Category Archives: Mission

CRISPR the Next Frontier in Gene Therapy Cancer Research

In our last blog, we described the progress and promise of CAR-T, an immunotherapy that has been particularly successful with leukemia and lymphoma. As a companion to CAR-T, and all forms of cell and gene therapies, CRISPR is the talk of the scientific town these days and is the next tcell_ssfrontier in genetic medicine.

CRISPR. Sounds like something that belongs in your kitchen, doesn’t it? This science is neither therapy nor treatment. It’s a fantastically precise tool that permits genetic editing. Once we completed mapping the human genome, we have the knowledge, and the technology, to effect change in the base DNA that makes up our unique identity. A small change in that code can make a huge difference in health and longevity.

What is CRISPR technology?

Clustered Regularly Interspaced Short Palindromic Repetition is the actual term, should you have an opportunity to prove your medical mettle in conversation. A simpler translation: molecular medical scissors used to repair or replace a portion of the genetic code. #genomics

The technology was discovered just three years ago when researchers, using a DNA cut and paste method using bacteria to protect against viruses, realized they could also edit human genes. In CRISPR, either the cancerous cell, once cut, will repair itself, or the body’s natural killer T-cells will take aim and destroy diseased cells. Unbelievably simple once the barriers caused by mutated or damaged genes are removed.DNA_scissors

Sounds like Sci-Fi? Well, it is a huge leap into the future. So new that scientists from around the world gathered earlier this year for the first international summit on human gene editing, in Washington DC, and agreed to avoid using the science for human genetic engineering. No test tube animals, no Frankenstein. On the other hand, CRISPR is being used to simulate cancers in laboratory animals, for the purpose of study, and much will be learned in this way.

CAR-T, Immunotherapy and CRISPR

CRISPR has the potential to battle not only disease, but also genetic disorders like allergies or neurological dysfunction. The opportunities are limitless. Our own Scientific Advisory Council member, Dr. Carl June, at the University of Pennsylvania, is heading a research team funded by Facebook financier Sean Parker. Dr. June aims to enhance his pioneering immunotherapy by making even more powerful killer T-cells using the CRISPR scissors. Cells will be edited to bypass cancer’s natural protective obstacles to treatment, known as checkpoint inhibitors. [See our blog on CAR-T.]

“This is the first example of CRISPR in clinical trials in humans, period,” Parker reported at the Forbes Philanthropy Summit in August. This sort of advancement may not have been possible without private funding.

 

gene_editing_ssThe race is on to refine the technique. In China, scientists are studying the potential for CRISPR for lung cancer and have secured approvals for first level trials. In the US, the FDA is expected to approve Phase I trials later this year, with patients suffering melanoma, sarcoma and myeloid cancers. The trials will examine toxicity and side effects as well as patient response.

Treating Cancer Patients

Are there challenges? Of course. Medical interventions, even perfected in the lab, must be refined in practice. Humans have been blessed with powerful immune systems that have been too often neutralized when confronted with cancers, but both CAR-T and CRISPR seek to change all that.

The future is happening right now, and ACGT is still the only non-profit in the nation dedicated exclusively to innovative cell and gene therapies for cancer. Stay tuned!

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Immunotherapy and Cell Therapy Research For Cancer Makes Headlines

In the third of a front page series in the The New York Times published July 31st – August 2nd, entitled “Setting the Body’s ‘Serial Killers’ Loose on Cancer,” science writer Andrew Pollack reported on Dr. Steven Rosenberg at the National Cancer Institute, a research physician who has committed half a century to the promise of immunotherapy. The article also highlighted the CAR-T immunotherapy treatment pioneered by Scientific Advisory Council members Dr. Carl June at the University of Pennsylvania and Dr. Michel Sadelain at Memorial Sloan-Kettering Cancer Center and referenced ACGT co-founders Edward and Barbara Netter.

ACGT Researchers Michel Sadelain and Carl June

Drs. Michel Sadelain and Carl June

ACGT has funded the research into CAR-T therapy from inception and we have seen incredible progress in refining this science as a whole new way of treating cancer, and the vast majority of patients enrolled in trials are experiencing long-lasting remissions.

What exactly is CAR-T cell therapy and how does it work?

Following is a simple summary of a complex process, and the Times printed a very useful diagram in Part I.

T-cells are a type of white blood cell whose sole function is to identify and destroy cells that threaten the body’s health. They are also known as killer cells, an apt description. Each T-cell has claw-like receptors that can lock onto antigens – protein fragments on the surface of abnormal cells. In a very simple blood draw, a patient’s own cells are extracted and then modified to target cancer cells before reinserting into the patient to awaken the immune system. The great beauty of this science is that those T-cells now know exactly where to attack. They go after the cancer – and only the cancer – so side effects are generally few, and mild, nothing like the toxic effects of chemotherapy drugs. And the treatment is usually one or two days, not weeks or months.

