Cancer researchers receive grant to develop immune-based therapies for deadly brain tumors

Researchers from the UCLA Brain Tumor Center received a $400,000 grant from the PHASE ONE Foundation to support their research in developing immunotherapies for glioblastoma, an aggressive and fast-growing type of brain tumor.

The grant, which is led by Dr. Linda Liau, chair of the neurosurgery department at the David Geffen School of Medicine at UCLA, and Dr. Timothy Cloughesy, a professor of neuro-oncology at the Geffen School of Medicine, helps fill an urgent need for novel and innovative approaches to better treat people diagnosed with the disease.

The average lifespan for someone with glioblastoma is often measured in months and less than 5% of people with glioblastoma live longer than five years. There are limited treatment options and there is a high probability that the tumor will come back after initial treatment. Currently, there is no standard therapy for recurrent glioblastoma.

“We are extremely grateful to receive this grant from PHASE ONE,” said Liau, who is also a scientist at UCLA’s Jonsson Comprehensive Cancer Center. “With this support, we have the potential to change the way glioblastoma is treated and provide patients with a new therapy that has the potential to support long-term survival.”

The funding will help open a clinical trial testing a combination treatment strategy using checkpoint inhibitors in conjunction with a personalized dendritic cell vaccine, which was developed by Liau at UCLA. The team hopes by combining the two treatments they will be able to create a new way to treat people with brain cancer, as well as develop new ways to track the immune response.

“We already have had preliminary success using checkpoint inhibitors to treat patients in a previous clinical trial,” said Cloughesy, who is also a scientist at the Jonsson Cancer Center. “By combining the two immune-based treatments, we hope to bring in more T-cells that will attack cancer cells that would otherwise go unnoticed by the body’s immune system.”

“Dr. Liau led the trial that gave my late husband seven healthy years, post terminal brain cancer diagnosis. At the time, she was a pioneer in the immunology approach to treatment, and I have watched her research progress to be even more effective and collaborative.  She is a uniquely brilliant, compassionate and forward-thinking doctor and researcher, and I am incredibly pleased that PHASE ONE is able to help fund her next wave of research.  Brain cancer is such a fierce and fickle disease, that requires aggressive, nimble and competent treatment – and her approach encompasses all of these qualities.  I’ve witnessed her first group of patients from trials in the early 2000s exceed life expectancy and continue to thrive, which is proof that this is working, and that her efforts using a layered approach to finding a cure should be continued,” said  Meritt Elliott, Board Member of PHASE ONE and co-founder of THE GREAT.

The Business of Clinical Trials: An Interview with Bartosz Chmielowski, MD, PhD

Dr. Bartosz Chmielowski, MD, PhD, is an Associate Professor of Medicine at UCLA. Currently involved in over 20 clinical trials, Dr. Chmielowski specializes in sarcoma, melanoma, and other types of skin cancers.

Q: Only 8% of cancer patients currently participate in clinical trials. Why do you think that number is so low?

In the US, you are paid for what you do, and not paid for what you don’t do. Physicians make money from seeing and treating their patients, but if a physician refers their patient to a clinical trial, or an academic center, they lose the income. This often leads physicians to recommend approved therapies that they can administer themselves.

There are also patients who are disinterested in joining a trial because trials are viewed as a medical experiment or a last resort. However, all trials are reviewed by scientific and ethical committees and approved for their target population. Notably, clinical trials are more time consuming due to more office visits and lab work, which means trial patients are under much closer supervision than patients on standard therapy.

Q: For those patients who don't know how to find a trial, what is the best resource for them to use?

The best resource is clinicaltrials.gov. Everyone who opens a trial in America must register with the website and include some basic information. However, it is still difficult for patients because they can use the site to search for trials under the name of their cancer, but then receive hundreds of results and not know which to choose.

Oncologists are not a perfect resource either because it's impossible for one to encompass knowledge of all open clinical trials while also running their practice. My recommendation is that it’s always better to go to an oncologist that specializes in the patient's disease.

Q: Is there a difference between trials run by drug companies vs. academic medical centers?

A main difference stems from multiple drug companies targeting the same common cancers, due to the high demand from patients. These companies use drugs of similar mechanisms and they all need patients to run their trials to get their drug approved first. This competition can slow the process down.

Q: What should a patient know when entering a Phase I trial?

The earlier the trial the more complicated it is. Doctors will enroll patients in cohorts because if you have a new drug you don't know the toxicity, and you don't want to expose so many people to the new drug all at once. Instead, you enroll a small number of patients at first, three or so, and if the patients don’t experience any side effects then you enroll the next three, and so on.

