Fielded: March 2015 Research Executed by: Survey Monkey Questions provided by: David Bernstein...

Fielded: March 2015Research Executed by: Survey MonkeyQuestions provided by: David BernsteinAnalysis provided by: David Bernstein

The Fly A Kite Foundation: DIPG Insights Survey

March 2015

The Fly A Kite Foundation: DIPG Insights Survey, March 2015

About the Fly A Kite FoundationThe Fly A Kite Foundation Inc. was founded in August 2014 as an IRS 501 (c)(3) non-profit charity by David and Deena Bernstein after the loss of their son Zachary. Zachary was an 11 year-old boy who was diagnosed with, and suffered from DIPG (Diffuse Intrinsic Pontine Glioma). DIPG is a type of brain tumor found in the Pons, the part of the brainstem near the lower back of the head near the top of the spinal cord. The Fly A Kite Foundation is dedicated to three main objectives:

Partners In ArtThe Fly A Kite Foundation provides custom art packages for children with pediatric brain cancers. These packages are intended to provide a creative and therapeutic outlet for children suffering from diminished motor function. We have partnered with Patricia Kearney, a Neuro-Occupational Therapist (Therapy Partner) who works for Transitions of Long Island (part of the North Shore-LIJ Health System), an outpatient rehabilitation center specializing in helping people recover from neurological injury or illness. Patricia designs these art packages based on the therapeutic needs of each child.

Partners In CareThe Fly A Kite Foundation seeks to provide parents of newly diagnosed patients with support and guidance as they try to define and navigate treatment plans. The members of The Fly A Kite Foundation are passionate third parties available to guide, organize, support, listen, identify treatment options, and help bridge communications between family and medical provider.

Partners In ResearchThe Fly A Kite Foundation, Inc. (FAKF) is an IRS 501 (C)(3) non-profit charity dedicated to educating, aiding and funding the search for a cure for brainstem glioma and other forms of pediatric brain cancer in the hopes of curing all cancers.The Foundation seeks to fund research investigating cures for pediatric brain cancers with a focus on Diffuse Intrinsic Pontine Glioma (DIPG). Our focus is to support translational and clinical research projects in those areas.


DIPG Insights Survey: Purpose

The Fly A Kite Foundation conducted its March 2015, DIPG Insights Survey in an effort to better understand the basic trends in DIPG. With the steady number of DIPG cases across the United States and the complexity of the disease, the Fly A Kite Foundation sought to gather information about the children who have passed or are still fighting DIPG.

With very little published data available on this disease, the Fly a Kite Foundation sought to gather information about the children who have battled, and those still battling, this disease. Does DIPG affect one race or gender more than another? Has the life expectancy changed over the past 20 years? We hope the results of the survey will foster a deeper understanding of this disease and lay a foundation for deeper, more complex research in the future.

The information has been analyzed and will be made available on The Fly A Kite Foundation website, Facebook & LinkedIn pages as well as the DIPG communities. We will also make the information available to medical clinicians so they can have a clearer understanding and profile of a DIPG patient.

Note: Although the methodology and fielded study meets the guidelines of the Marketing Research Association, this study should only be used for informational purposes. The Fly A Kite Foundation makes no guarantees that the research is 100% accurate or has been reviewed by a 3rd party research organization or analyst. This study was performed on the grounds of making a case to commission a full service research organization to field a much more complete and comprehensive study. The opinions expressed in the Executive Summary & Personal Observations page(s) are those of the Author, David Bernstein and only David Bernstein.


DIPG Insights Survey: Methodology

The Fly A Kite Foundation reached out to DIPG families via e-mail and online to social media DIPG groups of families of children who either passed away from DIPG or are still fighting the disease. We asked a series of 10 questions with the goal of obtaining 100 completed surveys. Within the DIPG medical community, it is estimated that the number of DIPG cases ranges between 200 – 300 in the US, on an annual basis. The survey pulled from a sample of approximately 750 known families (3 years of cases) with a 95% confidence level that the sample reflects the targeted population. We also factored a 6% margin of error that he answers by the respondents are “true value” and represent the DIPG community accurately. The following questions were asked …

• Does or did your son or daughter have a Diffuse Intrinsic Pontine Glioma (DIPG)?• What is your child’s gender?• What is your child's age?• What is your child's ethnic background?• What year was your child diagnosed with DIPG?• In which state or U.S. territory do you live?• If your child has passed, how long did he/she suffer with the disease from initial diagnosis?• If you child is fighting this disease, how long is he/she living with the disease?• With regard to information resources, which of the following sources do you use to learn about treatment options? • What has been the most challenging obstacle(s) when seeking treatment?

