What is a biotech company’s attempt to reclaim the “deafness” that leads to dementia and impaired quality of life through regenerative therapy? –GASINE

Sounds and words are difficult to hear or completely inaudibleDeafnessIs a decline in quality of lifeDementiaIt is also a dangerous disease. Exited from Massachusetts Institute of Technology in 2015Therapeutic FrequencyThe biotechnology company is focusing on “regenerative therapy” to treat these deafness.

Reverse Hearing Loss with Regenerative Therapy | MIT News | Massachusetts Institute of Technology

One of the causes of deafness is the conversion of sound vibrations into electrical signals which are transmitted to the brain.Hair cellsThere is damage. There are approximately 15,000 hair cells in an ear, and when hair cells are damaged by the effects of aging, noise, certain chemicals, antibiotics, etc., the most common deafness is sensory deafness. Once damaged, hair cells do not regenerate on their own, making it difficult to treat advanced sensorineural hearing loss.

However, the biotechnology company Frequency Therapeutics is a cell that is one step before it differentiates into hair cells, the inner ear.Progenitor cellsWe are trying to create damaged hair cells using small molecules that activate. “Voice perception is the number one goal for improving hearing and the number one need for patients to hear,” 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, more than 200 subjects in total received 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 improvement in hearing compared to a placebo-treated control group, Frequency Therapeutics identified this as a design issue in the study, from October 2022 to 11 During the month, we plan to conduct clinical trials with 124 additional subjects. .

“Hearing is a very important sensation. Hearing connects people to 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. have a great impact on society. »

Loose was enrolled in a doctorate in chemical engineering at the Massachusetts Institute of Technology in 2005, when he was a member of the chemical engineering department at the Massachusetts Institute of Technology.Robert LangerAt the professor’s meeting, I met David Lucchino, who was enrolled in the Sloan School of Business at the Massachusetts Institute of Technology. The two went on to launch a medical device company, Sempros BioSciences, which cost $80 million in 2012 (about 6.4 billion yen at this rate).Sold

Meanwhile, Langer, who brought Loose and Lucchino together, discovered around 2012 that small molecules that control intestinal stem cells, along with Karp, activate progenitor cells. The progenitor cells present in the inner ear differentiate into hair cells when the fetus is in the womb, but since they are dormant before birth, no hair cells are produced after birth. However, in 2012, the research team of Langer et al. 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 the patient’s cells, recombining the genes in the laboratory and returning them to the patient’s body, “the injection of small molecules into the inner ear to transform the progenitor cells into specific cells”. advantages. “Because tissues throughout the 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 previous clinical trials, subjects who had been deaf for 30 years said, “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. “It makes a lot of sense to them. Of course we need 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 frequency therapy research will improve progenitor cell activation technology and lead to the development of new treatments for deafness. With the resources and great science and technology devoted to the field of deafness treatment, Karp says that after 10 to 15 years, deafness treatment can restore vision in just 1 to 2 hours.LASIK surgery“I think we can see the same thing about LASIK surgery for deafness,” he said.

In addition, Frequency Therapeutics’ area of ​​research is not limited to the treatment of deafness, and immune cells attack nerve tissue, causing sensory and motor disturbances.Multiple sclerosisWe are also developing a cure for. In multiple sclerosis, nerve cellsAxonSurroundingsMyelinHowever, since progenitor cells differentiate into myelin-producing cells in the brain, multiple sclerosis can be treated by activating progenitor cells and increasing the rate of myelin production. Frequency Therapeutics has already confirmed the effect in animal experiments on mice, and is said to be planning to apply for clinical trials with the US Food and Drug Administration in 2023.

Karp had previously speculated that the therapeutic method of progenitor cell activation would have a wide range of applications. “Since we were considering 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 using small molecules to control local biology is just the tip of the iceberg,” Karp said.

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