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.

Working Together for A Brighter Future

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This week I had the great pleasure of speaking with Dr. Peter Adamson, Group Chair of the Children’s Oncology Group (COG). For those of you unfamiliar with COG, more than 90% of  children and adolescents diagnosed with cancer each year in the United States are cared for at Children’s Oncology Group member institutions. Their goal is to cure all children and adolescents with cancer, reduce the short and long-term complications of cancer treatments, and determine the causes and find ways to prevent childhood cancer. That matches our mission pretty well, so I was excited to learn where we might be able to collaborate.

COG is currently focused on collecting biospecimens and clinical data. In layman’s terms, they are collecting cancer specimens (tissue, blood, etc) as well ad information on the child’s diagnosis, treatment and outcome. They have collected a massive amount of data over the past 50 years. They have well over a million biospecimens! More than 350,000 patients have shared data with them. They have biorepositories and databases in different parts of the country and work with over 220 hospitals in the US & Canada.

I am very impressed by what they have accomplished, but ultimately, I believe that the infrastructure we are building can improve the work they are doing. Their focus is collecting the specimens and data. Our focus is taking those specimens and data and making them a perpetual resource backed by robust computational power to allow them to collaborate with other researchers and also analyze and visualize the data in new ways.

To give you some idea of the scale of the data, let’s look at the numbers. There are approximately 14,000 children a year diagnosed with cancer in the US. Collecting a biospecimen would cost somewhere in the range of $1,000. (The NIH currently values that at around $500, but the actual institutional cost is thought to be much higher, thus my $1,000 figure.) So just to collect the biospecimens for those patients, you are looking at $140,000 per year. However, that is just scratching the surface of what needs to be done. Those specimens have to be stored (visualize giant freezers with robots to access the individual samples), categorized, and matched with corresponding clinical records.

Traditionally, most hospitals and foundations have been unwilling and/or unable to invest in the infrastructure that it would take to compile this amount of data. COG demonstrated real vision by collecting this data and they have been able to use it to forward science. Dragon Master Foundation believes that additional computational power, or “big data” analytics, will help them find the cures they seek even faster.

Dr. Adamson said he felt Dragon Master Foundation is taking “a sophisticated look at the challenge.” We know that building this type of computational infrastructure will be expensive, but we also know that it will exponentially decrease the amount of time it takes for researchers to collect and query data. Faster answers to their questions means faster cures for us.

There is no doubt in my mind that we are building a resource that will improve cancer research. It ultimately will help cancer researchers throughout the US, and probably throughout the world. It will make the work they have been doing for years more relevant.

To learn more about Dragon Master Foundation, please visit http://www.dragonmasterfoundation.org. To learn more about the Children’s Oncology Group, please visit projecteverychild.org or childrensoncologygroup.org .

52 People You Need To Meet: #12 Shawn Ratcliffe

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What I Wish I Knew Before My Son Was Diagnosed with Brain Cancer

I wish I had known how big a role you truly play in the medical care of your loved one. TRUST YOUR INSTINCTS! They are nearly always right and you know your family member much better than the doctors. Also, make friends with the nurses – they can be a huge advocate when you need a different opinion or help in understanding the “system” or even getting past some of the quagmire you encounter with all the different specialists insisting on their unique treatment plan.  Ask to see the “chart” (if they will let you) so that you can better understand just what the doctors might be thinking. If you don’t feel comfortable with the answers you are given, keep asking, don’t be brushed aside as if you are too paranoid (“just being a Mom”), don’t back down, and know that the doctors are doing their best but in the situation of brain tumors their best is still greatly lacking in knowledge and expertise.  Every patient is different, and YOU are the best one to help your loved one. From diagnosis to death is a rollercoaster ride on every possible level, take one day at a time and always look for the positives.

