Stem cell therapy IND 13729 for amyotrophic lateral sclerosis (ALS) in clinical trials: TCA Cellular

IND 13729, a stem cell therapy for the treatment of amyotrophic lateral sclerosis (ALS), has been given approval for first-in-man studies.

The U.S. Food and Drug Administration has approved its adult stem cell protocol to conduct Phase I clinical trials to treat Amyotrophic Lateral Sclerosis (Lou Gehrig’s disease), TCA Cellular Therapy, LLC announced.

This is the second FDA-approved protocol for the treatment of ALS using stem cells in the country; and the first using adult stem cells from the same patient. The aim of the Phase I study is to  assess safety of  IND 13729, as a stem cell therapy for the treatment of amyotrophic lateral sclerosis (ALS) .

The adult stem cells will be taken from the patient’s bone marrow in a simple outpatient procedure.

The cells will then be processed in TCA-CT’s GMP laboratory and administered to the patient by spinal tap in one of TCA-CT’s facilities.

Recruitment for trial patients will commence in the next few weeks. The company anticipates moving into Phase II within a year.

ALS afflicts approximately 30,000 Americans. More people die of ALS than Huntington’s disease; and the fatalities nearly equal Multiple Sclerosis. The life expectancy of a patient diagnosed with ALS is two to five years.

“I hope that our trial, along with the combined efforts of scientists and patients, will pave the way to breaking the chains of this devastating disease,” stated Gabriel Lasala, M.D., president and CEO of TCA Cellular Therapy.

What is Amyotrophic Lateral Sclerosis?

Amyotrophic lateral sclerosis (ALS), sometimes called Lou Gehrig’s disease, is a rapidly progressive fatal neurological disease that attacks the nerve cells (neurons) responsible for controlling voluntary muscles.

In ALS, both the upper motor neurons and the lower motor neurons degenerate or die, ceasing to send messages to muscles. Unable to function, the muscles gradually weaken, waste away, and twitch.

Eventually the ability of the brain to start and control voluntary movement is lost.

Individuals with ALS lose their strength and the ability to move their arms, legs, and body. When muscles in the diaphragm and chest wall fail, individuals lose the ability to breathe without ventilatory support.

ALS does not affect a person’s ability to see, smell, taste, hear, or recognize touch, and it does not usually impair a person’s thinking or other cognitive abilities.

No cure has yet been found for ALS. However, the FDA has approved the first drug treatment for the disease—riluzole.  Riluzole is believed to reduce damage to motor neurons and prolongs survival by several months, mainly in those with difficulty swallowing.

CA Cellular Therapy, LLC (TCA Cellular) is a biotechnology, research and development company that intends to invent, develop, patent, and market cellular therapy products for medical applications.  An autologous combination stem cell product has been developed for regenerative medicine therapy.

TCA Cellular Therapy also owns controlling interest in LifeSource Cryobank, LLC, a company specializing in the cryopreservation of umbilical cord blood and other adult stem cells.

TCA Cellular Therapy has built a new facility equipped with Controlled Rate Freezing and Liquid Nitrogen (LN2) Storage systems.

The CBS 2100 Controlled Rate Freezing System, a fully computerized instrument, eliminates all variables associated with ultra low freezing to ensure maximum cell viability.

Cells under storage, both for short-term  and long-tem periods are preserved in CBS Isothermal freezers which provides ultra low storage temperatures (-190 degree C range)without contact of liquid nitrogen in the sample storage space.  This system eliminates the need to submerge samples in liquid nitrogen, thus eliminating the possibility of sample cross contamination.

In December, ReN001 stem-cell line, a experimental therapy that offers great promise to rejuvenate brain cells in stroke patients developed by ReNeuron, has been approved for clinical studies by UK government.

ReN001 stem-cell line will be administered in patients who suffered stroke early next year.

ReNeuron will be the first biotechnology to trial the use of fetal stem cells for treating stroke in humans. The stem cells extracted from fetal blood has been successfully tested  in rodents.

Earlier, a few other companies have tried stem cells derived from a patient’s bone marrow, fetal pig brain cells or tumors grown from germ eggs and sperm to treat stroke patients with little success.

Last year, Cell Therapeutics Corp, which was running a human trial using patients’ stem cells to stimulate growth of new brain cells in stroke patients, was asked to stop trials by North American regulators on safety reasons.

ReNeuron’s ReN001 stem-cell has been grown from a tissue sample taken in 2003 from the brain of an aborted fetus.

Fetal stem cells are grown from tissue taken from a fetus, which an embryo becomes eight weeks after conception.

ReNeuron can grow all the cells needed for treatment from the single tissue sample, and no need for more fetuses reports said.