You might be surprised to learn that only one percent of grant funding from the National Institutes of Health [NIH] is awarded to young researchers. In contrast, ACGT uses half of its funding for clinical trials and half of its funding goes to young scientists exploring revolutionary approaches to cancer treatment.

We are second to none in our admiration for the NIH, the world’s largest source of funding for medical research; however our ACGT Research Fellows concur with the general consensus that the red tape can be crippling and good research goes begging for support.

To make matters worse, public funding is a classic chicken and egg model: researchers have to prove their hypotheses before they are considered for NIH grants, but where will they find the financing to compile that data? Universities will fund a study for just so long before they too need an infusion of capital.

As a result, great minds are increasingly hesitant to commit to research and the brain drain will ultimately cost lives. Too many young scientists also face the denial of tenure if they cannot publish. Yet discovery laboratory research, which ACGT has emphasized from day one, lays the groundwork for most medical breakthroughs and the foundation for clinical trials.

A recent article in Philanthropy Magazine by Karl Zinsmeister, founder of the Almanac of American Philanthropy, endorsed the importance of private giving to ensure new forms of medicine. We couldn’t agree more. Discovery research has led to more innovative and effective treatments for cancer, and the need for funding is greater every day.

Zinsmeister also pointed out that donor-funded scientists produce high-impact papers at a much greater rate than similarly accomplished NIH-funded researchers, as reported by the National Bureau of Economic Research, because “philanthropy tolerates early failure, rewards long-term success and gives its appointees great freedom to experiment.”

Philanthropists have funded the most important medical breakthroughs of the 20th century, for example:

• Immigrant physician George Papanicolaou, in 1928, sponsored the development of the lifesaving test for cervical cancer, despite the disdain by the medical establishment.
• The John Hartford Foundation, created from the A&P supermarket fortune, was the leading funder of biomedical research from the 1950s to the 1970s
• And let us not forget the foundation of the Sloan Kettering Institute in 1956 by inventor Charles Kettering.

The trend continues in this millennium.

• Microsoft’s co-founder Paul Allen created a new lab with an initial $100 million gift to better understand how living cells relate to each other.
  Allen’s compatriot, Bill Gates, has given millions to combat worldwide disease.
• Internet entrepreneur Sean Parker donated $250 million from his Facebook proceeds to back cancer immunology. Parker demanded collaboration between six top cancer centers to improve the odds of success. ACGT’s funded scientists lead a third of the Parker Institute for Cancer Immunotherapy centers.
• Costco’s Sol Price funded research at Caltech to sequence DNA, which made the mapping of the human genome, and precision medicine, possible.
  Michael Milken founded the Prostate Cancer Foundation to support risk-taking research to achieve results sooner than later, and that success inspired FasterCures, to accelerate progress through greater efficiency.

Ratings for charitable organizations make it possible to ensure greater odds for a good return on investment. Each donation to Alliance for Cancer Gene Therapy goes directly to research because a separate fund supports administrative expenses. In contrast, university labs are burdened by overhead and onerous paperwork. Our prestigious Scientific Advisory Council makes certain that grants are awarded to the most promising researchers in North America, with few strings attached other than the dedication to results and proper reporting on progress. It is the generosity of ACGT donors that allow us all to truly fund miracles.

Giving, in the right way, makes miracles happen. Read more at Philanthropy Roundtable.

This fourth in our blog series on the progress of cell and gene therapy for cancer focuses on a treatment that has made huge strides in recent years: Viral Therapy.

What is Viral Therapy?

Using viruses to target disease is a science first contemplated over a hundred years ago, but was not possible until the technology advances we have now that enable genetic engineering. Oncolytic Viral Therapy [OV] made news last year at Duke University when a trial using a modified polio virus effectively destroyed Glioblastoma, a deadly brain cancer. Half the patients, in advanced stages, found themselves in remission. In other studies, a measles virus destroyed severe blood cancer and the maraba virus, related to rabies, reduced the size and spread of melanoma, lung and colon cancers, as well as select brain tumors.

How can a virus fight cancer?

