One Size Never Fits All

As poor as the American high school education system can be, it does have a grip on one concept that we could apply to the ALS cause.

Think of the four years and the differences in students between that first year and the fourth year. Think of the differences between student interests and their parents' interests. It's not rocket science that schools find activities and niches and educational approaches for first-year students that are vastly different from those supplied for seniors. Educators present different programs for parents than for the students themselves. Alumni are engaged very differently than sophomores. Neighbors of the school are included differently from seniors.

No school is foolish enough to toss up one educational program and then to expect all of the stakeholders to achieve at their highest levels.

Since the typical course of ALS takes around four years, perhaps we can learn from an analogy with the high school education experience. One size of advocacy engagement does not fit all.

Still "Indestructible"

The late Chicago filmmaker Ben Byer, who chronicled his fight against Amyotrophic Lateral Sclerosis, commonly known as Lou Gehrig's Disease, in the award-winning film "Indestructible," will be posthumously honored by the Chicago Muscular Dystrophy Association (MDA). The Ben Byer ALS Awareness Award will be presented at the Annual Benefit for ALS Research, A Toast to Life at 6 p.m Saturday at Enclave, 220 W. Chicago Ave.

 

For ticket information, visit  Toast To Life  or  Indestructible

ALS Gives One An Interesting Perspective On Healthcare Delivery

People with ALS and their caregivers have special insights into healthcare delivery and could add a valuable voice to the heathcare conversation that started at the White House yesterday.

The big White House powwow included key members of the Senate and Congress, representatives of associations of insurance companies, pharmaceutical companies, physicians, other healthcare professionals, independent citizens, etc. Many organizations representing consumers were there as were small business organizations and labor unions.

The focus was on healthcare reform, including cost reduction and increasing access to quality care. All of the stakeholders had a chance to be heard.

Once you've had the ALS experience, the inefficiencies in American healthcare delivery scream at you. The waste is sickening. The system doesn't deliver at the same pace that this very aggressive disease races through one's remaining years, and the system isn't exactly nimble enough to adapt to the need. It likes to do what it does.

Perhaps President Obama and our Senators and Congresspeople would benefit from some first-hand testimony on how healthcare delivery does not deliver. It's hard to build a truly better mousetrap if you don't confront the problems and their causes in the old mousetrap.

From the perspective of a caregiver to one with ALS, healthcare delivery today is much like package shipping was fifty years ago. You mailed the Easter package to be sent to your grandmother a few hundred miles away at the beginning of Lent, or it wouldn't make it by Easter. You had to wrap it with special paper and twine or it would be rejected. You had to buy insurance because there were pretty good odds that it would not show up in Cleveland or it would be damaged if it did get there. Today with improved logistics, better information systems, and effective competition, we have a choice of economical and effective ways to send a package to Cleveland. That is true progress.

PALS and CALS voices would be valuable for this discussion. We're not asking for the cure this time. We're simply willing to add some insight into out some areas where the system is failing expensively.

Call for ALS Demonstration in D.C. in May

Those involved with ALS research repeatedly make the point that the goal of getting effective treatments for ALS is directly a function of the amount of funding for ALS research. I've followed, off-and-on, both ALS research and ALS advocacy, since around 1981 and have seen far too many false starts and false promises, while during the same period, a disease like Aids has gone from being unknown to having very good treatments. So given this, I’d like to throw out the following suggestion for what I think would be effective advocacy:

Every year in May the ALS Association has Advocacy day in D.C. and lobbies Congressional offices (Congress is usually out of session during this time in my experience). While over the years they have advocated for some good and needed legislation, when it comes to any significant funding SPECIFICALLY for ALS, they have been very timid - nothing approaching what is needed or commensurate with a horrible disease like this with no real treatments whatsoever. So I propose that during ALS advocacy day in May, when many PALS and CALS will be in D.C. for this, that a protest demonstration be organized to DEMAND (not ask) MUCH greater federal funding dedicated to ALS research (which has always been disproportionately underfunded).

The Obama administration is pouring a lot of new money into the NIH for research, including through the just-passed stimulus package. I see no reason why ALS shouldn’t demand and get its fair share. But without a real advocacy push, I guarantee that ALS will, at best, just get some small increase, probably proportionate to the overall increase in funding (which likely wouldn’t even offset the decrease in inflation-adjusted funding that has occurred over the past few years).

Having observed and participated in protests, marches and other advocacy in the Nation’s Capitol for some decades now, I have seen the effectiveness of even small groups of protestors on the Capitol steps and other actions. The early disability advocates - most in wheelchairs - did this with great effect decades ago; to get laws and policies changed, leading up to the Americans with Disabilities Act. And the militant tactics of the early AIDS activists (groups like ACT UP) garnered attention and ultimately catalyzed public and private support for AIDS, at levels far beyond any justified on the basis of any medical, scientific or rational criteria. Following their lead, more militant Breast Cancer Activists blocked entrances on the Capitol, chanting that it was UNACCEPTABLE for breast cancer to be killing women; leading to funding increases that jumped many times in a short period of time. With somewhat less success, Prostate cancer advocates followed suit.

