Stem cells

Significant progress towards moving novel therapy into human clinical trials

California Stem Cell, Inc. (CSC) and Families of Spinal Muscular Atrophy (FSMA) announced that they have completed a formal pre-Investigational New Drug (Pre-IND) meeting with the Food and Drug Administration (FDA) for guidance on the clinical and regulatory pathway and requirements for submission of an IND to initiate human trials for a stem cell-derived motor neuron replacement therapy for Spinal Muscular Atrophy (SMA) Type I.
CSC, a leading stem cell therapeutics company, has developed a stem cell-derived motor neuron replacement product, for the treatment of SMA Type I. Pre-clinical studies, completed in collaboration with professor Hans Keirstead of the University of California, Irvine, have shown clinical proof of concept through the demonstration of functional benefit in animal models treated with CSC’s motor neuron replacement product, MotorGraft.

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Towards potential stem cell therapy

Families of Spinal Muscular Atrophy (FSMA), University of California, Irvine (UCI) Stem Cell Scientist; and California Stem Cell, Inc. (CSC) announce an extension to their collaboration to advance a potential stem cell therapy for SMA to human clinical trials.
The next stages of the collaboration will include the hiring of a Medical Director and a Clinical Research Coordinator at California Stem Cell, Inc. The two new positions will be responsible for coordinating the preparation of Investigational New Drug (IND) application and for overseeing and monitoring the resulting clinical trials. Together, the two individuals will spearhead and coordinate all aspects of the application for approval to begin, and preparations for, early phase clinical trials in SMA Type I.

The pivotal animal safety studies, required to support submission of an IND Application to the FDA, have recently been completed utilizing clinical grade human motor neurons manufactured by CSC. It is the intent of CSC to gain approval to begin human clinical trials for the use of these cells in development of a cell replacement therapy for SMA Type I. CSC is preparing for a final FDA pre-IND meeting to take place in the first half of 2009, keeping on track for a formal application by the end of 2009 for a Phase I/IIA clinical trial in SMA Type I.

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IPS cells

Since their discovery in 2006, the iPS cells, adult cells that have characteristics and potential similar to embryonic cells, are the happiness of specialists in stem cells. Here is a portrait of these promising cells.
To briefly describe the iPS cells (induced pluripotent stem cells) we can say that they are adult cells "rejuvenate" that behave like embryonic stem cells. Obviously, the scientific version of this description is somewhat more complex but still relatively simple. Here's what Marc Peschanski, director of Stem Cells (I-Stem), thinks: "What is amazing with iPS cells is the ease with which they are obtained".
In particular, the researchers start from an adult cell taken from a person, a cell of the teeth, skin, blood, etc.., in which introducing a genetic cocktail using retroviruses. Once reprogrammed, these cells are able to replicate indefinitely and differentiate into any cell in the body. In other words, possessing all the characteristics of embryonic stem cells, at least that is what the researchers are trying to prove.

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Motor neuron transplantation

FSMA has invested significant resources in alternative approaches that show promise to cure Spinal Muscular Atrophy rather than just treat the symptoms. In particular $1.5 Million to develop a motor neuron replacement therapy for SMA, with significant progress.
An initial investment in stem cell research in 2000 funded efficacy studies using motor neurons from mouse stem cells. Results show that this therapy can provide benefit to rodents with motor neuron disease: a highly significant finding. In 2005, additional FSMA funding lead to the first, highly-pure therapeutic population of human motor neurons for cellular replacement therapy for SMA. This program is now progressing on the path to IND in collaboration with the biotech firm California Stem Cell (CSC), and leading research centers at University of California-Irvine and Johns Hopkins University.

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