You may be thinking: why would I purposely insert a virus into my body? Viruses are harmless once they are decoded, and there are over 3,500 known viruses with the potential to be therapeutic agents. In laboratory research, viruses proved their ability to spread curative agents rapidly in cancer cells and, at the same time, signal the immune system of a threat, so the body responds with its own army of killer T-cells.

ACGT and OV

Dr. John Bell, ACGT Scientific Advisory Council member, and Research Fellow,  reported in April at the ACGT Scientific Symposium that viral therapy is a powerful 1-2 punch. “The virus attacks the tumor and the immune system comes in to finish the job.”

According to ACGT experts in the field, oncolytic viruses have an exemplary safety record. One of the greater success stories was a 49-year old patient at the end of a 10-year battle with myeloma who has been in remission for 2.5 years after the direct injection of a modified measles virus.

Dr. Bell and his colleagues at the forefront of OV describe viruses as “exquisitely selective” in targeting cancer cells and they are easily engineered to carry corrective genes to destroy or repair damaged genes.

Challenges and Potential

The great challenge ahead is to match the right virus to the cancer, as well as learn how to mass manufacture viral agents to treat large numbers of patients. Perhaps the greatest obstacle researchers face is that many tumors contain sensors that trigger an anti-viral response, to defend against the therapeutic influence of the virus. As we’ve reported before, cancer cells are tenacious in protecting themselves from interference from all types of treatment, including cell and gene therapies; however scientists are closing in on ways to destroy the cancer line of defense.

This science is moving rapidly. Researchers throughout the world are examining the intricate differences between types of cancers to ensure a virus’ ability to penetrate and replicate. At the same time, biotech and pharmaceutical firms are looking closely at the potential for this revolutionary treatment. Novartis, one of the largest drug companies in the world, is backing a therapy using an HIV virus with the hope of FDA approval within the year, and trials using Amgen’s Imlygic, an OV treatment for metastatic melanoma, used in combination with the chemotherapy drug Yervoy, improved life span. Stay tuned!

“There’s no question this field has altered the trajectory of cancer research – we’re moving towards a biologic approach and away from drug and radiation treatment.”    Dr. John Bell, Ottawa Hospital Research Institute

 

As we celebrate this wonderful season of giving, we want to thank all of our donors for helping support ACGT’s breakthrough research.  And we want to thank our researchers and their patients who are continually moving cell and gene therapies for cancer to the forefront of innovation in the treatment of this deadly disease.  2015 has been a wonderful year full of outstanding cell and gene therapy progress, and there is much to celebrate.  We continue to see more successful results with the use of T-cell therapy for the treatment of leukemia in both adult and pediatric patients.  Many of our researchers continue to achieve life-saving results including Dr. Carl June, Dr. Michel Sadelain, Dr. Laurence Cooper, Dr. Thomas Kipps to name a few. 

2015 has also been the year of oncolytic viruses (OV), as the FDA recently approved the first OV therapy for the treatment of blood cancer.  Many of ACGT’s scientists, including current ACGT grantee Dr. John Bell, Ottawa Hospital Research Institute have pioneered this gene therapy.  We have also witnessed the emergence of gene editing, and in London, it was successfully used on donor T-cells to treat a little girl in London who was dying of leukemia and is now cancer-free.   And just this month, suicide gene therapy was use to successfully kill prostate tumor cells.

All of these powerful therapies are being researched by ACGT Research Fellows and members of our esteemed Scientific Advisory Council.  But there is still much to do.  These therapies are still in their infancy and  need more support to get them into more patients, while keeping the costs of production down.  And we need more support for ACGT’s Young Investigator program which supports newly minted Assistant Professors conducting novel independent basic science using cell and gene therapies.

Again, thank you to all and best wishes for a happy, healthy 2016. 

 

 

In China, a patient whose life was close to its end was completely cured of leukemia by stimulating the immune system using CAR T-cell therapy. In the first study of its type, all cancer cells quickly disappeared. Immune-mediated treatment, pioneered in this country by ACGT scientists Dr. Carl June, University of Pennsylvania, and Dr. Michel Sadelain, Memorial-Sloan Kettering Cancer Center, among others, is rapidly proving more and more successful with thousands of patients in human trials.

