Some interesting CVID (and maybe SCID) developments

[Guest guest]

From (CVID)

I'm not sure if this news made lots of headlines, but one or two astute

people in my adult group picked up on some news releases today, and I

thought I'd pass on the info we've gathered.

I'm posting the articles below. This therapy, while still in the

testing phases (and not even yet approved as a drug by the FDA) does

hold some promise. Right now, the language of the journal articles is

still really technical and convoluted, but the MSNBC, CBS Marketwatch,

etc. summaries are pretty to the point, as is the website by the developer:

http://www.hgsi.com/products/blys.html

I have a lot of questions about the potential therapy -- its risks and

benefits, etc. -- but it does appear that *if* things go as planned, it

could be a way to stimulate the production of antibodies by B

cells/plasma cells in people with CVID or antibody deficiencies, and

perhaps in SCID patients post-BMT who have B cells which don't function

up to par. They are also saying it could be used in a number of other

populations (elderly patients, who lose B cell function with age; cancer

patients post-chemotherapy; other cancer patients with multiple

myeloma/chronic lymphocytic leukemia, etc). And the " opposite "

treatment may hold some promise in treating some autoimmune diseases,

because by blocking B-cell derived antibodies, you may be able to

prevent self-directed antibodies from being produced.

There are still many " ifs " and a lot more testing to go, but since the

media's going nuts (and some of the people in my adult group are, too),

I figured I'd share the info with these groups, as well. I'll send them

in a series of messages to follow.

In this one, here's the media reports:

Human Genome Sciences Confirms It Will Test Immune-Boosting Protein In Humans

Friday, June 23, 2000 04:28 AM

ROCKVILLE, Md. -(Dow )- Drug research-and-development company Human

Genome Sciences Inc. said it

would begin human tests of B-lymphocyte stimulator, a protein that is

one of the body's primary natural

weapons against infections.

The announcement confirmed a report in Friday's Wall Street Journal and

sent shares of Human Genome

(HGSI, news, msgs) higher.

The company said its plans follow a Food and Drug Administration review

of its investigational

new-drug application, which pharmaceutical companies must file before

conducting clinical trials of a drug in humans.

Human Genome Sciences said it would begin clinical trials of

B-lymphocyte stimulator, or BLyS, in

patients with common variable immunodeficiency, one of a group of

immune-system disorders characterized by increased susceptibility to

infections such as pneumonia, bronchitis and sinusitis. These patients

require lifelong treatment with antibodies to protect against recurrent

infections.

The news comes as Monday's much-anticipated announcement that scientists

have deciphered the entire

sequence of human genetic code is already being hailed as one of the

most remarkable feats in human history. But even more practical advances

are flowing from other genomic discoveries.

The BLyS compound is the fourth gene-based drug candidate developed by

Human Genome Sciences to

advance to human trials, expanding the biotechnology company's early

lead in the race to convert information from the human genome into new

drugs. No other company has more than one such drug.

The apparent breakthrough also follows & 's (JNJ, news,

msgs) recent disclosure that it

is close to human tests of a gene-based drug that targets a brain

receptor associated with memory and

attention. It also reflects the impact of new robotic and other

technology that is enabling drug companies to rapidly translate genomic

data into potential new medicines.

BLyS, a long-sought protein that was subject of an intense hunt by

several pharmaceutical companies,

appears to be particularly versatile. Rockville, Md.-based Human Genome

Sciences hopes to soon launch

additional studies of the substance to include certain cancers, patients

undergoing chemotherapy and organ transplants, and perhaps older people

whose immune systems have weakened with age.

" Genomics allowed us to find something that others couldn't find, " says

Dr. Haseltine, chief

executive officer of Human Genome Sciences. " From one discovery, we

think we will be able to treat a very broad array of diseases. "

There isn't any assurance, of course, that BLyS or any of the other

three Human Genome Sciences

proteins will pass muster in human trials and make it to market. The

majority of experimental medicines that enter clinical trials fail to

clear the FDA; thus, plenty of young biotech companies have foundered

after showing promise. While many scientists are hopeful that data from

the human genome will help improve on that performance, that too remains

a risky bet.

