BrainStorm will carry out the clinical trials, optimize and generally move towards having a commercial, FDA-approved procedure:
�� A Phase IIa clinical trial will be conducted to test the safety as well as trend towards efficacy of the NurOwn™ therapy for GDNF-producing cells procedure in two parallel human studies. The target population will be as set out as follows:
o A - small number of ALS patients in advance stage of the
disease who will undergo engraftment of GDNF-producing cells.
The cells will be injected intramuscular into one of the two
affected legs. Subjects will be monitored for adverse reactions
and effect on their motor ability.
o B - small group of PD patients receiving conventional treatment,
and/or PD patients not amenable to currently available therapies.
These individuals will be randomly placed in one of two groups.
One group will receive a placebo treatment, and the other will
undergo NurOwnTM therapy. Subjects will be monitored for
adverse reactions and the effect on their PD status, and as
surrogate assessment, imaging for brain production of dopamine.
�� Phase IIb trials designed to determine the effective treatment
quantity will involve a larger ALS and PD patient population (30 to 40
participants) aiming to show trends and enable dose assessment.
�� Phase III trials will involve a larger patient population (100 to 200
participants) and will be designed to test the efficacy of the full
NurOwn™ therapy for GDNF- and dopamine-producing cells.
BrainStorm will consider conducting clinical trials in the United States and other countries simultaneously to overcome time, budget and regulatory constraints.
Overview
