Cancer research is anything but stagnant, and with each new round of data on cancer comes a greater demand for the expansion of cancer care methods.

 chief medical officer Al Rizzo, MD, spoke with Andrea Mazzocchi, PhD, co-founder and chief executive officer of Known Medicine, and adjunct professor at Wake Forest University, about the news strategies being deployed in the world of cancer research.

 of their conversation touched on biomarkers, gene testing, targeted therapies, and new precision medicines. While some of those methods are new and very promising, they also present a very specific challenge.

For instance, biomarker testing has been used in upwards to 70% of patients in recent years, with the endpoint being 100% of the cancer patient population.

However, during the COVID-19 pandemic there had been a blatant disparity in medical knowledge and the efficacy of these new methods, which added to the challenge of reaching the level of deployment doctors hoped to achieve nationally.

“You know, we've been using chemo therapies for so many decades that to start to think about new targeted therapies can become overwhelming, and to really understand what those mutations, what targeted therapy means, and how do we build this into our new regimen can be quite challenging,” Mazzochi said.

Mazzocchi proposed that educating the public of these new methods through support systems could aid in ridding some patients of their hesitancy toward them, but she admitted some hospitals would need to make important changes to their infrastructure to adopt these systems.

Additionally, many of the new strategies that are being offered are time sensitive.

While the test result turnaround time for strategies such as gene testing have improved considerably (from a year or more to 6-8 weeks, generally), a patients’ status or condition can change quickly, which in turn presents problems with each treatment.

Cancer mutations presented a unique problem. If a patient’s cancer mutates and doctors are not immediately aware of the change, the targeted treatment they initially issued could be obsolete.

Mazzocchi urged doctors to be keenly aware of their patients’ conditions, and to issue appropriate treatments immediately to avoid complications.

Additionally, Mazzochi spoke of the new technologies that are being used to treat cancer in patients. She touched on ex vivo cell culture therapy, EGFR therapies, and the act of “taking the experiment out of the patient”.

In many ex vivo cell culture therapies, doctors can extract cell samples from tissue biopsies, tissue resections, and blood samples and test them on upwards to 100 different therapies. The results from those tests can them be transferred directly to a patient’s oncologist.

“This is really powerful because now the experiment is out of the patient (and) into a plate, and we can really see how they are responding (to) these chemo therapies, targeted therapies or immunotherapies will work for that specific patient,” Mazzochi said.

To learn more about newer technologies, genetic testing, therapies, and more referenced in the interview, watch the video above.

To hear all of Dr. Rizzo’s conversation with Dr. Mazzochi, listen to Lungcast below.

Podcasts

Dr. Mazzocchi talks of the promising new cancer care therapies and how to properly inform and care for patients with cancer who may be overwhelmed.

Introducing New Cancer Care Strategies to Patients

Mutations in cancer have always presented a challenge for doctors and patients alike. Lung cancer has several known mutations that have been extensively studied, with an ever-evolving list of inhibitors and therapies that followed suit.

In the newest episode of Lungcast, Andrea Mazzocchi, PhD, co-founder and chief executive officer of Known Medicine, and adjunct professor at Wake Forest University, sat down with host and American Lung Association (ALA) chief medical officer Al Rizzo, MD, to talk about the known mutations regarding lung cancer, the methods for treating these mutations, and immunotherapy and checkpoint inhibitors that can be used to determine cancer in patients.

Of the mutations mentioned, Dr. Mazzocchi noted that the EGFR mutation remained the most known. Approximately 10% of non-small cell lung cancer patients develop an EGFR mutation, which also affects approximately 50% of non-smokers as well, something that Mazzocchi said intrigued researchers.

Several treatments are available for EGFR positive patients, such as the therapy drug erlotnib.

During the discussion, Mazzocchi spoke of the importance of genetic testing, which could lead to more appropriate cancer treatment for patients.

“There’s a lot of opportunities to start using this genetic testing to start matching the patients with (the stages of cancer progression) as well as the mutations, and to what treatment would work best for them,” Mazzocchi said.

