Chris Lorson

#IAmScience: Leah Lepore

April 23, 2022 By Balslew, Karly (MU-Student) in #IAmScience Tags: biochemistry

By Karly Balslew | Bond LSC

Exploring new places and diving into the world of the unknown can be intimidating. At a young age, Leah Lepore was immersed in this world and grew to love it.

“The first time I left the country was to travel to Japan and it was an incredible culture shock,” said the current Chris Lorson lab member. “From my twelve-year-old perspective, I was learning about a different way of life but also drove my parents nuts because I would only eat McDonald’s while we were there.”

An avid traveler, Lepore loves immersing herself in new cultures and seeing the world from a different point of view. This has blended into other areas of life, especially when it comes to her passion for science.

“I think traveling is what formed this kind of wanderlust that I have, and that exploring unknown places gets you out of your day-to-day routine and puts things into perspective,” she said. “There’s so much in the world than what you see around you.”

Lepore is a sophomore majoring in biochemistry. She wasn’t new to working in a lab when she joined Bond LSC in summer of 2021. She worked at two other labs in high school, so she found it imperative to join a lab that would challenge and prepare her for the future. As a member of the Lorson lab and an employee of Shift Pharmaceuticals — Lorson’s business off-shoot of his research — she found that.

“It’s really interesting to see the industry science and the academic science be complimentary,” Lepore said.

At Shift Pharmaceutical, she works on Charcot-Marie-Tooth disease (CMT). CMT causes abnormalities in the nerves feet, legs, hands, and arms. This disease was unfamiliar to her before she joined, but that did not hinder her enthusiasm to learn more.

“What’s really interesting is that it doesn’t necessarily kill you, but it diminishes the quality of life because it is caused by this overexpression of protein,” she said. “It causes your limbs to contract but not able to relax while also impacting your balance and dexterity.”

Lepore uses gene silencing therapy with synthetic nucleotides that bind to mRNA in this research. mRNA codes for proteins and since there is an abundance of certain proteins in patients with CMT, she is trying to silence portions of mRNA that code for them.

Lepore aims to spread her desire to be involved in research as an ambassador for the Office of Undergraduate Research, and she encourages students to get involved as soon as possible. Many students are apprehensive to dive headfirst into working at a lab, but Lepore reassures that there is a place for everyone at Bond LSC.

“I find that everyone that I’ve met here is extremely intelligent, but also great at making anyone who hasn’t worked in a lab before feel extremely comfortable,” she said. “It’s a very welcoming and inviting atmosphere.”

When not in the lab or helping undergraduates, she focuses on fitness. At Orange Theory, Lepore lifts weights and runs on the treadmill, which helps her cope with stress and anxiety. No matter how hectic life might get, her positive attitude and ambitious personality shine through.

“Sometimes you have to realize that you’re not going to know everything even if you want to, but there are always going to be people that are willing to explain it to you,” she said. “However, not knowing everything is okay sometimes because it gives you room to learn and engage these critical skills that are not only going to help you in the lab but also in your day-to-day life.”

#IAmScience: Juliette Baker

March 11, 2022 By Balslew, Karly (MU-Student) in #IAmScience Tags: gene therapy

By Karly Balslew | Bond LSC

Science and art may feel like completely separate departments but for research scientist Juliette Baker, this couldn’t be further than the truth.

Her mom is a graphic designer and fueled her passion for art while Baker’s own love for science fused the two worlds together. She even drew a diagram for her master’s thesis on Amyotrophic Lateral Sclerosis (ALS).

“Honestly, people don’t think science and art overlap a ton,” Baker said. “But, I’m making tons of schematics for people to understand, and it’s all my artistic brain, really.”

At one point she considered becoming a science illustrator but views painting and art to be more of a de-stressor and hobby rather than a career path. When it comes to science, Baker wants to be more hands-on and focus on direct patient applicable research. However, her path wasn’t picture clear at the start of her academic career.

