National Foundation For Cancer Research - Research Milestones

Research Milestones

• Launching life-saving drugs such as Avastin. Thanks to an NFCR scientist, the drug Avastin has been used to save and extend the lives of nearly hundred thousand cancer patients. It all started with one discovery by NFCR scientist Dr. Harold Dvorak at Beth Israel Deaconess Medical Center. He discovered Vascular Endothelial Growth Factor (VEGF), a protein secreted by cells to start growth of new blood vessels-including vessels that bring blood to tumors for growth. By shutting off VEGF, scientists were able to cut down the growth of the tumor, often saving patients from reaching late-stage cancer. Avastin is currently used for treating patients with colorectal, lung, kidney, and brain (glioblastoma) cancers. Click here to learn more.

• Using basic chemistry to help cancer patients. NFCR scientist Dr. Csaba Horvath at Yale University, was the first to use a technique called High Performance Liquid Chromatography (HPLC) for life sciences and medicine; previously, it was used only for chemistry. HPLC is now used by nearly all pharmaceutical companies to develop new drugs.

• Groundbreaking targeted cancer therapies such as Iressa and Tarceva. Today, targeted therapy is all over the news - it is the new wave of cancer treatment. These therapies "target" or inhibit the abnormal gene or protein gone haywire that causes abnormal cell growth or cancer. Two of today's targeted therapies, Iressa and Tarceva, were made possible by the work of NFCR scientist Dr. Gordon Sato at the W. Alton Jones Cell Science Center Inc. He investigated and elucidated the role of Epidermal Growth Factor Receptor (EGFR), a growth molecule implicated in many cancers. His vital research led to development of Iressa and Tarceva, which block EGFR on cancer cells, and inhibit the growth signals for cancer. Today, these drugs are at the forefront of providing more individualized and effective treatment to cancer patients.

• Hormone therapies for breast cancer, including Tamoxifin and Aromatase Inhibitors. NFCR scientist Dr. Jack Gorski at University of Wisconsin at Madison provided the crucial groundwork for today's use of hormone therapies for breast cancer. His research helped us understand how the hormone, estrogen, interacts with its protein partner or receptor, and how the two together regulate cell functions such as growth. This work helped lead to the development of Tamoxifen and Aromatase Inhibitors-breast cancer drugs that block or prevent the growth signals of estrogen and its receptor.

• Drugs using Monoclonal Antibodies. A monoclonal antibody is a laboratory-produced molecule that's specifically engineered to attach to certain defects in your cancer cells. Monoclonal antibodies mimic the antibodies your body naturally produces as part of your immune system's response to germs, vaccines and other invaders. A new and improved methodology for constructing monoclonal antibodies (mAbs) was developed by Dr. Cesar Milstein at the University of Cambridge, England. This contributed significantly to the development of mAbs that are targeted anti-cancer drugs, such as Avastin, Herceptin, Erbitux, and many others.

• Cladribine, a first-line treatment for leukemia. The research of NFCR scientist Dr. Dennis Carson at the University of California, San Diego, led to the development of Cladribine, a landmark drug that can cure patients with hairy cell leukemia. Dr. Carson discovered that a purine nucleoside agent, which prevents cells from making DNA and RNA, can selectively kill hairy cell leukemia cells - a breakthrough discovery. Today, Cladribine remains the first-line treatment for this disease.

• Role of a healthy diet including vitamin B12 and Folic Acid to help reduce risk of cancer. The role of vitamin B12 and Folic Acid in preventing DNA damage was confirmed by NFCR scientist Dr. Bruce N. Ames, then at the University of California, Berkeley. His findings have since been translated into clear public policy recommendations on the role of a healthy diet in reducing cancer risk.

• Theory behind life-saving targeted cancer therapies. The theory of "Oncogene Addiction" was proposed by NFCR scientist, Dr. I. Bernard Weinstein at Columbia University. Though there are numerous genetic abnormalities in cancer cells, he proposed that cancer's deadly growth may be dependent on only one or a few cancer causing genes (oncogenes). New targeted therapies that specifically block signals from these oncogenes could help stop cancer while minimizing harmful effects to normal tissues. His theory provided the basis and guidelines for the development of many of today's targeted cancer therapies, including IressaTM, TarcevaTM, and GleevecTM.