A middle-aged woman is said to have been cured of leukemia after she was treated with CAR T-cell therapy. “The cancer cells have disappeared from her body. She is the first patient who has been completely cured of the disease using gene therapy,” said Professor Qian Cheng, director of the Bio-Treatment Center at the hospital in the city of Chongqing.

China has about four million patients that have been diagnosed with leukemia. Most patients are treated using chemo-therapy or bone marrow transplants, but CAR-T treatment is a gene therapy using modified T cells to fight cancer cells in leukemia patients. “CAR-T therapy is a much better option, because it can cut costs by at least 30 percent compared with bone marrow transplants, and is more likely to lead to a cure,” Professor Qian said

Six other patients, who are receiving the gene therapy in the same hospital, have shown improved conditions, according to Professor Qian. CAR-T gene therapy is still in the clinical trial phase in China and has only been administered in fewer than ten hospitals across China. Qian said his team is encouraged by the success and will further study dosing to improve the new therapy. .

The University of Texas’ MD Anderson Cancer Center — one of the most esteemed cancer laboratories and centers in the world — is in the vanguard of advancing and unlocking the amazing abilities of immunotherapy. And recently, MD Anderson Cancer Center’s work earned major plaudits and support: The hospital was awarded $100 million for its groundbreaking research.

At the center of the landmark science that garnered the financial support is Dr. Laurence Cooper, PhD, a Professor of Pediatrics and a Section Chief at MD Anderson Cancer Center. Dr. Cooper has been a ballast in the 21st century’s fight against cancer: A marquee name in the oncology world and an Alliance for Cancer Gene Therapy (ACGT) Young Investigator, his work has propelled the immunotherapy forward immensely and awarded hope and treatment to those who need it most. Working alongside Dr. Cooper is the esteemed Perry Hackett, PhD, a professor at the College of Biological Sciences at Minnesota. Dr. Cooper and Hackett explore the realm of immunotherapy that pits patients’ CAR T cells against invasive cancer cells. MD Anderson Cancer Center has published the scientific details of the project and partnership in their newsroom. It’s here that the center’s president, Dr. Ron DePinho, lauds immunotherapy as “one of the most exciting approaches with curative potential in oncology today.”

The generous support stems from synthetic biology company Intrexon Corporation and its oncology partner, ZIOPHARM Oncology, in the shape of a licensing agreement. The backing of these two companies augurs a new era for immunotherapy. As brilliant minds like Dr. Cooper and his colleagues further harness the power of immunotherapy, biotech companies are able to connect with laboratories and work to bring the discoveries to patients who need it dearly. Immunotherapy is a beacon in the war on cancer: It promises an effective treatment that can battle cancer cells without the harrowing side effects of chemotherapy, radiation or surgery. And that dovetails completely with ACGT’s mission: Discovering new ways to treat and defeat cancer, while also preserving patients’ quality of life and minimizing painful side effects. 

Dr. Cooper’s pedigree with ACGT dates back over one decade. In 2003, as an Assistant Professor of Hematology and Oncology at the City of Hope National Medical Center in Duarte, California, Dr. Cooper received a Young Investigator Grant. These ACGT grants support bright oncologists with novel ideas who display admirable promise and dedication. In 2006, Dr. Cooper transitioned to his role at MD Anderson Cancer Center. And ever since connecting with ACGT, Dr. Cooper has regularly assisted our foundation in evangelizing donors by spotlighting the amazing potential of immunotherapy. 

“The war on cancer has evolved… over these years,” Dr. Cooper said at a recent ACGT event in New York City, speaking to the growth and major successes of immunotherapy. The future of fighting cancer is immune-based therapy and gene therapy, he added. Indeed, the science has grown by leaps and bounds in just a few short decades, offering a renewed hope — and saving lives — to cancer patients who believed they had exhausted other treatment options. 

Dr. Cooper also understands the integral and indispensable role that philanthropy plays in the fight against cancer, particularly in the arena of immunotherapy. “It doesn’t fit the typical funding model,” he said at that same event, speaking to a room of donors, scientists and cancer survivors. “Philanthropy, thank goodness, has really stepped in and recognized the power.”

