Glenna Crooks

By Glenna Crooks. Rittenhouse Square in Philly, a holiday weekend and great weather made for the perfect place for light reading this weekend. I got magazines with the intention of doing just that – and did. It was great to be outside on warm, breezy days.

However, my mood soured about half way into Spirituality&Health, reading an article about a possible new cancer therapy.

It describes the observations of Mamdooh Ghoneum, PhD: cancer cells are attracted to, ‘eat’ heat-killed baker’s yeast and then die. That’s good news. It happens in labs and in mice, who apparently suffer no side effects. That’s good news, too. Approval for testing in other animals is pending. I hope he gets it. We need progress in the healing of people with cancer.

Why the sour mood? Dr. Ghoneum is hero enough for making the observation and following through with studies. He’ll be all the more heroic if he continues and learns from whatever comes next.

The article does not stop with the ‘scientist vs disease’ heroic tale, however. It goes further to paint an unfortunate and ill-informed, biased contrast – between an altruistic pioneer using his personal savings and fund raising efforts to find a cure vs a greedy, patent-dependent industry hungry to return to shareholders the $800M – $2B quoted as the cost of developing a medicine. It asserts that industry would never develop a ‘natural’ product, saying: “Nowadays, a cancer cure that is all natural, non-toxic, simple to administer and inexpensive to produce has become an economic non-starter.”

Drug Development and Approval. Dr. Ghoneum is currently at the drug ‘research’ phase. This is the easy part. Get past animal research and the ‘development’ phase begins. Move into humans and the costs pile up fast – so do the failures. He’ll face institutional review boards, numerous negotiations with FDA over appropriate surrogate markers and end points, challenges of getting patients into clinical trials, careful management of clinical research sites and exquisite documentation at every stage. This is the phase that washes out all but 1-2 of every 10,000 drugs discovered.

Add to that, this is a yeast product – a biological – which means he’ll have to satisfy FDA that there is ‘batch to batch’ consistency in the product as he scales up for clinical studies and, if the product succeeds, eventual market use.

Since he intends to develop a treatment safe, effective and affordable enough for poor people around the world, he will also face regulatory requirements, and perhaps clinical trials, in those nations as well.  

Patents. As for the product itself, natural products are not patentable, but under terms of the Orphan Drug Act, they can be granted market exclusivity for a period of seven years; I’m no expert on patents, but that is just as good.

Shaman Pharmaceuticals was a company devoted to doing just that – with intentions to return a portion of its profits to the people of the rain forests from which their products emerged. Unfortunately and as a testament to the challenges of development, after 10 years and $90M, their studies failed to pass muster at FDA. It wasn’t the intellectual property protection that got in their way; it was running out of money before they could finish the developmental requirements to satisfy FDA.

If his product works, he gets market exclusivity here in the US, then the sales of the product here can help support the costs of filing for regulatory approval in other countries.

Going it alone. It’s a shame he thinks he needs to. In ‘doing it the old fashioned way,’ he intends to use personal savings and fund raising to cover the costs and currently needs $300,000 to complete animal studies.

Of the big bucks to get a drug to patients, the biggest (bucks – and headaches) are yet to come. He needs help from an organization that has successfully accomplished what he is attempting and there are, frankly, lots of them. With 861 cancer medicines and vaccines under development are a large number of companies globally who might be interested in his approach and capable of advancing his cause more quickly. Every company is interested in new ideas from investigators.

Yes, big companies are interested in natural products: Merck has an agreement with the National Biodiversity Institute in Costa Rica, for example, paying up-front fees to have access to samples of plants and soil with the intention of studying their usefulness as therapies. If any approved therapies result, Merck will then pay royalties to Costa Rica on all sales worldwide.

If he’s wary of relationships with companies, several government agencies are available to help. The National Institutes of Health has three: the National Center for Complimentary and Alternative Medicine would be interested from the natural product perspective, the Office of Rare Disease Research from the market exclusivity offered natural products in the Orphan Drug Act (most cancers are rare disease as defined in the Act) and the National Cancer Institute, which screens compounds for anti-cancer activity. Since his product works against breast cancer cells, he can also turn to the Department of Defense, which has a breast cancer research program and rather substantial funding.

In addition, the FDA’s Office of Orphan Product Development not only provides technical assistance, but also funding and hand-holding through the rigorous FDA approval process.

Then what? Getting a product approved for use is sometimes the easiest step. After that, there are more hurdles. In the US, Oncologists will want to see published papers and results, as will those professional societies who develop treatment algorithms, health insurance companies and manage care formulary committees. Some of those groups will want not just safety and effectiveness demonstrations, but also cost-effectiveness and comparative effectiveness as well.

Helping the world. Dr. Ghoneum wants to help poor people around the world with an affordable medicine.  I hope he’ll reach out to find a partner with the skills and resources to make it happen.  If he does that, in my book he’ll be a real hero.

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