Research & Money

Kairit' s lovely face framed by her newly cut hair lit up the computer screen via skype.  She had received the protein powders and other supplements I had sent that she credits for keeping her feeling healthy. Healthy --  but for ALS. I wish I could go to the holistic pharmacy and buy some product that would keep the progression of the muscle-wasting disease at bay or even reverse its effects. She has been taking the Imuran drug for several months now in the hope that she has not ALS but MMN, multi-focal motor neuropathy.  The Imuran thus far has only worked to weaken her immune system but the motor neurons do not have any more life than 4 months ago. If anything she is weaker. In another eight weeks, Kairit will have a follow-up with her doctor at Tartu University Hospital. Whether or not she continues the drug will then be determined.

It seems of late the ALS Association has sent several notices of progress made in identifying genes and mutations, and other findings but there has been no "AHA" moment.  As with so many diseases that still need treatment options and cures, money makes the difference in research. The cancer treatments and cures for some types of cancer have resulted from billions of dollars and many years of scientific research. ALS is underfunded because, quite frankly, fewer people get the disease...and most die rather quickly. It does not place the same strain on the healthcare system as other terminal illnesses. It only matters to the ALS patients, their loved ones, and those dedicated medical professionals and scientists who want to make a difference in caring for patients stricken with this terrible disease. Every disease is difficult but ALS seems particularly cruel  -- to keep the mind vital while the body wastes away.

If you have never experienced ALS with a loved one....or any friend or acquaintance...it is hard to imagine the depths of despair that set in when told there is no treatment and no cure. I have struggled for months now with my emotions. Kairit is too young and vital to let go. My aunt Geri, a fashionable and energetic woman in her early 60's, died just a year and half after her diagnosis. The two sides of the family are not genetically related...Kairit being the youngest daughter of my half-sister, Aino...and Geri was my mother's younger sister. I was not as connected to Geri's ill fate because I did not speak with her regularly on skype. Her smiling face did not reach across the seas...grab my heart...and cause me to pray for riches to come my way so we could put the money towards research into a cure for ALS.

My Christmas Wish

My niece Ly, Kairit's sister, sent me this poem today:

May all beings have happiness
and the causes of happiness.

May all beings have no suffering
nor the causes of suffering.

May all beings dwell in supreme bliss
free from all sorrow and suffering.

And may all being rest in great equanimity
free from all attachment, addiction, indifference and aversion.

And may all beings wake up
to recognize their true self-nature.

An emptying glass...

There have been no words for a long time.

The lack of information is not unlike the lack of treatment and no cure when one is diagnosed with ALS. You might as well disappear into a black hole. Research has yielded some genetic markers that may one day lead to a cure but Kairit's glass is now less full. It will be too late for her. 

There is no good news these days. Kairit spends her days in bed and requires help to sit up. She is dependent on her mother and her boyfriend, Anto. Despite their blowup a few months ago, Anto has hung in and has been supportive. It has not been easy.

Kairit is in a very angry stage as she realizes that she is not going to improve. Her muscles continue to deteriorate and her mood darkens each day. There is no doctor, no treatment, no hope of getting better. Whatever is killing the motor neurons in her body is also killing Kairit's spirit.  The family feels a sense of frustration and the glass that was is now almost empty. Kairit insists on staying home for as long as possible. Without Anto, this would already be impossible as he is the only one strong enough to carry her to the WC and the shower. The desire of the patient can cause stress on everybody.  But love conquers some of the difficulties.

My half-sister, Aino, is also not well. She will soon undergo tests for severe stomach pain that has worsened over the years. Estonia's health care system needs an overhaul as well. It takes too long to see a doctor...too long to get tests...too long to get results....too long....while patients suffer and people die.

I am not in a particularly good mood. I try not to let the problems of loved ones so far away and so much out of my control overtake my optimistic self, but the holiday season of 2010 is not cheerful. What we need is a miracle.

