A promising therapy that may slow or stop Parkinson's progression is moving

forward. Today The Michael J. Fox Foundation (MJFF), the Van Andel Research
Institute (VARI) in Michigan and the Cure Parkinson's Trust (CPT) in the United
Kingdom announced plans to collaborate to assess the clinical use and
development of cancer drug nilotinib. Among the partners' goals: planning a
double-blind, placebo-controlled clinical trial of nilotinib, which MJFF hopes can
begin in 2017.
The announcement came in conjunction with today's publication, in the Journal
of Parkinson's Disease, of apaper on the first trial of nilotinib in people with
Parkinson's disease from a team at Georgetown University. An accompanying
editorial, "Nilotinib -- Differentiating the Hope from the Hype," presents the
research on nilotinib and its target, c-Abl (read more on this emerging protein of
interest below), as intriguing, but warns against patient use until we know more
about the drug's safety and efficacy for people with Parkinson's disease. (The
editorial was authored by MJFF CEO Todd Sherer, PhD; Richard Wyse, MD, director
of research and development at Cure Parkinson's Trust; and Patrik Brundin, MD,
PhD, director of the Van Andel Research Institute Center for Neurodegenerative
Science and editor of the journal.)
"It is impossible to extract definitive safety and valid efficacy signals from a small
open-label unblinded study (lacking a placebo control) in PD and dementia with
Lewy bodies," the authors write. "A major concerted effort is needed to
determine whether there is still hope that can match the hype for nilontinib in
alpha-synucleinopathies."
Here we cover some frequently asked questions about this drug and area of
research. Learn more in a special MJFF webinar on Tuesday, August 2 at 12 p.m.
ET.
What is nilotinib?
Nilotinib is a drug approved for chronic myelogenous leukemia, a cancer of the
white blood cells, under the brand name Tasigna. The medication inhibits a class
of certain proteins, including one called c-Abl, which is an emerging target for
Parkinson's research.
What is the connection between c-Abl protein and Parkinsons disease?
Higher levels of c-Abl are associated with Parkinson's disease. This means trouble
in a few different ways:
An MJFF-funded project showed that heightened c-Abl activity inhibits the
parkin protein. Parkin, when acting normally, goes around the cell and tags
unnecessary or dysfunctional proteins and mitochondria for degradation. When
parkin is not working correctly (perhaps because of high c-Abl levels), bad
proteins -- such as the key Parkinson's player alpha-synuclein -- and damaged
mitochondria can build up into toxic clumps and harm the cell. Other cellular
players that work with parkin (called substrates) also can become toxic to the
cell if parkin is not functioning correctly.
Also, last month another paper reported that deleting c-Abl from preclinical models reduced alpha-synuclein aggregation, while over-expressing c-Abl
led to the protein clumps. The research team on that paper showed a direct link
between c-Abl and alpha-synuclein, further supporting the role of c-Abl in
Parkinson's disease.
In addition to the role of c-Abl in regulating parkin and/or alpha-synuclein,
some researchers have demonstrated its involvement in dopamine-signaling
pathways.

