Monster Media 1994 #2/Monster Media No. 2 (Monster Media)(1994).ISO/text/nkcanews.zip/KCN942.TXT

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                              KIDNEY CANCER NEWS
                                Vol. V, No. II

                     (C) April 1994.  All Rights Reserved.

                      National Kidney Cancer Association
                              1234 Sherman Avenue
                           Evanston, Illinois  60202
                               708 - 332 - 1051



Gene for Kidney Cancer Isolated

A research team headed by scientists at the National Cancer Institute (NCI) has
identified the gene responsible for the most common type of kidney cancer.  The
finding is reported in the May 1994 issue of Nature Genetics.

The cancer, called sporadic (non-familial) clear cell carcinoma, accounts for
about 23,500 newly diagnosed cases of kidney cancer each year, or about 85
percent of all cases of the disease.  Currently, there are an estimated 75,000
kidney cancer patients in the U.S.

"With identification of this kidney cancer gene, it will be possible to develop
new methods to improve the diagnosis and treatment of the disease and
potentially to find way to prevent it," said W. Marston Linehan, M.D., of NCI's
Surgery Branch.  "The finding also will make it possible to develop a blood or
urine test to detect kidney cancer early when it is most treatable."

When detected in its earliest stages, the five-year relative survival rate for
kidney cancer is 86 percent.  If detected after it has spread to distant
organs, the survival rate is 10 to 20 percent at the end of two years.  In the
United States, about 8,400 people will die this year of clear cell carcinoma of
the kidney.

The damage or mutated gene responsible for sporadic clear cell carcinoma of the
kidney is a tumor suppressor gene located on the short arm of chromosome 3.
The protein produced by the gene appears to restrain normal growth.  The
researchers found that this gene is mutated and inactivated in 57 percent of
tumors from patients with sporadic, non-familial kidney cancer.

"The disease appears to fit the two-hit model for development of cancer, where
both copies of the critical gene are damaged or mutated," said co-investigator
Berton Zbar, M.D., chief of NCI's Laboratory of Immunobiology.  There are two
copies of every gene in most cells.  One normal copy of a gene is sufficient to
prevent development of cancer.  If both copies are damaged or mutated, (the
two-hit model) cancer may develop.

The researchers also found that the kidney cancer gene is affected early in the
development of the disease.  This finding is important, Dr. Linehan explained,
because its early presence makes it possible to consider development of
treatments to halt or reverse the progression of disease in its early stages.

The gene responsible for sporadic clear cell carcinoma in the same gene that
was identified last year as the cause of the inherited cancer syndrome called
von Hippel-Lindau (VHL) disease.  This research was conducted by Dr. Zbar and
Michael Lerman, M.D., Ph.D., of NCI's Laboratory of Immunobiology, in
collaboration with Dr. Linehan and colleagues.  People who have VHL disease are
predisposed to develop multiple tumors, including cancers of the kidney, eye,
brain, spinal cord, and adrenal glands.  Isolation of the VHL gene is now
leading to improved identification of carriers of the gene in affected families
to better manage care.

NKCA Involvement

Last fall, after the VHL gene was identified, Kidney Cancer News asked patients
to participate in the NCI's research.  All patients and family members of
patients were asked to contact the NKCA if:  (a) more than one person in the
family had kidney cancer, including deceased family members; or (b) if at least
one member of the family had kidney cancer and at least one other family member
had contracted or died of tumors of the eye, brain, spinal cord, ear, or
adrenal gland.

Over 20 families contacted the NKCA.  Each family was asked to provide some
medical information which the NKCA passed on to Dr. Linehan at the NCI.
Afterwards, he sent the NKCA the following note via electronic mail:

"The family information you sent has been a home run.  This morning we already
identified one family with three affected members and another with four."
Marston

Sometimes, a small amount of assistance at a critical time can lead to
important progress in research.  Dr. Linehan has again asked the NKCA for
assistance in identifying families with kidney cancer.

Patients Wanted

Kidney cancer patients or family members of patients should call the
Association at 708-332-1051.  The NKCA will provide a family medical history
questionnaire and information on participating in the NCI's research.  The NKCA
will also provide free information on kidney cancer and the genetics of kidney
cancer to callers.

When the questionnaires are returned to the NKCA, they are screened and sent to
the NCI in batches.  This saves the NCI the effort of dealing with individual
callers and NCI scientists are not tied up answering questions about kidney
cancer.  Once the questionnaires are received by the NCI, Dr. Linehan will call
those patients and families which seem to be most relevant to the research.

