Clinical trials news: September 2011 update

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

What's covered in this update?

We’ve focused on trials using embryonic stem cells or taking place in Europe, and cover spinal cord injuryStargardt's macular dystrophyage-related macular degeneration 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.



About clinical trials

Clinical trials are carried out in four phases:

  • Phase I and Phase II trials check on safety – they make sure the treatment does no harm to patients. 
  • Phase III or IV trials focus on assessing how effective the treatment is, and developing it into a therapy that can be made widely available.
More information on this website
Clinical trials FAQ
How research is turned into treatments

Embyronic stem cell clinical trials

In October 2010, the first trial of a therapy derived from human embryonic stem cells was launched in the USA. Soon afterwards in 2011, two other trials began.

Spinal cord injury

***Update November 2011: Geron announced it is halting this trial to focus on other areas of research. 

The US company Geron is conducting a safety study on a treatment for spinal cord injury. Geron has used embryonic stem cells to make a type of cell called oligodendrocyte progenitor cells – precursors of a kind of neural cell. These cells (called GRNOPC1) will be injected directly into the patient’s spinal cord 7-14 days after injury. Geron scientists hope the injected cells will release substances that help the healing process in the spine.

Key facts

Phase and objective: This is a phase I trial. The objective is to evaluate the safety of administering GRNOPC1 cells to patients with recent spinal cord injuries.
Dates: October 2010 – October 2012.
Enrollment status: Recruiting. Aims to enroll up to 10 patients. To date, 4 patients have been enrolled and treated.
More information on this study
More about spinal cord injury

Stargardt’s macular dystrophy

A safety study is underway in patients with advanced Stargardt's macular dystrophy, conducted by Advanced Cell Technology (ACT) in the USA. Stargardt’s macular dystrophy causes retinal pigment epithelial (RPE) cells in a part of the eye called the macula to stop working. This leads to problems with vision. The disease usually first appears in patients aged 10 to 20 years old. In its trial, Advanced Cell Technology is growing RPE cells from human embryonic stem cells in the lab. RPE cells are found at the back of the eye and their job is to provide nutrients and protection for the light-sensitive cells in the retina that allow us to see. ACT will transplant its lab-grown RPE cells into the eyes of patients with advanced Stargardt's macular dystrophy.

Key facts

Phase and objective: This is a phase I/II trial. The objective is to evaluate the safety of transplanting retinal pigment cells derived from human embryonic stem cells into the eye. A secondary objective is to examine how well the cells engraft (how well they attach to the surrounding tissue and survive).
Dates: April 2011– September 2013
Enrollment status: Recruiting. One patient has been injected. Aims to enroll up to 12 patients
More information on this study
More about Stargardt’s disease

Age-related macular degeneration

Human embryonic stem cell colonyHuman embryonic stem cell colony

Advanced Cell Technology (ACT) is also carrying out a safety study in patients with dry age- related macular degeneration (AMD), another eye condition that affects the tiny area at the back of the eye called the macula. AMD causes problems with central vision. The damage gets worse over time, and some patients experience severe loss of vision. AMD mostly affects people over 65. In its clinical trial for AMD, Advanced Cell Technology will use retinal pigment epithelial cells like the ones described in the trial for Stargardt’s disease above. RPE cells are also damaged in AMD. In the healthy eye, they provide nutrients and protection for the light-sensitive cells in the retina that allow us see. ACT will grow RPE cells from human embryonic stem cells and inject them into the eyes of patients with AMD.

Key facts

Phase and objective: This is a phase I/II trial. The objective is to evaluate the safety of transplanting retinal pigment cells derived from human embryonic stem cells into the eye. It will also try different doses of cells to see if any changes in vision can be detected.
Dates: April 2011– July 2013
Enrollment status: Recruiting. One patient has been injected. Aims to enroll up to 12 patients.
More information on this study
More about age-related macular degeneration

 

Stem cell clinical trials in Europe

In Europe, clinical trials of stem cell therapies are beginning to take place in several disease areas. Here are three of the latest.

Macular degeneration

The retinal pigment epithelium (RPE) cells derived from human embryonic stem cells by Advanced Cell Technology are also going to be evaluated in European patients. ACT has filed a European Clinical Trial Application for a phase I/II study on macular degeneration, but the trial has not started yet.

Key facts

Phase and objective: This is a phase I/II trial to evaluate the safety of transplanting retinal pigment cells derived from human embryonic stem cells into the eye.
Dates: Not started; awaiting approval.
Enrollment status: Not yet recruiting

Spinal cord injury

The spinal cord - a delicate structureThe spinal cord - a delicate structureIn December 2010 the Swiss regulatory agency for therapeutic products gave the go-ahead for a Phase I/II clinical trial on chronic spinal cord injury at the Balgrist University Hospital in Zurich, Switzerland.  The trial uses a type of stem cell developed by the company Stem Cells Inc. The cells are known as human central nervous system stem cells, or HuCNS-SC, and are derived from human fetal brain tissue. HuCNS-SC cells can make any of the three major kinds of neural cells found in the central nervous system. In the trial, HuCNS-SC cells will be transplanted into the spinal cord of patients at least six weeks after injury.

Key facts

Phase and objective: This is a phase I/II trial to evaluate the safety of transplanting HuCNS-SC cells into the spine of patients with chronic spinal injury.
Dates: March 2011–March 2016.
Enrollment status: Recruiting. Aims to enroll 12 patients.
More information on this study
More information about HuCNS-SC
More about spinal cord injury

Stroke

A pilot study of a neural stem cell therapy for stroke patients started in June 2010 at the Institute of Neurological Sciences at Glasgow University. It recruited its third patient in May 2011. The trial aims to assess safety. It uses a neural stem cell line (CTX cells) produced by the company ReNeuron. The cells are derived from human fetal tissue and are being injected directly into patients' brains. Patients will receive gradually increasing doses of cells as the trial continues, and will be assessed regularly to check for any adverse effects. Three patients have so far received their first injection, containing the lowest dose of cells that will be tested. In August 2011, a review of the findings so far found that no adverse effects of the cells have been observed and the trial can proceed to investigating a higher dose. Patients will be monitored for two years and will be invited to participate in a long-term follow-up trial for a further 8 years.

Key facts

Phase and objective: This is a phase I trial designed to evaluate the safety of a manufactured neural stem cell line (CTX cells) delivered by injection into the damaged brains of patients after a stroke.
Dates: Started June 2010
Enrollment status: Recruiting. First three patients have received their first injection. Aims to enroll 12 patients.
More information on this study
More about stroke

 

Find out more

Acknowledgements

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

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