By Elizabeth Simpson, elizabeth.simpson@pilotonline.com, The Virginian-Pilot
Sarah Piscitelli had barely gotten used to the idea she had an incurable disease – diabetes – before she was sitting in a Norfolk clinic having an experimental solution infused into her vein.
Was the liquid – in an opaque IV bag so she couldn’t see it – a potent mixture of stem cells or a powerless placebo?
She won’t find out for two years, but what she does know is she’s part of a clinical trial that has the potential to change lives.
The 23-year-old Virginia Beach woman was older than the norm when she was diagnosed in February with Type 1 diabetes, the less common variety usually diagnosed in childhood.
The timing of Piscitelli’s diagnosis placed her in a position to enroll in a national clinical trial to see whether infusions of stem cells can kick-start her body’s ability to make insulin.
She’s one of 60 people across the country who will test the idea. If successful, it could eventually change the lives of people in the early stages of the disease. Some 30,000 a year are diagnosed with Type 1.
She received her third and last infusion at an Eastern Virginia Medical School clinic Friday. The amount of insulin she’s had to use each day since the first dose in April has dropped.
But here’s the rub:
Neither she nor the researchers will know whether she’s getting the real deal or a placebo until the end of the two-year study.
Does she need less insulin because she’s in the so-called “honeymoon” period that new diabetics often experience after starting insulin?
Or are stem cells doing the hoped-for job of rejuvenating her pancreas?
“My mom tells me to pretend it’s the real thing,” she said, “because positive thinking itself can help.”
The study is being conducted by a Maryland company called Osiris Therapeutics that is developing stem-cell therapy to treat ailments such as Crohn’s disease, arthritis and heart disease.
The injectable solution the company is testing – Prochymal – is made of stem cells from bone marrow donated by adults, not human embryos.
The Strelitz Diabetes Center at EVMS is one of 20 sites recruiting volunteers with Type 1 diabetes for the trial.
Dayna Buskirk, director of clinical development for Osiris Therapeutics, would not reveal how many of the 60 are enrolled so far, nor how much the study costs.
However, the Juvenile Diabetes Research Foundation has seen enough promise in the idea that it donated $2 million to the effort last June.
Typically, if such a trial shows good results, it would be followed by another phase that would involve a larger number of people at even more clinical sites.
In Type 1 diabetes, a person’s immune system attacks and destroys insulin-producing cells in the pancreas. Insulin controls blood sugar, and poorly controlled levels can lead to heart disease, stroke, blindness, amputations, kidney disease and nerve damage.
Stem cells have characteristics particularly promising in treating autoimmune diseases such as diabetes.
One, they have the ability to transform into any type of tissue without the body rejecting them, and two, they travel to sites of inflammation. Once they get there, they can prevent further damage and even repair tissue.
“They sense where in the body something is going wrong, and they move in to see what they can do,” said Dr. Aaron Vinik, the Strelitz center’s scientific director, who’s overseeing the local part of the study. “If they find inflammation, they can mend the process if it’s early enough. They’re like subterfuge cells. They get in under the tent flap, and the body doesn’t recognize them as foreign.”
The trick is to call in those cellular troops soon enough. If it’s too late, the insulin-producing cells have already been destroyed.
The study is looking for people who are within two to 16 weeks of diagnosis. The peak age for diagnosis of Type 1 diabetes is 14, but because of consent and safety issues for still-developing children, the researchers are restricting this phase of the trial to adults 18 to 30.
Click link above for complete article.