Baker — who now works at Bond LSC in the lab of Chris Lorson — earned a bachelor’s degree in health science at Mizzou, and planned on going to med school. She worked six months as a medical assistant but realized shortly after that this career path wasn’t for her.

“I love science but what I loved was the research part,” Baker said. “So, I did my masters in a year and a half instead of two years to get back on track.”

After she received her master’s degree in biomedical science last year, an opportunity arose to work at Bond LSC, and she couldn’t refuse.

Baker explained that she had to find three people to comprise a committee for her master’s program. This committee would approve and decide if she would receive her degree. Baker added a fourth person, Chris Lorson, since her research project for her master’s thesis was a collaboration with him and her mentor, Nicole Nichols.

“He [Lorson] emailed me the day I got approved, I think it was before I even found out that I got approved, and said ‘so are you looking for a job?’”

Lorson, a principal investigator at Bond LSC, co-founded Shift Pharmaceuticals to focus on treatment for patients with spinal muscular atrophy (SMA), an inherited motor neuron disease. He was looking for a full-time researcher and Baker checked every box in the job description.

“They were looking for a full-time researcher and it was basically doing exactly what I learned to do my entire master’s so he thought it would be perfect if I could transition to working in his lab,” Baker said.

Baker primarily works for SHIFT but also helps with ongoing projects and aids other students in the Lorson lab.

“It was perfect because I was super worried about having to apply to jobs and figure out what I’m now qualified to do,” Baker said.

Currently, her research is on gene therapy treatments for patients with Charcot-Marie-Tooth disease (CMT). This inherited nerve disease causes abnormalities in the nerves in feet, hands, legs, and arms. She also helps with the SMA projects and SMARD1 project, a rare type of spinal muscular atrophy.

“Honestly this lab helped me realize that [gene therapy] is the kind of specialty that I want to stay in,” Baker said.

However, there is a stigma surrounding gene therapy and people are apprehensive when it comes to this type of treatment. One common misconception is that scientists are altering DNA or changing body composition, which Baker explains isn’t the case.

“What we are trying to do is to modify the production of the gene so it will go back to normal levels,” Baker said. “So, it’s not making the gene any different or adding a new gene that wasn’t there before, we are just modifying how your body is producing [the gene].”

Baker hopes the stigma surrounding gene therapy decreases over time as more people use it for different diseases, and it’ll become more accessible. The more attention this type of therapy receives, the cheaper it will become for people in the future.

“That’s the biggest thing that I hate about my job is that, yes, this treatment is going to be amazing and lifesaving, but it’s going to be expensive,” she said.

She plans on going back to school and getting her Ph.D. and one day working in the pharmaceutical industry. But for now, she enjoys working with Lorson and other undergraduates in the lab.

“The most rewarding part is definitely getting to see how my work and everything I do every day directly affects people with the diseases,” Baker said.

Bond LSC duo lands national fellowship by working to advance diversity in science

September 10, 2021 By Lauren Hines in #IAmScience Tags: diversity, HHMI, SACNAS

By Lauren Hines | Bond LSC

Sara Ricardez Hernandez starts her day in the Chris Lorson lab with a vibrant demeanor while wearing her jet-black lab coat. Within five minutes, the graduate student is already at her microscope.

Ricardez Hernandez’s eagerness has only enhanced since she and principal investigator Chris Lorson won the Howard Hughes Medical Institute (HHMI) Gilliam Fellowship on July 22. HHMI created the Gilliam Fellowship and its three-year funding to award and advance diverse and inclusive environments in science.

For Ricardez Hernandez, fostering a healthy, diverse lab environment hits home. Being from Mexico and president of the Society for Advancement of Chicanos/Hispanics and Native Americans in Science (SACNAS) chapter at MU, she knows minority students are often left out of the research career conversation and need opportunities to connect to research.

“I went to [University of Missouri-St. Louis], and I was the only Latina in my whole class,” Ricardez Hernandez said. “I also didn’t really have any mentors that looked like me. I was very interested in showing other people from the same background that they can also be scientists.”

