Our approach: ALPHA-602

The Unmet Need and a New Therapeutic Option in Amyotrophic Lateral Sclerosis (ALS)

Current Approaches and Therapeutic Issues

The current ALS treatment options have a modest impact on the disease, with some patients benefiting from a reduction in the rate of symptom progression. No curative therapy has yet been developed. As in the later stages of Alzheimer’s disease, treatment and care is best provided by a team of health care professionals including physicians, pharmacists, and physiotherapists, but eventually, home care and hospice nurses are required. These teams can design an individualized treatment plan and provide special equipment aimed at keeping people as mobile, comfortable, and independent as possible. Despite these professional support systems, a very significant burden of care still falls on family caregivers. In 2015, the economic burden of ALS was estimated at $279-479 M USD in the USA.*

The ACI Approach: Alpha-0600 Series

Progranulin is a natural protein that is expressed in several cell types in the central nervous system and in peripheral tissues and has a potent ability to protect neurons that are under stress. ACI is developing specific forms of progranulin in its Alpha-0600 Series of therapeutic treatments for ALS patients. Progranulin regulates cell survival and certain inflammatory processes. The protein plays a major role in regulating lysosomal function and microglial responses to disease. In pre-clinical studies, ACI has demonstrated that progranulin can correct the cellular defects caused by the altered patterns of TDP-43 and FUS proteins found in motor neurons of more than 90% of all ALS patients. ACI has been granted two EU patents relating to the treatment of neurodegenerative diseases using progranulin. Additionally, the FDA, has granted ACI Orphan Drug Designation for its progranulin product platform for the treatment of ALS.

For more information related to the clinical development for Alpha-0602 and Alpha-0603, please visit our page: Clinical Pipeline

*Rev Pharmacoecon Outcomes RES. 2015 Jun;15(3) 439-50