Hesperos Inc. is a small company based out of Orlando. But, with funding from the NIH NCATS center, they have made exceptional and innovative progress on a novel invention for rare disease indications. They created a very small, bioengineered model which, in 3-D, is able to mimic the biology of two rare neuromuscular diseases (multifocal motor neuropathy [MMN] and chronic inflammatory demyelinating polyneuropathy [CIDP]).

The team used the novel organ-on-a-chip model to demonstrate how different treatments could benefit these patient populations. The company submitted preclinical data to the FDA for clinical trial authorization. The organ-on-a-chip development was a huge step forward in the rare disease space. This is one of the first times this new model has been successfully used to file an Investigational New Drug Application to the FDA for a rare disease.

Considering that most rare diseases do not have an effective treatment and there are very limited animal models to study investigative treatments, these organ-on-a-chip models could be life-changing. It is incredibly promising that data using this new model was just submitted to the highest regulatory agency in the U.S., the FDA. This continues to demonstrate the potential of this therapy.

The Study

The Hesperos team was led by Dr. James Hickman. Their research was published in Advanced Therapeutics. 

The two neuromuscular diseases they included in this study both have some treatments, but they don’t have consistent therapeutic effects, and they’re certainly not a cure.

The team developed two chip models with two different types of cells. First, they created one using motoneurons which are responsible for transmitting messages from the brain to the muscles. Secondly, they created one using Schwann cells. These cells help the messages move at a faster pace from the brain to the muscles.

Therefore, the models will be able to help scientists understand if a treatment can help the transmission of these messages as well as the pace of these messages.

In the study, the team demonstrated that blood from patients with these rare neuromuscular diseases does lead to an immune system response which causes the signals to move very slowly. With the addition of a TNT0005 treatment, the immune reaction was blocked and the signaling of messages increased in pace.

Looking Forward

Right now, even rare disease clinical trials which have animal models rarely end successfully because well, animals aren’t people. Animal models don’t always tell us how real, human patients will respond to a drug.

Using the novel tissue chips, which use real human cells, we can better understand how a therapy may work in humans earlier on. Additionally, they can help us learn which specific patients may be more likely to respond to a specific treatment.

Not only does this provide us better information about potential treatments, but it minimizes risks to patients which can come from putting them in clinical trials which they aren’t suited for.

You can read more about this latest study here.