With nearly everything we do in life whether it is in business, sports, or even (if you are like me) everyday activities such as grocery shopping, we have to have strategies. (Yes, I really do have a strategy shopping for my milk and eggs at the grocery store but we don’t need to get into that). A better example is, if you are a coach of a football team, you don’t send your players out on the field without a strategy or you risk the chance losing the game; or worse someone will get hurt! Additionally, you wouldn’t use the same exact play every time you send your team out onto the field. That being said, Doctor Maryam Fouladi, a leading pediatric brain cancer oncologist at Cincinnati Children’s Hospital, has created a new and very effective strategy to add to our playbook of “The Homerun Cure”(Okay, maybe I should’ve used a baseball analogy instead of football, but I’m pretty sure you know what I mean). Fouladi’s project is called CONNECT (Collaborative Network of Neuro-Oncology Clinical Trials) Collaborative. It is essentially a network of neuro-oncology clinical trials performed at international sites by the world’s leaders in childhood brain cancer research and it was built on the network that was created by the DIPG Registry. You might even look at it as a virtual hospital for pediatric brain cancers.
I had the opportunity to meet with Dr. Fouladi and her colleague, Dr. Drissi, to discuss this exciting new collaborative as well as an upcoming trial. Fouladi very openly explained that the main goal of CONNECT Collaborative is to provide as many places for children with DIPG and other high grade gliomas to go for treatment. She reached out to 16 different institutions, who have all been partners in the past and know that they work very well together as they have the same mission. And each of them was enthusiastic and more than willing to come on board to find better and MORE treatment options, which will eventually lead to that homerun cure we so desperately need. The list of institutions participating is as follows: Baylor College of Medicine (USA), Children’s Nat’l Med Center (USA), Dana Farber Cancer Center (USA), Seattle Children’s(USA), Lurie Children’s (USA), Children’s Hospital of Colorado (USA), Great Ormond Street Hospital (UK),German Cancer Research Center (Germany), VU University Med Center (Netherlands), Hospital for Sick Children (Canada), Montreal Children’s (Canada), Lady Cilento Children’s ( Australia), Princess Margaret Hospital for Children ( Australia), Sydney Children’s (Australia), and McMaster Children’s (Canada), with Cincinnati Children’s (USA) as the lead operations center. It is important to point out that ALL of the sites involved are equal partners and can open all studies in the CONNECT Collaborative and can contribute new ideas for these trials. The doctors from each of these chosen institutions carry so much clout from the work they have done, that it has been significantly easier to get drug companies on board, thus far. That alone is a HUGE feat!
Currently there are clinical trials for patients with recurrent tumors and much larger trials through bigger consortia for newly-diagnosed patients as well as recurring patients. However, there is a huge gap in the middle where smaller feasibility studies NEED to be conducted in newly-diagnosed patients to see if the new treatment regimen being considered for much larger trials is tolerable and safe in order to push forward to larger studies. This will give families more hope and encouragement as now they will have more studies available to take part in. The CONNECT Collaborative bridges that gap between the newly-diagnosed and recurring patients.
With this new strategy, we will have better geographical options for families with kids who are in the fight. One of the goals is to make it so a family doesn’t have to pick up and temporarily move to a different country in order to have their child participate in a study. This alone eliminates a large portion of stress and fear these families face. Let alone the financial burden of traveling out of their own countries.
We are basically waging a war against DIPG and other high grade gliomas. And it is a World War. We need allies in other countries to come together, learn about the enemy (brain bancer) and defeat it! So by creating this connection between some of the world’s leading pediatric brain cancer researchers, we can effectively and efficiently speed up the process of achieving more accurate results! This also eliminates the possibility of repeating trials which is a waste of time and money. Additionally, advances in the trials will be made much faster! As we all know, time is of the essence with many of these brain cancers.
After speaking with Dr. Fouladi about what exactly the “CONNECT Collaborative” is, I learned the exciting news that Dr. Dewire and Dr. Drissi are beginning the first two studies for the CONNECT Collaborative! Dr. Dewire’s RAD001 and LEE011 study (previously funded by The Cure Starts Now) being the first and is currently taking enrollment. Dr. Drissi’s BMI-1 inhibitor study will be the second and is expected to begin hopefully in January or February. The protocol has been written, the drug company is on board and soon the doctors will send the proposal to the FDA for approval of this study.
What is BMI-1? BMI-1 is a protein found in cells of a normal brain. However, this particular protein is highly expressed in DIPG. Meaning there is significantly more BMI-1 proteins in the tumor cells than found in normal brain cells. While normal cells NEED controlled levels of BMI-1, in DIPG the levels are 2-26-folds higher. Anytime there is something abnormally high in a tumor that means the tumor probably NEEDS whatever it is to grow and survive.
When Dr. Drisssi looked back at published studies performed on tumor tissues, he noticed BMI-1 was highly expressed in all DIPG tumors. This was a very exciting find because this could mean that the protein could be a therapeutic target for the researchers to decrease which would then slow the tumor growth.