If a detailed study is made of a particular family, it usually sufficient for
every family member to get a simple blood test from his or her local doctor.
The blood is shipped to the appropriate NCI laboratory by the doctor.  (Due to
AIDS and other blood born diseases, it is important for all blood samples to be
properly packed and handled.)

Families which have the familial kidney cancer gene should be extra vigilant so
any new cases are diagnosed and treated early.  Besides helping themselves,
families which agree to participate in research will be helping scientists
develop diagnostic tests and new treatments for all forms of kidney cancer.

Nurses, Social Workers and Doctors Can Help Too

If you know any kidney cancer patients, call them and give them the National
Kidney Cancer Association phone number, 708-332-1051.  Urge them to contact the
NKCA.  You'll be performing a valuable service.  Supporting research.  Helping
patients get more information about their disease.  Perhaps even saving a life.

Health Care Reform Debate Intensifies

By Congressman John Porter of Illinois
Reprinted with Permission from Congressman Porter's Washington Report,
Vol. XV, No. 2, Spring 1994

The real work of health care reform legislation has begun in earnest.  At the
heart of this debate is one central question:  how much government do we want?

We should begin this discussion by remembering that America has the best health
care in the world.  We have the best facilities, the best medical personnel,
the highest technology, the most advanced biomedical research.  That's why
people from all over the world come here seeking treatment.

Of course, ours is the best system only for those who are fully in it.  Some
reform is clearly needed to provide health care access to those who are
excluded from coverage--including people dropped from or denied coverage
because of a pre-existing condition or a catastrophic illness.  But the Clinton
Plan, a 1,342-page prescription based on heavy government regulation and
control, is far too intrusive.

The difficulty is that while achieving health care reform requires finding
common ground, the President seems intent on an all-or-nothing strategy.  This
was evident in the ultimatum he issued during the State of the Union Address--
he would accept nothing short of universal coverage.

We might work toward universal coverage as an eventual goal, but requiring it
immediately means imposing a government mandate on businesses or individuals to
purchase it.  By making universal coverage a non-negotiable item, the President
is taking off the table a critical issue in this debate and preventing real
progress.

Rather than universal coverage, our objective should be ensuring universal
access to health care--e.g. no one should be denied coverage because of a pre-
existing condition or job change.  We should repeal Medicaid and use this money
to provide subsidies to the poor to obtain coverage.  We should not, however,
mandate that business pay for coverage for every employee.  In our fragile
economy, such a mandate would kill job creation.

The mandatory health alliances envisioned by the Clinton Plan are not only
purchasing cooperatives, but regulatory agencies.  In all, the Clinton proposal
would create an astounding 50,000 new positions, including a National Health
Board with sweeping powers to determine available services and to set prices.
This is unwarranted.  Purchasing alliances should be voluntary and have no
regulatory authority.

To control costs, the basic plan to which everyone should have access should
start with a modest package of services--for example, a plan similar to
standard option Blue Cross/Blue Shield, adding preventive and catastrophic care
(additional coverage could be purchased by individuals if they desire).  If we
go too far beyond this, we will end up with a plan that is far too expensive.
Let me be clear:  we can have the most extensive health care imaginable, as
long as we're willing to pay for it.  But to avoid adding to our already huge
deficits, we must not adopt any plan without adequate financing.

In addition, medical liability is adding to our nation's health care bills tens
of billions of dollars in wasteful defensive medicine--needless procedures
ordered by physicians to protect against potential lawsuits.  We must address
this problem by placing a reasonable cap on non-economic losses ("pain and
suffering") that will allow insurers to accurately rate these risks and hold
down premiums.  Other needed reforms include elimination of joint and several
liability and alternative resolution of liability claims.  Anti-trust reforms
are also important.  For example, hospitals should be allowed to enter into
agreements to share expensive equipment in a community, and physicians, within
certain limits, should be permitted to organize and negotiate with health care
provider networks.

Most importantly, we must retain private sector competition in any plan.  I've
said it before and it bears repeating:  after witnessing the collapse of the
former Soviet Union and other socialist systems, it is incredible that anyone
believes we would be better served by a health care system run or organized by
government.

The genius of America is private enterprise.  Choice, availability and quality-
-what Americans want from their health care system--are best ensured through
the marketplace.

