Personalized medicine, long in the works, is one step closer to bringing treatments tailored to each person.  At the 2010 BIO International Convention, the “Delivery & Manufacturing of Biologics and Drugs” track highlighted cell therapy and genetic medicine, presenting new procedures that bring the medical world one step closer to more accurate treatments.

Since the completion of the Human Genome Project in 2003, scientists have hoped to use genetic information to create medicine doctors could use according to what would be proven to work best foreach patient.

According to Edward Abrahams, executive director of the Personalized Medicine Coalition, many medications have been shown to not work efficiently in everyone. He said medications to prevent heart failure, known as beta blockers, are ineffective for 15 to 25 percent of people who take them. Statins, a type of cholesterol drug, do not work for 30 to 70 percent and some asthma medications do not work for 40 to 70 percent of those who take them.

With personalized medicine, individuals can be tested before they are prescribed a medication to see not only which kind would work best, but at what dose as well.

Abrahams called current medical practice “trial and error medicine.” He said it is hard to predict how each person will react to specific treatments and medicines, so adjustments often need to be made. Cell therapy, he said, can eliminate much of that guesswork.

“We need to find the right treatment for the right person at the right time,” Abrahams said.

Cell therapy involves introducing healthy cells into the tissue of an individual to treat a disease.

While there are several types of cell therapy, the most commonly known is stem cell therapy. Stem cells in bone marrow are currently used to treat leukemia. Current research is looking into their use for treatment in diseases like HIV, Alzheimer’s disease and spinal cord injuries.

Ronnda Bartel, vice president of technical operations for Aastrom Biosciences Inc., said the company is currently working on tissue repair cell, or TRC, technology, which increases the number of stem cells from bone marrow collected from the patient.

This procedure could be used to repair tissue in a patient who has cardiovascular diseases that have led to ischemia, or tissue damage due to a lack of oxygen and nutrients.

During the first part of the procedure, 50 milliliters of bone marrow containing stem cells is extracted from the patient’s hip, Bartel explained.  The stem cells are sent via overnight shipping to the laboratory in Ann Arbor,  Mich.  Because bone marrow is stable at room temperature, there are no special requirements for shipping, she said. The bone marrow extraction can take place in an outpatient clinic, which does not require overnight stay and can be done by a trained physician.

When the cells arrive, they are cultured for 12 days and then sent back to the patient’s hospital. Many different types of cells are added, Bartel said, such as macrophages, which are white blood cells from the immune system that digest bacteria and viruses.

“[We supply] the three legs of a stool you need to regenerate tissue,” Bartel said.

The biggest risk is contamination from other samples, she said. To avoid contamination, a “cell cassette” is used to keep the patient’s cell in a closed system and feed the cells into an automatic incubator. Automating as much of the process as possible is a key approach to making the system work.

“It’s basically plug and play,” Bartel said.

Two weeks after the bone marrow was extracted, the newly cultured cells are reinserted into the patient’s bone marrow through the hip  with a syringe  and promote tissue regeneration and the healing of tissue,

Bartel said. Currently, the technology is in the second of three clinical phases all new treatments and medications are required to go through before the FDA will approve sale to the public.

Edward Field, executive vice president and chief business officer for Aldagen Inc., said Aldagen is working on the development of a similar procedure, using a type of bone marrow cells called ALDHbr, which regulate gene production.

Field explained that 150 millilters of bone marrow is drawn from the hip at an outpatient clinic. The cells are purified, shipped and sent back to the center  within 48 hours of being received.

According to Field, the treatment is currently beginning Phase 2 trials and so far 40 percent of patients have no signs of ischemia after three months.

“This is the promise of regenerative medicine,” he said.

Dr. Raj Puri, director at the Division of Cellular and Gene Therapies at the Center for Biologics Evaluation & Research of the Food and Drug Administration, explained that there are two different cell sources for cell therapy: autologous cells, which come from the patient they will be used in, and allogenic cells, which are from donors.

While allogenic cells are useful in patients  who have few of their own healthy cells, Puri explained that they have some limitations.

Examples of the current limits include the capacity to manufacture such cells, as well as the limited shelf life of the cells. There is also limited scientific knowledge on how to characterize some cells, he said.

“There is an inherent variability because every patient’s cells are different,” Puri said. “We need to minimize mis-differentiated cells.”

Bartel said autologous cell therapy is beneficial because there are no ethical issues in using the patient’s own tissues and cells.

The biggest attraction to this type of therapy comes from being able to eliminate the need for suppression of the immune system. A person receiving donor cells will need to take medication to suppress their body’s immune system from rejecting these cells.

Abrahams said continuing research in cell therapy provides multiple benefits, such as safer, more effective drugs, faster cure times and more cost-effective health care. It can also reduce the potential for failed treatments, he added.

“We can increase patient compliance,” he said. “Patients don’t like to take drugs that don’t work.”

Dr. Linh Hoang, director of personalized medicine at Life Technologies, said that technology is only part of the equation. Economics, cost, ethical concerns, regulations and privacy all need to be considered, she said.

According to Hoang, the innovations will lead to more acceptance and drive cost down.

“The flood of genomics is just beginning,” she said. “You can’t treat what you don’t understand and can’t differentiate.”

Al Gore, former vice president and one of the keynote speakers at the conference, said health care quality will continue to improve and that stem cells hold

many promises.

He said that within five years, scientists are predicting that individual genomes will be able to be sequenced in 15 minutes and for less than $100.

“Personalized medicine is so exciting,” Gore said. “We confuse the unprecedented with the improbable. What we are facing is completely unprecedented.”

