The US Food and Drug Administration (FDA) has approved a new clinical trial of a groundbreaking strategy using stem cells for the treatment of MS (multiple sclerosis).
Researchers from the Tisch MS Research Center of New York say the FDA has granted approval to begin early clinical investigation (phase 1 trial) of autologous neural stem cells in the treatment of MS.
Multiple sclerosis is a chronic disease that attacks the central nervous system (the spinal cord, optic nerves and brain). Common symptoms are numbness of the limbs, but more severe cases can lead to paralysis and blindness.
According to the Multiple Sclerosis Foundation, there are currently between 350,000 to 500,000 people in the US who have been diagnosed with MS, and 200 people are diagnosed with the disease every week.
The new regenerative strategy will involve using autologous, mesenchymal stem cell-derived neural progenitor cells (MSC-NPs), which will be harvested from the bone marrow of 20 MS patients who meet the criteria for the trial.
The stem cells will then be injected into the cerebrospinal fluid that surrounds the spinal cords of the patients. The main objective of the clinical trial, the researchers say, is to determine the safety of the treatment, while the secondary objective is to measure the effectiveness.
The injections will be repeated at 3-month intervals, with their safety and efficacy frequently monitored through follow-up visits. After the final injection, patients will be monitored for up to 27 months.
Preclinical testing of this procedure showed that after injecting the stem cells, brain inflammation was reduced in seven MS patients, myelin was repaired (an insulating layer that forms around nerves), and protection of the neuronal structure and function of the brain was improved.
Dr. Saud Sadiq, senior research scientist at Tisch MS Research Center of New York, says:
"This study provides a hope that established disability may be reversed in MS."
Similar trials but with larger number of patients (phase 2) have already been under way in the UK. In 2011, UK scientists received Ј1 million from the MS Society and the UK Stem Cell Foundation (UKCSF) towards research investigating whether stem cells can slow, stop or reverse brain and spinal cord damage in MS patients.
Dr. Sadiq says of this most recent trial in the US:
"To my knowledge, this is the first FDA-approved stem cell trial in the United States to investigate direct injection of stem cells into the cerebrospinal fluid of MS patients, and represents an exciting advance in MS research and treatment."
The clinical trial will begin enrolling patients once ethical approval has been granted and funding secured.