Small Biotech Breaking Down Barriers in Autism Research

The future of autism treatments is increasingly growing more optimistic.  Biotechnology companies have seen a shift in focus from merely treating the symptoms that result from autism to more diligent research attacking the core issues in an attempt to hopefully one day eradicate the disease. 

While that possibility is still many years away, the reality of new data being compiled daily by pharmas, both large and small, to develop new therapies is here today.

Autism research is moving to the forefront in the biotechnology industry. With the cases of autism being diagnosed faster than any other developmental disease today, the importance of new research is imperative. There have been a number of reasons attributed within the industry to the lack of new developments in autism research.

One of the main reasons was the debate over what causes autism. While many people will argue that vaccines were a primary cause, this concept has been debunked when a special United States court ruled earlier this year that there was no conclusive evidence of a correlation between vaccines and autism rates. The American Medical Association, the American Academy of Pediatrics, the American College of Medical Toxicology, the National Academy of Sciences, the U.S. Food and Drug Administration, the Center for Disease Control and Prevention, and the World Health Organization all came out against the purported link between autism and vaccines as well. The research by Dr. Andrew Wakefield, the man who sparked the controversy, has never been replicated or proven to have any serious substance within the biotechnology industry. While this debate has lessened in intensity, the causes of autism still remain a mystery. Research has revealed several physical characteristics in the brain that run true through those stricken with the disease and it is this data is helping pave the way for the new generation of autism research and possible future treatments targeting the core issues.

A second reason has been the inability to define a reliable animal model to study. Serotonin depletion and decreased brain plasticity have been proven to be common features of the autistic brain, but replicating these characteristics has been a significant challenge. Cellceutix Corporation, a small biotechnology company that trades on the Nasdaq Bulletin Boards under the ticker CTIX, has recently become the first company to overcome this obstacle. Through injections of 5,7-dihydroxytyptamine (5,7-DHT) into the forebrain bundle on the day of birth of Wistar rat pups, Cellceutix researchers were able to chemically reproduce these key parameters of serotonin levels and plasticity. The rats were then orally treated with KM-391, a novel compound being developed by Cellceutix, for up to 90 days. The data collected showed significant increases in both serotonin levels and brain plasticity that more resembled that of a normally functioning brain. Now that a reliable animal model has been established for the first time, Cellceutix continues to move forward with their research and development of KM-391 and has released positive data in their research related to behavioral symptoms of autism being modified through KM-391 therapy.

While Cellceutix is in the forefront of the pack in revolutionary autism research, they are not alone. Large pharmaceutical companies have also realized not only the humanitarian benefits of the successful development of a compound focused on the core issues of autism, but also the financial benefits. With estimates for potential revenues ranging well into the billions of dollars per year, industry giants Novartis (NYSE: NVS) and Pfizer (NYSE: PFE) are also pursuing research in this area. Novartis has re-applied an existing compound overseas in a very small study targeting Fragile X Syndrome, but has publicly stated that, while optimistic, the study was small and requires much more extensive research to draw definitive conclusions. Pfizer has not disclosed research information, but announced last year that they have assembled a team of researchers to delve deeper into researching the root of autism and a possible course of action in developing new treatments.

Where autism was observed in 1 out of every 1,000 births ten years ago, its diagnosis rate has escalated dramatically recently to 1 out of every 110 births. It will affect more children this year than AIDS, cancer and diabetes combined. It is an area that has an extremely high level of unmet needs in the industry and requires even more attention that it is receiving. Whether a successful compound is developed by Cellceutix, Novartis, Pfizer or any other biotech that surfaces, these latest developments are providing a new hope for all those affected by autism. Even at expedited rates with no significant complications, human trials are still at least a year away, but the autism community is already showing support of the possibilities as researchers strive to develop their compounds.  The direction of focus has shifted and the research has begun, now it just will takes some time and support to one day have some viable options other than what is available today.

BiomedReports is not paid or compensated to report news and developments about publicly traded companies. Full disclosure can be read in the About Us Section

---

Add this page to your favorite Social Bookmarking websites