3 Changes Coming To Clinical Trials

Lessons Learned, Uncategorized

Clinical Trial Changes

If you have a “rare” cancer that doesn’t have a great standard of care, chances are you will be offered a clinical trial. To the general public (which is who we all are before that diagnosis is presented) clinical trials sound like a scary thing. To a cancer patient being told there is no cure, a clinical trial is a lifeline being tossed in a stormy sea. IF you catch it, it MIGHT help save your life.

So how do you pick a clinical trial? Well, first you have to find one that you qualify for. We’re going to assume that you have a doctor who is really helping you and is presenting you with some choices. So you have a couple of clinical trials and the “standard of care” to choose from. How do you decide? Right now, it’s a guessing game, but all of that will be changing rapidly as technology and open access data become more commonplace in the process.

  1. You will have more concrete data to help make decisions. I have seen some pretty impressive technology being developed by Clalit Research Institute in Israel that will help a doctor walk through a list of weighted questions with a patient that will help them make this difficult decision. (That program was developed using data made available from a clinical trial, and as more data becomes open access, I think we can expect to see more applications like this developed.) Each patient will be able to rate a list of possible side effects and based on their feedback, an algorithm will provide guidance on particular trials.
  2. You will know more about what “successful” patients look like. As data begins to become collected in one place, it is easier to compare patients on a genomic level. Researchers will be able to compile profiles of successful patients to help determine who has the greatest chance of success on a trial. My son participated in a clinical trial where one patient was doing really well. We had no idea if David would have the same results because there was very little data to tell us why the first patient was successful.
  3. The system will start to find you. Right now, clinical trials are found largely by patients and doctors sifting through websites like clinicaltrials.gov to find possible trials. In the future, doctors will enter your information into the computer, and then you will be pre-qualified based on your exact diagnosis and personal information. The computer will then present a list of potential trials that you can choose from.

This all might sound a little too good to be true, but the fact is, the infrastructure is already in place. Cavatica.org is an open access research platform that Dragon Master Foundation and others have been funding for more than three years now. It houses a patient’s full genome and biosamples from the patient, and sometimes the patient’s parents. It also links to the patient’s clinical records so we can have a longitudinal view of that patient. I believe it is the single most complete picture of a patient you can get, and we are working hard to make it available to everyone. (At the moment, it is largely working with pediatric brain cancer data, but the platform is built to expand as funding becomes available.)

Data like this can take a lot of the fear and guessing out of treatment, and it should lead us to more successful treatments and cures. We are on the cusp of a meaningful shift in cancer care, and I’m excited for this to start really impacting patients lives.

#Cavatica Cancer Research Database Has Launched!

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The day is finally here, and I am so excited! Today is the day the beta version of Cavatica launches to the cancer research world. A dream we had almost three years ago is coming true today. It’s not done. Technically, it will never be done. It will always be adding new patients and more information. Putting that aside, it isn’t as “done” as we want it to be. There is more functionality to add. There is plenty of DNA sequencing yet to be completed. But today, there is a pretty awesome database that is already unlike anything else cancer researchers have had access to before.

If you know a doctor or researcher, please tell them about this awesome resource. We will continue to build it out and make it better, but I think they will be blown away – even right now. (Say those last three words in your best Southern accent. That’s the only way to truly give them the emphasis they deserve.)

Here is the official press release:

The Children’s Brain Tumor Tissue Consortium (CBTTC) and the Pacific Pediatric NeuroOncology Consortium (PNOC) conclude Brain Cancer Awareness Month of May with the announcement of the Beta launch of Cavatica, a new cloud-based environment for securely storing, sharing and analyzing large volumes of pediatric brain tumor genomics data.

Cavatica will, for the first time, allow doctors, researchers and data scientists unparalleled access to pediatric brain tumor genomic data paired with a suite of analysis tools in a cloud computing environment that enables scalable, faster and more robust research. Upon its full release, Cavatica will host the largest standardized, integrated, and quality-controlled genomic database of pediatric brain cancer genomic data.