So once he knew BMI-1 is highly expressed in DIPG (and other cancers for that matter), his next step was to test living DIPG tumor cells that parents, like me and so many others have donated through autopsy. Drissi wanted to find out what drug could affect or inhibit the growth of BMI-1 levels. And guess what?! He found one! Even better, it is now in clinical trials for adults! However, it is not yet commercially available. Meaning it hadn’t been tested enough to assess its efficacy nor has it had safety studies in pediatrics. Therefore, he could only use it in the lab. When Dr. Drissi did use this inhibitor on living DIPG tumor cells he was incredibly surprised to learn that it did in fact kill a large portion of these tumor cells. DIPG has 3 sub-types and the BMI-1 inhibitor works on ALL of them!
Could this mean that BMI-1 is the mother ship or the driver of DIPG cancer cells and many other cancer cells? Now that we know BMI-1 protein plays such a huge role in these tumors and we found an inhibitor that can actually kill many of these cells, doctors need to figure out a safe way to use it to treat children. Amazingly enough, Dr. Drissi found another study currently taking place on adults. Remember, that BMI-1 is found in other cancers, not just DIPG. From the results of the adult studies we now know how to give the drug and what doses are safe, at least in adults. All very exciting! Even MORE exciting, the scientists who are conducting the adult studies were/are extremely willing to cooperate with Dr. Drissi as they understand the importance of advancing pediatric cancer treatments by making this inhibitor available for children.
One of the goals of Dr. Drissi’s new study is to lower levels of BMI-1, not completely get rid of it. As mentioned before, the normal brain needs some levels of this protein. So with this new inhibitor it will reduce the BMI-1 levels, making DIPG cells incredibly sensitive to therapies. We know that radiation shrinks DIPG tumors and we also know that the BMI-1 inhibitor makes DIPG cells more sensitive to radiation. Dr. Drissi’s study is to incorporate both radiation and the BMI-1 inhibitor making radiation more effective which may then sustain the lives of these precious children. This inhibitor also allows for potential reduction in radiation therapy doses. Let’s face it; we all have a love/hate relationship with radiation. And if the radiation dose doesn’t always get reduced it will at least be more effective than it is today.
Now a good question is: How do you safely deliver this drug? Most of us know getting past the Blood Bain Barrier has been a pretty difficult and frustrating issue. Well, I’m excited to share with you that there is some data showing that this particular inhibitor actually does cross that barrier! However, the proof will be in the upcoming study.
Sustaining a child’s life, but also allowing them to maintain quality of life is one of the major goals of this study and other studies in the CONNECT Collaborative. Most of us know that the average survival rate of kids with DIPG is only about 8-9 months. If we can push that for ALL or even MOST of these children to be over a year or a year and a half, then that alone is success. However, the ultimate goal through the CONNECT Collaborative using studies like Dr. Drissi’s upcoming BMI-1 inhibitor trial is to cure this disease. Until now, researchers have been making incremental steps and that just isn’t enough anymore.
And because of all of the hard work and generosity from the families who pushed for autopsies so they could donate their child’s tumor for research, The Cure Starts Now and chapters, foundations who are a part of the DIPG Collaborative, as well as every single advocate, business sponsorship, fundraising partner, and donor; in the last five years researchers have learned more about DIPG than they had in the fifty years prior! With all of the funding from all of the parties mentioned above, along with new forward thinking strategies like CONNECT Collaborative; I personally believe without a shadow of a doubt that a cure for DIPG and other high grade gliomas will happen much sooner than later, which will then finally lead us to the HOMERUN CURE FOR ALL CANCER! And when that day comes I will personally fund one last Carnival for the Cure (a fundraising event I do every June in Columbus, Indiana for childhood brain cancer research) which will require ZERO sponsors, ZERO dollars and will be FREE for all. And on that day we will join together in celebration of all of the beautiful children who have gone before us so that others will get the chance to live. That day will be the absolute BEST DAY EVER!
Currently there are clinical trials for patients with recurrent tumors and much larger trials through bigger consortia for newly-diagnosed patients as well as recurring patients. However, there is a huge gap in the middle where smaller feasibility studies NEED to be conducted in newly-diagnosed patients to see if the new treatment regimen being considered for much larger trials is tolerable and safe in order to push forward to larger studies. This will give families more hope and encouragement as now they will have more studies available to take part in. The CONNECT Collaborative bridges that gap between the newly-diagnosed and recurring patients.
With this new strategy, we will have better geographical options for families with kids who are in the fight. One of the goals is to make it so a family doesn’t have to pick up and temporarily move to a different country in order to have their child participate in a study. This alone eliminates a large portion of stress and fear these families face. Let alone the financial burden of traveling out of their own countries.
We are basically waging a war against DIPG and other high grade gliomas. And it is a World War. We need allies in other countries to come together, learn about the enemy (brain bancer) and defeat it! So by creating this connection between some of the world’s leading pediatric brain cancer researchers, we can effectively and efficiently speed up the process of achieving more accurate results! This also eliminates the possibility of repeating trials which is a waste of time and money. Additionally, advances in the trials will be made much faster! As we all know, time is of the essence with many of these brain cancers.
