Safety results were presented on 9 patients in an ongoing PI study to evaluate the safety of CUR’s spinal cord stem cells (HSSC’s), as well as the transplantation technique, in the treatment of ALS (amyotrophic lateral sclerosis, or Lou Gehrig’s disease).
In this group of 9 male patients, ranging in age from 37-to-66 years, with ALS duration at the time of surgery ranging from 1-to-12 years, the 1st 6 were non-ambulatory with complete paralysis. Of these, 4 out of 6 experienced transient neuropathic pain in the spinal dermatomes near the injected area within 24 hours of the procedure, which resolved with no treatment. 3 out of the 6 experienced adverse events secondary to immunosuppression, which were resolved via dose adjustment in 2 cases and discontinuance in the 3rd. 1 patient experienced a pulmonary embolism 19 days post-surgery secondary to an iliac vein thrombosis, unlikely related to the cells. All adverse events were resolved. In the group of ambulatory patients, all 3 remain ambulatory and experienced no serious adverse events secondary to surgery.
- Of these 9 patients, the first 6 (Cohort A) were all non-ambulatory with permanent paralysis. The 1st of these was treated on 1/20/10. Successive surgeries have followed at the rate of 1 every 1 to 2 months. The first 3 patients (Cohort A1) were treated with 5 unilateral HSSC injections in L2-L4 lumbar segments, while the next 3 patients (Cohort A2) received 10 bilateral injections in the same region. The next 3 patients (Cohort B) received 5 unilateral injections in the L2-L4 region. Since then, 2 more ambulatory patients have been transplanted, each receiving 10 injections in the same spinal region. Their data was not included in the interim results.
The Bottom Line: The trials will proceed as it was designed for transplantation to progress up to the cervical region of spinal cord for safety in the lumbar region. This data demonstrates (only) it is feasible to transplant stem cells directly into the spinal cord of patients with ALS using the procedure and device that was developed for this trial. This Phase I safety trial was designed to enroll up to 18 patients. If approved by the FDA and the trial’s safety monitoring board (NYSEARCA:SMB), the trial will progress to cervical transplantation in Q3/11. All 9 ALS patients remain alive and that there were no unresolved serious adverse reactions related to surgery.