Later this month, Jennifer and I will travel back to Texas Neurology in Dallas, Texas, for our final appointment of the Healey ALS Platform Trial. This visit will be a little bittersweet, however, as we were notified on Monday that the trial drug that I have been on for the last five months (Regimen A) has been suspended.
From the press release issued by Massachusetts General Hospital:
The decision to stop the zilucoplan regimen early was based on the recommendation of the DSMB following pre-specified criteria for early stopping that are evaluated as part of regular interim analyses. At the most recent interim analysis, zilucoplan was found to have a low probability of meaningfully slowing disease progression
“The platform trial includes early stopping rules and other adaptive features to accelerate the testing of investigational products for ALS,” says Merit Cudkowicz, MD, MSc, principal investigator and sponsor of the HEALEY ALS Platform Trial, director of the Sean M. Healey & AMG Center for ALS, chief of the Department of Neurology at MGH, and the Julieanne Dorn Professor of Neurology at Harvard Medical School. “While we are disappointed by the results of the zilucoplan regimen, we know it is important to have early read-outs when results are clear so that efforts can be quickly refocused on other investigational products. We have to move forward to find new treatments as quickly as possible. We are extremely grateful to the many participants in the trial and the members of our patient advisory group for their contributions to ALS science and therapy development.”
While this news is disappointing for sure, we went into this medical trial knowing that there could’ve been several outcomes, including: being randomly assigned to the placebo group with no medical benefit for me; being randomly assigned to the active drug study group with some medical benefit for me; or being part of the active drug group but still having no medical benefit for me. That being said, from day one we went into this project knowing that there could be no medical benefit for me but exceptional medical benefit for the entire ALS community.
When a drug is shown not to be beneficial, one can claim somewhat of a success since that failure allows science to eliminate one drug and move to the next.
So, what does this mean for me moving forward? First, I will no longer be starting my day with a shot to the belly! My final shot was administered on Monday, exactly twenty weeks into the twenty-four weeks I committed to. Halting the injections also means that we now have the option to explore participation in Regimen E of the HEALEY ALS Platform Trial or numerous other trials currently enrolling new participants. And if it turns out that I don’t qualify for any additional trials, I will be able to explore access to promising trial drugs—like AMX0035—through an Early Access Program (EAP).
Our final appointment at the end of this month with Texas Neurology will essentially be my exit interview. I cannot emphasize enough how wonderful the doctors, nurses, and staff of both Texas Neurology and the HEALEY ALS Platform Trial have been to both Jennifer and me.
AND, as you know, when one door closes another door opens.
The same day we received notice that Regimen A would be ended early, we received news that we have been accepted as a new patient for another exceptional neurologist, Dr. Bedlack who practices at Duke University. Dr. Bedlack is renowned for a non-traditional approach to tackling ALS. Not only does he use the tools available to other neurologists to combat ALS, but he also uses alternative methods, supplements, and off label applications of drugs that have shown positive outcomes for ALS patients. Getting an appointment with Dr. Bedlack is like getting a reservation at the best and newest restaurant in town. To say that he is a rock star in the ALS world is an understatement.
While in Texas at the end of this month, we also hope to reconnect with some old friends from my old Texas days, and enjoy spending time with Jennifer’s brother and his family.
Other important updates:
- The leg spasms which I noted in my last post have subsided somewhat thanks to a new prescription from my Hawai’i neurologist Dr. Vivian Chin. After about three weeks on the new drug, the horrendous night time leg spasm episodes have been reduced by about 90%, which has meant that both Jen and I have enjoyed better sleep for the first time in months.
- My custom built power wheelchair is in the process of being designed and built, and hopefully will be delivered in the next few months.
- We also purchased a light weight electric wheelchair that we can fold and place the trunk of the car allowing us additional flexibility to be out and about without depending on my walker, which I really can only use for very short distances.
- Our building board of directors approved our plans to renovate our bathroom to provide for a walk-in shower, and additional safety items to extend my independence and ensure that I don’t take a fall while in the bathroom.
