Life Extension Magazine®

Issue: Dec 2011

Bureaucratic Assault On New Cancer Therapies

One American dies every hour from melanoma. Non-Hodgkin lymphoma claimed over 20,000 US lives in 2010. Yet FDA barriers are delaying approval of innovative approaches that could have saved these fellow human beings. While technological advances are routine in virtually all disciplines, clinical medicine is the exception. It has devolved into a bureaucratic monstrosity that suffocates innovation while rewarding the politically well connected. How much longer will Americans tolerate a system that is a proven failure?

By William Faloon.

William Faloon
William Faloon
Bureaucratic Assault On New Cancer Therapies

One American dies every hour from melanoma.1 Incidences of this deadly skin cancer are on the increase.

Although no cure exists for advanced melanoma, virtually 100% of its victims can be saved if the malignancy is caught early.2

The problem is that dermatologists cannot accurately diagnose a melanoma based on a visual examination alone. A biopsy is needed to definitely ascertain if a skin lesion is a melanoma.

Biopsies require expenditure of money and time, along with some minor trauma. Patients and dermatologists have to make decisions as to whether a particular skin lesion warrants a biopsy.

In a clinical trial involving 23 dermatologists around the US, a hand-held non-invasive scanning device called MelaFind® was tested on suspicious lesions. Its accuracy in detecting melanoma was 98%—which equaled or bested the top doctors in avoiding unnecessary biopsies.3

MelaFind® was submitted for FDA approval in June 2009. Despite clinical trial results documenting its unprecedented (98%) ability to detect early stage melanoma, the FDA refused to approve it and insisted on further review.4,5

FDA's Lethal Illogic

According to bureaucratic illogic, the FDA was concerned that non-dermatologists might use MelaFind® to screen for melanoma, even though the company promised to only sell it to dermatologists. Since the FDA cannot regulate doctors, the agency felt that the best way of keeping MelaFind® out of the hands of non-dermatologists was to not approve it at all.

The FDA expressed other concerns such as its misuse by dermatologists. This is rather bizarre since dermatologists are the most highly trained in our society to diagnose and treat skin diseases, yet the FDA did not want them to use this new device that was 98% accurate in diagnosing melanoma.6,7

The Real World of Medical Practice

The Real World of Medical Practice

FDA bureaucrats live in a cave when it comes to the intricacies and expenses involved in the real-world medical setting.

Hurried people too often ignore suspicious skin lesions until it's too late.

Patients often go to their primary care doctor when it comes to suspicious skin lesions. More enlightened individuals visit a dermatologist.

Either way, medical efficiency comes into play when deciding whether a suspicious lesion should be biopsied or merely kept an eye on. A dermatologist charges for each biopsy, and there is a separate expense for the pathology lab. Return visits are often needed. All this adds up to medical costs that MelaFind® could otherwise render far more efficient.

MelaFind® is not 100% accurate. That means it may miss a few (2%) suspicious lesions that a dermatologist would then have to decide warranted a biopsy nonetheless. The FDA is using this as another reason not to approve it. Using this logic, MRI and ultrasound testing would never have been approved because these are not 100% reliable diagnostic tools either.

If ever approved, MelaFind® will provide doctors with an efficient method to detect early stage melanoma. According to some dermatologists, it has the potential to "save many lives."8

There were 68,130 new cases of melanoma in 2010 and 8,700 deaths.9 These numbers show that melanoma can be easily cured—if caught in the early stages before it infiltrates and metastasizes.

We at Life Extension® envision a day when devices like MelaFind® will be used in large screening campaigns where early stage melanomas can be easily detected and removed. None of this will happen as long as the FDA is allowed to erect bureaucratic barriers that suppress this kind of medical innovation.

FDA Approves Expensive Melanoma Drug

Cynical individuals might question the timing of the FDA's denial of MelaFind®, a device that could spare thousands of agonizing deaths from metastatic melanoma.

