Kadimastem Ltd., a biotechnology firm that develops cell therapies, said it has received promising interim results from a first group of patients treated with its new therapy for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrigs disease, a fatal neurodegenerative condition that causes the loss of muscle control.
Looks like we are on the right track, said Rami Epstein, the CEO of Kadimastem, in a phone interview. The interim results are important because they demonstrate that we have managed to inject the live cells into a human body without any treatment-related significant adverse effect, while also showing a measurable therapeutic effect. Our cell therapy managed to significantly slow down the disease progression and halt deterioration of the disease.
The cell therapy aims to slow or even halt the progression of the disease and improve patients quality of life and life expectancy, he said.
Get The Start-Up Israel's Daily Start-Up by email and never miss our top storiesFree Sign Up
ALS leads to muscle weakness, loss of motor function, paralysis, breathing problems, and eventually death. The average life expectancy of ALS patients is two to five years. According to the ALS Therapy Development Institute, there are approximately 450,000 ALS patients worldwide, 30,000 of them in the US. According to the ALS Foundation for Life, the annual average healthcare costs of an ALS patient in the US are estimated at US$ 200,000. Thus, the annual healthcare costs of ALS patients in the US alone amount to $6 billion.
Reserachers at Kedimastems Good Manufacturing Practice (GMP)-compliant production facilities in Nes Ziona, Israel (Hagit Stavinsky)
In a filing to the Tel Aviv Stock Exchange last week, the firm said that the treatment administered to one group of patients in a Phase 1/2a clinical trial held in Israel apparently caused a significant slowdown in the progress of the disease and was found to be safe.
ALS is a devastating disease and there is a critical need for new treatments to more effectively slow down or halt its rapid progression, said Dr. Marc Gotkine of the Department of Neurology at Hadassah Medical Center, Jerusalem, and the principal investigator of the trial. These preliminary results on a small subset of patients are encouraging, as the treatment seems to be safe, at this lower dose.
The initial data analysis appears to demonstrate a slowing down of the reported deterioration rate over the three months following treatment, he said.
This is a positive, but preliminary results and the final results should be awaited, analysts Frost & Sullivan said in an investor note filed with the Tel Aviv Stock Exchange.
Should the final results expected at the end of 2019 for this phase 1/2a trial also prove positive, this will position Kadimastem as a potential major player in the ALS market and will have positive effect also on firms value, the analysts said. This will also serve as proof of the potential of the companys platform which can be used to treat additional indications in the future.
Prof. Michel Revel, the CSO and founder of Kadimastem (Courtesy)
The company, whose shares are traded on the Tel Aviv Stock Exchange, was founded by Prof. Michel Revel, who serves as the companys chief scientific officer. Revel is an emeritus faculty member of the Weizmann Institute of Science and a winner of the Israel Prize, the nations greatest accolade. He is also the creator of Rebif, a multiple sclerosis drug now marketed by Merckand selling for billions of euros globally.
Revel founded Kadimastem in 2009 with Yossi Ben-Yossef, based on cell therapy research Revel had conducted at the Weizmann Institute. Such research focuses on live cells transplanted into the body to replace malfunctioning, disease-causing cells.
In the cell therapy methodology developed and implemented by Kadimastem, we take embryonic stem cells, expand them into large quantities, and differentiate them into specializing cells, he said. Our cells are injected into the patient, replacing and compensating for the biological activity of the damaged cells.
A researcher working in the lab of Kadimastem, in Nes Ziona, Israel (Hagit Stavinsky)
The firm operates in two main spheres: neurological diseases, with the aim of replacing nervous system cells that have stopped functioning; and type 1 diabetes, in which cells that secrete the insulin needed to absorb sugar stop functioning. We develop cells that secrete insulin and regulate blood sugar levels, he said.
In the neurological sphere, the firm has started clinical trials, still at very early stages and on a small group of patients, to replace astrocyte cells,which are responsible for supporting neurons that enable muscle activity. In ALS patients, as a result of dysfunctional astrocytes, the neurons degenerate and muscle control deteriorates as the disease progresses.
All of the ALS patients die eventually, said Epstein, as there is no known cure for the disease. As the disease progresses, patients eventually lose control of their respiratory muscles, including the diaphragm, causing severe respiratory complications, the most common cause of death for ALS patients.
In their work at Kadimastem, the researchers differentiated embryonic stem cells into astrocytes. Scientific literature has indicated that astrocyte cells play a substantial role in supporting malfunctioning cells in ALS and other neurodegenerative diseases, said Revel in a statement issued by the firm.
