The faces of ALS

I found this video on YouTube and was moved. There are so many suffering from this terrible disease and not enough awareness. Please take a moment to watch. It's sad, but we need to be motivated by these stories and honor their lives or memories by continuing the fight to find a cure.
(If you can't read the script on the video because of the advertisements -- the link is here.)

Day One

My daughter Lindsay and I went to the ALS Association Los Angeles Chapter office in Agoura Hills today armed with questions and filled with concern. My 39 year-old niece, Kairit Krikk, the youngest of my half-sister’s three daughters in Estonia, was diagnosed with Lou Gehrig’s disease a few months ago. Just over a year ago, the muscles in her legs began twitching involuntarily as though the brain was not getting the message that the muscles had done their work and now could rest. Her doctor in the capital city of Tallinn told her she was just nervous.

“Is there something in your life causing you anxiety, my dear?” asks the doctor. She prescribed Xanax.

When the feeling of needles and pins began in her lower body, Kairit was sent to Tartu University Hospital in the heart of Estonia, a facility similar to a teaching hospital/university in the USA but without the resources. Estonia is the smallest of the three Baltic States, about one-quarter the size of Texas, with a population of less than one and a half million people.

After a battery of tests, including two painful muscle biopsies after the lab determined that not enough tissue had been taken the first time, doctors at Tartu pondered for a while as though they could not believe the results. They determined that Kairit has the motor neuron disease called Amyotrophic Lateral Sclerosis, commonly known as Lou Gehrig’s disease.

The younger generation may not be familiar with Lou Gehrig. He was a famous Yankee baseball player who at age 36 was diagnosed with the disease in which the motor function of the central nervous system shuts down, causing muscle wasting and paralysis while the mind stays alert. Patients usually die within three to five years. The vibrant, healthy-looking young man bore his cruel fate with such grace. You can watch Gehrig’s farewell speech from July 4, 1939 below.

Kairit is also full of grace, a gentle, creative soul…an artist who painted and illustrated cards and children’s books but has now lost the ability to do so. Her ability to walk is nearly gone and her hands no longer work. She survives on a small government stipend for disabled persons. In November, her sister Hurmi sent out an email to family and friends explaining Kairit’s debilitating condition asking for donations for Kairit’s care and angels to hear the prayers of Kairit’s 19-year-old son, Kent, her family and her friends.

Her photo is above.

There are absolutely no support services in the small rural area where Kairit lives nor anywhere in Estonia. We have a standard of care in California but there are no therapeutic treatments other than the drug, Rilutek. It is very expensive, has side effects, and only extends life for 2-3 months. There are clinical trials going on but nothing of consequence has yet been discovered.

However, according to what I’ve read on the internet and from emails I’ve received from research scientists I have reached out to, there is lots of data available. The key to unlocking the mystery of ALS may be in having these best and brightest of minds together for a duration of time to look at, decipher, and brainstorm ideas.

Kairit has decided to try stem cell treatment in Germany at the X-Cell Center. I am going to meet her and her longtime partner Anto, in Duesseldorf in early March. Not enough is yet known about stem cell treatments but a clinical trial is being undertaken in the USA at Emory University Hospital in Atlanta headed by Dr. Eva Feldman from the U of Michigan. The first phase of the trial is to determine if the procedure is safe and will take two years to study before Phase Two begins.

Who has time? Not Kairit.

The X-Cell Center is doing stem cell treatment now. So, Kairit is putting on her game face. She will undergo bone marrow collection from her hip bone. The stem cells will be developed in the X-Cell lab overnight and then implanted via lumbar puncture. Cost is almost eight thousand euros. Once there, Kairit has the option to decide to try the one in which the stem cells are implanted in the occipital area closer to the brain. Right now she is not sure if she’s up to having a hole in the head…too new…only two patients thus far have tried it with no results to report thus far.

“No ALS patient we know of has been cured or benefitted significantly from any stem cell treatment to date," Fred Fisher, President of the Los Angeles Chapter of the ALS Association told us today. “However, stem cells may very well hold promise in the future.”

This, of course, was not what I wanted to hear. Fred Fisher is proud of the achievements thus far by the organization he heads and pointed out a number of legislative and standard of care successes, particularly in California. In late June they are planning a two-day symposium in Northern California to bring together researchers to discuss data and clinical trials going on. This is where my interest lies.

Dr. Marie Csete, an ALS researcher, to whom I was introduced via email and telephone by a mutual doctor friend, mused to me one day in conversation…"It would be great if we could have all these best and brightest in a setting together for several months to really devour all of the findings to date."

When I asked how much money that would take, she answered, “Oh, about twenty-five million would be good seed money.”

That’s what brought Lindsay and me to Agoura Hills today. I may be naïve but I think we need to try and raise the money. There’s hope in that scenario where scientists can lock horns and throw ideas around. They can discuss the results of what has been tried thus far. If they just focus on this one disease, and not have to look at other diseases at the same time in order to make a living, then maybe someone or some small group of doctors will have a lightbulb moment - that “AHA! I think we need to try this. This might work.”

I want a cure but I would temporarily accept any therapy that would treat ALS as a chronic disease halted in the early stages when a patient can still walk, use hands, talk, breathe and swallow.

Lance Keene, the VP of Development for ALS in Los Angeles, explained how we can set up our own family page on their existing website, which we are working on...

Our goal will be to raise $25 million, specifically to fund a significant international research symposium and the subsequent research that must be conducted following the symposium's findings and conclusions. If over 100-million people can watch a televised football game, surely we can find 2.5 million people willing to send $10 or more to help people like Kairit who right now have no hope. There are angels out there…they just need to know where they are needed!