The one thing that troubles me about my illness is that who will teach my daughter the lessons that I have learned over the years? Someone suggested that I write letters to her which she can read later in her life. I tried writing but couldn’t go further than writing ‘dear’. It was just hard for me to write because it made me realize that she will grow up without me and I couldn’t comprehend what she might be going through when she reads them. Nevertheless I still want my experiences to be there for her when she needs. This article on CNN might just be the answer. What do you think? Is ‘Dad Council’ something that could work for me? Trust me it is very hard to digest the fact that I might not be around when my daughter needs me the most.

I haven’t updated my blog for quite sometime now.  But fear not, I’m back and will be updating my blog regularly.  I needed sometime to adjust to the technological changes that I need to keep up with my illness.

This is really encouraging, there is going to be a new drug In the market soon. Finally an alternative to very expensive Rilutek, I’m so excited…

In Part 2 of the study, 92 subjects were re-randomized to receive daily doses of 50 mg or 300 mg of dexpramipexole for 24 weeks. In addition to results again suggesting a dose-dependent trend in slowing the rate of disease progression as measured by the ALSFRS-R, there was also a trend toward a survival benefit in the 300 mg group compared with the 50 mg group. In an exploratory test comparing subject rankings on the basis of mortality and functional outcomes, subjects in the 300 mg group had a significantly improved outcome compared with the 50 mg group.

Knopp Neurosciences Presents Further Encouraging Trends in Its Phase 2 Study of KNS-760704 (Dexpramipexole) in ALS – Technology | Centre Daily Times – State College, PA

This is awesome news!

In the first federal-government-approved clinical trial of its kind in the U.S., patients with amyotrophic lateral sclerosis (ALS) will be treated with their own stem cells.

The aim is to hopefully slow the progress of the fatal, neurodegenerative disease.

TCA Cellular Therapy of Covington, a privately held stem-cell research and development company, has signed up the first of six participants for Phase I of the trial recently approved by the U.S. Food and Drug Administration.

2theadvocate.com | Features | Clinic to participate in stem-cell trial — Baton Rouge, LA

A new potential treatment on the horizon. I’m so excited!

Cytokinetics, Incorporated (Nasdaq: CYTK) announced today that its fast skeletal muscle troponin activator, CK-2017357, has been granted orphan-drug designation by the U.S. Food and Drug Administration (FDA) for the potential treatment of amyotrophic lateral sclerosis (ALS), also commonly known as Lou Gehrig’s Disease. CK-2017357 is the lead drug candidate that has emerged from the company’s skeletal sarcomere activator program. Cytokinetics plans to initiate a Phase II Evidence of Effect clinical trial for CK-2017357 in ALS patients in the first half of 2010.
Orphan-drug designation is granted by the FDA Office of Orphan Drug Products Development to novel drugs or biologics that may treat a condition affecting less than 200,000 persons in the United States or occurs in more than 200,000 persons and for which there is no reasonable expectation that the cost of development and distribution of the drug will be recovered. The designation offers a number of potential incentives, which may include a seven-year period of U.S. marketing exclusivity from the date of marketing authorization, funding for clinical studies, study design assistance, waiver of FDA user fees, and tax credits for clinical research.

Press Releases – Cytokinetics

Isis Pharmaceuticals of Carlsbad, Calif., has begun a phase 1 clinical trial of its experimental compound ISIS-SOD1-Rx in people with familial (inherited) ALS caused by toxic SOD1 protein molecules.

“This is a watershed moment,” said R. Rodney Howell, a metabolic disease specialist and chairman of the MDA Board of Directors. “More than 30 people with familial ALS caused by mutations in the SOD1 gene soon will receive infusions of a SOD1 inhibitor directly into their central nervous systems.”

A so-called "antisense" compound, ISIS-SOD1-Rx is designed to block production of the toxic SOD1 (superoxide dismutase 1) protein in people who have developed ALS because of mutations in the SOD1 gene. Antisense compounds are pieces of genetic information that keep other genetic information from being processed.

SOD1 mutations account for approximately 1 percent to 3 percent of all cases of ALS (amyotrophic lateral sclerosis), and about 20 percent of all familial ALS cases.

The SOD1 form of ALS is perhaps the best-understood form of the disease and almost the only form used in animal-based ALS studies

ALS SOD1 Trial: A ‘Watershed Moment’ | Quest Magazine Online