As philanthropists — and now biotech companies like Intrexon Corporation ZIOPHARM Oncology, too — continue to support immunotherapy, we can watch a new era of better, more effective cancer treatment unfold. This deep investment in immunotherapy highlights the field’s bright future, and our ability to one day transform the perils of cancer into a relic.

 

At Alliance for Cancer Gene Therapy (ACGT), our vision is to transform cancer from a harrowing disease into a manageable diagnosis. We believe — along with the brilliant scientists who provide research, treatment and scientific counsel — that this can be unlocked through immunotherapy and cell and gene therapies. And so we are overjoyed that recently, immunotherapy has made another major stride forward in revolutionizing how we treat cancer.

That leap forward comes in the form of a Food and Drug Administration (FDA) approval. The FDA – which is tasked with taking a close look at new cancer treatment methods and drugs — recently granted accelerated approval to Opdivo (nivolumab), an immunotherapy drug for melanoma patients. Opdivo holds a particular importance: It will be used to treat melanoma patients who are not responding to other drugs, and who are not candidates for surgery. The heartening news came out on December 22, 2014.  

Paramount to Opdivo’s development and success is Dr. Lieping Chen, a brilliant oncologist who serves on the ACGT Scientific Advisory Council. Dr. Chen helps ACGT to bestow grants upon the world’s most talented and visionary oncologists — oncologists who have, in turn, developed breakthrough drugs, research and clinical trials, and saved lives. Dr. Chen is also the Director of Yale Cancer Center’s Immunology Program, and a Professor in Cancer Research and of Immunobiology. Dr. Chen’s work on Opdivo was carried out alongside scientists Arlene Sharpe and Gordon Freeman. Opdivo is made by biopharmaceutical company Bristol-Myers Squibb. 

Opdivo functions in that same, amazing way that other immunotherapy drugs do: It harnesses the patient’s immune system, allowing the body to fight off the invading cancer cells. This approach is often less harrowing than the other options, like chemotherapy, radiation and surgery. Opdivo’s ability to target melanoma specifically is also tremendous news. Melanoma is the fifth most common cancer in the United States, behind breast cancer, colon and rectal cancer, prostate cancer and lung cancer. Further, estimates from the National Cancer Institute posit that over 75,000 Americans were diagnosed with melanoma in 2014 — and that nearly 10,000 Americans perished from the cancer.

For us at ACGT — and all others following the saga of Opdivo — it is incredibly heartening to see the tremendous power of immunotherapy translate from the laboratory into the drug phase. It represents the future of how we understand cancer, and cancer’s transformation into a disease that we can manage while still maintain quality of life.

This fantastic Opdivo development comes on the heels of another great announcement: The FDA’s accelerated approval of Keytruda (pembrolizumab), the Merck drug that also employs immunotherapy to battle cancer, specifically melanoma. Keytruda’s potential and positive implications have been reported on by The Wall Street Journal, Barron’s, and FiercePharma. Indeed, media reports note Merck soon intends to expand Keytruda’s abilities to treat other cancer diagnoses, as well. 

These two drugs receiving the green light from the FDA designated 2014 as a watershed year for immunotherapy. The news also prepares us for an even bigger year: 2015 promises to hold so much spectacular news for the future of battling cancer. At ACGT, we look into the future eagerly and with a great deal of optimism. So many powerful factions — from scientists and donors to biotech and pharmaceutical companies — are uniting to eradicate cancer as we know it. 

We also wish to stress the importance individual charity can have. So many breakthroughs, like Dr. Chen’s and those of Opdivo and Keytruda, and financed by generous philanthropists and private contributors. These visionaries are making up for the dearth of financing from public entities. We invite you to learn more about donating, and how your gift can play a paramount role in stamping out cancer. 

Lymphyblastic leukemia is a destructive, painful blood cancer that progresses quickly and can be fatal within months if left untreated. And while treatments such as chemotherapy are currently commonly used, many patients suffer relapse, lowering the disease’s overall cure rate to between 30 and 40 percent.

But the genetic modification of T-lymphocytes is now emerging as a powerful approach to investigate T cell function. Alliance for Cancer Gene Therapy (ACGT) funded research grants for two investigators work in the area: that of Dr. Michel Sadelain, director of the Center for Cell Engineering & Gene Transfer and Gene Expression Laboratory at Memorial Sloan-Kettering Cancer Center, and Dr. Carl June, professor in Immunotherapy, Pathology and Laboratory Medicine at the University of Pennsylvania’s Abramson Cancer Center.