To recap: scientists believe that too much c-Abl hurts cells by messing with
parkin function, encouraging alpha-synuclein aggregation directly and/or
impacting dopamine signaling. With the evidence mounting, c-Abl is gaining
attention as a Parkinson's drug target.
What has the research told us about nilotinib?
Two studies in pre-clinical PD models from 2013 and 2014 showed protective
effects of nilotinib. And several other studies in pre-clinical PD models have
shown protective effects of inhibiting c-Abl. This provided impetus for testing
nilotinib in patients.
The trial results published today -- from a small, open-label (all knew they were
getting the drug) trial of nilotinib in people with advanced Parkinson's -- included
impact on spinal fluid measures of alpha-synuclein and imaging scans of
dopamine function.
The drug was well tolerated, and participants reported improvements in motor
skills and cognitive function. These are encouraging results; unfortunately,
researchers know that the likelihood of placebo effect is high in any open-label
Parkinson's clinical study. Nonetheless, MJFF deems these findings supportive of
continued, rigorous research in this area.
Should patients start taking nilotinib?
In short, no. We just don't know enough yet. Patients and clinicians are urged to
wait for further safety data before considering adding the drug to their treatment
regimens at this time. Much work remains to be done to validate the drug in a
clinical setting, and there is not yet enough information to assert with certainty
that it works in Parkinson's and, critically, that it is safe to take over the course of
a lifetime.
Cancer treatments are notoriously hard on the body. While people with
Parkinson's might take a significantly lower dose, we need to know the long-term
effects.
Why the lower dose for PD? In cancer treatment, you try to get that tagging and
degradation system working in overdrive to eat up everything in the cancer cells.
In Parkinson's, we just want the tagging system to work normally, so we might
not need as much drug. But we don't yet know whether a lower dose of nilotinib
will actually inhibit c-Abl in the brain.
Which leads to another question: How much drug gets the system working
enough to protect the cell but not so much as to harm it? We need to make sure
the drug is truly treating the Parkinson's process.
"Nilotinib does not get into brain that well, so one of the questions that I have is:
At the dose touted to be effective in humans, is c-Abl in brain cells being
inhibited?" says Ted Dawson, MD, PhD, of Johns Hopkins University and author of
the recent paper connecting c-Abl to alpha-synuclein. "And it has toxicities, so if
a patient is contemplating taking nilotinib, it should really be done in the setting
of a controlled clinical trial where you're appropriately monitored."
Are there other drugs similar to nilotinib?
There are other c-Abl inhibitors for cancer, but they either also come with harsh
side effects or don't pass the blood-brain barrier (a requirement to stop the
Parkinson's process).
So what are the next steps?
MJFF, VARI and CPT are collaborating on a therapeutic development program to
assess the safety and efficacy of nilotinib in people with PD. The program
includes the goal of planning of a double-blind, placebo-controlled (neither

researchers nor participants know who has gotten the drug or placebo) clinical
trial of nilotinib, which MJFF hopes can begin in 2017.
The partners plan to expand on early safety findings to better understand the
implications of long-term use of nilotinib and to rigorously vet early-stage preclinical and clinical findings such as around drug penetration into the brain and
the relationship between nilotinib dosing and c-Abl activity.
The sponsors of the first clinical trial also are planning a follow-up. These parallel
studies will help gather more data and provide independent findings for
comparison.
And the field is working on new c-Abl inhibitors that get in to the brain better
with fewer risks and side effects.
How can I learn more?
Join us for a special webinar on nilotinib on Tuesday, August 2 at 12 p.m. ET. We'll
answer questions on the science behind this potential treatment, findings thus
far, safety concerns, and next steps.
We'll continue to update the Parkinson's community on the MJFF/VARI/CPT
partnership through our blog, emails and social channels.
Create a profile on Fox Trial Finder or make sure your profile is up to date to be
notified if you are a likely match for this or other clinical trials in need of research
volunteers.

Tasigna (Nilotinib) is a leukemia drug that has recently

been tested for safety in a small, phase I clinical trial on
about a dozen Parkinsons disease patients. The study
had positive results that certainly warrant the
continuation to a phase II trial, however it is too early for
patients to seek treatment outside the setting of a
clinical trial. The study was very small, and it was not
placebo controlled. The clinical trial process was
designed primarily around patient safety, which is a
critical issue with a chemotherapy drug, and this process
should continue and more systematic evidence needs to

be collected before we can truly understand the impact

of this drug.
If you are interested in learning more about the results of
this clinical trial, visitGeorgetown University National
Parkinson Foundation Center of Excellence. To learn
about other clinical trials, check clinical trials.gov for
trials taking place in your area.

You can find

out more about NPF's National Medical Director, Dr.
Michael S. Okun, by also visiting the NPF Center of

Excellence, University of Florida Health Center for

Movement Disorders and Neurorestoration. Dr. Okun
is also the author of the Amazon #1 Parkinson's Best
Seller 10 Secrets to a Happier Life and 10
Breakthrough Therapies for Parkinson's Disease.

