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April 14, 2008

Worcester's Low-Tech Legacy Reaches Into The Future

Local biotech co. develops simple treatment with a complex twist

There has been a lot of enthusiasm and excitement about the growing concentration of biotech companies in the Worcester and MetroWest areas, with much of the attention based on the additional jobs and economic development they bring.

A focus on the business aspect of biotech is certainly justified, perhaps even more so these days as the dreaded "R" word rattles the doors and shakes the window panes of our economy. But the medical and biotech solutions these companies have already developed or the ones on their drawing boards should excite us just as much.

Think in terms of Robert Goddard's rocket experiments smoking from the basement of the Worcester Polytechnic Institute's physics building in 1907, or the development of the birth control pill in the 1950s by the Worcester Foundation for Experimental Biology in nearby Shrewsbury.

Worcester also birthed more utilitarian objects like the monkey wrench, the envelope folding machine, extruded steel wire (read barbed wire among other things) and lunch wagons that eventually became the diners we know and love.

In the past, Worcester was known as a place where new things were invented and made. And just as the area was the scene for these earlier innovations, it will likely be home to some amazing scientific developments as the years go by.

Imagine someday being able to take the specific DNA or RNA you lack, encapsulated in a natural substance, like yeast for example, for diseases such as asthma or rheumatoid arthritis. Or a pill made of RNA-interference packaged in yeast that could help silence a gene that caused a disease. Many people with severe asthma who are on a steroid-based regimen or those who suffer the pain and limited movement of rheumatoid arthritis would gladly swap the drugs they take for DNA in a yeast pill.

Taking Aim


Well, those are the future plans of start-up Targeted Cell Therapies LLC, a Worcester biotech company, although first it is working on a remedy and a possible cure for Gaucher's Disease. People affected by this disease lack an enzyme that breaks down a specific lipid, causing it to build up in the organs, bone marrow, brain and lungs. The most common manifestations are bone erosions and fractures.

Eric J. Evans, the company's CEO, said the company's founder Dr. Edward Ginns, a doctor at the University of Massachusetts Medical School with a practice that treats patients with lysomal storage and other diseases, has been working on using synthetic human DNA in Brewer's yeast so those with the disease will be able to take a pill that helps their bodies make the enzyme.

At some point, the company hopes to be able to teach the cells to make the enzyme on their own. After it perfects its answer to Gaucher's Disease, it will focus on osteoporosis. But for now, it's one step at a time.

The company has already been successful in using human DNA in mice with Gaucher's Disease, and soon it will begin clinical trials on humans. It hopes to be able to make the product available in about a year.

Of course more funding must be found: the company already received $500,000, but is looking for the next level of venture capital funds. The market for the drug is truly big business, although Evans said the startup will probably charge less than, say, Genzyme Corp. does for its Gaucher's Disease product, Cerezym.

Only 20,000 people world-wide are affected by the disease, but Genzyme recently forecast its sales of Cerezym in 2008 would be between $1.22 and $1.24 billion. That's compared to $1.13 billion in 2007. Genzyme is also partially located in MetroWest with three facilities in Framingham and one in Westborough.

Heck, there's even another biotech company here in Worcester whose mission is coming up with a solution for Gaucher's Disease: Glucadel Inc., with Dr. Gary Ostroff, who worked on the yeast delivery ideas with Ginns.

So as the biotech economy grows, so should the enthusiasm for the brain power and the answers developed for medical issues.

It would be pretty cool if Worcester becomes known outside the area once more as a hotbed of innovation and the home of answers for a better life.  

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