TO: Family and friends….
It has been a month since my last note to you about Phyl/Mom. While we have been in touch with many of you and thankfully visited with quite a few family members this month, I wanted to bring everyone up to date on where we are at with regards to ALS treatments. Its complicated…..
First, there are several theories on what causes ALS. There does not seem to be one single cause for all people. Also, the disease manifests itself in different ways for different people. The biggest difference is where it shows up first. There is a type of ALS called “bulbar” which starts with the throat (swallowing) and diaphragm (breathing). This is really bad. Fortunately, this is not our story.
The other type of ALS starts in the legs and sometime hands/arms. Phyl has lost a lot of muscle in her left leg, some in her right leg and a bit in her left hand. The uneven gait this produces has also created back pain. The good news is that symptom development has been relatively slow and for most ALS patients, the rate of development seems to be pretty steady over time. Some people have a very rapid loss of muscle control. Again, this is not our story.
So….. the treatments that have been developed and are in experimental stages right now address several of the potential causes. It is likely that the causes interact with each other, and our new best friend, the head of Neurology at UMass Medical Center in Worcester, says any effective treatment is likely to be a cocktail of several drugs. So that is what we are looking at. The potential causes and treatments are:
1. Excess glutamic acid (glutamate). This is a known cause for some ALS patients. Glutamate is a normal body amino acid found in all of us. It is exceedingly high in ALS folks and possibly a cause of motor neuron cell death. The standard ALS drug, Riluzol, is known to reduce glutamate and also delay symptom development. Phyl has been taking it for about a month. She also avoids foods with glutamate (and Chinese food which still has monosodium glutamate in it even though it is no longer legal). Riluzol is very safe and seems to have no side effects.
2. Neuroinflammation – the inflammation of neuron (nerve) cells is almost always seen in ALS patients. The cause of inflamed motor neurons may be external toxins, so we are eating mostly organic, non-processed foods and Belita has taken away all of our toxic cleaning fluids etc. Inflammation can also be caused by excessive free-radicals which oxidize cell components and may cause neuron death. These can come from diet but we also produce free radicals ourselves. She is eating foods high in anti-oxidants and also hopes to start with the new FDA approved drug, Radicava in October. This new drug is a free-radical scavenger and has been shown to slow symptom development in ALS patients. The downside to this drug is that it is administered by infusion into the veins which requires a visit to a medical center – at least at first. Over time, they may give her a port which would allow us to administer the drug at home. It is administered every day for two weeks and then two weeks off… forever. This assumes of course that insurance will pay!
3. Another possible cause is poor function and death of the cellular mitochondria (produces energy in the cell). I really don’t understand the mechanism for this one but there is an experimental drug, Amylyx 35, which will be available for clinical trial in a few weeks that may help. We have a telephone conference call scheduled on Monday to be evaluated for this trial, which includes twice daily medication (a dry powder mixed in water). This one is a combination of two different drugs which I can’t pronounce. Both have been shown to be safe individually and effective in slowing down disease progress. If we are accepted into the trial (and don’t get the placebo) Phyl will be one of 132 people who will test the drugs together for the first time. It seems to work in mice! These drugs are thought to be neuro-protective but the mechanism isn’t clear. Its a gamble and a hope. The folks are UMass Medical are actually pretty excited about this one. There are other experimental drugs but we have to do them one at a time so we are betting on Amylyx 35.
4. Another potential cause is heavy metal toxicity, but this is rare. Phyl is being tested because of her use of pottery glazes. It is unlikely, but the folks at UMass Medical are being really thorough. They have done extensive blood and DNA testing (cost $9,000…. thank God for insurance). More blood tests this week as well.
5. There are other possible causes but no scientific evidence for any. There are also lots of “quacks” who promise a cure. Fortunately the ALS community is strong and communicate with each other. There are several online forums that routinely examine all claims and tend to expose the scam artists. The medical community is supportive of anything we want to try, as long as it does no harm and doesn’t cost us the house. So we continue to explore….
6. One of the other potential treatments that may become available later this year would be stem cell infusions. Our research neurologist who has seen the preliminary data on a treatment called Nurown (they create stem cells from your own bone marrow) encouraged us to get involved in the Amylyx 35 study now and not to wait for the high-tech treatment – so we are following his advice. Maybe later….
Phyl is getting her routine therapeutic care from the Lahey ALS Clinic in Burlington, MA. We really liked the staff and will continue to see them every 3-4 months. She was also examined by the head of neurology at UMass Medical, which is a treatment hospital but specializes in research. That is where we will go for clinical trials. Finally, we saw a Functional Medicine doc last week and are working on cleaning up her diet and taking a few supplements that are believed to support overall health (probiotics, digestive enzymes, fish oil, and some super vitamins). Of course we also have Belita to give us guidance on how to eat healthy and we are making changes in what and how we eat. And today we picked up our first medical marijuana treatment which may have therapeutic value for ALS but is mostly for anxiety. This may allow her to quit the Xanax and help her sleep.
Our objective is to slow down the development of the disease. It is unlikely that any of these steps will cause a reversal of symptoms (although a miracle will be accepted and appreciated). She is losing functional muscles in her legs slowly. We hope to receive a foot brace this week which will provide support for her left leg and make it easier to walk. She also has a new Physical Therapist who she likes and will work on the back pain, that we believe is caused by an uneven gait while walking. She is walking okay… better if she holds my hand. We also have a rolling walker (rotavator) for use if she knows she will be on her feat a long time. So I think we are doing all we can.
There is a lot of research going on – so with these steps and a whole lot of prayers, I remain hopeful. In the mean time, we are planning on spending two weeks on the beach in Rhode Island in September and hopefully a trip to Arizona in late October or early November. If all goes well, I’d like to get to “Saint Somewhere” (warm) in February. All of these trips will depend on the scheduling of the Radicava infusions, so who knows.
This email has been much longer than I expected but I wanted you to know the whole story. If you have questions about any of the treatments or experiments, please let me know. I’m happy to tell you what I know…. as this study has become my life. Well, that and keeping my wife busy and entertained. This weekend Stuart and Gretel visited and we went to see Helen and Marty and family in Concord. We also saw a soccer game in Somerville (Noah was great in the game of course and Colin was trouble) and had dinner and Sunday bagels with Milt and Florrie. It was the best two days she has had since the diagnosis!
That’s all for now…. love you all.