Ricardez Hernandez found out about the fellowship in the middle of an online conference.

“I was super excited,” Ricardez Hernandez said. “I was like, ‘Oh my god!’ And then I went home, and I celebrated it with my fiancé.”

The $50,000 per year will be used to better understand disease and therapeutic developments for the infantile disease spinal muscular atrophy with respiratory distress type 1 (SMARD1). The genetic disorder occurs in children with mutations in the Immunoglobulin-μ-DNA Binding Protein 2 (Ighmbp2) gene.

The Lorson lab looks at SMARD1 in mice to better understand the disease and do preclinical testing of treatments, which fills much of Ricardez Hernandez’s day in the lab.

This involves doing anything from perfusing and dissecting mice tissue to staining mice diaphragms to see if there’s a disruption between the muscle and the muscle nerve cells. Often, Ricardez Hernandez visits the mouse room.

“That’s my favorite part of the day because I love mice,” Ricardez Hernandez said. “They’re so cute. We study an infantile disease, so we mostly use baby mice. These mice can die very prematurely, so if we treat them, they can be rescued.”

Lorson is frequently in and out of the lab helping lab members with their experiments. Lorson has been an important mentor and support system for Ricardez Hernandez even before she joined the lab in 2020.

“It was my first semester of my Ph.D., and I really wanted to go to the [SACNAS] national conference,” Ricardez Hernandez said. “We needed some funding, and [Lorson] being in the veterinary school was like, ‘Oh, I heard that you’re looking for funding. I know someone who can help you with that,’ and it was himself.”

Lorson’s mentorship may have rubbed off on Ricardez Hernandez a little since it’s now a passion in her daily life. Usually, she’ll work with undergraduates in the lab and meet with her mentees from the Maximizing Access to Research Careers/Initiative for Maximizing Student Diversity (MARC/IMSD) program after work. Then, she’ll help students as a teacher’s assistant for a microbiology class.

“She’s honestly an inspiration for any minorities seeking medicine, research, or higher education,” said Zayd Al Rawi, an undergraduate in the Lorson lab. “Although I come from a different background, being Middle Eastern, we share the commonality of being minorities in America, so I see her as a role model. She’s a true inspiration to minorities seeking their goals.”

The Gilliam Fellowship will help Ricardez Hernandez advance her research and gain her Ph.D. in molecular pathogenesis and therapeutics. Until then, Ricardez Hernandez will spend her afternoons helping students and quantifying data with her favorite show on in the background.

“Overall, [she’s] one of the best people, best researchers, and one of the best supportive mentors that I’ve met,” Al Rawi said. “I’m really glad that I’m in this lab and I’ve crossed paths with her. I know she’s going to continue doing great things in the future.”

More information on the HHMI Gilliam Fellowship can be found here, and information on the Mizzou SACNAS chapter can be found on their website.

#IAmScience Sara Ricardez Hernandez

May 7, 2021 By Becca Wolf in #IAmScience Tags: MARC/IMSD, SACNAS, spinal muscular atrophy

By Becca Wolf | Bond LSC

As an undergraduate student, Sara Ricardez Hernandez did not have mentors that exposed her to the many opportunities available for underrepresented students — like summer programs and other research initiatives — but now a graduate student and a Life Sciences fellow, Ricardez Hernandez wants to make sure that no one else is ever in that boat.

“I really like advocating for other people like myself. For example, the university that I went to for undergrad had very little mentoring for minority students, so I want to help people not only be able to get a Ph.D. but be even better scientists and have more opportunities than the ones I had,” said Ricardez Hernandez, a current member of the Chris Lorson lab at Bond Life Sciences Center.

She has not wasted any time. She is currently the vice president of Mizzou’s chapter of the Society for Advancement of Chicanos/Hispanics and Native Americans in Science (SACNAS). SACNAS is a program that provides networking and professional development opportunities for minority students.