While there are a variety of treatments for EGFR mutations, KRAZ mutation treatments remain more elusive. There are considerably less treatments for KRAZ mutations, but that is subject to change in the intervening years.

“Unfortunately, lung cancer affects so many patients, especially in comparison to other cancers, that there’s a lot of development around EGFR and KRAS mutations,” Mazzocchi said “A lot of these can be dictated or driven by these genetic tests and are a great option for patients who have been identified with mutations.”

Additional mutations were discussed in the episode, with potential off-label treatments referenced as well. Mazzocchi suggested that many of the therapies referenced would be treated in parallel with chemotherapies and immunotherapies.

Later in the discussion, Dr. Rizzo and Dr. Mazzocchi discussed the future of immunotherapy in cancer treatment.

Mazzocchi spoke of the “huge promise” that the current 5 immunotherapies presented. She noted that cancer cells tend to disrupt the immune cells with additional receptors that make it harder for immune cells to identify the threat. Luckily, the inhibitors used in immunotherapies can help properly identifying and attacking the cancer cells.

“That’s why these (immunotherapies) are so exciting,” Mazzochi said. “You’re really turning on your immune system.”

While the differences in safety between the PDR1 and CTL4 immunotherapies were noted, she added that there had been an “amazing response” by patients that include reductions in tumor size and quick recovery rates.

“I think there’s a huge amount of promise and a lot of development still to be had, but these treatments are hugely helpful for the population receiving them,” Mazzocchi said.

To learn more about genetic testing, immunotherapies, and microbiomes of cancer referenced in the interview, watch the video above.

To hear all of Dr. Rizzo’s engaging conversation with Dr. Mazzochi, listen to Lungcast below.

Articles

Dr. Mazzocchi discusses the limits and benefits of immunotherapies, as well as the importance of genetic testing in determining cancer diagnosis and treatment in patients.

The Promising Future of Immunotherapies in Cancer Research

In the burgeoning field of cancer research, precision medicine and biomarkers have proven to be crucial in helping to define the pathology of a patient’s cancer as well as possible treatments specific to their condition.

In the newest episode of Lungcast, American Lung Association (ALA) chief medical officer Al Rizzo, MD, spoke with Andrea Mazzocchi, PhD, co-founder and chief executive officer of Known Medicine, and adjunct professor at Wake Forest University, about the future of lung cancer treatment in relation to precision medicine and biomarkers.

Mazzocchi defined precision medicine and its significance in the world of cancer research.

“I think to really understand it we have to think of it as using a patient’s own tissue, their health records, or data surrounding them to then provide more patient specific information that really helps inform or correct your health care decision,” Mazzocchi said.

The practice works in tandem with defining biomarkers in patients. Doctors are no longer limited to studying the number of cells in a patient with cancer, they can also determine the size, how the cell is shaped, what’s inside the cell. 

Biomarkers can also aid in revealing the disease progression and behavior.

“I think what’s really interesting and important is that biomarkers can be used for every point along the timeline of cancer, from detection of cancer all the way to are we able to predict reoccurrence after someone’s in remission,” Mazzocchi said.

Several biomarkers were discussed during the interview, including genetic, epigenetic, proteomic, and phenotypic biomarkers. 

As referenced in the episode, genetic biomarkers may be the most well-known. Roughly 4000 genetic biomarkers exist, in addition to 100 mutations, some of which are treatable. Genetic biomarkers are often associated with genetic mutations and translocations and are identified using blood and tissue samples.

Others, such as epigenetic biomarkers, can be used to help understand the progression of a tumor and aid in early detection. The remaining biomarkers, proteomic and phenotypic, can be used to determine a patient’s response to specific treatment.

To learn more about the biomarkers referenced in the interview, watch the video above.

To hear all of Dr. Rizzo’s conversation with Dr. Mazzocchi on the future of cancer research, listen to Lungcast below.

Andrea Mazzocchi, PhD, explains how tissue and blood samples can be used to improve detection, progression, and treatment for cancer in patients. 

