China's Hopstem Receives Approval for Clinical Trials of Stem Cell Therapy for Stroke

The Chinese biotechnology company Hopstem Biotech has received approval to begin clinical trials in the US for a stem cell-based therapy to treat ischemic stroke. The green light comes following negotiations with the US Food and Drug Administration (FDA). This was reported by Reuters.

The company’s therapy, codenamed hNPC01, is designed to treat motor impairments following a stroke. Its action is based on stimulating the release of factors that support the brain’s natural recovery mechanisms, including the replacement of damaged neurons.

Hopstem has already completed Phase 1 trials in China with encouraging results. The company reports that they have reached an agreement with the FDA for a shorter clinical development path in the US.

Consequently, in December, the FDA granted hNPC01 Fast Track designation as a therapy with the potential to transform the treatment of ischemic stroke. Currently, the only approved therapies are thrombolytic drugs. These are administered within the first few hours after the onset of symptoms, with the goal of dissolving blood clots that block blood flow to the affected parts of the brain.

Clinical data indicates that approximately 50% to 70% of stroke patients suffer from varying degrees of motor dysfunction, with only 5% of them achieving full recovery.

While the potential of stem cells for stroke treatment has been discussed for years, Hopstem notes that hNPC01 is currently the most advanced therapy in clinical development. The company adds that the agreement reached with the FDA will significantly shorten the time to market for the product.

Phase 1 of the trial involved 23 patients who had experienced a stroke between six months and five years prior to their inclusion in the study. The results show that over an 18-month period, the stem cell treatment, implanted into the brain, led to sustained improvement in motor function. In patients with a two-year follow-up, the effect remained stable with no decline in efficacy. No signs of toxicity were detected, nor was there any evidence of tumor formation, which is one of the primary concerns with stem cell therapies.

According to Hopstem, the FDA will allow the company to bypass Phase 1 within the country and proceed directly to Phase 2 with an adaptive study design.

The company’s Chief Medical Officer, Dr. Shuning Zhang, commented that the FDA’s approval of the core elements of the protocol—including the patient population, dose selection, clinical endpoints, and statistical plan—without raising safety concerns, has removed key obstacles to the effective conduct of subsequent international studies.

Among the companies working on stem cell therapies for ischemic stroke is SanBio. Meanwhile, US-based Athersys and Stemedica Cell Technologies, which embarked on this path, have faced financial difficulties, leading to delays and even the suspension of their developments in the field.