The survey was promoted to and collected from the following online social media groups. All groups listed have a strong sample of DIPG families that have either lost a child or are on the process of battling this disease.

• Team Brooke (Healey) 4,394 members• DIPG Awareness of Family and Friends 2,096 members• Diffuse Intrinsic Pontine Glioma: DIPG/Brain tumor/Cancer Families 2,014 members• NATHAN's ARMY on a Mission Against DIPG 789 members• Strength For Maddox 617 members• DIPG Research group 296 members


Survey Questions

March 2015

The Fly A Kite Foundation: DIPG Insights SurveyFielded in March 2015, Based on 100 completed surveys. Location of DIPG reported cases from 1999 - 2015



75.35% lived with the disease between 0 – 12 month 75.01% are living with the disease between 4 – 19 months

28.13% are living with the disease 15 months longer

75.35%

75.01%

Comparing Children who have passed vs children that are currently battling

Organic food/natural path Burzynski clinic Herbalist, acupuncture Yahoo list serve Online medical research Work resources, medical resources (connections made through Dr visits) clinicaltrials.com Anything we could get ahold of Katie McKerracher book Support groups, Google searches Researched alternative treatments, cures and survivors


Unedited Verbatim Comments:

living with her dad and had no input lack of viable / reasonable options Chris was never treated, his tumor was too aggressive how to pick between a bunch of unproven options Had little options/confused on which path to go Nothing available since he had METS down spine at diagnosis. We were not told of the urgency of the situation and because of that, our son's treatment

was delayed. Limited options of treatment Information is confusing. Too hard to process the information and at the same time making

these difficult decisions. Is there a treatment? Seems no treatments are available There isn't many options regarding treatment. No one will treat an unstable tumor. Lack of effective treatment no treatment available Did not treat



Disease progressed so fast, it's hard to learn all you need in such a short time. Lack of information, lack of research options, lack of survival information, lack of detailed information (such as: cancer patients have a vitamin D deficiency) Actually having a treatment that would work We went in blind and was in such shock that we did exactly what our docs told us too. Then once we got

strength to get back on our feet and look for other options, we didn't qualify for anything because we already started with a treatment. Wish we knew of more options before starting with any treatment. Also our sons DIPG had spread to his spine so there was no trial we ever found him to qualify for.

Weren't many treatment options at the time of my son's diagnosis in 2005. Only option at that time was radiation.

lack of options with some degree of success No hopeful options limited treatment. And if your kid is doing well, they really don't want to do anything There was no cure...poor survival rate Treatment options limited. lack of effective treatments



If it was not for our personal network of contacts, we may not have ended up in good care at St. Jude. Our oncologist at Children's Hospital in Omaha recommended that we go home and make our daughter comfortable. The initial presentation of the diagnosis was unacceptable.

Choosing the best facilities-We managed after a few months Knowing that treatment was only palatine Watching the change of his lille life That there are any viable treatments available Wish had more options of clinical trial at the start Time off/paying household expenses we were able to seek treatment in our own city. But it didn't seem like our team knew much more about

what other facilities had as treatment options. also the clinical trial.gov site does not post results. So even if trying to look for better options, you don’t know what's most promising. if we had chosen to go elsewhere travel would have been an issue

1 hour drive in AZ, 2-3 hours in PA not a lot of options out there for treating DIPG right treatment wasn't available




Executive Summary

March 2015


DIPG Insights Survey: Executive Summary

After reviewing all the data, most of the answers did not raise any red flags, however, some interesting elements should be highlighted. In Q2, we asked, “What is your child’s Gender?” While the results of (49%) female and (51%) male were not alarming , it is important to note the closeness of these results. Historically, in published reports, medical speculation and strong estimates by the pediatric oncology community, indicated that the average age range for child with DIPG was between the ages of 4 – 10. As sourced on the DIPGRegistry.org, DIPG primarily affects children, with most diagnoses occurring between 5 and 7 years of age. After looking at the responses to question 3, “What is your child’s age?” the results show that (52%) of children between the ages of 4 – 7 have DIPG, (73%) are between the ages of 4 and 10 but (17%) are between the ages of 11 - 16+ years of age. Does this suggest that DIPG is skewing a bit higher in children between the ages of 11 – 16+ years of age?

Question 4 asked, “What is your child's ethnic background?” (89%) were white while a smaller population were American Indian, Asian, Hispanic and Italian.

