shine a light. grow a community. pay it forward.
June 20th, 2017 by

Edison High student’s senior project: Writing a book on what it’s like to be a cancer sibling

http://www.latimes.com/socal/daily-pilot/news/tn-dpt-me-butterfly-sisters-20170512-story.html

June 20th, 2017 by
June 20th, 2017 by

In precision medicine era legacy gifts of patient brain tissue reveal disease mechanisms and new therapeutic approaches.

https://www.scientificamerican.com/article/scientists-tackle-lethal-childhood-brain-cancer/

February 16th, 2016 by

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In its five years of existence, the McKenna Claire Foundation has granted over $1,000,000 to researchers working to find cures for pediatric brain cancer.

In 2015 alone, $225,00 was granted to Monje Lab at Stanford University, $75,000 was granted to Dr. Mark Souweidane of the Children’s Brain Tumor Project at Weill-Cornell Brain and Spine center, and a $10,000 partnering grant was entered into with St. Baldrick’s Foundation. 

At the Monje Lab, these funds provide much needed financial support for new in vivo experiments being conducted to test various drug combinations on live DIPG tumor cell lines. Each such experiment costs between $10,000 to $15,000 per drug combination. In addition, Dr. Monje studies the biological processes of the pediatric brain and their effect on the origins of pediatric brain tumors and the consequences of treatment.  This work is key in increasing the understanding of DIPG and according to Dr. Monje “will serve as the foundation of a clinical trial within a large national consortium” set to open to relapsed DIPG patients this year.

At the Weill-Cornell laboratories of Dr. Mark Souweidane, the granted funds are being used to further advance existing drug delivery methods to areas of the brain that have not been reachable by traditional methods.   Through a microscopic drug delivery system, Convection-Enhanced Delivery (CED),  Dr. Souweidane is working to strategically place therapeutic agents directly into areas that have resisted traditional treatment, thus reducing the potency of drugs therapies needed, and toxicities to patients through current drug delivery methods.

For the 3rd year in a row, McKenna Claire Foundation has also partnered with St. Baldrick’s Foundation to help jointly fund pediatric brain tumor research grants reviewed by the St. Baldrick’s advisory board, allowing more scientists access to much needed funding in order to further research.

These important grants would not have been possible without you.   Thank you to every McKenna Claire Foundation supporter and partner who, over the last four years, has helped raise desperately needed dollars for pediatric cancer research.  Because of the generosity and passion of our extended community, we are honored to support the researchers and physicians who continue this fight, accelerating the pediatric brain cancer solutions children and families so desperately need.

We look forward to seeing you in 2016

November 14th, 2014 by

The Monje Lab studies the developmental origins and unique biology of pediatric gliomas, especially DIPG.

Childhood brain development and pediatric gliomas

The myelinated infrastructure of the brain develops throughout childhood and adolescence, and discrete waves of developmental myelination correlate with the age and location of pediatric glioma formation. As mounting evidence from the Monje Lab and from others indicates that pediatric gliomas like DIPG arise from myelin-forming precursor cells, understanding the mechanisms that govern the growth and differentiation of these cells becomes paramount to discerning what drives pediatric glioma initiation and growth.  Recent work from the lab has demonstrated that neuronal activity robustly promotes the proliferation and differentiation of normal myelin forming precursor cells, ultimately modulating the white matter structure of active circuits in a manner that facilities neural circuit function of those brain networks most used by that individual. These results give clues to a possible powerful driver of growth in the glioma microenvironment, clues the lab is actively following up now. Moreover, these findings shed light on a previously under-recognized mechanism by which experience modulates brain structure and function, with implications not only for brain cancer but also for neuroregeneration in diseases of white matter such as multiple sclerosis.

Therapeutic targets in DIPG

The Monje lab has been developing, distributing and utilizing unique patient-derived DIPG cell culture and orthotopic xenograft mouse models in an effort of discover effective therapies for DIPG. As part of an international collaborative effort to screen DIPG cell lines for response to new and emerging targeted chemotherapy agents, we have identified a promising therapeutic candidate and have successfully negotiated access to this drug for a Phase I clinical trial for newly diagnosed and recurrent DIPG, anticipated to open in 2014-2015. We continue to study the unique molecular biology of DIPG, its cellular hetereogeneity and its interactions with the microenvironment of the childhood brain to identify additional effective therapeutic strategies.