Just a few weeks after saying no to MelaFind®, the FDA approved a new drug by pharmaceutical giant Bristol-Meyers Squibb to treat advanced melanoma trademarked Yervoy™.10

In a study involving 676 patients with advanced melanoma, those receiving Yervoy™ survived for 10 months compared to 6.4 months for those who did not receive it. Fourteen patients died from side effects caused by Yervoy™.11 The FDA hailed this as a breakthrough and granted approval for widely spread melanoma.

Shares of Bristol-Meyers Squibb surged as analysts predicted a $1.7 billion blockbuster.12 The reason so much money will be made is that it will cost each patient an astounding $120,000 for the four-course treatment.4 For what used to be the price of a nice home, one with terminal melanoma can buy an extra 108 days of life. The average cost per day of added life will be over $1,000—further exacerbating today's health care cost crisis!

The generic name for Yervoy™ is ipilimumab. If you ever wonder why you cannot pronounce the names of new compounds (like ipilimumab), it is because pharmaceutical companies intentionally create names that no one can readily comprehend. Pharmaceutical companies do this to make it harder for future generic drug makers to enter the market with lower-cost versions, since virtually no one can pronounce the generic name.

FDA Approves Expensive Melanoma Drug

The reason that Yervoy™ (ipilimumab) is expected to sell so well is that current FDA-approved therapies have not been shown to substantially extend survival, but the FDA allowed them to be used for decades anyway.

We are not against the FDA's approval of Yervoy™ as it may prove to work better when treating less advanced melanoma. What bothers us are the bureaucratic barriers. They are so cumbersome that few new therapies ever get approved. This enables companies to charge extortionist prices for the few that receive the FDA's coveted anointment.

FDA Fails to Approve Effective Lymphoma Drug

Each year about 65,000 Americans are diagnosed with non-Hodgkin lymphoma.13 It is estimated to have killed over 20,000 in the US in 2010.14 Jackie Kennedy Onassis died from non-Hodgkin lymphoma at the age of 64.15

Non-Hodgkin lymphoma is one of the few cancers where establishment medicine can brag about treatment breakthroughs. Over the past 50 years, survival rates have more than doubled.16 Often overlooked are long-term side effects like cumulative heart muscle damage that precludes long-term use of certain conventional treatments.17

Lymphoma patients who fail treatment with FDA-approved chemotherapy have a life expectancy measured in weeks or months.

A next-generation compound called pixantrone is designed to be less toxic and more effective than current anthracycline chemo drugs. It has successfully gone through phase I and phase II human clinical trials.

In 2004, a randomized phase III trial mandated by the FDA was initiated. Pixantrone was administered to non-Hodgkin lymphoma patients who had already failed conventional therapy. The control group received whatever their oncologist thought would work best for them.

In these extremely difficult-to-treat lymphoma patients, 20% of those receiving pixantrone showed a complete response, compared to only 6% receiving conventional care.18 A follow-up analysis of the data showed 24% of patients attaining complete response status as opposed to 7% in the standard group.19 These are unprecedented findings!

A complete response is not a cure, but it can buy a patient precious time in remission and the opportunity to identify potential curative therapies.

Despite almost four years of phase III clinical studies showing that pixantrone works three times better than what's available today, the FDA declined to approve it. In an FDA briefing as to why pixantrone was not approved, the FDA stated:

"The study was not stopped at a planned interim analysis and early study stopping invalidated the applicant's Special Protocol Assessment."20

The "Special Protocol Assessment" is an agreement between the FDA and a drug maker regarding how a clinical study should be done.21 It originally envisioned enrolling 320 patients over a 36-month time period. For various reasons, it took 45 months to recruit 140 patients.22 This is not unusual as some 60% of phase III studies do not meet patient recruitment objectives, but nonetheless generate statistically significant data that are used to approve a new drug.

