Dear Friend:

We wanted to give you an update on the extremely important and successful work that the Institute for Myeloma & Bone Cancer Research (IMBCR) has conducted during the past six months as we continue our groundbreaking research into finding a cure for multiple myeloma and other bone cancers.

IMBCR remains the only independent non-profit cancer research institute working to find improved treatment, and ultimately a cure, for multiple myeloma, a cancer of blood cells that reside in the bone marrow. While the focus of the research is multiple myeloma, many therapies discovered in our research laboratory can apply to the treatment of other cancers, particularly cancers that spread to the bone such as prostate, breast and lung cancer.

Because of our research, we have continued to develop new treatment regimens that have benefitted patients with multiple myeloma and other blood cancers. To continue our research, we need additional funds and we now ask for your financial support.

IMBCR continues to be involved in very exciting work. We have identified new treatments for myeloma, identified innovative ways to follow the disease, and completed research that has led to a better understanding of the biology of myeloma.

Our research has focused on three different areas, including B-cell maturation antigen (BCMA), Jak inhibitors and targeted genetic approaches.

• Serum levels of this protein show it to
o be a useful blood marker to predict outcomes and monitor the disease status of not only patients with myeloma, macroglobulinemia and chronic lymphocytic leukemia but also for those with amyloidosis.
o predict which patients with either MGUS, pre-myeloma, or smoldering myeloma are at risk to develop active myeloma.
o be extremely low in patients with immune deficiencies and associated with disease related complications in these patients. This likely represents the first blood test to both diagnose and predict clinical problems in patients with primary immune deficiencies.
o predict poor outcomes for patients with breast cancer when it is low and expect that this is likely to be the case in many other types of cancer that we are now testing.
o cause the occurrence of the immune defects that are a hallmark of myeloma. We are now working on ways to prevent this from occurring so that myeloma patients can have functional immune systems which will allow them to fend off infections and fight their myeloma more effectively. o interfere with the targeting of BCMA on tumor cells by new immune based therapies. Our results with an inhibitor of the enzyme, gamma secretase, that leads to the shedding of BCMA show that we can prevent it from being released off the myeloma cells. This is likely to pave the way for a multitude of studies to use this approach to reverse this “blocking” effect of the circulating BCMA which should improve the effectiveness of BCMA targeted approaches and reverse these patients’ immune deficiency.

2) Jak inhibitors
• Jakafi (ruxolitinib)
o increases the effectiveness of a variety of other drugs that are currently used to treat myeloma including the combination of Revlimid and steroids. A clinical trial was conducted with this combination for myeloma patients. It has shown that patients doing poorly on Revlimid and steroids respond with the addition of Jakafi, and the regimen has been very well tolerated. This is a whole new approach to treating myeloma and is likely to be applicable to a variety of other types of cancer.
o has several mechanisms through which it can help overcome the resistance of myeloma patients to their treatment. First, it prevents myeloma growth through effects on a type of white cell. This cell, called a macrophage, which is a ‘scavenger’ cell, can exist in two forms — one which increases tumor growth and the other which slows the growth of the myeloma. We have shown that Jakafi markedly reduces the number of macrophages that make the tumor grow, while increasing the types of these cells that eliminate the myeloma. We have also uncovered the specific genes that control this effect.
o markedly reduces a protein called MUC-1 that is responsible for resistance to Revlimid which helps explain the specific ability of Jakafi to overcome resistance to Revlimid in myeloma patients as shown in our clinical trial.
• The levels of proteins called checkpoint inhibitors including PD-L1 and PD-L2 as well as B7-H3 are very high in myeloma patients’ bone marrow and interfere with their immune systems ability to get rid of myeloma. We have now shown that Jakafi reduces the levels of these checkpoint inhibitor proteins; and, thus, restores these patients’ immune function so that they are much more capable of eliminating their myeloma. This is potentially an exciting new treatment approach that may boost the effectiveness of many immune-based treatments including antibodies and T-cells.

3) Targeted genetic approaches
• We have been working on a new, exciting approach to treat myeloma that will only eliminate the tumor cells without having any side effects. This is a treatment that will target the genetic material that is only present in the tumor cells allowing tumor specific killing without any side effects.

We are all very excited about the new discoveries that we have made to make the lives of patients with myeloma better. The advancements during this year at IMBCR would not have happened without your generous support.

We count on our many donors and financial supporters to continue with their remarkable efforts in funding our research and we have enclosed both a Pledge Card and a reply envelope for your use.

If it is more convenient, you can make a secure online credit card donation by visiting our website: Please click on the “Donate Now” button. You can also make a credit card donation by telephoning our main office number: (310) 623-1210. In advance, we thank you for your donation and, if you have any questions or comments, please do not hesitate to contact either of us at the above telephone number.

With thanks and best wishes for 2019,

James R. Berenson, M.D
President, Medical and Scientific Director

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