Families Of Spinal Muscular Atrophy Announces Latest Developments On Three SMA Drug Discovery Programs

Main Category: Muscular Dystrophy / ALS
Article Date: 20 Jun 2009 - 0:00 PDT

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Families of Spinal Muscular Atrophy has just released details of the latest developments for the Families of SMA funded therapeutic pipeline.

Families of SMA is currently funding three distinct drug discovery programs: 1) Quinazolines to boost gene expression; 2) Tetracyclines at Paratek Pharmaceuticals to correct gene splicing; and 3) Motor neuron replacement program at California Stem Cell and UCI. These three programs represent an investment of over $16 Million so far to build a SMA drug pipeline.

Quinazoline Program:

In June 2009, the IND-enabling safety studies were completed for the FSMA-directed Quinazoline program. This is the program previously worked on by Vertex Pharmaceuticals and deCODE genetics, and fully funded by Families of SMA.

The next step for this program is to request a pre-IND meeting with the FDA and to prepare the required data package. Families of SMA will be working over the next several months with toxicology and regulatory experts to prepare for this meeting. According to Jill Jarecki, Ph.D., Research Director FSMA, "This meeting will be an exciting milestone for the project, and an essential step in the path towards human clinical trials of the drug candidate."

Motor Neuron Replacement Program:

California Stem Cell (CSC) is preparing for a final FDA pre-IND meeting to take place in mid 2009. This is a critical step on the track to submitting a formal Investigational New Drug (IND) application to the FDA to begin clinical trials in SMA Type I. It is the intent of CSC to gain approval to begin FDA-approved clinical trials for the use of these cells in development of a cell replacement therapy for SMA Type I.

Numerous pre-clinical efficacy studies have been completed, demonstrating that the cells work. The pivotal animal safety study, required to support an application to begin clinical trials, was completed in October 2008. According to Chris Airriess, Ph.D., Chief Operating Officer, California Stem Cell, Inc., "CSC is now preparing for a final FDA pre-IND meeting to take place in mid 2009, keeping us on track for a formal IND application to begin a Phase I/IIA clinical trial in SMA Type I."

Tetracycline Program:

According to Paul Higgins, Ph.D., Director, Inflammation Drug Discovery, Paratek Pharmaceuticals, "FSMA has supported the research at Paratek for the previous three years for our work on tetracycline derivatives as a potential treatment of SMA. These compounds are intended to correct SMN2 splicing and in turn increase SMN protein levels.

Using funding from FSMA to generate the preliminary data for a grant application, Paratek has been awarded a multi-million dollar U01 grant from the NINDS to continue our research that FSMA funded for the past 3 years."

The overall goal for this project is to develop a drug candidate for SMA resulting in an Investigational New Drug (IND) application with the Food and Drug Administration (FDA) within 4 to 5 years.

These three announcements were all made at the 25th Anniversary SMA Conference. A record of over 900 families and researchers were present at the conference.

About Families of SMA:

Families of Spinal Muscular Atrophy is dedicated to creating a treatment and cure by:

Funding and advancing a comprehensive research program; Supporting SMA families through networking, information and services; Improving care for all SMA patients; Educating health professionals and the public about SMA; Enlisting government support for SMA; Embracing all touched by SMA in a caring community.

Our vision is a world where Spinal Muscular Atrophy is treatable and curable.

The goal of the organization for 2009 is to have raised $50 million for SMA research. Families of SMA funds and directs the leading SMA research programs. Families of SMA has created hope for our community that did not exist in 1984. Our support comes from generous individual donations and numerous fundraising events held by volunteer families and our chapters.

Our successful results and progress from basic research to drug discovery programs to clinical trials provides real hope for families and patients:

-Families of SMA has funded 5 multi-center clinical trials for existing drugs that have potential for SMA.

-FSMA has directed and funded the leading new drug development program for a therapy specially designed to treat SMA.

-Families of SMA is building a pipeline of drug discovery programs based on our investments in basic research.

-FSMA has invested significant resources into alternative approaches that show promise to cure SMA.

Source: Families of Spinal Muscular Atrophy

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