Because of Ezra

What’s Going on in Neuroblastoma Research – 2013 NMTRC Symposium

The NMTRC (Neuroblastoma & Medulloblastoma Translational Research Consortium) is a group of 18 universities and children’s hospitals headquartered in Grand Rapids, MI which offer a nationwide network of childhood cancer clinical trials. The group is chaired by Dr Giselle Sholler. These trials are based on the research from a group of closely collaborating investigators who are linked with laboratory programs developing novel therapies for high-risk neuroblastoma and medulloblastoma.

They’re one of the groups Because of Ezra supports financially under our mission of funding relevant, patient-affecting research into a cure for neuroblastoma. The NMTRC hosts an annual symposium, where the members meet to discuss the previous year’s work and future directions. The 2013 symposium was this past Monday and Tuesday (May 6-7) at the Wyndham hotel in Lake Buena Vista, Orlando, FL. Video will be on YouTube within the next week or two (and we’ll mention when it is), but we wanted to share some of the experiences here first. You can watch the entire conference on YouTube if you’d like, separated by discussion.

The NMTRC is unique in the level of community between the physicians, scientists, and families (both in treatment, out of treatment, and those running foundations). We largely attend the same presentations, dinners, and have structured open conversations about the work being done. The depth of collaboration is refreshing and inspiring. When you have parents whose children are ON a trial participating in the same conversations as the scientists who proposed the trial, the organizations funding it, and the physicians carrying them out… it’s an incredibly effective synergy.

The work being done is truly remarkable – the first ever FDA approved personalized medicine trial for pediatric cancer, preventative trials to reduce the rate of relapse (in this case with a drug called DFMO), immunotherapy, and more. One of the most interesting presentations was by David Krag, MD, who is the SD Ireland Professor of Surgery at the University of Vermont (yes, he said, surgeons do science, too!). Titled Personalized Antibody – Timeline to Clinical Impact, Dr Krag discussed an exciting method of harnessing the body’s own immune system to create personalized antibodies specifically for an individual’s neuroblastoma tumor. It should be going to trial this year.

Personalized medicine is a concept at the core of what the NMTRC is doing. It has the huge benefit of being much less toxic (and much more targeted) than the standard chemotherapy regimens currently prescribed for neuroblastoma. These current standards are not effective (survival rates for stage 4 neuroblastoma hover at 40%, with relapsed neuroblastoma still having no known cure), and include rounds of treatment with what physicians call “kitchen sink chemo.” Partnerships between the NMTRC and the Translational Genomics Research Institue and Dell have pushed forward much of this work by analyzing individual patient’s tumors to find treatment options specific to their disease.

We got to spend some great time with non-profits also working toward a cure for neuroblastoma – and families who are battling it now. Some of the children represented included Will Lacey, Melina Riniolo, Brooke Hester, Saoirse Fitzgerald, Abigail Goss, Daxton Blanford, Chase Ringler - and there were even more. These children are each reasons we fight, and reasons why continuing the battle against neuroblastoma is so very important. Neuroblastoma CAN and WILL be beaten – and it is going to take time, passion, awareness, and funding. We are so, so, so, so grateful to have your support in making this a reality.

Giving Away $100,000 in 2012

Today marks the day we’ve been without Ezra as long as we were with him. 800 days from the day he died, after living exactly 800 days. It’s a strange day for us.

We thought it would be fitting to do Because of Ezra’s giving for 2012 on this day – this is only something we could have done Because of Ezra. So this morning, Robyn and I presented a gift from Because of Ezra in the amount of $75,000 to the neuroblastoma work being done by Dr Giselle Sholler at the Van Andel Institute in Grand Rapids, Michigan. Our gift was matched by an incredibly generous local family trust, meaning the total gift will be $150,000. This is specifically to fund a trial using DFMO, an investigational new drug which prevents relapse of neuroblastoma. We personally know many of the children on this trial, and it’s a drug which has no significant side effects, and so far has been extremely effective. 50% of children who fight neuroblastoma to the point of no evidence of disease relapse – and there is no current cure for relapsed nb. Preventing relapse would be a huge accomplishment. Dr. Giselle Sholler received the gift along with the CEO of the Van Andel Institute, David Van Andel.

We are honored to be able to contribute to work which is making a huge difference in children’s lives who are fighting the disease Ezra died of. In all their names, and from the bottom of Robyn and I’s hearts – thank YOU for supporting what we’re doing Because of Ezra. We couldn’t have done this today without all your support raising awareness and funds. Our board members Marissa Devins, Erica Copeland, and Derek Pupello (and their spouses) all deserve a huge thank you as well – they are incredible people who make doing all this easier, and we are so grateful to have them on board. They are also amazing friends.

Late in December, we also supported the work being done by Dr Robert Seeger and the team at NANT in Los Angeles, with a $25,000 gift. NANT is doing some great work, including a study on the biology of neuroblastoma, and we’re honored to support them as well.

Through your help Because of Ezra was able to give $100,000 in 2012 to researchers, physicians, and scientists making a difference in survival today for children fighting neuroblastoma. Thank you, thank you, thank you.

Robyn mentioned raising awareness and giving toward a cure is a way for her to continue being Ezra’s mother – to keep honoring his name, to keep fighting the fight he started for us. We know the hurt neuroblastoma brings to a family, and to be able to take that away from someone in Ezra’s name has so much meaning for us.

We look forward to doing even more to cure this horrible disease in 2013 – and telling more people about the need, the fight, and the ways to cure this. It is possible, and with people like you helping us, we know we can do this.

Thank you.