In a small scale study, Georgetown University Medical Center researchers have discovered that a
drug used to treat leukemia improves the symptoms of Parkinson's disease.
In the early phase I clinical trial, 12 patients received nilotinib, an FDA-approved drug for
leukemia. The drug improved cognition, motor skills, and non-motor function in patients with
Parkinson's disease and Lewy body dementia. In addition, it led to statistically significant and
encouraging changes in toxic proteins linked to disease progression.
Charbel Moussa, director of Georgetown's Laboratory of Dementia and Parkinsonism, and
Fernando Pagan, director of Georgetown's Movement Disorders Program, led the trial, which was
supported by the Georgetown-Howard Universities Center for Clinical and Translational Science.
Preliminary data were presented at Neuroscience 2015, the annual meeting of the Society for
Neuroscience, in October.
Moussa conducted the preclinical research that led to the discovery of nilotinib for the treatment
of neuro-degenerative diseases, then partnered with Pagan for the clinical study. "To my
knowledge, this study represents the first time a therapy appears to reverseto a greater or lesser
degree depending on stage of diseasecognitive and motor decline in patients with these
neurodegenerative disorders," says Pagan.
Investigators report that the six-month study of nilotinib, a treatment for chronic myelogenous
leukemia or CML, produced benefit for all study patients who completed the trial (11 of 12), with
11 patients reporting meaningful clinical improvements. The study's primary objective was to test
safety. Researchers say that use of nilotinib, in doses much smaller than those used to treat
cancer, was welltolerated with no serious side effects.
Moussa and Pagan found that the drug penetrates the blood-brain barrier in amounts greater
than dopamine drugs, but the observed efficacy in cognition, motor skills, and nonmotor function
improvement for many patients was the most dramatic result. The investigators report that one
individual confined to a wheelchair was able to walk again; three others who could not talk were
able to hold conversations.

Alan Hoffman, a retired professor, was diagnosed with Parkinson's disease in 1997 and has
participated in several clinical trials with no benefit, he says, until he enrolled in Pagan's study.
Before nilotinib, he was unable to do simple household tasks, but after the trial he said, "Now, I
empty the garbage, unload the dishwasher, load the washer and the dryer, set the table, even take
responsibility for grilling."
In the three weeks prior to enrolling in the study, Hoffman says he fell eight times, but only fell
once during six months on the study.
His speech has improved, as has his thinking. "My wife says it's life-changing for her and for my
children and grandchildren," Hoffman says. "To say that nilotinib has made a change in our lives
is a huge understatement."
While the results of the small trial are promising, they should be interpreted with appropriate
circumspection there was no control group for comparison. Also, nilotinib was not compared
with a placebo or other medications used to treat Parkinson's in the study.
Moussa and other Georgetown researchers are planning larger clinical trials with nilotinib for
patients with Parkinson's and other similar diseases including Alzheimer's disease, likely to begin
this year. Moussa is an inventor on a Georgetown University patent application for use of
nilotinib for the treatment of neurodegenerative disease.
Support for the next phase of the Parkinson's trial includes a recent $1 million gift from the
Lasky-Barajas Family Fund to the Nilotinib Clinical Research Fund in the Translational
Neurotherapeutics Program at Georgetown University Medical Center. The phase II Alzheimer's
trial also continues to receive funding from individuals and organizations including the
Alzheimer's Drug Discovery Foundation.
GEORGETOWN UNIVERSITY MEDICAL CENTER
Suite 120
4000 Reservoir Road, N.W., Washington D.C. 20057
Phone: (202) 687.0100

