Ten patients with severe heart failure were told they had between six months and two years to live. Most of them are still alive today, around eight years after receiving experimental stem cell injections, and the scientist behind the treatment has now chosen Abu Dhabi to run the world’s next pivotal controlled trial of the therapy.
Sir Martin Evans, who won the Nobel Prize in 2007 for his discovery of embryonic stem cells, made the announcement on Wednesday at the Abu Dhabi Future Health Summit.
Evans, founder of UK biotechnology company Cardiogeni, said Abu Dhabi’s Department of Health had offered the regulatory support and encouragement he had been unable to find elsewhere.
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“What we found in Abu Dhabi is a health authority who is happy and supportive and will help us make sure all the regulations are properly in place, but also encourage us,” he said. “They’re not restrictive as we found elsewhere, but they are encouraging.”
The treatment, known as CLXR-001, uses mesenchymal stem cells derived from bone marrow, which are injected directly into the heart muscle during coronary artery bypass surgery. The cells do not permanently settle in the body. Instead, Evans said, they appear to deliver a biochemical signal that reawakens dormant heart muscle.
“We’re using these cells to deliver some sort of message to the heart muscle which is damaged but able to go back into full function,” he said.
Evans explained that during a heart attack, some muscle cells die entirely and form scar tissue, while others enter a state of hibernation, still alive but no longer beating. The injected cells appeared to bring these hibernating muscles back to life. “What we seem to have been able to do is to have a positive effect on these reserve muscles to come back into use again,” he said.
The European Phase IIa trial showed statistically significant improvements in all measured outcomes, including heart function, reduction in scarring and quality of life. The new Abu Dhabi trial will be larger and double-blind, meaning neither patients, doctors nor researchers will know who receives the treatment and who receives a dummy injection, meeting the standards required for full regulatory approval worldwide.
Evans was direct about what the trial represents. “Today’s protocol is a tryout, this is the Model T Ford only. It’s going to be much better in the future,” he said. If successful, Abu Dhabi may become the first health authority in the world to approve the therapy for widespread use, with Evans noting that the number of patients worldwide who could benefit is enormous.
The Abu Dhabi Future Health Summit continues under the patronage of Sheikh Khalid bin Mohamed bin Zayed Al Nahyan, Crown Prince of Abu Dhabi.
Source: Khaleej Times
