(Complimentary Newsletter from Target Health Inc.)
21 June 2004
I. WHAT'S NEW?
Goings On at Target Health - DIA and BIO-IT World
II. HISTORY OF MEDICINE
Anthrax
III. DIABETES
Lipitor Halves Stroke Risk in Diabetics
IV. CELLULAR THERAPY
Treating Heart Failure With Cells Grown in Culture
V. OPHTHALMOLOGY
Treatment of Ocular Hypertension in African Americans
VI. PEDIATRICS
Gene Therapy for Alpha-Mannosidosis
VII. FDA
Time May Be Up For Albuterol Metered-Dose Inhalers Using Chlorofluorocarbons
(CFCs)
Goings On at Target Health
- DIA and BIO-IT World
Target Health would like to welcome our new
readers we recently met in Washington. Target Health had a booth at the annual
meeting of the Drug Information Association (DIA) in Washington, where we met
many new and old colleagues. Target Health was also a sponsor of BIO-IT World's
Best Practices Awards dinner at the Washington Press Club. A good time was had
by all. A new publication, entitled, Meeting the Challenges
of Internet-based Clinical Trials, was just featured in the June edition of
Applied Clinical Trials. For more information about Target Health, please
contact Dr. Jules T. Mitchel (julesmitchel@targethealth.com).
Anthrax
Until recently, anthrax has been described as a veterinary disease of minor
importance to clinical medicine. Anthrax is known to cause occasional occupational
infections in single cases or clusters. Anthrax's potential for rapid and
widespread epidemic transmission under natural circumstances has not been
widely appreciated. A little-known 1770 epidemic that killed 15,000 people
in Saint-Domingue (modern Haiti) was probably intestinal anthrax. The epidemic
spread rapidly throughout the colony in association with consumption of uncooked
beef. Large-scale, highly fatal epidemics of anthrax may occur under unusual
but natural circumstances. Historical information may not only provide important
clues about epidemic development but may also raise awareness about bioterrorism
potential.
III. DIABETES
Lipitor Halves Stroke Risk
in Diabetics
Type II diabetes, which typically occurs in
adulthood and is closely linked with obesity, is one of the world's fastest
growing health problems. Type II diabetes is closely related to cardiovascular
disease, with two out of three sufferers dying from heart disease and stroke.
According to the International Diabetes Federation, there are 194 million Type
II diabetics worldwide. According to the results of a study, presented at the
annual meeting of the American Diabetes Association in Orlando, Lipitor
(atorvastatin; Pfizer), halved the risk of stroke in patients with diabetes and
cut cardiovascular events, including heart attacks, by more than a third.
According to the investigators, the clear benefits mean that physicians should
now consider giving so-called statin drugs routinely to patients with diabetes.
This could open up a huge new market for the cholesterol-lowering drugs,
already the world's top-selling medicines, and Lipitor in particular, which has
annual sales of $10 billion. A similar study last year on Merck & Co Inc's
rival statin Zocor showed it cut the risk of heart attack and stroke by a
third. The study showed that a 10 mg dose of Lipitor, reduced cardiovascular
events by 37% in diabetics with no previous history of cardiovascular disease,
while the incidence of stroke fell by 48%. Current best practice is to use
statins in diabetics only when they have elevated cholesterol levels or
established heart disease. According to the authors, the clinical trial
demonstrated that a much wider group of patients would actually benefit from
statin use, and that statins may become a third leg of a strategy that already
includes treatment for blood sugar levels and high blood pressure.
Treating Heart Failure With
Cells Grown in Culture
Five million Americans suffer from heart failure, a slow and often fatal
weakening of the heart muscle. Various drugs or devices can help ease symptoms
and slow the decline somewhat, but the prognosis for advanced cases remains
dismal. Now, according to the June 2004 Forbes Magazine, Genzyme and Medtronic
are teaming up to attack heart failure on a whole new front. They are testing a
radical new cell therapy technique that injects new cells into damaged heart
muscle in an effort to slow or halt the decline and help restore the heart's
functioning. Genyzme has been testing a method that involves transplanting
immature skeletal muscle cells called myoblasts into damaged areas of the
heart. The other technique is a high-tech catheter from Medtronic that can
snake through a vein in the leg all the way to the surface of the heart without
surgery. The aim of the collaboration is to devise a simple outpatient
procedure that would inject functioning new muscle cells into damaged areas of
the heart. To deliver new cell therapies to the heart, Medtronic's TransAccess
catheter system uses a needle guided by a tiny ultrasound transducer.