Pixatrone Not Owned by Big Pharma

Unlike Bristol-Meyers Squibb, which had the wherewithal to fund a huge clinical study and whose stock soared in response to the FDA's approval of Yervoy™,23 the maker of pixantrone's auditors handed management a notice that the company (Cell Therapeutics) may not be able to continue as a going concern in response to the FDA's refusal to approve their drug.24,25

Cell Therapeutics' initial challenge in enrolling enough study subjects was convincing oncologists and hematologists that pixantrone might work. Patient incentive to participate was minimized because the FDA mandated that only 50% of the study subjects would receive the promising drug (pixantrone).

Despite these limitations, Cell Therapeutics believes it generated statistically significant data and has taken the unusual step of appealing the FDA's denial of pixantrone. The reason appeals are seldom filed is fear of FDA retaliation on future drug applications. In the case of Cell Therapeutics, which is not part of Big Pharma, it may have little to risk in asking for a common-sense review of the impressive data it generated in terminally ill lymphoma patients.

The inability of Cell Thera-peutics to adhere to the FDA's impossible-to-achieve dictates is an example of a federal agency that went out of its way to railroad a promising cancer therapy.

How Life Extension is Helping to Accelerate Melanoma Treatment

Scientists supported by the Life Extension Foundation® long ago discovered a novel method of treating advanced melanoma (stages 3 and 4).

In an FDA-approved clinical trial, a topical cream (called imiquimod) is applied to the exposed tumor twice a day for a total of six weeks. At weeks two and four, the doctors expose the area to an infrared laser. The topical imiquimod cream binds with receptors on cancer cells and stimulates them to activate proteins that "broadcast" the presence of the tumor cells to the immune system. In essence, the patient's own tumor cells become a unique anti-tumor vaccine.

The laser portion of the treatment is designed to hyper-activate the imiquimod with the objective of inducing a systemic immune response against metastatic melanoma cells. Imiquimod is an FDA-approved drug and this is an FDA-approved clinical trial. To inquire about participating, call Meghan Dubina, MD, at (312) 695-6786 or email [email protected] (This study is referenced by the ClinicalTrials.gov identifier: NCT00758797. Study details can be found on the FDA-approved clinical trials website: www.clinicaltrials.gov.)

You may not qualify to participate in this clinical study or may not want to be bothered with the bureaucratic red tape. Fortunately, this same protocol is being done in the Bahamas for melanoma, and a modified version is being studied to treat breast cancer. In order for this treatment to be administered, a tumor lesion must be present near the surface of your skin, such as a breast lump, a chest wall breast lesion, or a superficial melanoma tumor. To inquire about clinical programs being offered in the Bahamas, call the International Strategic Cancer Alliance (ISCA) at 610-628-3419 or visit www.is-canceralliance.com

The Real Issue…

The way this country tolerates FDA behavior, it is as if only large pharmaceutical companies are capable of discovering effective new drugs. Those without deep pockets are often shut out of today's Byzantine approval process, where it can cost over $100 million to have a new compound "approved" for sale.

Most troubling is what this is doing to medical innovation across the entire spectrum. We at Life Extension know of pioneering physicians who have discovered and are utilizing novel therapeutic protocols to treat the diseases of aging. Yet these inventions have virtually no chance of making it out of these private practices because of FDA overregulation.

To see how much more efficient an unregulated environment functions, look no further than the breakthroughs that have been made in the treatment of AIDS. This disease appeared in America around 1980. It took several years just to identify the HIV virus as the cause. In the first half of the 1980s, virtually everyone who contracted AIDS died within 1-2 years.

The difference was that AIDS activists were acutely aware that FDA-mandated randomized clinical trials were the roadblock to the discovery of effective therapies. Unlike cancer support groups who too often capitulate to FDA suppression, AIDS activists rebelled and forced the FDA to back down from restricting any therapy that might be effective.

Removed from the artificial constraints of controlled trials designed by uncaring and incompetent bureaucrats, front-line doctors and researchers were able to collect data from actual medical practice on AIDS patients and had the flexibility of trying whatever therapy might work. Life Extension® partnered with these groups early on and witnessed the miraculous results that occurred when doctors could prescribe therapies without regard to FDA dictates.

