+ Breast and Ovarian Cancer Outreach Program, Sharsheret

Breast and Ovarian Cancer Outreach Program
Grant Amount: $50,000

Sharsheret is a national nonprofit organization supporting young women and their families facing breast cancer. PHASE ONE's grant will help Sharsheret expand their outreach throughout Los Angeles medical centers and their mission to support women of all backgrounds through individualized connections with networks of peers and health professionals. This grant will also help Sharsheret provide individual patient kits which include:
• Busy Box® for mothers facing cancer
• Best Face Forward® for those impacted by the cosmetic side effects of treatment
• Newly Diagnosed kit
• Thriving Again® survivorship kit
• Financial Wellness Toolkit

+ Continuation of Testicular Cancer Awareness Education Grant with LAUSD

Los Angeles Unified School District (LAUSD) Testicular Cancer Program
Grant Amount: $52,034

In 2017, PHASE ONE funded and helped create a pilot program with LAUSD that educated high school students about testicular cancer. After receiving many accolades and positive results, LAUSD will be introducing and expanding the program to additional campuses across the district for the 2018-2019 school year.

+ Social Media Lab Pilot Project + Clinical Trial Recruitment, Mina S. Sedrak, MD

Dr. Mina Sedrak
City of Hope
"Social media lab pilot project: Tapping into Twitter to rethink how we recruit patients to clinical trials"
Grant Amount: $58,080

Clinical trials are essential for translating scientific discoveries into new treatments, but only 3 – 4% of adult cancer patients participate annually in therapeutic clinical trials. How people get information is changing dramatically and it may be necessary to use new modes of online communication through social media to tap into health communication effectively. With this grant, Dr. Mina Sedrak, Assistant Professor, Department of Medical Oncology & Therapeutics Research, will bring scientific rigor and insight to study online communication and social networks, develop new tools and methods for mining the cancer ecosystem online, and understand how interpersonal connections influence personal health choices and actions.

The use of social media for cancer research is in its infancy. Its value and direct application remains to be seen and warrants further exploration. The research Dr. Sedrak and his team are doing is highly innovative because, just like the medium itself, the tools used to conduct social media research are incompletely characterized and evolving. These insights will lead to novel approaches for dissemination of information to improve treatment and support for cancer patients and survivors and create improved public awareness of clinical trials. This work will provide the essential foundation for further research in this new area.

+ Advocacy Education Program, Friends of Cancer Research

Friends of Cancer Research
Online Educational Platform "Progress for Patients"
Grant Amount: $15,000

In order to make the greatest impact, patients and advocates need a complete knowledge and understanding of the drug development process. That’s why, in partnership with Friends of Cancer Research, PHASE ONE is pleased to be able to offer a new course and tool for patient advocates - Through the Advocacy Education program, advocates will acquire the necessary tools to effectively communicate with drug researchers, developers, and regulators enabling them to make the connections necessary to engage with all sectors who need to be better guided by patient input. PHASE ONE provided a grant to Friends of Cancer Research and has joined the Advocacy Alliance. Please visit to learn how you can be a better advocate for yourself, a family member, or a friend.

+ Testicular Cancer Awareness Education Grant with AYA@USC, James Hu, MD and Terry Church

Testicular Cancer Awareness Initiative of the Adolescent and Young Adult Cancer
Grant Amount: $25,000

Funds from “PHASE ONE’s Tia Palermo Memorial Endowment” support three different programs:

Tia’s Hope (, which provides gifts and support to children at hospitals throughout the country, PHASE ONE grants that it funds within the foundation’s original mission.

Small size grants that typically do not exceed $50,000.00 to support research, treatment and cancer awareness programs in our community that can be outside of the original PHASE ONE mission.

PHASE ONE’s first endowment grant was made in 2015 to the USC Norris Adolescent and Young Adult (AYA) cancer program’s new testicular cancer awareness initiative. The initiative is a collaborative community project with the Los Angeles Unified School District (LAUSD) and the University of California, Los Angeles (UCLA).

With the funds from the Phase One grant, in the fall of 2015, AYA initiated a pilot program with the LAUSD and UCLA aimed at increasing awareness of testicular cancer and among school-age students.

In the past several decades, cancers occurring in childhood have been the focus of cancer research and treatment programs worldwide. Unfortunately less attention has been given to adolescent and young adult (AYA) patients (ages 15-39) and cancer remains the number one disease killer within this age group. Because testicular cancer is one of the most common cancers diagnosed in young men, increasing awareness and early detection is a top educational priority of the initiative.

LAUSD is the nation’s second largest school district with an annual enrollment of approximately 651,000 kids in 1,188 schools. The district provides a semester-long comprehensive health education courses to all Grade 7 and 9 students who attend its schools. The courses are taught by credentialed health teachers. While the currently adopted curriculum used by the LAUSD health course contains content related to testicular cancer, the health textbook is ten years old, which is three years past a standard new adoption.

The initiative developed new project based health lessons focused on testicular cancer awareness to complement the current LAUSD health curriculum. Following the completion of the testicular cancer curriculum, professional development, technical assistance, and follow up support to the LAUSD health educators on project and content as they implement and incorporate these materials into their program, is planned. The newly developed curriculum was then piloted in the fall of 2015 in a minimum of three schools with the goal of expanding it to additional schools the following year and ultimately to every school in the LAUSD system.

PHASE ONE is proud to provide the seed grant of $25,000 to fund this new testicular cancer awareness initiative that ultimately will help educate and provide greater awareness to every child in the LAUSD.




