In the
articles in this series, I explained what a clinical trial is and how to access
one. Currently, there are 437,173 research studies registered with clinicaltrials.gov. They are
conducted in more than 220 countries, with just under half being done within
the U.S.A. in all 50 states. They offer hope to patients with serious illnesses,
yet finding and participating in one is a daunting process. My vision is to
offer members of our community hope through a chesed organization
that would walk with them during this
very frightening time in their and their family’s lives. I call
this new entity Path to Hope, for now, and its purpose would be to provide the
expertise and energy to support, guide, and advise patients and their families
as they search for and enter clinical trials. I pray that such a non-profit
will be formed soon.
I would like to share
more details about clinical trials. Let’s take a brief look at resources,
finding trials, and knowing what to ask. One resource is the clinicaltrials.gov
webpage, a good link to all things clinical trials: https://clinicaltrials.gov/ct2/about-studies/learn.
I especially like the list of 18 questions. (Of course it would be “chai,” a number of hope!) As we explored
in a previous article, the patient and his or her family need to consider why
they are participating in a clinical trial. Is it to seek a possible beneficial
outcome (therapeutic intervention) or perhaps to create meaning from their
suffering for the greater good of humanity and science (the observational or
possible placebo arm of a treatment trial)? The purpose of their participation
will help guide the questions. For rare diseases without immediate life-threatening
consequences, the opportunity to gain specific medical knowledge about the
condition might be quite rewarding.
An oncology
resource is found on the American Society of Clinical Oncology’s website, on
their research and advocacy page: https://www.cancer.net/research-and-advocacy. Their clinical
trials page can provide more information on placebo use in trials as well as
informational videos: https://www.cancer.net/research-and-advocacy/clinical-trials. Again, it is
important to have someone with healthcare experience review the information
with the patient and family to provide proper understanding of the content and
context.
Many of the professional societies have patient resources,
as does the National Cancer Institute itself; for example, the Office of
Advocacy Relations, https://www.cancer.gov/about-nci/organization/oar. I worked with them for several years to bring in cancer
survivors to speak to my colleagues and interns – plus, they know many of the
medical investigators working from within the clinical trials. A good starting
place for rare diseases might be this website at NIH: https://rarediseases.info.nih.gov/; there is a link below for clinical trials. You never know
which track will bring the patient closer to a yeshuah, and all angles need to be explored.
It’s important to
remember that clinical trials are research driven by hypotheses, and the
studies are required to take care to follow human subject protections. An
aspect of these protections is that the benefits outweigh the risks, and that, through
a process of informed consent, the patient understands the risks, potential
harms, and potential benefits of participation. “Protectzia,” or knowing the right person, will not necessarily
allow one to enter a trial. Both eligibility and ineligibility need to be met,
or the science and learning derived from the trial can be compromised. That
could put all participants in a trial at risk of futility of effort creating an
imbalance in the benefit-to-risk profile of the study.
In the end,
clinical trials are always bigger than the individual participants, although
each person enters them very much with their individuality and humanity at
stake. I recognize this might be hard to accept in our Jewish culture, where one
Jew will bend over backwards for another Jew. However, in these situations
there is very little wiggle room. I will say that there is a time and place for
everything, and it is the preemptive work of an organization such as Path to
Hope that could create opportunities for those in need.
An exception to
the no-wiggle room rule can be found with the FDA’s Expanded Access program.
The intent is not for research, rather, the program provides investigational
medical products so that patients with a serious disease or condition who do
not have comparable or satisfactory alternative therapies to treat the disease
or condition have access to them for emergency or urgent situations. More about
the program can be found here: https://www.fda.gov/drugs/investigational-new-drug-ind-application/physicians-how-request-single-patient-expanded-access-compassionate-use. FDA partnered
with the non-profit Reagan-Udall Foundation to create an online navigator which
can be accessed here: https://navigator.reaganudall.org/expanded-access-navigator. This expanded
access program does require the participation of one’s physician to request the
medical product and guide treatment.