Kadimastems lab in Nes Ziona, Israel (Hagit Stavinsky)
The patients had these cells injected into their spinal cord, where they secreted various factors that act as goodies, said Epstein. In animal trials, these cells created a protective layer around damaged neurons preventing further deterioration and maintaining and supporting the remaining neuron and muscle activity.
The cells were also able to secrete factors that remove toxic materials accumulated on the damaged neurons, thus acting as a garbage truck, taking toxic and damaging materials away from the neurons, Epstein said.
Now, the Phase 1/2a clinical trial conducted in humans is the first one ever, worldwide, in which live astrocyte cells, our own proprietary AstroRx cells, have been transplanted into humans, he said.
The trial, which started last year, is being conducted at the Hadassah Ein Kerem Medical Center in Jerusalem on 21 patients divided into four groups.
Each group receives our AstroRx cells, Epstein explained, saying the firm is very excited about the interim results of the first group, which got the lowest dose of the cells some 100 million cells injected into the spinal cord fluid of each patient.
The results are very significant for ALS patients and for us, he said. We hope that in the other groups we will see improved or prolonged efficacy and also safety. Kadimastem has advanced the quest to find a treatment for ALS but has also shown that the company has the ability to differentiate cells into clinical grade functional cells, fit for injection and also effective in humans.
A second cohort of patients, forming part of the same ongoing Phase 1/2a clinical trial, will be getting 250 million cells; group three will get two doses of 250 million cells each, to see if repeated treatments will yield enhanced results; and the fourth group is expected to receive a dose of 500 million AstroRx cells.
The journey ahead is still very long, Epstein emphasized, and it will be years before a drug will be made available to patients. The current Phase 1/2a trial is expected to conclude at the end of 2020, and then an additional trial will start, hopefully with a larger group of patients and in a number of medical centers.
Shares of the company have declined 2.1% in the past 12 months, bringing its market value to NIS 74 million ($21.2 million), as of end of day Wednesday.
- Naturally Treat & Heal The Cause of Pain with Regenerative Therapies - The Mountaineer - May 15th, 2021
- CAR T-Cell Therapy UCARTCS1A Shows Early Activity in Relapsed/Refractory Myeloma - OncLive - May 15th, 2021
- Rinri Therapeutics Secures Innovate UK Funding Grant for 3.2m Project to Advance its Novel Cell-Based Therapy to Restore Hearing Loss - PRNewswire - May 15th, 2021
- With $52 Million Series A, Appia Bio Anticipates a Bright Future in Cell Therapy - BioSpace - May 15th, 2021
- Genenta Phase I/II Glioblastoma Data at ASGCT Show Temferon Delivered Tumor-Focused Interferon ExpressionData presented at the 2021 American Society... - May 15th, 2021
- Safety and Efficacy of Consolidation Therapy with Ipilimumab Plus Nivolumab after Autologous Stem Cell Transplantation - DocWire News - May 15th, 2021
- Fascination with stem cell sensation leads to Fulbright scholarship - Monash University - May 15th, 2021
- Omeros' Narsoplimab Pivotal Trial Data to Be Shared as an Oral Presentation at the European Hematology Association Congress - Business Wire - May 15th, 2021
- Munshi Explains Staging, Prognosis, and Treatment for a Patient With Acute Graft-vs-Host-Disease - Targeted Oncology - May 15th, 2021
- KD-PACE Salvage Therapy for Aggressive Relapsed Refractory Multiple Myeloma, Plasma Cell Leukemia and Extramedullary Myeloma - DocWire News - May 15th, 2021
- Icy Microneedle Patch Delivers Cell Therapy, Then Melts - Freethink - May 15th, 2021
- FDA Responds to Creative Medical Technology Holdings Regarding Its ImmCelz IND for Stroke Treatment - BioSpace - May 15th, 2021
- Global Stem Cell and Platelet Rich Plasma (PRP) Alopecia Therapies Market 2021 Type and End-use Industry 2026 Orange County Hair Restoration Center,... - May 15th, 2021
