Health care innovations: good therapy for Ohio and beyond

From the futuristic enclaves of genetic engineering to easing the administration of medicine, health care innovations abound across the Buckeye State. Some aim to help patients daily in gentle ways while others are waging battles on the front lines against disease.
 
The future of gene therapy: disease-fighting factories
 
Therapeutics is the medical treatment of disease. However, the "science of healing" an illness does not always hit its target. Cleveland's Copernicus Therapeutics, Inc. asks what would happen if DNA could be delivered via a non-viral delivery system? What if patients served as a kind of factory for their own disease-fighting agents?
 
The biotechnology company has established a platform that makes the process of gene therapy effective and safe, says CEO and President Dr. Robert Moen. The company's products treat cystic fibrosis, retinitis pigmentosa, macular degeneration, diabetic retinopathy, Parkinson’s disease and cancer.
 
As Moen explains, Copernicus can inject DNA into the back of an eye to remedy genetic mutations that cause blindness. Compaction of single DNA molecules produces a stable gene transfer system, enabling the efficient uptake of drugs by specific cells and tissues in the eye. Through this "blueprint," cells then make the proper protein to allow them to work, eliminating the mutation and restoring the patient's vision.
 
This form of gene therapy has only been tested in mice, but the company CEO points to the possibility of a special DNA-delivering nose spray that could one day treat Alzheimer's disease and other neurological ailments without the need for surgery.
 
"The DNA goes into the brain through the nose, and the brain can make the necessary proteins itself," Moen says.
 
Copernicus was founded in 1995 by three faculty members at Case Western Reserve University, receiving funding help from the technology-based economic development initiative Ohio Third Frontier and various foundations looking to harness gene therapy for neurological diseases. It is now located in the BioEnterprise business incubator in Cleveland.
 
The marketplace for the technology is unstable due to the risk factors involved with gene therapy as well as the expense of clinical trials on humans. Still, the non-viral approach to delivering therapeutics continues to draw funders due to its potential. Unlike a viral method, the company's gene therapy will not be staunched by virus-blocking antibodies in the bloodstream.
 
Moen, who has a biochemistry degree from Harvard University, believes this science can be harnessed to treat the otherwise untreatable. "It's an exciting field," he says.
 
Creating genes in the fast lane
 
Athens-based First Biotech Inc. (FBTI) is not directly involved in gene creation, but the startup's founder believes he has discovered a way for others to construct new genes swiftly and efficiently for applications in medicine and more.
 
FBTI produces innovative reagents for research in biotechnology. The process, called Unrestricted Mutagenesis and Cloning (URMAC), is designed to save the time and effort associated with building new genes. Company founder Louay Hallak developed the process. 
 
Hallak's background as a research scientist motivated him to discover the technique that sped up the usually sluggish gene creation process. Since its inception February 2011, FBTI has manufactured a DNA mutagenesis kit that can be used to accelerate the creation of protein therapeutics and viral vectors for the treatment of genetic illnesses like multiple sclerosis.
 
The kit circumvents the time-consuming sub-cloning process that transfers a cloned DNA fragment from one vector to another. This allows for faster biochemical reactions and direct manipulation of large DNA sequences in a matter of days instead of the weeks or months common with the existing technique. The process can also be applied to agriculture for improving crop yield or the quality of grain.
 
"We're trying to learn the function of these genes," says Hallak. "We take the genes out, put it in a vector, then try to correct the genes through the vector."
 
FBTI is currently operating out of Ohio University’s Innovation Center. The company received pre-seed funding approval from TechGrowth Ohio, in addition to an earlier grant from TechColumbus.
 
Hallak, a Columbus resident, has spent the last two years improving his technology. Beta testing resulted in positive feedback, and FBTI is now talking distribution with companies in the U.S. and abroad.
 
"Right now we're just setting foot in the market," he says, "then we'll expand our customer base."
 
'Pharm'-ville
 
On the more conventional side of the equation, P&C Pharma out of Columbus aims to provide personalized medications for easier patient administration. The tech venture wears its health care aspirations on its sleeve; P&C stands for Patients' and Consumers'. Patients are one group that founder Joe D'Silva believes has been largely ignored when it comes to easy dosing of their medicines.
 
D'Silva is a native of India who received his Ph.D. in pharmaceutics from The Ohio State University. He founded P&C in 2000, and now shuttles between his home in Philadelphia and the TechColumbus Business Incubator where his company is headquartered.
 
His two-person operation is built on one key question:  "How can you give a person the exact medication they require at the right time and dose they want?" D'Sliva asks rhetorically.
 
Trouble-free administration of medications and hard-to-swallow vitamins is a topic D'Silva had been thinking about throughout a 20-year career in pharmaceuticals. He witnessed numerous cases of patients unable to ingest capsules, with their plight combated by a pharmacist who would crush tablets into a liquid using a mortar and pestle. The solution, D'Silva says, was ineffective, as a mortar and pestle technique does not take into account texture, flavor or even proper dosage of the medication.
 
Enter P&C’s first invention - the INSTA Formulation System. The automated device compounds tablets and capsules into flavored liquids, easing the lives of patients that cannot swallow pills and have no access to appropriate liquid products. This is particularly important to someone with Parkinson's disease, which can effect swallowing due to loss of control of the mouth and throat muscles, notes D'Silva. P&C's technology navigates this by turning pills into a nectar-like liquid that Parkinson's patients can swallow without aspirating.
 
Ultimately, the INSTA system "changes the pattern of how people can be looked after," D'Silva says. "They can get therapy in a holistic fashion."
 
The product is for use by pharmacists and nurses at the point of care, and has been granted patents in the U.S., Europe., Canada and Japan. D'Sliva's device applies to our four-footed friends well, with veterinarians able to use the technology to care for their animal patients. P&C is currently raising funding after receiving grant money from the states of Ohio and Pennsylvania. D'Silva is confident his creation can take root in the Buckeye State.
 
"We are personalizing these medical products," he says. "This is a very unique niche."