+ Glioblastoma, UCLA, Linda M. Liau, MD, PhD, MBA and Timothy F. Cloughesy, MD

Dr. Linda M. Liau and Dr. Timothy F. Cloughesy
"Revolutionizing Glioblastoma Treatment: Understanding the anti-tumor immune response to develop novel immunotherapy for glioblastoma"
Grant Amount: $400,000

Drs. Liau and Cloughesy co-direct the UCLA Brain Tumor Center, which is one of five brain cancer programs nationwide to be designated a Specialized Program of Research Excellence by the National Cancer Institute. This trial will focus on adult patients with recurring glioblastoma to develop rational combinations of immunotherapeutic strategies and understand the dynamics of the anti-tumor immune response. The specific treatment includes neoadjuvant immunotherapy with a PD-1 antibody blockade and dendritic cell vaccination.

+ Head and Neck Cancer Surgical Instrument, UCLA, Maie St. John, MD

Dr. Maie St. John
"Precision cancer surgery using Dynamic Optical Contract Imaging"
Grant Amount: $297,440

Dr. St. John worked with a team from the engineering department at UCLA and created a tool that uses non-invasive imaging to detect between normal and cancerous tissue. This intraoperative instrument is the first of its kind and will reduce the devastating side effects of surgical resection of a tumor and improve the precision of cancerous tissue that is removed, leading to a potential impact on cancer mortality.

+ Pediatric Liver Cancer, UCLA, Noah Federman, MD

Dr. Noah Federman
"Precision pediatric liver cancer therapy using high-throughput drug screening of patient-derived tumor organoids"
Grant Amount: $100,000

Liver cancer is rare in children, adolescents, and young adults, but when aggressive malignancies are found, the patients often require intense chemotherapy, aggressive surgery, and a potential liver transplant. This trial will use a unique approach to personalized medicine, by screening primary-culture-3D tumor organoids from surgically removed pediatric liver cancer specimens. This method will allow hundreds to thousands of drugs to be tested directly on cancer tissues harvested from patients. These results will lead to proven and individualized treatment based on specific tumor and personal characteristics.

+ Urinary Tract Urothelial Carcinoma, UCLA, Karim Chamie, MD

Dr. Karim Chamie
"Upper Urinary Tract Urothelial Carcinoma"
Grant Amount: $277,386

In 2013, PHASE ONE funded a Phase I trial led by Dr. Chamie testing a groundbreaking new treatment: a hydrogel polymer to target chemotherapy in urothelial cancer. This treatment is now in an international Phase III trial. Building on the success of the 2013 trial, Dr. Chamie now plans to use this targeted therapy in patients with metastatic urothelial carcinoma, adding checkpoint inhibitors and immune stimulators to the polymer. A locally delivered treatment would be a paradigm shift, not only in the way checkpoint inhibitors are administered, but also in the way physicians can manage the care of metastatic urothelial carcinoma.

+ Rhabdomyosarcoma Pediatric Cancer, UCLA, Noah Federman, MD

Dr. Noah Federman & Dr. Arun Singh
"A Phase 1/2 Study Of Novel HDAC inhibitor Mocetinostat in combination with vinorelbine in relapsed/refractory Rhabdomyosarcoma"
Grant Amount: $250,000

Rhabdomyosarcoma, a tumor that starts in skeletal muscle, is a rare and devastating disease mostly found in children. New approaches are needed for patients who relapse on standard treatments as there have not been any new treatment developments in this area in 30 years and only chemotherapies are currently available. Moreover, the lack of good model systems, limited funding for this rare disease and a lack of interest from pharmaceutical companies have hindered drug development in this area.

Through high throughput profiling of cancer cells in the Translational Oncology Research Labs at UCLA, a new candidate drug has been identified that may have efficacy in this patient population. We have done additional research to validate this finding and have isolated a combination treatment approach with this new molecule that would be a valid clinical strategy in the refractory rhabdomyosarcoma population. We have garnered approval to use this medicine and have put together a clinical trial for the use of this medicine. Teaming up with Phase One has been instrumental in providing much needed funding to execute this clinical trial and bring a novel drug combination to rhabdomyosarcoma patients with few choices.

+ Glioblastoma, John Wayne Cancer Institute at Providence Saint John’s Health Center, Santosh Kesari, MD

Dr. Santosh Kesari
John Wayne Cancer Institute at Providence Saint John's Health Center
"Using personalized medicine to change the standard of care: Clinical trial of precision immunotherapy in the neoadjuvant setting for patients with glioblastoma"
Grant Amount: $299,442

Patients with high-grade gliomas represent a population with a large unmet medical need, and many drugs have failed to be effective enough to change the standard of care over the past 20 years. Standard therapy includes surgery followed by radiation and chemotherapy. However, radiation can cause immunosuppression, cognitive decline, and other unwanted side effects, as well as promote more aggressive tumor recurrence. Traditionally, new drugs in development are not tested in clinical trials until after the tumor has recurred at least once. At this point, the tumor is highly resistant to all therapies and the patient’s immune system is compromised. New strategies are needed to understand how drugs affect individual tumors and the immune system to kill cancer. Therefore, early testing of new therapies is likely to be more effective when the immune system is still intact. Here we plan to use the immune system to kill cancer (immunotherapy) based on individual tumor and patient characteristics (precision), before standard radiation therapy (neoadjuvant).