Working with Seven Bridges, a biomedical data analysis company, the eight CBTTC site members and 15 member hospitals of PNOC are further fulfilling their commitment earlier this year to the White House Precision Medicine Initiative (PMI) with the launch of the Beta version of Cavatica being announced today. The Beta release will be open for subscribed end-user input and will be iteratively enhanced by ongoing implementation of advanced platform features and deposition of additional data sets over the coming months. These datasets will include additional pediatric cancer supporting pan-cancer pediatric data analysis in partnership with additional consortia. including the SU2C-St. Baldrick’s Pediatric Cancer Dream Team.

Sign up for access to the Beta release of Cavatica is available for researchers and data scientists by going to cavatica.org.

For more information or to schedule an interview, please contact the CBTTC at CBTTCadmin@email.chop.edu

It’s Kind of a Big Deal

Dragon Master Foundation
Wish I knew who to credit for this pic because it is awesome.

Wish I knew who to credit for this pic because it is awesome.

We get a lot of questions about Dragon Master Foundation, and whenever I have the chance to talk to someone about it, the response is amazing. They always end up saying “Wow, that’s such a big deal!” People are so generous with their support once they understand the project. The problem is, a lot of people don’t understand what we are doing and why it is needed. So I thought I’d take a moment to explain a little bit about what makes this project so special.

When David was sick, we were inside hospitals for days at a time watching people do their jobs. Technology is everywhere – from the patient bedside to databases in some unseen corner of the building. However, all of that technology seems to be locked inside each institution, with very little ability to share information from one hospital to the next.

It is like  being a horse with blinders on. You can only see a small part what’s really out there. You get a myopic view of the world. Unfortunately, that is the world most cancer doctors and researchers face. They long for more information, but it is largely out of their reach.

You may be thinking, “But what about the internet? Can’t they just send their information back and forth?” The short answer is no. Between HIPAA, different technology formats, and the sheer size of data, even the most collaborative hospitals have trouble sharing all the information researchers want to access. Collaboration would mean that a database would quickly need to warehouse petabytes of of information – a task that has only been tackled by the likes of the NSA or Google in the past.

It is an overwhelming task, to be sure, but for the first time in history, it is possible. It is possible to house genetic information and clinical data in one place so that researchers can really see the “big picture” of a patient’s health and furthermore, they can compare that patient to other patients. They can start to see why a drug works for one patient and not another. They can start to make sense out of things that are seemingly random.

It will be four years this September since we were dropped into this cancer world. I’m not a doctor or a researcher, but I’ve talked to as many as I could over that time, and every one of them has said a database like this would be an asset to them. EVERY ONE OF THEM.

And yet, we continue to spend money on tiny projects that help a single researcher or a single hospital. Please don’t misunderstand. Every researcher needs funding. Every hospital needs more help. But this is a situation of not being able to see the forrest for the trees. We need to build an infrastructure for the research data if we ever hope to move at a pace that is faster than cancer.

The good news is, we have made amazing progress. We have joined forces with the Children’s Brain Tumor Tissue Consortium, Children’s Hospital of Philadelphia, Children’s Hospital of Pittsburgh, Chicago’s Lurie Children’s Hospital, and Children’s Hospital of Seattle to take the database they are working on and grow it to a scale that can help pediatric and adult patients. The data is already being collected, which is a great and wonderful thing. However, it means that we are already at a place where we need vast amounts of funding in order to continue to grow.

I wake up every morning more sure that this database will change the way they do medical research. I have hope that people will begin to understand the vision that that this database represents, and that they will focus on helping us build it. You ABSOLUTELY CAN make a HUGE difference in the fight against cancer. Please share the mission of Dragon Master Foundation. Like us on Facebook ( http://www.facebook.com/DragonMasterFoundation ). Follow us on Twitter (@dragonmasterfdn and/or @amandahaddock ). Host a grass-roots fundraising event. Something as simple as dining out at a local restaurant that will donate proceeds can be a huge help with both raising money and raising awareness. Cancer is a beast that is taking lives. You can be a dragon master. Please join us today!