Human Genome rallies on news of trial

Study to target people with immune problems

By Greg Morcroft, CBS.MarketWatch.com

Last Update: 1:04 PM ET Jun 23, 2000 NewsWatch

Latest headlines

NEW YORK (CBS.MW) -- Human Genome's rally continued early Friday

afternoon as the shares rose more than 9 percent after it outlined human

clinical trials of B-lymphocyte stimulator, or BLyS, a protein that may

help patients with immune system problems.

The shares recently traded at 148, up 15 or more than 11 percent.

Human Genome said its plans follow a review of the company's

Investigational New Drug application with the U.S. Food and Drug

Administration.

Merrill Lynch analyst Todd , in a research report to clients, said

BLyS represents the company's third genomics derived drug to enter into

clinical trials.

" Although anticipated, the announcement illustrates the company's

ability to continue to drive long term shareholder value, " said

in his note. " The announcement that the company has received approval to

begin BlyS clinical trials bolsters our confidence in HGS's early stage

pipeline. "

BLyS is a protein made by the human body that stimulates the production

of antibodies, which fight disease.

Patients in the trial will be sufferers of Common Variable

Immunodeficiency, or CVID, one of a group of

primary immunodeficiency disorders that are characterized by increased

susceptibility to infection.

Jerry Winkelstein, a professor at s Hopkins University said in a

press release issued by Human Genome (HGSI: news, msgs) " Patients with

Common Variable Immunodeficiency are unable to make adequate amounts of

their own antibodies. As a result, they have to depend on antibody

treatments for the rest of their life. BLyS may restore their ability to

produce antibodies again, which would be a very important advance in

their treatment. "

[Guest guest]

,

Thank you for the articles. Very interesting.

Have you tried the Ametop?

Jan

wrote:

> >From (CVID)

>

> I'm not sure if this news made lots of headlines, but one or two astute

> people in my adult group picked up on some news releases today, and I

> thought I'd pass on the info we've gathered.

>

> I'm posting the articles below. This therapy, while still in the

> testing phases (and not even yet approved as a drug by the FDA) does

> hold some promise. Right now, the language of the journal articles is

> still really technical and convoluted, but the MSNBC, CBS Marketwatch,

> etc. summaries are pretty to the point, as is the website by the developer:

> http://www.hgsi.com/products/blys.html

>

> I have a lot of questions about the potential therapy -- its risks and

> benefits, etc. -- but it does appear that *if* things go as planned, it

> could be a way to stimulate the production of antibodies by B

> cells/plasma cells in people with CVID or antibody deficiencies, and

> perhaps in SCID patients post-BMT who have B cells which don't function

> up to par. They are also saying it could be used in a number of other

> populations (elderly patients, who lose B cell function with age; cancer

> patients post-chemotherapy; other cancer patients with multiple

> myeloma/chronic lymphocytic leukemia, etc). And the " opposite "

> treatment may hold some promise in treating some autoimmune diseases,

> because by blocking B-cell derived antibodies, you may be able to

> prevent self-directed antibodies from being produced.

>

> There are still many " ifs " and a lot more testing to go, but since the

> media's going nuts (and some of the people in my adult group are, too),

> I figured I'd share the info with these groups, as well. I'll send them

> in a series of messages to follow.

>

> In this one, here's the media reports:

>

> Human Genome Sciences Confirms It Will Test Immune-Boosting Protein In Humans

> Friday, June 23, 2000 04:28 AM

>

> ROCKVILLE, Md. -(Dow )- Drug research-and-development company Human

> Genome Sciences Inc. said it

> would begin human tests of B-lymphocyte stimulator, a protein that is

> one of the body's primary natural

> weapons against infections.

>

> The announcement confirmed a report in Friday's Wall Street Journal and

> sent shares of Human Genome

> (HGSI, news, msgs) higher.

>

> The company said its plans follow a Food and Drug Administration review

> of its investigational

> new-drug application, which pharmaceutical companies must file before

> conducting clinical trials of a drug in humans.