When Life Extension attempted to introduce this same strategy to dying cancer patients, the FDA stood in the way and said absolutely not!

Low CoQ10 Levels Associated with 790% Increased Risk of Melanoma Metastasis
Dermatologist removing mole
Dermatologist removing mole

In a study published in the Journal of the American Academy of Dermatology, plasma coenzyme Q10 levels were measured in 117 consecutive melanoma patients upon enrollment. One hundred twenty five matched volunteers without any clinically suspected pigmented lesions were utilized as the control group.

Researchers found that CoQ10 levels were significantly lower in melanoma patients compared to control subjects. Further, it was noted that for melanoma patients with CoQ10 blood levels of less than 0.6 mg per liter, the risk of developing metastatic disease increased by 790%, compared to those melanoma patients with blood levels of 0.6 mg per liter or higher. In addition, melanoma patients with higher blood levels had a metastasis-free interval that was almost double compared to patients with lower levels.29

Of the 82 patients with low CoQ10 levels, 17 died during the study, compared to none of the 35 patients with higher CoQ10. CoQ10 levels did not vary by sex.29

Levels of CoQ10 correlated well with tumor thickness, which is currently the best indicator of melanoma progression. Specifically, lower CoQ10 levels correlated with increased tumor thickness and poorer prognosis.29

The study notes that abnormally low plasma levels of CoQ10 previously have been known in patients with cancer of the breast, lung, and pancreas. This study may be the first to indicate that lower blood levels of CoQ10 can have an extremely adverse effect. The lead author of the study concluded that analysis of their findings suggested baseline CoQ10 levels are a powerful and independent prognostic factor that can be used to estimate risk for melanoma progression.

Statin drugs are known to lower CoQ10 levels. Will we find that melanoma progression is another side effect of statins? If so, this side effect can be readily overcome with CoQ10 supplements.

Look at the Difference between AIDS and Cancer

Those afflicted with AIDS today are prescribed an armamentarium of medications and take huge quantities of dietary supplements to keep their infections under control. What used to be a near-certain death sentence has turned into a manageable chronic disease for most people. That happened more than a decade ago!

Contrast this with cancer, where a melanoma drug that gives patients an extra 108 days of life (that costs $120, 00010) is hailed as a breakthrough in 2011. Americans have been dying of melanoma for hundreds of years.

The FDA's approval of expensive and mediocre drugs like Yervoy™ and suppression of common-sense approaches (like MelaFind® and pixantrone) are stark examples of the FDA's bureaucratic assault on novel cancer therapies.

At the June 2011 conference of the American Society of Clinical Oncology (ASCO), the results from several human studies were announced about new compounds that prolong the lives of advanced melanoma patients.26 Despite an unusual amount of enthusiasm shown by oncology researchers, it may take years before the FDA will allow combinations of these compounds to be used in desperately ill melanoma patients… who are dying at the rate of one each hour.

One of the new targeted melanoma drugs featured at the June ASCO meeting is called vemurafenib and is being developed by Roche Holding AG and Daiichi Sankyo's Plexxikon unit.27 It inhibits a mutated form of a gene called BRAF found in more than half of patients with advanced melanoma.27 It has virtually no benefit on patients with a normal version of the gene.

Results from a 675-patient trial showed that those taking vemurafenib were 63% less likely to die over a six-month period compared to those taking chemotherapy called dacarbazine.28 The median time before the disease progressed for patients on vemurafenib was 5.3 months compared with 1.6 months on dacarbazine chemotherapy.

Based on this trial, we believe that melanoma patients with a mutated BRAF gene should have been allowed immediate access to vemurafenib if they were willing to sign a disclaimer acknowledging that it is not yet FDA-approved. Instead, thousands of melanoma patients are dying prematurely in the FDA's waiting room.