Personalized medicine, long in the works, is one step closer to bringing treatments tailored to each person.  At the 2010 BIO International Convention, the “Delivery & Manufacturing of Biologics and Drugs” track highlighted cell therapy and genetic medicine, presenting new procedures that bring the medical world one step closer to more accurate treatments.

Since the completion of the Human Genome Project in 2003, scientists have hoped to use genetic information to create medicine doctors could use according to what would be proven to work best for

each patient.

According to Edward Abrahams, executive director of the Personalized Medicine Coalition, many medications have been shown to not work efficiently in everyone. He said medications to prevent heart failure, known as beta blockers, are ineffective for 15 to 25 percent of people who take them. Statins, a type of cholesterol drug, do not work for 30 to 70 percent and some asthma medications do not work for 40 to 70 percent of those who take them.

With personalized medicine, individuals can be tested before they are prescribed a medication to see not only which kind would work best, but at what dose as well.

Abrahams called current medical practice “trial and error medicine.” He said it is hard to predict how each person will react to specific treatments and medicines, so adjustments often need to be made. Cell therapy, he said, can eliminate much of

that guesswork.

“We need to find the right treatment for the right person at the right time,”

Abrahams said.

Cell therapy involves introducing healthy cells into the tissue of an individual to treat a disease.

While there are several types of cell therapy, the most commonly known is stem cell therapy. Stem cells in bone marrow are currently used to treat leukemia. Current research is looking into their use for treatment in diseases like HIV, Alzheimer’s disease and spinal

cord injuries.

Ronnda Bartel, vice president of technical operations for Aastrom Biosciences Inc., said the company is currently working on tissue repair cell, or TRC, technology, which increases the number of stem cells from bone marrow collected from

the patient.

This procedure could be used to repair tissue in a patient who has cardiovascular diseases that have led to ischemia, or tissue damage due to a lack of oxygen

and nutrients.

During the first part of the procedure, 50 milliliters of bone marrow containing stem cells is extracted from the patient’s hip,

Bartel explained.  The stem cells are sent via

overnight shipping to the laboratory in Ann Arbor,  Mich.  Because bone marrow is stable at room temperature, there are no special requirements for shipping, she said. The bone marrow extraction can take place in an outpatient clinic, which does not require overnight stay and can be done by a

trained physician.

When the cells arrive, they are cultured for 12 days and then sent back to the patient’s hospital. Many different types of cells are added, Bartel said, such as macrophages, which are white blood cells from the immune system that digest bacteria and viruses.

“[We supply] the three legs of a stool you need to regenerate tissue,” Bartel said.

The biggest risk is contamination from other samples, she said. To avoid contamination, a “cell cassette” is used to keep the patient’s cell in a closed system and feed the cells into an automatic incubator.  Automating as much of the process as possible is a key approach to making the system work.

“It’s basically plug and play,” Bartel said.

Two weeks after the bone marrow was extracted, the newly cultured cells are reinserted into the patient’s bone marrow through the hip  with a syringe  and promote tissue regeneration and the healing of tissue, Bartel said. Currently, the technology is in the second of three clinical phases all new treatments and medications are required to go through before the FDA will approve sale to

the public.

Edward Field, executive vice president and chief business officer for Aldagen Inc., said Aldagen is working on the development of a similar procedure, using a type of bone marrow cells called ALDHbr, which regulate gene production.

wField explained that 150 milliliters of bone marrow is drawn from the hip at an outpatient clinic. The cells are purified, shipped and sent back to the center within 48 hours of being received. According to Field, the treatment is currently beginning Phase II trials and so far 40 percent of patients have no signs of ischemia after three months.

“This is the promise of regenerative medicine,” he said.

Dr. Raj Puri, director at the Division of Cellular and Gene Therapies at Center for Biologics Evaluation & Research for the Food and Drug Administration, explained that there are two different cell sources for cell therapy: autologous cells, which come from the patient they will be used in, and allogenic cells, which are from donors.

While allogenic cells are useful in patients who have few of their own healthy cells, Puri explained that they have some limitations. Examples of the current limits include the capacity to manufacture such cells, as well as the limited shelf life of the cells. There is also limited scientific knowledge on how to characterize some cells, he said.

“There is an inherent variability because every patient’s cells are different,” Puri said. “We need to minimize mis-differentiated cells.”

Bartel said autologous cell therapy is beneficial because there are no ethical issues in using the patient’s own tissues and cells. The biggest attraction to this type of therapy comes from being able to eliminate the need for suppression of the immune system. A person receiving donor cells will need to take medication to suppress their body’s immune system from rejecting these cells.

Abrahams said continuing research in cell therapy provides multiple benefits, such as safer, more effective drugs, faster cure times, more cost-effective health care and reducing the potential for failed treatments.

“We can increase patient compliance,” he said. “Patients don’t like to take drugs that don’t work.”

Dr. Linh Hoang, director of personalized medicine at Life Technologies, said that technology is only part of the equation. Economics, cost, ethical concerns, regulations and privacy all need to be considered, she said. According to Hoang, the innovations will lead to more acceptance and drive cost down.

‘The flood of genomics is just beginning,” she said. “You can’t treat what you don’t understand and can’t differentiate.”

Al Gore, former vice president and one of the keynote speakers at the conference, said health care quality will continue to improve and that stem cells hold many promises. He said that within five years, scientists are predicting that individual genomes will be able to be sequenced for less than $100 and in 15 minutes.

“Personalized medicine is so exciting,” Gore said. “We confuse the unprecedented with the improbable. What we are facing is completely unprecedented.”