IU researcher develops new therapy for treatment of ALS

Bri Heron, technology marketing manager at Indiana University’s Innovation and Commercialization Office, contributed the following story.

Indiana University researcher Chandler Walker, Ph.D., has developed a new stem cell-based secretome therapy that influences both the central and peripheral components of Amyotrophic Lateral Sclerosis pathology. Secretome refers to the secretions of tissue or cells that can be harvested for therapeutic or other purposes.

One in 50,000 people has ALS, a rare neurodegenerative disease that primarily affects the nerve cells that control voluntary muscle movement and causes the gradual degeneration and death of upper and lower motor neurons in the central nervous system. Death of motor neurons is a hallmark of ALS, but other cells in the brain and spinal cord, as well as peripheral myelinating cells and skeletal muscle, all play a role in disease progression.

The average survival time after diagnosis of ALS is three to five years, and there is currently no cure or treatment to stop or reverse disease progression. While a few FDA-approved drugs provide marginal therapeutic benefits for patients, they primarily only target the central nervous system while the peripheral nervous system continues to degenerate.

“Getting an effective therapy to people with ALS sooner than later is our top priority,” said Walker, an associate professor at the IU School of Dentistry and adjunct associate professor of anatomy, cell biology and physiology at the IU School of Medicine. Given the rapid progression of ALS, time is of utmost concern for afflicted individuals, their families and caretakers. With the limited efficacy of current approved treatments, having a new therapy that could slow disease progression and prolong life would positively affect thousands of individuals in the United States alone.

Walker’s research, published in Neural Regeneration Research, has shown therapeutic benefits at various stages of disease progression in an ALS mouse model. Administering the treatment delayed symptom progression, reduced neuromuscular degeneration and extended survival time. Daily systemic administration of the therapy allows for targeting of both central and peripheral characteristics of disease progression. This approach sheds light on the effects of the treatment on specific types of disease pathology as well as the long-term therapeutic benefits of adipose-derived stem cell (ASC) secretome therapy.

“For neural disorders and diseases like ALS, a multi-factorial approach to therapy is the most beneficial for patients,” Walker said. As ALS negatively affects neurons in the brain and spinal cord, as well as peripheral tissues such as skeletal muscle and immune cells, targeting just one organ or set of tissues is insufficient for meaningly impacting disease progression. Walker’s ASC secretome therapy contains hundreds of growth factors and other pro-regenerative and protective components that affect the wide range of areas of the body impacted by ALS. Furthermore, neurological conditions other than ALS also involve central and peripheral pathologies. “This secretome therapy could also have a therapeutic affect for other neurodegenerative diseases such as Alzheimer’s or Parkinson’s,” he said.

Walker and his team will continue their studies on the therapeutic composition of the secretome, hoping to eventually produce a clinic-ready therapy for ALS patients, thanks to $700,000 in funding from Neurodegenerative Disease Research, a nonprofit organization supporting researchers studying disease pathology and therapeutic development in ALS.

Walker has also partnered with IU School of Medicine’s Cell and Gene Therapy Manufacturing Center to optimize and produce ASC secretome for FDA approval and a Phase I clinical trial. He is optimistic that the team will have clinical-grade ASC secretome ready for clinical application in the next six to 12 months.

Walker has worked with the Innovation and Commercialization Office for a couple of other inventions, and he participated in ICO’s IU Idea to Startup Pitch Competition last October.

“I am thankful that I was able to participate in the pitch competition as it showed me a different way of presenting my ALS research,” Walker said. “I am able to apply what I learn from ICO along with the feedback from the competition to move our secretome therapy closer to the patients that need it.”