Exhibition showcases quest to pair patients with novel search for cure


Staff Writer

People with obscure, degenerative nerve disorders with no known cures. A biologist exploring the use of fruit flies as an alternative to mice to test drug compounds. A designer who makes his own laser cutter. All of them have collaborated for a unique exhibition that could one day change the way new drugs are developed.

In a project infusing design perspectives into biosciences, Kyoto Institute of Technology’s Kyoto Design Lab invited Frank Kolkman, a Dutch designer from the Royal College of Art in London, for three months last fall to develop a prototype for a kit to test possible drugs to treat Charcot-Marie-Tooth Disease (CMT).

CMT refers to a group of nerve disorders that cause mobility and sensation disabilities over a long period of time. A rare hereditary condition, it affects 2.8 million people worldwide, according to U.S.-based patient support group CMTA. In Japan, researchers estimate at least 2,000 people have the disease.

Drug companies have to date not shown interest in developing cures for CMT, as the disease does not affect a large number of people and its impact is not as severe as other ailments, said Julia Cassim, a design professor at KIT who led the project.

Cassim matched Kolkman with Masamitsu Yamaguchi, a professor of applied biology at the university who has used fruit flies to test drug candidates. Fruit flies share 70 percent of their genes with humans, and are cheaper and easier to handle than mice, researchers say.

It was Yamaguchi who decided the project should target finding cures for CMT, Cassim said.

“He wanted to concentrate on CMT because it’s a very ‘unsexy’ disease,” Cassim said.

“Unlike AIDS or Ebola or other big illnesses, CMT is obscure. It’s a rare condition. As you go through life (with CMT), you gain disability but you are not going to die. But the problem is, there’s no cure for it.”

Kolkman, an all-round designer who can write software and also make prototypes, spent the first few weeks at KIT intensively studying Yamaguchi’s research on creating a strain of fruit flies that carry the genetic characteristics of CMT patients through gene analyses.

CMT flies move more slowly than healthy flies as they, just like humans with CMT, are physically impaired.

Yamaguchi has traditionally compared the movements of normal flies with genetically modified, or sick, flies by putting them in test tubes and shaking them to assess how fast the they can crawl back up the tubes.

Kolkman, however, came up with the idea of creating a round cassette with 20 radially arranged pockets in which normal flies and transgenic flies reflecting a CMT patient’s genes are placed in alternate rows. In the far end of each row, fly food mixed with a randomly picked chemical compound is dispensed.

Placed on a device that resembles an LP record player, the cassette then spins the flies every 15 minutes to the far edge of the rows by centrifugal force, where they are fed a variety of drug candidates. If CMT flies in particular rows become more active, it could mean those compounds worked to improve their condition.

Under the plan formulated by project members, the cassettes would be sealed, packaged and shipped to CMT patients, who could take part in the research by observing how the preprogrammed device works.

The device is also equipped with cameras to monitor the flies, with the recorded data then sent to researchers via a cloud server.

Compared with traditional drug development approaches, where candidate compounds are tested in research labs far away from patients, the screening kit delivery system would allow patients to monitor firsthand at home which drugs are working on flies containing their own genes.

Keiko Ota, a 41-year-old CMT patient from Nara Prefecture, said she found the project — though merely an idea and a business model at this point — fascinating, primarily because it would give patients a participatory role.

She is one of three CMT patients who have taken part in the project by offering early feedback on the team’s ideas.

“I found it groundbreaking that the project presented a way for patients to be actively engaged in the development of a new drug,” said Ota, who was diagnosed with CMT at age 2 and has seen her condition slowly worsen over time. In the absence of an effective drug therapy, Ota, who now uses a wheelchair, visits a rehabilitation clinic regularly to train her muscles.

“It also felt real to see how a package containing flies that carry my genes would react to chemical compounds right in front of my eyes,” she said.

For the drug-screening kit to become a reality, it needs to overcome regulatory hurdles. Currently, genetically modified animals and insects used in research cannot be taken out of labs.

Also, drugmakers need to come on board.

Cassim said the university will start marketing the idea to drugmakers, noting the system can offer similar solutions to other rare diseases that have long been neglected.

“This project centers on CMT but is asking a bigger question,” Cassim said. “How can we interest drug companies to pursue research into very obscure conditions, which are nevertheless horrible conditions?”

Designs for Flies, presented by KIT’s Kyoto Design Lab, will be exhibited at the Museum of Kyoto Feb. 18 through Feb. 21. For more information, visit jtim.es/XSrw0.