“We try to help students by hosting workshops and inviting different diverse researchers so students see that they can succeed in academia and become principal investigators too,” Ricardez Hernandez said. “When I first arrived at Mizzou, I knew they had a chapter here and I was very excited about that. I even contacted some people before classes started and I was like, ‘Hey, I’m really interested in becoming a member’ — I had been president for the Hispanic Latino Association in undergrad — so I knew that I wanted to keep helping students.”

Ricardez Hernandez also serves as a graduate student mentor with MARC/IMSD (Maximizing Access to Research Careers/Initiative for Maximizing Student Diversity) at Mizzou. She currently mentors six undergraduate students where she provides support and advice regarding their research, academics, and any personal issues that may be barriers to their success.

“One of my biggest passions is mentoring and trying to push higher education into a more equitable future,” Ricardez Hernandez said. “Historically, higher education has lacked diversity, so we do not see a lot of role models for minority students like professors or PIs. Showing students that, ‘Hey, I’m here at Mizzou, I’m a minority as well and I’m passionate about what I’m doing,’ can demonstrate to undergrads that they can do that too.”

Ricardez Hernandez’s energy is contagious.

“Her mentees think highly of her. She is transparent and vulnerable in sharing her own previous challenges and that makes her so relatable,” Brian Booton, Undergraduate Director of MARC/IMSD at Mizzou, said. “I think we sometimes put mentors on a pedestal and think of them not struggling themselves or that they’ve never had difficulties, but I think she’s really authentic with the ways in which she’s grown. She’s able to reflect on those lessons and reframe them as social capital making her a very effective mentor.”

Ricardez Hernandez extends her passion for education in the lab and her studies as well. She is in the Molecular Pathogenesis and Therapeutics Program where she studies neuromuscular infantile diseases. She is currently working on the characterization of respiratory defects in spinal muscular atrophy with respiratory distress type 1 (SMARD1) and the applications for potential therapeutics.

“I think it keeps me going. Sometimes it’s hard being a graduate student, and not everything works out all the time or you have a lot of things going on but studying diseases like SMARD1 makes me want to come to lab every day and keep doing what I’m doing,” Ricardez Hernandez said.

With a few years left at Mizzou, Ricardez Hernandez plans on helping as many people as she can.

“She’s a real advocate and champion for her mentees,” Booton said. “She’s so approachable and genuine and she’s been a great asset to the program.”

#IAmScience Caley Smith

December 18, 2020 By Lauren Hines in #IAmScience Tags: spinal muscular atrophy

By Lauren Hines | Bond LSC

With her cheerful and friendly demeanor, graduate student Caley Smith can transform any rigid lab into a place of warmth and scientific excitement.

In 2017, Smith was searching for a lab on campus where she could explore the world of genetics.

“It was probably not until high school biology class where I got my first real lecture on genetics and genetic diseases,” Smith said. “It was more about understanding. You have this gene, and this mutation happens, and this disease happens. That I always found super fascinating.”

By the time she finished her rotation of labs on campus, she found her place in the Chris Lorson lab at Bond Life Sciences Center.

“When I was rotating there, there was a real sense of community,” Smith said.

Not only did the Lorson lab provide the place and expertise for her to study genetics, but it was also scientists like Eric Villalón, former postdoctoral fellow in the Lorson lab, who welcomed her.

“Right away she was pretty much a blank open book,” said Villalón, current postdoctoral fellow at Johns Hopkins University School of Medicine. “She was open to anything, and she was very curious to learn about the science in the lab. She was very eager to learn all the techniques that we used in the lab to do the research.”

The Lorson lab studies spinal muscular atrophy (SMA), which is a genetic disorder that occurs in children missing the survival motor neuron one gene. The disorder is a leading genetic cause of infantile death worldwide and causes muscle weakness, respiratory failure and premature death. Spinal muscular atrophy with respiratory distress type 1 (SMARD1) is a rare type of SMA caused by a different gene mutation with respiratory distress as the first symptom.

