In November 2020, the FDA granted accelerated approval to DANYELZA (naxitamab-gqgk), a humanized, monoclonal antibody that targets the ganglioside GD2. DANYELZA was approved for use, in combination with granulocyte-macrophage colony-stimulating factor (“GM-CSF”), in the treatment of pediatric patients one year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy.

Development of this drug began over 30 years ago in the laboratory of MSK physician-scientist Nai-Kong Cheung. Together with the MSK Kids Neuroblastoma team, support from clinical researchers, as well as funding from philanthropic groups such as the Band of Parents, Dr. Cheung worked tirelessly to develop this treatment. The Office of Technology Development worked alongside Dr.Cheung for over a decade to find a suitable commercial development partner to make this antibody available to children in need beyond MSK. The project also received support from MSK’s Technology Development Fund, which provides critical funding for projects with strong commercial potential.

In 2015, the Office of Technology Development was able to execute a license with Y-mAbs Therapeutics, paving the way for DANYELZA’s FDA approval. Thomas Gad, the founder, Chairman and President of YmAbs, along with Claus Møller, Chief Executive Officer of Y-mAbs, built a commercial team around the product, and successfully advanced it to market approval.

The drug apalutamide, or Erleada™, is used to treat people with prostate cancer that has not spread to other parts of the body and no longer responds to a medical or surgical treatment that lowers testosterone. It has been shown to delay the spread and progression of castration-resistant prostate cancer. Apalutamide is marketed as Erleada™ by the Janssen Pharmaceutical Companies of Johnson & Johnson.  

Erleada™ is a next-generation androgen receptor (AR) inhibitor that binds directly to the ligand-binding domain of the AR. It works by inhibiting AR nuclear translocation and DNA binding and impeding AR-mediated transcription. Erleada™ was approved by the US Food and Drug Administration in February 2018 and is the first FDA-approved therapy to treat patients with nonmetastatic castration-resistant prostate cancer.

The targeted therapy Folotyn™, also known as pralatrexate, is the first drug ever to be approved for the treatment of peripheral T cell lymphoma (PTCL), a group of rare but highly aggressive blood cancers that often become resistant to standard treatments. 

In the United States, the disease accounts for approximately 10 to 15 percent of all newly diagnosed cases of non-Hodgkin lymphoma, or about 5,600 people per year.

Investigators at Memorial Sloan Kettering discovered that tumor cells are capable of taking in natural folate as well as methotrexate, a chemotherapy drug similar to folic acid that has been used for decades to treat cancer. They also found that the uptake mechanism relies on the function of a protein called plasma membrane transporter, or RFC-1, which cancer cells often produce in high amounts. Research has found that in noncancerous cells, RFC-1 is less abundant and does not mediate the same transport function.

Building upon this discovery, the Memorial Sloan Kettering team collaborated with researchers at SRI International and Southern Research Institute to develop a targeted therapy that exploits the tumor cells’ dependence on RFC-1 for folate intake. Their work resulted in the development of pralatrexate, which was studied in patients for the first time at Memorial Sloan Kettering.

After encouraging trial results, Memorial Sloan Kettering’s Office of Technology Development licensed the drug to Allos Therapeutics, Inc., for further clinical testing.

In September 2009, Folotyn™ was approved for the treatment of PTCL by the FDA under accelerated approval, which allows for earlier approval of drugs that address unmet medical needs.

Filgrastim – sold under a number of brand names and most commonly known as Neupogen^® – is a drug that stimulates the body’s production of white blood cells called neutrophils. It has been used in more than one million people around the world – mainly to treat neutrophil depletion caused by treatment with chemotherapy.

The use of chemotherapy has been vital in saving and extending the lives of cancer patients. However, in addition to killing cancer cells, many chemotherapy drugs destroy neutrophils and other white blood cells, leaving patients susceptible to infection and sometimes unable to undergo additional chemotherapy treatments.

In the 1970s, Memorial Sloan Kettering researchers isolated a naturally occurring protein called G-CSF, or granulocyte colony stimulating factor, and showed that it can stimulate the bone marrow to produce more neutrophils. In subsequent studies, the Memorial Sloan Kettering team demonstrated that filgrastim – an analog of G-CSF – could be used safely in patients to speed up the body’s renewal of neutrophils after chemotherapy. Filgrastim treatment was shown to reduce the frequency and severity of infections in these patients, and to help them recover faster after chemotherapy.

Since 1991, Filgrastim has been marketed by Amgen as Neupogen^®. Today, it is produced by a number of companies worldwide. In many hospitals, the drug has become the standard of care for cancer patients receiving chemotherapy or a bone marrow transplant. It is also used to increase the production of blood-forming stem cells in people who donate blood or bone marrow to transplant patients.

Thyrogen^®, or recombinant human thyroid–stimulating hormone, is used to test for recurrences of thyroid cancer in patients who have undergone a thyroidectomy. Prior to the approval of Thyrogen^®, patients tested for recurrences had to temporarily be put on thyroid hormone withdrawal, which can be debilitating. To date, Thyrogen^®, which was licensed to Genzyme, has been taken by more than 300,000 patients and is sold in 50 countries worldwide.

The immune system has an inherent ability to recognize and attack tumors, but in many cases this response is ineffective. Cytokines – cell signaling molecules that assist in the maturation and replication of several cell types in the immune system – have been shown to increase the effect of the antitumor immune response.

Scientists at Memorial Sloan Kettering demonstrated that Interleukin-2, or IL-2, is among the most effective cytokines for this purpose. Marketed by Novartis Oncology as Proleukin^® (aldesleukin) for injection, IL-2 was approved for the treatment of metastatic renal cell carcinoma in 1992, and for metastatic melanoma in 1998.

Approximately 3,000 to 5,000 patients are treated with Proleukin^® each year in the United States.