 Long-term consequences of childhood cancer therapy on the developing brain

Children who survive childhood cancer therapy frequently experience life-long and progressive deficits in cognitive function. Damage to neural precursor cell populations responsible for brain development, maintenance and plasticity, including myelin-forming precursor cells, is thought to underlie much of the cognitive dysfunction that follows cancer therapy. The Monje Lab studies the mechanisms responsible for this precursor cell dysfunction and seeks to harness and regulate mechanisms of myelin plasticity in neuroregenerative strategies aimed at improving cognition after cancer therapy.

For more information, please visit the Monje Lab website

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August 28th, 2014 by

PCN2014Group

 

Lisa Roberts
MCF Board Member 

In early August I got to spend a week roaming the campus of my dream school…Stanford University.  Although I have long held Stanford in high esteem, my perspective became much more personal when, back in 2011, McKenna was diagnosed with DIPG.  It was during that time that I first learned of Dr. Michelle Monje, a well respected pediatric brain cancer researcher and Assistant Professor of Neurology at Stanford.  Only six months after Macky’s diagnosis, there was a heartbreaking reason to work directly with Dr. Monje…to help coordinate the Wetzel family’s generous, yet incredibly painful, decision to donate Macky’s tumor tissue to Dr. Monje’s lab at Stanford for further research.  This was how I became personally aware of Dr. Monje’s profound compassion for her patients and their families as well as her ground breaking and innovative research aimed at finding a cure for pediatric brain cancer.

 

An informal part of Dr. Monje’s work involves partnering with and updating the private foundations that help to fund research conducted by her lab, as well as, that of other renowned researchers and institutions all over the world.  To that end, Dr. Monje and Stanford graciously agreed to host the 2014 Pediatric Cancer Nanocourse in order to educate pediatric brain cancer advocates on the current science and research developments in DIPG pathobiology, diagnosis and treatment as well provide opportunities to strengthen the relationships between foundations and researchers as we work together for a cure.  A “nanocourse” is basically just a fancy word for “crash course” which is a very accurate description of my week at Stanford!

 

For five days we listened as leading doctors and researcher’s presented on a myriad of topics related to pediatric brain cancer…from it’s theorized origin, to current research findings and developing clinical trials, to treatment standards of care, as well as (the devastating) late term effects of treatment on long term survivors.  While I couldn’t possibly summarize all that we learned here in this post, here are my Top Ten important take-aways from the week…

 

 

1.         Brain Tumor Tissue Donation is Critical

 

Although the total number of brain tumor tissue donations is still fairly small, such donations have played a critical role in developing new and novel approaches to pediatric brain cancer treatment.  In fact, the propagation of these tumor cell lines over the past three years has led to more advancements in the area of DIPG research than in the last thirty years combined!  With that said, at present only ~25% of tumor tissue harvested will be successfully cultured allowing for the propagation of cell lines.  Dr. Monje and other researchers have learned a great deal in a short period of time about the factors that increase or decrease the potential for creating a successful cell line from tissue donation and are working hard to ensure that every donation can become a durable cell  culture.

 

2.         With DIPG Tumor Tissue Donation Increasing, There is More Hope for Developing     Clinical Trials for Effective Treatment

 

Clinical Trials are the process by which drugs or other treatment interventions are tested for safety and efficacy.  They are divided into four classes:

 

Phase I:  A new drug or treatment is tested in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.  The actual efficacy of the drug is not tested in the phase.

Phase II: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.

Phase III: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.

Phase IV: Studies are done after the drug or treatment has been marketed to gather information on the drug’s effect in various populations and any side effects associated with long-term use.

 

There has never been a Phase III or IV DIPG clinical trial in the United States.  In other words, there has NEVER been a drug developed that shows any efficacy in the treatment of DIPG.

 

Historically, DIPG and other brain stem tumors are some of the most difficult of all brain cancers to treat.  With the recent availability of tumor tissue for research there is finally real hope for accelerating the clinical trial process and therefore the development of treatments that can effectively target these tumors.

 

3.         Tumor Cell Lines Have Already Led to Breakthrough Research

 

The breakthrough availability of tumor cell lines has enabled researchers to test these cancers against a panel of 60 known chemotherapy agents to look for patterns of efficacy and develop strategies for creating clinical trials that test new combinations of chemotherapy drugs to attack these tumors with greater success.  As a result of this work, clinical trials are already in process for testing these theories and creating hope on the horizon.