+ Interdisciplinary Undergraduate Track with Focus on Health Innovation, USC Iovine & Young, Peter Kuhn, MD

Dr. Peter Kuhn
USC Iovine & Young Academy
"Undergraduate degree program at the USC Jimmy Iovine and Andre Young Academy for Arts, Technology and the Business Innovation in partnership with the Convergence Science Initiative in Cancer."
Grant Amount: $425,654

The purpose of this new degree program is to expand the USC Iovine and Young Academy's innovative immersive cancer module to a full-scale four-year undergraduate track that applies the Academy's proven interdisciplinary educational model to the field of health to drive innovation in patient care. The undergraduate track is looking to launch as early as Fall 2019.

With seed funding from PHASE ONE, the Academy will build on the success and momentum of its immersive cancer module and broaden its interdisciplinary academic curriculum and co-curricular offerings in partnership with USC's Convergent Science Initiative in Cancer (CSI-Cancer) led by Dr. Peter Kuhn and the USC Dornsife College of Letters, Arts and Sciences. Students will be immersed in an information-rich, hands-on learning ecosystem where they interact with teams of physicians, scientists, cancer patients, UX/UI designers, and graduate students to develop innovative health solutions to improve patient outcomes.

"The future of cancer research rests on interdisciplinary innovation and collaboration," said Dr. Peter Kuhn, a dean's professor of biological sciences for the USC Dornsife College of Letters, Arts and Sciences, professor of medicine for the Keck School of Medicine, professor of biomedical engineering and professor of aerospace & mechanical engineering for the Viterbi School of Engineering and founding faculty for the Michelson Center for Convergent Bioscience.

+ Triple Negative Breast Cancer, City of Hope, Yuan Yuan, MD

City of Hope
"Biomarker correlative studies for a trial of immunotherapy in combination with a selective androgen receptor modulator in patients with advanced androgen receptor positive triple negative breast cancer"
Grant Amount: $150,655

Triple negative breast cancers (TNBC) (estrogen, progesterone and HER2 negative), are typically not responsive to targeted therapies, can be more aggressive, and are more likely to recur than other breast cancer subtypes. To counter these challenges, TNBCs have been suggested to be promising targets for immunotherapy, as well as targeting the Androgen Receptor in Androgen Receptor Positive TNBC, after it was found that this tumor type is highly infiltrated by immune cells. Extending the reach of a current phase 2 study for treating these patients with a combination of immunotherapy along with the androgen receptor modulator, this work will analyze immune cell characteristics and biomarkers before and after treatment in order to predict response. Once these predictive biomarkers are identified, this will help assess patient’s response and potential efficacy of immunotherapy.

+ Neuroblastoma, Children’s Hospital Los Angeles, Araz Marachelian, MD

Children’s Hospital Los Angeles
"Immunotherapy with Natural Killer Cells for Treatment of Neuroblastoma"
Grant Amount: $61,416

Neuroblastoma is the most common extra-cranial solid tumor in children. Patients with high-risk features continue to have an expected survival of 50% despite intensive therapy. The addition of immunotherapy utilizing the recently FDA approved anti-GD2 mAb ch14.18 Unituxin has improved outcome. However, long-term disease free survival remains a significant problem and improving immunotherapy further is needed for complete disease eradication of disease. Adoptive immunotherapy with natural killer (NK) cells has emerged as a promising anti-cancer treatment with broad anti-cancer potential. Until recently, the clinical efficacy and effective application of NK cell immunotherapy has been limited by the inability to obtain sufficient cell numbers for adoptive transfer. We have developed a novel expansion method for NK cells that solves this problem using artificial antigen presenting cells to express stimulatory molecules that promote sustained proliferation of NK cells in the laboratory. This expansion can be performed from blood from a simple venipuncture that can then be sent to the central laboratory. This phase 1 study will be the first study to combine cellular therapy with Unituxin in the treatment of patients during which we will identify the maximum tolerated dose of NK cells to be given with Unituxin and determine the immune effects of the combination therapy for patients with neuroblastoma. It will also be the first multi-center pediatric trial with cellular therapy and will help pave the way for other cellular therapy trials in pediatrics. The trial will be conducted through the NANT consortium (, a multi-institution consortium (centered at CHLA) whose goal is identify new therapies for patients with relapsed and refractory neuroblastoma and to improve their outcome. If successful, this therapy could then be utilized for all high-risk neuroblastoma patients to eradicate residual disease. The cost of this trial exceeds $1 million, but funding from Phase One foundation would be used as a component of the budget.

+ Pancreatic Cancer, Cedars-Sinai Medical Center, Richard Tuli, MD, PhD

Cedars-Sinai Medical Center
"A Phase 1 Study Of Veliparib (ABT-­‐888) in Combination with Gemcitabine and IMRT in Patients with Locally Advanced Pancreatic Cancer"
Grant Amount: $323,423

Pancreatic adenocarcinoma is a devastating disease with the lowest survival rate of any solid cancer. Gemcitabine chemotherapy and radiotherapy given concurrently remain the mainstay treatments for patients with locally advanced pancreatic cancer, which cannot be removed by surgery, but also has not spread to other areas of the body. Both gemcitabine and radiotherapy kill tumor cells by interfering with their DNA, the genetic blueprint. Veliparib is a poly (ADP-ribose) polymerase (PARP)1/2 inhibitor; PARP1/2 proteins are also involved in DNA repair. Studies have shown that inhibiting PARP1/2 activity can significantly enhance the effects of radiotherapy and chemotherapy in killing tumor cells. Our own laboratory work has shown excellent synergy between veliparib (a PARP inhibitor), radiotherapy and gemcitabine in killing pancreatic cancer cells grown in culture, and in prolonging survival in mice with pancreatic tumors.

