Expanded access program for SLS-005 begins treating first ALS patient
EAP for adults ineligible for clinical trials, including SLS-005 arm of HEALEY study
An expanded access program for the experimental treatment SLS-005 has dosed its first participant, according to Seelos Therapeutics, the therapy’s developer.
The expanded access program, or EAP, is an open-label study allowing access to SLS-005 for people with amyotrophic lateral sclerosis (ALS) who are not eligible for ongoing clinical trials, including an ongoing Phase 2/3 study of this potential therapy.
Conducted in collaboration with the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital, the program expects to enroll about 70 adults who will be treated for six months. Contact information for the SLS-005 clinical program is available on a Seelos webpage.
Trehalose in SLS-005Â may help nerve cells to clear toxic proteins
“Initiating this EAP is an important event for Seelos and for patients in need of therapy who cannot participate in existing clinical trials,” Raj Mehra, PhD, chairman and CEO of Seelos, said in a company press release.
SLS-005 contains a naturally occurring sugar molecule called trehalose that is given as an infusion into the bloodstream. The therapy can enter the brain and is thought to promote autophagy, a recycling process used by cells to break down complex molecules they no longer need.
By activating this process, SLS-005 may help to clear the toxic clumps of proteins in nerve cells that are believed to help drive ALS. The therapy has been designated an orphan drug, a status that aims to incentivize the development of treatments for rare disorders, in the U.S. and the European Union.
SLS-005 is currently part of the HEALEY platform trial (NCT04297683), a clinical trial run by scientists at Massachusetts General to simultaneously test several potential ALS treatments against a shared placebo.
The trial’s SLS-005 arm (NCT05136885) has finished enrolling 160 people with ALS. Three-quarters of the participants are being treated with weekly injections of SLS-005, while the rest are getting weekly placebo injections. Its main goal is to assess whether treatment slows disease progression, as measured by changes in ALS Functional Rating Scale-Revised (ALSFRS-R) scores, and results are expected later this year.
The EAP is being conducted in parallel to the HEALEY trial, and it is funded by a grant from the National Institute of Neurological Disorders and Stroke (NINDS), given under the Accelerating Access to Critical Therapies for ALS Act (ACT for ALS).
“We are grateful for the support from the ALS community, the Healey platform team and for the funding from NINDS,” Mehra said.
Seelos is also developing SLS-005 as a potential treatment for spinocerebellar ataxia (SCA), a genetic neurological disorder. The company recently announced, in a separate press release, that it is pausing enrollment of a clinical trial testing SLS-005 in SCA.
This decision was made purely due to business considerations, according to Seelos, and will allow more resources to be focused toward developing the therapy for ALS.
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