Initially, Medtronic and Genzyme will work to complete an ongoing 300-patient
trial in Europe. The study will test whether injecting skeletal myoblasts into
damaged areas of the heart during cardiac bypass surgery helps improve symptoms
and reduces hospitalizations in heart failure patients. Results from this trial
are due in three years. The Genzyme approach involves taking a muscle biopsy
from a patient's thigh and then growing the tissue sample in the lab for three
weeks until there are 600 million to 800 million myoblasts. The myoblasts then
are transplanted into damaged areas of the heart during surgery. The initial
trial on ten patients showed hints of efficacy, but because there was no
control group, it is impossible to tell whether the improvements seen were due
to the cell transplant or to the bypass surgery the patients also received.
V. OPHTHALMOLOGY
Treatment of Ocular Hypertension in African Americans
Primary open-angle glaucoma (POAG) affects about 2.2 million Americans age
40 and over, half of whom are not aware they have the disease. It is estimated
that between three and six million people in the U.S. are at increased risk
for developing POAG, representing between four and seven percent of the population
above age 40. Vision loss from glaucoma occurs when the optic nerve is damaged.
In most cases, elevated eye pressure, also called ocular hypertension, contributes
to this damage. This causes gradual loss of peripheral (side) vision. As the
disease progresses, the field of vision gradually narrows and blindness can
result. Glaucoma has no early symptoms, and by the time people experience
problems with their vision, they usually have a significant amount of optic
nerve damage. However, if detected early, glaucoma can usually be controlled
and serious vision loss prevented. Comprehensive dilated eye examinations
are recommended at least once every two years for African Americans over age
40 and all people over age 60. A study, published in the Archives of Ophthalmology
(2004;122:813-820), was performed to evaluate the safety and efficacy of topical
ocular hypotensive medication in delaying or preventing the onset of POAG
among African American. The study was known as the Ocular Hypertension Treatment
Study. It is well-known that the prevalence of glaucoma is higher in African
Americans than in whites. Study participants were between 40 and 80 years
of age, had intraocular pressure between 24 and 32 mm Hg in one eye and between
21 and 32 mm Hg in the other eye, and had no evidence of glaucomatous structural
or functional damage by standard clinical measures. Participants were randomized
to either the observation group or medication group. Of the 1,636 participants
randomized, 408 were self-identified as African American. The main outcome
measure was the development of reproducible visual field abnormality and/or
reproducible optic disc deterioration attributed to POAG. Study results showed
that among African Americans, 17 (8.4%) of 203 in the medication group developed
POAG during the study (median follow-up, 78 months) compared with 33 (16.1%)
of 205 participants in the observation group (hazard ratio, 0.50; P = .02).
According to the authors, topical ocular hypotensive therapy is effective
in delaying or preventing the onset of POAG in African American individuals
who have ocular hypertension.
VI. PEDIATRICS
Gene Therapy for Alpha-Mannosidosis
Alpha-mannosidosis (AM) encompasses a continuum of clinical findings from
an early lethal form to less symptomatic forms initially diagnosed in children.
Patients with a more severe phenotype have early death, mainly due to infections;
those with a milder phenotype have mild-to-moderate mental retardation, reduced
hearing, characteristic coarse features, immunodeficiency, clinical or radiographic
skeletal abnormalities, and primary central nervous system disease, mainly
cerebellar involvement causing ataxia. The disease is also frequently associated
with corneal opacities, aseptic destructive arthritis, and metabolic myopathy.
AM is insidiously progressive and patients may live into the sixth decade.
The diagnosis of AM relies on demonstration of deficient acid AM enzyme activity
in peripheral blood leukocytes or other nucleated cells such as fibroblasts.