My Grandmother's Funeral

At age 13, I stood over the casket of my grandmother, who had died a horrific death from melanoma. She was only 54 and suffered terribly as metastatic lesions invaded every part of her body.

Her death was preventable, as she ignored a melanoma lesion on her leg for many years.

At that funeral in 1968, no one would have predicted that more Americans than ever would be dying of melanoma in 2011—43 years later! Like others back then, our family believed that medicine would advance and find a cure for cancer, just like antibiotics wiped out most bacterial infections.

While major technological advances are routine in virtually all disciplines, clinical medicine is the exception. It has devolved into a bureaucratic monstrosity that suffocates innovation while rewarding the politically well-connected.

How much longer will Americans tolerate a system that is a proven failure?

As a member of the Life Extension Foundation®, you help support our ongoing campaign to educate the public that most deaths today could be avoided if it were not for the bureaucratic assault on novel therapies erected by our own government.

For longer life,

For Longer Life

William Faloon

As this article was being finalized… FDA Partially Capitulates on MelaFind®

In response to intense legal and political pressure put on the FDA, a limited conditional approval has just been granted for the MelaFind® skin cancer detection device.30

This pending approval comes after a seven-year battle between the company that makes MelaFind® and the FDA.

After FDA rejected MelaFind® last year, the company filed a citizen's petition with FDA Commissioner Margaret Hamburg seeking to overturn the denial.

The House of Representatives held a hearing in the summer of 2011 where the FDA's top device regulator acknowledged the agency mishandled the MelaFind® application. The error occurred when the FDA denied approval of MelaFind® before it held a meeting of its own scientific advisors—talk about bureaucratic mix-up!

Pharmocracy

This does not mean that MelaFind® will definitely become available, but the FDA is at least moving off its refusal to approve it at all. MelaFind® did win approval in early September in 27 European nations.

In order for MelaFind® to be approved in the US, the FDA needs to agree on the device's final labeling, a user guide, details of a training program for doctors, and the design of a post-approval clinical trial.

The CEO of the company that makes MelaFind® was uncertain about when the FDA would approve MelaFind® and was careful to downplay if and when the company can begin selling the device. He acknowledged that discussions with the FDA were still going "back and forth" and therefore not complete.

Before MelaFind® can be sold in the US, the FDA wants additional "beta tests" with doctors to be conducted to make technical and usability improvements to the device. The FDA insists that MelaFind®'s label be longer and more complicated than the company ever envisioned.

Until these issues are resolved, MelaFind® will not be allowed on the American market.

There are examples of other products in the past that received this kind of conditional FDA approval but never made it to the market, though it seems the political heat has forced the FDA in a direction regarding MelaFind® that it previously refused to consider.

If you ever wonder why medical advances take so long and then cost so much, the expense and delay in pushing MelaFind® through the FDA's cumbersome bureaucracy provides a stark example.


References

1. Available at: http://www.melanomafoundation.org/facts/statistics.htm. Accessed May 18, 2011.

2. Available at: http://www.projectmelanoma.com/a-national-challenge.php. Accessed May 18, 2011.

3. Monheit G, Cognetta AB, Ferris L, et al. The performance of MelaFind: a prospective multicenter study. Arch Dermatol. 2011 Feb;147(2):188-94.

4. Available at: http://www.marketwire.com/press-release/MELA-Sciences-Announces-FDA-Panel-Review-MelaFind-PMA-
Application-on-August-26-2010-NASDAQ-MELA-1266362.htm. Accessed May 18, 2011.

5. Available at: http://www.thestreet.com/story/10944267/3/biotech-calendar-fda-drug-approvals-in-2011.html. Accessed May 18, 2011.

6. Available at: http://www.medpagetoday.com/Dermatology/SkinCancer/23448. Accessed May 19, 2011.

7. Available at: http://www.modernmedicine.com/modernmedicine/Modern+Medicine+Now/MelaFind-device-raises-
concerns-among-some-dermato/ArticleStandard/Article/detail/706390. Accessed May 19, 2011.