- AND today we were the happy recipients of an electric hoyer lift being loaned to us by our local chapter of the ALS Association. Should I ever take another fall, Jennifer can use it to safely get me up from the floor. And over time, as my mobility lessons further, it can be used to help transfer me from bed to my wheelchair, the shower, and other furniture.
As always, thanks for reading and following along on this journey. Your well-wishes and messages of encouragement mean more than you know. While our days may not always be easy, we continue to be buoyed by your support.
Thank you so much, Jose, for your contribution to the fight against ALS; your participation in the platform trial with Zilucoplan was instrumental for society to ultimately beat ALS.
And, as you reminded us: as one door closes, another opens; onward to Dr. Bedlack, at the best restaurant in town! Years ago, I was working at Duke University (marine biology education at Duke’s marine lab in Beaufort). Terrific educators and researchers throughout the University! So, while in Durham, I hope you’ll have a chance to spend some time in coastal NC…maybe even a small plane flight over the salt marshes and barrier islands.
all the best,
So true! We only have the medicine we have today because of selfless and brave people like you, Jose. Hoping the doctor at Duke has an option that will be a difference maker for you, and others. Mahalo for keeping us involved even when the news is not what we all hoped. Much aloha, Suzanne
Your bravery and grateful attitude are inspiring. Best of luck with new regimen.
“Getting an appointment with Dr. Bedlack is like getting a reservation at the best and newest restaurant in town. To say that he is a rock star in the ALS world is an understatement.” Love the sound of this! Go Team José!
You read my mind one door closes another will open. Keep up the fight Jose and informing us on your voyage, you are breaking ground where no man has gone before ! Lots of love your brother Tony
Keep the boat ROCKING Pirate, love ya and always will, brother.
Judith K Lee
“I have not failed. I’ve just found 10,000 ways that won’t work.” Thomas Edison — You have contributed to the invention of the light bulb which will brighten the lives of thousands of ALS patients in the future.
I am inspired by your faith and optimism! I’m sorry Zilucoplan wasn’t able to help, but the fight you and Jennifer have will continue!! Congratulations on being accepted to Dr. Bedlack’s crew! You will love him! He and ALS TDI have a great partnership, especially with the ALS reversal program! I am rooting for you from the Mainland!!
A river cuts through rock not because if it’s power but because of its persistence.
Would love to see you both when you are in Dallas this month. Let me know when and see if we can work something out.
Jose, a Warrior. I can only hope to be as brave and relentless as you; thank you for sharing your courage. It is an inspiration. My prayers and thoughts are with you, your family and your doctors. Thank you for the light you and your family emanate. You are a victor.
As we enter this Lenten season which is a time for reflection, positive change, healing, and increased prayers, I ask all believers to join Jose and Jennifer’s courageous battle to fight and defeat this challenging disease. It is amazing how your story has touched and changed the lives of those on your “team”. It certainly has for me and I am so grateful and proud that our paths have crossed. As cancer survivors for over 10 years now, both my wife and I can attest to the power of prayer. May Jesus’ healing hands continue to guide you and your medical team. Saludos, Lou and Nicza Carrión
That’s MY BROTHER!!! 💜 So proud of him. What a frgn trooper. I love the positivity and high spirited energy both you and Jen exude! It’s contagious! Thank you!
Thanks for sharing and showing what superman’s strength and bravery looks like.
Jose, your smile is such an inspiration and shows your great positive spirit. You are definitely making a difference to the ALS community by your willingness to try new treatments. May you be blessed
with a positive outcome along the way. As always, thank you for sharing your journey.
Blessings, Jean McIntosh
Go Jose! We are all rooting for you out here in radio-land! I hope you can feel the Aloha we are all sending you!
Jose, reading your update gave me a new appreciation for the word “warrior”! Thank you for your courageous fight to help find a cure and treatment to ALS. May God continue to provide you with his blessings and grace. Pirate Strong 🙏🏼🏴☠️