Based upon these promising results and with the generous support of the PHASE ONE Foundation and Diane V. Allen, Dr. Richard Tuli and his team are currently enrolling patients in a first-in-human, investigational clinical study (phase I) to test the safety and efficacy of veliparib in combination with gemcitabine and radiotherapy in patients with locally advanced pancreatic cancer. Prior to and during therapy, patient tumor and blood samples will also be tested for levels of different DNA repair proteins and mutations, such as BRCA1/2. This molecular “signature” will hopefully serve as a “biomarker” to predict how the patient and tumor are responding to treatment. We are hopeful that we will be able to select out the subset of favorable patients who respond to this regimen, and design future studies (phase 2/3) by “personalizing” treatments using PARP1/2 inhibitors.

+ Brain Cancer, City of Hope, Jana Portnow, MD

City of Hope
"Phase I Study of CD-Expressing Neural Stem Cells in Combination with Oral 5-FC in Patients with Recurrent High Grade Gliomas"
Grant Amount: $66,000

“My first trial patient – Jenny, from Seattle, had glioblastoma. She didn’t think the treatment could help her, but she wanted to give hope to other patients who could be helped. Jenny died two months after treatment, but her involvement kick-started the study. News coverage of her situation drew participant interest from all over.” Dr. Jana Portnow

Treating brain tumors with chemotherapy presents special challenges because the blood-brain barrier restricts which chemicals can reach the vast majority of our brain cells. The barrier consists of a network of junctions between our capillaries and the brain cells that they supply with blood. These junctions possess an electrical resistance that permits water, certain gases, lipids, glucose and some amino acids to pass through, but other substances are blocked – creating a significant hurdle to delivering medication.

Phase ONE Foundation immediately saw the potential benefits of Dr. Jana Portnow’s proposal to use neural stem cells to circumvent the blood-brain barrier and deliver targeted chemotherapy to tumor sites.

This novel treatment involves surgically infusing specially designed stem cells into the brain, and then waiting one week for them to migrate to the tumor sites – to which they are naturally drawn. The patient is then given an anti-fungal compound that is known to be able to pass through the blood-brain barrier. Once it reaches the tumor, the stem cells convert the compound into a chemotherapy drug that acts against the tumor.

Stem cell therapies have typically been used to replace or regenerate damaged tissue; in this case, they are creating a gateway to cellular repair that is unique to the treatment needs of the brain.

The trial began in 2010 with the first-in-human study and concluded in January of 2013 with 15 enrolled patients. In addition to the groundbreaking work being conducted within the brain, Dr. Portnow’s team also tested whether they could use a microcatheter to provide a continuous supply of stem cells to the brain in order to eliminate the need for a surgical infusion each time. Indeed, their work with the microcatheters was able to document proof-of-concept.

“I got involved in research because I can’t imagine how any oncologist cannot be involved in it, given the difficulties you witness during treatment, said Dr. Portnow. “I intended to be an oncologist but, after working with so many patients, I realized that I had to try to do more to help them.”

Work continues in this promising field, with the intent that another trial group will soon be able to undergo treatment with microcatheter delivery of stem cells.

PHASE ONE’s support for Dr. Portnow’s stem cell therapy is dramatically improving our arsenal of responses to this form of cancer with a therapy that seems to have no significant side effects. This treatment can work alongside traditional therapies as well, and provide an additional layer of defense.

In 2014, brain cancer was the focus of PHASE ONE’s award-winning video – EveryONE Can Make a Difference. After its premiere, guests at the Gala were able to donate directly to Dr. Portnow’s work using their smartphones — an addition $67,000.

Funding is limited for this area of study, so Phase ONE Foundation’s support has been pivotal in advancing its progress in recent years. Without these resources – and the additional funds that have been leveraged with them, stem cell treatment of brain tumors might not have emerged as the promising therapy that it is.

+ Hamamatsu Nanozoomer 2.0 HT-Slide Scanner, City of Hope

City of Hope
Grant Amount: $75,000

PHASE ONE board member and former chair, Tia Palermo, passed away in 2012 following an 11-year battle with cancer. PHASE ONE wanted to honor her memory, and along with generous donations from friends and family, funded an extraordinary piece of equipment called the Hamamatsu NanoZoomer 2.0 HT-slide scanner at City of Hope, where Tia had been treated.

The T Cell Therapeutic Research Laboratory (TCTRL), directed by Tia’s physician Dr. Stephen Forman, is developing novel immune-based therapies for the treatment of cancer—specifically for the treatment of hematological malignancies, gynecological cancers and brain tumors. Their pre-clinical and clinical research program focuses on engineering human T cells to recognize and destroy tumor cells, and, in first-in-human clinical trials, in which a patient’s own reprogrammed tumor-specific T cells are transferred back to that patient, have observed encouraging initial results. A current technical challenge for the research program is the high-throughput evaluation of human and mouse tumor and organ histology both before and after therapeutic intervention with T cells that have been genetically modified to attack tumors. To address this technical limitation, the goal was to purchase the Hamamatsu NanoZoomer 2.0-HT slide scanner (C9600-13)with fluorescence imaging module (L11600-21). This equipment has helped evaluate the amount of T cell and/or tumor cell engraftment in their in vivo models of cancer therapy, and the surface marker/antigen profiles of individual cells both in their in vivo cancer models as well as on primary human tumor samples. Specifically, this slide scanner and imager provides cell-level resolution critical for determining the frequencies of varying patterns of antigen expression, mapping antigen distributions between cells and on individual cells both before and after adoptive T cell therapy in clinical trials and/or animal experiments.