Question 5 asked, “What year was your child diagnosed with DIPG?” (83.83%) reported that their child was diagnosed between 2010 – 2015. From the years 2012 – 2013, (40.40%) of the children were diagnosed and from 2014 to current date, (29.29%) were diagnosed. We will be watching closely to see if the number of cases grow in 2015 vs the prior years.


DIPG Insights Survey: Executive Summary

In question 6, we asked “In what state or US territory do you live?” While the number of cases plotted on the map appears to be spread across the country, there is a concentration of cases on the West Coast (CA) (12.12% ) and the East Coast (NY, NJ, PA) (27.34%).

When we analyze questions Q7 and Q8 together, (Q7) “If your child has passed, how long did he/she suffer with the disease from initial diagnosis?” and (Q8) “If your child is still fighting this disease, how long is he/she living with the disease?” Looking at the 2 charts side by side, (75.35%) lived with the disease between 0 – 12 month. When comparing this to Q8, (75.01%) are living with the disease between 4 – 19 months, or you can say that (28.13%) are living with the disease 15 months longer. I suspect the rationale would be the advancement of medical intervention and new drugs to market.

Question 9 asked “What source do you use to learn about treatment options (multiple options)?” (61.86%) use their current physician while the second largest are Non-Hospital Websites (57.73%) and Facebook (52.58%).

Question 10 asked “What has been the most challenging obstacles(s) when seeking treatment (multiple options)?” The single largest concern at (38.46%) is, “Didn’t know where to go for help and was uninformed”. It was also interesting that the medical provider was “Not informative” at (21.98%). If you read through the list of other comments comprising (41.76%) of responses, they include…”Confusion”, “Didn’t know where to go”, “lack of treatment options”, “Disease hit us so fast”.


Opinions and Observations

March 2015

• Funding• Accessibility

• Information• Standard of Care

• Palliative Care• Media and Public Relations


DIPG Insights Survey: Personal Observations

There is no denying that DIPG is a horrible disease that takes the lives of our precious children at a very young and innocent age. The data support the historic age range that children between the ages of 4 – 7 are still the primary age group but these is some speculation that younger adults are also diagnosed with this disease as well. Has this gone undetected in the past? Is there a possibility that teens are now starting to be diagnosed this disease more and more?

There has been speculation that environmental issues may play an important role. With 100 plotted cases, there seems to be a wide range of cases across many states. There is a higher concentration on the West Coast (total population 38 million) and East Coast – NY, PA, NJ (41 million) as compared to MN (5 million) or any other Midwest state. Could this simply be that NY and CA have a higher population rates than many of the other states so the number of cases would increase because population is 3 to 4 times greater? Although this study is limited, I do not suspect environmental factors play a role in children and young adults diagnosed with DIPG.

My interpretation of the data, personal observations after countless conversations with grieving families, I’ve identified what I think are the biggest hurdles in DIPG and necessary steps we as a committed and unified group need to take to learn, decide and execute the changes needed for the families of children newly diagnosed and those that have lost.

Funding…We simply need to get more funding into the labs of researchers who are doing great things behind the scenes. For 2014, The National Cancer Institute (NCI) budget is $ 4.9 billion. It is anticipated that ALL childhood cancers will receive only 4% of that sum or $ 195 million. DIPG will receive much less. With recent news of new paths for drugs delivery, new drugs to markets, more trials conducted, Immunotherapy, vaccine and gene therapies, and personalized medicine, now more then ever is the right time to invest in the study and cure for DIPG. Within the last few years the level of progress has magnified substantially given then resurgence of focused efforts by clinicians, higher profile cases, additional outside funding and progressive medical treatment.



Accessibility – We need to find a way to get better medical and palliative care to regions where families feel abandoned or uninformed. I do believe there is a correlation between progressive hospitals and the survival rates of patients. More importantly, I think more sophisticated hospitals and cities offer better early detection, treatments options, consultative medical advise and palliative care for the entire family. With a very limited number of DIPG Cases diagnosed on an annual basis, many of the remote hospitals lack training, early detection and resources to provide families the necessary tools for treatment and care. There needs to continue to be a collaborative discussion among the more experienced medical professional and doctors that are seeing cases for the 1st time. The allowance of more experience medical teams should be available for consultative discussion and onsite treatment whether remote or not. Families often time spend tens of thousands of dollars on travel and living cost to be connected to a more progressive hospital. Technology should be able to bridge the gap and offer better insight and collaborative discussion among clinicians.

