The genetic code that forms the basis of all life is remarkably composed of just four components, or bases: adenine, cytosine, guanine and thymine, or A, C, G and T.
By manipulating that genetic code, researchers backed by the nonprofit Alliance for Cancer Gene Therapy — ACGT for short — are making headway against acute forms of cancer that patients have fought for years, often in the face of overwhelming odds.
Stamford-based ACGT was formed in 2001 and began funding research and clinical trials in 2002.
In just over 10 years since, the group has received about 780 grant requests from 166 scientific institutions, awarding 39 grants totaling $23 million to researchers studying gene therapy as a method of attacking multiple types of cancer.
In 2004, one award went to Dr. Carl June, who leads a research team at the Abramson Family Cancer Research Institute at the University of Pennsylvania.
June and the university in August 2011 issued preliminary results of a study that aims to show whether genetically engineered T-cells, a type of white blood cell, can be used to defeat cancer, with additional study results released last month.
Out of the 10 adults and two children who participated in clinical trials administered by June’s team – all of whom had advanced forms of leukemia – nine currently show their disease in remission. Two out of the first three patients who were treated remain healthy and in full remission after more than two years, according to ACGT.
The sample size is small, but the results are “overwhelming” nonetheless, said Barbara Netter, president and co-founder of ACGT.
“As it turned out, Dr. June made an enormous, stunning breakthrough in lymphocytic leukemia,” Netter said.
That more than two-thirds of the participants are in remission “is a huge thing, and that is proof of concept,” Netter said. “And we are very excited about it because we gave them the seed money and set them on this course.”
One of the world’s largest biopharmaceutical companies, it seems, agrees.
Novartis A.G., which is based in Switzerland and has its U.S. headquarters in East Hanover, N.J., has committed $20 million to the construction of a research center on the University of Pennsylvania campus with the goal of bringing the gene therapy treatment to market.
Now, Netter said of the researchers who have teamed with ACGT, “They all seem to be putting their heads around, ‘Where next, what next?’”
Netter and her husband, Edward, who passed away in 2011 after serving in executive roles with Geneve Corp., Independence Holding Co., and The Aristotle Corp., founded ACGT after their daughter-in-law died from breast cancer.
“We, like many others, felt very helpless,” Netter said. “We decided that we would venture and make this something we would work toward. Every little step is important.”
Now, she says, “We are the only institution that concentrates exclusively on cell and gene therapy, and all the money we take in goes directly to research.” She said fundraising efforts started slow, with the couple contributing some capital of their own, but that the recent successes of several studies funded by ACGT have led to increased support from donors.
“As more people hear about it, we have continued to grow it,” Netter said. “Individuals, companies, corporations — we do have a following now.”
Netter said she is hoping to carry on and build the organization in her husband’s stead.
“We wouldn’t be at this point if not for my husband,” she said. “I’m trying to carry out his legacy.”