Sunday Morning Science

It is a LAMposium tradition that on Sunday morning a brief summary of the scientific portions of LAMposium is given to the LAM patients, family, and friends. This year the challenge of translating the science into terms for the lay person fell to Dr. Greg Downey, the Basic Science Chair for The LAM Foundation International Research Conference. In his introduction, Dr. Downey commented that this year's conference included some evolving and developing areas of science, some of which had no prior exposure to LAM.

Dr. Downey divided his presentation into several categories: Signaling, Lymphatics, Hormones, Cancer and Metastasis, Origin of the LAM Cell, and Clinicals (biomarkers, trial design for rare diseases). What follows are just some of the conference highlights.

SIGNALING

Major inroads have been made into understanding how LAM and Tuberous Sclerosis cause disease. This opens the potential for finding existing drugs that might be used to treat LAM. Data from the CAST trial in Cincinnati showed that the effects of Sirolimus might be reversed once the drug was stopped. It may be that the LAM cells are still there and are not being killed.

Reports were made at the conference about several different drug categories that when added to Sirolimus might prevent this reverse and actually kill the LAM cells. Prolactin, a reproductive hormone in women might affect the progression of LAM and therefore be a potential therapeutic target.

Presentations were made on new technologies that could be applied to the study of LAM signaling pathways and the rapid screening of potential therapeutic compounds and their interactions with the LAM pathways. This could dramatically speed up the examination of drugs and their effects on LAM.

LYMPHATICS

Study of the lymphatic system really only started about 10-12 years ago. This is really the first year that scientists from this field and now looking at potential applications in the field of LAM.

Technology is now available that allows for the study of the lymphatic system in living persons. This opens up a whole new area in studying the development and progression of LAM.

HORMONES

There are almost certainly female hormones involved in the development of LAM. It is now known that there are two types of estrogen receptors that may have different effects. Medications are now being developed that could selectively inhibit one or the other of these receptors and might be of interest in the treatment of LAM.

A presentation was made that showed that estrogen might be preventing a process that would result in the death of LAM cells.

ORIGIN OF THE LAM CELL

A group from Japan has done some microscopic studies of the uterus that found found the presence of LAM cells. Additional studies looking for the same cells in the uteri of women without LAM are now underway (the negative controls).

BIOMARKERS

Results from the studies on the use of VEGF-D as a biomarker were presented which showed that a VEGF-D at a specific level along with a high resolution CT and other compatible symptoms is diagnostic for both sporadic and TSC-LAM. Laboratory certifications will be needed to minimize laboratory test variation and standardize the testing procedure.

Research continues for additional biomarkers that could be used for tracking the effects of therapies in clinical trials.

CLINICAL TRIALS FOR RARE DISEASES

Due to the small number of women with LAM, it can be very difficult to set up and conduct clinical trials. In order to speed up the ability to conduct clinical trials for LAM, alternative methods are being discussed. One is the N=1 trial in which a single patient goes on and off the trial drug several times and acts as their own control. This needs much more discussion and study to make sure that stastically powerful trials can be conducted.

Dr. Downey emphasized the need to plan, finish, and analyze clinical trials correctly. It can be difficult for those of us wanting fast results but is necessary for obtaining reliable results that will be acceptable to the scientific and clinical community.


Mary Harbaugh

SNL (Saturday Night LAM)

It's just a bunch of us gals (and a few token guys) sitting around and gabbing about how great the gala was, as well as some new shoe purchases that were made prior to the big night.

This year's gala was done a bit differently. It took on more of a casual flair. Some DDS's (delectable dining stations), no assigned seating, and Tony the tiger (he was more of a kitten) in the DJ spotlight. A few of the cougars in the audience were checking him out. (You know who you are...)

The host/keynote speaker was CNN's Senior Business Correspondent Ali Velshi, who must have been an auctioneer in a former life. He managed to motivate and connect with the audience on so many different levels. The LAM Foundation felt fortunate (no pun intended in today's economic climate) to have him here as a speaker. If you were one of the lucky ones who walked out with a signed book of Ali Velshi's, read it and learn from it. If you were one of the folks who walked out with a signed Ali Velshi bookmark, let us know where he landed. Ali said he wished more auctions worked "backwards" the way this one did and said he was inspired by everyone in the room.

