Professional Philanthropy Network Benefits Beat Nb!

Last Wednesday, we were given a fun-filled happy hour at the Hyde Out, courtesy of the Professional Philanthropy Network (PPN). This group benefits charities once a month and brings together the charity and the professional community in Tampa Bay, and we are grateful that they chose us for the month of February. This money will directly help us give brave hope to kids with cancer, through effective and innovative clinical trials. We always appreciate the love and support that the Tampa community gives us.

We also auctioned off a signed Ryan Callahan (Tampa Bay Lightning) jersey – pretty cool!

An album of photos from the evening can be found here.

If you attended and took any photos, we’d love to see them! Email us at janine@beatnb.org, or tag @beatnb on Twitter or Facebook when you share. Thanks!

600 family members in Cape Cod

Yesterday Beat Nb ran the Falmouth Road Race, for the 5th year in a row. It was a perfect day on Cape Cod, high 70s with a breeze rolling off the water through most of the run.

The people are awesome. This was my first time running Falmouth, with over 11,000 runners showed up for the 44th annual 7.1 mile race. 200 of them were running with Beat Nb to help us save kids with cancer (the second largest group running). For pretty much the entire 7 mile stretch, supporters were lined up along the road cheering runners on, giving out water, spraying us with their garden hoses, and smiling.

Right around 5.5 miles was the official Beat Nb cheer area, with what must have been a hundred green shirts and happy faces giving us that extra boost of motivation toward the finish line. I got messages from people who weren’t even on our team, noticing that huge green mass and having seen our running shirts throughout the run, complimenting us on our crowd.

Our crew here is a family. The amount of hugs and high fives and cheers going around welcomed us in with open arms, and Robyn and I both felt right at home. This group has raised $275,000+ so far, with fundraising pages open until November to hit our $300k goal. There are multiple families of neuroblastoma kids represented in the runners and supporters, and many who are here because one of those stories has touched their heart in a deep enough way to raise thousands of dollars and run 7 miles in the heat. My heartfelt thanks to you.

Robyn and I lost our son Ezra to neuroblastoma in 2010. Not a day goes by we don’t think of him. To see so many people pouring their effort into something which allows other families to stay whole, through clinical research which is saving these kids lives, means the world to us both. And to the families who were there yesterday. All us neuroblastoma parents are deeply grateful knowing you spend your family’s time working to better ours.

The post race party was a blast. Especially since there was no running involved! We had about 600 RSVPs, and Pat Lacey got at least partly soaked with freezing water as I dumped it mostly on his shoes (note: next year get more people to help lift the giant tub). Jack Eichel from the Buffalo Sabres (and Boston University) was there signing pucks and photos, and hanging with the tons of kids there who love hockey. Whoops – I wore my Tampa Bay Lightning hat. Thanks for being cool about it, Jack. 😉 It was great to have you there.

I think the most common thing I heard as I introduced Robyn and myself to everyone who has been training for Falmouth for weeks and months, was “oh, Kyle Matthews! You’re the email guy!” Ha, thanks – I’m glad to finally meet you all in person, and proud to be a part of this family now.

I didn’t run fast, but I finished the race. We’re ready to do this even bigger next year, and continue growing with our mission of beating neuroblastoma always in plain view and hard focus. There is a magic to the group we have amassed these past 6 years, in Boston and Braintree, in Tampa Bay, in San Diego, in Buffalo, in Grand Rapids, and across the country.

It is heart that cements us, and you could feel it when we stood yesterday outside that tent on the Cape Cod Fairgrounds. We are saving kids’ lives. This isn’t lofty rhetoric – the kids were there yesterday. In person, and on posters throughout the fairgrounds. These kids are beating a cancer which historically is bad news for a family, for a kid, as it was for my family. This is because of your help. Because of the incredible researchers we work with at the NMTRC. Because we are all standing up and saying, no, this isn’t right, and we won’t stand for it.

Thank you. Let’s keep at it. Let’s Beat Nb.