For us, it started out like any other Saturday morning.  Matt and I had breakfast, no one else was up yet. He was off to get his eyes checked as we were sure that was what was causing his headaches. Not long after he arrived at the Dr’s office he sent me a text “They want me to get an MRI”. I was stunned, and in my heart, I knew we were headed for something scary.  Within a few hours, we heard the words that would change our lives forever. “A very large mass on the right front lobe of your brain. It is cancer.” He was 20 years old.  By Sunday afternoon, 24 hours later, the tennis ball size tumor had been removed, or at least 90% of it. Matty bounced back like a true warrior, despite major pain and swelling, and on Thursday afternoon, he and I walked out of the hospital, confident that the worst was behind us.  Naïve, I know.

Within a few weeks, the swelling was so bad that they had to put in a VP shunt.  Within about a month, they removed the shunt as it had caused massive infection.  He was now experiencing Grand Mal seizures, loss of memory and functionality.  Shortly after that, he was fired from his job, and we added major anxiety and depression to our list of issues.    Basically, everything that could go wrong did go wrong.  He continued to focus on school, and within just over a year, he graduated top of his class at ITT Tech. It was truly a highlight of his life.   After 6 weeks of radiation, 11 months of chemo, and 5 stays in the hospital we finally heard the words “no evidence of tumor”, and it looked like we were finally getting a break!

With his confidence at an all-time high, he accepted a job in San Francisco, California, and we moved him out there.  I was, of course, devastated and proud all at the same time.  There were many struggles during the 18 months he was there.  He had just gotten his dream job working for Google when we realized the infection had never really gone away, and two more surgeries would be required.  The first was to remove the infected brain plate and start him on aggressive antibiotics.  Of course, during that time the cancer returned, and by the time they were able to replace the brain plate, the tumor had grown to the size of a fist.  It was stage 4 and angry.  The doctor was so aggressive in the recession of the tumor for the fifth and final surgery that Matty lost half of his eyesight in both eyes, the use of his entire left side, his short term memory, and some of his cognitive skills.  We managed to get him home to Valley Center, Kansas after an extensive stay in the hospital and quickly signed up for a clinical trial through Mayo Clinic.  But to no avail.

Just days after his 24th birthday, we were told there was nothing more they could do.  Matty wanted to go home and party with his family and friends and that is just what we did.  For the 3 weeks and 3 days he was with us, we opened our home to anyone, anytime.  Friends and family came from all over the country to spend time with him, reminisce about the good times and embrace the present, soaking in every moment with him. It was such a difficult time filled with tears, laughter, heartbreak, stress, and always love. Lots of love.

Matthew came into this world on a Sunday morning at 8:53am as a content, happy baby boy and left on a Sunday morning at 8:35am as a peaceful young man ready to let go of this life and seek out new adventures. It was a true privilege to be with my son for both. I cherish the memories that help me never forget, help me learn, help me survive.

During his entire fight my son displayed a kindness and gentleness that I could only watch, I felt none of that.  He always thanked everyone for their assistance and never lost his patience, despite severe pain at times.  He was constantly trying to help people feel comfortable with his cancer, with a ready quip or comeback to make them laugh.  Often times when a nurse finally arrived with pain meds to help with a horrible headache, he would purpose marriage and make them know he appreciated their efforts. I, on the other hand, was tenacious if not demanding when it came to his care and pain management.  His last effort to help others was to donate his body to science.  It was his hope that somehow he just might help find a cure so others would not have to endure what he did.

