August 2018

Radicava is the big news in the ALS community this year. Approved by the FDA in August, I began my infusions in September. I have continued to have them regularly since then. My FVC (Forced Vital Capacity-breathing) improved from 28% to 40%. I realize there are no good biomarkers in ALS progression but FVC is an empirical measurement and I have held steady at 40% for 11 months. We do not know if this is a direct result of the Radicava or perhaps by other drugs or even something else entirely. I am grateful, and I still use the Trilogy ventilator frequently.

My hands continue to deteriorate. Typing, what was one of the a few things I could do, has become especially difficult. Even using the controls on my power wheelchair has become a challenge. I no longer go swimming, and I miss it. Eating is an effort, but I can still both speak and swallow. I have found softer foods are easier for me, and many of the foods I enjoy are no longer possible. This is yet another loss to ALS. I had a bout with aspiration pneumonia in July. The intense difficulty in breathing was frightening and enlightening of the path I am on and future difficulties I will face. I managed to stay out of the hospital and have fully recovered. I have learned to be more careful when swallowing.

The ALS monster is relentless. Simple tasks such a typing an email reply are most difficult, sometimes impossible, time consuming and exhausting. If my hand drops off my lap. I cannot pick it back up. I cannot scratch my ear; it is so frustrating! I am constantly just out of energy. The good news is I have survived over 6 years with this terminal disease. The bad news is I am about to start my 7th year with this terminal disease.

My friends here help me in so many ways. They take me to many of my daily infusion appointments and to fun things outside of the house. Their unconditional love and support are extraordinary gifts…to me and to Brian.

Hurricane Irma invaded Southwest Florida in September. We were forced to evacuate. This is no small task for those of us with ALS with all the support equipment required. We made the 20- hour drive to and from Maryland with the insistence and help of our children. We stayed at Elizabeth’s and were able to spend time with the grandchildren. Time with family was the only good part of the whole event.

With Stephen Finger and Stephen Winthrop at the ALS Community Workshop before the FDA

I continue with my advocacy work. For the fifth year in a row I spoke for the Russell Sage interdisciplinary ALS conference. This year there were 400 students from several health disciplines in attendance. I have also participated on the ALSA committee to develop a survey to measure the burden of ALS patients and caregivers. This will be presented to the FDA as a Voices from the Patients report.

For two years I have been a member of the committee to develop a Guidance document regarding ALS drug development for the FDA and industry. I was invited to speak before the FDA in DC in July. Our goal is to help streamline clinical trial processes for FDA approval. ALS has so varied a clinical presentation, trajectory, and no widely accepted objective clinical biomarkers. This makes the reliance on Randomized Clinical Trials counterproductive in the development of potential drug therapies. We are patients with very limited time and no treatment options to stop the progression of the horrible disease. We, ALS patients, are willing to accept more risk! We want to move the ball forward, to advance the process of developing treatment. We must do better to accommodate future patients who will have so few options and so little time to wait.

Our third granddaughter, Josephine Ruth, is due in September. What a joy my entire family is to me! Life is difficult but I continue to focus on the blessings I have.

Visit us at this us at this year’s research donation site: Over the past 5 years we have proudly raised over $200,000 for ALS research.

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© Madeline Kennedy 2018