Novartis cancer drug to be tested for Parkinson’s disease

Lab work

A blood cancer drug called Tasigna (nilotinib) marketed by Swiss Big Pharma Novartis will be tested in Parkinson’s patients in the hope that it may produce disease-modifying effects.

The trial, which is slated to begin next year, will be run through a collab between three groups: The Michael J. Fox Foundation, the Van Andel Research Institute and the Cure Parkinson's Trust.

There have already been some early clinical tests on the drug for Parkinson’s--specifically a small, 6-month Phase I safety study, although it was not set up for efficacy and was not placebo-controlled.

Training Course

BioBasics: Biotech For The Non-Scientist

BioBasics: Biotech for the Non-Scientist is a two-day course for those who want to better understand the science driving the industry. The course starts with basic scientific concepts and quickly delves into the causes of genetic and infectious disease and the therapeutic strategies used to mitigate disease. The latest innovations in immunotherapies, gene therapy, checkpoint inhibitors, CAR-T and more are explained.

But the study, undertaken by researchers at Georgetown University Medical Center, said in their preliminary findings in 2015 that the use of Tasigna (at much lower doses than used for cancer patients) led to “statistically significant and encouraging changes in toxic proteins linked to disease progression.” This study also sought to trial its effects on Lewy body dementia.

Tasigna works by forcing cancer cells into autophagy (the death of tumor cells). For the Georgetown study, they used less than half the dose for chronic myeloid leukemia patients. At this smaller dose, autophagy was produced for a few hours--which was enough to clean out the cells but without killing them.

The drug is given daily, so the proteins that build up again should be cleared when given again the next day, the researchers said at the time. Preclinical models have also been trialled for the past few years with Tasigna in PD.

But as this study was only set up to primarily assess safety--and was only in a dozen patients--the three groups are hoping to build on this work in their new Parkinson’s trial.

In a statement, they said they would help design and co-fund a double-blind, placebo-controlled trial of the drug to be used in Parkinson’s to assess it efficacy, and whether it can alter the disease and/or the symptoms of the condition.

The organizations urge caution however, saying patients and doctors should not rush out to start using this drug off-label in the hope that it can help, given that there are so few treatments on the market for this chronic disease, as much more study needs to be done.

In conjunction with its trial announcement, an editorial was published in the Journal of Parkinson's Disease, co-authored by Cure Parkinson’s Trust, Van Andel Research Institute, and the Michael J. Fox Foundation, alongside with others, which discussed the limitations of the Georgetown study and argued for the need for a second, more detailed trial.

In the piece, they note that a placebo effect for the Phase I trial cannot be ruled out given the tiny sample size, and note the Black Box warning for Tasigna could mean higher side effects rates in a larger patient population--although a lower dose usage may reduce this risk.

It also said that Tasigna is incredibly expensive (U.S. cost is $10,360 a month for 800mg daily) with the authors estimating that around 200 Parkinson’s patients across a number of countries are now using the drug off-label for their condition--numbers they say may have risen since the Georgetown study.

The authors conclude however that “definitive proof is now required […] to whether nilotinib is safe in PD and DLB, and whether the hints of possible improvement are apparent in a larger group of patients when compared to placebo.”

The new placebo-controlled trial will begin in 2017.

- read the Journal of Parkinson's Disease’s editorial
- check out the release

Related Articles:
FDA's internal review of Acadia's Parkinson's drug raises safety, benefit concerns
Johns Hopkins researchers reveal novel protein and involvement in Parkinson’s disease

Suggested Articles

The Chan Zuckerberg Initiative is awarding $17 million for advances in digital microscopy to allow researchers to view the subtleties of disease.

The drug failed to clearly outperform a generic used as a control, leaving room to doubt whether vibegron poses a big threat to Astellas’ Myrbetriq.

The level of interest in gene therapies has been laid bare by a report on Biogen’s pursuit of Nightstar Therapeutics.