My wish is for everyone to enjoy today, whatever it brings, and make the most of it.   My son is gone in body, but he lives on in our hearts and our memories.  I am a better person having experienced this pain and heartache, no matter how awful it has seemed at times. It has softened the rough edges, dimmed the harsh blacks and whites and helped me focus more on the moment. I have finally come to a sort of peace with the fact that I’m alive and healthy, despite my desperate pleas that I be allowed to somehow take his place.  Matthew is my hero, and I just hope that I can make him as proud of me as I am of him!  Live, Laugh, Love

 

 

 

 

52 People You Need To Meet: #10 Karen Simonds

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There are many things that I wish I’d known before my brother, Jon, and my husband, Andy, were diagnosed with GBM brain cancer. First, because I think of this all the time, I really wish I’d learned to skip rocks from my brother. He tried to teach me so many times, and it was the last outdoor activity I did with him before he died. When he showed me, I could do it. On my own, not so much. My last truly happy memory of my brother was when we were on the Sacramento River with his kids and mine. We had a really good time even though he was having difficulty walking. We shared memories of our childhood and all the fun times we’d had on that river. I wish I could skip rocks.

The second thing I wish I knew before my husband was diagnosed was not to form opinions so quickly. I remember thinking when my brother was so sick that there were so many people around (hello – I was one of them!) and that I didn’t know them so therefore, who were they? Well, now that my husband is bedridden, we cherish the close friends, from all aspects of our lives, who stop by. It may be his work friends; it may be our daughters’ friends who grew up with us; it may be a school friend of mine, but all of them are equally important and none of them are “family.”

I wish I’d known to cherish, truly cherish, the little things. There was the coffee my husband made and delivered to me every day while I was still in bed. There were the hikes he took us on and the “secret adventures” he planned. There were camping trips and picnics. There were the gifts of a mountain bike, fly rod and kayak . All of which I adore and none of which I realized how very much I actually wanted. Now that he isn’t able to participate in these activities it breaks my heart that I didn’t stop and soak in every moment.

I wish I’d known that it is okay to accept help from friends. It’s taken me a long, long time to get this lesson. People want to help. I would want to help. Ask for help. It’s okay, and people are more than willing to give their time and talents to help a family in need. Some of the most incredible gifts have been things I would have never thought of. There was the shoveled driveway, the Christmas lights that came down, the money given for prom dresses just because the giver thought our girls deserved extra special dresses, the counseling sessions that were miraculously paid for, the case of wine that showed up, the cards, the phone calls from people from our past, friends that showed up on our front door, the school counselors helping the girls, all gifts from the heart. It’s been incredible and so very hard to accept with grace.

I’d wish I had known what a difficult job it is to research treatments and clinical trials and alternative nutritional information. I would have been reading these books a long time ago and changing my family’s diet (even though we eat quite well!) years ago. It’s important to stay on top of new treatments so that you are able to question your doctors and understand your options. I assumed that this was something I didn’t have to worry about because the doctors would do it (and in our case, they did) but that’s not always true.

I’ve always had a lot of respect for medical professionals, but now, they are my heroes. Really. From the first doctors and nurses we had here in Boise to those at MD Anderson and UC San Francisco, we have hit the jackpot. These people are amazingly kind, compassionate, and skilled. Oh, and underpaid. Coming from an elementary school teacher, that’s saying something! However, what I wish I had known and what I now tell others, is that when all else fails, go with your gut and ask lots of questions. It helps to bring someone along to take notes. We always, from the very first appointment, had someone to take notes. They know who they are and they are invaluable.

Another thing I wish I had known is how very much I despise the statement, “God only gives you what you can handle.” Really? Because I’m kind of at my limit and so are my parents, Andy’s mom, his brother, and our kids. Don’t say that. Ever. I know people mean well but that just might cause me to reach out and scratch some eyes out. I. Can’t. Handle. Any. More. The end.

I wish I had known that my brother and my husband were/are absolutely amazing human beings who never for one second deserved this and who both fought this so bravely. Of course, I always loved both of them, but I really wish I had realized what absolutely amazing people they were/are. Both of them put their family first and during the brain cancer years, always, always supported other people with the disease – even when they were barely hanging on themselves. Both of them cared more about others, and both of them said they wouldn’t have changed a thing. Crazy. I would have changed a lot of things if I could. I guess that’s why it’s not my time yet. I haven’t learned true compassion, and I haven’t learned to skip rocks.