The Hamamatsu unit combines high capacity (210 slides per run) for array tomography mapping of antigen expression relative to tumor structures, high resolution (0.46μm/pixel or 0.23 μm/pixel), and high sensitivity withrelatively increased speed with TDI (Time Delay Integration) line scanning. Cell-level resolution is critical for determining frequencies of varying patterns of antigen expression, mapping antigen distributions between cells and on individual cells. The COH Light Microscope and Digital Imaging Core, while well-equipped for conventional confocal microscopy, lacks the capability for scanning wide fields at high spatial resolution and high-throughput. Overall, this slidescanner and imaging unit is extremely important for their research mission of developing cancer immunotherapies.

+ Ewing’s Sarcoma, City of Hope, Anna Pawloska, MD

City of Hope
Ewing’s Sarcoma
Grant Amount: $37,000

Ewing’s sarcoma is the second most common primary bone tumor in children and adolescents. While advances in treatment have increased survival rates for children with localized disease to nearly 70 percent, fewer than 30 percent of patients who experience relapse or the spread of their cancer survive. In addition to these grim statistics, there is no nationally accepted “gold standard” of treatment for patients with advanced Ewing’s sarcoma. More effective therapies are urgently needed to save the lives of young patients.

City of Hope will be launching a five-year pilot clinical trial to study the effectiveness of a promising treatment for high-risk patients with advanced Ewing’s sarcoma: whole-body MRI-guided radiation therapy followed by autologous hematopoietic cell transplant, with a novel combination of high-dose chemotherapy. Led by Anna Pawlowska, M.D., Clinical Professor of Pediatrics, the proposed study will assess whether this therapy is feasible, safe and likely to increase patient survival rates. A similar approach tested in Germany and Austria increased children’s survival rate to 70 percent after two years and 56 percent after three years with minimal toxicity, but this approach has never been tested in the United States.

In a novel approach, guests at the 2013 PHASE ONE Gala DIRECTLY donated $37,000 to help fund part of this five-year trial by supporting MRI tests for patients not covered by their insurance and/or support a portion of the salary of a Clinical Research Assistant. This critical seed funding would help young patients with Ewing’s sarcoma access a promising new therapy now and start collecting the patient data needed to secure additional public and private funding for future years of the trial.

+ Kidney Cancer, UCLA, Joseph Riss, MD

University of California Los Angeles
Department of Urology
Kidney Cancer
Grant Amount: $310,000

Despite advancement in targeted treatments, metastatic kidney cancer poses a therapeutic challenge due to its resistance to conventional models of treatment. As long-term survival for patients with metastatic kidney cancer remains poor, UCLA Urology’s Allan Pantuck, MD and Arie Belldegrun, MD, along with Joseph Riss, PhD, have been working for years to develop innovative methods of therapy. With the assistance of critical funding provided by the PHASE ONE Foundation, this research team developed a kidney cancer vaccine that was recently approved by the FDA and given to the first patient in February 2013. Even with this grand milestone, the researchers must now monitor the effectiveness of the vaccine, especially given the kidney tumor’s resistance to current therapeutics. With the continuous support of the PHASE ONE Foundation, Dr. Riss and his team will use previously identified indicators to develop diagnostic tools for monitoring the new treatment and aim to develop a next-generation vaccine that will combat tumor resistance in metastatic kidney cancer.

+ Urothelial Cancer, UCLA, Karim Chamie, MD

University of California Los Angeles
Department of Urology
Urothelial Cancer
Grant Amount: $142,350

Dr. Karim Chamie, with UCLA Urology, is working to combat the rapid increase in recent years of urothelial cancer by investigating a potentially new treatment method involving a hydrogel polymer (chemical compound), which is liquid at room temperature, mixed with chemotherapy to treat this difficult-to-detect and highly reoccurring form of cancer. The polymer-chemotherapy combination takes on the shape of the cavity of interest (such as a kidney) at body temperature and has the potential to deliver chemotherapy to the targeted site while minimizing side-effects. The polymer can also be mixed with a dye that binds to tumor cells to facilitate detection of tumor cells within a specific organ. Traditionally, what has been most challenging in treating this type of cancer is the ability to deliver the chemotherapeutic agent at a high concentration level to the exact tumor site. This new discovery has the potential to overcome the previous challenge by using the polymer-chemotherapy directly to the targeted site. Dr. Chamie and his team are optimistic in pursing this study and are grateful to the PHASE ONE Foundation for supporting these efforts.

+ Melanoma, UCLA, Antoni Ribas, MD

University of California Los Angeles
Grant Amount: $252,674

With the support of the PHASE ONE Foundation Dr. Antoni Ribas and his team will conduct a phase 1 clinical trial (MART-1 F5 TCR Engineered Adoptive Cell Transfer Therapy with CTLA4 Blockade) with the goal of genetically programming the human immune system to efficiently target cancer cells in patients with advanced stage melanoma, the deadliest of skin cancers. This clinical trial builds upon the experience at UCLA over the past 3 years in the use of T cell receptor (TCR) engineered adoptive cell transfer therapy. This therapy is manufactured in-house and results in the generation of a large army of immune system cells that are genetically redirected to recognize and kill melanoma. With this therapy we have noted high initial response rates, but tumors come back. A major limitation has been a progressive decrease in the antitumor functionality of the TCR engineered immune cells. The antibody tremelimumab blocks a major negative regulator in immune cells and will be tested with the goal of maintaining the cancer killing functionality of the TCR engineered lymphocytes given to patients. This protocol has administrative approval by the UCLA regulatory committees and it has already been filed and approved by the Food and Drug Administration.

