NEW YORK, Nov. 8, 2012 /PRNewswire/ --Stemline Therapeutics, Inc., a clinical-stage biopharmaceutical company developing oncology therapeutics that target both cancer stem cells (CSCs) and tumor bulk, announced today that a heavily pre-treated patient with a drug-refractory and recurrent blastic plasmacytoid dendritic cell neoplasm (BPDCN) achieved a complete response (CR) following treatment with Stemline's SL-401. BPDCN is a rare and aggressive hematologic cancer which is generally unresponsive to standard treatment regimens. SL-401 is Stemline's lead therapeutic being developed to treat acute myeloid leukemia (AML) and other hematologic cancers.

The patient, a 40-year-old male, developed progressive BPDCN with malignant cells in his blood and bone marrow, which resulted in low blood cell counts despite receiving several intensive high-dose chemotherapy regimens, including bone marrow transplantation. After developing progressively worsening BPDCN following failure of multiple standard drug regimens, his physicians referred him to the MD Anderson Cancer Center in Houston, Texas and the Texas A&M Health Science Center College of Medicine at Scott and White Cancer Research Institute in Temple, Texas, to receive treatment with Stemline's SL-401 in a Phase 1/2 clinical trial. The patient was treated with five daily doses of SL-401, a targeted therapy directed specifically to cells that overexpress the interleukin-3 receptor (IL-3R) on their cell surface. BPDCN is known to overexpress IL-3R. SL-401 has previously been shown to possess potent anti-BPDCN activity in preclinical models. Thirty days after SL-401 treatment, the patient achieved a CR, with no evidence of malignant BPDCN cells in his bone marrow or bloodstream and his blood cell counts; in particular, his absolute neutrophil and platelet counts returned to normal levels. Furthermore, he did not experience any serious side effects from SL-401 treatment. "It is always very gratifying to observe a CR and negligible side effects following use of a new agent in patients with cancers, in which approved treatments are of little to no value," said Dr. Eric Rowinsky, Chief Medical Officer and Head of Research and Development at Stemline Therapeutics. "Since we believe the explanation for this prominent clinical response and lack of side effects is SL-401's specific targeting of IL-3R, which is overexpressed on BPDCN and other hematologic malignancies, Stemline will continue to evaluate and potentially register SL-401 in BPDCN."

BPDCN is a relatively uncommon hematologic cancer of plasmacytoid dendritic cells, which are specialized immune cells that circulate throughout the bloodstream, bone marrow, and many other organs. In BPDCN, the cancer cells typically reside and grow in the skin and bone marrow. Their growth in the bone marrow results in decreased blood cell counts, thereby causing serious infections, bleeding, and invariably death. Although BPDCN can be controlled for brief periods with standard chemotherapy that is used to treat other hematologic cancers, meaningful clinical responses are rare and such treatment often produces serious side effects. There are no therapies approved to treat BPDCN. BPDCN cells express high levels of IL-3R on their cell surface, which served as the rationale for treating the patient with SL-401, which specifically targets the IL-3R. SL-401 has also demonstrated clinical activity in other hematologic malignancies that overexpress IL-3R, including AML and myelodysplastic syndrome (MDS). Other malignancies in which high levels of the IL-3R are expressed include Hodgkin's and non-Hodgkin's lymphoma, chronic myeloid leukemia (CML), and multiple myeloma. Preclinical studies of SL-401 in these cancers have demonstrated favorable anticancer activity. SL-401 is currently being advanced into a registration-directed Phase 2b study in advanced stage AML.

About Stemline Therapeutics, Inc.

Stemline Therapeutics, Inc. is a clinical stage biopharmaceutical company developing novel oncology therapeutics that target both cancer stem cells (CSCs) as well as the tumor bulk. Among Stemline's drug candidates are SL-401 and SL-701, both of which have demonstrated single agent clinical activity in Phase 1/2 studies of advanced cancer patients. In a multicenter Phase 1/2 trial in patients with advanced acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS), SL-401 demonstrated single agent activity, including durable complete responses (CRs), and an overall survival (OS) improvement relative to historical data in the most heavily pretreated AML patients. In addition, SL-401 was well-tolerated and was not toxic to the bone marrow. SL-401 is being advanced into later stage trials in advanced AML. In Phase 1/2 trials, Stemline's second clinical stage therapeutic, SL-701, has demonstrated single agent activity including durable CRs and partial responses (PRs) in adult patients with refractory or recurrent glioblastoma and pediatric patients with malignant glioma, as well as an OS benefit in adult patients with refractory or recurrent glioblastoma compared with historical data. SL-701 is now poised for later stage trials in pediatric and adult patients with advanced brain cancer. Stemline is also developing a broad portfolio of preclinical small molecules and antibodies for a variety of solid and hematological cancer types. Many of these compounds have derived from the Company's proprietary discovery platform, StemScreen. For more information, please visit the Company's website at http://www.stemline.com.

Stemline Contact:

Mark Jacobson Director, Corporate Development Stemline Therapeutics, Inc. 750 Lexington Avenue Sixth Floor New York, NY 10022 Tel: 646-502-2307 Email: mjacobson@stemline.com http://www.stemline.com

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Stemline Therapeutics' Lead Clinical Candidate SL-401 Induces a Complete Response in a Patient with a Drug-Refractory ...

EASTERN SHORE, Md. (WJZ) The miracle of stem cells changes the life of a little boy from the Eastern Shore.

Adam May has the amazing story of a mother and the choice she made moments after her son was born.

