The microscopic particle with limitless potential
In the film Extraordinary Measures, venture capitalist John Cowley (Brendan Fraser), with two of his children suffering from Pompe disease—which causes muscular atrophy and in its extreme form, respiratory failure and death—races against time to raise millions to further the research for a cure.
He consults Dr. Robert Stonehill (Harrison Ford), who has developed a therapy that employs a replacement enzyme, allowing the body to process stored sugar so that muscle, heart and liver functions improve. The company they start is bought by a large biotech company, and the children's lives are saved. The film depicts the real life story of John Cowley and his biotech firm Amicus.
In an exciting sequel to this story, Dr. Sathy Balu-Iyer, a researcher at the University at Buffalo, has developed proprietary nanoparticle technology that provides even more hope to sufferers of unwanted immune response against life-saving protein drugs used to treat ailments such as Pompe disease, hemophilia and other autoimmune disorders.
In the past, if someone developed one of these disorders, once their body rejected the treatment due to the development of antibodies that attached to and neutralized the protein drugs, they were out of options other than palliative care. Now, when a healthy treatment enzyme is delivered via the nanoparticle, the body accepts it as native, suppressing an immune response and allowing sufferers to live healthy lives.
Dr. Balu-Iyer developed the technology four years ago, after finding that the same cell membrane material—phosphatidylserine—that triggered the body to clean up dead cells also taught the immune system to tolerate it. He found that therapeutic proteins could be delivered via the nanoparticle, giving the body the ability to reduce, prevent and reverse an unwanted immune response, since the body recognizes it as “self.”
The nanoparticle has applications ranging from autoimmune suppression to gene therapy. For example, people with hemophilia A lack a blood-clotting agent called Factor VIII. They may suffer bleeding in the joints and GI tract and can become severely debilitated. The first line therapy for this disease is protein replacement using recombinant Factor VIII. Up to thirty percent of patients can develop an unwanted immune response in the form of the development of antibodies, leaving them with no treatment option.
Dr. Balu-Iyer has found that administration of nanoparticles containing Factor VIII to a preclinical hemophilia A model helps inhibits the production of antibodies against treatment and maximizes the effectiveness of this critical therapy. In addition, this nanoparticle can also reverse an unwanted immune response.
“Unlike conventional vaccination, this new technology, called 'reverse vaccination,' selectively desensitizes the human immune system to a therapeutic protein,” Dr. Balu-Iyer says.
Dr. Balu-Iyer was originally inspired to begin his research by reading a magazine article while on a flight to an American Association of Pharmaceutical Scientists conference. The article was written by the mother of a boy who had run out of treatment options for his hemophilia.
The extensive research behind the nanoparticle was funded by grants from the National Institutes of Health, the National Heart Lung and Blood Institute and SUNY's Technology Accelerator Fund (TAF).
TAF helps faculty inventors and scientists along the curve of transforming their research into market-ready technologies. In Balu-Iyer's case, the TAF investment will enable his research team to conduct preclinical “proof of concept” studies to prove the nanoparticle's effectiveness in gene therapy and to meet the scientific requirements of several potential partners in the pharmaceutical industry.
Dr. Balu-Iyer's patent for the nanoparticle technology is licensed to Amherst, NY-based Zoetic Pharmaceuticals, founded by John J. Seman, Chief Executive Officer, and Sven A. Beushausen, Chief Scientific Officer, in 2014. Zoetic is a start-up that collaborates with the University at Buffalo and hopes to employ immune tolerance technologies such as the nanoparticle to reduce reactions to therapeutic proteins, alleviate autoimmune diseases and improve gene therapy. They are currently seeking to develop partnerships with large pharmaceutical firms, among them John Cowley's Amicus.
“This product represents a new strategy for tolerance induction for hemophilia A patients that could significantly reduce costs and improve their quality of life,” says John Seman. “We believe the hemophilia community, and the health care system, will welcome a new approach to addressing one of the most serious and costly complications of managing hemophilia.”
One of Dr. Balu-Iyer's main goals, like John Cowley, is to help give treatment, and hope, to sufferers of debilitating autoimmune diseases like Pompe disease, rheumatoid arthritis and celiac disease who have run out of options. This exciting story is still in its fledgling stages, but with some more research, development, and the proper marketing and placement via Zoetic, this story, like the movie Extraordinary Measures, should have a happy ending, where autoimmune sufferers can live normal lives.
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