Not that this is the most rational or equitable way to make funding decisions, but this is how the game is played - more politics than science or medicine. Also, do not forget that in the early 80's, militant AIDS activists who disrupted board meetings were not exactly a sympathetic group, unlike PALS, who would certainly be seen this way by the media and the public. And yet those early AIDS militants were damn effective in forcing both policy changes in drug studies, etc. and, of course, in convincing the government to pour massive amounts of funding into AIDS research.

And yet, over the 28 years I’ve followed ALS advocacy, except for the March of Faces (do they even still exist?) demonstrations outside of the FDA office to maintain access to Myotrophin in the 90s, I’ve not seen any comparable militant or effective ALS advocacy - at least in terms of affecting federal funding. The MDA even shunned asking for federal funding for decades, until just a few years ago, even though the federal government is by far the biggest funder and potential funder of medical research, especially at a time like this, when private funding sources are generally not in much shape to be shelling out dollars for anything.

So what do people say? Mid May is coming up fast, but with organizing over the internet it should be possible to put together an effective protest/demonstration; one which might draw on many of the PALS and CALS who will already be in town for ALS Advocacy day. By now, it should be clear that doing the same things the same old way has simply produced the same old results, which in this case has meant the same basic, anemic level of funding for ALS, even as the cost of research has usually gone up faster than inflation, meaning that the same level of funding is really a decrease in real dollars.

I’ve heard the arguments against such actions, but don’t buy them for the most part. If most PALS are too sick or otherwise unable to attend such an event, there are CALS, family member and friends of CALS and all of the above for PALS no longer with us. All told, there must be hundreds of thousands of persons in the U.S. who know, knew or have been affected by ALS, directly or indirectly. And for such advocacy, large numbers, while always nice, are not as necessary. If half those who come every year for ALS advocacy day participated, that probably would be sufficient to gain the crucial media attention and force Congresspersons to go on the record, making some statement. Plus, there are a fair number of people who worry they may have ALS, not to mention some number who have related motor neuron disorders or similar one’s, like Multifocal Motor Neuropathy - all of whom could be invited to join in.

BTW, I recently heard the Governor of South Carolina, Mark Sanford, mention that his father had ALS, so maybe he could be a champion.

Thanks for reading

Hopeseeker

ALS or FDA

Which of these three letters scares you the most?

The FDA has done it again! Last December the FDA cleared the way for Neuralstem, Inc. to conduct clinical trials on ALS (amyotrophic lateral sclerosis) patients by injecting stem cells into their spinal cord. The company had filed an Investigational New Drug (IND) application with the FDA to begin a clinical trial to treat ALS. The plan is to treat ALS patients through spinal injections of stem cells via its patented Human Neural Stem Cell Technology at Emory University.

Last week the FDA put a hold on this clinical trial and said it needed more information about the manufacturing capabilities, and all studies and injection methods for treatment. Once again, as with other therapies, the FDA has asked for a modified protocol including a patient's eligibility to participate. How much more qualified can ALS patients be? If the IND describes the clinical trial that is directed solely at ALS what then is their alternative. Could it be that the FDA does not understand that having ALS is already a death sentence. The legal waivers to participate in any clinical trial could never match the horrors of ALS.

For the past 21 years, The Life Extension Foundation has compiled evidence indicating that the FDA is the number one cause of death in the United States. The FDA causes Americans to die by:

• Delaying the introduction of life-saving therapies
• Suppressing safe methods of preventing disease
• Causing the price of drugs to be so high that some Americans do without
• Denying Americans access to effective drugs approved in other countries
• Intimidating those who develop innovative methods to treat disease
• Censoring medical information that would let consumers protect their health
• Censoring medical information that would better educate doctors
• Failing to protect the safety of our food
• Misleading the public about scientific methods to increase longevity

A startling number of reports reveal the FDA is in far worse shape than originally thought. Few people comprehend that they are likely to suffer and die prematurely as a result of FDA’s failures. The greatest threat the FDA poses to our health is the fact that the agency functions as a roadblock to the development of breakthrough medical therapies. Innovation in medicine is stifled by FDA red tape, which is why Americans continue to die from diseases that long ago might have been cured.

Tell Congress we need an HHS Secretary now.
The clock is ticking on comprehensive health care and FDA reform - and for millions of Americans, time has already run out. Leaving us without a strong advocate in Washington is simply unacceptable.

Americans cannot afford to wait any longer. Tell Congress to work together for swift confirmation of Kathleen Sebelius as Secretary of Health and Human Services.

