First-ever “reprogrammed” stem cells for treating heart disease
Scientists in Japan now have the “go signal” to use the “reprogrammed” stem cells in treating people who have heart disease. The experimental therapy will be applied to three people in Japan next year.
The “reprogrammed” stem cells treatment is part of the revolutionary reprogramming technique and is only the second clinical pilot trial of the induced pluripotent stem (iPS) cells.
Yoshiki Sawa, a cardiac surgeon at the Osaka University explains that the treatment is created by inducing cells from body tissues to bring them back to an embryonic-like state. “It is in this form where they can develop into other cell types,” says Dr. Sawa.
During the treatment process, doctors will take thin sheets of tissues from iPS cells and implant them onto the defective human heart. These tissue sheets are expected to regenerate the damaged parts of the heart, which are usually caused by plaque build-up.
Thomas Eschenhagen, a pharmacologist at the University of Hamburg, Germany, and chair of the German Centre for Cardiovascular Research is very excited to present the treatment to a wider public. “I am confident that the world is similarly eager to learn this new treatment considering that many groups of scientists are gearing towards the same direction,” says Eschenhagen.
When all goes well in 2019, the team will seek approval to conduct a clinical trial in a larger group of patients. Subsequently, the team will make it commercially available for the public under Japan’s accelerated system for regenerative medicine.
The treatment was first introduced in 2014 with the goal of expanding the availability of revolutionary life-saving medical procedures in Japan. However, the government has halted the system pending the determination of its safety and efficacy in treating heart diseases. It was suggested that sufficient data must first be established to prove that the procedure works before selling it out to the public.
Experimentation Process Explained
During the experimental stage, Dr. Saw and his colleagues utilized the iPS cells to generate 100 million heart-muscle cells. The team grafted around 0.1mm thick and 4cm long cells from these sheets and implanted them on a pig’s heart.
The studies have revealed that the implantation improved the organ’s function. However, since the implants do not literally integrate into the heart, the better approach is to release growth factors to help regenerate damaged muscles.
“The advantage of using iPS cells is their ability to generate their own cellular matrix and maintain it without the infusion of foreign materials, thereby maintaining its originality,” says Dr. Sawa. “In contrast with engineered tissues, these are a much smarter way to deliver cells.”
Dr. Sawa and his teammates have been conducting the study for over 15 years now. Wolfram-Hubertus Zimmermann, a pharmacologist at the University Medical Centre Göttingen in Germany who helped in developing the treatment says that the latest trial will be based on Dr. Sawa’s studies.
Zimmerman is hopeful that the treatment will be successful so that they can hold a clinical trial at a larger scale. “This lets scientists bypass a more expansive testing, rather than mere pilot trials,” says Zimmerman.
Insufficient data to support commercial sale
Many researchers are still not convinced regarding the introduction of “reprogrammed” stem cell treatment for heart disease in the commercial market. Even if the treatment will be successful, there will always be some risks associated with it.
The government says that if Dr. Sawa’s team is planning to make the treatment commercially available, the efficacy of the new treatment must outweigh the risks. Thus, if people give up some existing treatments in favor of this new system, there is a 100 percent assurance that it will work.
Yoshiki Yui, a cardiologist at Japan’s Kyoto University also suggests that the researchers must demonstrate the efficacy of their treatment at a larger population through a controlled clinical trial. The evaluation process should also observe ethical regulations and practices, and must pass the standards of demonstrating efficacy in medical researches.
“At present, the biggest problem we’re facing is the lack of an adequate system for evaluation and controlled trial,” says Yui.
Meanwhile, a spokesperson for the health ministry in Japan argues that the system is already sufficient because even if the treatment is proven to be safe and effective, the researchers will have to continue demonstrating its efficacy to obtain approval for commercial use.
Dr. Sawa agrees that a controlled clinical trial is still necessary even though it is not legally required under Japan’s rules and regulations. “The Ministry of Health has already approved our treatment to be tested in three patients next year which means that we’ve passed scientific and ethical standards,” says Dr. Sawa. “Whether it is entirely effective or not, that’s what we need to find out.”