Improving Cancer Care with Precision Medicine and Biomarkers 

 Avicenna’s impact on precision medicine


 The state of lung cancer precision medicine


Lung cancer treatment is on the precipice of unheralded advancement in its field. The intersection of advanced diagnostics, targeted immunotherapy, and implementation of artificial intelligence-borne pathology has created a possibility of concise, individualized care.

Or, as many refer to it, precision medicine.

In this newest episode of Lungcast, American Lung Association (ALA) chief medical officer Al Rizzo, MD, sits down with Andrea Mazzocchi, PhD, co-founder and chief executive officer of Known Medicine, and adjunct professor at Wake Forest University.

After reviewing the ever-long beneficial teachings of Persian scientist Avicenna and his influence on modern precision medicine, Rizzo and Mazzocchi discuss the ins and outs of emerging lung cancer tools and capabilities.

Lungcast is a monthly respiratory health podcast series from the ALA, produced by 

Subscribe or listen to Lungcast on your favorite platforms:

https://open.spotify.com/show/1F3u1hKrwxi9snSQ0q3nti

https://podcasts.apple.com/us/podcast/lungcast/id1524712350

The co-founder and CEO of Known Medicine dissects the art and science of how profiling biomarkers, epigenetics and the microenvironment may provide the synergy needed to revolutionize the oncologic landscape.

The Future of Lung Cancer Precision Medicine with Dr. Andrea Mazzocchi

US prevalence is ~1 in 2500, or 130,000 individuals.

There is no cure for the disease; patients may be managed with supportive therapy.

It is rarely life-threatening, and muscles for vital functions are not typically implicated.

Common early symptoms include weakness or paralysis of the foot and lower leg muscles.

Nerve pain may range from mild to severe.

Some patients may need foot/leg braces or other orthopedic devices.

It is very interesting for everybody to know that this disease has been described over 100 years ago. But to date, no drug has been approved.

I am glad to be part of a treatment team that involves rehab therapists, physiatrists, child neurologists, foot and ankle surgeons, podiatrists, and people whose expertise is in designing ankle braces that can stabilize a dropped foot.

It is not uncommon that patients take medication to treat many of the symptoms — including pain, fatigue, depression. 

Unless there is a clear family history, in many cases it is underdiagnosed.

Having a medication for CMT will dramatically increase recognition of patients by doctors.

 refers to a group of hereditary and degenerative disorders characterized by damaged nerves in the arms and legs. Although there are currently no approved treatments for this condition, patients typically do not have lowered life expectancy.

Of course, quality of life can be negatively impacted, especially if disease onset occurs during adolescence and early adulthood.

The nerve damage can cause weakness in the legs, ankles, and feet, and individuals may experience paralysis in any of their limbs. Tripping and falling are frequent, as are deformities of the feet — often a source of embarrassment and anxiety.

 Florian Thomas, MD, of Hackensack University Medical Center & Hackensack Meridian School of Medicine, and Adrian Hepner, MD, Chief Medical Officer of Pharnext, explained the basic facts about the disease, highlighting the patient experience with CMT.

They talked about how it is often managed and what that care team may look like.

Central to this discussion was the research and treatment landscape, with particular emphasis on their ongoing Phase III study and the impact that a potential FDA approval can have on the lives of hopeful patients.

Thomas and Hepner also mentioned the need for greater advocacy and how increased treatment options can tie into higher awareness levels for this disease.

Listen to The Rare Disease Report on your favorite podcast platforms, including

Florian Thomas, MD, and Adrian Hepner, MD, cover the basics of the degenerative nerve disorder.

The Rare Disease Report Podcast: Charcot-Marie-Tooth Disease

0:15 Intro
1:05 Returning to rivaroxaban
2:12 VOYAGER PAD Subgroup Analysis
4:42 Marc Bonaca, MD, MPH
29:13 Outro

1,533 patients with critical limb ischemia (CLI), with or without diabetes

 2.5 mg twice-daily rivaroxaban plus aspirin, versus placebo plus aspirin, for the reduction of composite acute limb ischemia, vascular-related major amputation, myocardial infarction, ischemic stroke, or cardiovascular-related death

 Major bleeding per Thrombolysis in Myocardial Infarction (TIMI); major bleeding per International Society on Thrombosis and Haemostasis (ISTH)

 Rivaroxaban plus aspirin was associated with a 5.5% reduction in absolute composite endpoint risk reduction in patients with diabetes and CLI, with consistent safety outcomes.