A touching ceremony made for tears in both Sue Byrnes' and Aaron Dichter's eyes (and many others as well), as Aaron was honored for all of the hard work he's done for the foundation through the years. I'm pretty sure Aaron cried more than Sue, as he's a self-proclaimed "emotional wussy."

The drinks were flowing, and in a virtually unheard of event, both Dr. Frank McCormack and Tom Laurenzi put on their dancing shoes. Rumor has it, (actually there were witnesses) Dr. Greg Downey ended up making an appearance in Catherine Lawrence's lamb costume. And then it was four hooves to the dance floor. Some might say he was the black sheep of the doctors' group but the patients loved his spirit. He certainly came in like a lion, but went out like a lamb. And right about now, I should admit that I'm being fed these lines by some friends in the room. Bottom line, the dancing and chatting went on until they closed down the room.

Now that the party is over, comes the silence of the LAM's. Until tomorrow.

We look forward to seeing many more of you at LAMposium 2010!

Eden Pontz
Madeline Nolan
Barb Laurenzi
Tom Laurenzi
Tish Davey
Catherine Lawrence
Laura Lentz
Chris Lentz
Chris Hughes
Brigid Higgins
Laureen McCorkle

We Gotta Laugh! It's A Matter of Life and Breath!

For Catherine Lawrence, the Chief Laughter Officer of "Survival of the Funniest," LAM is a laughing matter. Actually, most everything is for her. Which means, there's a likely chance that if you attended her session, that you had a good laugh or two. Or three. Or more.

This crazy Canadian (and I say that with the utmost respect) believes that laughter and humor are vital survival skills to help you ride out the stresses and challenges you face dealing with LAM and life in general. I couldn't agree with her more.

She started attendees off with some big belly laughs. That is, she had everyone work to laugh hard from down low. It's amazing how infectious laughter really is. What may have started off as work likely soon came quite naturally. Did you know that one hundred deep belly laughs is about the same as working out for ten minutes on the rowing machine? And, I'm pretty sure your face looks a heck of a lot better during laughing than rowing!

Catherine wants to make sure that we always go to the place of "a ha" moments, or rather, "ha ha" moments because, she says, laughter is the currency of hope. (Speaking of laughter and currency, she's printed up some fantastic aprons and t-shirts with the slogan, "Nice Pair" right underneath a picture of two strategically placed lungs. They're reasonably priced and guaranteed to get you some extra attention.)

Painting LAM in the most positive way, whether it's using that three-letter acronym to remember that "Laughter Always Matters" or "Laughter Attracts Miracles", or whatever your brain conjures up, she wants us to take responsibility for and improve, our laugh life.

For anyone who dared not laugh in the early part of the session, she pulled out all the stops towards the end, re-emerging dressed up in a full lamb costume. Actually, it was a lamb costume with a red super-cape. Soon afterwards, all in attendance were up from their chairs and flying as they recited words from an Alicia Keys song, "Oh yes, I am Superwoman." Even the men there were superwomen for a shining moment.

And as she took questions, she was always ready with a comeback. Witness this exchange:
Attendee: "Well, aside from your bunny costume, I did want to ask..."
Catherine: "Oh, you think this is a bunny suit? No problem, after all, it is the Easter season, and the suit was on sale. And, well, if I put my apron on now, I'm a Playboy Bunny."

Laugh on lady!
HAHAHAHAHAHAHAHAHAHAHAHAHAHAHAHAHAHAHAHAHA

Eden Pontz

MILES Trial

I forgot to mention yesterday that MILES Trial Coordinators from the Boston, NIH, Tyler, Cincinnati, Cleveland Denver and UCLA sites came to LAMposium to be available to answer questions from patients about the MILES Trial. It has been extremely helpful for those who are considering enrollment or wanting more information.

Mary Harbaugh

My chest hurts, can you help me?