This may cause a delay in starting the new treatment, as a trial may be open, but if a patient comes just after the first three patients were enrolled, then the next slot may not be open for another month. We don't want to leave patients in limbo, and that's why keeping all their doctors and specialists informed is key.

Q: Are there any positive takeaways you noticed from your own practice?

My practice is busier and busier and it’s not necessarily that we have more patients being diagnosed with cancer, but because our patients are living longer - and this is what we always want! Five or seven years ago if a patient was diagnosed with metastatic melanoma, only 25% of them survived one year. Now it's 75% of patients who survive one year, so of course the patients come back to the clinic, making the clinic busier because of our success!

Success with Sharsheret

Last June, PHASE ONE gave Sharsheret a grant of $50,000 for their pilot program distributing free kits to women recently diagnosed with ovarian and breast cancer. Sharsheret is a national not-for-profit organization that improves the lives of Jewish women and families living with or at increased genetic risk for breast or ovarian cancer through personalized support and saves lives through educational outreach. They also specifically aim to highlight and educate the medical community on the unique experiences of Jewish women with these cancers, as there is a large genetic and cultural component.

As a result of our funding, over the last year Sharsheret has expanded its network and formed partnerships with more local medical practices and cancer centers. Sharsheret provides multiple types of kits, including Financial Wellness Toolkits, Best Face Forward Kits, and Busy Boxes - each tailored to a different part of the healing and self care process for women with cancer. They streamlined the kit distribution process by having one standard mailing piece across all centers, which included sample kits and information packets for patients.

The outlook from this pilot year is optimistic. Sharsheret exceeded their goal, distributing 372 kits! The feedback from the women who received support has been extremely positive. 

            “I want to thank you so much for sending all the information and goodies. My scalp was so dry after the chemo, and I looked in the box of cosmetics you sent, and there was scalp oil in there. It was really helpful! When I got the diagnosis, it was shocking, terrifying, and overwhelming. The support from family, friends, and organizations helped me get through all of it, and I can’t thank you enough!” – Breast Cancer Patient, Stage 3

 The Best Face Forward Kit was one of the most requested. This supports the idea that cosmetic and wellness resources are a crucial part of the cancer journey and process. Informed by the success of initiatives including the PHASE ONE-funded program, Sharsheret is launching a new program, Best Face Forward 2.0. Its aim is to subsidize non-medical services that are important for a woman’s body image and quality of life.

Additionally, thanks to our seed funding, Sharsheret has gained recognition and been invited to join the Susan G. Komen of Los Angeles Diversity Committee, as well as the California Dialogue on Cancer’s Cancer Plan Treatment and Survivorship Subcommittee. This has led to a two-year partnership with Cedars-Sinai Medical Center to expand community health education and develop a Community Cancer Education Toolkit.  With the backing of PHASE ONE, Sharsheret has been able to further establish itself in the Los Angeles region and can reach more individuals than ever before. PHASE ONE looks forward to future partnerships with Sharsheret.

Meet our new Board Chair!

We are so excited to announce that Brad Meadow is the new Board Chair of PHASE ONE! Below is a brief interview to get to know Brad and see what’s ahead for our organization. (Photo above: Brad and Jodi Meadow at the 2019 One for All Gala, Cindy Gold Photography)

Q: How long have you been involved with PHASE ONE?

A: I have been on the Board since moving back to Los Angeles in 2012. But I have been supporting and following PHASE ONE since it was founded 20 years ago by my sister and brother-in-law.

Q: What other roles have you served as for PHASE ONE?

A: I have been on the Development Committee, the Speaker Series Committee, and I was one of the individuals who organized the first PHASE ONE Young Person event a few years ago.

Q: What made you get more involved in PHASE ONE?

A: I am continually inspired by the achievements that our Founders and Board members, both past and present, have accomplished over the years. I want to do my part in keeping that momentum going and expanding the breadth and reach of our organization.

Q: What are your goals as the new Chair of the Board?

A: Mainly to just continue the amazing legacy that our Founder and previous Board chairs have built and use that as the foundation for additional growth and opportunities for the organization.

Q: What is one ongoing project you are most excited about?

A: I am most excited about expanding the awareness and reach of PHASE ONE to a larger pool of donors, including corporate and other institutional support that we have not previously tapped into that much.

Q: What’s your favorite PHASE ONE Gala memory?

A: My favorite memory was when my brother-in-law Rick (who was my sister’s new boyfriend at the time) won a Mini Cooper at PHASE ONE’s 2007 Gala at the Beverly Wilshire. It was a pretty funny “welcome to the family” moment!

Q: What is your favorite music to listen to while you work?

A: I am a big Van Morrison fan and love all classic rock in general.

