Drug Candidate for ALS

Background:

Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's Disease, is a progressive neuromuscular disease for which there is no cure. The disease attacks motor neurons that run from the brain to the spinal cord and from there to the muscles throughout the body. As these motor neurons die, patients lose the ability to control their muscle movement. Eventually, they also may lose the ability to move, eat, speak and even breathe. The vast majority (~90%) of ALS cases have no family history of the disease (i.e. it is not genetic). Currently, there are a few drugs that are FDA approved to treat the disease, but these drugs are only marginally effective.

Technology Overview:

This University at Albany innovation involves the discovery of a group of RNA aptamers that inhibit AMPA-subtype glutamate ion channel receptors. AMPA receptors are expressed in the brain and are indispensable for brain development and activity necessary for such things as learning and memory. One theory for why these receptors are critical to understanding and treating ALS is that when AMPA receptors are abnormally expressed and/or malfunction, they allow calcium to flux into motor neurons, causing neurodegeneration and motor neuron death. The RNA aptamers developed here show promising effectiveness at stopping motor neuron death and even reviving dying ones.

Advantages:

This ALS drug candidate offers a unique approach to treatment that is quite different from traditional organic synthesis and offers compelling advantages such as:

• High potency rivaling existing small molecule inhibitors.
• Unprecedented conformational selectivity.
• Unmatched water solubility compared to any drug molecules or inhibitors reported to date.

Applications:

The RNA inhibitors of AMPA receptors described here are applicable to the treatment of ALS, though the platform technology could also be used to develop therapies for other neurological diseases and disorders.

Intellectual Property Summary:

U.S. patent 20190010498 for Chemically modified AMPA receptor RNA aptamers was issued on Jan 10, 2019.

Stage of Development:

Technology Readiness Level (TRL) 5: Technology validated in relevant environment (preliminary data from animal model studies show efficacy).

Licensing Status:

This technology is available for licensing. Globally, over 225,000 people live with ALS with no cure on the horizon and limited treatment options. As an ALS therapy, this technology has the potential to be licensed by pharmaceutical companies developing therapies in this space. Other pharma companies working on neurodegenerative diseases may also be interested in the platform technology to develop new therapies for diseases beyond ALS.