If you are having a chest tube, thoracotomy, pleurodesis, etc. this is the seminar for you.

Dr. Hammam Akbik is the Pain Service Director at the University of Cincinnati and it is no surprise that he was voted the best pain physician in Cincinnati for 2006-2007. He spoke to us last year about Pain Management. He shows such respect for the patient and made it clear that we are never at the end of the road in pain management. There is always hope. No one should ever be allowed to live in pain for the rest of their life.

There are several possible reasons for post thoracotomy, post pleurodesis pain that becomes chronic: 1) tissue destruction from the disease, 2) trauma to the intercostal nerve which can occur if the surgeon is not careful and puts a retractor on the nerve that runs on the bottom edge of the rib where the nerve is located, 3) scar formation after surgery that pulls on the lung, 4) rib fracture either from surgery or from extreme bouts of coughing, 4) costochondrial separation (a separation in the costochondrial joint. It can also come from a large chest tube if it is put on the lower edge of the ribs or after a significant bout of pneumonia that results in scar tissue.

The pain can sometimes last for months to years.

The best treatment for pain occurs in being pre-emptive. Medication often takes hours to be fully effective and should be done well prior to the procedure so that pain does not get started in the first place.

The placement of an epidural 24 hours prior to surgery decreases the chances of severe pain 75%. Pain control can be continuous and/or under patient control. The problem with only patient control is that when you sleep, no medication is delivered. The epidural requires the placement of a catheter in the back in the epidural space around the spinal cord in an area with the margin of error being 4-6 mm. If you cannot get a pre-emptive epidural he recommends going to another hospital.

Many different drugs can be administered through the epidural so if you don't respond to one drug, it should be discarded and another tried. Every patient responds differently to the drugs.

If you are not finding relief from your pain even after leaving the hospital, there are several other possibilities. There are Intra/Sub-Pleural catheters that can be left in place for a few days to weeks. Nerve blocks and steroids are also a possibility. It might be that once the nerve regenerates, you will no longer have pain.

Another possibility is a Port-A-Cath into the epidural space that can be inserted in the office.

Local anasthetics can be delivered by a catheter into the wound for up to 6 days. You can adjust the rate of delivery and pull it out yourself when used up.

TENS units can be used to alter your perception of the pain so it is tolerable.

If all these possibilities fail a catheter can be implanted into the spine or a spinal cord stimulator which emits an electrical field can be tried.

A question was asked about bedside pleurodesis which many know can be extremely painful. This is a situation where the pre-pleurodesis should be used followed by several days of post-pleurodesis pain control.

I think most of us would agree that we would like to each have our own clone of Dr. Akbik that to help us with our pain management issues!

Mary Harbaugh

The Opportunities of Illness!

Actually despite the blog title, Tiffany Christensen, author of "Sick Girl Speaks", narrowed that concept down for us in a session she spearheaded called, "Ten Opportunities of Illness." It's a mindset that most people likely don't have--thinking of their illness as an opportunity--but perhaps it's one that we all should embrace once we're ready.

As she says, "There's no denying the impact physical disabilities can have on one's professional life, relationships and self-esteem...at the same time, there is a side to illness that we often ignore: The gifts."

She enlightened us with ten key opportunities, and had session attendees break up into smaller groups where they were asked to come up with the challenges they faced, and some solutions (big and small) to those challenges. She usually does this program during a ten-hour session, but did a condensed version for us that lost none of its "nutritional value."

Although it would have been swell to be there, even if you weren't, there are things to take away. So, here's to sharing some of the discussion highlights:

Opportunity #1: I AM NOT ILLNESS
The thrust of the thought is the challenge to connect with the separation between body, mind and spirit. What does it mean to "be sick"? Our body is sick, but there is a place within us that is never sick, never scared and never tired.
Challenges: Trying not to dwell on your symptoms
Strategies: Focus on the positive and the "well" parts of your body and mind, be thankful each day and having a spirit of giving, ask others what you can do for them instead of what they can do for you, remember life is all in the attitude.

