Before new treatments can reach patients, they must be tested in clinical trials. This is our second brief update on some stem-cell-related trials currently under way or recently approved.

What's covered in this update?

This update looks at trials for amyotrophic lateral sclerosis (ALS) or motor neurone disease, as well as the latest news on how the trials we told you about in September are progressing. We’ve included details of one or two new trials for some of the conditions we focussed on last time: spinal injury, Stargardt’s macular dystrophy and stroke. We’ll post more updates on other themes in future, so tell us if there’s an area you really want to know about.

Amyotrophic lateral sclerosis (ALS) or motor neurone disease

ALS trial in Atlanta, USA

The company Neuralstem Inc and researchers at Emory University in Atlanta, USA have received approval from the Food and Drug Administration (FDA) to advance to the second stage of their trial investigating the safety of using human neural stem cells to treat patients with amyotrophic lateral sclerosis (ALS). Amyotrophic lateral sclerosis is also known as motor neurone disease, or sometimes Lou Gehrig’s disease. In ALS, the nerve cells that control movement degenerate and die. These nerve cells are found both in the spinal cord and in the brain.

The Neuralstem Inc/Emory clinical trial started in January 2010 and is designed to assess the safety of implanting neural stem cells derived from human fetal tissue into the spinal cord in up to 18 people with ALS. The first 12 patients received neural stem cells in the lumbar, or lower, region of the spinal cord. Following a review of the safety data in autumn 2011, the FDA granted approval to transplant neural stem cells in the cervical (upper) region of the spinal cord.

Key facts

Phase and objective: This is a phase I trial. The objective is to evaluate the feasibility and safety of transplanting human spinal-cord-derived neural stem cells into the spinal cord of patients with amyotrophic lateral sclerosis.
Dates: January 2010 – October 2012
Enrollment status: Recruiting. 12 patients already recruited. Aims to enroll up to18 patients.
More information on this study
More about Amyotrophic Lateral Sclerosis

ALS trial in China

The State Food and Drug Administration in China authorised a phase II trial on the use of umbilical cord mesenchymal; stem cells in amyotrophic lateral sclerosis. The cells will be injected by lumbar puncture: a hollow needle is inserted between the bones of the lower back into the fluid around the lower part of the spinal cord. The trial is being run by the General Hospital of Chinese Armed Police Forces. The researchers hope that the injected stem cells will release small proteins called trophic factors that help keep motorneurons healthy and working properly.

Key facts

Phase and objective: This is a phase II trial. The objective is to evaluate the safety and efficacy of transplanting umbilical cord mesenchymal stem cells by lumbar puncture into patients with ALS.
Dates: January 2012 – April 2015  
Enrollment status: Not yet open for participant recruitment. Aims to enroll up to 30 patients.
More information on this study
More about Amyotrophic Lateral Sclerosis

Progress news on trials from our last update

In our September 2011 clinical trials update we described trials for macular degeneration, spinal injury and stroke. Some of these trials have moved forward or stopped, and additional trials are getting underway for some of these conditions. Below is an update on progress.

Stargardt’s macular dystrophy

In September we told you about a safety study being carried out by the company Advanced Cell Technology (ACT) in the USA for patients with advanced Stargardt’s macular dystrophy. ACT now has authorization to start a trial in the UK using the same type of cells as in the US. These cells are called retinal pigment epithelium (RPE) cells and are made from human embryonic stem cells. The UK trial will be a phase I/II study and will be carried out at the Moorfields Eye Hospital NHS Foundation Trust in London, UK.

Key facts

Phase and objective: This is a phase I/II trial to evaluate the safety and tolerability of transplanting retinal pigment epithelial cells derived from human embryonic stem cells into the eye.
Dates: November 2011 – December 2013.
Enrollment status: Recruiting. Aims to enroll up to 12 patients
More about this study
More about Stargardt’s disease
September 2011 clinical trials update from EuroStemCell

Spinal cord injury

Geron trial

In October 2011, Geron presented the safety data from four patients who received the lowest intended dose of cells called GRNOPC1 in the company’s phase I clinical trial for spinal injury. The cells did not cause any serious problems. However, in November 2011 Geron announced it is halting this trial to focus on other areas of research. 

Press release from Geron about it’s first results from the trial
News article on halting of this trial

StemCells Inc. trial

In December 2011, StemCells Inc announced that the first cohort of patients with chronic spinal injury had been transplanted with human central nervous system stem cells (HuCNS-SC) produced by the company. This phase I/II trial is taking place at the Balgrist University Hospital in Zurich in Switzerland and started in March 2011. Its aim is to evaluate the safety of transplanting HuCNS-SC cells into the spine of patients with chronic spinal injury. The trial is currently recruiting patients and the company has opened up enrollment to include patients in the USA and Canada.

Press release from Stem Cells Inc on progress and patient enrollment
Previous clinical trials update from EuroStemCell

New spinal injury trial in India

The US and Indian company TotipotentRX is conducting a study in India using bone marrow stem cells in spinal injury patients.  The stem cells will be derived from the patient’s bone marrow and transplanted back into the spine.  This is known as an autologous transplant – the transplanted cells come from the patient’s own body. It is hoped that the bone marrow stem cells will help reduce the extent of damage to tissues after spinal injury, and encourage repair of nerves.

Key facts

Phase and objective: This is a phase I/II trial. The objective is to evaluate the of autologous bone-marrow-derived stem cell transplantation in patients with spinal cord injury. A secondary objective is to look for evidence of a positive effect of the transplant.
Dates: October 2011 – October 2013
Enrollment status: Recruiting. Aims to enroll up to 15 patients.
More information on this study
More about spinal cord injury

Stroke

ReNeuron trial

ReNeuron’s pilot study of it’s neural stem cell therapy ReN001 for stroke patients started in June 2010 at the Institute of Neurological Sciences at Glasgow University and has now recruited 6 patients. In late October 2011, the data collected from the first four patients were reviewed by the trial independent data safety board and no cell-related problems have been reported to date. ReNeuron expects that all remaining patients in the clinical trial will be treated over the course of 2012. 

Press release from ReNeuron about the trial's progress

Find out more

• Our FAQ on clinical trials and stem cell treatments
• Patient Handbook on stem cell therapies from the International Society for Stem Cell Research
• ClinicalTrials.gov: clinical trials database
• EU Clinical Trials Register: European clinical trials database

Acknowledgements

Christine Kubiak from ECRIN researched and helped put together this update.
Lead image: ©iStockphoto.com/Photo-Dave

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