CAR T-cells Of course there is a snag in the approach – nothing in medical science happens overnight. Cancer cells have what are called Checkpoint Inhibitors that defend against any interference, even from the immune system. So T-cells have to bypass those inhibitors and that’s the more delicate part of the process. When this doesn’t work, side effects can be severe – in fact, the immune system can turn on itself, known as an autoimmune response, which can be severe, and the treatment may not work. The future of CAR-T relies on shutting down checkpoints and there is a lot of optimism that this will be accomplished sooner than later.

CAR-T has been proven extremely effective with leukemia and lymphomas and other blood cancers, and some pediatric types as well. Further study and human trials are necessary to improve the effectiveness of the treatment on solid cancers, and ACGT scientists are working to make this happen.

Promise of Cell and Gene Therapies for Cancer

The real future, and the great promise of cell therapies like CAR-T, is to match the cancer profile to the treatment design and tremendous progress is being made toward this end. In this way, the checkpoints on each cancer in each individual patient will be more easily identified, and neutralized, and the treatment will be more reliably effective.

Immunotherapy is the future and is happening right now and ACGT is still the only non-profit in the nation dedicated exclusively to cell and gene therapies for cancer. Stay tuned!

 

An Amazing, Unprecedented Documentary on Cancer

At Alliance for Cancer Gene Therapy (ACGT), we strive to revolutionize how we treat and understand cancer. The scientists we fund, and the donors who share their resources, envision a world where a cancer diagnosis doesn’t signal hopelessness. They imagine a world where cancer is a manageable disease — one that doesn’t require surgery, radiation or the harrowing side effects that come with a course of chemotherapy.

We also know ACGT is part of a larger narrative: The war on cancer. It’s a war that has touched countless lives, and involves so many brave patients and dedicated researchers. Cell and gene therapies — the revolutionary treatments that harness the human immune system to effectively battle cancer cells — are showing more promise than ever. And that promise will be featured in an upcoming television event.

This week, PBS is debuting an unprecedented documentary: a six-hour, three-part program produced by legendary filmmaker Ken Burns that traces the history of cancer and explores what lies ahead. Titled “Cancer: The Emperor of All Maladies,” the program is based on the Pulitzer Prize-winning book of the same name, penned by Dr. Siddhartha Mukherjee. Dr. Mukherjee is an author and physician who has dedicated himself to battling cancer – he both treats patients and works diligently to uncover new and innovative ways to conquer cancer.

“Cancer: The Emperor of All Maladies” is an ambitious project — it’s “the most comprehensive documentary on a single disease ever made,” according to PBS. The program will explore cancer’s appearance and impact thousands of years ago; it will spotlight the efforts in the 20th century to understand, control and cure cancer; and it will detail the cell and gene therapy work being done today that holds tremendous promise and hope.

ACGT invites and encourages our supporters to tune in. The documentary dovetails with our mission, our motivations and our accomplishments. And it culminates on an optimistic note: “The series also features the current status of cancer knowledge and treatment — the dawn of an era in which cancer may become a chronic or curable illness rather than its historic death sentence in some forms,” PBS explains.

We’re also tremendously proud that “Cancer: The Emperor of All Maladies” includes a few faces familiar to the ACGT community: Dr. Carl June, an ACGT grantee, immunotherapy pioneer, and member of our Scientific Advisory Council, and Emily Whitehead, the amazing young girl who — with the help of Dr. June — was able to fully recover from near fatal leukemia. Both Dr. June and Emily Whitehead make appearances in the film, and share their stories.

Their stories are inextricably linked to ACGT. Dr. Carl June — who has carried out watershed breakthroughs during his time at the University of Pennsylvania Perelman School of Medicine — received an ACGT grant a few years ago to battle blood cancers. It was through this work that Dr. June was able to save Emily’s life — and make history. For those interested in learning more, read The New York Times story.

This won’t be the duo’s first time on the small screen: In February, Dr. June and Emily Whitehead were featured in the VICE/HBO documentary “Killing Cancer,” which focused on our ability to now weaponize the human immune system against cancer. Other ACGT personalities were featured prominently, as well: 2013 grantee Dr. John Bell and Scientific Advisory Council member Dr. Stephen Russell.

Dr. June and Emily Whitehead’s inclusion in “Cancer: The Emperor of All Maladies”— along with their appearances in “Killing Cancer” — are a testament to ACGT and so much of what we stand for: conquering cancer, optimism and the power of private philanthropy.

For those who share our mission and passion, please tune into the PBS documentary this week. The program will be showing Monday, Tuesday and Wednesday; broadcast details can be found here.

The Importance of Philanthropy in Our Fight Against Cancer

Progress in the war against cancer involves so many dimensions – the ingenuity and dedication of physicians and oncologists, new and improved technologies for battling the disease, and proper funding to accelerate our research and make for more functional laboratories.