 

 

4.         The Study of Epigenetics Is Crucial to Understanding DIPG

 

Epigenetic research (the study of how gene expression is controlled within a cell) has led to the breakthrough identification of certain histone and other mutations that are present in pediatric high grade gliomas.  Further research is looking at how these mutations operate within DIPG tumors and at identifying the cell of origin for DIPG tumors.  Once determined, this information can be used to develop therapeutic strategies for treating DIPG.

 

5.         Research Labs Require a Variety of Forms of Financial Support

 

“Indirect costs” are like a “tax” that the research lab must pay to the University in order to accept the grant funds. The Universities at which the research is being conducted will charge the researcher a percentage of the total grant funds to cover the “indirect costs” of performing the research (like keeping the lights on and other lab infrastructure services that the University provides), so if the granting Foundation does not cover these “indirect costs” then the researcher must pay these charges from other sources. If they cannot find other funds to cover the indirect costs, they cannot afford to accept the grant.  For this reason, “indirect costs” are ideally built into the grant research budget… disallowing “indirect costs” penalizes the researcher for doing the work of that grant.

 

In the last three years, the McKenna Claire Foundation has donated nearly $410,000 to the Monje Lab, which not only provides “unrestricted funds” for DIPG research (funds Dr. Monje has flexibility in determining exactly how to allocate), but in addition, specific grants to cover a variety of administrative costs that support her research…from the salary of a research assistant whose primary responsibility is to administer the maintenance and distribution of the cell lines propagated by the Monje Lab, to costs associated with cell line shipping, to the costs associated with new tissue harvest.  By covering these “non research”, administrative, costs the McKenna Claire Foundation is helping to ensure that the cell lines created from donated tumor tissue will not only be studied at Stanford, but at leading institutions all over the world.  Already, McKenna’s tumor cell line has been distributed to more than 3 dozen researchers in countries as far as Australia, France and Spain The more researchers who work with these lines the faster we can advance progress!

 

6.         Funding the Technology to Conduct Research is Just as Important as the             Research Itself

 

Similarly, it is often difficult for researchers to obtain grant funding for “capital expenditures” e.g. one time expenses related to the purchase of equipment.  The McKenna Claire Foundation has helped address this funding shortage by providing a grant for the purchase of cutting edge technology that allows creating “slices” of cultured mouse brain, thereby keeping the tumor microenvironment intact to study in a  dish.

 

7.         Creative Ideas are Necessary to Close The Valley of Death

 

One in five pediatric patients do not survive childhood cancer and these patients have had the same chance of survival for over 40 years.  Because drug development is fueled widely based on market forces, science has outpaced treatment development, particularly for children.   A gap between basic science and the development of clinical trials, known as the “preclinical gap” or “the valley of death”  too often prevents new drugs for promising targets from reaching children.  Creative and innovative approaches by “outside the box” thinkers like Dr. Charles Keller of the Children’s Cancer Therapy Development Institute) and Nancy Goodman, author of the “Creating Hope Act”, are critical to bridge this gap and bring drugs developed specifically for children to trial and market faster.

8.         Advocacy for Careis as Necessary as Cure

 

Although the ultimate goal is to find a cure for pediatric brain cancer, we at the McKenna Claire Foundation understand this quest is a marathon, not a sprint, and “baby steps” are an important part of the journey.  One of the ways pediatric brain cancer families and foundations can assist in this process is to continue to inquire and advocate for the development of better palliative treatment options for inoperable, terminal brain tumors.  The 2014 Nanocourse participants have committed to following up on promising early clinical trials that present alternatives to the standard treatment of administering dexamethasone to alleviate the symptoms of brain tumor growth and the swelling caused by surgical intervention or radiation therapy.  These alternatives aim to provide the same benefits of dexamethasone without the devastating side effects so many pediatric brain tumor patients must endure.