Sign the petition to be delivered to the Senate.

A New Gene for ALS

What sharing your genetics could mean for research
In today’s issue of the journal Science two papers describe the discovery of a new gene for ALS (you can read the abstracts here and here). 95% of ALS cases are sporadic, i.e. we don’t know what causes them, but for 5% of patients the disease runs in their family (known as familial ALS, FALS). Until today, there was only one major causative gene that we knew about, called SOD1, which accounted for 20% of familial cases. Today’s new discovery of the gene FUS (also known as ALS6) accounts for an additional 5% of familial cases and was the result of an international collaboration between scientists in Boston, London, and Sydney. This is very exciting for research because the more we know about what causes ALS, the better our chances of finding an effective treatment through better understanding of the pathways involved in motor neuron degeneration.

Here at PatientsLikeMe, we’ve recently upgraded our ALS platform to capture data on familial ALS patients’ known genetic mutations. The goal is to help familial ALS patients find another patient like them, and to enhance understanding of the phenotype of each mutation, e.g. if different types of mutation cause a faster or slower disease progression. Ultimately our aim is to try and establish whether there might be any treatments that have a differential effect on patients with different disease-causing mutations. There are examples of this already known in other diseases; for instance the presence of absence of the Philadelphia chromosome in chronic myelogenous leukemia (CML) predicts whether the patient will respond to the drug Gleevec. Although there is currently only a single effective treatment for ALS (Rilutek), there are a number of trials underway investigating the potential of drugs for patients with specific gene mutations.
source & data Patients Like Me

Researchers identify ALS gene mutation - Type 6

Early study is key to latest discovery

CHICAGO --- Research that has discovered a new gene whose mutations cause 5 percent of inherited cases of ALS (amyotrophic lateral sclerosis) is part of a national study led by the Northwestern University Feinberg School of Medicine.

The study reported in Science today (Feb. 27) points to a common cellular deficiency in the fatal neurological disorder, said Teepu Siddique, M.D., Les Turner ALS Foundation/Herbert C. Wenske Foundation Professor in the Davee Department of Neurology and Clinical Neurosciences and Department of Cell and Molecular Biology and Director of the Division of Neuromuscular Medicine at the Feinberg School.

The new research is part of a national collaboration directed by Siddique, the principal investigator for the "Genetics of ALS" project funded at Feinberg by the National Institutes of Health.

Earlier research by Siddique and colleagues extended the genetic knowledge of familial (inherited) ALS by identifying the first and second ALS genes (the SOD1 gene in 1993 and the ALSIN gene in 2001), in addition to identifying loci on chromosomes 9, 15, 16, and X.
The study published today discovered aFUS/TLS gene mutations in ALS families collected through efforts of the NIH-funded multi-center project and included among others a large Italian family previously studied by Siddique and Cortelli.

ALS affects the motor neurons in the central nervous system. As motor neurons die, the brain's ability to send signals to the body's muscles is compromised. This leads to loss of voluntary muscle movement, paralysis and eventually death from respiratory failure. The cause of most cases of ALS is not known.

"The purpose of this national study is to understand what triggers the death of motor neurons in order to find new cellular models of ALS, with the ultimate goal of advancing research that leads to a treatment for this fatal disease," Siddique said. "Approximately 10 percent of ALS cases are inherited."

"The discovery of this gene mutation shows new kinds of molecular defects that damage motor neurons and it implicates defective pathways previously identified in other genetic forms of ALS," said Siddique.

The new findings were reported in Science by the University of Massachusetts Medical School, one of three institutions that collaborate with Siddique on the national study. Other authors from the consortium include M.A. Pericak-Vnace (University of Miami and Jonathan Haines (Vanderbilt University). Robert H. Brown Jr., M.D., chair and professor of neurology at University of Massachusetts Medical School, was senior investigator of the study and lead author of the Science paper.

In addition to funding from the NIH, Siddique's ALS research is supported by the Les Turner ALS Foundation, Vena E. Schaff ALS Research Fund, Harold Post Research Professorship, Herbert and Florence C. Wenske Foundation, Ralph and Marian Falk Medical Research Trust, The David C. Asselin MD Memorial Fund, Les Turner ALS Foundation/Herbert C. Wenske Foundation Professorship, Help America Foundation and the ALS Therapy Alliance, Inc.
source & data;Les Turner ALS Foundation

The Art Of Fundraising Vs. The Science Of Curing A Disease

Augie's Quest has announced that the $18 million fundraising goal over the last three years has been met.

The ALSA About-Us webpage indicates, "during the past decade, we have committed close to $40 million toward ALS research."

Not-for-profits' marketing materials all tout their relentlessness and and certainly nobody is leaving stones unturned. The financial footprint is used to distinguish an organization's effectiveness. Presenting a perception of a big financial footprint in research certainly helps attract new donors; however, the numbers we see are terribly difficult to understand.