Dr. Sadelain’s research on T cell engineering and artificial antigen was bolstered by a 2004-2007 ACGT research grant for a program that investigated the therapeutic potential of genetically-targeted T cells in leukemia and lymphoma treatments. This Phase 1 of clinical trials research followed Sadelain’s demonstration that blood T cells can be genetically redirected to vanquish B-cell tumors – a discovery that demonstrated for the first time that genetically modified human T cells can induce enduring remission.

Following his ACGT-funded research, Sadelain’s treatment went through the subsequent phases of clinical trials and the JCAR015 drug – which came out of that research and was developed by Sadelain and his Sloan-Kettering colleague Renier Brentjens – was granted Breakthrough drug status by the FDA earlier this year.  This “breakthrough” status, reserved for treatments for serious or life-threatening conditions, expedites the four phases of clinical trials and final FDA approval.

Sadelain is also a scientific founder of Juno Therapeutics, a biotech company focused on cell-based cancer immunotherapies. The company recently released its IPO at the very end of 2014 and closed up more than 45% on its first day of trading, the Wall Street Journal  reported.

Sadelain’s fellow ACGT-funded investigator Dr. Carl June  has made similarly groundbreaking strides in cell therapy treatment for lymphyblastic leukemia.

ACGT funded Dr. June’s early work in 2004, which studied whether the immune system’s T cells can be altered genetically so that they become “guided missiles” that kill tumor cells. The clinical trial used genetically modified versions of the patient’s own T cells, and showed remission for up to a year in a small group of patients – several others of whom are now in complete remission. Since then, Dr. June and his team at the University of Pennsylvania Abramson Cancer Center have made great strides for the treatment of advanced chronic lymphocytic leukemia, culminating most recently with the FDA’s breakthrough designation for a new  therapy .

The designation was awarded after 27 of the 30 patients in the studies achieved a complete remission a month after receiving an infusion of these engineered cells, and 78 percent of patients were still alive following the trial in 2012.

ACGT continues to fund research, which is carefully selected by its esteemed Scientific Advisory Council.  By funding this breakthrough research, ACGT strives to reduce the painful side effects of cancer treatments, and allow patients to live longer, better quality lives .

Show your support for breakthrough cancer treatments.  Help us lead the charge!  Donate today! 

 

 

In the spirit of #GivingTuesday, a global day dedicated to giving back on December 2, Alliance for Cancer Gene Therapy (ACGT) asks donors to support their favorite causes in the spirit of the holiday season.

Here at ACGT, the nation’s only nonprofit dedicated exclusively to cell and gene therapies for cancer, we’re particularly excited about #GivingTuesday this year, as we’re unveiling our new #NotImmune campaign.

#NotImmune aims to put a spotlight on the need for funding research, so cancer can become a more manageable disease and patients can receive quality treatment with fewer painful side effects.

Cancer is an aggressive disease for which the treatments often come with devastating side effects.  Cell and gene therapies, such as immunotherapy treatments for cancer, attack the cancer, not the patient.  It’s a powerful message and a game changer for those undergoing cancer treatments.  We want to fund research that will bring these types of treatments to all.

#NotImmune is a call to action – an opportunity for people to show family, friends and colleagues that they support breakthrough research, such as immunotherapy treatments, that use the body’s own immune system to attack the cancer while minimizing side effects.

We’re fortunate to have funded researchers early on, selected by our esteemed Scientific Advisory Council, who are now considered pioneers in the fields of cell and gene therapy, and to have an active and engaged community of supporters.  Cancer is something that impacts patients, their families and the community and we invite everyone to join us in showing they’re not immune to the cause.

With your support, we help Investigators to uncover breakthrough treatments that make cancer a more manageable disease and allow patients to lead longer and fuller lives.

No one is immune to cancer, no one should be immune to the cause.  Attack the cancer, not the patient. One-hundred percent of all donations go directly to research.

Support Cancer Research!

Share your story using #NotImmune and tell us why cancer research matters to you!