New ALS Treatment Shows Promising Survival Benefits

Understanding Amyotrophic Lateral Sclerosis (ALS)

Amyotrophic lateral sclerosis, commonly known as ALS or Lou Gehrig’s disease, is a progressive neurological disorder that impacts nerve cells in both the brain and spinal cord. This disease leads to muscle weakness and a loss of motor control. Initial symptoms often include muscle twitching, weakness in limbs, and slurred speech. As the condition progresses, it increasingly hampers the ability to move, speak, eat, and breathe.

CENTAUR Trial Findings

Recent results from the CENTAUR trial reveal that patients treated with the experimental drug AMX0035 experienced up to six and a half months longer survival compared to those receiving a placebo. The study, led by Dr. Sabrina Paganoni at the Healey & AMG Center for ALS at Massachusetts General Hospital, suggests that this therapy offers both functional and survival advantages for ALS patients.

Current ALS Treatment Landscape

Currently, there are only two approved treatments for ALS: riluzole and edaravone. Riluzole has been established to prolong survival, while the effects of edaravone on survival remain unclear.

Trial Design and Methodology

In the randomized CENTAUR trial, participants were assigned to receive either AMX0035 (3-g PB and 1-g TURSO per sachet) or a matching placebo in a two-to-one ratio. The treatment was administered twice daily, either orally or via a feeding tube, for a planned duration of six months. Patients who qualified for the open-label extension (OLE) had the opportunity to receive the active drug for approximately thirty months, with ninety-two percent of the study population choosing to enroll. The research team focused on assessing the long-term safety and efficacy of AMX0035 during follow-up.

Survival Analysis Results

The trial included 135 randomized patients, revealing a median overall survival of 25 months for those treated with AMX0035, in contrast to 18.5 months for the placebo group. This translates to a 44% reduced risk of death for patients receiving the treatment. Furthermore, a nearly three-year survival analysis encompassing all CENTAUR participants, regardless of continued long-term treatment, demonstrated a 6.5-month increase in median survival for those treated with AMX0035 compared to the placebo.

Conclusion

These findings underscore the potential of AMX0035 as a treatment that not only extends survival but also enhances functional abilities in patients with ALS.

Reference

Paganoni S., Hendrix S., Dickson SP., Knowlton N., Macklin EA., Berry JD., Cudkowicz M.E. Long-Term Survival of Participants in the CENTAUR Trial of Sodium Phenylbutyrate-Taurursodiol in ALS. Muscle Nerve. 2020 Oct 16. doi: 10.1002/mus.27091.