“My natural curiosity and wanting to know why gradually turned into ‘I want to help these kids,’” Smith said.

Smith focuses on characterizing mouse models for SMARD1 based on human-derived protein mutations, so then researchers can do preclinical testing of treatments to measure their effectiveness and expand their knowledge on SMARD1. Models like these didn’t exist prior to the Lorson lab.

“Since it’s very rare, rarer than SMA, there isn’t a lot of research or knowledge,” Smith said. “Rather, there are no treatment options. With SMA, there are three FDA approved treatment options and with SMARD1, there’s nothing really. It’s just palliative care measures.”

Working with the mice in the Lorson lab also allows Smith to see the effects of her work in real-time.

“By doing this animal work and treating these mice, I can directly see these mice get better,” Smith said. “It’s fulfilling in that way because it helps me see that connection of what I’m doing in a very preclinical setting to see how it could potentially benefit kids down the line.”

Over the past three years at the lab, Smith’s unsatiated curiosity has treated her well in the field. However, she has narrowed her focus to her own scientific questions.

“She has grown to think critically and independently about her science,” Villalón said. “She has grown a lot from a very young wannabe scientist to the real scientist that she is now. She has been fully independent for the past year and a half or two.”

Now, Smith is performing her own experiments and mentoring others. Maybe she’ll even help someone find their own place in the world of science.

#IAmScience Mona Kacher

October 30, 2020 By Lauren Hines in #IAmScience Tags: spinal muscular atrophy

By Lauren Hines | Bond LSC

Years ago, when career technician Mona Kacher was still in school, her science teacher asked their students who wanted to dissect a salamander first. Some students were hesitant, others excited, but no one more excited than Kacher who had already raised her hand.

Originally, Kacher was a medical technician in an Army hospital lab. However, when she left the Army, she was encouraged by her then father-in-law to apply for a senior research position at Dalton Cardiovascular Research Center. There, the lab manager asked if she knew anything about the chemistry analyzer they had just bought.

She knew it inside and out.

“So, that started an unintentional career in research, and I’ve stayed with it ever since,” Kacher said.

In her 31 years at Mizzou, Kacher has been bouncing around campus as a senior technician and research specialist working in various labs and disciplines.

“As you know, PIs sometimes lose their funding or funding comes and goes,” Kacher said. “So, I’ve had to piece together a career on campus, moving around a little bit.”

Kacher has worked in oncology, veterinary pathobiology, diabetes, hypertension, Lyme disease, HIV-AIDS and malaria research. She has been listed as a co-author of 20 scientific manuscripts that also include her data.

“Mona’s wide variety of disciplines that she has been exposed to throughout her career absolutely helps strengthen the Lorson lab,” said Chris Lorson, principal investigator at Bond Life Sciences Center. “She is able to draw on techniques she has performed in the past and apply them to various research needs.”

Kacher has been at the Chris Lorson lab at BLSC for the past two years. She’s the only technician there, so she assists others and helps teach methods and procedures to graduate and undergraduate students. Kacher also maintains five mice colonies for Lorson’s gene therapy work regarding Spinal Muscle Atrophy (SMA).

SMA is a genetic disorder that occurs in children missing the survival motor neuron-one gene. The disorder is the leading genetic cause of infantile death worldwide and causes muscle weakness, respiratory failure and premature death.

“SMA has likely affected members of my own family,” Kacher said. “It’s a very sad situation for a lot of families, and working in research that may directly improve the outcomes of the lives for some of these little sick children is very rewarding.”

Kacher is currently working on projects that test treatment methods for SMA and other neurodegenerative diseases. In these projects, infant mice the size of gummy bears receive a spinal injection with the treatment. Spinal injections are also involved in some projects.

“She is focused and hard-working,” Lorson said. “She understands the need for flexibility in the world of research and will persevere until the task is completed. She has a wonderfully positive attitude and a genuine concern for the well-being of those around her.”

Similar to how her research reaches a range of disciplines, her interests are just as varied.