crucial clinical trial of the most promising new treatment

for Parkinsons disease in decades might be delayed because of a

feud between a key scientist and the influential Michael J. Fox
Foundation.
The prominent foundation the richest nonprofit seeking to
cure the crippling neurological disorder initially wanted to
collaborate on a study with the Georgetown University
researcher. His preliminary findings last year had buoyed
patients hopes for the first Parkinsons medicine that might
reverse some of their debilitating symptoms.
The trial was supposed to begin in October, but Fox and the
Georgetown team had a bitter falling out, and its unclear
whether Georgetown will be able to obtain the medicine from its
manufacturer so that the study can proceed. Fox, meanwhile, is
moving forward on its own, and has established a separate group
to study the same drug. That trial wont start until about a year
from now a long wait for millions of Parkinsons patients
worldwide.
We thought it was a scientific discussion, but (the foundation)
had alternate motives, Dr. Charbel Moussa, the Georgetown

scientist, told STAT. Fox engaged in a premeditated attempt to

cut us out, he said. Some people think they are the owners of
the conversation, the owners of the scientific debate, the owners
of Parkinsons research.
Todd Sherer, chief executive of Fox, denied Moussas
accusations. We acted in good faith to reach an agreement with
Georgetown, he said, noting that the foundation has a long
record of credibility and authority in its collaborations with
hundreds of researchers.
STAT learned of the dispute from members of the Parkinsons
research community, and subsequently examined published
scientific papers and emails and research proposals that passed
between Georgetown, Fox, and the foundations collaborators.

READ MORE
The remote control that can help Parkinsons patients

Conflicts over credit and control of research are far from rare in
the chase for medical breakthroughs. But rarely do such fights
with high stakes for professional stature, organizational prestige,
and fundraising erupt into public view.
The trial offers Moussa, a little-known scientist, a chance to vault
into the top ranks of researchers, while Fox and its collaborators
could reinforce their standing and tap into a bonanza of
donations from wealthy philanthropists and worried patients.
The episode also highlights the tension between scientists and
foundations that are no longer content simply to dole out money.
Following the lead of the colossal Bill & Melinda Gates
Foundation, major medical research philanthropies increasingly
seek to coordinate or manage studies, or control details of how
they are done vexing many grant recipients.
In this case, the clash involves nilotinib, a cancer drug tested by
Moussa and his colleagues in 12 Parkinsons patients many of
whom described substantial relief from symptoms such as
shuffling gait and cognitive decline. Although the trial was small,

the results announced last year thrilled doctors and patients who
have waited decades for a glimmer of success.
Soon after, Moussa said, the foundation, started by actor and
Parkinsons patient Michael J. Fox, approached him about
funding his next steps. Moussa said he already had separate
funding, and approval from the US Food and Drug
Administration and a university research review board to
conduct a Phase 2 trial a larger test of the drugs safety and
effectiveness at Georgetown. He hoped that Fox would
underwrite costs for other test locations, which would give the
results more credibility.
Discussions between the sides lasted about six months, during
which Fox officials reviewed and asked for changes in the
Georgetown study design. Moussa disagreed with some requests
involving verification of concepts he said had been demonstrated
in the initial study. The modifications would delay us for one
year, and this was not good for patients, Moussa said.
The talks fell apart when Brian Fiske, the foundations senior
vice president for research, told Novartis, nilotinibs maker, that
Fox disavowed the Georgetown project.
Whether they [Novartis] will continue to review your proposal is
of course up to Novartis, but we made it clear that this was
submitted without our knowledge or commitment to support, he
wrote to Moussa in a June 17 email provided to STAT by the
Georgetown scientist.
Moussa said he hopes Novartis eventually will donate the drug
valued at several million dollars for the proposed 75-patient
trial. He said Fiskes intervention seemed designed to sabotage
his proposal.
Soon after, Fox announced its own Phase 2 trial. Sherer said he
encouraged Novartis to supply nilotinib to all researchers
including his foundation and its partners from two other
nonprofits.

