What is the attempt of a biotechnology company to recover 'deafness' that leads to dementia and deterioration of QOL by regenerative treatment?

Deafness , which makes sounds and words hard to hear or completely inaudible, is a dangerous illness that can lead to poor quality of life and dementia . A biotechnology company called Frequency Therapeutics , spun out of the Massachusetts Institute of Technology in 2015, is focusing on 'regenerative therapies' to treat such deafness.

Reversing hearing loss with regenerative therapy | MIT News | Massachusetts Institute of Technology

One of the causes of deafness is damage to hair cells that convert sound vibrations into electrical signals that are transmitted to the brain. There are about 15,000 hair cells in one ear, and when hair cells are damaged by the effects of aging, noise, certain chemicals, antibiotics, etc., the most common deafness is sensory deafness. You will have hearing loss. Once damaged, hair cells do not self-regenerate, making it difficult to treat advanced sensorineural hearing loss.

However, biotechnology company Frequency Therapeutics is trying to create damaged hair cells by using small molecules that activate inner ear progenitor cells , which are cells just before they differentiate into hair cells. 'Voice perception is the first goal to improve hearing and the number one need to hear from patients,' said Chris Loose, co-founder and chief price officer of Frequency Therapeutics.

Frequency Therapeutics is already in clinical trials, and as of March 2022, in three different clinical trials, a total of more than 200 subjects were given small molecules that activate progenitor cells and were clinically tested. It has been confirmed that there is a significant improvement in speech perception. Although another study failed to show an improvement in hearing compared to a placebo-treated control, Frequency Therapeutics identified this as a design issue in the study, from October 2022 to 11 Over the month, we plan to conduct clinical trials with an additional 124 subjects.

'Hearing is a very important sensation. Hearing connects people with the community and fosters a sense of identity,' said Jeff Karp, co-founder of Frequency Therapeutics and professor of anesthesiology at Brigham and Women's Hospital. I think the possibility of restoring hearing will have a big impact on society. '

Loose was enrolled in the Massachusetts Institute of Technology's Sloan School of Business in 2005 with the help of Professor

Robert Langer of the Massachusetts Institute of Technology's Department of Chemical Engineering in 2005. I met him. The two later launched the medical device company Semprus BioSciences, which was sold in 2012 for $ 80 million (about 6.4 billion yen at the rate at the time).

Meanwhile, Langer, who brought together Loose and Lucchino, discovered around 2012 that small molecules that control intestinal stem cells, along with Karp, activate progenitor cells. Progenitor cells present in the inner ear differentiate into hair cells when the fetus is in the womb, but because they are dormant before birth, no hair cells are produced after birth. However, in 2012, Langer et al.'S research team succeeded in transforming thousands of progenitor cells into hair cells using small molecules, albeit in a laboratory environment. So Langer contacted Loose and Lucchino again and founded Frequency Therapeutics in 2015 with Lucchino as CEO.

Compared to gene therapy, which involves extracting patient cells, recombining genes in the laboratory, and returning them to the patient's body, 'injecting small molecules into the inner ear to transform progenitor cells into specific cells.' The Frequency Therapeutics approach seems to have many advantages. 'Because the tissues of the whole body contain progenitor cells, the Frequency Therapeutics approach has a wide range of uses, and we believe this is the future of regenerative medicine,' Loose said.

In past clinical trials, subjects who had been suffering from deafness for 30 years reported, 'I was able to hear for the first time what children were saying in a crowded restaurant I visited after treatment,' Langer said. There are cases. 'This makes a lot of sense to them. Of course, we have to help more people, but the fact that we were able to help a small group alone is really impressive to me.' Langer commented.

In the future, Karp believes that research on Frequency Therapeutics will improve the technology for activating progenitor cells and lead to the development of new treatments for deafness. Karp wonders if the resources and great science and technology devoted to the field of deafness treatment make deafness treatment 10 to 15 years later like

LASIK surgery that can restore vision in just 1 to 2 hours of treatment. 'I think we can see the same thing about LASIK surgery for deafness,' he said.

Furthermore, the research field of Frequency Therapeutics is not limited to the treatment of deafness, and we are also developing therapeutic agents for multiple sclerosis, in which immune cells attack nerve tissue and cause sensory and motor disorders. In multiple sclerosis, the myelin sheath that surrounds the axons of nerve cells is attacked, but the precursor cells differentiate into myelin-producing cells in the brain, activating the precursor cells to increase the speed of myelin production. By raising it, there is a possibility that multiple sclerosis can be treated. Frequency Therapeutics has already confirmed the effect in animal experiments on mice, and it is said that it plans to apply for clinical trials to the US Food and Drug Administration in 2023.

Karp had previously assumed that the therapeutic method of activating progenitor cells would have a wide range of applications. 'Since we were envisioning small molecule treatments, we intended to make this treatment a platform that could be applied to multiple tissues. We are currently working on research on myelin formation. But given what we can do by using small molecules to control local biology, this is just the tip of the iceberg, 'Karp said.

in Science, Posted by log1h_ik