Kacher and her wife started a business named, Shivers Shaved Ice and Shivers Coffee & Donuts in 2017. They owned it for several years until Kacher went back to MU.

In addition, Kacher likes to make jewelry and art. She also plays guitar and used to sing at weddings and in the Columbia Chorale. She still sings with MU’s Choral Union.

All of this creativity seeps into BLSC where she has colorful toys on her desk and a rainbow of organizers on lab shelves.

“Gosh, well, I think I’ve certainly brightened up the lab,” Kacher said.

With so many positions across campus, Kacher believes this one will be her last. She plans on retiring in a few years.

“I enjoy a lot of variety in life,” Kacher said. “So, being able to contribute data and small pieces of information in this field or that field, knowing that all the little pieces together are what makes a cure or moves scientists into the next direction for studying that disease or that condition is very rewarding.”

#IAmScience Monir Shababi

November 2, 2018 By Erica Overfelt in #IAmScience Tags: spinal muscular atrophy

By Erica Overfelt | Bond LSC

Research Assistant Professor Monir Shababi and mothers of SMARD children started out as friends on Facebook. Now, they’re friends for a lifetime.

It was an unexpected turn for someone who spends most of her professional life in a lab.

SMARD1 is an acronym for the rare genetic neuromuscular disease spinal muscular atrophy with respiratory distress, which has been the focus of Shababi’s research for the past five years. While it shares part of its name with the better known spinal muscular atrophy (SMA), the disease-causing gene is not the same. Both diseases target the lumbar motor neurons and cause muscle atrophy, diaphragmatic paralysis and respiratory complications are the first and most devastating symptom of SMARD.

“When my first paper about gene therapy for SMARD1 disease was published, a mother of the SMARD child contacted me and she informed other moms about my research,” Shababi said. “I never thought it would happen this way, you never imagine there would be a personal impact with your publications.”

Shababi ran into the obstacle of funding. Her once Facebook friends were there to help Shababi face this drawback.

“Within the second year of my National Institute of Health funding for SMARD1 gene therapy, I discussed the research with the mothers of SMARD kids, Jill Sims and Lisa Porter,” Shababi said. “I told them about the limited funding options in the future. Sims and Porter family were extremely supportive and generous. Dr. Sims and his wife (Jill’s in-laws) donated a large sum of money to Mizzou Give Direct, MU’s crowdfunding website exclusively for my SMARD research, establishing a webpage for donations with the goal of curing SMARD. Their gift changed the course of many lives in the most meaningful way.”

Shababi met the Sims daughter Catherine a few weeks ago. Catherine is five years old and lives with the disease.

“It had the most amazing personal impact on my work, which I was looking all along in my professional life,” Shababi said. “This is not just a job, it is not just research, it is not about publishing papers; it is affecting real lives.”

Shababi — who works with Dr. Chris Lorson at Bond LSC — began her life career in science at the University of Tabriz in Iran studying plant pathology. She moved to America with her husband, who accepted a research job in MU. She then got her masters and Ph.D. through the university, finishing her Ph.D. in Dr. James Schoelz lab studying gene regulation in a plant virus. She then had two postdoctoral positions working on gene regulation in yeast and bacteria.

“My postdoc research on yeast and bacteria was not satisfying enough; I wasn’t feeling the impact on the society that I was looking for,” Shababi said. “I even thought about changing careers until I contacted Dr. Lorson. His research involved hereditary genetic disease in humans and using virus as viral vectors to deliver the missing gene. I thought this research will surely have an impact on human lives. In Lorson’s lab, I found the enthusiasm and passion which led to four funded grants, 18 publications, a book chapter and most importantly, a financial gift to MU.”

Even if Shababi doesn’t get the expected results, every day in the lab is a new victory for her. She has developed a new mouse model for SMARD1 and injects them with the therapeutic virus at different times with different dosages to determine the therapeutic window and proper dosing for the vector. Shababi previously tested only black mice but now tests new white mice born with SMARD.