• Treating breast cancer patients who have Progesterone Receptors (PR) with Tamoxifin. The vital role of Progesterone Receptors (PR), the protein partner of the progesterone hormone, in abnormal growth in breast cancer was confirmed by NFCR scientist Dr. Kathryn Horwitz at University of Colorado Health Science Center. Dr. Horwitz developed PR detection tools, which are now routinely used to test for breast cancer in patients. These findings have also had a profound impact on directing the wide clinical usage of Tamoxifen, the drug which blocks PR, in breast cancer patients.

• Stopping the spread of cancer. Six Metastasis Suppressor Genes that stop the spread or metastasis of cancer have been identified by a team of collaborators led by NFCR scientist Dr. Danny Welch, Director of NFCR Center for Metastasis Research, at the University of Alabama, Birmingham. These findings opened an entirely new avenue for the development of drugs that could stop cancer from spreading, holding great promise for controlling the seemingly uncontrollable cancer cell in a variety of cancer types.

• Improving chemo and radiation. Imagine devices so small that one thousand of them can fit side by side within the period at the end of this sentence! That's how small the nanoparticles are that NFCR scientist Dr. Esther Chang is working with at her lab at Georgetown University Lombardi Comprehensive Cancer Center. "Decorated" with a cancer-fighting agent, these nanoparticles are able to locate hidden tumor cells and deliver their payload: a fully functioning copy of a gene that suppresses the spread of cancer. Moreover, this novel drug delivery system significantly enhances a tumor's sensitivity to chemo and radiation therapy.

• Using the power of personal computers to fight cancer. The world's largest supercomputer for discovering drugs - called, "The Screensaver-Lifesaver Project" - was launched by NFCR scientist Dr. W. Graham Richards, Director of the NFCR Center for Computational Drug Discovery at the University of Oxford, England. The Project used the idle PC power from 3.5 million individual computers worldwide to screen potential drug candidates against 12 cancer protein targets. This public endeavor resulted in tens of thousands of lead compounds for further biological testing, significantly reducing the time needed in the initial phase of anti-cancer drug development.

• Personalized medicine for patients with lung cancer. Sometimes, the most challenging task of a physician is to determine which patients will benefit most from which treatments. Fortunately for lung cancer patients, NFCR has made that decision easier. NFCR scientist Dr. Daniel Haber at the Massachusetts General Hospital discovered a specific mutation that affects about 10% of all lung cancer patients. This discovery means that it is now possible to identify patients who are most likely to benefit from the drug IressaTM, providing crucial guidance for personalized medicine for patients with lung cancer.

• A new technology that is helping develop better cancer drugs. A new technology framework that allows the design and synthesis of β-peptide Inhibitors was developed by NFCR scientist Dr. Alanna Schepartz, Director of the NFCR Center for Anti-Cancer Drug Design and Discovery at Yale University. Whereas other drugs may have failed, β-peptide inhibitors are able to specifically inhibit or target almost any protein-to-protein interactions occurring in diseases. This new technology is tremendously improving the current capacity of drug development.

• Delaying or preventing the recurrence of ovarian cancer. Ovarian cancer continues to claim the lives of three out of four women with the disease, due mainly to the persistence of drug-resistant cancer cells that survive despite standard chemotherapy. These resistant cancer cells can remain dormant or "asleep" for years, only to awaken later and grow progressively until they cause the death of the patient. NFCR scientist Dr. Robert C. Bast, Jr., at the M.D. Anderson Cancer Center, discovered a gene called ARHI plays a critical role in the survival of these dormant cancer cells. The team further developed a new experimental model in which ARHI can be switched on and off to closely mimic the actual tumor dormancy and re-growth that occurs in humans. This model will help cancer researchers open the door to the development of new treatments that eliminate these cells before they can become reactivated in the body.

