Safety/Efficacy Study for the Treatment of Amyotrophic Lateral Sclerosis (ALS)

This is a Phase I, single center, prospective, non-randomized, open label, safety and efficacy study of the infusion of autologous bone marrow-derived stem cells, in six patients suffering from Amyotrophic Lateral Sclerosis according to established criteria (1), (2) with a moderate to severe diagnosis of Amyotrophic Lateral Sclerosis according to the World Federation of Neurology El Escorial criteria.

The primary purpose of this study is to evaluate the safety of the infusion procedure, as assessed by absence of complications at the infusion  site or the appearance of new neurologic deficit not attributed to the natural progression of the disease.

Secondary outcomes will include

• neurological evidence of trends toward a slowing down of the decline of the forced vital capacity (FVC) (3) and of the functional rating scale (ALS-FRS) scores, as assessed at 3-month intervals,
• patient evaluation that the treatment was effective and consider the possibility of a new cell product stem cell infusion.
• evidence of a decline of the maximum voluntary isometric contraction-arm (MVIC-arm) and MVIC-grip Z (4) scores

Subjects will undergo an aspiration of bone marrow from the iliac crest for preparation of the cellular product.

On the day of infusion, the investigational product will be injected into the patient's intrathecal space.

After cell infusion patients will be followed at WK 2, MN 1, MN 2, MN 6 and a long-term follow up at MN 12 in the clinic and/or office. Electromyographic (EMG) studies, functional rating scale (FRS), Forced vital capacity (FVC),  and maximum voluntary isometric contraction-arm (MVIC-arm) and MVIC-grip Z scores will have been used to assess the status of the disease before (historical record acceptable if done within three months of Screening) and during the 12-month study period after  the cell infusion.