If you ran and took photos, we’d love to see them! Email us at kyle@beatnb.org, or tag @beatnb on Twitter or Facebook when you share. Thanks!

Beating Nb

In 2010, I founded Beat NB so there would be a consistent platform for all of you to support our mission to dramatically impact survival rates for children with neuroblastoma. I took this step out of necessity, because I wanted to fund treatments that could save my son – and every kid just like him – who had been told “there is nothing more we can do”.

Six years later, and we have raised and donated millions of dollars to research and clinical trials.

We have taken part in the creation of a national consortium of children’s hospitals to help kids all across the US. And we have all watched as my son continues to live his life, thanks to your support.

When I founded this organization my hope may have seemed indefensible to some, but as we stand here today so much more has been achieved than even I could have hoped for. For my son, and for every child on this planet with neuroblastoma – or about to be diagnosed with this deadly cancer.

And as I look at all that has been accomplished, I look ahead with even bigger dreams. Dreams for the day that we not only cure all of these children, but we do so without all of the devastating effects from the current ineffective and barbaric treatments we force these infants and toddlers to endure. And the need to achieve this goal, knowing that today is not soon enough, necessitates another bold step. I am proud to say that today is a new day for Beat NB – a day we’ll look back on when this disease comes with the promise of a cure, as an important step in helping to make that a reality.

It is my honor and privilege to introduce you all to my good friend Kyle Matthews, as he takes on the role of Executive Director of Beat NB and fills you in on what we have in store, and how you can help make our dream a reality.

Patrick Lacey

Today, we’re taking a huge step toward beating neuroblastoma. Four neuroblastoma nonprofits are joining together into a single entity, Beat Nb. We have a few other groups joining in the coming months, and hope to continue this model to bring dozens of families fighting neuroblastoma together into one collective fist in the face of neuroblastoma childhood cancer.

There’s a made-up thing called the cancer club, or cancer parents club, which we become members of when our kids are diagnosed. When Ezra was diagnosed, people told me “the neuroblastoma community is close. You’ll get to know many of the parents and groups.” I’d just been told my son had cancer, and thought that was a stupid thing to say. I wanted nothing to do with this community.

But as time went by (and I’ve said this before), I fell terribly in love with the childhood cancer world. We were surrounded by extremes: strength, resilience, tragedy, heartbreak. For years, Robyn and I have been honored to have many incredible friends whose children have also battled, or are battling neuroblastoma.

Over the past several months, we’ve been working behind the scenes to bring many of us even closer together. Here’s a common story (and, my own). A son or daughter is diagnosed with cancer, and the family builds a nonprofit to make a difference for the next family. There are many groups like this, built on passion, experience, and hope. We’re all doing the same work, in different cities, waving the same flag with different names. And while our individual voices are loud, as cheesy as this sounds… united we will be inescapable.

So we are coming together. Today, Because of Ezra (Tampa), Beat NB (Boston), Melina’s White Light (Buffalo), and Max’s Ring of Fire (San Diego) are forming a new Beat Nb. We will be a united entity, and other groups are joining soon. I will remain Executive Director, and our current Associate Director Noelle Muniz will retain her position, too. Pat Lacey will keep his role as President of the Board. We are, today, one of the largest neuroblastoma-focused nonprofits.

There’s a piece of me that feels the sting of not using Ezra’s name in this new group, but for Robyn and I, what we’re doing now isn’t for Ezra. We cherish talking about and remembering Ezra, and knowing the ripples from his life continue to change other people’s lives. But we’ve always recognized, what we’re doing now is for families who are where we were the day they told us Ezra had cancer. That day, I jumped in the car and drove home to pack a couple bags, since we’d “be here for a while.” My body shook and I screamed at the sky, my face soaked with burning tears, the entire way home. Thirteen months later, the early morning Ezra died, I cried less. Tears are a part of my life now, but that night I was empty and shocked. That is why we do this – so no parent ever has to know that feeling because of neuroblastoma. We were called Because of Ezra, but we were always Because of _____.

