Gene therapy: Local group seeks ‘answers to cancer’

There may not be a cure for cancer just yet, but the Alliance for Cancer Gene Therapy (ACGT), a nonprofit established by a Greenwich couple that’s dedicated to gene therapy, is making big strides in treatment.

The Stamford-based organization is the only nonprofit in the United States dedicated exclusively to cell and gene cancer therapy research. One hundred percent of all contributions to ACGT go directly to research and fund grants with leading scientists in the country, representing 28 prestigious medical institutions. Many feel that because of this ACGT has played a major role in what many doctors believe may be the key to healing those plagued by the disease.

The concept behind ACGT was developed in 2001 when town residents Edward and Barbara Netter attended a lecture about the beginnings of gene therapy presented by Savio Woo at Mt. Sinai School of Medicine in New York City.

In an interview with the Post, Ms. Netter said she and her late husband realized “this really is a key to perhaps finding the answers to cancer” and immediately sought to put their “energies and resources into it on hope and faith.” After a year of fund raising, the couple officially embarked on their mission to provide grants to the nation’s leading scientific “investigators” for cancer gene therapy research.

With the help of ACGT’s Scientific Advisory Council, a group composed of leading scientists and doctors who conduct rigorous reviews of grants to ensure that the most promising cancer gene therapy projects are given funding, the Netters awarded more than $22 million in grants within the first 10 years of launching their organization.

The latest strides in gene therapy projects funded by ACGT, however, are the most exciting the organization has seen, Ms. Netter said. The alliance played a significant part in the recent leukemia study pioneered by scientists at the Perelman School of Medicine at the University of Pennsylvania. ACGT provided the initial funds for the study, which has found success using immune-mediated gene therapy for leukemia and lymphoma.

According to Ms. Netter, the therapy used involves removing immune cells from the body of the patient, bioengineering and strengthening them, then reinfusing them into the patient using a gutted out HIV vector, which reprograms the patient’s immune system genetically to kill cancer. The T cells directly target and kill cancer cells and circulate through the body for at least a few years, and Ms. Netter called it “an enormous breakthrough.”

Initial ACGT grants for the immune mediated gene therapy project were awarded in 2004 to Carl June of the Abramson Family Cancer Research Institute at the University of Pennsylvania and to Michel Sadelain, of Memorial Sloan-Kettering Cancer Center, Gene Therapy and Gene Expression Laboratory, in New York City. Preliminary results were issued by Dr. June in August 2011, with additional results released in December 2012, delivering some of the most promising results seen to date in the search for a cure.

The clinical trial participants, all of whom had advanced cancers, included 10 adult patients with chronic lymphocytic leukemia who were treated at the Hospital of the University of Pennsylvania and two children with acute lymphoblastic leukemia who were treated at the Children’s Hospital of Philadelphia. Two of the first three adult patients treated with the protocol remained healthy and in full remission more than two years after their treatment, with the engineered cells still circulating in their bodies. Currently, nine out of the 12 participants show their disease in remission.

Perhaps the most amazing story of recovery, Ms. Netter said, was that of now 7-year-old Emma Whitehead, who was on the brink of death, suffering from acute lymphoblastic leukemia. After trying traditional cancer treatments like chemotherapy without any improvement, Emma’s parents decided to try the “experimental” T-cell therapy. And although the treatment nearly killed Emma, her health rapidly improved and she emerged cancer-free, Ms. Netter said. The young girl is now out of the hospital and leading the life of a regular second grader, with eight months of remission from the disease under her belt.

To the amazement of all involved, including Ms. Netter, it became apparent that immune-mediated gene therapy could be an extraordinary success in pediatrics, in addition to treating adults with cancer. With each patient, doctors are discovering more useful information, proving the Netters’ goal wasn’t merely a dream, Ms. Netter said.

The Netters always knew that funding experimental research would be an uphill battle but seized the opportunity to make a real difference, even after realizing that major pharmaceutical companies wouldn’t assist them until they had a truly marketable product, Ms. Netter said. Yet Mr. Netter, who passed away in 2011, was “so convinced [gene therapy] was the answer,” she said, adding that if he could see the success achieved in the recent University of Pennsylvania trial, “he would have a huge smile on his face.”

Perhaps eliciting another smile from Mr. Netter would be the agreement the University of Pennsylvania has reached with multinational pharmaceutical company Novartis.

The company, a major drug manufacturer, announced over the summer that it would fund additional research at the university to further study the immune-mediated gene therapies and has acquired exclusive rights to market the treatment. The hope, Ms. Netter said, is that the additional funding will allow doctors and scientists to test the treatment with other forms of the disease such as brain, breast and pancreatic cancers. Mr. Netter always believed that nonprofit organizations would work with for-profits one day, she said. “He really was a man ahead of his time.”

As for the future of ACGT, things look more encouraging than ever. The organization is currently funding 17 clinical trials in cancer cell and gene therapy targeting numerous types of the disease, including ovarian cancer, brain cancer, breast cancer, multiple myeloma, lung cancer, neuroblastoma, and prostate cancer.

Although it is unclear whether cell and gene therapy treatments will be as successful at wiping out other forms of cancer as they were with leukemia, ACGT will continue to raise money for research grants “until we really find that word ‘cure,’” Ms. Netter said. Both doctors and scientists have started to find “success they never dreamt of having” in the realm of cancer treatment and ACGT is looking forward to more “good news” in the future, she said.

ACGT is still in great need of funding. To donate, or to learn more about ACGT, visit acgtfoundation.org or call 203-358-8000.

 

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