- Allogeneic Mesenchymal Stem Cell Segment Is Expected To Lead In the Global Rheumatoid Arthritis Stem Cell Therapy Market over the Forecast Period,... - May 15th, 2021
- Advanced Therapy Medicinal Products Market Size Worth $21.2 Billion By 2028: Grand View Research, Inc. - PRNewswire - May 15th, 2021
- New connection between metabolism and red blood cell development - Harvard Gazette - May 15th, 2021
- Heart attack recovery aided by injecting heart muscle cells that overexpress cyclin D2 - The Mix - May 15th, 2021
- Vertex and CRISPR Therapeutics to Present New Clinical Data - GlobeNewswire - May 15th, 2021
- Rocket Pharmaceuticals Presents Positive Clinical Data from Fanconi Anemia, Leukocyte Adhesion Deficiency-I, and Pyruvate Kinase Deficiency Programs... - May 15th, 2021
- Stem Cell Therapy Global Market Report 2021: COVID-19 Growth And Change To 2030 - Yahoo Finance - May 3rd, 2021
- Stem cell therapy for COPD: Cost, effectiveness, and more - Medical News Today - May 3rd, 2021
- Thomas Smeenk on Hemostemix's autologous stem cell therapy technology and why some call it 'the fountain of youth' - InvestorIntel - May 3rd, 2021
- Mesoblast says its stem cell treatment saved lives of severely ill COVID patients - Stockhead - May 3rd, 2021
- Antiviral T cells protected, viable as off-the-shelf treatment for painful complication stem cells - Microbioz India - May 3rd, 2021
- Investigating CAR T-Cell Therapy for Use in Different Disease Types - Targeted Oncology - May 3rd, 2021
- Should scientists be allowed to grow human embryos in a dish beyond 14 days? Is it scientifically important or morally wrong? - USA TODAY - May 3rd, 2021
- Hope, hype and exploitation the wild history of stem cell science - ABC News - May 3rd, 2021
- Brave New World is being reinvented with synthetic embryosand the right reasons - SYFY WIRE - May 3rd, 2021
- Stem Cell Therapy Market Trends Evaluation 2021 By Leading Players Updates, Consumer-Demand, Key Strategies, Consumption, Industry Development, Market... - May 3rd, 2021
- STEM NOLA Teams with Obatala Science & CellSpring to Expose Students to Careers and Medical Research at Biotech Startups - PRNewswire - May 3rd, 2021
- Breakthrough gene therapies in sight | UNC-Chapel Hill - UNC Chapell Hill - May 3rd, 2021
- FDA's Abecma Approval a 'Great Blessing' for Patients with Multiple Myeloma - Curetoday.com - May 3rd, 2021
- Enhanced inhibition of tumor growth using TRAIL-overexpressing adipose-derived stem cells in combination with the chemotherapeutic agent CPT-11 in... - May 3rd, 2021
- Global Stem Cell Therapy Market 2020-2027 Trends, industry vertical, along with the geography, delivering valuable insights | Reportspedia Clark... - May 3rd, 2021
- Rheumatoid Arthritis Stem Cell Therapy Market Report 2021 by Global Key Players, Types, Applications, Countries, Size, Forecast to 2027 Clark County... - May 3rd, 2021
- CRISPRoff Reversible Epigenome Editing Method Could Enable Safer, More Precise Therapeutics - GenomeWeb - May 3rd, 2021
- Cell Therapy Market To Expand At An expeditious Growth Rate From 2021-2027 | Thermo Fisher Scientific, Nanofiber Solutions, Advanced Biomatrix,... - May 3rd, 2021
- FTC continues to crack down on companies peddling fake COVID treatments and cures - HamletHub - May 3rd, 2021
- ThermoGenesis : The History of Cell and Gene Therapy - marketscreener.com - April 18th, 2021
- Russell Health Honored in Global Business Leaders Magazine's '20 Leading Companies of the Year 2021' - PRNewswire - April 18th, 2021
- Durable B-ALL Control With Allogeneic Transplant After CAR T-Cell Therapy - Cancer Therapy Advisor - April 18th, 2021
- CRISPR lauds easy scale-out of cell therapy - BioProcess Insider - BioProcess Insider - April 18th, 2021
- NeoProgen, Inc. Receives First Granted Patent for Method to Treat Cardiac Conditions using Neonatal Heart-derived Medicinal Signaling Cells (nMSCs)... - April 18th, 2021
- CRISPR gene therapy for sickle cell disease approved by the FDA - BioNews - April 18th, 2021
- Man's heart healed by stem cell therapy and love of an old flame - Leeds Live - April 18th, 2021
- Autologous Stem Cell Based Therapies Market Professional Report 2021 Witness Robust Expansion by 2026 SoccerNurds - SoccerNurds - April 18th, 2021