• Discovery that lycopenes can help reduce the risk of some cancers. Lycopene is the famous red pigment found in tomatoes and other fruits and vegetables. Dr. Helmut Sies at Heinrich-Heine-Universität, in Heidelberg, Germany, discovered lycopene is a powerful anti-oxidant and further demonstrated that consumption of lycopene can help prevent the risk of skin cancer. This finding led to an enhanced public awareness of a maintaining a healthy diet for cancer prevention.

• Immunotherapy to treat leukemia. A new type of immunotherapy using genetically engineered human immune cells to treat leukemia was discovered by NFCR scientist Dr. Laurence Cooper at the M.D. Anderson Cancer Center. Dr. Cooper also developed a new technology that allows quick and cost-effective manufacturing of therapeutic immune cells, meaning faster progress in translating this novel immunotherapy to cancer patients.

• Traditional Chinese Herbal Medicine formula. The Traditional Chinese Herbal Medicine formula, called PHY906, was analyzed and clinically tested for its potential as an effective addition to chemotherapy by NFCR scientist Dr. Yung-Chi Cheng at Yale University. PHY906 could become one of the first FDA-approved oral herbal medicines for anti-cancer treatment in patients with colon, liver, or pancreatic cancer.

• A new technology that is 14,000 times faster than other technologies. NFCR scientists Drs. W. Graham Richards and Pedro J. Ballester at the NFCR Center for Computational Drug Discovery at the University of Oxford, England, have developed a new technology that finds drug-like molecules within a huge database in only a few hours rather than after a few years. The software, known as "Ultrafast Shape Recognition", is up to 14,000 times faster than other technologies. The software has been licensed and may soon be available to the wider scientific community, significantly accelerating the pace of drug development.

• Growing new blood cells. A set of microRNAs -or tiny but important pieces of cellular RNA- was discovered by Dr. Curt Civin, formerly at Johns Hopkins University (now at the University of Maryland). These particular microRNAs work as powerful "Master Switches" that are able to switch on and off critical genes to keep adult blood-forming stem cells in their primitive state. This breakthrough discovery may one day enable scientists to grow new blood cells for transplant into patients with cancer and other bone marrow disorders.

• New treatment for brain tumors. A major problem with brain tumors occurs when the tumor adapts and becomes resistant to treatment, and the treatment no longer works. NFCR scientist Dr. Webster K. Cavenee at the Ludwig Institute for Cancer Research has found a way to inhibit a receptor that helps tumors in one type of brain cancer, glioblastoma, become resistant. This advancement may lead to a new way to overcome tumor resistance to treatment and offer a more effective therapy for this highly aggressive brain tumor.

• Customizing treatment for colorectal cancer patients. Surrogate Markers for evaluating treatment efficacy of the anti-angiogenic drug AvastinTM were developed by Dr. Rakesh Jain at the Massachusetts General Hospital. Clinical tests of these markers in colorectal cancer patients have shown promising results. With further testing, the surrogate markers Dr. Jain developed may soon enable oncologists to customize the anti-angiogenic therapy for their individual patients to achieve optimal results.

• 1.6 billion different human sFv antibody-displaying phages. A library containing 1.6 billion different human sFv antibody-displaying phages was established by Dr. Wayne Marasco, Director of the NFCR Center for Therapeutic Antibody Engineering at the Dana-Farber Cancer Institute. This huge library is a tremendous resource for developing Monoclonal Antibody-based Targeted Cancer Therapies.

• 20,000 high quality fresh frozen cancer tissue samples. A state-of-the-art Tissue Bank was established in partnership with Tianjin Medical University Cancer Institute and Hospital in Tianjin, China. To date, the Tissue Bank has collected and systematically catalogued nearly 20,000 high quality fresh frozen cancer tissue samples and more than 7,000 blood samples, in addition to over 400,000 paraffin imbedded samples stored historically. This is a very precious resource that allows scientists to classify tumors based on their molecular signatures and identify tumor biomarkers for the development of early diagnostic tools and personalized targeted cancer therapies.

