Leukemia & Lymphoma Society Inc

A) research programs we will continue to support research through our innovative and integrated funding programs, until every patient has a safe and effective therapy. in fiscal year 2020, lls supported research in the u.S., canada and 8 other countries with a total research disbursement of approximately 26.8 million. research funding was distributed across all blood cancers. beat aml master trial beginning november 2016, lls launched the beat aml master trial, a collaborative clinical trial testing several novel targeted therapies for patients with acute myeloid leukemia (aml) designed to facilitate fda approval of new drugs and change the treatment paradigm for patients diagnosed with aml by developing more individualized, effective treatment approaches. the master trial involves collaborations with multiple medical institutions, drug companies, a genomic provider, a clinical research organization, and the fda, all of whom have committed to working collaboratively. at the end of 2020, the beat aml trial had enrolled 1,000 patients. our critical role lls programs accelerate relevant research outcomes by: - building a focused research work-force: assuring the next round of breakthroughs requires that young investigators be encouraged to work in blood cancer research fields. - turning discoveries into new therapies: fundamental new findings can be translated into safe and effective treatments that can ultimately prolong and enhance patient lives. - supporting synergy: large grants and contracts enable scientists in academia and the private-sector to collaborate, combining resources and expertise to produce more and faster advances. - filling a void: research projects that are high-risk and/or address rare cancers are less likely to be funded by government agencies or for-profit companies, but may provide important advances. - speeding new treatments to patients: partnering with biotechnology and pharmaceutical companies can advance promising therapies through clinical testing, faster. past advances made with lls research funding generous donors have helped lls support research that has already benefited blood cancer patients and many others. advances include: - multi-drug therapies that are more effective than treatments with single anti-cancer agents, - bone marrow / stem cell transplantation and supportive care treatments for patients who relapse despite the best available therapy, and, - tests that distinguish specific characteristics of particular blood cancers for accurate diagnosis of cancer subtypes, and for "risk stratification" to select an optimal therapy. targeted therapy research discovering the molecular abnormalities that cause particular types of blood cancer has been useful in diagnosis and risk stratification, and in new "targeted drug" development. lls-funded investigators have helped advance molecularly targeted treatments that can selectively kill blood cancer cells versus normal cells. many of these new treatments benefit not only blood cancer patients, but also patients with other diseases. for example: - gleevec is fda-approved for patients of all ages with chronic myeloid leukemia (cml), and is also approved for patients with one form of acute lymphoid leukemia (all), myelodysplastic syndromes (mds), myeloproliferative disorders and rare forms of stomach and skin cancers. related drugs, sprycel and tasigna, are approved for patients who do not benefit from gleevec. one or more of these drugs are also showing promise for patients with various lymphomas, acute myeloid leukemia (aml), chronic lymphocytic leukemia (cll), and other cancers, including brain, breast, head-and-neck, lung, pancreatic, and prostate cancers, and patients with other diseases including alzheimer's, asthma and pulmonary hypertension. - rituxan was the first fda-approved, anti-cancer antibody drug, developed for patients with forms of b-cell non-hodgkin lymphoma (nhl). it is now also approved for cll patients and as a "maintenance" therapy for follicular lymphoma patients, and showing promise for patients with all and after stem cell transplantation. in addition, it is approved for treating patients with severe rheumatoid arthritis and two other types of autoimmune diseases. a related antibody drug, arzerra, is approved for cll patients and showing wider promise. - velcade, thalidomid and revlimid are fda-approved for patients with myeloma and are also helping some patients with hodgkin lymphoma and nhl. krypolis was recently approved for myeloma patients for whom at least two prior therapies were insufficient. one or more of these drugs are now being tested for patients with t-cell and b-cell forms of lymphoma, acute leukemias, as well as aids-related kaposi sarcoma and brain, breast, colorectal, head-and-neck, kidney, liver, lung, ovarian and prostate cancers, and alzheimer's disease. - istodax, zolinza, dacogen and vidaza target small chemical, "epigenetic" changes. the first two drugs are approved for patients with peripheral t-cell lymphomas; the latter drugs are approved for mds patients. one or more of these drugs are being tested for patients with all, aml, cml, cll, myeloma and forms of nhl, after stem cell transplantation, and for patients with breast, brain, kidney, colorectal, head-and-neck, lung, stomach, prostate and ovarian cancers, melanoma as well as sickle cell disease and persistent hiv infections. - adcetris was approved in 2011, and in january 2012. it is an antibody- drug conjugate that combines an anti-cd30 antibody and the cytotoxic drug monomethyl auristatin e (mmae). it is an anti-neoplastic agent used in the treatment of hodgkin lymphoma after failure of autologous stem cell transplant or those who are not eligible for asct after failure of at least 2 mutagen chemotherapy regimens. adcetris was also approved for systemic anaplastic large cell lymphoma with failure of at least one prior treatment. - gazyva is a humanized monoclonal antibody used as a combination treatment with chlorambucil to treat patients with untreated chronic lymphocytic leukemia. it was approved by the fda in november 2013 and by the eha in july 2014. - imbruvica is an oral small molecule inhibitor against btk kinase. it was first approved by the us fda on november 13, 2013 for the treatment of mantle cell lymphoma patients who have received at least one prior treatment. on feb. 12, 2014 the us fda expanded the approved use of the drug to chronic lymphocytic leukemia (cll) patients who have received at least one prior treatment. additionally, it received further expansion to treat 17p deletion in cll with or without prior therapy. - zydelig is an oral small molecule inhibitor that blocks the delta isoform of the enzyme phosphoinositide 3-kinase. it was approved by the fda in july 2014 to treat relapsed/refractory cll in combination with rituxan. it was also approved to use as a monotherapy for relapsed mantle cell lymphoma and follicular lymphoma. other active research directions lls-funded researchers are also exploring other areas of research that hold promise for patients: - novel stem cell transplantation procedures: these include so-called "mini" transplants that use less toxic pre-transplant treatments and engineered donor cells that help reduce post-transplant complications, making these potentially curative treatments available to more patients. - immunotherapies: including antibodies, vaccines and engineered immune cells, these targeted therapies help a patient's immune system fight infections and kill residual cancer cells, prolonging remissions, and perhaps one day replacing toxic chemotherapies. - diagnostics: new technologies make it possible to characterize the abnormalities in individual cancer cases in molecular detail. this information can be used to help choose the best possible treatment for each patient, especially as more targeted therapies become available. - quality of life research: these studies increase our understanding of how specific treatments can cause debilitating side-effects, including late- effects, and which patients are at risk for developing these complications, so that they can be better managed or even prevented. driving research to address unmet medical needs lls continues to solicit and support research focused on improving blood cancer patients' quality of life after today's curative therapies. also in 2018, for the seventh year, lls actively recruited research proposals in six other underdeveloped research areas in which progress is likely to improve outcomes for patients with particularly urgent needs. new research is focused on: - development of novel therapeutic strategies for patients with non- cutaneous t-cell lymphoproliferative disorders - develop novel targeted therapies for cll patients, with real curative potential - develop novel treatment strategies for mds and aml patients - develop novel targeted therapies for patients with high-risk myeloma - development of new-targeted therapies fo