After speaking with Dr. Fouladi about what exactly the “CONNECT Collaborative” is, I learned the exciting news that Dr. Dewire and Dr. Drissi are beginning the first two studies for the CONNECT Collaborative! Dr. Dewire’s RAD001 and LEE011 study (previously funded by The Cure Starts Now) being the first and is currently taking enrollment. Dr. Drissi’s BMI-1 inhibitor study will be the second and is expected to begin hopefully in January or February. The protocol has been written, the drug company is on board and soon the doctors will send the proposal to the FDA for approval of this study.
What is BMI-1? BMI-1 is a protein found in cells of a normal brain. However, this particular protein is highly expressed in DIPG. Meaning there is significantly more BMI-1 proteins in the tumor cells than found in normal brain cells. While normal cells NEED controlled levels of BMI-1, in DIPG the levels are 2-26-folds higher. Anytime there is something abnormally high in a tumor that means the tumor probably NEEDS whatever it is to grow and survive.
When Dr. Drisssi looked back at published studies performed on tumor tissues, he noticed BMI-1 was highly expressed in all DIPG tumors. This was a very exciting find because this could mean that the protein could be a therapeutic target for the researchers to decrease which would then slow the tumor growth.
So once he knew BMI-1 is highly expressed in DIPG (and other cancers for that matter), his next step was to test living DIPG tumor cells that parents, like me and so many others have donated through autopsy. Drissi wanted to find out what drug could affect or inhibit the growth of BMI-1 levels. And guess what?! He found one! Even better, it is now in clinical trials for adults! However, it is not yet commercially available. Meaning it hadn’t been tested enough to assess its efficacy nor has it had safety studies in pediatrics. Therefore, he could only use it in the lab. When Dr. Drissi did use this inhibitor on living DIPG tumor cells he was incredibly surprised to learn that it did in fact kill a large portion of these tumor cells. DIPG has 3 sub-types and the BMI-1 inhibitor works on ALL of them!
Could this mean that BMI-1 is the mother ship or the driver of DIPG cancer cells and many other cancer cells? Now that we know BMI-1 protein plays such a huge role in these tumors and we found an inhibitor that can actually kill many of these cells, doctors need to figure out a safe way to use it to treat children. Amazingly enough, Dr. Drissi found another study currently taking place on adults. Remember, that BMI-1 is found in other cancers, not just DIPG. From the results of the adult studies we now know how to give the drug and what doses are safe, at least in adults. All very exciting! Even MORE exciting, the scientists who are conducting the adult studies were/are extremely willing to cooperate with Dr. Drissi as they understand the importance of advancing pediatric cancer treatments by making this inhibitor available for children.
One of the goals of Dr. Drissi’s new study is to lower levels of BMI-1, not completely get rid of it. As mentioned before, the normal brain needs some levels of this protein. So with this new inhibitor it will reduce the BMI-1 levels, making DIPG cells incredibly sensitive to therapies. We know that radiation shrinks DIPG tumors and we also know that the BMI-1 inhibitor makes DIPG cells more sensitive to radiation. Dr. Drissi’s study is to incorporate both radiation and the BMI-1 inhibitor making radiation more effective which may then sustain the lives of these precious children. This inhibitor also allows for potential reduction in radiation therapy doses. Let’s face it; we all have a love/hate relationship with radiation. And if the radiation dose doesn’t always get reduced it will at least be more effective than it is today.
Now a good question is: How do you safely deliver this drug? Most of us know getting past the Blood Bain Barrier has been a pretty difficult and frustrating issue. Well, I’m excited to share with you that there is some data showing that this particular inhibitor actually does cross that barrier! However, the proof will be in the upcoming study.
Sustaining a child’s life, but also allowing them to maintain quality of life is one of the major goals of this study and other studies in the CONNECT Collaborative. Most of us know that the average survival rate of kids with DIPG is only about 8-9 months. If we can push that for ALL or even MOST of these children to be over a year or a year and a half, then that alone is success. However, the ultimate goal through the CONNECT Collaborative using studies like Dr. Drissi’s upcoming BMI-1 inhibitor trial is to cure this disease. Until now, researchers have been making incremental steps and that just isn’t enough anymore.
And because of all of the hard work and generosity from the families who pushed for autopsies so they could donate their child’s tumor for research, The Cure Starts Now and chapters, foundations who are a part of the DIPG Collaborative, as well as every single advocate, business sponsorship, fundraising partner, and donor; in the last five years researchers have learned more about DIPG than they had in the fifty years prior! With all of the funding from all of the parties mentioned above, along with new forward thinking strategies like CONNECT Collaborative; I personally believe without a shadow of a doubt that a cure for DIPG and other high grade gliomas will happen much sooner than later, which will then finally lead us to the HOMERUN CURE FOR ALL CANCER! And when that day comes I will personally fund one last Carnival for the Cure (a fundraising event I do every June in Columbus, Indiana for childhood brain cancer research) which will require ZERO sponsors, ZERO dollars and will be FREE for all. And on that day we will join together in celebration of all of the beautiful children who have gone before us so that others will get the chance to live. That day will be the absolute BEST DAY EVER!
