Tag Archives: FDA

Geron gets FDA OK for embryonic stem-cell based therapy

From BizJournals.com

The U.S. Food and Drug Administration has granted clearance of an investigational new drug application that lets Geron Corp. move forward with the world’s first study of a human embryonic stem cell based therapy in man, the company said late Thursday.

Menlo Park-based Geron (NASDAQ:GERN) said it plans to initiate a Phase I multi-center trial that is designed to establish the safety of the drug GRNOPC1 in patients with “complete” American Spinal Injury Association grade A subacute thoracic spinal cord injuries.

“The FDA’s clearance of our GRNOPC1 IND is one of Geron’s most significant accomplishments to date,” said Dr. Thomas B. Okarma, Geron’s president and CEO. “This marks the beginning of what is potentially a new chapter in medical therapeutics — one that reaches beyond pills to a new level of healing: the restoration of organ and tissue function achieved by the injection of healthy replacement cells.”

The ultimate goal for the use of GRNOPC1 is restore spinal cord function by injecting hESC-derived oligodendrocyte progenitor cells directly into the patient’s injured spinal cord.”

Geron funded the original research at the University of Wisconsin-Madison that led to the first isolation of hESCs. The production of oligodendrocytes from hESCs is covered by patent rights licensed to Geron from the University of California.

FDA Approves Drug that Boosts Stem Cell Yield for Bone Marrow Transplants

From FDA.gov

The U.S. Food and Drug Administration today approved Mozobil (plerixafor), a drug that helps increase the number of blood stem cells for bone marrow transplantation in patients with certain forms of blood cancer.

Mozobil is intended to be used in combination with the growth factor granulocyte-colony stimulating factor (G-CSF), for treatment of adults with multiple myeloma or non-Hodgkin’s lymphomas. Multiple myeloma is cancer of the plasma cell, a cell in the bone marrow that produces antibodies to help fight infection and disease. Non-Hodgkin lymphomas are a diverse group of blood cell cancers derived from lymphocytes, a type of white blood cell.

Prior to receiving high-dose chemotherapy or radiation therapy, patients with these forms of cancer sometimes undergo a procedure known as apheresis in which blood stem cells are collected and stored for reinfusion after therapy. G-CSF is commonly administered to help release and collect stem cells from the bone marrow. Mozobil is an injectable drug that, when used in combination with G-CSF, boosts the number of stem cells released from the bone marrow into the blood stream.

“Collecting the millions of cells needed for a bone marrow transplant can take hours or days,” said Richard Pazdur, M.D., director, Office of Oncology Drug Products, Center for Drug Evaluation and Research, FDA. “Mobozil provides a new therapeutic option for patients with certain types of blood cancers by increasing the number of stem cells collected in a given time period to be reinfused after therapy.”

In two randomized clinical trials – one in patients with non-Hodgkin’s lymphoma, the other with multiple myeloma – Mozobil combined with G-CSF increased the number of stem cells available for collection and transplantation compared with patients receiving G-CSF alone.

The most commonly reported adverse reactions in these trials and other smaller studies were diarrhea, nausea, fatigue, injection site reactions, headaches, joint pain, dizziness and vomiting.

Mozobil is manufactured by Genzyme Corp., Cambridge, Mass.

StemCells, Inc. Receives FDA Approval to Initiate Clinical Trial of HuCNS-SC(R) Cells in a Myelin Disease

StemCells, Inc.  (STEM) today announced that it has received approval from the U.S. Food and Drug Administration (FDA) to initiate a clinical trial of the Company’s proprietary HuCNS-SC product candidate (purified human neural stem cells) to treat Pelizaeus-Merzbacher Disease (PMD), a fatal brain disorder that mainly affects young children. This Phase I trial is designed to evaluate the safety and preliminary efficacy of HuCNS-SC cells as a treatment for PMD. Currently, there are no approved treatments for this disease.

This is the Company’s second FDA-approved clinical trial to evaluate HuCNS-SC cells as a potential treatment for neurodegenerative diseases. The first such study was the Company’s Phase I clinical trial of HuCNS-SC cells to treat neuronal ceroid lipofuscinoses (NCL), or Batten disease. The Phase I NCL trial will be completed in January 2009.

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