>

> Human Genome Sciences said it would begin clinical trials of

> B-lymphocyte stimulator, or BLyS, in

> patients with common variable immunodeficiency, one of a group of

> immune-system disorders characterized by increased susceptibility to

> infections such as pneumonia, bronchitis and sinusitis. These patients

> require lifelong treatment with antibodies to protect against recurrent

infections.

>

> The news comes as Monday's much-anticipated announcement that scientists

> have deciphered the entire

> sequence of human genetic code is already being hailed as one of the

> most remarkable feats in human history. But even more practical advances

> are flowing from other genomic discoveries.

>

> The BLyS compound is the fourth gene-based drug candidate developed by

> Human Genome Sciences to

> advance to human trials, expanding the biotechnology company's early

> lead in the race to convert information from the human genome into new

> drugs. No other company has more than one such drug.

>

> The apparent breakthrough also follows & 's (JNJ, news,

> msgs) recent disclosure that it

> is close to human tests of a gene-based drug that targets a brain

> receptor associated with memory and

> attention. It also reflects the impact of new robotic and other

> technology that is enabling drug companies to rapidly translate genomic

> data into potential new medicines.

>

> BLyS, a long-sought protein that was subject of an intense hunt by

> several pharmaceutical companies,

> appears to be particularly versatile. Rockville, Md.-based Human Genome

> Sciences hopes to soon launch

> additional studies of the substance to include certain cancers, patients

> undergoing chemotherapy and organ transplants, and perhaps older people

> whose immune systems have weakened with age.

>

> " Genomics allowed us to find something that others couldn't find, " says

> Dr. Haseltine, chief

> executive officer of Human Genome Sciences. " From one discovery, we

> think we will be able to treat a very broad array of diseases. "

>

> There isn't any assurance, of course, that BLyS or any of the other

> three Human Genome Sciences

> proteins will pass muster in human trials and make it to market. The

> majority of experimental medicines that enter clinical trials fail to

> clear the FDA; thus, plenty of young biotech companies have foundered

> after showing promise. While many scientists are hopeful that data from

> the human genome will help improve on that performance, that too remains

> a risky bet.

>

>

> Human Genome rallies on news of trial

> Study to target people with immune problems

>

> By Greg Morcroft, CBS.MarketWatch.com

> Last Update: 1:04 PM ET Jun 23, 2000 NewsWatch

> Latest headlines

>

> NEW YORK (CBS.MW) -- Human Genome's rally continued early Friday

> afternoon as the shares rose more than 9 percent after it outlined human

> clinical trials of B-lymphocyte stimulator, or BLyS, a protein that may

> help patients with immune system problems.

>

> The shares recently traded at 148, up 15 or more than 11 percent.

>

> Human Genome said its plans follow a review of the company's

> Investigational New Drug application with the U.S. Food and Drug

> Administration.

>

> Merrill Lynch analyst Todd , in a research report to clients, said

> BLyS represents the company's third genomics derived drug to enter into

> clinical trials.

>

> " Although anticipated, the announcement illustrates the company's

> ability to continue to drive long term shareholder value, " said

> in his note. " The announcement that the company has received approval to

> begin BlyS clinical trials bolsters our confidence in HGS's early stage

pipeline. "

>

> BLyS is a protein made by the human body that stimulates the production

> of antibodies, which fight disease.

>

> Patients in the trial will be sufferers of Common Variable

> Immunodeficiency, or CVID, one of a group of

> primary immunodeficiency disorders that are characterized by increased

> susceptibility to infection.

>

> Jerry Winkelstein, a professor at s Hopkins University said in a

> press release issued by Human Genome (HGSI: news, msgs) " Patients with

> Common Variable Immunodeficiency are unable to make adequate amounts of

> their own antibodies. As a result, they have to depend on antibody

> treatments for the rest of their life. BLyS may restore their ability to

> produce antibodies again, which would be a very important advance in

> their treatment. "

>

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Primary Immune Deficiency. Opinions or medical advice stated here are the sole

responsibility of the poster and should not be taken as professional advice.