Key Collaborators In Amyotrophic Lateral Sclerosis Dose Escalation Study

Armen Hareyan's picture

Amyotrophic Lateral Sclerosis

Avicena Group announced the key collaborators in the recently initiated dose escalation trial to evaluate the Company's ALS-02 drug candidate in subjects with Amyotrophic Lateral Sclerosis.

ALS-02 has shown neuroprotective effects that may potentially slow the progression of this neurodegenerative disease.

The dose-escalation study is being led by principal investigator Allitia DiBernardo, M.D., at the Neurology Clinical Trials Unit (NCTU) at Massachusetts General Hospital (MGH). In addition to MGH, the trial is being supported with funds donated by the Harvard Center for Neurodegeneration and Repair, and the ALS Therapy Alliance. Avicena, as part of its ongoing collaboration with MGH and both of these foundations, will supply ALS-02 for evaluation in the trial.


"MGH continues to be an important collaborator for us," stated Belinda Tsao-Nivaggioli, Ph.D., CEO of Avicena. "We look forward to strengthening our partnership with MGH and the foundations which have generously provided the funding for this trial."

"As we mentioned last week, the purpose of this study is to identify the optimal dose of ALS-02 for use in later-stage clinical trials. MGH is expected to enroll six patients with sporadic ALS to establish the serum pharmacokinetics (PK) of ALS-02 at three doses. By mid-2007, we anticipate results from this trial that could uncover the optimal dose for further evaluation."

This clinical study is an open-label, single-center, dose escalation study designed to establish the serum pharmacokinetics (PK) of ALS-02 at three doses (5 gm twice-daily ("BID"), 10 gm BID, and 15 gm BID) and to determine the optimal dose for later stage clinical studies. Principal Investigator Allitia DiBernardo, M.D., will lead the trial at the Neurology Clinical Trails Unit (NCTU) at Massachusetts General Hospital.

In addition to establishing an optimal dose for further study, the trial will also evaluate brain concentration levels of ALS-02 pre- and post- treatment via MR spectra (MRS) measurement. During the seven-week study, patients with sporadic ALS will receive escalating doses of ALS-02 for three weeks. PK analysis will be performed at the end of each week and MRS imaging will be conducted throughout the study to measure changes in brain metabolite levels.

ALS is a neurodegenerative disease that attacks the motor neurons of the brain and spinal cord that are responsible for voluntary muscle movement. As these motor neurons degenerate, their ability to send impulses to the muscle fibers is compromised. Once a patient's muscles no longer receive the messages that they require to function, muscles begin to atrophy. With an incidence rate of approximately 1 in 10,000, ALS affects roughly 30,000 Americans at any given time. Each year, approximately 5,600 new cases of ALS are diagnosed. The two types of ALS are "sporadic ALS," which accounts for approximately 85% - 90% of all cases; and "familial ALS," which accounts for the remaining 10% - 15% of patients.