Opportunity #2: TAKING CONTROL
If you spend enough time in healthcare settings, it's almost impossible to ignore the value of patient empowerment through patient advocacy.
Challenges: There's a lot of doctors and healthcare professionals out there to choose from, and a lot of them know very little, if anything, about LAM. There's also potentially a lot of medications to challenge us as well.
Strategies: First find a doctor who knows about the disease, or a doctor who is WILLING TO LEARN along with you. Educate yourself about the different medications out there, what each one of them does, and don't hesitate to ask a nurse or doctor to clearly explain about each one of them. (If they won't explain, ask for another nurse or doctor!)

Opportunity #3: STILL BODY, FORWARD MIND
As our bodies become less able, look for strategic ways to use your limitations as a motivation to explore the unexplored.
Challenges: Fear of trying the unknown, fear of failure
Strategies: Start off by keeping it simple, enroll in an online class, try an art--be it cross stitching or something else, keep your mind running--crosswords or soduku anyone? Keep your internal playground up and running.

Opportunity #4: SELF-HELP
Coping with pain and other physical discomforts can be overwhelming. Are there other techniques outside of Western medications that can aid in decreasing discomfort?
Challenges: Sifting the good from the bad, finding practitioners who really know what they're doing
Strategies: Look into the holistic approach, but make sure that recommendations work with what your traditional doctor has perscribed, get support from one another, pay more attention to yourself--ok maybe you can't go on a vacation to relax, but can you have a staycation in which you do something small but special for yourself? Network in your local area to find out who has gotten good responses, look into what your insurance actually covers as it may be more than you thought!

Opportunity #5: WHY ME
People facing illness often find themselves in a crisis of faith. Here's an opportunity to explore our faith deeper and more authentically.
Challenges: Defining what your faith means to you in the first place
Strategies: Accept help from others, allow others to be a part of your life experience and let them help you.

Opportunity #6: EQUAL OPPORTUNITY COMPASSION
People dealing with illness may feel depressed and cheated. When is it time to transform self-pity into compassion for the self?
Challenges: going through the different stages of grief
Strategies: Take care of yourself, remember you aren't the only one dealing with something like this-there could be somebody who's dealing with something worse, get involved, volunteers, have faith--whatever that means to you, be kind to everyone because we're all fighting some sort of battle

Opportunity #7: LOVING THROUGH ILLNESS
Difficulty can make people grow apart or grow stronger together. Despite illness, relationships remain a two-way street.
Challenges: Keeping the relationship together
Strategies: Talk about things you might not have talked about together before, listen to what the other people are saying, accept that everyone has a different role and accept them for what they are and not what you expect them to be

Opportunity #8: TRANSFORMING THE ILLNESS IDENTITY CRISIS
There is something that happenes when a person becomes sick and loses their sense of purpose. Those around the patient have difficulty knowing how to best relate to them.
Challenges: Re-discovering your sense of purpose and allowing others to connect with that evolving identity
Strategies: Get your family to accept your illness if they haven't already, realize and identify what your new sense of purpose is and allow it to come organically

Opportunity #9: SAYING GOODBYE MY WAY
There is one undeniable benefit to being sick: the opportunity to plan ahead.
Challenges: Most family and friends don't want to hear it. Some may be too young to understand it.
Strategies: Educate them, give them brochures, send them to the LAM Foundation website, tell them it's okay, and what you want to be able to do, photograph or videotape things about yourself for them to remember when you're gone, document things--write them down.

Opportunity #10: GRATITUDE WITHOUT LIMITS
After a person comes to a place of acceptance with illness, the next step is moving into gratitude--by consciously embracing the unique experiences of living on earth.
Challenges: Having to work or go about your normal life while still dealing with things, a lack of knowledge about what to do if/when you need to change the way you've been living
Strategies: Keep a gratitude journal, start by appreciating the little things, work to adapt your energy so it's used differently.

As I had a moment to chat with Tiffany after the session I pointed out how many times alone that I've made promises to myself to do the kinds of things that were highlighted during the session. She told me that her therapist once gave her a good piece of advice saying, "Write yourself a note saying REMEMBER TO REMEMBER."