The President should understand this and see the handwriting on the wall:  his
plan will not be adopted.  He should take this opportunity to bring us together
and support minimal yet essential reforms necessary to make the world's best
health care system more affordable and accessible to all Americans.

Drug Access and Emergency IND's

The Food and Drug Administration (FDA) is charged with regulating food,
therapeutics (pharmaceuticals and biologics), vaccines, diagnostic kits, blood
supplies, and medical devices.  Access to new drugs and therapies is controlled
by the FDA through clinical trials leading to formal marketing approval.

For several years, patients with life threatening diseases have pushed the U.S.
Food and Drug Administration to provide greater access to new treatments.  The
National Kidney Cancer Association has been a strong advocate of expedited FDA
procedures for new cancer treatments and easier patient access to new
therapies.

During the last half of 1993, the FDA started to respond to the needs of cancer
patients.  It created two new full time staff positions in the Office of AIDS
and Special Health Issues.  These positions were filled by Patty Delaney and
Ellen Cutler, well qualified FDA employees with prior experience in oncology
and public communications.  Their assignment is to provide better liaison
between the FDA and cancer organizations as well as patients, and to coordinate
cancer related activities with the FDA and Public Health Service agencies.

Approved Drugs and Clinical Trials

Typically, cancer patients are treated with approved therapies or with an
Investigational New Drug through a clinical trial.  In kidney cancer,
interleukin-2 is the only FDA approved treatment for advanced metastatic
disease.  It is marketed by Chiron Corporation under the tradename Proleukin.
Some kidney cancer patients are also treated with drugs which are FDA approved
for diseases other than kidney cancer.  The most common drug of this type is
interferon-alpha which is made by several companies, most notably Schering
under the tradename Intron-A.

Many patients are also treated with Investigational New Drugs and therapies
through clinical trials.  It is not uncommon to see kidney cancer patients in
trials treated with IL-4, IL-6, gamma interferon, GM-CSF, and other agents.
Before these products enter human clinical trials, they have been researched
with laboratory tests and in animals.  They have not yet been approved by the
FDA for general marketing, however, they have demonstrated enough efficacy and
safety to justify warrant use in clinical trials.

Emergency IND's

Patients who are not eligible for clinical trials may be eligible to receive
drugs and therapies which are not yet FDA approved by having their doctor apply
to the FDA for an Emergency IND (Investigational New Drug).  Many patients and
physicians may not be familiar with the process of applying for an Emergency
IND.  But it really is quite simple and may be extremely useful in the care of
patients.

Who?

An Emergency IND starts with a single patient and a single doctor.  The doctor
makes a request to the FDA for an Emergency IND and proposes a course of
treatment.  If the Emergency IND is granted, it authorizes the doctor to treat
the patient with a drug or therapy which is not yet FDA approved.  In the case
of a cancer drug or therapy, the physician can contact Ellen Cutler at the FDA
by calling 301-443-0104.  The physician will be directed to the appropriate
division within the FDA where the request for an Emergency IND will be
reviewed.

When?

Seeking an Emergency IND is especially appropriate under the following
circumstances:  (1) when the patient is no longer responding to approved
therapies and no other adequate treatment is available; (2) when a patient is
not eligible for a clinical trial but would be an appropriate candidate for
treatment with a drug that is being tested in a clinical trial; (3) when the
most appropriate drug or therapy for the patient is not currently available
through a clinical trial (i.e. a drug that was tested in a clinical trial but
is no longer available); and (4) when a promising drug has been through pre-
clinical testing but has not yet entered into human clinical trials.

One or more of these conditions may indicate that it is appropriate to seek an
Emergency IND for a patient.  In making a request, it is always important (1)
to state those conditions which support the request and (2) to submit
information which supports the proposed course of treatment.  For example, a
doctor can submit a medical journal article on the drug and protocol.  For a
drug or therapy not yet tested clinically, a doctor must submit pre-clinical
animal research data, toxicity data and manufacturing information assuring the
identity, quality, purity, and strength of the drug (e.g. a certificate of
analysis).

What is Submitted?

The physician needs to submit information to the FDA review division so the
agency can make an informed decision and assess the appropriateness of the
request.  This information includes:

(1) The name of the drug and the indication for which it is being sought.  A
statement regarding the supplier of the drug (source of the drug, such as the
drug company which makes it or the NCI which distributes it.)

