Kadimastem Plans Phase 1/2a Clinical Trial of Cell Therapy for ALS

The Israeli biotechnology company Kadimastem is planning a Phase 1/2a clinical trial of a cell therapy for ALS after regulators gave it the go-ahead.

Kadimastem’s therapy, AstroRx, consists of cells called astrocytes that are injected into cerebrospinal fluid. The star-shaped cells, which surround nerve cells in the brain and spinal cord, are derived from human stem cells that produce neurotrophic factors. These are small molecules that support the growth, survival, and differentiation of developing and mature nerve cells.

Studies in animals showed that AstroRx can delay the start of ALS, maintain muscle function and increase survival. The research also demonstrated that the therapy was safe, displaying no signs of toxicity or a tendency to generate tumors.

The trial (NCT03482050) will cover 21 ALS patients. The Hadassah Ein-Kerem Medical Center’s Department of Neurology will conduct it. Recruitment will begin soon, Kadimastem said.

Participants will receive a single administration of AstroRx. It will be delivered in escalating doses — low, medium and high — or two consecutive medium doses, with time between each.

The 11-month trial’s primary objective will be to see how safe the therapy is, and how well patients can tolerate it.

Researchers will also assess its effectiveness. Measures they will use include changes in patients’ scores on the ALS functional rating scale and changes in their respiratory muscle strength, hand grip strength and limb muscle strength. Another measure will be changes in patients’ quality of life.

“We are proud to announce the start of this vision turning into reality, with the receipt of the Ministry of Health’s approval for commencing our clinical trial in ALS,” Yossi Ben-Yossef, the CEO of Kadimastem, said in a press release.

“The commencement of the clinical trial is the most significant event since the founding of the company,” he said. “It is a great achievement for us, both in the company’s reaching the clinical stage, as well as in the successful development of a revolutionary, groundbreaking treatment.” The treatment is based “on a unique approach in the cell-based medicine field, which demonstrated excellent preliminary results, attesting to the treatment’s efficacy and safety,” he added.

Michel Revel, the company’s chief scientist, said “we see in this cell-based product high hopes for effective treatment of ALS, which damages motor neurons [movement nerve cells]. AstroRx is made up of young astrocytes, which are manufactured by the company from pluripotent stem cells, and which have shown high efficacy in protecting the survivability and functioning of motor neurons in ALS [animal] models.”