• New drug for pancreatic cancer. The urokinase inhibitor, UK122, was developed by Dr. Daniel Von Hoff, Director of the NFCR Center for Targeted Cancer Therapies at TGen in Phoenix, Arizona. UK122 is a molecular therapeutic which shows strong anti-cancer effects in preclinical tests. It could become a new and better drug for treating pancreatic cancer - one of the most deadly cancers.

• Detecting cancer earlier. Novel cancer biomarkers such as CRIP1 that are ideal for cancer early detection have been discovered by Dr. James Basilion, Director of the NFCR Center for Molecular Imaging at Case Western Reserve University. New Molecular Imaging Tools are now being developed to visualize these markers and allow cancer to be detected at early and more treatable stages.

• Preventative and/or treatment effects against liver cancer, highly aggressive lung cancer, melanoma and leukemia. A new class of chemical compounds named Synthetic Triterpenoids has been developed by Dr. Michael Sporn at Dartmouth Medical School. These compounds have shown potent preventative and/or treatment effects against liver cancer, highly aggressive lung cancer, melanoma and leukemia. One of these triterpenoids, CDDO-Methyl ester, is now in a Phase II clinical trial for the treatment of pancreatic cancer. This completely new group of molecules may bring more effective therapeutics to patients with a variety of cancers.

• Novel Virotherapy for lung cancer patients. A new generation of the therapeutic virus, KTR-27, was developed by Dr. Feng Yao at Brigham and Women's Hospital. KTR-27 is capable of killing cancer cells and is also tightly controlled to minimize harmful effects to healthy cells. Further optimization of this innovative anti-cancer therapeutic may lead to a novel Virotherapy for lung cancer patients.

• Improving surgical removal of brain tumors. A Molecular Imaging Technology that uses tumor biomarkers to visualize glioblastoma during surgery was developed by Dr. James Basilion, Director of the NFCR Center for Molecular Imaging at Case Western Reserve University. This novel technique improves surgical removal of infiltrated glioblastoma and may increase survival rates of patients with this very aggressive brain tumor.

• Overcoming resistance to Taxol. The molecular mechanisms of Tumor Resistance to chemotherapy drug Taxol have been determined by Dr. Susan B. Horwitz at Albert Einstein College of Medicine. Dr. Horwitz also investigated natural products Epothilones and Discodermolide which may overcome this problem. To date, over a million cancer patients worldwide have received Taxol for treating their tumors. Dr. Horwitz's discoveries may make a significant difference for those whose tumor become resistant to Taxol.

• New drug for leukemia, brain tumors, and small cell lung cancer. A new drug, laromustine, has been designed and synthesized by Dr. Alan Sartorelli at Yale University School of Medicine. laromustine has entered clinical trials for treatment of acute myeloid leukemia, brain tumors, and small cell lung cancer. To maximize the clinical benefits of laromustine, Dr. Sartorelli has also developed a simple and accurate laboratory test, the AGT assay, to help oncologists choose patients who will most likely benefit from treatment with laromustine. This is a significant step toward personalized treatment of patients with this novel therapy.

• Quality control checkpoints in the cell. A previously unknown "Quality Control" process that human cells use to produce defect-free proteins in the body has been discovered by Dr. Paul Schimmel at the Scripps Research Institute. This discovery provides the first evidence of the existence of three different quality control checkpoints in the cell, and explains for the first time how these checkpoints identify and correct errors occurring during protein production. This breakthrough could enable scientists to discover the underlying causes of cancer and other diseases and develop novel approaches for treatment.

• Mutations in one-fourth of all human cancers. Anti-RAS Intracellular Antibodies that prevent aberrant RAS signaling inside cancer cells have been developed by Dr. Terence H. Rabbitts at the Leeds Institute of Molecular Medicine, England. Oncogenic RAS mutation underlies the pathogenesis of more than one-fourth of all human cancers, and this breakthrough research may lead to improved treatment for many types of cancer, including pancreatic, lung, colon cancer, and leukemia.