And that sentiment is behind our Because of _____ program. We are changing the neuroblastoma treatment paradigm. The work we’re funding is in over 25 hospitals and universities across the country, and more internationally. The NMTRC, our partner research group, has shown “unprecedented” results with their clinical trials, and we are motivated knowing the story IS changing. The Because of _____ program invites other families to join us as well. We need you. Like I said, we are loud with one voice, or with 5, but with 50 families? Neuroblastoma will be beaten. If you founded a neuroblastoma nonprofit focusing on research, and are interested in joining us, I’d love to talk. Actually, I’d love to talk either way. The neuroblastoma community is close.

It’s important to me to mention our friends who, like us, have lost their children since Robyn and I entered the childhood cancer world. There are too many to name here. There are pieces of your kids (from a photo on our wall, to a blossom on our nightstand, to a t-shirt hanging in our closet) all over Robyn and I’s life. Our tears and our love are with you.

I wrote a post after Ezra died titled “everything matters.” As I look back on the past years of friendships being built with people like the Riniolos, the Mikulaks, and the Laceys, I see it all being groundwork for what we’re doing today. I say this too often – I hate that we share neuroblastoma. Still, to paraphrase an old quote, “give me a dozen passionate families and I’ll change the world.” We will beat neuroblastoma. It won’t be fast enough, and the stakes are too high. But we will build a time where every diagnosis comes with a promise that we can beat this.

If you’d like to know more, we’re pretty open about how we operate and what we’re up to. Please, send us a message. And as always, thanks to everyone who supports our work. It’s making a difference.

We will Beat Nb.

Kyle Matthews

Max

Melina

Ezra

Will

Yes, Beat Nb is a 501(c)(3) nonprofit organization.  Our federal EIN # is 27-2314549.
We feel the name Beat Nb (beat neuroblastoma) is more inclusive and speaks directly to our mission.  While it is important to honor each of our kids’ legacies and names, we all share the same goal of beating neuroblastoma.  We will still be sharing the stories of our children, including Max’s, Melina’s, and Ezra’s.
Yes. Beat Nb will continue to work closely with the NMTRC, a research consortium of more than 25 hospitals across the world focused on treating and curing neuroblastoma, chaired by Dr. Giselle Sholler. You can learn more about the specific trials we fund here.
No. Nothing will change. If you were a recurring supporter of any of these groups, your recurring monthly or annual donation will automatically be transferred to the new Beat Nb entity. You will now receive receipts and annual giving letters from Beat Nb.
The “Because of _____” program is still an active and important program within this new combined Beat Nb organization.  The Because of _____ program was designed as a way for families to tell their child’s story and raise funds for neuroblastoma cancer research without having to start their own charity. Beat Nb provides a fundraising platform for these families through their own web page on our site as well as infrastructure under our 501(c)(3).  To learn more about the Because of _____ program, please visit: beatnb.org/because-of.
You can see our staff and combined board of directors here.
Yes.  We will still have the same special fundraising events in your city including Cure Me, I’m Irish (Boston, Buffalo), The Falmouth Road Race (Boston), Touch a Truck San Diego, Karaoke for the Kure (LA, Tampa, Denver, NYC), Mini Golf Big Cause (Tampa) and more!

#SofiaStrong finishes upfront cancer treatment!

A week ago, we hung out at All Children’s Hospital in St Pete to see 3 year old Sofia Anderson ring the oncology floor bell. All Children’s does this every time a kid finishes upfront cancer treatment, and Friday marked the end of over a year of treatment for Sofia – she’s cancer free!

One of the main things Because of Ezra is focusing our research funding on is stopping neuroblastoma relapse. Historically, over half of kids who beat neuroblastoma relapse – but through the work of the NMTRC, a 25+ hospital national research consortium we work closely with, we’re changing that story. Sofia’s next step is getting on the DFMO trial we’re helping to fund, which we hope is stopping relapse. Results of the DFMO trials which have already been completed are hugely promising, and we’re thrilled Sofia will be able to get on this trial at the same hospital Ezra was treated at 6 years ago, All Children’s Hospital.