* * *
Finding trials is an arduous process. Because the patient and his or her immediate family are
often exhausted and just barely coping day to day, there has to be an advocate
for the patient who is willing to be devoted in terms of time and effort. The
patient’s physician needs to be an advocate and willing to try new avenues of
treatment. Families might find leads with patient advocacy groups, such as the
QuadW for osteosarcoma (https://www.quadw.org/) and BCAN for bladder cancer (https://bcan.org/). I have worked with both and come away inspired each
time. Online forums have proliferated, but it’s important to recognize that
experiences shared on these forums are anecdotal rather than proven science.
Still, they make for good leads and further investigation. All this takes time
and patience to compile sufficient information to begin to generate a picture
of hope.
In keeping with my vision for Path to Hope, such an
organization would do a lot of this important but demanding work for later
presentation to the patient and his or her family. Additionally, the
peer-to-peer mentorship suggested as part of the organization would provide
personal resources, emotional support, and perhaps shortcuts to finding the
best path forward.
* * *
As an example of
the hope seen in clinical trials, a family friend sent me a Baltimore Sun article (August 31, 2022,
Meredith Cohn) that reports on a cancer patient who was suffering from
glioblastoma, a brain cancer with very poor prognosis. What’s remarkable is that
this was a phase 1 trial where the primary outcome was to show safety, not any
effectiveness of treatment. Yet, in this trial, there were effective results
for most of the 14 participants. Larger trials will be needed to show that this
phase 1 trial is a sustainable strategy offering long-term hope. (https://www.baltimoresun.com/health/bs-hs-glioblastoma-trial-20220831-l76szrun5rgo3f4nhiaik2srhe-story.html)
Although I have
focused on cancer clinical trials in these articles, they amount to only
one-third of trials that are recruiting participants in the U.S.A. The
remaining two-thirds (>16,000) are investigations into other diseases and
conditions. For example, recently it was reported by Newsmax in the journal
Lancet (https://www.newsmax.com/health/health-news/eczema-severe-children/2022/09/20/id/1088260/) that children
with severe eczema could be treated with a manufactured monoclonal antibody
called dupilumab, which
provided significant relief of the symptoms. The safety profile and side
effects seem to be minimal; however, the cost can be significant.
This trial caught my eye because I have a young grandson
who suffers from severe eczema; indeed, his parents were already on the hunt
for this treatment. Over the past few months of treatment, the little guy’s
skin symptoms have significantly decreased in severity. The biggest issue is
not the cost (Medicare) but the inoculation itself; a less invasive means needs
to be created for administration of the antibody that might be more favorable
for children.
I will close this series of articles on clinical trials
with the hope and expectation that a better path can be created for those
suffering from diseases where the current standard of care has not led to a
cure or a significant diminution of symptoms. Should you share this vision of hope
for these patients, please contact me at dan.edelman@gmail.com, and please support Path to Hope. Thank you for your
attention to this important matter.
Daniel Edelman received his BTL from Ner Israel Rabbinical
College, his M.S. in Applied Molecular Biology from University of MD Baltimore
County, and his Ph.D. in Medical Pathology from University of MD Baltimore. Dr.
Edelman worked at the Food and Drug Administration (FDA) as a Scientific
Reviewer for a brief stint before moving to the NCI/NIH for 13 years as a
laboratory manager of a clinical laboratory supporting NCI cancer research and
clinical trials. While at NIH, he ran a science bridge program for seven years
for dozens of young Orthodox Jewish women seeking a mentored pathway into the
sciences; many have gone on to careers in science and medical fields.
Currently, Dr. Edelman is a Biologist/Policy Analyst at the FDA as part of the
Personalized Medicine Team where he and his colleagues seek to bring the right
test, to the right patient, at the right time, for the use of the right drug.
The views expressed in this series do not necessarily represent the views of
the FDA or the United States government.