• EGCG in green tea. The anti-cancer effects of EGCG, a component in green tea, were identified by Dr. I. Bernard Weinstein at Columbia University. This finding has led to a multi-institutional randomized clinical trial on green tea extract Polyphenon E, which is enriched with EGCG, for its cancer-preventing effects in people with Barrett's esophagus, a condition that increases the risk of esophageal cancer. Further investigation on EGCG may well lead to the development of effective therapeutic agents for esophageal cancer prevention.

• Tracking cancer cells in the bloodstream. A new cutting-edge microchip-based device called the CTC-chip was developed by Dr. Daniel Haber and colleagues at Massachusetts General Hospital. The CTC-chip allows researchers to capture even a minute number of circulating tumor cells (CTCs) that have entered the bloodstream and track their genetic changes in real-time. This new technology makes it possible for non-invasive and continuous monitoring of the genetic profile of tumor cells during treatment, enabling doctors to tailor therapy to individual cancer patients.

• Improving targeted therapies such as GleevecTM, Tarceva TM, Sutent TM, and Nexavar TM. New gene signatures that hold promise for improving treatment efficacy of available targeted anti-cancer therapies were identified by researchers at the NFCR Center for Targeted Cancer Therapies at the Translational Genomics Research Institute, directed by Dr. Daniel Von Hoff. The genes identified are under further investigation and may significantly improve patients' responses to commonly used targeted therapies such as GleevecTM, Tarceva TM, Sutent TM, and Nexavar TM.

• Online DrugFinder. A new online "DrugFinder" service was jointly launched by NFCR and its partner, Inhibox, a computational drug discovery company located at Oxford, UK. Using cutting-edge computational technology, this online screening service will identify possible hits in terms of new drug candidates, greatly accelerating the speed of drug discovery.

• Prostate cancer gene therapy. An innovative gene therapy to treat prostate cancer was developed by Dr. Paul Fisher at Virginia Commonwealth University. This new therapeutic approach uses a genetically re-programmed virus with a smart control system that ensures that the viruses specifically destroy tumor cells and leave normal cells unharmed. Dr. Fisher's gene therapy could be especially beneficial to patients with metastatic prostate cancer.

• Stopping lethal invastion of brain cancer. Gene signatures correlated to cell migration and invasion of glioblastoma were identified by Dr. Stanley Cohen at Stanford University School of Medicine. These findings provide novel molecular targets around which new therapies may be developed to stop the lethal invasion of this very aggressive type of brain cancer.

• A new experimental model for studying ovarian cancer was developed by Dr. Robert Bast, Jr., at M.D. Anderson Cancer Center. Ovarian cancer continues to kill seventy-five percent of all women diagnosed, largely due to the chemotherapy-resistant cells that can remain dormant for years and then awaken to grow progressively and kill the patients. Dr. Bast's new model enables scientists to further understand ovarian cancer and develop effective strategies to prevent these dormant cells from rebounding.

• Vaccine for advanced kidney cancer. A new cancer vaccine developed by Dr. Howard Kaufman, formerly at Columbia University (now at Mount Sinai University), can boost a patient's immune reactions to kill the tumor cells. This promising new treatment has shown favorable results for patients with advanced kidney cancer in a Phase II clinical trial.

• ER+ Breast Cancer. Genes that may predict if a patient with ER+ Breast Cancer will respond to hormone therapy have been identified by Dr. Kathryn Horwitz at University of Colorado Health Science Center. Dr. Horwitz's work provides crucial information that doctors need to make treatment decisions for their patients, and thus help to avoid wasting precious time in the treatment process.

• Lowering the chance of rejection of donor bone marrow transplantation. A treatment strategy to lower the chance of rejection of donor bone marrow transplantation has been developed by Dr. Curt Civin, formerly at Johns Hopkins University (now at University of Maryland). With further testing, this new strategy could prove useful in making donor bone marrow transplantation a safer approach to replenishing cancer patients' vital blood-forming system which has been destroyed by high-dose chemotherapy.

• Suppressing tumor growth. SARI, a new gene that may significantly suppress tumor growth, was discovered by Dr. Paul Fisher at Virginia Commonwealth University. Dr. Fisher's discovery may provide valuable insight into tumor suppression and may lead to a new anti-cancer gene therapy for treatment of multiple types of cancer.