Novartis spokeswoman Julie Masow would say only that the

company is in discussions with Georgetown about the possibility
of supporting this study.
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Sherer said Fox contacted Novartis because Moussa told the

drug maker that the foundation was funding his work before an
agreement was final. Moussa called that characterization
completely false. He said he now thinks Fox had planned for
some time to delay Georgetowns efforts, to give its own trial a
lead in the race to prove nilotinibs value.
The reason why its so hard-fought is that there are some people
who believe that this is the drug that could be the first to
demonstrate an ability to slow the progress of Parkinsons
disease, a potentially towering achievement, said Peter Schmidt,
senior vice president of the National Parkinson Foundation, a
smaller charity that also funds research.
Beyond the finger-pointing, there are real scientific differences
about how to construct the best test of nilotinib.
There are people who believe that a better-run trial might
demonstrate positive findings. Thus, there are good and noble
reasons for a funding agency to want to be involved in organizing
an important trial, Schmidt said. He offered the historical
example of the National Foundation for Infantile Paralysis (later,
called March of Dimes), which pulled control of the final polio
vaccine trial from its inventor, Jonas Salk, in favor of a different
leader.
But Schmidt cautioned against unnecessary delays of a Phase 2
trial. The question of whether or not nilotinib will make a
difference is one of great urgency to people with Parkinsons
today, he said. Anything that slows down getting the answer to
that question is hurting patients.

The dominant player

Michael J. Foxs star power has built the foundation into a
fundraising juggernaut. Rather than directing scientific
programs, he focuses on boosting public awareness and
donations assisted by fellow celebrities, such as Tina Fey, Paul
Simon, and Julianna Margulies, with whom Fox starred on The
Good Wife. Moguls from Silicon Valley, rather than Hollywood,
have given the most, including $25 million or more in annual
contributions from Google cofounder Sergey Brin and his exwife, 23andMe founder Anne Wojcicki.
The foundation awarded nearly $88 million in research grants
last year, dwarfing combined support by all other Parkinsons
nonprofits. Fox has gained far-reaching influence by funding
leading academic, corporate, and even National Institutes of
Health researchers.

READ MORE
New Parkinsons drug could reduce hallucinations, but at what cost?

Sherer, 43, joined the foundation in 2004 after completing a PhD

in neuroscience and postdoctoral training. He built the
foundations scientific team that assesses and steers research
priorities before being elevated to CEO in 2011. In a news
release at the time, Michael J. Fox praised Sherers uncommon
ability to look at therapeutic progress from both the scientist and
the patient perspectives, and how his research peers
willingly follow him in new and challenging directions.
Since Sherer took the top job,the foundation has funded about
1,000 grants. Most were calculated to the dollar, akin to contract

invoices and unlike conventional grants awarded for a body of

work or project. Fox spokeswoman Christina Brdey said grant
managers approve specific research budgets and approaches.
Philanthropies find that this kind of contract research is
acceptable to researchers, given the difficulty in securing federal
funds, Schmidt said.
Allan Tobin, director emeritus of the UCLA Brain Research
Institute, helped start the CHDI Foundation, which funds studies
on Huntingtons disease, another neurodegenerative disorder. He
said CHDI also actively shaped grantee projects to help ensure
the best results.
This hands-on approach is ascending in medical research
philanthropy, Tobin said, and on the whole works well, although
some CHDI-supported researchers objected to the foundations
scientific and financial management.
Fox sometimes parses large projects into many grants,
maintaining overall control, as it has done for a large
observational study to define physical or biological signs of
Parkinsons. Working with the studys principal investigator, Fox
officials manage the studys protocol, governance, financing,
expansion, promotion, (and) recruitment, Brdey said in an
email.
Fox is spreading $60 million over dozens of testing sites and
research programs for that project. Brdey compared the
foundations role to that played by the National Institute on
Aging on a similar Alzheimers project.
Leading a trial for the most important Parkinsons treatment in
more than 40 years could bring the Fox Foundation valuable
cachet and more donations, according to executives of other
philanthropies.
The March of Dimes took advantage of their direct involvement
in the development of the polio vaccine to continue as a
fundraising powerhouse, despite having achieved their principal

