Researchers Announce Promising Results in Study of Regenerative Medicine-Based Treatment for Complete Cervical Spinal Cord Injury

Asterias Biotherapeutics Inc., a California-based biotechnology company focused on regenerative medicine, announced interim clinical trial data today, revealing motor improvement in patients with complete cervical spinal cord injury who were treated with a higher dose of the company’s regenerative-medicine based treatment.

Shepherd Center is one of the sites for the SCiSTAR Phase 1/2a clinical trial, and one of Shepherd’s study participants was among the cohort, or group, treated with a 10-million-cell dose of Asterias’ AST-OPC1 (oligodendrocyte progenitor cells derived from federally approved stem cell lines). Previous study participants received a 2-million-cell dose to assess safety.

While Asterias noted that it is early in the study, results show four of the five patients in the higher-dose group have gained at least one motor level of improvement within 90 days of treatment. Also, as targeted in the study, two of the five patients in this group have achieved two motor levels of improvement on at least one side of their body, the company reported.

“Shepherd Center clinicians and researchers are encouraged by these interim positive results and hopeful that this clinical trial will result in a treatment that will improve the lives of people with spinal cord injury,” said Donald P. Leslie, M.D., medical director of Shepherd Center and the principal investigator for Shepherd Center for this clinical trial.

Steve Cartt, chief executive officer of Asterias, said: “The results to date in the 10-million-cell cohort, while still early, demonstrate meaningful improvement in motor function, particularly in the use of a patient’s hands, fingers and arms, which is critically important for a patient’s quality of life and ability to function independently. We are quite encouraged by this first look at efficacy results and look forward to reporting six-month efficacy data in January 2017.

“We have also just recently been cleared to begin enrolling a new cohort and administering to these new patients a much higher dose of 20 million cells,” Cartt added. “We look forward to begin evaluating efficacy results in this higher-dose cohort in the coming months.”

Researchers are measuring patient improvements with the ISNCSCI neurological classification scale widely used to quantify functional status of patients with spinal cord injury (SCI). Previous research shows that patients with complete cervical SCI who show two motor levels of improvement on at least one side may regain the ability to perform daily activities such as feeding, dressing and bathing.

The independent Spinal Cord Outcomes Partnership Endeavor (SCOPE) recommended the SCiSTAR study’s efficacy target based on recently published data (Steeves et al., Topics in Spinal Cord Injury Rehabilitation, 2012). SCOPE is affiliated with the American Spinal Injury Association, which is headquartered at Shepherd Center.

The SCiStar study is funded in part by a $14.3 million grant from the California Institute for Regenerative Medicine (CIRM).

Edward Wirth, M.D., Ph.D., chief medical officer of Asterias, presented the study data at the 55th Annual Scientific Meeting of the International Spinal Cord Society (ISCoS) in Vienna, Austria, and included the following highlights:

Efficacy and Safety Results: Cohort 2 dosed with 10 million cells

• Five out of five patients dosed with 10 million cells have exhibited improved upper-extremity motor scores (UEMS) relative to their baseline scores.
• At Day 90 of follow up, four of four patients dosed have improved one motor level on at least one side, two of four patients have improved two motor levels on at least one side and one patient has improved two motor levels on both sides.
• The average UEMS improvement at Day 90 for the four patients that have reached this follow up was 9.5 points.
• The results to date from Cohort 2 show no serious adverse events related to AST-OPC1, the injection procedure or immunosuppression with low-dose tacrolimus. In addition, data from the study indicate that AST-OPC1 can be safely administered to patients in the subacute period after severe cervical spinal cord injury.

Efficacy and Safety Results: Cohort 1 dosed with 2 million cells

• Three out of three patients (including one from Shepherd Center) have exhibited improved upper-extremity motor scores (UEMS) relative to their baseline scores.
• The average UEMS improvement for the three patients was 5.0 points at Day 90, and they continued to improve an average of 7.0 points at one year after treatment.
• Despite the very low dose used in this safety cohort, at one year of follow up, one patient in Cohort 1 has improved one motor level on one side, and two patients have improved one motor level on both sides.
• The 12-month results from Cohort 1 show no serious adverse events related to AST-OPC1, the injection procedure or immunosuppression with low-dose tacrolimus. In addition, data from the study indicate that AST-OPC1 can be safely administered to patients in the subacute period after severe cervical spinal cord injury.

The results from Cohort 2 dosed with 10 million cells compared to those from Cohort 1 dosed with 2 million cells may also begin to support a dose response as patients dosed in the earlier safety cohort with 2 million cells saw an average upper-extremity motor score (arms, hands, fingers) increase of 5.0 at Day 90, while the 10 million cell cohort has experienced an average increase of 9.5 at Day 90, Asterias officials reported. The company expects to have data evaluating the efficacy results after implantation of 20 million AST-OPC1 cells in complete cervical spinal cord injury patients later in 2017.

For additional information on the presentation data presented, please see the ISCoS conference presentation on the Asterias website at: http://asteriasbiotherapeutics.com/wp-content/uploads/2016/09/Wirth-ISCoS-14SEP2016-talk-FINAL.pdf.

Data evaluating the efficacy results six months after implantation of 10 million AST-OPC1 cells in complete cervical spinal cord injury patients will be available in January 2017 and will focus on improvement in physical functioning of the upper extremities (fingers, hands and arms) of each treated patient using scoring on the International Standards for Neurological Classification of Spinal Cord Injury (ISNCSCI scale).

Each year in the United States, more than 17,000 people sustain a spinal cord injury. These injuries can be devastating to quality of life and the ability to function independently. Lifetime healthcare costs for people with SCI can often approach $5 million. Improvements in functional capabilities of people with spinal cord injury can result in significant improvements in daily living, less required care by an attendant and lower cost of care for people with SCI.

About the SCiStar Trial

The SCiStar trial is an open-label, single-arm trial testing three sequential escalating doses of AST-OPC1 administered at up to 20 million AST-OPC1 cells in as many as 35 patients with sub-acute, C-5 to C-7, motor complete (AIS-A or AIS-B) cervical SCI. AST-OPC1 is administered 14 to 30 days post-injury. Patients are being followed by neurological exams and imaging methods to assess the safety and activity of the product.

Additional information on the Phase 1/2a study, including trial sites and eligibility criteria, can be found at www.clinicaltrials.gov, using Identifier NCT02302157, and at the SCiStar study website (www.scistarstudy.com).

All clinical trial participants at Shepherd Center must be patients who are admitted to Shepherd Center for rehabilitation. Also, clinical trial eligibility requirements apply. Medical professionals are invited to promptly refer patients for assessment to determine whether they are appropriate for admission to Shepherd Center. Contact Shepherd Center Admissions at 800-SHEPHERD (800-743-7437) or admissions@shepherd.org.

For more information about Asterias, visit www.asteriasbiotherapeutics.com.