What Are You Doing To Cure Cancer? You May Be Surprised At How Much Power You Have In This Battle.

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This morning I woke with a start and was completely wide awake. I am not a morning person, so this was very unusual for me. I had been awakened by my dream. In my dream, I was able to think a little more freely about some questions as they relate to cancer research, and I woke up super excited to put those questions down on paper.

The timing is perfect as four board members from Dragon Master Foundation are traveling to Philadelphia this week to meet with the Childhood Brain Tumor Tissue Consortium. This is the second meeting for Richard and I, but the first time we are able to take other board members. It is significant because the progress we can make in person is more collaborative than what we can discover over the phone. Rapport has been built, and I believe both teams are eager to forge a bond that will help us move this big data project forward.

I can’t really share every thing that is going through my mind, but there is a central issue that I think is key for all of us to understand. It starts with Ted Kennedy.

Ted Kennedy was diagnosed with GBM in 2008. He went to the best doctors money could buy, and he died anyway. There is much to be learned about brain cancer. My son, David, was diagnosed in 2010, and he died in 2012. He also had amazing doctors, and unlike Mr. Kennedy, David had great health and youth on his side. It didn’t help. The doctors and researchers didn’t have any new information to work with that might make a difference. There has been little progress in brain cancer research in the last few decades. However, in the last couple of years, scientists have discovered that GBM is not just one disease. There are at least four subtypes. It is easy to understand that these subtypes might all react differently to different treatments. Right?

And now for the really important part. If there are four subtypes of GBM that have just recently been discovered, what other things might we have been missing in the “big picture”? You see, we are in a revolutionary time in both science and technology. And one can barely keep up with the other. Researchers are pushing to the edge of their worlds – eager to go farther, faster. Data engineers are using bigger sets of data in new and innovative ways – eager to go farther, faster. But there are precious few resources being given to bring these two sets of people together. Imagine a researcher writing out all the questions he or she is trying to answer. There may be hundreds of possible scenarios they want to investigate. It would take a whole lab full of people months, or maybe even years, to answer all the questions. They might not ever be able to answer the questions because they simply don’t have access to enough data.

That is where the data engineers come in. They can take the data and program the computer to find similarities and differences, greatly reducing the number of man hours needed to get answers. The questions are hard, but the answers could be easy.

However, before any of that can happen, we have to build the database. The exciting thing for us is that the team at CBTTC has already begun doing that. They have started “small”, with childhood brain tumor patients, but their vision is big. We believe it is going to be able to scale quickly to include adult tumors and then even beyond brain cancer. This is the way research will be done in the future, and we are poised to make it happen now. NOW. And the faster this database is built, the faster they can save lives.

To say we are working with a sense of urgency is an understatement. It woke me up this morning. I think about it every day. I truly believe the only thing standing between us and a cure for cancer is this lack of a database. So we are going to build it, but we truly, desperately need your help.

Everyone can do something, and right now the thing we are going to need most is resources to build the database. We need citizens like you to care about a cure for cancer right now. You need to ask your hospitals if they are sharing data. Don’t accept no for an answer. We need to build this database big enough that they can all share -which is going to take a lot of money. The good news is that the major expense is in building the database. Once the resource is built, ongoing expenses will be a fraction of the start-up costs. So the help we need in the beginning will diminish over time. When the database is fully functional, it will save money elsewhere that could be re-allocated to fund the database.

Please share this story with everyone you know. It is so important that we get this database built. It is too late for my son, but there are so many other lives at stake. We can’t afford to wait for someone else to fix this problem. We have to do it ourselves. Now.

If you’d like to donate to the project, you can make a tax-deductible donation here:
http://www.dragonmasterfoundation.org/get-involved/

If you would like to plan a fundraiser in your area, please let me know and we can help you get started. We CAN do this!

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