Researchers at Memorial Sloan Kettering and Columbia University demonstrated that HDAC inhibitors – drugs that target gene-regulating enzymes called histone deacetyleases – can induce tumor regression. HDAC inhibitors are believed to work by activating certain genes that suppress cancer development in normal cells, but may be repressed in tumor cells by histone deacetylease activity.

Suberoylanilide hydroxamic acid, or Zolinza^®, was the first of several HDAC inhibitors developed for the treatment of cancer. Marketed by Merck, Zolinza^® was approved in the United States for the treatment of advanced refractory cutaneous T cell lymphoma in 2006, and is in clinical trials for several other indications, including cancers of the breast, kidney, and colon.

DNA cloning, a commonly used method in the field of molecular biology, traditionally required a number of steps that were very time-consuming. Memorial Sloan Kettering scientists developed a way to eliminate many of these steps, reducing the bulk of the process to a highly efficient five-minute reaction.

Marketed by Invitrogen as TOPO^® PCR Cloning technology, the method takes advantage of an enzyme called topoisomerase I, which is present in the vaccinia virus.

TOPO^® PCR Cloning kits include standard PCR primers and a standard linearized vector with topoisomerase attached to both ends. The enzymes essentially replace themselves with the insert to be cloned, covalently attaching it to the vector. TOPO^® PCR cloning does not require gel or column purification of the insert, post-PCR modification — such as digestion, blunting, or dephosphorylation — or the addition of adapters.

Currently, there are 90 different products that incorporate TOPO^® Cloning technology, and TOPO^® Cloning products are sold in more than 80 countries.

Acute promyelocytic leukemia (APL) is an aggressive type of acute myeloid leukemia (AML). It happens when there are too many of the blood-forming cells called promyelocytes in the blood and bone marrow. A buildup of promyelocytes leads to a shortage of other kinds of blood cells, including red cells, white cells, and platelets.

Many people with APL are first treated with a drug called all-trans-retinoic acid (ATRA), also called tretinoin (Vesanoid®). ATRA is given in combination with another drug called arsenic trioxide (Trisenox®). Both of these drugs were pioneered at Memorial Sloan Kettering Cancer Center. 

Tumors can cause bone resorption leading to hypercalcemia, a condition caused by excessive levels of calcium in the blood. Ten to 20 percent of all cancer patients suffer from hypercalcemia, which can induce nausea, dehydration, stupors, and even comas. For some cancers, including breast and lung cancer, 30 to 40 percent of patients are hypercalcemic.

Memorial Sloan Kettering scientists demonstrated that a compound called gallium nitrate inhibits bone resorption and is an effective treatment for cancer-related hypercalcemia. Today, gallium nitrate is sold by Genta under the name Ganite^®.

Acute promyelocytic leukemia (APL) is a rare form of leukemia characterized by the accumulation of abnormal white blood cells in the bone marrow and blood. Patients with the disease often suffer from anemia, susceptibility to infections, bleeding, and hemorrhaging.

Twenty to 30 percent of patients receiving the standard treatment – all-trans retinoic acid usually in combination with an anthracycline – eventually suffer relapse. Arsenic trioxide, marketed as Trisenox^® by Cephalon, was approved by the FDA in 2000 as a second line of treatment in APL.

The anticancer drug clofarabine is approved for refractory or recurring acute lymphoblastic leukemia, and is in clinical development for the treatment of several other cancers. The drug is marketed by Genzyme as Clolar^® in the United States. In Europe, it is marketed by Bioenvision, a subsidiary of Genzyme, under the trade name Evoltra^®.

Clofarabine is a nucleoside analog that inhibits DNA synthesis and repair. It also disrupts the integrity of the mitochondrial membrane, inducing cancer cells to undergo programmed cell death.

Clofarabine was designed to retain anticancer activity while reducing the neurotoxicity associated with similar drugs. It is jointly owned by Memorial Sloan Kettering and the Southern Research Institute.

Hairy cell leukemia (HCL) is a rare type of indolent leukemia. Its name derives from the characteristic appearance of cytoplasmic projections on malignant lymphocytes.

About 600 people are diagnosed with HCL each year in the United States.

Memorial Sloan Kettering scientists discovered a number of antigenic markers that are characteristic of HCL. Marketed by BD Biosciences, these markers, known as CD22 and CD11c, are used in diagnostic tests for the disease.

In some cancers – including cancers of the lung, breast, uterus, and ovary, as well as non-Hodgkin lymphoma – a small percentage of patients have seemingly unrelated neurologic symptoms generally referred to as paraneoplastic neurologic syndromes. These symptoms include ataxia of the limbs and trunk, involuntary eye movements, and difficulty with speech articulation.

Paraneoplastic neurologic syndromes are believed to be caused by autoimmune reactions, in which patients produce antibodies against antigens expressed on the surface of tumor cells and as well as some noncancerous cells in the cerebellum.

Memorial Sloan Kettering scientists identified several of these antigens and used them to create diagnostic tests for paraneoplastic neurologic syndromes. These tests are marketed by Athena Diagnostics.

Canine oral melanoma is a common type of cancer in dogs, and the most common malignant tumor of the dog’s mouth. The disease is highly aggressive and frequently spreads to distant organs, including the lymph nodes, liver, lungs, and kidneys. The disease often spreads in spite of treatment with radiation and surgery, which in the past were the most common treatments for canine oral melanoma, resulting in death within five to six months.

Building upon their research on a human melanoma vaccine, Memorial Sloan Kettering scientists partnered with Merial to develop ONCEPT™, the first and only USDA-approved therapeutic vaccine for the treatment of canine cancer. ONCEPT™, which became available to veterinary oncologists in 2010, has been shown to extend the survival of dogs with advanced stages of the disease.