We’ve become good friends with Catalina and Patrick Anderson, Sofia’s parents, and Cat has joined Because of Ezra as the founding member of our Parent Advisory Board. Hanging out with their son Aiden and Sofia is always a good time. Plus, our five year old Charley may have a small crush on Sofia. The Andersons have also helped us launch our Because of _____ program, raising awareness and fundraising Because of Sofia. They’re a force to be reckoned with against neuroblastoma, and we’re so grateful to be beating up this stupid cancer with them.

Giving $250,800 to NMTRC neuroblastoma trials

To close out 2015, I wrote a post sharing I’d be going full time with Because of Ezra in 2016. For my first official day “on the job” (although I’ve held this job since we founded in 2011 – just part time), we headed to Grand Rapids, MI, to present a check to the NMTRC toward their neuroblastoma trials.

Last year, Because of Ezra hit some incredible milestones in our fight against neuroblastoma and childhood cancer.  We hired a new Assistant Director (Noelle Muniz), helped facilitate the opening of a new clinical trial at All Children’s Hospital, launched our Because of _____ program to better partner with families fighting cancer, held two hugely successful fundraiser events in Los Angeles and Tampa, and were once again the beneficiary of the second annual Brighthouse Networks Enterprise Solutions Charity Classic!

Through the generous support of people like yourself, we were honored to give $250,800 (Ezra lived 800 days) toward two promising trials. The first, a continuation of the DFMO trials we’ve been funding, hopes to stop neuroblastoma relapse. The paper being published this year on the previous DFMO trials the NMTRC has completed will show promising results. We are proud to be a part of continuing the push to make this treatment available to all children battling neuroblastoma.

The second trial, called PEDS-PLAN, is an innovative new paradigm in treating childhood cancer using computers. Partnering with Dell, the NMTRC has been looking at genomics (or precision / personalized medicine) for over 5 years now. PEDS-PLAN is a new trial which opened in September of 2015 bringing all that knowledge and experience into using precision medicine in frontline treatment for neuroblastoma – an approach that’s never been done, and just a few years ago wouldn’t have been possible. If you’re interested to know more, read our article on how we’re beating childhood cancer with computers.

Please, be changed because of this. Recognize the flawed world we live in, and work to help the ones you can. Really work. Recognize the hurt in the world, and recognize the grace that gives us a chance to still be beautiful.

For the past five years, we’ve filled our free time with Because of Ezra, raising what we think is an impressive amount of awareness, and funds for research (more importantly – research with incredible RESULTS). At the end of our first year, we gave $30,000 to research. We kept just $5k in the bank, to operate and grow our organization. That small investment has led to so many more people becoming aware of the need for better treatment, children beating neuroblastoma, so much more knowledge gained, and many families whose stories will be different than ours. Today, we have given over $700,000 to patient-affecting childhood cancer research. We are beyond thrilled to see what happens as we continue to grow.

We can only succeed because of the powerful and passionate group surrounding us. It is important, of course, our work raising awareness and funds. But the story-changing, cancer beating research we and other advocacy groups help to fund is being done by over 25 hospitals, along with scores of physicians, scientists, researchers, technicians, administrators, nurses, and many others. We work closely with the NMTRC, chaired by Dr Giselle Sholler. We spend time with many families beating cancer today, and many who have lost children. We listen to how each and every treatment is working, and how it is affecting kids’ lives. We tell the stories, and we participate in the global conversation of how to move treatment forward, from perspectives of basic science all the way to federal and international policy.

We’re just getting started. Thank you for all your support to this point, and I look forward to continuing to be a growing fist in the face of childhood cancer. Join us.


Kyle Matthews, Ezra’s dad

2015 Yearend Summary: A Letter from Beat Nb Co-Founder, Patrick Lacey

Dear Beat NB Supporters –

Thanks to your extraordinary support over the past calendar year, we were able to send $800,000 to fund research and clinical trials in 2015.