 Investigators observed 23% of patients with CLI and diabetes had experienced amputation, versus just 8% of patients with CLI.

“The risk in this population is really daunting. I mean, there’s an extremely high event rate, there’s a huge unmet need, and the efficacy of rivaroxaban is consistent and really associated with very large absolute risk reductions.”

New subgroup analyses from the VOYAGER PAD trial suggest 2.5 mg rivaroxaban plus aspirin provides consistent benefit in reducing risk of possibly fatal events in patients with 

The findings, presented by Marc Bonaca, MD, MPH, of CPC Clinical Research at the 

American Diabetes Association (ADA) 2021 Scientific Sessions

 this week, contribute to cardiovascular care on multiple levels.

First, it helps define the scope and scale of burden patients with comorbid diabetes and peripheral artery disease (PAD) face in limb and cardiac events—even when compared to patients with lone PAD.

Next—and most importantly—it establishes opportunity of care for millions of at-risk patients with a proven vascular therapy.

In the fourth episode of Heart Trials, Bonaca joined 

during ADA 2021 to share perspective on the newest VOYAGER PAD analysis.

Along the way, Bonaca touched on the burdens of PAD on patients with diabetes, the continuation of VOYAGER PAD assessment, and the pursuit of patient-impacting clinical outcomes.

Listen to Heart Trials on your favorite podcast platforms, including 

The study author joins to highlight new ADA 2021 findings showing rivaroxaban's benefit for a subpopulation of diabetic patients.

VOYAGER PAD, Revisited with Marc Bonaca, MD, MPH

Prevalence estimated to be 1-2/100,000 births

The disorder is passed on through an autosomal recessive fashion

Affected individuals are typically born blind at birth

Electroretinography (ERG) can be utilized in diagnosis

Absence or reduction of the electrical activity in the retina is an important marker of the disease 

These children typically present with roving eyes. They tend not to fix on a parent and have delayed visual maturation. In the case of Leber's Congenital Amaurosis, they have progressive visual loss. 

Parental enthusiam for intervention is very high in this disease because each parent knows that they contributed to the problem. Although, I try not to emphasize any of those things because nobody can really control what the genes are that their children get.

Very few [patients] are actually on the wall chart in terms of their visual acuity by the time they are 20.

The only way to really treat this disease is to treat the mutation 

Patient with Leber’s Congenital Amaurosis (LCA)—a 

 inherited eye disorder that primarily affects the retina—have few to no treatment options, and the condition itself can certainly be burdensome for patient and families.

The most common symptoms associated with the recessive genetic disorder is severe visual impairment and blindness. Patients may also experience photophobia, strabismus, enophthalmos, and extreme farsightedness among other ocular symptoms.

According to the National Organization for Rare Disorders (NORD), “at least 27 genes are implicated” in the monogenic disease. The most common form of the disease is LCA 10, caused by mutations in the CEP290 gene. 

features Stephen Russell, MD, of the Institute for Vision Research at the University of Iowa. He discussed recent data presented at this year’s annual 

Association for Research in Vision and Ophthalmology (ARVO

) meeting that support the therapeutic potential of investigational oligonucleotide sepofarsen for LCA 10.

Russel further explained the causes behind LCA and LCA 10, the quality of life in affected patients, current screening practices, as well as trajectories in research for this domain.

He offered insight into treatment strategies for these patients and reflected on the future of care for those living with LCA 10 and other mutations.

Listen to The Rare Disease Report on your favorite podcast platforms, including 

Stephen Russell, MD, talks about promising new data that has the potential to provide a breakthrough for treating the most common mutation of the rare genetic eye disease.

The Rare Disease Report Podcast: Leber's Congenital Amaurosis 

, diet may play an increasingly important role as a therapy.

