UsAgainstAlzheimer’s Proposes Key Measures to Advance Treatment Development

UsAgainstAlzheimer’s Proposes Key Measures to Advance Treatment Development

As the disappointment over the failure of solanezumab begins to wane, George Vradenburg, co-founder and chairman of UsAgainstAlzheimer’s, seeks to address the efforts needed to continue research and clinical development for Alzheimer’s disease.

Despite the inability of Eli Lilly to bring the goods to the table, Vradenburg applauded Lilly’s efforts in the open letter, along with that of patients and caregivers who contributed to the solanezumab trials. The studies, he argued, brought valuable knowledge that will help any future drug development efforts.

Vradenburg specifically pointed to a number of factors that he believes are crucial. First, he stated that Alzheimer’s is a disease that needs aggressive treatment. Comparing Alzheimer’s to cancer, he suggested that affected patients and their families are prepared to take the consequences of more aggressive treatments if they can reap the benefits of a slowed or prevented disease progression.

Second, he stated that the current approach to Alzheimer’s clinical trials makes a system in which the time before a drug can become available to the patients become unacceptably long. The failure of past trials has made developers target increasingly earlier disease stages. But these trials become both costly and lengthy, with an estimated time to completion of up to eight years.

The UsAgainstAlzheimer’s chairman emphasized that even if such a trial become successful in producing a drug, healthcare payers, such as Medicare, may not want to pay for the treatment until they have “real-life” evidence of its benefits. Outside the U.S., an expensive therapy may be even more inaccessible. To make matters worse, Vradenburg said that physicians are not well enough equipped to assess which patients may benefit from a precision treatment.

Finally, Vradenburg is of the opinion that the research community should not give up on symptomatic treatment approaches. Given the complexity of Alzheimer’s disease, he stated that it is unlikely that any single compound will hold the answer to treating the condition. Combining preventive and symptomatic treatments, as well as broadening research efforts to include compounds that do not target amyloid-beta, are crucial steps, he maintained, specifically mentioning therapies targeting tau or inflammatory processes.

But Vradenburg, as a representative of the proactive organization UsAgainstAlzheimer’s, did not just point out the challenges. In a list of nine points, he also suggested specific actions that are needed to advance the field.

  • As recognition and support of researchers and trial participants is an important motivator, he suggested that the Alzheimer’s community should applaud Eli Lilly’s efforts.
  • Commit resources that are proportional to the problem, and hold everyone accountable. “Alzheimer’s is a cancer-size problem requiring cancer-size investments,” Vradenburg wrote, urging the U.S. and its president-elect to make good on the goal of stopping the disease by 2025. This, he suggested, should be done by showing international leadership and funding commitments. He suggested that every government in the world should contribute with funding the equivalent of 1 percent of their Alzheimer’s care costs (which in the U.S. was $2.3 billion, according to data from 2016).
  • A point of particular importance is the needed transformation of the clinical trial system. Both length and cost of trials need to be minimized by new trial designs, rapid recruitment, and testing of combination therapies.
  • Working with physicians, the rates of early and accurate diagnoses need to be increased. This step includes more efforts on biomarker development. With the help of big data, researchers need to work to develop cheap and easily administered algorithms for identification of patients.
  • Develop combination therapies. Comparing both the fields of cancer and HIV treatment, Vradenburg again underscored that it is highly unlikely for a single drug to successfully treat Alzheimer’s disease.
  • In a point extending from the previous one, he also stressed the importance of diversifying theories and drug targets. Also, research participation needs to be more variable, including subjects of varying ethnic, socioeconomic, and educational background.
  • Data sharing is one of the steps that may be most effective in advancing research  efforts.
  • Researchers and regulators need to agree on the type of goals clinical trials need to meet. The solanezumab trial did away with the co-primary goal of improving function and focused solely on cognition; a discussion of this practice is needed.
  • Finally, ensuring access is a key goal for any new treatments.

For all of these points, Vradenburg outlined the steps currently taken to address the issues. The entire letter can be read here.

One comment

  1. Lance V. Packer says:

    I commend Mr. Vradenburg for his Open Letter anaysis of recent clinical trials and his recommendations for future needs. Although not directly mentioned but implied, a needed addition to his list is the inclusion of off-label prescription of approved FDA drugs which show experimental potential for repurposed use with symptomatic Alzheimer’s patients. There are many which research has shown to have great potential but individual pysicians, hospital treatment centers, and university research centers are hesitant and actually refuse to consider such an approach to AD. I have personal experience in trying to find someone or an organization which is willing to prescribe such medications for my wife. The usual response, if any, is “We don’t do that”, in spite of the specific drugs and research evidence I cite for them. I don’t argue, but my thoughts are, “My wife is condemned to die, but you won’t even try to help? Isn’t that the essence of medical practice?” Try responding that way to a cancer patient and see what kind of uproar would ensue; but somehow it’s OK for AD patients to be turned away.
    Aside from the personal loss looming over me, I think the AD research community is missing a huge opportunity for solving some of its problems. Without getting into a lot of detail, such off-label prescription is a great opportunity to acquire huge amounts of data for inclusion in big-data banks for analysis as well as treatment potential for inviduals. It would need standardization (at some point) of input and output and to be available for physicians and institutions alike, as a self-feeding process from which recommendations and new discoveries could be made. It’s good to have preventative research but the millions of existing patients cannot be ignored, morally or for their potential information input for research. The problem has to be attacked from both ends of the spectrum. Moreover, with immediate prescription available and a clear explanation of potential upside and downsides of the drug, I guarantee that AD patients and caregivers will be very willing to volunteer for a chance to have some possible positive effect of participating in an experiment, in contrast to the current problem of low trial recruitment. It’s quite simple: there’s something in it for them, right now. It’s called “Hope,” and we all live better with hope. If it isn’t personally successful, there’s always another drug possibility and maybe it will help someone else–that’s a kind of hope that motivates the willingness to try. Somehow, this message hasn’t gotten through to the profession as a whole. It needs to be.

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