Eden Pontz

Early Morning Harmony

It's always a good way to start your day in perfect harmony. I almost never start my days off like that, but today I thought I'd buck the trend by signing up for one of the Doc Talk sessions with Pat Rubadeau, PhD--because she was teaching the harmonica. Why harmonica? Plain and simple, it's good for breathing because playing it exercises the lungs. (Cue the patriotic Americana music.)

Pat blazed down the hallway, proudly wearing her pink, "Can You Say Lymphangioleiomyomatisis?" t-shirt, and apologized for showing up one minute late to the session explaining, "Sorry, I had to go to the bathroom. You can't play harmonica if you have to go." Hmmm. I excused myself made a break for the ladies room not wanting to risk what might happen if I played without the pit stop.

Back in the session, Pat said that most of us breathe quite shallowly and don't use our lungs to the full potential. She added that when we have respiratory issues, we're even more likely to breathe less effectively. But playing harmonica requires taking big, deep breaths and exhaling and inhaling with control--giving your diaphragm a good workout. And, it's definitely good for some laughs, which, coincidentally, is another way to work out your lungs, and a great way to start the day.

Pat explained the basics of our harmonicas (we'd been able to buy them from her), saying they were in the Key of C. Then she gave us some great news--reading music isn't required to play. You just need to be able to count from 1 to 10. Fantastic. Not only would I be able to try this, but when I got home, I might even convince my husband to sit around our fireplace and play harmonica cowboy to my cowgirl, while I slow-cooked some baked beans on the stove top, and my daughter made a mess roasting marshmallows in the fire, pretending it was a real campfire.

Pat usually teaches a class of about 20 students, all seniors, and she said they bitch and complain all the time over what selections they want to play, but that they teach her a lot on a regular basis. She said some days, half the class plays the harmonica upside down, and she's positive that someday they're destined to perform on a public street corner. She was less sure about a performance at Carnegie Hall. Phew, the pressure is off for us to do well. So we got started.

The trick, she said, is to blow slowly. (Hey--I know what some of you are thinking--but don't go there. It's not even lunchtime.) Try to put the harmonica as far in your mouth as you can (don't eat it!) so you get the most air going through it. There was no waiting to exhale here--we went for it. Right away, you could see whether you're breathing through your diaphragm or not, because you can feel the difference in how much air you get out if you're breathing through your chest instead. Sure, Pat said it's really hard, you may get frustrated, but one day all of the sudden you'll be able to play. (And by this, I mean play a song that someone else could recognize.) She told us to sit up on the edge of our seats and unbutton our jeans to allow for a big belly breath. (I was sure I'd be good at that, seeing that I've got the belly to work with.) She asked us how many bronchodialators we're on and added that she's just taken a "hit" right before class. As I don't use them, I'm not sure what I was missing, but Pat was smiling, so it must have been good. Pat said she used to play the French Horn, and at that time, it was all about getting it right. Not anymore--now for her it's just about having fun and getting exercise at the same time.

And then, we had our Kum ba yah moment. Literally. We played Kum ba yah. And you know what? There was a song in there. Really. I was sure that together, we could be playing the local street corner by afternoon. We moved through some old classics, "Oh My Darling Clementine", "On Top of Old Smokey", "America" and others. By the time we got to "Go Tell Aunt Rhody", I was pretty sure that if I had to "go tell Aunt Rhody the old grey goose is dead" that the goose would no longer be rolling over in its grave.

If you want to try it for yourself--I suggest you sign up at LAMposium 2010!

Eden Pontz
LAM Patient

LAMposium 2009--We've Arrvied!!

I'm always a little nervous when flying, but it has nothing to do with the airplane. It's more an underlying fear (and I realize a highly irrational frear) of whether it could lead to another pneumothorax, and the need for another chest tube. For me, having chest tubes was a more painful experience than childbirth. Oh sure, the doctors would say that's HIGHLY unlikely, but remember, along with childbirth comes that oh-so-convenient amnesia allowing us gals to be willing to give birth all over again. I'm pretty sure there's no such thing as "chest tube amnesia". I'm pleased to report all went well, and here I am at LAMposium again this year!