From Bad to Worse

Kairit fell yesterday on the way to the bathroom in her apartment.  Her left leg completely twisted and bent backwards.  It is fortunate that Anto was there. He called for an ambulance right away. Kairit was taken to the local hospital where an x-ray showed she had torn the ligaments and tissue in her leg up to the knee.  The doctors sent her home with a brace and told her to keep her leg elevated and still for the next week. They recommended ice packs and offered her pain medication. Kairit refused the medication because she is concerned about the interaction with the Imuran.

I only heard about this terrible turn of events this morning as I skyped with my sister-in-law Margit and then Kairit's younger sister, Hurmi.  Health care in the USA is often determined by one's insurance coverage. It is no different in Estonia where universal coverage gets you some help but private coverage may get you more.  In addition, many of the doctors are not motivated to help when care is needed. One of my nieces near Tallinn had a fever for two weeks and despite calling her doctor requesting an appointment citing her fever, stuffy nose, blocked ears and pain in her face, it wasn't until she was well in her third week of discomfort that the doctor reluctantly gave her time. He then prescribed antibiotics after which she was better in a few days. In the meantime, she had lost more than two weeks of work and suffered unnecessarily.

In Kairit's case, she is only one of three patients in Estonia (with a population under 2.5 million people) who have ever been diagnosed with ALS.  There is little knowledge among doctors there about the disease and patient care and no incentive to learn more.  Kairit's doctors at Tartu University Hospital are doing the best they can but it is also up to patients to advocate for themselves and the country's citizens to demand more.

With that in mind, I suggested that despite it being a weekend, that Hurmi try to reach Kairit's main physician at Tartu to make her aware of Kairit's accident and acute pain and her reluctance to take any pain medication until discussing it with her doctor.  ALS is not a painful disease, although horrific, but taking strong medication usually given to cancer patients and then having severe pain from an injury and the psychological curveball in trying to stay positive is almost too much for Kairit to bear.

Life has gone from living in one room....to a nightmare.

A Visit to Estonia

The past two weeks have been a reality check in Estonia. Kairit's life now takes place in one room of her comfortable apartment in the small town of Haapsalu. My visit confirmed suspicion that Kairit's optimistic nature and sense of humor belied the truth of her situation. When her boyfriend Anto is available, he carries Kairit into the shower that has been newly redone to suit a disabled person for bathing. However, one needs to be able to step up about six inches to get into the shower

room and Kairit is unable to do that.

Over the past few months, Kairit's mother, my half-sister, Aino, has taken on more and more responsibility caring for her. She can still walk a short distance to the bathroom with the help of a walker and a strong hand guiding her. Her gait, however, is less steady. Kairit is also having trouble sitting up in bed without help. She has less use of her hands and her fingers appear to be thinner and atrophied. When she's on her laptop computer, she uses her curled fingers to type on the keyboard.

To make matters worse, she and Anto have used up their mutual goodwill and their relationship is tenuous. Kairit understands that he is healthy and well and needs to move forward with his life. She does not want to be a burden to him or unkind but sometimes her anger at her situation gets the best of her. Anto has been a good help but the time is coming when her physical situation will require more trained and able caregivers. Aino will care for her daughter as long as possible with help and occasional weekend relief from another daughter who lives an hour away but works and has a young son. Kairit wants to stay home as long as possible but ALS is taking its toll physically, mentally and emotionally.

There is also still no consensus on her diagnosis. One of her doctors believes that it is yet possible that she has MMN (multifocal motor neuropathy) and has prescribed a drug called Imuran. The drug that is often used by some cancer patients is being used off-label here. She may experience nausea, dizziness, and other symptoms or she may have no reaction at all. My understanding is that if the drug helps her in a 4-5 month period, then she has the autoimmune system disease. The drug could itself cause cancer in the long term but Kairit felt it was worth trying for a few months to see if anything changed in regard to the motor neurons. In any case, neither ALS or MMN is a good diagnosis although with MMN one can live longer. In Kairit's case, does that mean a continuation of life in a room or would she be able to work, get out of her apartment, and lead a more normal existence? Or is MMN simply three different letters to a similar health issue?

Life in a room. Eat. Sleep. Watch TV. Read. Google. Email. Occasional visits from friends. Wait to deteriorate. Cry...while loved ones also cry and hearts break.

The fight continues...