Xander McKinley was a beautiful babybut challenging. The newborn didnt eat or sleep well, and by two-years-old, he couldnt walk or even crawl.

Something just wasnt right, said Xanders mother, Jennifer McKinley.

Jennifer McKinley got the news every parent fears. Xander had cerebral palsya brain condition that slows motor functions.

Adam: Did you ever fear he would never have a normal life? Jennifer: We thought he wasnt going to be able to walk. It was heart-wrenching, that we knew this was permanent.

Turns out, it wasnt permanent. That young boy frustrated by immobility can now stand on his own, and even take a few stepsafter a groundbreaking experimental stem cell transfusion.

Adam: Arent you walking better now? Xander: Yeah. Adam: How good does that feel? Xander: Great!

The six-year-old can finally climb a fence with his brother.

Adam: Youre so fast now Xander: Yeah!

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Stem Cells Help Md. Boy With Cerebral Palsy Walk

ScienceDaily (Nov. 8, 2012) Canadian and Italian stem cell researchers have discovered a new "master control gene" for human blood stem cells and found that manipulating its levels could potentially create a way to expand these cells for clinical use.

The findings, published November 8 online ahead of print in Cell Stem Cell, usher in a new paradigm for the regulation of human blood stem cells, says co-principal investigator Dr. John Dick, who holds a Canada Research Chair in Stem Cell Biology and is a Senior Scientist at University Health Network's McEwen Centre for Regenerative Medicine and Ontario Cancer Institute (OCI), the research arm of the Princess Margaret Cancer Centre. He is also a Professor in the Department of Molecular Genetics, University of Toronto.

"For the first time in human blood stem cells, we have established that a new class of non-coding RNA called miRNA represents a new tactic for manipulating these cells, which opens the door to expanding them for therapeutic uses," says Dr. Dick.

In 2011, Dr. Dick isolated a human blood stem cell in its purest form -- as a single stem cell capable of regenerating the entire blood system -- paving the way for clinical uses. He also pioneered the cancer stem cell field by identifying leukemia stem cells in 1994 and colon cancer stem cells in 2007.

OCI lead author Dr. Eric Lechman says the research team removed a master control gene -- microRNA 126 (miR-126) -- that normally governs the expression of hundreds of other genes by keeping them silenced, which in turn keeps the stem cells in a non-dividing dormant state. The method was to introduce excess numbers of miR-126 binding sites into the stem cells by using a specially designed viral vector.

"The virus acted like a sponge and mopped up the specific miRNA in the cells. This enabled the expression of normally repressed genes to become prominent, after which we observed a long-term expansion of the blood stem cells without exhaustion or malignant transformation," says Dr. Lechman.

Adds Dr. Dick: "We've shown that if you remove the miRNA you can expand the stem cells while keeping their identity intact. That's the key to long-term stem cell expansion for use with patients." The co-principal investigator was Dr. Luigi Naldini, Director, of the San Raffaele Telethon Institute for Gene Therapy, Milan.

Dr. Dick's research was funded by the Canadian Institutes of Health Research, the Canadian Cancer Society, the Terry Fox Foundation, Genome Canada through the Ontario Genomics Institute, the Ontario Institute for Cancer Research, the Canada Research Chair Program, the Ontario Ministry of Health and Long-Term Care, the Canada Foundation of Innovation, as well as The Princess Margaret Cancer Foundation.

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Stem cell scientists discover potential way to expand cells for use with patients

Every week in his clinic at the University of Michigan, neurologist Joseph Corey, M.D., Ph.D., treats patients whose nerves are dying or shrinking due to disease or injury. He sees the pain, the loss of ability and the other effects that nerve-destroying conditions cause - and wishes he could give patients more effective treatments than what's available, or regenerate their nerves.

Then he heads to his research lab at the VA Ann Arbor Healthcare System, where his team is working toward that exact goal.

In new research published in several recent papers, Corey and his colleagues from the U-M Medical School, VAAAHS and the University of California, San Francisco report success in developing polymer nanofiber technologies for understanding how nerves form, why they don't reconnect after injury, and what can be done to prevent or slow damage.

Using polymer nanofibers thinner than human hairs as scaffolds, researchers coaxed a particular type of brain cell to wrap around nanofibers that mimic the shape and size of nerves found in the body.

They've even managed to encourage the process of myelination - the formation of a protective coating that guards larger nerve fibers from damage. They began to see multiple concentric layers of the protective substance called myelin start to form, just as they do in the body. Together with the laboratory team of their collaborator Jonah Chan at UCSF, the authors reported the findings in Nature Methods.

The research involves oligodendrocytes, which are the supporting actors to neurons - the "stars" of the central nervous system. Without oligodendrocytes, central nervous system neurons can't effectively transmit the electrical signals that control everything from muscle movement to brain function.

Oligodendrocytes are the type of cells typically affected by multiple sclerosis, and loss of myelin is a hallmark of that debilitating disease.

The researchers have also determined the optimum diameter for the nanofibers to support this process - giving important new clues to answer the question of why some nerves are myelinated and some aren't.

While they haven't yet created fully functioning "nerves in a dish," the researchers believe their work offers a new way to study nerves and test treatment possibilities. Corey, an assistant professor of neurology and biomedical engineering at the U-M Medical School and researcher in the VA Geriatrics Research, Education and Clinical Center, explains that the thin fibers are crucial for the success of the work.

"If it's about the same length and diameter as a neuron, the nerve cells follow it and their shape and location conform to it," he says. "Essentially, these fibers are the same size as a neuron."

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Stem cells and nanofibers stimulating nerve research