Testicular cancer awareness in a large urban school system: Evaluation of pilot program

In 2017, PHASE ONE funded and helped create a pilot program with LAUSD that educated high school students about testicular cancer. Due to such positive response, PHASE ONE funded the program again in 2018 to help introduce and expand the program into new schools in the district. Below you can read an abstract of the pilot program’s evaluation from the 2018 Annual ASCO Meeting, made available by the ASCO Meeting Library.

“Testicular cancer awareness in a large urban school system: Evaluation of pilot program.”

Authors: Kathleen Ruccione, Aaron Plant, Emerald Snow, Timothy Kordic, James Hu, Terry David Church, Stuart E. Siegel; Department of Doctoral Programs, School of Nursing, Azusa Pacific University, Azusa, CA; Sentient Research, West Covina, CA; Los Angeles Unified School District, Los Angeles, CA; University of Southern California Norris Comprehensive Cancer Center, Los Angeles, CA; University of Southern...

Background: Testicular cancer is the most common cancer among males age 15-34. Lack of understanding and awareness of testicular cancer and self-examination are barriers to early detection. A program aimed at increasing testicular cancer awareness and self-examination was created and implemented in the LAUSD in 2017. Effectiveness of this program is presented here.

Methods: Twelve classrooms at six high schools (34%-99% Hispanic) participated in the one-week program. Students completed a questionnaire (n = 1,382) before the program began and an identical questionnaire upon program completion (n = 1,338). The 16-item instrument measured knowledge and attitudes toward testicular cancer and self-examination. Male students completed three additional items about self-examination, self-efficacy, comfort speaking to a health provider, and self-examination in the past month. A 16-item teacher questionnaire was administered at the end of the program.

Results: Seven of nine knowledge/awareness items had statistically significant increases from the pre-test to post-test survey. The percentage of students strongly agreeing that testicular self-examination is important for men’s health increased from 53.8% to 75.5% (p < .001). Among male participants, there were increases in the percentage who strongly agreed they could recognize signs of testicular cancer (14.5% vs. 36.6%; p < .001) and comfort in speaking to a health provider about their testicular health also increased (27.4% vs. 36.0%; p < .001). The percentage of male students who had performed a testicular self-examination in the past month increased from 29.1% to 48.2% (p < .001). The teacher survey revealed a high level of satisfaction with the quality and content of the lessons, as well as possible ways to improve the program.

Conclusions: The results of this pilot program were promising. The evaluation showed increases in knowledge, awareness, and attitudes, and a nearly 20% increase in testicular self-examination among males. Next steps will include refinements to the program, followed by implementation with a larger sample with a more rigorous study design to determine if wide-scale dissemination of the program throughout the district and beyond is warranted.

PHASE ONE Helps Sharsheret Expand Work With Los Angeles Medical Centers

We are pleased to announce that Phase One has awarded a Community Grant to Sharsheret. This significant grant will allow Sharsheret to expand their work in reaching patients directly in Los Angeles-area medical centers while they’re receiving treatment, educating doctors about the unique concerns of their Jewish patients and providing them with Sharsheret’s free kits during their cancer journey.

“Sharsheret understands the importance of having a medical team that not only provides the highest level of care, but is also intimately knowledgeable about the unique issues and concerns of each of their patients,” said Sharsheret California Regional Director Jenna Fields. “Thanks to the Phase One Foundation’s grant, Sharsheret will be able to more deeply engage with patients in LA area medical centers and practices, providing critical psychosocial support for women and their families.”

The concerns of Jewish women facing breast and ovarian cancer include increased genetic risk and issues related to genetic testing; use of the ritual bath as it relates to spirituality, healing, and renewal; life with cancer in a close-knit community; and the effects of treatment decisions on fertility and child-rearing.

Healthcare professionals will provide patients with Sharsheret’s tailored kits, ensuring patients receive the resources they need when they need it the most.

Kits 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; and
Financial Wellness Toolkit.

About Sharsheret
Sharsheret, Hebrew for chain, a national cancer organization with three offices (California, Florida, and New Jersey), serves 80,000 women, families, health care professionals, community leaders, and students, in all 50 states. Sharsheret creates a safe community for women facing breast cancer and ovarian cancer and their families at every stage of life and at every stage of cancer – from before diagnosis, during treatment and into the survivorship years. While expertise is focused on young women and Jewish families, more than 15% of those served are not Jewish. All Sharsheret programs serve all women and men.

Women in Cancer: An Interview with Alexandra Drakaki, MD PhD

Dr. Alexandra Drakaki, MD, PHD, is a primary investigator of several clinical trials at UCLA.  She has achieved a number of successes in hematology and oncology, which have lead to significant breakthroughs and approval of novel drugs in the field. Fortunately, Dr. Drakaki made some time to answer a few questions for PHASE ONE. 

