While new phones, tablets and other electronic devices are all the rage this year, at the end of the day, these gifts do not necessarily represent the true spirit of the season. Material possessions provide entertainment and might give us a sense of status, but the true meaning of the holidays seems to be lost. I feel this year should be different. This year we should take time to reflect on what is important and what we can each do to make our world better.
I’ve said it before and I will continue to declare that the only people who are going to change the outcome for CLL patients are CLL patients. So this year include yourself on your gift list. It is okay to be selfish when your heart is in the right place. And tell your friends and family that instead of receiving a fruit basket or bottle of wine, a gift to CLL Global Research Foundation would have a positive impact on your life.
From my point of view, CLL Global is a gift from patients to CLL researchers and me in particular. Their generosity has enabled us to move the ball forward so that we are now in a position to discuss the potential cure of CLL. I am tired of having my optimism challenged by CLL, and I am ready to reciprocate your gifts by discovering the puzzle piece that will eliminate any suffering from this disease. Two major discoveries have led to realistic optimism that the cure is near.
A series of compounds have been developed which dramatically reduce the amount of CLL in the body. These compounds inhibit the B-cell receptor signaling pathway, a very important aspect of CLL cell survival, by selectively blocking enzymes that are important in the production and survival of CLL cells. These inhibitors have been discussed in recent copies of the Research Momentum newsletter. Unlike current standard treatments for CLL, these inhibitors are targeted and therefore do not damage the immune system. The B-cell receptor inhibitors CAL-101 and PCI-32765 are the most advanced in development and both are being tested in clinical trials. These agents are able to render normal blood counts, a small amount of leukemia and may serve as a primer for immunotherapy.
The other hot topic these days is chimeric antigen receptors (CARs). CARs are receptors that are genetically attached to T-cells, which are a type of immune cell. They train the T-cells to seek out a specific molecule and destroy the cell to which the molecule is attached. Investigators from the University of Pennsylvania, Drs. Carl June and Bruce Levine, have led the pack with a clinical trial using (CARs). Three patients participated in the UPenn trial and had a dramatic reduction in the amount of CLL in their bodies. In two out of the three cases no CLL could be identified.
The UPenn CAR was successful, but as with any new invention, the prototype usually has some kinks. The target molecule for the UPenn CAR was CD19 which is also present on normal B-cells. This causes the normal B-cells and the immune system to be severely damaged in already immune-compromised patients. Also, the technology used at UPenn may prove to be cost prohibitive.
For the last few months, I have been meeting frequently with researchers who just might have the answers to jump over the hurdles of the CAR prototype. Some years ago, Drs. Thomas Kipps (University of California, San Diego) and Bill Wierda (MD Anderson) discovered that the ROR1 protein is almost exclusively expressed on CLL cells and not on normal cells. Therefore, a ROR1 CAR should be effective in eliminating CLL cells while leaving normal immune cells alone. By using a technology developed by members of Dr. Laurence Cooper’s laboratory team at MD Anderson Cancer Center, the cost of manufacturing the ROR1 CAR can be significantly reduced. While there is still a substantial amount of work to be done, it is hoped that clinical trials will be mounted in 2012.
Clinical research is very costly. However, the reward is great. Current funding from major agencies such as the National Institutes of Health (NIH) is insufficient to move the research quickly. This is why philanthropic support is essential. Many of you, your families and friends have gifted the CLL Global Research Foundation. All past gifts have been appreciated and have led to great rewards. The majority of donations continue to be from repeat donors who have supported the cause for many years.
I now have the temerity to ask for those that can do so to increase their gifting. We need to establish a fund of $1 million per year for the next three years to support the development of CARs. We also need to continue funding the quality research we have been supporting from day one. Researchers are continuing to make beneficial discoveries in the area of genetics which helps us better understand the biology of CLL. The new B-cell receptor inhibitors are already having a dramatic effect for patients, and we have only conducted phase I trials. We have a lot we want to put on our plate in 2012, but we need you to be involved in order to accomplish our goals.
Whatever you can do to support this endeavor will be sincerely appreciated. Share the true meaning of the season with others. Spread the word about our mission. Do it with passion. Your family wants you to be cured, you want other generations of CLL patients to be cured, and I want to hold true to my word. Whatever you can give, however you can contribute, will be a gift to the world that will indeed also be a gift to yourself. A cure for CLL will be the best gift of all.
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