The Multiple Myeloma Research Foundation (MMRF) is a charitable organization dedicated to multiple myeloma, an incurable blood cancer. The MMRF runs as if it were a for-profit business, expecting high returns from the money the organization raises from donors. [1]
MMRF was founded in 1998 by twin sisters Kathy Giusti and Karen Andrews, following Kathy's diagnosis with multiple myeloma. Giusti, a pharmaceutical company executive and Harvard Business School Alum, wanted to encourage researchers to develop treatments for multiple myeloma by using business models rather than academic models of drug development. [1]
MMRF is a private funder of multiple myeloma research, having raised over $120 million since its inception to contribute funding to more than 120 laboratories worldwide. [2] MMRF funding contributes to diverse research strategies to yield long-, mid-, and short-term results in an effort to deliver better treatments to patients faster: basic science programs to better understand the disease and identify new druggable targets through genomics and proteomics research; validation programs to prioritize new compounds and combinations based on key targets; and clinical trials conducted at a number of myeloma centers.[ citation needed ]
In 2009, the MMRF funded research into 30 compounds at the pre-clinical stage. [3] By 2013 it had raised more than $250 million and its work has helped gain approval of six new drugs to treat the disease. [4]
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Thalidomide, sold under the brand names Contergan and Thalomid among others, is an oral medication used to treat a number of cancers, graft-versus-host disease, and many skin disorders. While thalidomide has been used in a number of HIV-associated conditions, such use is associated with increased levels of the virus.
Multiple myeloma (MM), also known as plasma cell myeloma and simply myeloma, is a cancer of plasma cells, a type of white blood cell that normally produces antibodies. Often, no symptoms are noticed initially. As it progresses, bone pain, anemia, kidney dysfunction, and infections may occur. Complications may include hypercalcemia and amyloidosis.
An orphan drug is a pharmaceutical agent that is developed to treat certain rare medical conditions. An orphan drug would not be profitable to produce without government assistance, due to the small population of patients affected by the conditions. The conditions that orphan drugs are used to treat are referred to as orphan diseases. The assignment of orphan status to a disease and to drugs developed to treat it is a matter of public policy that depends on the legislation of the country.
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Kathy Giusti is a business leader and a healthcare disrupter. She is a two-time cancer survivor having been diagnosed with multiple myeloma and breast cancer. Kathy Co-Founded the Multiple Myeloma Research Foundation where she served as CEO and president for nearly two decades. She also co-chaired the Harvard Business School (HBS) Kraft Precision Medicine Accelerator, which she helped found, as a Senior Fellow at Harvard Business School.
The mission of the Multiple Myeloma Research Consortium (MMRC) is to champion collaboration with and integration across academia and industry and to focus on speed and innovation to bring the most promising multiple myeloma treatments to patients faster. It was founded by Kathy Giusti, a myeloma patient and founder and chief executive officer of the Multiple Myeloma Research Foundation (MMRF).
Tipifarnib is a farnesyltransferase inhibitor. Farnesyltransferase inhibitors block the activity of the farnesyltransferase enzyme by inhibiting prenylation of the CAAX tail motif, which ultimately prevents Ras from binding to the membrane, rendering it inactive.
Rodger O. Riney is an American billionaire and the founder of Scottrade, a stockbrokerage that was acquired by TD Ameritrade in 2017.
The ALS Therapy Development Institute is a non-profit biotechnology research organization focused on finding treatments for amyotrophic lateral sclerosis (ALS). With a staff including more than 30 scientists, it operates a research and development program centered on ALS.
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Genta Incorporated was a biopharmaceutical company started in La Jolla, California, which discovered and developed innovative drugs for the treatment of patients with cancer. Founded in 1989 by a highly skilled entrepreneur, the company focused on a novel technology known as antisense, which targets gene products that are associated with the onset and progression of serious diseases. At that time, only Ionis Pharmaceuticals, Inc. was conducting significant research with this technology. Antisense is a short span of oligonucleotides – modified DNA structures ranging from about 12-24 bases that selectively bind to specific RNA. The intent is to block expression of an aberrant protein that contributes to the disease of interest. Genta in-licensed three different antisense molecules that blocked Bcl-2, a fibroblast growth factor (FGF), and the gene c-myb, respectively.
Daratumumab, sold under the brand name Darzalex, is an anti-cancer monoclonal antibody medication. It binds to CD38, which is overexpressed in multiple myeloma cells. Daratumumab was originally developed by Genmab, but it is now being jointly developed by Genmab along with the Johnson & Johnson subsidiary Janssen Biotech, which acquired worldwide commercialization rights to the drug from Genmab.
A MEK inhibitor is a chemical or drug that inhibits the mitogen-activated protein kinase kinase enzymes MEK1 and/or MEK2. They can be used to affect the MAPK/ERK pathway which is often overactive in some cancers.
Cereblon E3 ligase modulators, also known as immunomodulatory imide drugs (IMiDs), are a class of immunomodulatory drugs containing an imide group. The IMiD class includes thalidomide and its analogues. These drugs may also be referred to as 'Cereblon modulators'. Cereblon (CRBN) is the protein targeted by this class of drugs.
Melphalan flufenamide, sold under the brand names Pepaxto and Pepaxti, is an anticancer medication used to treat multiple myeloma.
An osteolytic lesion is a softened section of a patient's bone formed as a symptom of specific diseases, including breast cancer and multiple myeloma. This softened area appears as a hole on X-ray scans due to decreased bone density, although many other diseases are associated with this symptom. Osteolytic lesions can cause pain, increased risk of bone fracture, and spinal cord compression. These lesions can be treated using biophosphonates or radiation, though new solutions are being tested in clinical trials.
Verseon International Corporation is a clinical-stage pharmaceutical company based in Fremont, California. It is developing several drug discovery programs in cardiometabolic diseases and cancer featuring drug candidates that represent novel chemical matter designed using molecular physics and artificial intelligence.
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Irene Ghobrial is an American-Egyptian physician who is a professor at the Dana–Farber Cancer Institute and Harvard Medical School, where her research investigates the progression of multiple myeloma. She is interested in why certain patients with monoclonal gammopathy of undetermined significance (MGUS) and smoldering multiple myeloma develop B cell malignancies. She leads the Stand Up To Cancer multiple myeloma dream team.
