Therapeutics

On March 21, 2004, in Therapeutics
Vaccine research and trials have been much in the news. An experimental immunotherapy vaccine has
eliminated non-small-cell lung cancer in some advanced-stage patients, HPV vaccines in trial could prevent cervical
cancer in thousands of women, and a breakthrough in understanding the proteomics of the AIDS virus could lead to
an effective AIDS vaccine.

Blood was also much in evidence, with a new method of automating the process
of blood-typing under development,
proof that stem cells from umbilical cord
blood
can turn into healthy brain and heart cells in children with cancer
and metabolic diseases, and the successful freeze-drying of blood platelets, which
will have significant benefits in wound treatments and pharmaceuticals.

The long march of angiogenesis
inhibitor drugs
is nearing its goal of treating and preventing a multitude
of diseases. Monoclonal antibody drugs, too, continue their roll with promising trial results against
multiple sclerosis.

Other interesting developments:

  • Lab rats have been definitively cured of diabetes by the
    implantation of embryonic pig islet cells.
  • An “IV drill” alternative to the IV
    needle is claimed to represent “the new standard of care in emergency medicine.”
  • A genetic therapy that has dramatically increased muscle size and strength in
    rats is attracting strong interest from sports trainers. Unauthorized human
    trials are probably imminent if not already underway.
  • A new genetic discovery could lead to drugs that would extend the average human lifespan to
    150
    without giving up fast food.
Immunotherapy Vaccine Cures Lung
Cancer

An experimental vaccine called GVAX eliminated (for at least three years)
non-small cell lung cancer in three of 43 patients and slowed its spread in
others, according to findings just published in the Journal of the National
Cancer Institute. FDA approval is at least three years away, however. The
vaccine uses cancer cells taken from the patient and genetically modified to
make them more “noticeable” to the immune system. A similar approach has shown
promise against skin and renal cell cancer, reports the Associate Press.

In the study, the cancer disappeared in three of ten advanced-stage patients,
two of whom had previously undergone chemotherapy, which failed — as it almost
always does for advanced-stage patients. In the other advanced-stage patients
the cancer did not spread for almost five months to more than two years. For
patients in the early stage, the vaccine did not make much difference against
the cancer. The vaccine had no side effects.

Reference: Associated Press (1004). “Researchers:
New Vaccine Can Stop Lung Cancer
.” Washington Post, February 19.

HPV Vaccine

A vaccine undergoing clinical trials among 20,000 women worldwide for four
strains (6, 11, 16 and 18) of human papilloma virus (HPV) could be on the market
within three years. A similar vaccine for just HPV-16 alone (reported
in the Digest in January 2003
) provided complete protection against
that strain of the virus. If that proves to be true for the present vaccine,
writes Kristen Philipkoski in Wired, it would have a dramatic effect on
women’s health care and save millions of dollars in health care. Even if “only
moderately effective, it could save 1,300 lives and prevent more than 3,300
cases of cervical cancer over the lifetime of 2 million women.”

However, those benefits will only be achieved if the vaccine achieves wide
insurance coverage.

Reference: (2004). “Taking Aim at a
Stealthy STD
.” Wired News, February 25.

New Approach to an AIDS Vaccine

HIV/AIDS researchers are ecstatic about the discovery by their Harvard
colleagues of the reason why some monkeys are resistant to infection with the
AIDS virus. The discovery points to a new and highly promising strategy for
blocking HIV in people, says the Associated Press.

It turns out the monkeys have a protein that prevents the virus from shedding
its protective coat after it enters a healthy cell, preventing it from breaking
out of its shell to wreak havoc. Humans have a variant of the protein, and it
may be possible to design a drug that makes it work better.

Reference: Associated Press (2004). “Protein Keeps
Monkeys AIDS-Free
.” Wired News, February 25.

Automated Blood Typing

A startup company aims to develop a new method for automating blood typing.
It will initially focus on a method for replacing the largely manual,
labor-intensive process for typing donated blood and the blood of patients
receiving transfusions, using “antibody phage display” to isolate monoclonal
antibodies from humans to produce blood typing reagents that will eliminate the
need for shaking or spinning blood samples and looking for the clumping that
indicates blood type.

The new technique will search for the presence of unique pieces of DNA
associated with the antibody to determine blood type. The company hopes
eventually to use the same technology to develop diagnostic products and
therapeutic treatments for certain types of autoimmune diseases.

Reference: George, John (2004). “Firm
to automate typing of blood: PhenoTech born of funding from bio greenhouse
.”
Philadelphia Business Journal, February 6.

Cord Blood for Stem Cell Therapies

Duke University Medical Center researchers have shown conclusively that stem
cells from umbilical cord blood turned into healthy brain and heart cells in
children with cancer and metabolic diseases who received infusions. Stem cells
from umbilical cord blood, extracted from tissue otherwise discarded after a
childbirth, do not have the ethical baggage of embryonic stem cells and are
younger and more mutable than adult stem cells.

The scientists, reports Sarah Avery in the News & Observer, “hope
to devise a direct injection of stem cells into the brain, speeding the process
of regeneration. Someday, the procedure could be used to treat spinal cord
injuries and other problems.”

Reference: Avery, Sarah (2004). “Cord blood
shown to repair cells
.” News & Observer, February 23.

Freeze-dried Blood

Platelets — the cells present in blood that clump to form clots — are in
high demand for treating patients with bleeding diseases or undergoing
chemotherapy, but their use has been limited by a short shelf-life. A method has
now been devised of freeze-drying platelets without destroying their proteins
and cell membranes, to give them a shelf life of three months. They could be
used for transfusions and in wound-healing gels or bandages, and in the
liposomes used by pharmaceutical and biotechnology companies to encapsulate
freeze-dried drugs.

