Clinical grade human astrocytes (the nervous system supporting cells) for the treatment of ALS and other neurodegenerative diseases
Received FDA clearance to commence a phase IIa clinical trial in the US according to the Investigational New Drug (IND) application that was submitted in February 2023.
A first in human phase I/IIa clinical trial was previously conducted by the company in Israel (2020), in which AstroRx® was injected into the spinal cord fluid using a standard lumbar puncture procedure
In this phase I/IIa clinical trial Kadimastem demonstrated a good safety profile and clinically meaningful decline in the disease progression during the first 3-months follow-up period.
Clinical grade pancreatic islet cells which produce and secrete insulin and glucagon in response to blood glucose levels
A unique micro-encapsulation technology, integrated within IsletRx, protects the islet cells from the host immune system, this allowing prolonged therapeutic effect overcoming a major challenge faced by the Cell Therapy industry
AstroRx® for ALS
Kadimastem Earns FDA Approval of its IND Application For its US Multi-Site Phase IIa Clinical Trial of AstroRx® to Treat ALS
AstroRx® - Functional Healthy Astrocytes to Protect ALS-Diseased Motor Neurons.
ALS is a rapidly progressive and fatal neurological disease, causing death on average within 3-5 years from time of diagnosis. The disease attacks motor neurons responsible for controlling muscles and leads to paralysis and impaired breathing and swallowing. Multiple studies have demonstrated that astrocytes play a crucial role in ALS disease progression. In ALS patients, the astrocytes malfunction and attack the neurons.
Kadimastem's innovative approach is to use fully differentiated astrocytes as a regenerative medicine therapy for ALS – AstroRx® to slow the progression of the disease and improve the patient's life quality and life expectancy. AstroRx® is administered directly into the patient’s spinal cord fluid by a standard intrathecal injection under local anesthesia. Pre-clinical studies in ALS animal models showed that AstroRx® is a safe treatment that provides systemic support to damaged motor neurons, increasing the life expectancy and motor functions.
Kadimastem has received FDA clearance to commence a phase IIa clinical trial in the US according to the Investigational New Drug (IND) application that was submitted in February 2023. The trial will test its lead neurological cell therapy product AstroRx® in repeated dosing of three-month intervals for the treatment of patients with ALS.
The planned clinical trial follows a first in human phase I/IIa clinical trial previously conducted by the company in Israel, in which AstroRx® was injected into the spinal cord fluid using a standard lumbar puncture procedure. In this phase I/IIa clinical trial Kadimastem demonstrated a good safety profile and clinically meaningful decline in the disease progression during the first 3-months follow-up period.
What makes Kadimastem unique in its approach to cell therapy?
“Most cell-based therapies are likely to be autologous, where stem cells are harvested from a specific tissue of a patient and cultured to create large quantities over many weeks and then returned to the same patient.
Moreover, the cells had been taken from a patient that had already manifested the disease. Another major hurdle of an autologous solution is the need for a lengthy procedure for each patient and the inability to create a ready-to-use product.
We are focused on our two main cell therapy products we invest all our efforts to bringing “off-the-shelf” products to the clinic in the best quality. We accumulated significant knowledge on cell growth, scalability, freezing methodologies, cell characterization, and we are open to new collaborations that can help improve and progress the regenerative medicine field.
One key issue is getting them from the bench to the bedside without compromising safety and efficacy. The successful commercialization of cellular therapeutics requires extending the shelf-life of these therapies through a developmental process called cryopreserved. We are moving fast with this process with AstroRx® and IsletRx. This development will allow banking of large quantities of cell products for broader distribution supporting the next advanced clinical trials and marketing phase.”
Dr. Michal Izrael, PhD
Clinical Trial on ALS Patients using our Innovative Cell Therapy
The Company conducted a phase 1/2a clinical trial on ALS patients
The trial was conducted at Hadassah Ein Kerem Hospital in Jerusalem a world leading center in the field of ALS
The trial included 10 ALS patients
The Company has completed treatment (cell transplantation of AstroRx®) in 10 patients of Cohort A and B
The company has received approval from the MOH of Israel to commence our phase I/IIa clinical trial in ALS patients
Results of Cohort A and interim results for the Cohort B have been published on our press release page.
6 Mo. results for the Cohort B were published in December of 2020
"The results of the clinical trial demonstrated a high safety profile of AstroRx® cells. In addition, the results showed clinical meaningful decline in disease progression as measured by ALS Functional Rating Scale-Revised (ALSFRS-R) during the first 3 months after AstroRx® intrathecal transplantation in both tested doses of AstroRx® cells. We are now moving forward with all the activities needed to test multiple injections of AstroRx cells in our next clinical trials.” Michal Izrael PhD.
The Company reports that study objectives have been achieved, as study results show safe delivery of IsletRx and demonstrate efficacy manifested by prolonged normalized blood sugar levels in treated immunocompetent diabetic mice throughout the duration of the preclinical study (3 months). Furthermore, no disease or treatment related complications were observed, and all treated animals remained healthy throughout study duration. In comparison, a control group of non-treated diabetic mice presented severe hyperglycemia, leading to the death of the non-treated mice within 40 days.
IsletRx for Diabetes
We Have Reached Successful Pre-clinical results of our cell Therapy Treatment for Insulin-Dependent Diabetes
Diabetes mellitus is a chronic disorder characterized by high blood glucose levels and glucose intolerance, due to insulin deficiency, impaired effectiveness of insulin action, or both. Diabetes is classified on the basis of cause and clinical presentation into two major types: type 1 and type 2 diabetes. In Type 1 diabetes pancreatic islets are destroyed, causing deficiency in insulin production and secretion. It currently requires daily glucose monitoring and injection of exogenous insulin. Type 2 diabetes results from insulin resistance, in which the body fails to use insulin properly.
Kadimastem is focused on the generation and manufacturing of pancreatic insulin secreting islets (IsletRx) from embryonic stem cells for the treatment of insulin dependent diabetes such as Type 1 Diabetes. This program is in pre-clinical phase and we are moving fast down the regulatory path to start human trials
Kadimastem's product IsletRx contains pancreatic islet cells which produce and secrete insulin and glucagon in response to blood glucose levels. IsletRx is produced utilizing proprietary method of the Company, which includes a unique stage for enrichment for functionally active cells using newly specific cell surface biomarkers. In addition, a unique micro-encapsulation technology, integrated within IsletRx, protects the islet cells from the host immune system, without the need for potentially toxic immunosuppressive drug treatment and allows for prolonged therapeutic effect, demonstrated in pre-clinical studies. IsletRx is intended for the treatment of Insulin Dependent Diabetes, aiming to free patients from continuous monitoring of blood sugar levels and repeated insulin injections.
The number of patients with diabetes was estimated to be over 366 million worldwide in 2011 and is projected to be more than 552 million in 2030. The American Diabetes Association (ADA) estimated the total annual costs of diabetes to be US$223.5 billion (in the US alone). The global cost is estimated to be US$465 billion annually and will grow to about US$510 billion by 2030.