“As opposed to black mice, these white SMARD mice do not have the genetic modifiers to survive for a long period of time,” Shababi said. “They usually die by day 20, so we administer the gene replacement therapy at different days post birth or different dose and analyze the survival and overall health. Even a few days elongated survival is a huge improvement and you can tell if that time point or dose was still efficient or not.”

To move the research further toward clinical trials, the lab aims to use a more advanced model such as a human primate.

“We are hoping to advance from the mice research to an animal closer to humans,” Shababi said.

With hopeful new advancements in the future, there is only a positive outlook for the future of treatment.

“The pain and suffering of SMARD children and their parents are real and heartbreaking,” Shababi said. “This research builds a foundation for the future clinical trials creating optimism and hope for the SMARD community.”

#IAmScience Toni Morcos

October 12, 2018 By Erica Overfelt in #IAmScience Tags: spinal muscular atrophy

By Erica Overfelt | Bond LSC

Science and spaghetti are an unlikely combination.

For Toni Morcos, this combination led her to research.

“Growing up, my dad researched muscular dystrophy and my mom was an OB-GYN,” Morcos said. “Every night, my mom would talk about her surgeries that day then my dad would talk about this gene targeting thing. I would be eight years old, just eating spaghetti wondering what the heck they are talking about. And now I’m here studying biology just to understand and be a part of that dinner table conversation.”

Morcos is more than just understanding what the heck they were talking about. Working in Chris Lorson’s lab at Bond LSC, she studies spinal muscular atrophy (SMA). SMA is a genetic disease that most often affects the nervous system of children, causing muscle movement to deteriorate and leads to death.

“Most of what I do in the lab is work with the mice; we study them by injecting them with these different quantities of treatments and chemicals,” Morcos said. “They are naturally sick with SMA. I weigh them all and track their weights and track their outcomes to see how they do.”

This research means creating new treatments to help those affected by SMA to save more lives at a sustainable price.

“There is one treatment that is on the market and they have a monopoly,” Morcos said. “The last figure I saw for it was around $500,000 annually per patient, and no one can afford that. We can’t even, as college students, fathom that much money. It is really important to get another treatment out to drive down costs so more lives can be saved.”

Morcos’ family dinners may not have been like most families, but those talks over spaghetti caused her to advocate for others and science.

She plans on going to medical school where she wants to help those in the health care system. In that light, she studies Arabic alongside biology and currently volunteers through refugee and immigration services. As an in-home tutor she teaches English to refugees and helps them become more culturally educated on American systems. And one of the most difficult of those systems to navigate is the health care system.

“There are translators, but sometimes they’re incorrect,” Morcos said. “I don’t understand everything the translator says in Arabic, but a translator once told a kid to take Tylenol when the doctor said ibuprofen or Advil. That kind of difference is small, but when I can catch something like that you don’t know how often that’s happening.”

This pursuit of new knowledge and desire to understand drives Morcos in life.

“If you stop asking questions or stop looking for answers, as a society, you start to go backwards,” she said. “A problem we have now is people not wanting to know the answers. It’s beyond the lab, its beyond research, people have stopped asking the hard questions and get stuck in their ways. Change isn’t a bad thing, it’s progress. That’s true in science, research and culture.”

A shift in focus: Lorson moves basic research to drug development

July 25, 2018 By Erica Overfelt in Research Tags: #MizzouResearch, #Shift

By Roger Meissen & Erica Overfelt | Bond LSC

It takes a lot to move a discovery from lab bench to an application that can provide therapeutic benefits to those suffering from disease.

Bond LSC’s Chris Lorson is making moves to bridge that gap with the start of Shift Pharmaceuticals. With its formation in March 2017, Lorson adds co-founder and Chief Science Officer of the company to his list of titles that include Bond LSC investigator, professor of veterinary pathobiology and associate dean for research and graduate studies.

