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.

Thursday, February 28, 2013

Picture This

Picture a web meeting.  The people on the monitors will be a panel of people with ALS and caregivers.  The people at the table will be FDA officials.  Each FDA person has six minutes to give his or her perspectives on ALS drug development and approval to the panel of patients and caregivers.


Monday, February 25, 2013

This Is What I Had To Say At The FDA Today

Thank you for the opportunity to speak directly to the FDA today. To have so many different stakeholders in the room together hearing directly from one another is unprecedented and appreciated.

Today I will share a few observations on information gaps that I think hold us back and three simple ideas to help fill them.

Some Background

We lost Mom to ALS 16 years ago. She desperately wanted to be in a clinical trial. She had no delusions of beating the grim reaper. She simply wanted to contribute so that a young mother or father would not be slapped with a disease that wasn’t a fair fight. She managed to qualify for a trial -- no easy feat for a 78-year-old woman with bulbar-onset ALS. She was ready to start. Then there came a delay. It had to do with the drug company, the FDA, and the pudding that was being used to deliver the experimental medicine. During that protracted delay, Mom died, waiting for the pudding.

Some Observations

Every stakeholder in this room knows his or her own gig well, be it caregiver, a person w ALS, an FDA representative, a scientist, a pharmaceutical company employee, a physician, our not-for-profit staffs. Our problem is that each of us has some big information gaps between our own jobs that we know so well and the other stakeholders' jobs.

Today I heard about the wonderfulness of biomarkers, yet I envisioned a thought-cloud popping up somewhere above the FDA panel that said, "Fine. Bring us one." Today I heard about the urgency of EAPs, yet I imagined another thought-cloud appearing over a pharma person in the back of the room saying, "And why would you buy our cow when you can get your milk for free?" The thought clouds that I see may be way out of line. It’s difficult to know since there are such large information gaps between what I know well and what the FDA knows and what pharmaceutical companies know.

New drug ideas for ALS are announced with trumpets and promise. Then the mysteries begin. Clinical trial design seems to happen behind a big, heavy, black velvet curtain. We know there are things going on back there. We hear occasional noises and sense motion, but we don't know who does what. We suspect that patients and caregivers aren’t even in the cast. When we ask questions, the answer is often, "We're waiting for the FDA." I sometimes envision an impish thought-cloud that fades in right behind those words with the unspoken, "Oh, and we're trying to raise $2 million dollars."

The heavy black curtain finally opens and the trial is at last ready for enrollment. The eligibility criteria sometimes mystify. Sheaves of papers are signed. Patients work hard to fill the trial with peers because they really do understand that every day is precious. The curtain closes again. Even those who have put their own skin in the game by volunteering for the trial are sent to the audience side of the curtain. They have become "subjects."

And we wait. And when the curtain is finally raised again, we have yet to see anything worth a standing ovation.

Some Ideas

1. Make every clinical trial participant a valued project-team member who therefore has a right to know the accurate status of the project and who is carrying the ball today.

2. Give every clinical trial participant the right to sign a paper that says I allow, or better yet, I demand that my clinical trial data be available to other scientists working on ALS.

3. Have patients and caregivers "at the table" during trial designs… and “at the table” needs to mean something different than we healthies picture – a conference room with a nice table. With ALS, gravity is the enemy, travel is difficult or impossible, and technology is enabling. We need a different kind of table. People with ALS and caregivers don’t have all the answers, but they’ll ask some darned good questions. They’ll talk to you about things like –

• Can’t we cut the travel during trials themselves. After all, who isn’t within yards of a Walgreen or CVS that can take blood samples these days? And you should really ask the kids about Skype.

• OR Why isn’t there isn’t a standard-of-care issue when you decide to exclude DPS patients?

• OR How about capturing a 10-second patient video daily from an iPad on the kitchen table that will give scientists a lot of context that the FRS doesn’t capture?

• OR Every ALS trial is an efficacy trial regardless of what the phase says it is. Ask any patient. Ask any sponsor. Why can’t we be honest with each other? Can we have larger, more definitive Phase II trials? How about a special ALS phase – Phase Red for ALS?

• OR Why aren’t scientific data gathered at clinics that can serve as control data … or even historical trajectories for trial volunteers?
Some Conclusions

Stakeholders need to stop working behind their velvet curtains, only to come out at press-release time. We need to have all stakeholders at a new kind of table. Information gaps will close.

We all want good science. We want treatments quickly. We want for them to be good business for pharmaceutical companies whose investments are essential.

Today is an important day in ALS history. It needs to be more than important. Today needs to be opening day for a new way of doing business.