8. Available at: http://online.wsj.com/article/SB10001424052748704559904576230562290013904.html. Accessed May 20, 2011.

9. Available at: http://seer.cancer.gov/statfacts/html/melan.html. Accessed May 20, 2011.

10. Available at: http://www.bloomberg.com/news/2011-03-25/bristol-myers-squibb-wins-u-s-fda-approval-for-new-
melanoma-medicine.html. Accessed May 20, 2011.

11. Hodi FS, O'Day SJ, McDermott DF, et al. Improved survival with ipilimumab in patients with metastatic melanoma. N Engl J Med. 2010 Aug 19;363(8):711-23.

12. Available at: http://www.mmm-online.com/fda-approves-bms-skin-cancer-biologic-yervoy/article/199258/. Accessed May 21, 2011.

13. Available at: http://www.non-hodgkins-lymphoma-cancer.org/news/non-hodgkins-news0026.htm. Accessed May 20, 2011.

14. Available at: http://seer.cancer.gov/statfacts/html/nhl.html. Accessed May 23, 2011.

15. Available at: http://www.nytimes.com/learning/general/onthisday/bday/0728.html Accessed May 23, 2011.

16. Available at: http://www.lls.org/diseaseinformation/getinformationsupport/factsstatistics/nonhodgkinlymphoma/. Accessed May 23, 2011.

17. Available at: http://www.drugs.com/health-guide/non-hodgkin-lymphoma.html. Accessed May 24, 2011.

18. Available at: http://www.drugs.com/clinical_trials/cell-therapeutics-pixantrone-phase-iii-extend-pivotal-trial-successful-achieving-
primary-endpoint-6168.html. Accessed May 24, 2011.

19. Available at: http://www.fda.gov/downloads/advisorycommittees/committeesmeetingmaterials/drugs/
oncologicdrugsadvisorycommittee/ucm199560.pdf. Accessed May 25, 2011.

20. Available at: http://online.wsj.com/article/SB10001424052748703766704576009512990553104.html. Accessed May 26, 2011.

21. Available at: http://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/
OncologicDrugsAdvisoryCommittee/UCM204335.pdf. Accessed May 26, 2011.

22. Available at: http://www.pharmalot.com/2010/02/the-fda-and-special-protocal-assessments/. Accessed May 26, 2011.

23. Available at: http://www.bloomberg.com/news/2011-03-25/bristol-myers-squibb-wins-u-s-fda-approval-for-new-
melanoma-medicine.html. Accessed May 30, 2011.

24. Available at: http://seekingalpha.com/article/198401-fda-s-early-verdict-on-cell-therapeutics-pixuvri-comes-as-no-surprise. Accessed May 30, 2011.

25. Available at: http://www.ipdatadepot.com/archives/ipp070402.pdf. (pg. 13/45). Accessed May 30, 2011.

26. Available at: http://online.wsj.com/article/SB10001424052702304432304576367802580935000.html?
mod=WSJ_hp_MIDDLENexttoWhatsNewsTop. Accessed May 31, 2011.

27. Available at: http://www.bloomberg.com/news/2011-05-25/roche-leads-deadly-skin-cancer-turnaround-as-dozen-
drugs-coming.html. Accessed May 31, 2011.

28. Chapman PB, Hauschild A, Robert C, et al. Improved survival with vemurafenib in melanoma with BRAF V600E mutation. N Engl J Med. 2011 Jun 10 30;364(26);2507-16.

29. Rusciani L, Proietti I, Rusciani A, et al. Low plasma coenzyme Q10 levels as an independent prognostic factor for melanoma progression. J Am Acad Dermatol. Feb 2006;54(2):234-41.

30. Burton TJ. New tool in skin-cancer fight. FDA reversal clears path for a device that helps doctors diagnose melanoma. Wall Street Journal. September 26, 2011.

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