2012 NMTRC Symposium

You may wonder where the money you donate to Because of Ezra goes. A large portion of our outgoing financial support is to the NMTRC, or Neuroblastoma Medulloblastoma Translational Research Consortium.

Chaired by Dr Giselle Sholler, the NMTRC is a group of 18 universities and children’s hospitals headquartered at Van Andel Research Institute that offer a nationwide network of childhood cancer clinical trials. These trials are based on the research from a group of closely collaborating investigators who are linked with laboratory programs developing novel therapies for high-risk neuroblastoma and medulloblastoma.

Each year, the NMTRC members and supporters meet up at the NMTRC Symposium to have a “state of the union” so to speak of the past year’s work. This year’s symposium was in Austin, TX this past Wednesday and Thursday (May 16-17), and Because of Ezra founders Kyle and Robyn Matthews attended the event for our second year.

One of the most refreshing parts of this group is the community aspect – over 100 physicians, researchers, scientists, and family members show up each year to meet all in one place. It’s very collaborative - we share meals together, stories, laughs, tears, and of course talks of progress made in the past year. The NMTRC is a passionate group of extremely bright individuals making a difference – and we’re very proud to be a part and toss our dollars in the hat.

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 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.

Another important and exciting new trial is centered around DFMO, a drug taken orally. This drug can be taken by kids who have reached no evidence of neuroblastoma disease – to prevent relapse. Relapse is highly likely, and there are very few current options. Will Lacey, son of Friends of Will founder Pat Lacey, has been taking DFMO since May of 2012 with zero side effects, and normal blood counts.

This is incredible work. What’s even more amazing is the targeted medicine trias are being pioneered in pediatric cancer instead of adults. Since the 1980s, the FDA has approved only one new pediatric cancer treatment, compared with 50 treatments approved for adult cancer in this same timeframe. There is still a lack of attention and funding on pediatric cancer.

We need to beat this.

Making a Difference – Giving Away $30,000 from 2011

Because of Ezra seeks to fill some of the large gaps in pediatric cancer research funding and focus on these solid-mass cancers. Our goal is to raise money to support relevant research and clinical trials with physicians and researchers who can help make a difference in survival today. You’ve probably heard us say that before. But what does it mean? Who are these people we support?

We’ve just finished up doing our 2011 year end reports (letters to come to all you folks who donated!). Thanks to all of you, our supporters, we were able to give $30,000 to researchers who are in fact making a difference in survival today. I thought I’d highlight the two organizations we chose to support this year.

Dr Giselle Sholler | Van Andel Institue / Neuroblastoma and Medulloblastoma Translational Research Consortium

We first met Dr Sholler when working through Ezra’s treatment in Orlando, in October of 2010. She was working on a trial of a drug called Nifurtimox (now in Phase II) in treating neuroblastoma and was extremely helpful in getting us approved for use of the drug on a compassionate use basis. Robyn and I remember her answering our calls and emails (and calling and emailing us) into the late hours of the night trying to get Ezra approved; and she’d never met us before.

Since then we’ve met with Giselle and her team multiple times, and are incredibly impressed and inspired by her passion, focus, and intellect. She has recently moved from her position with the Vermont Cancer Center to co-directing the VARI Pediatric Cancer Translational Research Program at the Van Andel Institue in Grand Rapids, Michigan. The VARI program will enable large-scale projects that will use the latest genomics and molecular analysis techniques to identify the best treatment options for children based on the individual biology of their tumor, a process often referred to as “personalized medicine.”

We’re proud to count Dr Sholler a personal friend and a partner of Because of Ezra.

Dr Robert Seeger | Los Angeles Children’s Hospital / New Approaches to Neuroblastoma Therapy

Dr Bob Seeger (no, not this guy) is another incredible researcher. As head of the New Approaches to Neuroblastoma Therapy (NANT) Consortium, Dr Seeger works on developing and testing new therapies that will be targeted specifically to neuroblastoma cells, and therefore improve the outcome for children with advanced neuroblastoma with fewer side effects.

We first met Dr Seeger in LA in 2010 when one of our board members reached out to him for an understanding of what was being researched in high risk neuroblastoma. While low and intermediate risk nb have very high cure rates, high risk (stage 4) still has a dismally low survival rate. Dr Seeger has a fiery attention on fixing this. We’ve since spent more time with him and the team in LA, including touring their lab. They are doing exciting work.

We’re also proud to count Dr Seeger a friend and a partner of Because of Ezra.

Hopefully that gives you all an idea of the people and organizations we are partnering with in order to snuff out neuroblastoma. It is a beast of a disease which needs awareness raised in order to further promote research, and funding to continue that research. Your help makes a huge difference – every $10, $20 or $1,000 comes together to be what we like to call “a growing fist in the face of pediatric cancer.”

Thank you. 

CaseFactori – Cases with a Cause

CaseFactori is a brand new (they just launched Saturday) company which sells iPhone and iPad cases. They’re a bit different though – a portion of every sale they make goes to charity! After meeting us through a review on ModMyi.com, they’ve added Because of Ezra to their list of (now 5) supported charities. With so many iProducts out there, and hundreds of accessory companies, it’s exciting to see a company coming out supporting charity.

Driven by their compassion for people and the love for revolutionary products, Case Factori unites your generous support with organizations that are making substantial changes in the lives of others. While Case Factori cares a great deal about the products they want to bring to each of their customers, they dedicate their hearts fully to connecting each customer to a cause that might be close to their hearts. They care about those movements, and believe in the changes each organization continues to make throughout the world.

So check them out if you need a new case!