According to an article published in the Journal of Pediatrics (2004;144:569-573),
a study was performed to determine the efficacy of hematopoietic stem cell
transplantation (HCT) for ameliorating the clinical manifestations of AM.
For the study, four patients with AM underwent allogeneic HCT at the University
of Minnesota. Diagnosis was established by assay of leukocyte AM activity
levels. Physical features, donor engraftment, leukocyte AM activity, neuropsychologic
function, and hearing were monitored before and after transplantation, with
follow-up ranging from 1 to 6 years. Results showed that all four patients
showed slowing of their neurocognitive development and sensorineural hearing
loss before HCT. All patients are alive, with normalization of leukocyte enzyme
activity after HCT. Intellectual function stabilized, with improvement in
adaptive skills and verbal memory function in 3 of 4 patients. Hearing has
improved to normal or near normal for speech frequencies in 3 patients. No
new skeletal abnormalities developed. At was concluded that HCT can halt the
progressive cognitive loss in patients with AM and that early diagnosis and
treatment with HCT is critical for optimal results.
TARGET HEALTH excels in Regulatory Affairs and works closely with
many of its clients and FDA and performs all FDA submissions. TARGET HEALTH
receives daily updates of new developments at FDA. Each week, highlights of
what is going on at FDA are shared to assure that new information is
expeditiously made available.
Time May Be Up For
Albuterol Metered-Dose Inhalers Using Chlorofluorocarbons (CFCs)
FDA is seeking public comment on a proposal
for withdrawing "essential-use" status for albuterol metered-dose
inhalers (MDIs) using chlorofluorocarbon (CFC) propellants. This notice is
being issued because two alternatives, non-CFC-based, albuterol MDIs are now
available. If the essential-use designation is removed, albuterol MDIs
containing an ozone-depleting substance (ODS) could not be marketed after an
appropriate transition period. FDA has tentatively concluded that the two
non-CFC based albuterol products will be satisfactory alternatives to the
albuterol MDIs containing ODS. CFCs have been commonly used as propellants for
various pressurized products including MDI treatments for asthma and chronic
obstructive pulmonary disease (COPD), which includes emphysema and chronic
bronchitis. Since 1978, the use of CFC-emitting aerosol products in the United
States has been generally banned because of increasing evidence that CFCs
contribute to the depletion of the earth's protective ozone layer. As a result
of an international agreement established through the Montreal Protocol on
Substances that Deplete the Ozone Layer and the U.S. Clean Air Act, CFC
production and importation have also been banned for all commercial purposes in
the United States since January 1996. The only exceptions to these bans are
products that are considered medically essential with no suitable alternatives.
Albuterol CFC-containing MDIs for asthma and COPD have been considered
exceptions from the ban. However, under the Montreal Protocol and the Clean Air
Act we are expected to phase out these products when suitable alternatives are
available. Although there are compelling reasons to phase out the use of these
products, as a public health agency FDA must also weigh the potential public
health impacts of such an action. The agency is providing a 60-day public
comment period from date of publication on this matter. For more information
about our capabilities in regulatory affairs, please contact Dr. Jules T.
Mitchel (julesmitchel@targethealth.com).
VIII.
TARGET HEALTH
TARGET HEALTH INC. (www.targethealth.com) is a full service
e*CRO with fulltime staff dedicated to all aspects of drug and device
development. Areas of expertise include Regulatory Affairs, comprising, but not
limited to, IND, IDE, NDA, PMA and 510(k) submissions, execution of Clinical
Trials, Project Management, Biostatistics and Data Management, Web Trials,
utilizing Target e*CRF¢â¢â, our proprietary Internet-based Remote Data Entry
(I-RDE¢â) system, and Medical Writing. TARGET HEALTH's Pharmaceutical Advisory
Dream Team (PADT) assists companies in strategic planning from Discovery to
Market Launch. Let us help you on your next project.
TARGET HEALTH INC.
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Dr. Jules T. Mitchel, President (julesmitchel@targethealth.com)
Ms Joyce Hays, CEO (joycehays@targethealth.com)
©2004 Target Health Inc. All rights reserved