+ Brain Cancer, University of Michigan School of Medicine, P.R. Lowenstein, MD, PhD & Maria G. Castro, MD, PhD

University of Michigan School of Medicine
Department of Neurosurgery and Cell and Development Biology Brain Cancer
Grant Amount: $216,000

“PHASE ONE has thus provided the green light to move the dreams of two basic scientists into the challenging arena of clinical trials.” Drs. Pedro Lowenstein and Maria Castro

Highly malignant brain tumors are uniformly lethal. Patients usually survive less than 2 years post-diagnosis, with very few still alive 5 years later. In spite of constant developments in surgery, radiotherapy and chemotherapy a breakthrough for this particularly aggressive cancer has not been yet discovered.

Over the last ten years Dr. Pedro Lowenstein and Dr.Maria Castro have developed a novel approach for the treatment of brain tumors. Based on an evolutionary and developmental understanding of the structure and function of the brain immune system, they decided to restitute to the brain those immune cells normally absent from it, as a potential new approach to treat brain tumors. The immune cells normally absent from the brain are those in charge of turning on the anti-tumor immune response. Their absence from the brain helps us explain why normally anti-brain tumor immune responses are ineffective. By attracting those specific immune cells to the brain and the brain tumors, they expect them to identify brain tumor antigens and unleash a therapeutically effective immune attack to eliminate brain tumors.

To achieve this Drs. Lowenstein and Castro have turned adenoviruses which normally can cause lung infections and other diseases into therapeutic agents. They have engineered the viruses to deliver directly to the brain and the brain tumors a protein that will recruit the missing immune cells to the brain to start a clinically effective immune response. As their work uses viruses as agents of treatment to induce immune responses, their therapeutic approach is described as a “combined gene-and immune therapy”. Following more than ten years of development and experimental testing they are now ready to start clinical trials in patients suffering from glioblastoma multiforme.

With very generous support from the PHASE ONE Foundation, they are now on the threshold of starting the first ever clinical trials of this new therapeutic approach for the treatment of deadly highly malignant brain tumors. The proposed trial, which was approved by the Food and Drug Administration on April 7, 2011 is currently undergoing the final evaluations by the Institutional Biosafety Committee, and the Institutional Review Board for Clinical Trials. They expect these evaluations to be finalized within the next few weeks. Once our trial has cleared all necessary Committee reviews, it will be ready to start.

“PHASE ONE has thus provided the green light to move the dreams of two basic scientists into the challenging arena of clinical trials. As the trial starts enrolling patients we will look forward to keeping PHASE ONE abreast of all therapeutic developments. We hope, and for the sake of the patients, we pray, that treating the first patients may represent in Winston Churchill’s prophetic words, Now this is not the end. It is not even the beginning of the end. But it is, perhaps, the end of the beginning.”

+ Leukemia, Children’s Hospital Los Angeles, Stuart E. Siegel, MD

Grant Amount: $410,000

In February 2010, PHASE ONE granted $410,000 over two years to fund a Phase I study at Children’s Hospital Los Angeles. The study, led by Dr. Stuart E. Siegel, is of AC220 for children with relapsed or refractory acute lymphoblastic leukemia (ALL) or acute myeloid leukemia (AML).

This grant will provide an opportunity in getting a second generation FLT3 inhibitor drug tested in a pediatric population that truly focuses on the concept of FLT3 inhibition in high-risk ALL and AML patents. The goal of the study is to obtain sufficient laboratory and clinical data to enable the design of a larger Phase II study. Results from this study would also leverage funding to support the Phase II part of this project from both the National Cancer Institute, as well as the pharmaceutical company Ambit that makes the drug.

+ Lung Cancer Initiative, Premiere Oncology Foundation

Lung Cancer Initiative
Lung Cancer
Grant Amount: $266,500

Premiere Oncology Foundation, a non-profit dedicated to cancer research, integrative medicine, patient/family support services and community education, will utilize the grant to support a comprehensive lung cancer program to include multi-disciplinary research.

Lung cancer is the world’s top cancer killer, claiming more than 1.3 million lives per year, yet few financial resources have been dedicated to this disease. The Premiere Oncology Foundation Lung Cancer Initiative focuses on the use of FDA-sanctioned novel treatment regimens in collaboration with experts in the field of lung cancer detection and treatment. The program will foster dialog among practitioners caring for lung cancer patients, and provide a means for seamless interaction with medical and radiation oncologists, thoracic surgeons, radiologists, pulmonologists and primary care physicians caring for these patients.

Dedicated to whole-patient care and recognized for expediting clinical trials, Premiere’s flagship cancer center in Santa Monica offers patients the newest cutting-edge treatments, rapid access to novel, internationally available therapies and integrative medicine all under one roof. Additionally, Premiere provides services in its Scottsdale, Arizona center and research affiliation with Pacific Hematology & Oncology Associates in San Diego.