Whether you donated, hosted a fundraising event, sponsored an event, attended “Cure Me I’m Irish,” or ran in the Falmouth Road Race, you contributed to Beat NB’s accomplishments in 2015. Below is a list of updates to summarize the work done this year in the clinical trials consortium (NMTRC), which is just one aspect of the efforts funded by our organization, in addition to all of the ongoing trials that were opened before January 1st, thanks to prior funding.

1. Preventing high-risk NB children from relapsing. This is an area where we feel we can have the most dramatic impact on survival in the short-term. We hope to have phase II clinical trial results published early in the New Year, and the results are very promising.

2. The first ever clinical trial using genomic medicine – along with standard chemotherapy – for newly diagnosed children with high-risk neuroblastoma. This trial was funded by DELL, and is also using DFMO to hopefully prevent any children from relapsing. This is part of the longer-term strategy to, not only increase survival, but to work on decreasing the toxicity of the current standard of care.

3. A new trial was opened for NB kids, combining tolcapone and oxaliplatin for those kids battling relapsed or refractory disease.

4. Six new agents were tested in the lab, with the two most promising agents looking to move into phase I clinical trials in 2016.

5. The consortium has added 8 new sites – thus bringing the treatment closer to more children across the United States.

In addition to my duties with Beat NB, I am honored to continue to serve on the Executive Committee for the consortium, which is funded by your support. In addition to this committee, earlier in 2015 I was also appointed to the role of Executive Advisor to the NMTRC chair. Both roles allow me a unique look into exactly how our funds are being used, and that transparency only further fuels my desire to lead Beat NB to do more.

This work is where the tide will be turned – and your support is making it all possible.

Thank you for all you have made happen and it is with great enthusiasm that I look ahead to making 2016 a year to remember.

Sincerely,

Patrick Lacey

How we’re beating kids’ cancer with computers.

This 5 minute video is an abridged version of the article below.

A history of the NMTRC’s genomic-based treatment for childhood cancer, and a new paradigm for today.

The launch of a new clinical trial called PEDS-PLAN in September of 2015 marks a paradigm shift in childhood cancer treatment and precision medicine — the first time DNA and RNA sequencing are being done in newly diagnosed patients. This article will take a look at the last five years of genomic research in childhood cancer done by the Neuroblastoma Medullablastoma Translational Research Consortium (NMTRC), and what it means for the future of precision medicine.

If you follow the clinical research world, you’ve heard of personalized medicine, or precision medicine, or molecularly guided therapy (MGT), or genomics. They’re different terms for what is generally the same concept — personalizing treatment per patient based on specific genetic or molecular analysis. In childhood cancer, genomics is being used to beat cancer on a kid by kid basis.

Source: Forbes.

Beat Nb is dedicated to finding and funding a cure for neuroblastoma and other types of childhood cancers. We have directed over $5 million directly to patient-affecting childhood cancer research.

We helped to found, and now fund, the NMTRC, an international consortium of 25 hospitals (and growing) conducting clinical trials and research with a shared goal to ours — beating neuroblastoma and other childhood cancers. Much of the NMTRC’s work, in partnership with Dell and TGen, has been in leading genomics research in childhood cancer. (Full disclosure: this author sits on the Executive Board for the NMTRC, and Beat Nb supports various NMTRC trials financially.)

Some background on genomics: the human genome was first completely mapped in 2003. It took 10 years, and $1 billion. Today, in 2015, TGen can process a patient’s genome in 6 hours. The cost is in the thousands. The sharply dropping costs and speed of genomics have opened a new world of research. There is much work to be done still — we could map the genome in 2003, and now can do so quickly and cost effectively, but what do we do with that data? That is what is being researched worldwide now — across many malady types.