After a quick check-in at the front desk, I went up to get registered for LAMposium. I was met with hugs and smiles from folks that I hadn't seen in many months, and yet it was like seeing old friends. And then I was faced with my first dilemma--what color free tote bag should I chose? The pink and blue were summertime cute, for sure, but being from New York, I couldn't get past thinking about the dirt it would face once I returned home, so I went with the more practical burgundy colored bag. I'll check out the goodies inside late tonight.

I tend to be a talker (lord knows my husband would say that's an understatement), but today I tried to do some listening. So, I thought I'd share some of the things "overheard" at Friday's LAMposium. If you weren't able to come this year, let these challenge you and serve as a tease for some fun that you'll have if you attend in the future. I'll let you do the guessing as to who might have said them:

"I'm not a Lammie, I'm more of a Laminator."

"Paul Yoder--You'd make a very long, lean bookmark."

"I'm just going to put this flower in my hair tonight, and then I CAN BE a centerpiece!"

"I've booked a massage at 7:30pm. I hope we're done dinner by then because Hercules is coming to my room." (Note: Lucky for her, dinner was done early!)

"There is not a martini big enough for me right now!"

"You got a nice pair....of lungs."

"Is there a doctor in the house?"

"I got some great news tonight. My house hasn't burned down since I've been here!"

First woman: "You should really try acupuncture for that ailment."
Second woman: "How about Botox, honey?"

"I feel like I'm in a room of oxygen sisters."

"Let's challenge each other to live harder, better and with the intensity life deserves.....AMEN!"

All right, those last two quotes actually came from the evening's keynote speaker, Tiffany Christensen, author of "Sick Girl Speaks." She spoke all right, and she was sick--the cool kind of sick. Here's a woman who totally gets it. After all, she's had it, done it, been through it. But she's living life as it should be lived. If you missed her speech, you can still read her book.

Now I'm heading up to my room for a heavenly night's sleep.


Eden Pontz
LAM Patient

Dr. Frank McCormack's Talk

Dr. McCormack went over the importance of clinical trials and the specifics of the MILES Trial-much of this information can be found on the Foundation website (http://www.thelamfoundation.org/).

One important change is to how a definitive diagnosis for LAM can be made that would not require a biopsy. In addition to a CT scan consistent with LAM, a VEGF-D level greater than or equal to 800pg/ml may be acceptable. So if not wanting to go through a biopsy to become eligible for the trial has been a barrier, contact your study coordinator to see if this is a possibility.

In response to a question, Dr. McCormack also made a clarification about the $22 million dollar grant received by the University of Cincinnati. The grant was to the University for translational research not to LAM. However, LAM could benefit because treatment studies for LAM could be conducted there.

Time to go. More tomorrow.

Mary Harbaugh

Starting Out

Blogging is a brand new experience for me so bear with me. I have been asked to try and give the scientific side to the presentations.

We are just getting started with sessions and so far it has been getting to know each other, meet old friends, and make new friends. We just finished with the Roundtables on special topics-I was at the Asymptomatic/Slow Progression table. It was an opportunity to share our experiences and to know we are not alone. We could ask and answer many of the questions that if we hesitate to ask our doctors-they might look at us more than a little strangely.

After a break Dr. McCormack will be talking about the MILES Trial and clinical trials in general so later today I will share his comments.

Tomorrow, Dr. Taveira-DaSilva will be talking about the effect of exercise (our favorite!) on LAM and then I hope to attend some of the scientific sessions.

It is humbling to know that so many great minds are so devoted to finding answers and effective treatments for LAM. We inspire them and they inspire us!

More later.

Mary Harbaugh
LAM Patient
Northern Region LAM Liaison

Welcome

Hello and welcome to the LAMposium 2009 blog! We are glad you will be joining us to follow our journey through LAMposium. This blog is set up for a few bloggers to post about their experiences at the conference. Enjoy...and we hope you can make it to Cincinnati next year.