+ PHASE ONE Endowed Chair, The Samuel Oschin Comprehensive Cancer Institute

Phase One Endowed Chair
Cedars-Sinai Medical Center
Grant Amount: $2,000,000

“The history of Cedars is quite long, but for 90 years of that history or more they didn’t have an academically oriented cancer center… This is the first era where the organization has had a conceptually guided…benchmark, if you will, a nationally reviewed…program.” Dr. Steven Piantadosi

Phase ONE’s funding objective emphasizes promising early-stage research, but it also recognizes the importance of fostering systemic change in our nation’s cancer research infrastructure. Our grant to establish the Phase ONE Endowed Chair at the Samual Oschin Comprehensive Cancer Institute (SOCCI) at Cedars-Sinai Medical Center in 2007 has been instrumental in propelling its move toward conducting comprehensive phase one research. Researcher Steven Piantadosi, MD, Ph.D, was recruited from Johns-Hopkins Medical Center in 2008 with the offer of the Phase ONE Endowed Chair. A renowned cancer researcher who spent part of his diverse career at the National Institutes for Health, his immediate goals were to prepare Cedars-Sinai’s to apply for a National Cancer Institute (NCI) designation, and to foster a cultural shift within the medical center to emphasize the value and importance of phase one cancer research for patients. Over the long term, Dr. Piantadosi envisions SOCCI as a leading center for phase one cancer research in the nation. Since accepting the Phase ONE chair, Dr. Piantadosi has made significant progress towards these goals. With this new spotlight on SOCCI, Cedars-Sinai has already recruited a number of physicians with an interest or specialty in bench research. “Not only getting them here, but also getting them to interact in a constructive way with each other,” said Dr. Piantadosi. “I think the personality of cancer or care or investigation here has changed as a result of that mission.” He added, “I think it is better. I think it is more scientific. I think it is better connected nationally with both clinical care standards and research activities nationally.” Indeed, the growing influence of research at this medical center is resonating throughout traditional centers of excellence, and Phase ONE’s early investment has been integral to this growing momentum. Through Dr. Piantadosi’s efforts, the number of Cedars-Sinai patients enrolled in cancer trials has doubled in the past seven years, and will double again in the next four years. He expects to apply for an NCI designation as early as 2018, and a successful application will facilitate access to resources that will rapidly escalate phase one research at Cedars-Sinai. Phase ONE’s early investment and partnership with SOCCI is not just expanding access to one promising study, it has helped to create a platform for countless, future studies with wide-ranging and potentially life-saving benefits.

+ Phase I Solid Tumor Program, City of Hope, Yun Yen, MD, PhD

Comprehensive Cancer Center
Grant Amount: $345,000

The City of Hope Division of Medical Oncology and its Department of Clinical and Molecular Pharmacology, directed by Yun Yen MD, PhD, is the City of Hope Comprehensive Cancer Center equivalent to a Phase I Solid Tumor Program. In this program is the exciting translation of molecules from the laboratory to the clinic.

City of Hope will use the PHASE ONE funds to support novel Phase I clinical trials developed in collaboration with the Department of Clinical and Molecular Pharmacology. These resources would be utilized to fund novel protocols or existing protocols where additional funds could translate findings from the laboratory to the clinic. Support for novel “NEW” programs (25–50K/ year for two years) in Clinical and Molecular Pharmacology that can reach the clinic during the two years of the award, and bring innovation from our laboratories will be the highest priority.

The City of Hope Comprehensive Cancer Center’s Phase I Program has a commitment to the Developmental Therapeutics and Hematologic Malignancies Programs directed by Yun Yen MD, PhD, Richard Jove PhD, and Steven Forman MD. City of Hope will establish competitive awards (25–50K/year for two years) that combine a laboratory and clinical Principal Investigator that translate laboratory observations into the clinic during this period. Programs in Solid Tumors and Hematologic Malignancies including lymphomas, in both adults and children would be included.

+ Prostate Cancer, UCLA, Kuk-Wha Lee, MD, PhD

University of California Los Angeles
Grant Amount: $250,000

Kuk-Wha Lee, M.D. PhD. is a graduate of the Medical Scientist Training program at Loma Linda University School of Medicine, obtaining her PhD and MD. Her clinical training is in pediatrics and she was fellowship trained in Pediatric Endocrinology at the Mattel Children’s Hospital at UCLA. In training she was the recipient of the prestigious Giannini Foundation Fellowship, Stein-Oppenheimer Award, and the LWPES Clinical Scholar Award for her research. In addition, she is the recipient of the UCLA Prostate Cancer SPORE Career Development Award for her research in cellular and animal models of human prostate cancer. Her laboratory studies the biological functions of Insulin-like Growth Factor Binding Protein-3 (IGFBP-3) and its binding partners in cancer biology, specifically prostate cancer. Specifically, she studies the molecular mechanisms by which this molecule enters cells; targets specific areas within cancer cells (e.g. the nucleus and mitochondria); and initiates the programmed cell death cascade. Unique assays in their laboratory have been developed to study serum levels of these molecules and also have developed unique transgenic and knockout mouse models of human disease. Based on preliminary work published from their laboratory the plan is to initiate a Phase I trial of IGFBP-3 in men with prostate cancer in the near future.

+ Lung Cancer, City of Hope, Karen Reckamp, MD, MS

City of Hope
University of California Los Angeles
Grant Amount: $500,000

“A few other investigative teams have benefited [from our work] around the world. The results are globally helpful.” Dr. Karen Reckamp

Cancer treatment breakthroughs typically occur with the discoveries of new drugs or radiology techniques but, thanks to the groundbreaking work of Dr. Karen Reckamp, we are witnessing the development of an innovative lung cancer treatment based on the well-known arthritis medication Celebrex.

Celebrex was introduced in 1998 as an anti-inflammatory. While at the University of California, Los Angeles, Dr. Reckamp approached Phase ONE Foundation with a novel proposal for treating lung cancer with Celebrex to boost the body’s own immune response to cancerous tissues. After completion of the pre-clinical work at UCLA, Dr. Reckamp moved the trial to the City of Hope where she oversaw the phase one study with seven enrolled patients.