Genomics in cancer is exciting. Cancer kills because a group of cells are mutated, and keep duplicating without stopping. Your body is supposed to maintain a state of equilibrium (called homeostasis) as you go about your life. Cancer cells ignore this, and keep duplicating. The mutations that make cancer cells keep going are what cancer genomics look at. And the goal is to then treat with a drug targeted to that specific mutation — knocking out the tumor with a precise medicine, rather than a blanket chemo hoping to destroy the tumor.

While sequencing can be done at most hospitals today, it is important to understand what depth of sequencing is done for each child, and how this information is being interpreted. The NMTRC’s molecular tumor board has developed significant expertise over the last 5 years in the analysis of the genomic sequencing, to make the most informed clinical decisions for children. This is truly the key to best personalized care, taking into account the previous medical treatments, the current condition of the patient and wishes of the family.

The NMTRC, chaired by Dr Giselle Sholler, first started clinically looking at genomics in childhood cancer with a trial opened in 2010. They’d noticed in treating children with neuroblastoma since 2005, kids all had very different tumor profiles. With genomics starting to be clinically relevant, a trial idea was formed.

Dr Giselle Sholler, Chair of the NMTRC, and one of her patients. Photo credit: Spectrum Health.

The pilot trial (NCT01109238, completed), opened in April 2010, enrolling 5 children to test if it was possible to support “real-time” (quick enough to treat with) treatment decisions through mRNA expression data genome-wide in neuroblastoma biopsies. At the end of that trial, the process was able to be completed in 12 days. This was the first genomic-based personalized medicine childhood cancer trial in neuroblastoma. (Results published here). It’s important to note, the most accurate and deepest data for determining best treatments comes when using both RNA and DNA expression and sequencing. However, in 2010 the tech wasn’t yet able to support doing all of that quickly enough to get “real-time” data for making treatment decisions. But RNA expression could be done quick enough, so it was time for a new trial, one actually treating kids.

How a drug prediction report was generated in the NCT01109238 study. Source: Journal of Cancer Therapy, 2012, 3, 602–612

The following year, in 2011, a Phase I trial (NMTRC 001, NCT01355679, completed) opened with a larger enrollment of 14 children fighting neuroblastoma, to test the feasibility and safety of actually treating kids with these individualized therapies. (Results published here). Now, the NMTRC was treating children using the data generated by their RNA expression profiles (what the first trial tested if was possible). In addition, as research, DNA mutation panels were done (these look at 50 common DNA mutations for cancer) to see if they’d be feasible / useful to add to the data gathered.

A significant partnership with Dell (along with an initial commitment of $4m, which has continued to grow over the years), brought Dell’s cloud supercomputing power to help analyze the data.

In May of 2012, we had written a blog update about the rapidly increasing speed and relevance of using genomics in realtime treatment, after discussions at the 2012 NMTRC Symposium:

This year focused heavily on personalized medicine trials, or targeted treatments. To explain, a little history — chemotherapy, which was only discovered in 1942, is the main treatment for cancer. Chemotherapy is poison — in fact, the first chemo was mustard gas, which was injected into patients after folks during World War 1 noticed it suppressed blood production. Because cancer grows faster than normal cells, the chemo kills cancer faster than it kills the body (is the hope), and then the body recovers (is the hope). You know all those pictures you see of bald, skinny, hollow eyed cancer patients? That’s not cancer doing that — it’s chemo. Of course, it’s the best we’ve had until now.

Since mapping the human genome, we can now attempt to target therapy. The NMTRC’s work is focusing on (among other things — and this is a high level overview) taking a biopsy of a tumor, identifying it’s mutations, and treating with a chemotherapy [sic — this could be various drugs] which will target ONLY these mutations — making the treatment go from systemic (poisoning the entire body) to targeted (poisoning only the tumors). It’s showing a lot of promise.

There’s a LOT of information, and up until recently it could take 17 days from biopsy to a list of targeted drugs (these are FDA approved drugs). Dell has stepped up this year to a long-term commitment to the NMTRC’s work with millions of dollars of hardware and systems analyzing. This has already cut over 2/3 of the processing time down — from 17 days to roughly 5 days.