If we were to put together the actual annual annual expenditure on ALS research in 2008, we would probably see much smaller numbers than we perceive with the three-year and ten-year millions. Oh, and we need to make sure that we take out any double counting when one organization gives a research grant to another.

The ALS-research bottom line is terribly small in the big medical research arena. That fact is often lost in the unfortunately necessary fundraising marketing techniques that involve multi-year, cumulative numbers that are hard for many of us to normalize.

Much more needs to be invested. Are the billboards on the highway to a cure distracting us from this big problem?

Dandruff Steals The Headlines... Besides, You're Not Likely To Have This Disease

http://www.southcoasttoday.com/apps/pbcs.dll/article?AID=/20090223/LIFE/902230304/-1/ENTERTAIN

From Dr. Donohue's "To Your Good Health" column at www.southcoasttoday.com ... way under the featured question on dandruff...

DEAR DR. DONOHUE: My father was 67 when he passed away in 1989 from ALS, Lou Gehrig's disease. He had it for only two years. I am 60 years old and a Vietnam veteran from 1968. I had extensive nerve and muscle wounds in both legs and my left arm from the war. I get muscle spasms in my legs and am concerned that it might be ALS. Is ALS hereditary?
— B.W.

Only in a few cases, 5 percent to 10 percent, does heredity play a major part in ALS. ALS is amyotrophic lateral sclerosis. It's a dying off of nerve cells in the brain and spinal cord that control muscle movement. In its final stages, it leaves a person a prisoner of his own body, unable to move and often unable to swallow or talk. The initial symptom of ALS is muscle weakness, not muscle spasms. You're not likely to have this illness.

Readers may write to Dr. Donohue or request an order form of available health newsletters at P.O. Box 536475, Orlando, FL 32853-6475.

Dr. D's response is correct from the textbook. The part that is missing is that since veterans have an astronomically increased risk of ALS, perhaps B.W. might want to touch base with a VA neurologist to check out whatever his problem may be.

"You're not likely to have this illness," is a sentence that thousands of people with ALS have heard from primary care physicians before being diagnosed correctly by knowledgeable neurologists.

Support Stem Cell Research

Stem cell research has great potential to treat, or even cure many fatal illnesses, including ALS. America has not pursued stem cell research with the level of vigor because it basically boils down to this: a small but vocal minority in the country believe stem cell research is baby butchery akin to legalized abortion, and for the last eight years a president who agreed, or simply didn't want to tick that small minority off, was in office. A lot of other politicians who should have known better heard words like "snowflake babies" and "abortion" and stayed out of sight with no comment. America and its scientists have waited long enough to show the world what they can do.

Not for much longer. Millions of Americans and hundreds of millions more worldwide who have been afflicted by fatal or terminal illnesses can actually begin to imagine, they can hope. They can dream of the incurable becoming cured.

Contact President Obama and urge him to remove the current restrictions which limit stem cell research. With your help, we can ensure that the ALS research community can pursue every opportunity to find a treatment and cure for Lou Gehrig's disease as soon as possible.

Welcome, at last, to the 21st century.

Take Time and Take Action.

Disease For Sale By Owner... Or At Least Foreclose On It And Get It Out Of Our Lives

Every year the National Association of Realtors has its mid-year meeting in Washington, DC, in May. It's like the ALSA advocacy conference and day except it's bigger, and, well... they're Realtors. This year they'll be hungry Realtors.

In the past ALS advocates have sometimes hit Capitol Hill on the same day as the Realtors. Those highly tagged, badged, and pinned brokers and salespeople are not a shy lot. They are there in droves. They fill up elevators and legislators' offices. They know how to make sales, and they seem to be driven to get to legislators at all costs. They glance at ALS advocates' nametags, but the cause is hard to read. They see people in wheelchairs and think about that accessible property they have back in Anytown, USA.

You may wonder why in the world Realtors want (and seem to get) so much face time with our legislators. You may wonder why Realtors approach legislators so aggressively and relentlessly. What could they possibly have to talk about? We have a matter of life and death to discuss. What drives relentless Realtors to Washington?

Take a look at the stimulus package. That answers the questions. There are some incredible incentives to get the housing market moving and to sell that accessible property in Anytown. Cha-ching.

The people in the wheelchairs at the backs of the elevators need to be heard, too. The competition is stiff. The Realtors got what they wanted.

Change We Need

There is a very telling statement in this ALSA document --
http://capwiz.com/alsa/attachments/ALS_Association_NIH_Transition_Statement_FINAL_Attachement_.pdf

"Over the past six years, funding for the NIH has been flat and, in recent years, funding for ALS research through the NIH has decreased."

So we're not even been treading water relative to NIH priorities?