9.         The Late Term Effects of Pediatric Cancer Treatment are Devastating

 

The late effects of cancer treatment on pediatric cancer patients are quite simply, devastating.  Although patients diagnosed with DIPG do not currently survive long enough to fully experience these effects, as treatments are developed, it is a problem the DIPG community hopes to face one day soon.  About 1 in 250 adults is a childhood cancer survivor, and of those, 65% of them survived a brain or other central nervous system (CNS) tumor.  One of the most effective and widely used treatments for CNS cancer is radiation therapy (XRT).  Because the human brain takes decades to fully develop, children who receive brain XRT are particularly at risk for serious, long term, side effects.  These survivors are less likely to obtain a driver’s license, marry, hold down a job, or live independently as adults and significantly more likely to require special education services, suffer from depression, thyroid gland, reproductive organ and growth hormone disorders, hearing or vision impairments, secondary cancers and have a higher incidence of stroke compared to their healthy peers.  Concurrent to the importance of finding a cure for pediatric brain cancer is the critical importance of understanding how brain cancer treatment affects the developing brain (a subject The Monje Lab is also working on…) so that scientists may develop better ways to protect pediatric patients from experiencing these devastating side effects and treat them more effectively when they do.

 

and lastly…

 

10.       The Difference is in the People

 

The doctors, researchers and advocates I met during the Stanford Nanocourse are nothing short of heroic.   It is truly breathtaking to be in a room full of parents who have lost the most precious thing in the world to them…their child…then add to the mix doctors and researchers who have dedicated their entire careers to finding a cure for pediatric cancers, and you may then begin to understand how humbled I was to be in their midst.  It is hard to put into words how inspired I was by their, across the board, selfless dedication, compassion and commitment to making a difference in the world of pediatric brain cancer.  It was my distinct privilege to learn along side these amazing individuals, to hear their heartbreaking yet beautiful journeys with pediatric cancer and the triumphs and frustrations of researching one of life’s most mysterious and deadliest cancers.  We laughed, we cried, we learned, we pondered, but mostly we came together for a common cause…to grow the community that is shining a light on pediatric brain cancer to find a cure…And although I’d give anything to have never even heard of DIPG, I left Stanford full of hope and forever changed by what I learned and the partnerships forged during my time there.

 

My heartfelt thanks to Dr. Monje, the Wetzel family, the McKenna Claire Foundation and all of the presenters and participants of the 2014 Pediatric Cancer Nanocourse for this once in a lifetime opportunity…

April 25th, 2014 by

04|20|2014

Last week was a good week for DIPG research. After years of few advancements in the understanding and treatment of the disease, a new crop of research has emerged which shines a light on the working biology of the normal pediatric brain and its affect on DIPG tumors, as well as the origins of the tumor itself. MCF is proud to support Dr. Michelle Monje and Dr. Mark Souweidane, two of the leading researchers in this field who both had major papers published in scientific journals last week.

Dr. Monje, of Stanford University, was cited in not one, but two papers last week. The first is a collaborative effort reporting the identification of mutations in the ACVR1 gene in some DIPG tumors. Dr Monje was also the lead author of a paper showing that “neuronal activity causes changes in myelin (cells that insulate nerve fibers and make them more efficient).” As Stanford’s School of Medicine Newsletter says, this is an area of interest for Dr. Monje and other DIPG researchers because:

“The cancer, which usually strikes children between 5 and 9 years old and is inevitably fatal, occurs when the brain myelination that normally takes place as kids become more physically coordinated goes awry, and the brain cells grow out of control. “

To read more about the study click here.

Dr. Mark Souweidane, of Weill Cornell Brain and Spine Center, is a co-author (along with some of the leading names in pediatric brain tumor research) on a new paper about DIPG. A collaborative, multi-institution research group announced that they have discovered three distinct types of DIPG – showing it is not a single disease at all, but rather three different diseases. With this knowledge, researchers will now be able to choose therapeutic agents that have the best chances of success against an individual child’s particular type of tumor.

To read more click here.

Your generous support and participation in MCF events has helped enable researchers to begin to understand the complexity of this disease. While it is exciting to see progress, there is still a long and difficult road ahead of us as we search for a cure. Research needs funding now more than ever to allow these discoveries to become a source of real hope for children and families

“The funding model used by the McKenna Claire Foundation, investing in the research group more than in a specific project, provides the resources we need to act quickly when a promising new direction arises from the data. This allows us to be nimble in our research, and hopefully to move the field closer to effective therapies for DIPG.”
-Dr. Michelle Monje

April 1st, 2014 by

Two years ago, three months after losing McKenna, we sat down with some good friends around a kitchen table and made the decision to honor her by starting a foundation in her name.  At that point, the details were a little hazy.

