig. This July 4th will mark the 70th anniversary of that historic day in baseball and it would be the perfect opportunity for fans of our great American pastime and for the MLB franchises to stand up together in a statement to ALS that we will NOT be defeated. We WILL find a cure. We WILL honor the memory of Lou Gehrig and everyone who has been touched by this terrible disease. The time is now and we CAN do something to make this happen. Every 4th of July millions of baseball fans gather in stadiums, around televisions and around radios to cheer on their favorite teams. Let's take this opportunity to remind people that every single day this disease is taking lives. Let's rebroadcast Lou Gehrig's farewell speech. Let's give fans an opportunity to remember his legacy and donate to this worthy, but neglected cause. We need to make the 4th of July to ALS what Labor Day is to the MDA. This should be our holiday. This is our chance to raise in 24 hours what we could never hope to raise in a years worth of smaller fund raisers. Let's organize volunteers to collect donations at baseball fields across the country. Let's contribute that money to ALS research and let's knock one out of the park for our friends and family struggling against this disease. LET'S KNOCK OUT ALS! We are petitioning you that July 4th 2009, the 70th anniversary of Lou Gehrig's farewell speech and every July 4th from this day forward be declared National ALS Awareness Day and that opportunities be provided at baseball fields across the country for people to donate to ALS research. Sincerely, The Undersigned Click Here to Sign PetitionPetition Link

Friday, December 5, 2008

PatientsLikeMe Hits The Media

  1. Mentioned on Diane Rehm on NPR this week in a conversation on information in healthcare
  2. Featured in Business Week
  3. To be covered on CBS Evening News tonight (December 5)

Here's hoping that ALS is always mentioned as the compelling reason for the creation of .

ALS Registry Act - Authorization Doesn't Get The Job Done

The ALS Registry Act passed a few months ago was a big, difficult victory, but authorization alone doesn't cut the mustard. Without funding, authorization is a hollow victory. Nobody at the CDC will be counting noses and retaining ALS data until some funding is assigned to the ALS Registry.

The new administration seems to be in listening mode, and now is the time for us to give them an earful about ALS and the need to get the registry funded and implemented.

From this morning's (December 5) Wall Street Journal --

... Mr. Daschle will say the Obama administration wants input
from supporters -- as a way of getting average Americans to feel connected to
the process and invested in its success. At some point, the administration may
seek to mobilize supporters to lobby Congress or otherwise build support for a
plan.... Already, the Obama transition office has posted two health-care
videos on its Web site,,
and it is soliciting comments and ideas from people via the site.

Please use this opportunity to express the importance of implementing the ALS Registry to HHS Secretary-elect Daschle and the Obama administration at and "Submit Your Ideas." Today they are listening. By January, they may be so overwhelmed with problems that it will be much harder to get ALS on their radar.

Monday, December 1, 2008

ALSA 2009 Advocacy Priorities Being Formulated

ALSA chapters are in the process of assembling their top three issues for advocacy for 2009. Chapters' top three issues must be submitted to the ALSA Washington, DC, office by December 12.

Below includes items in the menu of choices which was provided to chapters. Perhaps your local chapter would appreciate hearing your thoughts on your top three priorities.


2008 / 111th Congress Advocacy Listening Tour Issues

National ALS Registry
· Implement the ALS Registry Act, which establishes a national ALS registry at the Centers for Disease Control and Prevention.
· List ALS as the cause of death on death certificates.

· Increase ALS research funding in general – National Institutes of Health, Department of Defense, Veterans Affairs, Centers for Disease Control.
· Stem cell research, including relaxing federal restrictions, expediting human trials.