Animal studies have begun but it will take three or four years before humans
can be treated with freeze-dried platelets. The US military, which has never had
platelets available on the battlefield, has helped fund the research. The
researchers are also planning to study the application of their freeze-drying
technique to red blood cells and vaccines.

Reference: Lamb, Celia (2004). “UC
Davis finds market for freeze-dried blood platelets
.” Sacramento Business
Journal, February 6.

Drugs Starting to Fulfill Promise

The FDA’s approval of Avastin for colon cancer validates over three
decades of perseverance in the pursuit of angiogenic inhibitor drug therapies
and offers promise of angiogenic drugs for other cancers and for age-related
macular degeneration. Angiogenesis inhibitors are already in final testing for
more than a dozen cancers, including kidney and gastrointestinal cancers. In a
year-long study of nearly 1,200 people, the angiogenic drug Macugen
prevented significant loss of vision in 70 percent of patients, compared with 55
percent who got either a placebo or standard treatment, and improved vision in
six percent of patients, compared with two percent of patients on placebo or
standard treatment. Angiogenics also are being tested or the treatment of
retinopathy, psoriasis, endometriosis, rheumatoid arthritis, and Alzheimer’s
disease.

Preliminary research is also underway to see whether angiogenesis inhibitors
given to healthy people at risk of cancer could prevent the disease, for which
there is some evidence from Down Syndrome patients, who rarely get solid tumors
and have high levels of a natural angiogenesis inhibitor.

Reference: Dembner, Alice (2004). “Colon
cancer drug seen as long-awaited victory
.” Boston Globe, February 26.

Antegren for MS on Fast Track for
Approval

Biogen Idec intends to apply for US Food and Drug Administration (FDA)
approval of the monoclonal antibody drug Antegren (natalizumab) for
multiple sclerosis a year ahead of schedule, after achieving promising results
only one year into a two-year trial.

Reference: Krasner, Jeffrey (2004). “Biogen
Idec says MS drug is on fast track
.” Boston Globe, February 19.

Potential Cure for Diabetes Type I

Embryonic pig insulin cells transplanted into diabetic rats produced insulin
for the remainder of the rats’ natural lifetimes and were not rejected even by
rats in a control group not given anti-rejection drugs. Essentially, the
treatment cured diabetes. Pig insulin is already used successfully in type 1
diabetic humans.

The absence of rejection in the control group is thought to have been the
result of removing the pig embryo’s entire pancreas before its cells had time to
develop rejection-causing proteins, and the effectiveness of the treatment to
have been the result of implanting the entire pancreas into the peritoneal
membrane, whose veins empty directly into the liver, which is how insulin is
secreted normally. Primate testing could take place this year.

Other scientists have genetically modified pigs to remove the genes that
cause humans to reject the pigs’ cells. Tests of islet cells taken from such
transgenic pigs could begin in humans by the end of the year, reports
Wired‘s Kristen Philipkoski.

Reference: Philipkoski, Kristen (2004). “Pigs May Hold Key
to Diabetes
.” Wired News, February 27.

FDA Approval Soon for New IV Device

The VidaPort IV drill we mentioned in
September
that eliminates the time-consuming task of finding a usable vein
in trauma patients and instead drills a hole directly into the patient’s bone,
where fluids can flow directly into the marrow, is expected to “clear the FDA
any day now and move into quick pre-market trials,” according to VidaCare’s
president as of early February. He thinks it will become “the new standard of
care in emergency medicine.”

Reference: Thomas, Mike W. (2004). “VidaCare
expects to roll-out new medical device soon
.” San Antonio Business Journal,
February 6.

Genetic Therapy Builds Muscle

Injecting a virus carrying the gene for insulin-like growth factor 1 into lab
rats caused their target muscles to grow in size and strength by 15 to 30
percent. Putting the rats through an exercise program then doubled their muscle
strength. This research could lead to a therapy to increase muscle strength for
the elderly and muscular dystrophy patients, though safety concerns preclude
human clinical trials for at least several years.

If the behavior of athletes and their bosses with respect to
performance-enhancing drugs is anything to go by, then genetic manipulation is
probably going to happen. According to an Associated Press report, “word of the
research has reached the sports community and [one of the researchers] said that
half of the e-mails he receives now come from athletes or trainers wanting to
get information about the muscle-building therapy.” Given the money involved in
sports and sports medicine today, the sports world will likely quietly get to
human clinical trials, probably in third-world countries, long before the world
of legitimate medical research.

Reference: Associated Press (2004). “Gene Therapy Shows
Its Muscle
.” Wired News, February 16.

Longevity

“Worms, mice and primates all have been shown to appear to be younger on
severe diets,” reports Kristen Philipkoski in Wired, even in death.
Extreme caloric restriction may thus be both a life extender and an antidote to
diseases associated with aging such as Alzheimer’s, diabetes, cardiovascular
disease, and osteoporosis.

MIT researchers have identified two key genes that regulate aging in humans.
Manipulating the same genes in worms doubled their lives. One of the genes,
SIRT1, represses the activity of the other when the organism is taking in fewer
calories, and mitigates against cell damage from physical stress. The first gene
is thus “a putative regulator of lifespan in mammals and therefore a potential
target for future therapy in humans as well,” according to a University of
California researcher.

The discovery could lead to drugs that extend the average human lifespan to
150, without going on an extreme diet.

Reference: Philipkoski, Kristen (2004). “Stay Fat and Live
Long
.” Wired News, February 5.

 

 

 

 

 

 

 

 

 

 

 

 

 

 

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