January 1, 1970 (Vol. , No. )

Zachary N. N. Russ Bioengineering graduate student UC Berkeley

Navigating the Legal and Psychological Minefields of Developing an Entirely New Class of Vaccines

“It is courage based on confidence, not daring, and it is confidence based on experience.” – Dr. Jonas Salk, developer of the first safe and effective polio vaccine.

If you are reading this, you are no doubt aware that you and I and untold millions owe our health—if not our lives—to the successful application of numerous vaccines and their creators. Take measles, for just one example: The number of new cases in the U.S. dropped from more than 760,000 in 1958 to fewer than 150 annually during the period 2000–2007, and in the process, the number of deaths per year dropped from 450 to zero. Virtually all the cases of measles that did crop up were among the unvaccinated.

Polio, whooping cough, smallpox, and several other virulent diseases are all but eliminated in the developed world because of vaccines. These vaccines, which must be administered early in life for maximum utility, all have one serious side effect: hysteria.

In Vaccines We Trust

Having never experienced a neighbor’s child who comes down with measles and a few years later succumbs to the neurodegenerative complications of the disease, some parents have less appreciation for the vital protection provided by the routine application of vaccines. There is a disconnect between doing P to prevent Q—such a recommendation is backed only by the authority of the physician, the school board, and the scientific literature.

Mistrust of conventional medicine, pharma, or government agencies can lead to rejection of vaccination even in the face of overwhelming evidence. Indeed, that has been the case across the world, from Nigeria, where claims of vaccines made by the federal government to sterilize and kill the population have severely hampered polio eradication efforts, to the U.S., where mistrust of conventional medicine leads people to choose measles over vaccination on the word of celebrities and pseudoscientists.

It really is a question of trust—without an understanding of the science behind vaccines, you’re left with someone being told to dose their children for a disease they do not have. Being that the only exposure to vaccines many people have is childhood vaccination, we have a rare event with no basis of experience to buffer it from association with whatever happens afterward. So, if a recently vaccinated child becomes sick, one would be more likely to associate the sickness with the vaccine rather than their playmates who have been passing around colds.

Fear wasn’t always in charge. The “courage” that Dr. Salk is referring to in the above quote is his decision to test his vaccine on himself and his family, knowing that he could not in good conscience ask other families to do the same if he had not. The parents of over a million school children, the 1954 “Polio Pioneers,” followed his lead, volunteering their own families to receive the experimental vaccine to prevent the dreaded polio disease. Even today, many parents embrace vernacular vaccination by holding “chicken pox parties” that, by embracing the usually mild disease in infancy, usually prevent more significant complications of the disease at an older age.

So why revisit the topic now? The profile of vaccine medicine is dramatically changing. The previously cited vaccines are prophylactic. Now, a broad new class of vaccine—the therapeutic vaccine, which is given to treat an existing disease—is about to transform medicine, with ramifications perhaps as dramatic as the prophylactic vaccines have had.

We stand at the threshold of a new era in medicine. Therapeutic vaccines, previously rare, will soon explode, with many new dramatic avenues for treating serious illnesses. This could be a new golden era for medicinal advancement.

But this glowing vision faces a potential impediment that may stall progress in its tracks. In its current term, the Supreme Court is considering a case that will decide the constitutionality of the NCVIA’s no-fault vaccine compensation program. If the Supremes decide in favor of the plaintiffs, we could very well see a storm of lawsuits against a number of childhood vaccine manufacturers, with ominous implications for the introduction of new therapeutic vaccines, setting back this promising field of medicine for years if not decades.

Controlling the (Previously) Uncontrollable

But for a moment, let’s go back to the future. The field of therapeutic vaccines—such as cancer vaccines—is reaching maturity. Indeed, the provocatively named “Provenge” vaccine has just been approved by the FDA, and several other autologous, personalized treatments as well as nonautologous cancer vaccines are in Phase II and III trials.

These vaccines use innovative approaches that retrain the immune system to better attack cancerous cells by targeting certain receptors. Still other vaccines will treat existing infections, such as one that has shown promise in reducing chronic obstructive pulmonary disease exacerbations by encouraging T cells to recruit neutrophils to the airways and consume resident pathogens.

Another set of vaccines will treat addiction by encouraging the immune system to trap drugs with antibodies before the drugs can reach their receptors—these include TA-CD for cocaine and NicVAX for nicotine.

In a remarkable flash of brilliance, another set of vaccines are actually inverse-vaccines—vaccines aimed at decreasing immune response to a particular antigen. Perhaps these vaccines would be better described as immune modulators. These include vaccines to treat multiple sclerosis (Bayhill Therapeutics’ BHT-3009), diabetes (Diamyd’s drug called Diamyd®), and allergies (Cytos Biotechnology’s CYT003-QbG10).

As protein and genetic engineering capabilities grow so too will the ability to control or fine-tune the immune system and use innovative means to treat and prevent disease. All these vaccines show a number of advantages over other chemical methods, namely, longer periods between doses and fewer side effects.

It’s good to be in control. Tuning the immune system to better do its job of protecting us from environmental threats while not destroying us in the process is certainly a good thing.

What’s Not to Like?

Cool, so vaccines are about to go therapeutic in a very big way, very soon. What does that have to do with childhood vaccines? What’s the connection with the current SCOTUS case regarding the 1986 National Childhood Vaccine Injury Act (NCVIA)?

The NCVIA was passed in response to lawsuits that bankrupted several vaccine manufacturers. The collapse of these companies and their subsequent exit from the market resulted in serious shortages and rationing of vaccines, with a potentially devastating impact on the health of American children and an incalculable cost to society.

Legislation was introduced to protect the vital work of the remaining childhood vaccine manufacturers and healthcare providers while also addressing (and compensating) the obvious distress of parents of children with severe medical conditions that may (or may not) have been caused by said vaccines.

Practicing Safe Selectivity

No one has suggested that therapeutic vaccines will need NCVIA-type protection. Indeed, the rabies vaccine has survived for decades without such protection. A vaccine by patients who are already sick with a serious disease has a smaller patient population and is far less likely to be randomly associated with some other coincidental event.

Some interesting questions are raised, though: Will we see parents who use vaccines to treat their diseases but refuse vaccination for their children? If the NCVIA is (partially) invalidated, will the antivaccination movement take off, and if that happens will it affect the market reception of these therapeutic vaccines as well? They are, after all, intimately linked in both mechanism and name.

Alternatively, will the wider range of therapeutic vaccines actually erode resistance to childhood vaccination, as vaccine experiences become more commonplace with parents finding treatment for their own maladies in vaccines?

Clearly, we need continued NCVIA-level protection (or better) for childhood vaccine-makers whatever the Supremes decide in the case before it. And, though they are safer and have fewer side-effects than alternative treatments, therapeutic vaccines may still need additional demonstrations of purity and safety—not for the FDA but for the public.

Thus, the question is: To be proactive in providing the proof and sensitivity that will enable an exciting new era in medicine to excel, or not to be?

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