More men and women die each year from lung cancer than any other form. It takes the lives of more people than colon, breast and prostate cancer combined, and the survival rate has remained at a discouraging 15% for far too long. This cancer manifests itself as an inflammation of the lung tissues. Under normal circumstances, our body’s immune system is designed to attack the source of the inflammation and restore our good health. In this case, lung cancer cells can mask themselves from our immune system; our body never identifies the problem and the cancer grows unchecked.

Dr. Reckamp’s research and trial determined that Celebrex’s anti-inflammatory effects could reveal the cancer cells to our immune systems. Once they were unmasked, the body’s natural immune response was triggered and the cancer cells were attacked, leading to a reduction in cancer tissue.

Just 10 years ago there was little interest and a scarcity of funding for this area of research. Because of results like we are seeing from this research – which is the direct result of Phase ONE Foundation’s support, there are now other immune-modulating drugs in development that may prove even more effective in treating lung cancer.

“The goal of this study was not to observe tumor shrinkage directly, but to record the activation of the patients’ immune systems – which was observed in direct response to the treatment,” said Dr. Reckamp.

Phase two trials, which tested the efficacy of Celebrex and another drug, are already complete. As of 2015, a phase three study is underway with Celebrex and the same secondary drug, but in differing dosages. Because Celebrex was already approved by the FDA, the process for studying its secondary effects was launched more quickly than in a typical trial.

Funding from Phase ONE Foundation made it easier to secure additional grants from other supporters, and the fact that Dr. Reckamp was at both UCLA and the City of Hope during the course of this study meant that the two institutions could collaborate and enroll more patients in the trial.

+ Molecular Research, UCLA, Charles Sawyer, MD and Ingo Mellinghoff, MD

University of California Los Angeles
Jonsson Comprehensive Cancer Center
Grant Amount: $250,000

UCLA Jonsson Comprehensive Cancer Center Hematology/Oncology Translational Scientists working on molecular mechanisms that underlie the development and spread of cancer.

+ Gastro-Intestinal Malignancy, USC, Heinz-Josef Lenz, MD & Daniel Vallboehmer, MD

University of Southern California
Norris Comprehensive Cancer Center
Gastro-Intestinal Malignancy
Grant Amount: $250,000

USC/Norris Comprehensive Cancer Center Specialists, Dr. Heinz-Josef Lenz and Dr. Daniel Vallboehmer, in Gastro-Intestinal Malignancy searching for molecular markers to predict prognosis and response to therapy.

+ Kidney Cancer, UCLA Arie Belldegrun, MD & Alan Pantuck, MD

University of California Los Angeles
Kidney Cancer
Grant Amount: $250,000

UCLA Department of Urology, Dr. Arie Belldegrun and Dr. Alan Pantuck, specializing in New Treatments for Kidney and other genitourinary cancers, particularly vaccine therapies directed against newly identified targets.

+ Prostate Cancer, Cedars-Sinai Medical Center, David B. Agus, MD & Mitchell Gross, MD

Cedars-Sinai Medical Center
Prostate Cancer
Grant Amount: $250,000

Cedars-Sinai Prostate Cancer Program, with Dr. David Agus and Dr.Mitchell Gross, are looking at innovative treatments for prostate cancer and using information technology systems to identify molecular markers for response to therapy.

+ PT/CT Scan Research, Cancer Institute Medical Group, Lee Rosen, MD

Cancer Institute Medical Group
Grant Amount: $276,250

In 2003, Dr. Lee Rosen conducted a Phase I study of a medication that could theoretically shut down the development of new blood vessels from which tumors grow and possibly spread. Using a series of radiographic assessments including CT/MRI/Bone scans, etc. as well as conventional scans such as CT/MRI, Dr. Rosen and his colleagues at the Cancer Institute Medical Group, examined the response of their patients to this new medication and to what degree. He also found the use of PET scans, which look at changes in tumor metabolic activity rather than size, is very effective, PHASE ONE has been sponsoring a trial which will look at whether a combination of PET/CT scans can aid in the assessment of the drug’s efficacy more quickly than conventional CT scanning alone.

+ Lymphoma, UCLA Jonsson Comprehensive Cancer Center, Sven de Vos, MD, PhD, Christos Emmanouilades, MD, William H. McBride, PhD, DSC & Jonathan W. Said, MD

University of California Los Angeles
Jonsson Comprehensive Cancer Center
B-Cell Lymphomas
Grant Amount: $150,000

PHASE ONE sponsored an Indisciplinary Grant to study the use of the Proteasome Inhibitor PS-341 in the treatment of refractory diffuse large B-cell lymphomas in terms of clinical efficacy, mechanisms of resistance and new models of combination therapy. The study was conducted by principal investigator, Sven de Vos, M.D., and co-investigators, Christos Emmanouilides, M.D., William H. McBride, M.D. and Jonathan W. Said, M.D. at the Jonsson Comprehensive Cancer Center.

+ Sponsored Recruitment of Clinical Research Physician, UCLA, Carolyn Britten, MD

University of California Los Angeles
Jonsson Comprehensive Cancer Center
Clinical Research Physician
Grant Amount: $175,000

PHASE ONE sponsored the recruitment of a full-time clinical research physician for a full year. Highly respected oncologist, Dr. Carolyn Britten, joined the faculty of the Jonsson Comprehensive Cancer Center in early 2001 and PHASE ONE funded her research for one year. Dr. Britten is involved in four promising Phase I trials testing new and unique therapeutic strategies in signal transduction and angiogenesis.