The old script has failed. It has been the ultimate tragedy. Let’s produce something different starting today. Let’s raise the curtains, light the lights, and perform like there’s no tomorrow!

Friday, February 22, 2013

Here Are The Basics On Monday's FDA ALS Hearing

The background --

The agenda of six-minute speakers --

The link to listen on Monday, February 25, 9am-5pm EST --

And most important of all, the link to leave your comments --!documentDetail;D=FDA-2013-N-0035-0001

You have until March 25 to leave written comments.

If you are in the DC area and can show up, you don't have to pre-register to show up on Monday as an observer.

If you can't be there, we hope you'll listen and submit your own comments online.  This is a opportunity too precious to squander!

The "Be Careful" Look Is Seared In My Mind

I once said to a gentleman with ALS, "Be careful," as we parted ways.

He gave me a look that I'll never forget.  It was a combination of messages.  The minute I saw his face, I knew that I had uttered pretty stupid words.

I wish that every person at the FDA could see that look that I saw.

At the ALS Part 15 Hearing on Monday, we hope that they at least get the message in words.

Friday, February 15, 2013

Nobody Speaks For A Patient As Well As A Patient- Chapter II

The following survey was written by people with ALS for people with ALS to assemble data for their comments to the FDA on February 25, regarding risk.

Please help it find patients who have something to say.

Nobody Speaks For A Patient As Well As A Patient

"I hope the FDA hearing overflows with ALS patients, caregivers and health professionals. I hope the system for electronic comments breaks down under the weight of comments. Because, as people living with ALS, we need to make a big noise."
Catherine Wolf, A Woman With ALS

It's Getting Curiouser

How long will it take to prove to the FDA that a biomarker works for ALS and a drug so that we can shorten the clinical trial time for that drug in ALS?

Why did I feel like Lewis Carroll when I wrote that question?

Wednesday, February 13, 2013

Every Impression Helps

Senator Murkowski of Alaska, gets that.  She wore an ALS sunflower pin and an ALS wristband to the State of the Union speech last night.

If only everyone involved with ALS grasped the importance of those impressions and conversation-starters.

Sunday, February 10, 2013

We Often Like To Blame The IRS...

...when the problem really is the tax code.

We often like to blame the FDA, when the problem really is __________, __________, and __________, not to mention __________.  (fill in the blanks)

"We have met the enemy, and he is us."

Friday, February 8, 2013

Was The Dexpramipexole Thud A Blessing In Disguise?

A month ago we heard a gigantic thud when the big Phase III clinical trial for dexpramipexole showed that it was a dud for ALS.

"The important thing we're learning about ALS is it's a spectrum of diseases," Lucie Bruijn, chief scientist at The ALS Association, said recently. "There are some who respond to treatment and some who don't; at the moment, we lump all the people together and that might be one of the reasons why the trials are failing."

Did dex fail because a subpopulation that it could have helped was not sufficiently represented in the big Phase III trial?

Today stakeholders are buzzing about finding new ways of identifying subpopulations and testing drug candidates for ALS.  It's a rather refreshing buzz and the gigantic dex disappointment has been credited as a big reason for the FDA Part 15 Hearing on ALS on February 25.

What if last month we had been told that dexpramipexole had shown even modest efficacy in that clinical trial?  There would be an ecstatic push to make it available for people with ALS, just as there was with riluzole in 1995.  There would be celebrations.  We would be breaking our arms patting ourselves on the back. Would we be challenging clinical trial design?  Would we be asking if the trial represented subpopulations sufficiently?  Would we be questioning the validity of what we do if it had generated the news we wanted to hear?


Wednesday, February 6, 2013

It's Wonderful To Live In A Country Where The Public Is Asked To Speak Up

On Monday, February 25, the FDA will hold a Part 15 Hearing dedicated to ALS.

Here's the scoop --

You may attend and make comments; however, you must submit your request to speak before the end of the day on Friday, February 8!

The more people who give the FDA their thoughts on drug development and regulation, the more likely we are to have some creative solutions.  The status quo has failed.   The more patients and caregivers who speak, the more likely they are to have influence over changing the status quo.

Please speak up, in person at the FDA in Silver Spring, MD, on February 25 if you are able.  And if you have ALS, even though they have not yet made accommodations for remote participation, the FDA needs to know that remote participation is a reasonable accommodation to let you speak up.

Finally, there is an open written comments period for the month after the hearing for all to chime in.

Please, we need to speak up.  Thanks, all.

Sunday, February 3, 2013


OJ Brigance and Steve Gleason at Super Bowl XLVII

Saturday, February 2, 2013