mission objective with the vaccine, Schmidt said in an email. It

is very powerful to be able to say to a prospective donor, We are
doing this, rather than, We are funding this.
Decades of frustration
Parkinsons afflicts about 1 million Americans, who face
increasing tremors, stiffness, movement problems, and often
mental decline. It has no cure.Parkinsons is associated with a
reduced supply of dopamine a naturally occurring chemical in
the brain that helps nerve cells talk to each other.
Since the 1970s, to relieve some movement symptoms, many
patients have taken levodopa, also called L-dopa, which
replenishes dopamine. But L-dopa does not reverse the course of
the disease and helps less over time, so scientists have long
sought alternatives.
Nilotinib, a blood cancer medicine, has long been considered for
its potential to help Parkinsons patients. Lab and animal studies
suggested that the drug could protect dopamine-producing cells
in people. But its toxicity made researchers nervous. It carries
the Food and Drug Administrations black box warning
indicating possibly life-threatening side effects.

READ MORE
FDA urged to place warnings on Parkinsons drugs about compulsive behaviors

Georgetown scientists, who had been deeply involved in basic

research exploring nilotinibs potential, felt confident that a
small human safety trial at far lower doses than used by cancer
patients would be safe to try.

Their results, presented at a meeting last October of the Society

for Neuroscience, exceeded every expectation.
Nilotinib appeared to double patients dopamine production,
reduce the death of dopamine-producing nerve cells, and clear
from those cells a toxic protein associated with Parkinsons and a
related disorder, dementia with Lewy bodies.
Relief from symptoms seemed equally striking among the 11
patients who completed the trial. Alan Hoffman, 75, a retired
university professor who participated in the trial, said in an
interview that by 2014, his Parkinsons had become devastating.
He suffered frequent falls and stiff, frozen muscles. After a life
of intellectual curiosity, his powers of concentration were
reduced to scanning news headlines.
Nilotinib proved transformative, Hoffman wrote in his blog. I
was walking steadily. My speech was louder and clearer, I was
no longer falling, I was less confused about things in general,
and my life was considerably better, he said. I first read the
David McCullough book on the Wright Brothers and then
progressed to others. I was thrilled!
When the study ended, Hoffman regressed sharply, he told STAT.
After he secured nilotinib from Canada at a discount from the
$10,000 monthly cost charged in the United States, his
symptoms again began to taper off.
Such stories prompted doctors around the world to begin
prescribing the drug for some of their Parkinsons patients,
though it had not been approved or proven safe and effective for
that condition.
A few participants in Georgetowns Phase 1 safety trial
experienced health problems that might have been caused by
nilotinib, and the study had serious limits no control group or
double-blind design, in which neither patients nor researchers
know who gets the drug and who gets a sugar pill. Such features
can help verify benefits attributed to nilotinib and possible side

effects. Moussa and his colleagues cautioned that the hopeful

results must be confirmed in a larger Phase 2 trial.
Quest for collaboration
Moussa, 43, said he acquired his love of science and medicine by
accompanying his father, a large-animal veterinarian, on his
rounds to farms in their native Lebanon. He earned his MD and
PhD degrees in Australia, and came to Georgetown in 2003.
Since my first year in college, it always fascinated me how the
brain works. But there are few answers, he said. Neuroscience
is one of the areas in medicine today where you can really make
a contribution.
Fox had rejected his request to underwrite the Phase 1 nilotinib
trial. So when we announced the results and Michael J. Fox
Foundation contacted me, it was an exciting moment and
validation, he said.
Moussa confidentially shared his groups test plans, proposal to
Novartis, and other details. Both sides expected Fox to provide
funds.
Moussa provided STAT a February email from Fox research
director Marco Baptista, who wrote, MJFF is willing to work
with you to design such a study and fund it. I can ensure you
that I have the full support of MJFF senior management to
overcome any obstacles so that together we can make this
happen.
Dr. Patrik Brundin, a top scientist at the Van Andel Research
Institute in Michigan, and Dr. Richard Wyse, research director at
the United Kingdom-based Cure Parkinsons Trust, also
approached Georgetown about possible collaborations. In an
email, Wyse praised Moussa for convincing top global experts
that low-dose nilotinib was likely to be safe
for Parkinsons patients.

