A look back to the future in ALS research
Twenty-twenty four was a busy year at NDR! We made incredible progress, but we still have far to go. We thank all who support our work, either with donations or science. We thank the staff that volunteered their time for the cause, Laura, Julie and Bill and greatly thank those that funded us and allowed us to continue the vision, a cure for ALS.
We said good bye to several members of the research team, some retired, some moved on to loftier positions, and some are still at the bench with other projects. A BIG thank-you for those that are staying on in an advisory role: Bob Naviaux and David Borchelt. Sadly, we said goodbye to some friends with ALS, especially missed is Richard Helms. Our focus was taking FC-12738 into clinical trial to treat ALS patients and it is bittersweet we didn’t get the study going in time for our friends with ALS, but they knew we would get there. We just ran out of time.
2024 highlights
Let me first say we owe a huge debt of gratitude to Anne Lindblad. She is a wealth of information on clinical trials (formerly co-founder of Emmes) and has a particular affinity for finding errors in documents, even if they span a hundred or more pages. She has been by our side every day since the start of the clinical work. We were introduced by Richard Helms prior to his diagnosis and we have equal admiration for him. The next year promises to be document-heavy as we move from normal subjects to ALS patients.
We partnered with Rho, a contract organization that puts most of the pieces together to enroll normal subjects into a trial. The slow start from contract to enrollment was attributed to logistics, government boxes to check off, but mainly a team change at Rho. We were back on track by February and the Site enrolled the first sentinel subjects to receive FC-12738 in the single ascending dose study (SAD) FC-US-001 Clinical Trials.gov.
Our FDA compliant drug was received from our manufacturer and began the FDA requirement of validation in this country. By a kittens whisker the analysis was finished by the time Subject 100-001 finished screening to enroll. Thank you to our partner for many years Allay Pharmaceuticals LLC, especially Patrick Allay LLC.
The last cohort was dosed and sent home without any issues by July. The final report is being prepared and is a priority in 2025. We learned from the study that the drug is tolerated well in people and behaves just as the pre-clinical data promised Pre-Clinical Paper! FC-12738 is absorbed and present in the serum in a dose dependent relationship. The half-life is 2 hours. The next goal is to show FC-12738 will do what we expect in diseased people and animals. That is a requirement to obtain Orphan Drug Designation. We have pieces for the acceptance but fall short of ALS patient dosing. Obtaining ODD is a 2025 goal.
A large project, you just can’t imagine, was getting a name for our drug…The Overlords of the naming process are The World Health Organization, in Geneva. In order to apply for a name you have to pick one. The name can’t be in use ANYWHERE in the Universe and some combination of letters can’t be used lest they infringe on decency in some far off land! Everything I proposed seemed to mean something offensive in Swahili! We hired Brand Institute to help and in August ‘23 they set to work. How about Helminotide…no! Nor Helminetide. We left them to their work and submitted five names and by December ‘23 one made the cut! It’s top secret until it is published in the “List 132 of proposed INN” sometime in the summer of ‘25. Shockingly, 17 months to name our baby! Stay tuned!
We teamed up with Hesperos Inc and tested FC-12738 on their Human-on-a-Chip platform. We got clinically meaningful results and hope the ODD center feels the same way. Hesperos helped us with dosage and a path forward, they truly share our vision. Stay tuned for updates in our collaboration with them in 2025. The work completed by Bob Naviaux continues to bear fruit, we hope to add his analyte profile into our ALS studies and show this may be a good diagnostic. We will let you know when he publishes the work…soon!
In other big news we are partnering with the AskHelpU Platform in an international collaboration. ALS is a worldwide, unresolved problem and this is a beneficial relationship for both organizations. We are hammering out the details and have hopes that the results of our work will be available in 6 months.
And finally, some bad news-good news! A collaborator diagnosed a horse disease in a cheetah! Unfortunately, this is a life altering disease for Nyambe. His options were amputation (both hind limbs) or euthanasia. Dr. Diane Schiereck contacted us for help after seeing the case. We recognized some key immune issues that sound a lot like what we target with FC-12738 and our work in horses. Fortunately, we had a few doses available! She rushed down on Christmas Eve and took the gift back to Panther Ridge Conservation Center in Loxahatchee Florida. He responded quickly! Some improvements in just 3 days! Nyambe is on the mend and is going to complete his recovery with some skin grafts. You can expect the link to our scientific paper/case report in 2025. I like to think this success would have brought a smile to Richard. And thank-you Nyambe!
We will post our accomplishments as they happen, we expect some big leaps forward!
Our year in numbers:
8, 840+ man hours working on ALS
$1,400,502 donated to FINDING A CURE: THANK YOU!
5 Studies, including clinical trials
New services launched
What's ahead for us:
Treating ALS patients
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