Fortunately, we have no shortage of brilliant minds intent on uncovering pain-free treatments and unlocking the key to remission. There is, however, a dearth of funds. While cell and gene therapy has established itself as a beacon of hope for those affected by cancer, public funding remains low. With sequesters and slashed budgets, the support that cell and gene therapies for cancer so sorely needs, often received through National Institutes of Health (NIH) grants, is at an all-time low.

Still, top scientists and researchers are able to uncover breakthrough discoveries and create life-saving clinical trials through the power of philanthropy. Private individuals and foundations have stepped in to fill public funding gaps and finance unprecedented medical developments on a regular basis. Large gifts to cancer institutions and research centers are becoming more frequent, and more and more supporters are rallying to conquer cancer through personal donations.

Late last year at an Alliance for Cancer Gene Therapy (ACGT) event at the Harvard Club in New York City, Dr. Laurence Cooper – M.D. and Ph.D. at The MD Anderson Cancer Center and a recipient of ACGT’s 2003 Young Investigator Award – put it eloquently: “There’s never been a better time to be an oncologist,” he said. “Philanthropy has truly stepped in and recognized the power of cell and gene therapy.”

Dr. Cooper was speaking directly to individuals who had made a difference: donors and board members at our foundation. Dr. Cooper was joined by Doctors Carl June of the University of Pennsylvania, Michel Sadelain of Memorial Sloan Kettering Cancer Center, and Savio Woo of The Mount Sinai School of Medicine, who all spoke about the power and potential of cell and gene therapies for cancer. In 2012, a trial at the University of Pennsylvania by Dr. June saved the life of Emma Whitehead, an eight-year-old who was battling leukemia. Now in remission, Emma is sharing her journey online. In addition to Emma, Dr. June has placed dozens of other patients in remission.

Cell and gene therapies have the potential to combat a variety of cancers – your support, and our scientists’ research, can allow physicians to successfully treat blood cancers, brain cancers, and pediatric cancers. Indeed, it already has.

Today, philanthropy is going to extraordinary lengths in the fight against cancer. Our donors are funding scientists and doctors like Carl June, Michel Sadelain, Laurence Cooper and Savio Woo, who are fast becoming household names in the realm of oncology – and, most important of all, saving lives and providing hope. This is a new and inspiring age in cancer treatment, and donors are making it possible.

I’m continually amazed at the generosity and foresight of our supporters. Last month, ACGT was the beneficiary of a gift from sixth-grade students at Greenwich’s Stanwich School. Those five young women, who raised funds through a bake sale and donated all proceeds to Alliance for Cancer Gene Therapy, are contributing to something great: a future of pain-free cancer treatment and remission.

This summer, donors from around Connecticut and beyond will gather for the annual Greenwich-Stamford Swim Across America event. Held Saturday, June 21 at Alliance for Cancer Gene Therapy’s headquarters in Stamford, families and friends affected by cancer will play a personal role in uncovering a treatment method that eschews the suffering associated with radiation and chemotherapy.

In the non-profit world, the generosity of many individuals makes the difference.  In advancing cell and gene therapies for cancer, this is certainly the case.

 

—Barbara Netter, President and Co-Founder of Alliance for Cancer Gene Therapy (ACGT)

Cutting Edge Web Site for Cutting Edge Research

tcells_largeAs you may have noticed, ACGT has revamped its web site!

In this ever-changing technical landscape, we want to be sure we can deliver you breakthroughs in the cancer field, progress of ACGT researchers, updates on clinical trials, creative ways to fundraise for ACGT, details about our funding partners, why you should donate to ACGT, and get information to you as quickly as possible. Please take some time and look at our Progress & Impact page, where the facts and figures still tell us that every minute of every day two people discover they have cancer, that 1 of 4 Americans will develop cancer at some point in time, that current treatments for cancer have hardly changed in the last fifty years and that the choices — chemotherapy, radiation and surgery — can be nearly as debilitating as the disease.

ACGT is the only public charity in the nation dedicated exclusively to the study of cell and gene therapies for cancer. ACGT supports research into treatments that directly attack the cancer, and only the cancer.  To date, we have funded 44 grants at 31 institutions in the US and Canada for both discovery and clinical translation.  ACGT researchers are triggering the immune system to detect and destroy cancers, researchers are strangulating the blood supply to kill cancer cells, or injecting reengineered genes from the patient to repair damaged or defective genes.

Gene therapies are also used in conjunction with chemotherapy to improve response rates. Rapid progress in technology and molecular medicine are paving the way to the greatest advance in cancer treatment in fifty years. As ACGT Scientific Advisory Council Michel Sadelain remarked at an ACGT event in October 2013, “we are on the cusp of a golden age of cancer prevention and treatment, thanks to the promise of recent discoveries.”

Margaret C. Cianci, Executive Director, ACGT
Ms. Cianci invites your comments and opinions.
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