Information: As families scramble to make sense of the initial diagnosis, I think the DIPG communities need to have a single reference that ALL foundations, hospitals, online communities, and sources could share. Many families get mixed messages, inconsistent messages and often times, wrong information. Many find themselves going to friends, family and social networks that often times provide a myriad of treatment options that are self prescribed without any medical oversight. Families are taking treatment into their own hands which ultimately is more harmful than the initial diagnosis.

I think we need to adopt a single source of information, that can be shared among all regions and communities. This should be a working, living, breathing entity that shares the latest information, treatment and drug options. What is the overall standard of treatment? There is either information overload (i.e., trials and drug choices) or not enough information. Families need to make very quick decisions when it comes to care. Information can not be fragmented. It needs to be concise, directed and clear.



Standard of Care: As outlined on the DIPGRegistry.org, the standard of care for a DIPG patient when newly diagnosed is as followed. While essentially palliative, focal radiotherapy remains the primary approach to treating DIPG and can temporarily stabilize or improve symptoms and show improvement on neuroimaging for most children. Conventional dosing ranges from 54-60 Gy, and standard treatment generally consists of 1.8-Gy fractions delivered once daily, 5 days a week, for a cumulative target dose of 54 Gy. Hyper fractionated doses up to 72 Gy have not shown efficacy in pediatric patients, and hypo fractionated radiotherapy may lead to slightly inferior outcomes. More patients demonstrate a clinical response to radiotherapy (85%) than an evaluable radiological response (50%), but little or no correlation has been shown between radiological response and either clinical response or survival. The benefits of radiotherapy are generally of short duration.

While radiation seems to be the standard of care for more than 20 years it is proved to repress the symptom for several months after treatment but certainly not a cure all. I think the greatest minds in DIPG need to adopt a NEW standard of care that provided Radiation, Drug Intervention (based on the biology) and a biopsy (when available). Although the last 2 are not without risks, I think a study should be done to demine what phase 3 trials have very limited side effects, will improve the quality of life for the child and give the family as much time as available in the remaining months to years they have left. While I understand there are misdiagnosis, unique circumstances and factor beyond our control that would prevent a child from being placed on the new standard of care, I think the Standard of care choices are extremely limiting and provided little to no longer term benefit than in the past. Give all we know about the biology of the tumors and the 300 plus drugs on the market, we clearly need to go back to the drawing board and reevaluate what we define standard of care. Let that be our new normal while we continue to push for a cure.



Palliative Care– The truly unfortunate issue with DIPG is there continues to be no cure. Often times, families jump at anything that “may make a difference”. Their “Hail Marry” approaches to medicine may be more destructive then beneficial. No family ever wants to have regrets. Families need options, to maintain hope and to know there are sensitive, consultative medical professionals there, holding their hand and guiding them every step of the way. I always say, “your oncologist should be your best friend. If they are not, you need a new oncologist.” The medical community needs to be trained in the palliative side of medicine. Where the survival rate of DIPG stand today, 10 out of 10 times, the hard and transparent conversations will need to be had. Having a medical team trained to deal with the emotional side of care is equally if not more valuable then the scientific side.

Media and Public Relations: DIPG awareness has been growing aggressively over the last few years. With high profile cases like Lauren Hill, or former Chicago Bears running back Adrian Peterson who received the news that 6-year-old son A.J., was diagnosed (DIPG). These cases for a few more are gaining national attention from the HuffingtonPost, CNN, NFL.com, ESPNgo.com, SportingNews.com and The View. This is not to diminish the local coverage families obtain by their local press and media outlets. There is not better time to work with a National PR agency to help spread the word on a National Level. Media outlets, celebrity endorsements, team affiliations are all beneficial to the overall exposure of DIPG awareness. This is a disease that needs to be fought on a national stage. While local coverage Is valuable, they do not have the reach and exposure to new organizations that touch millions of families every day. I think all the foundations, organizations, hospitals and labs need to allocate a % of revenue's towards a national outreach program. The days of holding up big checks for $ 25,000, $ 50,000 and $ 100,000, while nice and certainly beneficial, that only serves one main focus…the preservation on 1 foundation or organization. This is a disease that can not be won by 1 organization but consortium of organizations that strategically pool resources to speak the word. “Cure DIPG.”

Fly A Kite FoundationDavid BernsteinFounder and [email protected]

mailto:[email protected]

http://www.flyakitefoundation.org/

Fielded: March 2015 Research Executed by: Survey Monkey Questions provided by: David Bernstein...

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