Kairit and I spoke a few days ago.  She had been to see her doctors at Tartu University Hospital. .)  It seems one of her doctors is still not yet sure that Kairit has ALS. They want her to try what she calls a "chemistry" treatment called cyclophosphamide (cytoxan), a chemotherapy drug normally used for some types of cancer. It is also used in treating MMN or multifocal motor neuropathy. There are many possible side effects to the cyclophosphamide

treatment.

Kairit then sent me the following email...

"I think I'm not doing this. I'm sooo weak. I need my good blood and good bone marrow.

And I need to eat and never be sick and throw up. I really don't know- can I throw up or do I suffocate in the first 5 minutes? Experiments?

Doc say- the chemistry I need do in to the vein and that I need to do it again in 6 months.

But I have 2 months to thinking about this. Today and tomorrow I know- I'm not doing this. If I need to die, then without throwing up. :)))  And - maybe stem cells started their work. I have time for this."

In the meantime, I will continue to send her protein powders and vitamins and mineral supplements to keep her body as strong as possible. She feels well, but for the muscle weakness in her hands and legs.

I cannot advise her as to what to do. I understand the reluctance at trying such difficult treatment that can cause toxicity and so much harm to one's organs. Life is precious but if the treatment is not a cure but ravages your body while possibly giving you some more time…

What quality of life is there?  What price?  These are questions only the patient can answer. I wish my niece the gift of time lived as well as possible.

"Hurry up, hurry up!"

My heart is breaking for my half-sister, Aino Krikk. She has already lived through so much pain in her life. Her father (my father) was forced to leave his first family behind during WWII when the Soviet army marched into Estonia in September, 1944. Had he not left at the urging of his wife (Aino's mother) the entire family may have been killed instantly or perished in Siberia. Aino and her three brothers and mother were led to believe that Paul Leek had died when his documents and wedding ring were found the nest day. Years later in 1962 they learned that he had remarried and lived in Toronto, Canada. Aino felt great sadness and feelings of abandonment by her father....and life in an occupied homeland under communist rule was hard and dark. But nothing can compare to watching her youngest daughter, 39-year-old Kairit,, on death's march with a disease in which muscles waste away. It is as if one's heart is whipped every day.

I spoke with Kairit this morning. Her disposition remains stable but her body is not. She is getting weaker. She is losing her ability to walk, even awkwardly. Her hands are weaker. The February stem cell treatment gave her a shot of optimism, but it did not have all of the right ingredients. I watched an episode of Charlie Rose that dealt with inventions and science. He quoted Einstein who said something like "If you ask the right question, you have 95% of the answer. " What is the right question about ALS? What is the answer?

I told Kairit about the event held this past Saturday at the Century Plaza Hotel, a fundraiser for the ALS Association that honored some notables in the fight against ALS. They included actress and ALS celebrity spokeswoman Kate Linder, whose brother-in-law had ALS; Jim Barber, an ALS patient and attorney who advocates with great success for patient rights; Dean Rasmussen, whose father had ALS, who is a tireless advocate, generous benefactor and meaningful fundraiser, and Dr. Clive Svendsen, PH.D, who is the Director of Regenerative Medicine at Cedars Sinai Medical Center that provides ALS care and research into the cause, treatment, prevention and cure for ALS. Dr. Svendsen illustrated with the use of slides the exciting promise of stem cell research for treatment of ALS.

I shout, "Hurry up, hurry up!"

I had the good fortune to sit next to a doctor working with Cambria, a pharmaceutical company that is developing a drug for ALS. It is not a cure but one that has shown in mouse studies to prolong life longer than Rilutek, the only drug currently available. Clinical trials are next.

I shout, "Hurry up, hurry up!"

I know, of course, that science does not hurry up. Sometimes there are "aha" moments that lead to further results but it's generally a long and tedious road, especially when it involves the NIH, the FDA, or any government institution. On one hand, they protect. On the other, they hold back on the side of safety. One death in a clinical trial, even if the patient was at death's door, can set back progress by years. In a world where just about everything is rushing at us at warp speed, cures for diseases seem to be in analog.

I shout, "Hurry up, hurry up!"