But talks between Moussa and the possible collaborators

foundered in June after he learned that Fox officials had
approached Novartis about Georgetowns proposal behind his
back. Those officials also pressured Moussa not to speak with
Novartis independently. Fiske, the Fox vice president, wrote in an
email that Moussa should only have submitted to Novartis a
proposal approved by the foundation, a process the foundation
thought Georgetown had agreed to follow.
Moussa bristled at Fiskes admonition. Your email appears to
suggest that you are in control of our study, Moussa replied.
We do not feel that we have to wait for your approval to
continue our engagement with Novartis. We have come a very
long way to get the drug donated and we are interested in
engaging MJFF for funding. We do not believe that one thing
depends on the other in any way, shape, or form.
Fiske responded that given these challenges in
communications with Novartis, the foundation was reassessing
the ability of MJFF and Georgetown to continue our collaboration
and are reviewing other options we have for independently
evaluating nilotinib.
In interviews, Brundin and Sherer described their differences
with Georgetown as technical ones concerning study design and
priorities for the Phase 2 trial.
Moussa said Fox officials never formally notified him that his
grant proposal had been rejected. In July, he published
the results of the initial trial in the Journal of Parkinsons
Disease, which is co-edited by Brundin. The article appeared
alongside an editorial by Brundin, Wyse, and Sherer, which said
the Georgetown study substantiates a new direction but
castigated the intense media exposure when the results were
announced last fall as an object lesson on how not to report a
small clinical trial that has no placebo control.

The editorial concluded that a new clinical trial of nilotinib was

warranted and that it should involve leaders in (Parkinsons)
clinical trial design and be conducted at multiple sites.
Moussa read the editorial as an attempt to disparage his groups
work and planned trial. Without Foxs funding, it would be
conducted at only one site.
The commentary made no mention about what the authors were
planning next.
A gatekeeper moment
The same day the editorial appeared, the three authors
announced in an optimistic press release their mutual
collaboration on a multisite, Phase 2 nilotinib trial.
Moussa said he had no idea it was coming. This is a huge
endorsement of what we are doing at Georgetown University, but
why they would want to cut us out of the process, I dont
understand, he said. Now they are racing us to get the credit.
Brundin said he didnt understand such concerns. If nilotinib
does become important, he said, most credit would go to basic
scientists who uncovered its potential. That includes Moussa,
who ironically, would benefit tremendously if there is a second
trial (unconnected to Georgetown) that shows nilotinib works,
because it will have so much more credibility Brundin said.
Georgetown has applied for a patent, with Moussa as inventor,
on using nilotinib to treat neurological disorders, and could earn
licensing fees should the medicine be approved for that use.
Moussa contends that Brundin, Wyse, and the Fox Foundation
improperly took advantage of their access to confidential
materials about his research to plan their own study. This
included prepublication versions of Georgetowns journal article,
detailed plans for his Phase 2 study, and his proposal to Novartis
that showed the scientific explanation for the drug donation

request. For months, Moussa said, Fox wanted to get more data
out of me.
Sherer and Brundin said nothing improper occurred. Their own
study plan, Brundin said, was based on publicly available
scientific reports, including (Georgetowns) own report, which is
published, of course, in the journal.
Leigh Turner, a bioethics professor at the University of
Minnesota, said that in general, funders and journal editors
should exercise care to be above reproach in their handling of
insider information.
You have these gatekeeper moments, where it is possible to take
advantage of positions like that, to gobble up as much
information as possible, and repurpose it for your own
advantage, he said.
Charles Piller can be reached
at charles.piller@statnews.com
Follow Charles on Twitter @cpiller