· Encourage the development of products to treat ALS through FDA drug development process, including FDA Critical Path/Reagan Udall Foundation.
· Ensure that efforts to establish a new approval process for generic biologics do not hinder access to new drugs or drug development.
· The Access Act, legislation that would ban the use of placebos in clinical trials and allow terminally ill patients to purchase investigational drugs once the patients have exhausted existing treatment options and are not eligible for any clinical trials.
· Other issue (please explain)

Veterans Issues
· Ensure implementation of new rules for veterans with ALS that make ALS a service connected disease and establish expedited procedures for veterans to access benefits.
· Assistance accessing/navigating the VA system.
· Develop partnerships for clinical care with the VA, in terms of both research and practice.
· Change functional criteria needed to qualify for adaptive housing grants.
· Other veteran’s issue (please explain)

Long-Term Care Services
· Promote respite care and respite care legislation, including securing appropriations for the Lifespan Respite Care Act.
· Provide Medicare and private coverage of in-home custodial care services and home health aides.
· Change Medicare’s homebound requirements.
· Reimburse family caregivers.
· Establish caregiving tax credits separate from the deduction for medical expenses.
· Provide health benefits to family caregivers.
· Improve access to long-term care insurance at both the state and federal level.
· Encourage home-based care.
· Other long-term care issue (please explain).

Social Security
· Educate local Social Security Administration personnel about the rules for ALS.
· Eliminate the 5-month waiting period for SSDI (disability benefit).
· Eliminate the "20/40" rule under which a person with ALS must have worked five of the previous 10 years in order to qualify for SSDI.
· Other Social Security (please explain)

Medicare Coverage of Durable Medical Equipment (DME)
· Oppose reductions in reimbursement for Durable Medical Equipment.
· Improve coverage for communication/speech generating devices in general.
· Oppose Medicare changes that would eliminate the ‘first month purchase option’ for customized wheelchairs.

· Improve coverage criteria for wheelchairs, including power chairs.
· Improve coverage for bipaps.
· Improve coverage for lift chairs, transfer benches, grab bars, shower chairs, toilet safety frames, toilet seats for bathrooms and kitchens.
· Improve coverage for air-fluidized mattresses and pressure mattresses.
· Improve coverage for DME in general.
· Mandate private coverage for DME needed by PALS.
· Establish caps on out-of-pocket expenses for durable medical equipment.
· Improve coverage for SLP AAC evaluations.
· Other DME issue (please explain)

General Medicare/Medicaid
· Streamline/reduce excessive and burdensome paperwork requirements; address the inefficiencies of Medicare and private insurance.
Estimated percentage of PALS/caregivers/others impacted by the issue in your area: _____
· Educate Medicare and hospice providers that Rilutek is not an aggressive treatment for ALS and should not be a reason for denial of hospice services.
· Speed processing of Medicare claims.

· Other Medicare issue (please explain)
· Other Medicaid (please explain)

Medicare Prescription Drug Benefit
· Improve the current drug coverage to eliminate the coverage gap (‘doughnut hole’).
· Limit the use of utilization management tools such cost sharing, tiering, therapeutic/generic substitutions, quantity limits and other policies of Medicare prescription drug plans.
· Ensure Medicare coverage of specific prescription drugs (Rilutek is currently covered). Please list other drugs that should be covered.
· Ensure Medicare coverage of drugs prescribed off label.
§ Other Medicare Prescription Drug Benefit issue (please explain)

Other Issues
· Charitable giving incentives - allow non-itemizers to deduct charitable gifts.
· Obtain grants to educate nursing homes on how to care for ALS.

· Obtain grants to establish ALS specific nursing facilities.
· Obtain grants to provide transportation assistance and other support to homebound patients.
· Obtain grants to provide social worker consultation services to PALS and families to assist in identifying services.
· Provide counseling for PALS and caregivers to help them cope with the disease.
· Provide legislative protections to prevent abuse and neglect of PALS.
· Ensure adequate supply of flu vaccine and enforcement of CDC guidelines for triage.
· Monitor World Trade Organization activities that impact the availability and dosage of vitamins and supplements that can be acquired without a prescription.
· Support legislation that would adjust the federal financial aid formula for PALS so that the value of their home equity is deducted from the tuition assistance formulas.

§ Other issue (please explain)