(2) The medical history of the patient for which the drug or therapy is
intended.  This history should include information on any prior therapy and
response, and the rationale for the proposed treatment.  The rationale may be
strengthened by including any research information on the patient's tumor, such
as results of an in vitro drug test with the proposed agent on the patient's
tumor.

(3) The protocol detailing the proposed treatment plan (dose, route of
administration, and duration).  The doctor should also include procedures for
monitoring the patient and planned modifications in the event of intolerable
toxicity or unforeseen side effects.

(4) A statement that informed consent will be obtained prior to initiating
treatment.  The application should include a copy of the consent form which has
been drafted for the patient's signature, or signed by the patient.

(5)  A statement of the physician's qualifications as an investigator should be
included.  A copy of his or her curriculum vita is sufficient.

(6) If the physician has obtained approval of the IRB (Institutional Review
Board) at his institution, a statement to that effect should be included with
the application.  At the very least, a statement should be included that the
IRB will be advised of the treatment within five days.

The completed application for an Emergency IND can be FAXed to the appropriate
FDA division.

FDA Response

The FDA recognizes that a request for an Emergency IND is urgent and the agency
will try to respond as quickly as possible.  Typical response time is 24 to 48
hours, and same day approvals are not uncommon, particularly when the request
is well documented and complete.

If the FDA approves the application, response is made by letter acknowledging
the issuance of the IND number with instructions on how to complete the
process.  Someone from the FDA will call the physician directly and will
verbally provide the IND number.

Physician Response

The physician then calls the supplier of the drug or therapy, provides the IND
number, and the drug is shipped, usually by express shipping service.  The
physician then treats the patient according to his plan.

As part of the process, upon receipt of the FDA letter, the physician is
required to fill out FDA Form 1571 and provide a complete copy of the original
correspondence.  When treatment is completed, the physician should submit the
results of treatment to the FDA and request withdrawal of the IND.

In some cases, when treatment is extended, the physician is required to submit
an annual report on the patient's progress.  Some suppliers also request a
follow up report in return for providing the drug.

Regulatory Restraints

Clinical investigators' responsibilities when using investigational drugs are
well defined by FDA regulations (21 CFR Part 312), including:  (1) use of the
drug or therapy only in accordance with the treatment plan submitted; (2) use
of the drug or therapy only in the named patient under the doctor's personal
supervision or under the supervision of other investigators who are responsible
to him or her; (3) assurance that an IRB reviews or approves the study; and (4)
obtaining proper informed consent from the patient or from his or her legal
representative.

Under the regulations, the physician is precluded from:  (1) giving the drug to
another physician not responsible to him or her, (2) giving the drug to
patients other than the one named in the application, and (3) giving the drug
to a physician in another institution for use on his or her patients.

Limitations

The Emergency IND is a very special application because its relates to an
individual patient who is confronting an immediate medical crisis.  Hopefully,
treatment under the Emergency IND will be successful.

However, even if the IND is granted and treatment proves successful, the
company producing the drug or therapy may not find the results useful.  When a
physician provides a new drug or therapy in an emergency situation, the
protocol may be tailored to the specific patient.  It may not be applicable to
the overall population of patients with the same disease.

Other Benefits

When used appropriately, the Emergency IND can benefit patients and advance the
development of new drugs and therapies, particularly if information from the
Emergency IND makes its way into the scientific community as well as back to
companies and the FDA.

Patients, physicians, researchers, companies, and the FDA itself should give
careful consideration to using Emergency IND's.  These applications are useful
tools which can help individual patients and provide insights which help all
patients.

Get In Touch

For more information, contact:  Ellen Cutler, Food and Drug Administration HF-
12, Parklawn Building Room 12A40, 5600 Fishers Lane, Rockville, MD 20857, 301-
443-0104, FAX [phone].

Clinical Trials on NKCA BBS

The NKCA computer bulletin board system (BBS) now has all currently active
clinical trials online which are listed in PDQ.  Three file areas have been
established for clinical trials in renal carcinoma, transitional cell
carcinoma, and Wilms tumor.

The complete long description for each trial is available for viewing online or
downloading.  There is also a compressed file in each file area which contains
all clinical trials combined.  All files in these areas are updated monthly as
the National Cancer Institute updates its Physician Data Query (PDQ) system.