- Stem Cell Therapy Market Analysis 2021: Size, Share, Sales, Growth, Revenue, Type, Application & Forecast To 2027 SoccerNurds - SoccerNurds - April 18th, 2021
- Mastitis therapy explored | AG | kmaland.com - KMAland - April 18th, 2021
- CAR T-Cell Therapy Enters the Arena in Multiple Myeloma With Ongoing Research Ahead - OncLive - April 18th, 2021
- Leukemia Cutis: Symptoms and Treatment - Healthline - April 18th, 2021
- Stem Cell Therapy Market is Set to Witness Impressive Growth | Top Players Osiris Therapeutics, Inc. (US), MEDIPOST Co., Ltd. KSU | The Sentinel... - April 18th, 2021
- Global Stem Cell Therapy Market 2020 SWOT Analysis, Growth Rate, and Market Share By Type, Application and Global Forecast 2027 - Los Hijos de la... - April 18th, 2021
- Stem Cell Therapy Market Potential Growth, Size, Share, Demand and Analysis of Key Players Research Forecasts to 2025 - The Courier - March 8th, 2021
- Animal Stem Cell Therapy Market Size, Industry Analysis, Growth Factors, Trends, and Regional Forecast to 2027 - The Courier - March 8th, 2021
- Pericardial Injection Effective, Less Invasive Way to Get Regenerative Therapies to Heart - NC State News - March 8th, 2021
- Biomarkers Help Predict the Role of Chemotherapy in Biologic Aging - OncLive - March 8th, 2021
- Glial Cell Discovery Could Point the Way to Motor Neuron Disease Therapies - Technology Networks - March 8th, 2021
- Mouth Sores from Chemo: Symptoms, Causes, and Treatments - Healthline - February 19th, 2021
- ThermoGenesis Holdings Begins a Year of Celebration of its 35th Anniversary in the Cell Banking and Cell Therapy Industry - PRNewswire - February 19th, 2021
- Meet the women hoping to recruit more stem cells donors from Black communities - CTV News - February 19th, 2021
- CAR T-Cell Therapy Carves Out a Role in Multiple Myeloma - OncLive - February 19th, 2021
- AI project to power the future of stem cell manufacturing - TechCentral.ie - February 17th, 2021
- Promising Results for Stem Cell Treatment of Degenerative ... - February 14th, 2021
- Stem Cell Therapy for Heart Disease - Cleveland Clinic - February 14th, 2021
- Stem Cell Therapy for Stroke Recovery | University of ... - February 14th, 2021
- Stem Cell Treatment: Future of Modern Medicine | Science Times - February 14th, 2021
- Researchers may have found a miracle cure for baldness - BGR - February 14th, 2021
- Types of leukemia: Prevalence, treatment options, and prognosis - Medical News Today - February 14th, 2021
- Jasper Therapeutics Announces Launch of New Clinical Trial with National Heart, Lung, and Blood Institute to Evaluate JSP191 in Sickle Cell Disease -... - February 14th, 2021
- Global Stem Cell Therapy Market Set to Reach USD 214.5 Million by 2024 - The Courier - February 14th, 2021
- Keep it Flowing: Combating COVID-19 Blood Shortages in Cancer Treatment - Curetoday.com - February 14th, 2021
- Network-based screen in iPSC-derived cells reveals therapeutic candidate for heart valve disease - Science - February 14th, 2021
- AlloVir Research Presented at the 2021 Transplantation & Cellular Therapy Meeting Digital Experience - Business Wire - February 14th, 2021
- Orchard Therapeutics talks the benefits of HSC therapy - BioProcess Insider - February 14th, 2021
- Protein Discovery in the Development of New Hearing Hair Cells Could Lead to Treatments for Hearing Loss - SciTechDaily - February 7th, 2021
- Stem cells efficacy confirmed in treating ototoxic hearing loss - Korea Biomedical Review - February 4th, 2021
- Groundbreaking Trial Shows Umbilical-Cord Derived Mesenchymal Stem Cell Infusions Save Lives of Sickest COVID-19 Patients - HospiMedica - January 22nd, 2021
- Massive Growth in Canine Stem Cell Therapy Market Set to Witness Huge Growth by 2027| VETSTEM BIOPHARMA, Cell Therapy Sciences, Regeneus, Aratana... - January 22nd, 2021
- Autologous stem cell transplantation versus bortezomib for the first line treatment of systemic light chain amyloidosis in the UK - DocWire News - January 22nd, 2021
- Promising Steps Toward Retinal Cell Transplants to Fight Blindness - HealthDay News - January 22nd, 2021