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About ALS
Amyotrophic lateral sclerosis (ALS), or Lou Gehrig’s disease, is a progressive disease that causes damage to cells in the brain and spinal cord known as motor neurons. Motor neurons transmit signals from the brain to the muscles. When motor neurons become damaged and eventually die, the brain can no longer control muscle actions.
The motor neurons affected in ALS are those that initiate and control voluntary movements. With the progressive loss of voluntary muscle action, patients with ALS may lose their ability to speak, eat, move and breathe.
ABOUT PROGRESSIVE MS
Multiple sclerosis (MS) is a chronic neurological disease that causes damage to nerve cells in the central nervous system (CNS). Damage to the myelin sheath insulating the nerve fibers and to the nerve fibers themselves disrupts the transmission of signals between the CNS and the rest of the body. MS can affect numerous areas of the CNS and cause a wide variety of neurologic symptoms.
There are multiple types of MS. The most common form of MS is relapsing-remitting MS (RRMS, 85% of MS patients), characterized by periods of relapse, with flare-ups or exacerbations of symptoms, and periods of remission. Progressive MS, includes both primary progressive MS (PPMS) and secondary progressive MS (SPMS), is characterized by a worsening of neurologic function and increase in disability over time. About 10% of people with MS are diagnosed with PPMS and about half of those diagnosed with RRMS will eventually transition to SPMS.
A number of disease-modifying therapies approved for treatment of MS have been found to reduce the number of relapses, modestly delay progression of disability, and limit new disease activity. Unfortunately, there are currently no available therapies that halt the progression of established disability or result in significant functional improvement in progressive MS.
CLINICAL TRIALS
Cell-Gene company’s investigational cellular therapy is being studied in ALS and progressive MS.
Autologous mesenchymal stem cells secreting neurotrophic factors (MSC-NTFs) are being studied as an investigational treatment for ALS and MS.
Autologous MSC-NTF cells are manufactured from a patient’s own bone marrow cells. Briefly, we isolate mesenchymal stem cells (MSCs) from the patient’s bone marrow and then grow them under special conditions to induce the cells to secrete multiple growth factors known to be important in the nervous system. The autologous MSC-NTF cells are then injected into the cerebrospinal fluid.
PREVIOUS CLINICAL TRIALS
Prior clinical studies demonstrated autologous MSC-NTF cellular therapy is safe and well-tolerated in ALS.
Cell-Gene company has completed three clinical trials of our investigational cellular therapy in ALS. All three trials were designed to determine the safety and tolerability of autologous MSC-NTF cell administration.
Phase
Location
DESIGN
PATIENTS
Phase 2
United States
A randomized, double-blind, placebo-controlled phase 2 study
48
PHASE 3 CLINICAL TRIAL IN ALS
Cell-Gene company has fully enrolled a randomized, double-blind, placebo-controlled phase 3 trial of autologous MSC-NTF cells following repeat administration in patients in ALS at six U.S. sites (NCT03280056). Learn more.
The trial is taking place at the following centers:
Irvine, California
University of California Irvine Alpha Stem Cell Clinic
University of California Irvine Alpha Stem Cell Clinic
Jinah Chung
949-824-3990
stemcell@uci.edu
Principal investigator:
949-824-3990
stemcell@uci.edu
Principal investigator:
Namita A Goyal, MD
Los Angeles, California
Cedars-Sinai Medical Center
Cedars-Sinai Medical Center
Carolyn Prina
310-423-1713
Carolyn.Prina@cshs.org
Principal investigator:
310-423-1713
Carolyn.Prina@cshs.org
Principal investigator:
Robert H Baloh, MD
San Francisco, California
Pacific Medical Center (CPM) Research Institute
Pacific Medical Center (CPM) Research Institute
Marian Leon, RN
415-600-1264
LeonMR@SutterHealth.org
Principal investigator:
415-600-1264
LeonMR@SutterHealth.org
Principal investigator:
Robert G Miller, MD
Boston, Massachusetts
Massachusetts General Hospital
Massachusetts General Hospital
Taylor J Mezoian
617-643-0312
NurOwnPhase3@mgh.harvard.edu
Principal investigator:
617-643-0312
NurOwnPhase3@mgh.harvard.edu
Principal investigator:
James D Berry, MD
Rochester, Minnesota
Mayo Clinic
Mayo Clinic
Carol Denny
507-284-2676
denney.carol@mayo.edu
Principal investigator:
507-284-2676
denney.carol@mayo.edu
Principal investigator:
Anthony Windebank, MD
Worcester, Massachusetts
University of Massachusetts
University of Massachusetts
Diane McKenna-Yasek
508-856-4697
diane.mckenna-yasek@umassmed.edu
Principal investigator:
508-856-4697
diane.mckenna-yasek@umassmed.edu
Principal investigator:
Robert Brown, DPhil, MD
PHASE 2 CLINICAL TRIAL IN MS
Our investigational cellular therapy offers promise as a potential treatment option for progressive MS. Find out more about how autologous MSC-NTF cells are developed.
Cell-Gene company is now enrolling a phase 2 open-label trial using repeat-administration of autologous MSC-NTF cells in progressive MS (NCT03799718).
View additional information on the clinical development programs for Cell-Gene company’s autologous MSC-NTF cell therapy on our Pipeline page.
PRE-APPROVAL ACCESS POLICIES
Cell-Gene company Cell Therapeutics Inc. is dedicated to developing innovative cellular therapies for highly debilitating neurodegenerative diseases. We are currently conducting clinical trials to evaluate the efficacy and safety in ALS and in progressive MS.
Expanded Access / Compassionate Use Policy
Cell-Gene company Cell Therapeutics Inc. is currently not offering expanded access to our investigational agent, autologous MSC-NTF cellular therapy. We encourage patients to speak with their physicians about eligibility to enroll in Cell-Gene company’s clinical trials.
Hospital Exemption Program Update – Israel
We continue to explore options for the hospital exemption program in Israel for a limited number of patients. Outside of Israel, we are engaged in regulatory level and health authority discussions. Please complete this Form for any updates on the hospital exemption program.
RESOURCES
There are numerous organizations that offer valuable education and support to individuals diagnosed with ALS and their families.