The NKCA BBS has grown.  It now has over 250 files of information on kidney
cancer and cancer in general.  Access to the NKCA BBS is free.  All patients,
physicians, family members, and others are welcome on the system.  It also
supports electronic mail to the NKCA and allows patients and physicians to
exchange messages.

The telephone number of the computer is 708-332-1052.  Baud rates to 14.4K, 8
data bits, 1 stop bit, no parity, no echo, full duplex, ANSI terminal
emulation.  The system is accessible with most communications software products
including Procomm on IBM-compatible PC's and MicroPhone on Apple computers.

Even if you don't own a computer, you may be able to access the system.  Do you
have a friend who has a computer?  Your doctor or your local public library may
also access the NKCA BBS for you.  If you have any technical problems or
questions, please call the NKCA office at 708-332-1051.  A special edition of
Kidney Cancer News will be issued shortly on the NKCA BBS.

Second Opinion

Patients often have problems getting proper reimbursement from their insurance
company.  If your insurance company rejects a claim, resubmit it.  A different
claims reviewer may put it through.  If the claim is still rejected, appeal the
claim.  Every insurance company has an appeals process.

To successfully appeal a rejected claim, do your homework.  Know the medical
reasons for the claim and for the care you received.  Provide your insurance
company with medical evidence supporting your claim, including medical journal
articles indicating the appropriateness of your treatment.  Send this
information to your insurance company's medical director along with your policy
number and other information about the claim.

Keep accurate records and copies of all medical bills and correspondence with
your insurance company.  If the company remains unresponsive, you can file a
complaint with your state insurance commission.

If the company remains unresponsive, you should contact your state bar
association and get the names of attorneys in your area who might help you sue
your insurance company.  Some attorneys may take your case on a contingency fee
basis, meaning they get paid only if they win for you.  If you cannot afford an
attorney, your state bar association can direct you to legal agencies which
help people in your situation.

Know your insurance policy.  Read it, and understand what is covered and what
is not covered.  Reimbursement depends heavily upon the language of your
policy.  If your health insurance is through an employer sponsored plan, ask
your employer for information on the plan.

If you are having problems getting reimbursement, tell your employer about your
problem.  Many companies have a personnel or human resources office which will
help you.  Companies pay big bucks for their health insurance programs.  No
reputable company wants their employees "stiffed" by their insurance carrier.
Your employer wouldn't accept bad product from a supplier.  Why should they
accept bad coverage from an insurance supplier?  Several members of the NKCA
have received reimbursement or pre-authorization for reimbursement when their
employers asked an insurance company to pay a claim.

Annual Convention Coming Up

July 22 and 23 are the dates for the NKCA Annual Convention of patients and
physicians.  Featured speakers include:  Dr. McClellan Walther, a member of the
National Cancer Institute team which identified the kidney cancer gene, will
talk on the genetics of kidney cancer.  Dr. Keith Block on nutrition as
adjuvant therapy.  Noted economist Eugene Lerner on financial strategies for
cancer families.  Dr. Ronald Bukowski on immune response in kidney cancer, and
many more.

This year's registration fee is $ 125 per person, and includes all sessions and
meals.  An invitation will be sent to all patients, family members, and
physicians involved in the Association.  If you want an invitation, simply call
the NKCA office at 708-332-1051.

Join the NKCA

Patients, family members, physicians and others involved in kidney cancer
should be members of the NKCA.  This Association is the only national
organization specifically dedicated to helping kidney cancer patients.

The Association is your voice in Washington, with insurance companies and
employers.  It also provides information to patients and physicians, and the
NKCA sponsors research on kidney cancer.

The suggested annual donation for membership is $ 125.  However, no one is
turned away.  Give what you can.  Become active in the Association.  What you
learn just might save a life, even your own.  Just look at what you have
learned in this edition of Kidney Cancer News!  The address is National Kidney
Cancer Association, 1234 Sherman Avenue, Evanston, IL  60202.

Do You Know a Kidney Cancer Patient?

If you know a kidney cancer patient, tell them about the Association and give
them the NKCA phone number, 708-332-1051.  They will receive free information
on kidney cancer, a free subscription to Kidney Cancer News, invitations to all
patient meetings, and other assistance.

If you are a kidney cancer patient, get your doctor to tell other patients
about the NKCA.  Also, call the Association and give us the complete name and
address of your doctor.  The NKCA will give him a free subscription to Kidney
Cancer News.  The larger and stronger the NKCA, the more it can do to help you.