A new study "Diet to Induce Remission in Crohn's Disease," or DINE-CD, compared the Specific Carbohydrate DIet and the Mediterranean Diet in terms of efficacy and safety in patients. 

As it turned out, both diets met symptomatic remission in nearly half of patients, in 43.5% of patients following a Mediterranean style diet and 46.5% of patients following the Specific Carbohydrate Diet.

In this episode of the DocTalk podcast, lead investigator of the DINE-CD, James Lewis, MD, Chief of Gastroenterology at Penn Presbyterian Medical Center, discussed the reasons behind the diets chosen and what they might ultimately mean for care for patients with IBD. 

He noted that the Mediterranean Diet was associated with lower risk of Crohn's Disease in studies that look at pre-illness diet, as well as improved symptoms and quality of life in IBD patients.

"It has also been associated with many other health benefits, particularly in reduction in cardiovascular disease and some malignancies," Lewis said. "Those could be viewed as an added benefit to the Mediterranean Diet, because even if it didn't help their Crohn's Disease, many people would get health benefits following it." 

He also touched on how he describes the treatment to patients, as an adjunct to traditional IBD therapies that may have benefits for symptoms of Crohn's Disease. 

"In a small amount of people, it may be able to suffice as a sole therapy for your Crohn's Disease, but you need to go into it eyes open, willing to change course if it is not meeting the goals of therapy," Lewis said.

Both the Mediterranean Diet and the Specific Carbohydrate diet achieved nearly 50% symptomatic remission in patients with IBD. 

James Lewis, MD: Diet as Treatment in Patients with Crohn's Disease

There remains a need to develop new treatments for pediatric patients with 

attention deficit/hyperactivity disorder (ADHD)

, particularly in extended release formulation.

One such option might be viloxazine (Qelbree), which gained US Food and Drug Administration approval in April for pediatric ADHD patients between 6-17 years old.

In a double-blind study that included 297 patients 12-17 years of age who were diagnosed with ADHD, researchers found patients treated with viloxazine 400 mg treatment reached statistical significance compared to placebo in the primary endpoint, change from baseline in ADHD-RS-5 total score. Participants receiving the study drug had a -18.3 LS mean change from baseline (

 = .0082) vs. LS mean change of -13.2 from baseline for those receiving placebo.

In this episode of the DocTalk Podcast, Ann C. Childress, MD, Center for Psychiatry and Behavioral Medicine, explained the promising results of viloxazine and why it could be a game changer for pediatric ADHD patients.

Childress said the extended release formulation should help reduce the social stigma for students taking medication during the school day.

However, when looking at the state of ADHD care there are some reasons to remain concerned. Childress said there is still a need to develop new treatments, particularly for pediatric patients, and there is reason to believe this patient group may have been particularly impacted by the circumstances of the COVID-19 pandemic.

Viloxazine was approved in April by the FDA for pediatric ADHD patients between 6-17 years old. 

Ann C. Childress, MD: The Promise of Viloxazine for Pediatric ADHD

 Dr. Kevin Walton and e-cigarette research


E-cigarette devices are among the most divisive products introduced in the United States this century.

Their potential—fluctuating between a safe alternative to combustible cigarettes, and an altogether more harmful product than cigarettes, depending on who is asked—is not fully met a decade-plus into availability in a country where smoking-induced 

As many experts weigh their possible value as a nicotine delivery alternative, and others denounce their availability, regulation, and marketing toward younger users, some are are still seeking a refined e-cigarette product: safer, satisfactory, and better understood by scientists than what’s currently available.

This month’s episode of Lungcast, features Kevin Walton, PhD, Chief of the Clinical Research Grants Branch at the NIH’s National Institute on Drug Abuse (NIDA).

Walton joins Al Rizzo, MD, Chief Medical Officer of the American Lung Association (ALA), for a discussion on the currently understood science of e-cigarettes—and what questions remain in research.

Prior to their discussion, Rizzo highlighted Dr. Alan H. Hart, and his contribution to science, literature, and LGBTQ+ advocacy and awareness, in alignment with Pride Month.