Radicava

Yesterday, Deb added Radicava to her treatment regimen. Radicava, known as "liquid Platinum" is delivered intravenously, and it is one of two FDA-approved drugs used to treat the symptoms of ALS. One of the these treatments, Riluzole, has an oral and a suspension form of delivery. Riluzole works by decreasing the body's levels of glutamate, an amino acid that affects nerves that send messages from your brain to your muscles. People with amyotrophic lateral sclerosis may have very high levels of glutamate, which can damage these nerve cells. Radicava works by reducing the oxidative stress in the body. People with ALS have high levels of oxidative stress. Radicava is administered via intravenous infusions. Initially, patients have a daily infusion for two weeks and then have two weeks rest.

Okay, so anyone without a PhD in Biochemistry or not currently practicing medicine is wondering "What the heck is all that?". I am not going to attempt to delve into the cell chemistry for how existing medications work, because that is above my pay grade. I will say, however, that both Riluzole and Radicava can be considered first generation drugs for the treatment of ALS, and each of these has demonstrated the ability to slow disease progression by 25-50%. They are both targeting symptoms of the disease, rather than the source, and seeking to interact with some of the effects of its progression.

There are two principal reasons why we began Radicava now and not last Fall. First, suspension-delivered drugs usually remove a patient from consideration for trials and are a major hit to quality of life due to their administration; and most of the interesting trials currently in the pipeline are somewhat more sophisticated drugs that are targeting genetic solutions, whereby "switches" once identified can be turned off in a patient. These are still in Phase II and III (efficacy/dosage), but they are one of the strong benefits of working with a research center like MGH/Healey. Based on the forward calendar, we anticipate being able to qualify for such trials in the coming few quarters, and Radicava would no longer disqualify Deb. Yes, a bit of jumbled logic to balance, but we work closely on this stuff. The second issue is approval due to cost (back to my reference to Platinum). As Deb has managed so much of the family's medical care over the years, she pushed and pushed to get this thru the red tape at Cigna.

Some of you may also know that Deb takes Delsym, an over-the-counter cough suppressant that contains dextromethorphan polistirex. One of the interesting things, for those who have followed the "off label" discussions on Covid treatments, is that Delsym can be taken at a pretty high dosing level and has some beneficial effects on speech. Deb has been using Delsym since late in 2019, and it seems to have been having a positive effect. We are always interested in two-packs of Delsym which are basically half-price at Costco! If you go to Costco during these times, let us know! We buy a lot and are happy to join your cart and pay you back.

One of the things that cannot be denied in all of the discussion on drugs is that none of them can arrive fast enough for patients with ALS. Deb has eagerly raised her hand to participate in trials, something we learned is actually rare among patients; but that is, in Las Vegas terms, a bit of a "crapshoot", since efficacy has not been fully proven until FDA-approved. There are many innovations taking place at Healey, one being the "ALS Platform Trial" which groups participants into a collection of drugs and provides for "open label" administration (placebo groups get to take the drug following the first segment) and more rapid cycling across a series of treatments. Because this platform is competitive, the research team believes the patients are getting closer to helpful therapies faster. We are waiting to hear if we win the platform lottery. It is competitive. Very competitive.

So much of this is unknowable, and we just need to put our heads down and plow forward. For now, Radicava will be a 14-day "on" and 14-day "off" process, and we are getting more proficient connecting IV's and maintaining a clean home environment. Deb received an in-home placement of a midline yesterday, a catheter that runs inside her vein from the crook of her arm up and just past her shoulder. Hopefully, the treatment of Covid will be in such a place that such an impermanent delivery can be replaced by a hospital-placed "port" that is better suited to long-term administration of Radicava.

I hope this update was helpful. We are so excited to be trying it, because these discrete projects keep us focused on beating the invisible enemy. We are also hopeful to be released from the house, and we love hearing from you, especially scheduling short Zoom calls that fill our home with laughter and the sounds of voices of those we love. If you have not written to Deb to schedule a Zoom call, please do it soon. They are very therapeutic.