(photo credit: Courtesy)
A patient with amyotrophic lateral sclerosis (ALS, or Lou Gehrig’s disease) has testified to the effectiveness of
the stem cell-based treatment for the disease being developed by Brainstorm Cell
In a letter obtained by Globes, Avihai Kremer said: “I
was diagnosed [with ALS] seven years ago at age 29. Today I am almost completely
paralyzed and can’t walk, talk or eat (I use a feeding tube). I am writing this
letter using my head. ALS/MND [motor neuron disease] has no effective treatment,
and diagnosis means a death sentence even today, a decade into the 21st
“Nevertheless, I was fortunate to be the first to get the
stem-cells treatment of Brainstorm Cell Therapeutics two and a half months ago
[October 2010] at c in Jerusalem, Israel. The procedure was
simple and quick. Within 24 hours I was back home. Two and a half months later,
I can testify that the Brainstorm treatment is completely safe and that my
condition didn’t deteriorate in that time. In fact, not only did the disease
seem to slow/stop in that time period, but preliminary follow-up tests done at
Hadassah showed slight improvement in several physical functions such as
breathing and speech.”
Kremer is the first patient to be treated with
Brainstorm Cell’s drug, NurOwn, on the basis of a special approval. The
treatment is not part of the clinical trials that are conducted to establish
statistical analyses of a drug’s efficacy and safety. The company is currently
conducting a Phase I/II clinical trial of the drug.
Treatment with a drug
without US Food and Drug Administration (FDA) or Health Ministry approval of a
single patient is an extraordinary procedure, only given in incurable diseases.
Such cases are usually handled at the medical center that is also conducting the
clinical trial, and the results are not always sent to the company that is
developing the drug.
Brainstorm Cell initiated the clinical trial of
NurOwn last month and is currently selecting patients for
If the treatment is successful, it could be a historic
achievement, as there is no treatment for ALS, and sufferers die within two to
five years. However, success in a single patient often cannot be replicated and
is statistically meaningless in a clinical trial, and it has no bearing on
approval of the drug.
Nonetheless, Kremer’s letter is the first
indication of possible success of Brainstorm Cell’s treatment – provided that
the clinical trial achieves similar results. A successful treatment opens the $1
billion a year ALS market before the company.
Brainstorm Cell declined to
comment on the report.
Brainstorm Cell’s share price closed at $0.44 on
Wall Street on Wednesday, giving a market cap of $52.6 million.