Q: What is the best cancer prevention tip you have ever shared and/or received?

Cancer is the disease of our time. People used to die from infections and cardiovascular disease in theirs forties and fifties, but now they live longer so they have higher risk of developing cancer. If we knew how to prevent it, nobody would have had cancer; however we know a few "tips." Lifestyle intervention strategies that may help to decrease risk of several cancers include avoiding smoking, alcohol, and obesity. Good sleep, meditation, and exercise play an important role as well in decreasing cancer recurrences as proven by many clinical trials in certain types of cancer, like breast cancer. However, my favorite tip that I ever received is: "Laughing is the best cancer prevention strategy."

Q: Do you have a favorite patient survivor story?

There are many wonderful stories. One of my special ones, was Ms. X. I met her in July 2014. She was in her late forties at the time, a beautiful lady full of life. As soon as I introduced myself to her as her new oncologist, I then gave her the bad news that her cancer had recurred and it was going to be terminal. Our relationship went through waves as you can imagine. She refused to accept the news. Her only son had just graduated from high school - she had to be there for him. We looked through the few available studies at the time for her rare cancer and eventually I enrolled her in a Phase I clinical trial. Time went by and we bonded like family. Her widespread cancer melted away within the first few cycles and now we are getting ready for her son's graduation from college. She is going to be proud, but most importantly, she is going to be there, four years later...cancer free!
 

Q: Is there a problem with conventional cancer screenings for certain types of cancer? 

In 2018, the simple answer is, "no." There is no problem with conventional screenings for certain types of cancer. Although, I would say the bigger issue is the lack of  screening modalities for cancer. One of the first screening tests, that positively proved itself with life saving results for early detection, was the Papanikolaou test for cervical cancer, followed by mammograms for  breast cancer, colonoscopies for colorectal cancer, PSA for prostate cancer (with a lot of debate about its use since it may identify "insignificant" cancers), and recently a CT of the chest for lung cancer screenings in high risk patients. Accordingly, and as you can see, the major problem in detecting cancer at an early stage is due to the lack of screening modalities for most cancer types. Although, there is hope that with the way research is progressing, we will be able to detect malignancies at an earlier stage through a simple urine, stool or blood test.

The Future of Urotheliar Cancer: An Interview with Dr. Karim Chamie. A 2013 PHASE ONE grant recipient

In 2013, PHASE ONE granted Dr. Karim Chamie, with UCLA Urology, a grant for $145,350 to help combat the rapid increase in urothelial carcinoma. Here is a recent update:

Q: Would you briefly describe your research?

In July 2012, I was appointed as Assistant Professor of Urology and my first grant out of fellowship was a PHASE ONE Foundation Award. I was awarded for the very innovative, but also very risky work of developing a novel reverse polymer gel matrix. This matrix is liquid in cold temperatures and solidifies at body temperature. Therefore, it can be mixed with any chemotherapeutic agent, while on ice, and quickly solidifies within a cavity of interest in the human body. The thought was to instill this into any cavity of interest to better deliver site-specific, anti-cancer therapy. PHASE ONE funded pre-clinical (mouse and porcine models) and clinical trials (compassionate use in humans) instilling this gel mixed with mitomycin C (a common chemotherapy for low-grade urothelial carcinoma) into the upper urothelial tract (renal pelvis and ureter). We have presented our findings at the American Urological Association, Society of Urologic Oncology, and at European Association of Urology meetings. Our findings have been published in peer-reviewed journals and have directly led to an IND application and a pivotal market-directed study—an international multi-institution phase III trial clinical trial measuring the efficacy of this novel compound for patients with low-grade upper tract urothelial carcinoma. The PHASE ONE Foundation has been essential in seeing this idea develop into a Phase III clinical trial. 

Q: What are the next steps?

We are now expanding the indication for high-grade urothelial carcinoma by examining the utility of combining a checkpoint inhibitor and the hydrogel polymer as local treatment. We have submitted a research proposal to the NCI through the investigative grant mechanism to determine the permeability and the subsequent effects on immune cell infiltrates of a checkpoint inhibitor that is delivered intravesically in a MB49 murine bladder cancer model. Preliminary data suggests that the checkpoint inhibitor does penetrate the bladder wall and demonstrate local efficacy without systemic toxicity. 

Q: What are your primary goals with your research?

My long-term goal is to develop, implement, and disseminate interventions that reduce the burden of disease for patients with genitourinary malignancies. With my recent promotion to Associate Professor, I want to thank the PHASE ONE Foundation for playing an instrumental role in early in my career.