During the past fifteen years there has been ever-increasing interest in scientific research in the role of microbiome in human health.
According to a recent article in Forbes India, this state-of-the art research is on target to deliver treatments for a wide range of illnesses. A microbiome-derived drug is now being developed to treat childhood asthma.
Other illnesses that may be targets for microbiome therapies are autism, cancer, digestive ailments, and obviously food allergies.
In fact, over fifty thousand scientific papers have been written about the effect of microbiomes in the past five years. Certain types of gut bacteria appear to stimulate or suppress the body’s immune responses. Others have been found to fend off microbes that cause disease.
Earlier Test Results with Mouse Models
In 2018, three studies provided evidence that intestinal microbiome affects the outcome of melanoma. Results of one study showed that patients with the most varied microbiomes responded most favorably to immunotherapy.
Other findings involved lean mice that gained weight after they received gut microbiome cells from obese mice.
In another test, mice that were injected with gut bacteria taken from human marathon runners were able to run long distances.
About Clostridium difficile (C. diff)
The disease affects a half million people in the U.S. each year, with approximately fifteen thousand deaths. Once these statistics were published in the New England Journal of Medicine in 2013, investors came to the rescue.
The results were astounding. Ninety-four percent of C. diff patients in a randomized trial recovered following fecal transplants.
A Novel Bank
Mark Smith, co-founder of the promising startup Finch Therapeutics, describes being unable to help a C. diff patient when he was a microbiology grad student.
As a result, he created OpenBiome, a non-profit that is similar to a blood bank but instead supplies human feces for transplantation. To date, the bank has supplied stool to more than 1,200 hospitals that performed over 53,000 fecal transplants.
Smith and his company have partnered with Takeda, the pharmaceutical giant, in an effort to develop drugs for Crohn’s and ulcerative colitis disease. A total of ten million people worldwide are diagnosed with these diseases.
Smith says that his company is using a technique called “reverse translation”. Instead of beginning their research using animal models, Finch is using a “human-first” technique. The researchers analyze the stool of patients after they have received fecal transplants and have recovered.
A Full-Spectrum C. diff Capsule
Smith hopes to have a C. diff capsule by mid 2020. He calls it a “full-spectum” of bacteria from a human stool sample taken from a fully-recovered patient.
The sample is freeze-dried and considered a fecal transplant but actually delivered in a single pill.
The company is also in the process of developing a drug to treat autism. More than one million people are diagnosed with autism in the United States but to date there are no drugs that treat the disorder.
The Gut-Brain Axis
Sarkis Mazmanian, at Caltech, is perhaps one of the most talented microbiologists in microbiome research. He is said to be exploring the “gut-brain axis.”
In 2008 Mazmanian and his Caltech colleague turned their attention to autism and the need to develop a treatment for the sixty percent of autistic children who experienced digestive problems. Mazmanian had already received recognition for treating IBD in mice with human bacteria from the gut.
Mazmanian’s partner succumbed to brain cancer in 2014. He continued their research at Caltech working with one thousand germ-free lab mice.
The mice were kept in sterile conditions encased in rectangular bubbles. They were fed food with gut microbes added. The effect produced tremors and other motor problems that conformed with human Parkinson’s symptoms.
In 2016, Mazmanian was approached by a venture capital company, Axial Biotherapeutics, that raised millions for his project. Axial is developing synthetic drugs composed of small molecules that absorb gut-bacteria byproducts (metabolites) that promote symptoms of autism.
The company is also developing a drug that will treat the digestive problems associated with Parkinson’s.
Funding From the Bill & Melinda Gates Foundation
Bernat Olle, an MIT Ph.D, founded the microbiome drug developer Vedanta Biosciences based in Cambridge, Massachusetts. The company has accrued funding of $112 million which includes ten million from the Gates Foundation.
Approximately 200 million children in developing countries are suffering from malnutrition with 1.5 million deaths each year. The Gates Foundation has invested in Vedanta’s efforts to develop a bacteria-derived drug to defeat childhood malnutrition.
Another finding is that malnourished children, even when fed properly, cannot gain weight. The premise is that their gut microbiota have developed abnormally. Researchers believe that treating the children with strains of beneficial gut bacterial may correct the imbalance.
Billions Pouring Into Microbiome Research
There are estimates that beginning in 2014, over five billion dollars have been invested in microbiome research. Mark Zuckerberg, Bill Gates, and Vinod Khosla, are among the many investors who are supporting microbiome research and funding startups.
A New York biotechnology analyst recently commented that if any of the microbiome projects are successful, the impact on the drug markets for most of the major diseases would translate into billions.