June 12, 2024

Courage and Connections: Dan Doctoroff and Stella Sarraf Discuss ALS, Synapses, and Spinogenix’s Innovative Approach

By: Stella Sarraf, Ph.D., CEO and founder of Spinogenix

Amyotrophic lateral sclerosis (ALS), commonly known as Lou Gehrig’s disease, was first described over 150 years ago. Despite decades of research, it remains an incurable disease with limited treatment options. ALS is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord, leading to the gradual loss of muscle control, making it increasingly difficult for affected individuals to move, speak, eat, and eventually breathe.

Thanks to advocates like Dan Doctoroff, we are finally making strides toward overcoming the barriers to ALS treatment. Dan, the founder and CEO of Target ALS, has been instrumental in advancing ALS research through collaboration, funding, and the necessary resources to accelerate progress toward clinical trials.

Hear Dan share about the mission of Target ALS and the importance of collaboration:

Dan Doctoroff became a hometown hero in New York City for his work in rebuilding after 9/11. He brings the same lessons he learned throughout his career on resilience and perseverance to Target ALS where he has pioneered a new collaborative approach to ALS research that focuses on accelerating scientific research into successful clinical trials.

My connection with Dan stems from our shared mission to bring hope to ALS patients. At Spinogenix, we are developing therapeutics that work at the synaptic level to restore functions lost to disease. While ALS is often viewed as a motor disease, my collaborators and I chose to instead look at ALS more holistically, which drew us to the conclusion that the brain is a fundamental and key factor involved in the disease. Within the brain, there are connections between neurons called synapses through which signals flow from one neuron to another. These connections underpin the fundamental basis for neural communication, enabling cognition, movement, perception, memory and other faculties of mind. When these synapses deteriorate, a broad spectrum of diseases can emerge. We hypothesized that by targeting the brain and repairing synapses, we could potentially restore lost functions and provide substantial benefits to people battling ALS, like Dan.

Listen to Stella discuss Spinogenix’s work at the synaptic level to regenerate synapses:

Stella Sarraf, Ph.D., discusses the diverse symptoms ALS and why Spinogenix is developing a platform of small molecules that work at the synaptic level to regenerate synapses and transform the lives for people battling diseases involving synapse loss and dysfunction.

Over the years, research has increasingly highlighted the importance of synapses in neurodegenerative diseases like ALS. Like Dan, I was inspired to change this paradigm due to experiencing the personal battle of my father with Parkinson’s disease, a neurodegenerative disease. My goal in founding Spinogenix was to bring patients and their families struggling with neurodegenerative diseases new hope. Having started my career at the bench as a chemist at Merck, I was determined to help develop a better solution for the treatment of these conditions with the development of a small molecule treatment. An oral pill simplifies drug administration, improves compliance, and significantly reduces costs, all while targeting synapses to make a real impact on ALS.

After years of development and with the help of incredible and talented trailblazing teams at Spinogenix and collaborators, we have developed a platform of oral, once-daily therapeutics with the remarkable ability to restore synaptic function in neurodegenerative diseases.

I am excited that we are now treating ALS patients in clinical trials with our lead therapeutic, SPG302, having recently completed a Phase 1 safety study in healthy volunteers in Australia. We also recently gained FDA acceptance of our U.S. IND for SPG302 in ALS to expand that trial to the U.S. This marks a significant milestone in the Spinogenix journey – we as a team feel honored to continue this vision and celebrate that we have reached this benchmark to expand to U.S. human trials, emphasizing that our bold approach is no longer just a science experiment.

Learn more about the progress of SPG302 in clinical trials and the hope it represents for patients:

Hear Stella Sarraf, Ph.D., share the progress of SPG302 in clinical trials and plans for U.S. expansion.

At Spinogenix, our goal is to advance therapeutics for patients where synapses are the critical focus, and where we can have the most impact. We are excited about our ongoing trials with SPG302 in ALS and are planning to expand into Alzheimer’s and schizophrenia, where synaptic dysfunction also plays a significant role.

Together with dedicated advocates like Dan Doctoroff, we are committed to making a meaningful difference in the lives of those affected by ALS and other neurodegenerative diseases.

Synapse loss in Alzheimer’s disease and how SPG302 can help

Courage and Connections: Dan Doctoroff and Stella Sarraf Discuss ALS, Synapses, and Spinogenix’s Innovative Approach

Using EEG and TMS to capture treatment-related Brain Signals in Spinogenix Drug Trials