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Shri ThanedarShri Thanedar, Ph.D., is the CEO and chief chemist of Avomeen Analytical Services. A veteran of the CRO industry, Thanedar has more than 25 years of experience supporting companies nationwide with services including method development and validation, chemical characterization, deformulation, extractable and leachable testing, stability and release testing, litigation support, and failure analysis services.

Breakthroughs in scientific research are always heralded as great accomplishments, but progress in Alzheimer research is never fast enough for those whose family members suffer the effects of the disease. A 2012 study sparked intense interest when research revealed a possible new treatment option using the drug bexarotene. This drug, created and prescribed for skin cancer treatment, showed astounding results in the initial study and was spoken of as a “miracle” drug. The Alzheimer community caught fire with this spark of hope. However, quickly the hope was turned to murky doubt as follow up studies were unable to repeat the miraculous outcomes. Still, many cling to this discovery with a desperate passion while ethical debates and medical worries build.

The initial study reported signs of memory restoration in the test rodents as well as 50% clearance of amyloid plaques in only three days. As amyloid plaque is thought to be one of the determining features of Alzheimer disease, this speedy change was monumental. This discovery astounded the researchers, and within days people were clamoring for drug prescriptions for their family members. Four other laboratories conducted follow up studies to verify the results. To the disappointment of those following the progress, these researchers did not see the same results that caused the drug to be labeled a miracle. But they did confirm increased memory and a decrease in soluble amyloid-β, a less aggravated form of the peptide. This confirmed the potential of the drug as a viable treatment, especially for use at the onset of Alzheimer disease.  Sponsored studies are now in progress to evaluate the drug’s use for treatment of Alzheimer disease.

Though the research is progressing at a steady pace, most individuals feel years of waiting simply are not an option. In fact many are looking to bexarotene as a last hope. As it is an FDA-approved drug on the current market, it has already been through the pharmaceutical product development process. The drug, however, has extreme side effects and is most frequently prescribed at low doses for only short periods. The implied use for Alzheimer disease might require long-term treatment, which has unknown consequences. Yet even after consistent warnings from the doctors, medical experts, and the initial researchers at Case Western University, many individuals are turning to off-label use of the drug. These individuals say that, if anything can help, it is worth the risk. Phrases such as “what do we have to lose” and “willing to try anything” scatter the blogs and support forums. People lash out in anger against research regulations and the time projected before an answer will be found. Individuals chronicle their attempts at self-trial while hundreds, maybe thousands, wait with baited breath to read the next update hoping that someone, anyone, will see a breakthrough.

This type of public response concerns medical professionals. The primary worry is for the patients misusing the drug who run a high risk for severe and lasting side effects. The secondary concern is for the research itself. Widespread experimental use of the drug may lead the public to form their own staunch opinion that could undermine the true results once the necessary clinical testing is complete.

As the testing continues, debates wage on, not only regarding the medical implications but the ethics of desperation versus patient’s best interest. While doctors have the responsibility to protect their patients from harmful misuse of drugs, if possible benefits seem to outweigh the risks, how does one qualify “best interest.” The next months will be monumental for this study and will hopefully assuage the concerns on both sides. For now Alzheimer patients and family are urged to wait, painful as it is, until the necessary testing is concluded.