We knew we wanted to fund research, but also wanted to involve the community that had supported us during her illness.  We wanted to help “shine a light” on the problem of pediatric brain cancer, but we also wanted to show how others “shine” when given the option to help.  Dave and I can honestly say we had no idea what we were getting into.  We were in a fog of grief and just knew we needed to move forward in a way that would help offer other children and families who were faced with the diagnosis of brain cancer.

Thanks to those amazing friends around that table, the foundation found its footing and gained traction.  The road has not been easy for any of us.  There have been struggles, frustrations, and misunderstandings.  But more importantly, there has been love, strength, and community that has blossomed from the depths of our despair.  This community has grown to include not just those who physically surround us in our hometown, but to others who have heard McKenna’s story and taken our little girl to heart.

From corporate partners like Chevron, whose patrons have embraced an unknown philanthropy, to the children holding lemonade stands in McKenna’s name, and everything in between, you all have done something amazing.

Because of you, over one million dollars has been raised for pediatric cancer in just two short years.  Of this, over $500,000 dollars has already been put into the hands of researchers who are making great strides in decoding the biology of DIPG and developing new, more effective treatments that may soon offer children more hope than they have ever had before.  Partnerships with established pediatric cancer foundations, such as St. Baldrick’s, have been formed in order to develop a prototype that allows smaller foundations to work together to crowd source funding for cures.  McKenna’s cell lines have been distributed to researchers world-wide to provide much needed material for in vitro and in vivo studies.  Because of the support you have provided, there are two scientific projects in Dr. Michelle Monje’s lab at Stanford University investigating the neurodevelopmental origins of DIPG and potential treatment strategies that are both in the process of publication.  As well, Dr. Monje and her lab are working collaboratively with DIPG researchers internationally who are sharing findings in order to expedite hope to those families who are currently told there is none.

None of this would be happening without you.  Whether you have contributed your money, your time, or your talent, we cannot adequately express our appreciation.  We are thankful that you remember our Macky.  She would be humbled, as we are, by the community that has embraced her.  We hope that you will continue with us on this journey, as there is still a long road to travel and so many children waiting for us to get to our destination – a cure.

April 1st, 2014 by

This year we celebrated McKenna’s birthday a day early by visiting some of the lemonade stands held during our2nd Annual Stand Up and Shine event.  With over 40 stands being held from the Hamptons to Hawaii, we could think of no better way to celebrate our girl than to visit so many amazing kids doing their part to help find a cure for pediatric brain cancer.  Although donations are still coming in, these entreprenurial kids raised well over $5,000 dollars.  It made us smile to see the joy and laughter surrounding every stand we visited and received pictures from.  Your emails and stories warmed our hearts, and we just know that Macky was shining down on each and every one of you.  We are looking forward to growing this community and including even more of our future humanitarians/business leaders involved in next year’s event.

April 1st, 2014 by

Along with our promise to McKenna to find a cure for DIPG, we here at McKenna Claire have also promised to do our part to raise awareness and be advocates for all childhood cancers.  With the thought that our voices and our funds are more powerful when united, we began looking for a way to partner with other foundations in order to make funding and advocacy more effective.

We were thrilled when we found St. Baldrick’s receptive to the partnership concept, effectively allowing small foundations to continue to honor their children and their cause while helping to make a bigger impact on research than working alone.  To that end, McKenna Claire Foundation has partnered with St. Baldrick’s, donating $50,000 to of the $115,000 grant given to fund research by Dr. Christopher Gamper of Johns Hopkins Kimmel Cancer Center’s.  Dr. Gamper’s research focuses on immunotherapy and decreasing the late effects of treatment by reducing the need for chemotherapy and radiation for children with cancer. “Pediatric oncologists are coming close to understanding how to harness the power of the immune system to help fight and cure cancer,” said Dr. Gamper. “We believe that using drugs that block DNA methylation will enhance anti-cancer vaccines and will use our extended St. Baldrick’s funding to help us test this theory.”  Dr. Gamper adds, “Harnessing the immune system to help fight cancer holds the promise of curing difficult-to-treat cancers while sparing children from the toxicity of chemotherapy and radiation.”

We are grateful to St. Baldrick’s for launching this program, and it is our hope that as we continue to build on the partnership concept that we will be able to deliver more effective, less invasive treatments to our children who so desperately need it.

For more information please visit: http://www.stbaldricks.org/blog/post/St-Baldricks- Foundation-partners-with-the-McKenna-Claire-Foundation

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