Shift Pharmaceuticals builds off of years of progress the Lorson Lab has made in understanding spinal muscular atrophy (SMA), which is the leading genetic cause of infant deaths. The disease causes neurons to die, leading to muscle failure, including those that affect walking, arm movement, and respiratory function. While SMA is technically a rare disease, it is remarkably common, affecting nearly 1/10,000 births.

“It’s a devastating disease for patients and families; while the primary defect in is nerves, this leads to problems in muscles, bones, and other vital systems,” Lorson said. “Historically, the majority of kids who develop SMA do not survive beyond 3-5 years.”

Lorson knew his research had potential for drug development, and MU’s Office of Technology Management & Industry Relations (OTMIR) pursued patent protection for the technology. This process helps safeguard the innovations resulting from the research and allows MU to better attract commercial interest to develop and market medical treatments originating from the technology. But it took a partnership with co-founder Steve O’Connor to get the ball rolling. O’Connor is MU’s Entrepreneur in Residence and has significant experience in starting drug development businesses and now serves as CEO of Shift.

“I never knew how to start a company,” Lorson said. “I would argue most academics don’t – this isn’t part of our traditional training. I sat around not knowing what to do, realizing I had this thing that could do a lot, but the practical steps of setting up a biotech start-up were beyond me.”

“Steve thought this technology sounded really cool, so in late March he submitted paperwork and by the end of March we were a company. Shift’s first grant was submitted 3 days later.”

The goal of Shift Pharmaceuticals is to move their lead compound into the clinic for SMA.

“When you look at the disease, it’s not just one cell type and not just one clinical type of patient,” Lorson said. “It really is a disease that is complex and the idea is to bring more options to the fight.”

With the start of any new business, money is always a necessity. Funding from the advocacy group CURE SMA provided the initial funding for the discovery of this compound, while several other foundations including FightSMA, the Gwendolyn Strong Foundation, and Muscular Dystrophy Association have further contributed to the pre-clinical development. MU’s OTMIR negotiated an option agreement with Shift, giving them the exclusive rights to the technology, which helped them obtain a recent $2.73 million grant from the Department of Defense Congressionally Directed Medical Research Program (CDMRP). This will move the company toward the first phase of investigating a new drug.

The root of SMA

Lorson has spent most of his scientific career chasing the underlying causes of SMA.

That search focused in on a few key genes in those suffering from the disease. Two genes — named survival motor neuron-1 and -2 (SMN1 and SMN2, respectively) — are central to SMA development. In patients, SMN1 is mutated and doesn’t process enough of a key protein (SMN) that helps neurons function. While SMN2 acts as a backup gene for this function, a miniscule change in the SMN2 gene causes it to make less SMN protein than required by the body.

In 2016, the Lorson Lab at Bond LSC produced a compound that increases the lifespan of SMA mice . They targeted the back-up gene, SMN2, to produce more functional protein and discovered an increase of protein causing a significant lifespan extension in treated mice. This discovery showed promise for creating a cure for those with SMA.

Shift Pharmaceuticals will be working on developing a drug that builds off Lorson’s work and targets all forms of SMA.

Shift’s first two employees, Mizzou alumnus Paul Morcos and UMKC alumnae Diane Beatty, will help move toward that goal. With Morcos in research and development and Beatty negotiating regulatory affairs, the company hopes to move the drug toward FDA approval.

Thanks to the recent Department of Defense grant, the next step looks to testing in larger animals and at things Lorson did not consider before.

“The experiments we will be doing are not academic in nature, rather, they are focused on the singular goal of preparing our lead compound for an FDA submission. From a traditional academic lab perspective, these might sound rather boring,” Lorson said. “All of these things are not things you do in an academic setting, but that is exactly the point. This is drug development, not the quest for another paper.”

Within the Business Incubator, MU will provide space for the company as well for their research. This sort of partnership is just another part that may one day help translate vital basic research into future treatment.

“Almost everybody has the possibility of doing something that is translational, it is just envisioning it in a different way,” Lorson said.