Building on the past three years of research and continuing at, for the clinical research world, a rapid pace, a third personalized medicine trial by the NMTRC (NMTRC 008, NCT01802567, ongoing, but not recruiting participants) was opened, accruing 48 kids. The partnership with Dell suggested the hope to complete an RNA expression profile, a DNA Mutation Panel, genomic analysis and report generation, a tumor board held with treatment decision, and treatment review completed and start of treatment — all within 21 days of a child’s biopsy/surgical resection date. It worked.

Source: Dell.

Between 2013 and 2014, Dell continued to increase technology and speed, and where sequencing an entire DNA exome (important because it tests allyour DNA mutations, not just a common set) used to take 2 months (not fast enough to be used while treating children, or “clinically relevant”), it could now be completed in 2 weeks. Additionally, TGen became the first CLIA certified lab to have RNA sequencing available, in September of 2014. CLIA certification is needed to use RNA sequencing for treatment decisions. These major advances led the NMTRC to open a new trial.

In 2014, a 4th trial was opened (NMTRC 009, NCT02162732, currently recruiting participants). 48 children with cancer would be the goal to test again, now with full DNA exomes being completed, as well as RNA sequencing (remember, sequencing is a much deeper look than expression profile). This study would determine feasibility of using tumor samples to assess genomic sequencing using predictive modeling to make real-time treatment decisions for children with relapsed/refractory cancers. The entire DNA exome was now being sequenced (more powerful than a DNA mutation panel alone), RNA was sequenced, and the data was analyzed and a treatment decision made.

Watch this video to see how TGen and Dell are collaborating with Dr Sholler and the NMTRC to beat kids’ cancer.


A new paradigm in treating children with neuroblastoma.

In 2012, the ASCO Post wrote:

Although Dr. Sholler’s trials are the first attempt in personalized medicine for children with relapsed disease, if successful, genomic-based medicine for pediatric patients with cancer may eventually be used in the front-line setting, where the greatest chance for cure may be possible.

Today, that goal is being realized. Just 5 years after the NMTRC began clinical trials to research moleculary guided therapy in childhood cancer, in September of 2015 a new trial opened incorporating MGT into upfront therapy (PEDS-PLAN, NCT02559778, currently recruiting participants). This trial represents a new paradigm in treating children with neuroblastoma, based on and uniting the work the NMTRC has done with personalized medicine and a drug called DFMO, which hopes to stop neuroblastoma relapse. The trial, called Pediatric Precision Laboratory Advanced Neuroblastoma Therapy (PEDS-PLAN), suggests using molecularly guided therapy in combination with current standard therapy (COG protocol), followed by maintenance therapy with DFMO in subjects with newly diagnosed high risk neuroblastoma.

This is the first time DNA and RNA sequencing are being done in newly diagnosed patients.

A targeted agent (drug) is added to standard of care therapy. Because it is known from experience the expression profiles of tumors change throughout a child’s treatment, they do the RNA and DNA sequencing again after the 4th cycle of chemotherapy, when the tumor is removed. Using precision medicine in this way, in upfront therapy, truly gives the greatest chance for a cure. It’s expensive — full exome sequencing currently costs around $12,000, and RNA sequencing around $7,200. Dell has graciously committed to covering these costs during this trial.

Genomics is an exciting weapon in battling disease, and precision medicine has an army of advocates moving it forward. Not the least of these is thePrecision Medicine Initiative launched in January of 2015 by President Obama. The Precision Medicine Initiative’s mission is to enable a new era of medicine through research, technology, and policies that empower patients, researchers, and providers to work together toward development of individualized treatments.


Others are also carrying this work forward, even in childhood cancer. St. Jude and Washington University have launched the Pediatric Cancer Genome Project. Columbia University Medical Center is now sequencingevery pediatric cancer patient’s tumor. As the NMTRC marks five years into childhood cancer genomics research, more continue to join the battle.

A new trial, the NEPENTHE trial (Next-generation PersonalizedNeuroblastoma Therapy) is slated to begin in 2016. However, this trial is using only a DNA mutation panel for the results, not full exome sequencing. Their RNA sequencing is not being done in a CLIA certified lab, and therefore won’t be used to make treatment decision — critical in pediatrics, given the low mutation rate. Scientifically, this study will answer the target question for those drugs, but a concern will be how well these single or double agent treatments without chemo will affect relapsed neuroblastoma.

Collaboration

Collaboration is crucial to moving work like this forward. The NMTRC now has trials operating in over 25 institutions across the US and internationally. Partners like TGen in Phoenix and Dell in Austin add to the passion and expertise being directed to beating childhood cancer with genomics.

Precision medicine generates vast amounts of data. Converting that data into actionable (and effective) clinical outcomes is a very nuanced and learned approach. The physicians and scientists in the NMTRC continue to learn how best to make use of these new approaches, having worked through the process over 80 times now (data, rules, tumor boards to determine best treatment options).

Mature rules have been developed to determine what pathways and drugs are clinically relevant in neuroblastoma. Technical partners are refining analytics and sequencing processes, and speed keeps increasing. Advances in mobile and cloud computing will continue to offer more relevant and available data, and easier ways to digest it for use in the clinic (treating kids). The next five years are just as exciting as what’s happened already.

I wish Ezra could have been diagnosed today instead of in 2009. And of course I don’t mean that — if we’re granting wishes I’d wish he never experienced a single minute knowing what cancer feels like. I can’t help but feel proud, and energized, seeing the huge leaps in treatment and research in the 6 years since Ezra was diagnosed with stage 4 neuroblastoma. Robyn and I know in some small way, Ezra’s name will one day be one of so many whose stories led to a cure. With incredible and caring physicians and resaerchers like those in the NMTRC, and other passionate advocates like ourselves, we will beat neuroblastoma.


If you’d like to be a part of this work being done to cure childhood cancer, we welcome your involvement. If you are a parent seeking information on any of these clinical trials (or anyone else with a comment), you can reach us at beatnb.org/contact. You can support financially at beatnb.org/give. Beat Nb is a 501(c)(3) tax-exempt organization with the Internal Revenue Service. Our EIN is 27-2314549.

Brighthouse Networks Enterprise Solutions 2nd Annual Charity Classic brings in $90,000 to support childhood cancer research!

Craig Cowden of Brighthouse Enterprise presents a check to Robyn Matthews of Because of Ezra

Brighthouse Networks Enterprise Solutions (BHNES) chose Because of Ezra as the beneficiary for their 2nd annual Charity Classic golf tournament. We spent the day at the beautiful Saddlebrook Resort in Wesley Chapel, FL. Yesterday, Brighthouse Networks Enterprise Solutions presented Because of Ezra with a check for $90,000! This money goes straight to childhood cancer research. We are so incredibly grateful.

BHNES have been incredible supporters of Because of Ezra – yesterday’s event marked over $170,000 they’ve raised toward beating childhood cancer with us. Between BHNES and their incredible patrons – the many sponsors of the event, we’ve been humbled at the support. BHNES have connected with the research we are doing with the NMTRC, and our cause in a deep way, and know our story and mission well. It is an honor to be partnered with a group who delves into our workings and sees the promise this research is showing.Over 100 golfers came and the feedback was the same as last year – many golfers said this was one of the best tournaments they’ve ever been to. Sherrin Smyers of Eventrics did a brilliant job of event planning. We owe huge thanks to Craig Cowden, Brian Zelenka, and the entire team at Brighthouse Networks Enterprise Solutions. These are caring professionals.

As I’m writing this, I’m in a conference room with a dozen doctors, parent advocates, and medical professionals discussing one of the most promising trials we’re helping to fund. The results are groundbreaking, and this room is full of people who are truly bringing hope to families who are fighting neuroblastoma today, and those who will tomorrow.

Brighthouse Networks Enterprise Solutions, and all our supporters, are making this possible. We can’t say enough how deeply thankful we are. It takes all of us. Thank you.